image:Alliance for Cancer Gene Therapy (ACGT) Scientific Advisory Council Chair Michael T. Lotze, MD, of the University of Pittsburgh, will be honored with the 2021 Society for Immunotherapy of Cancer (SITC) Lifetime Achievement Award at the SITC 36th Annual Meeting, Nov. 10-14, in Washington D.C. view more

Credit: University of Pittsburgh

Alliance for Cancer Gene Therapy (ACGT) Scientific Advisory Council Chair Michael T. Lotze, MD, of the University of Pittsburgh, will be honored with the 2021 Society for Immunotherapy of Cancer (SITC) Lifetime Achievement Award at the SITC 36th Annual Meeting, Nov. 10-14, in Washington D.C.

Dr. Lotze is currently Chief Cellular Therapy Officer of Nurix Therapeuticsand professor of surgery, immunology and bioengineering at the University of Pittsburgh School of Medicine. He is widely regarded as the leader in exploring cancer as a disorder of cell death and is devising novel strategies to approach the disease in this context. He initiated the first approved gene therapy protocols at the National Institutes of Health and has treated more than 100 patients on gene therapy protocols at the University of Pittsburgh. He is the co-inventor of 10 patents in dendritic cell vaccines and antigen discovery, and author of more than 500 scientific papers and chapters in basic and applied tumor immunology and cytokine biology.

Dr. Lotze leads the 14 members of the ACGT Scientific Advisory Council in rigorously reviewing and monitoring the research selected for funding by ACGT. The importance and value that the Council contributes to the ACGT funding process distinguishes ACGT from many other funding agencies. Council members are among the most accomplished thought-leaders in the field of cancer cell and gene therapy. They are experienced scientists whose decades of research and patient care have elevated them into important leadership positions at top institutions across the U.S. and in Canada.

Society for Immunotherapy of Cancer is a member-driven organization dedicated to improving cancer patient outcomes by advancing the science and application of cancer immunotherapy through educational programs that foster scientific exchange and collaboration. Learn more about SITC at http://www.sitcancer.org.

Alliance for Cancer Gene Therapy

For more than 20 years, Alliance for Cancer Gene Therapy (ACGT) has funded research that is bringing innovative treatment options to people living with deadly cancers treatments that save lives and offer new hope to all cancer patients. The organization funds researchers who are pioneering the potential of cancer cell and gene therapy talented visionaries whose scientific advancements are driving the development of groundbreaking treatments for ovarian, prostate, sarcoma, glioblastoma, melanoma and pancreatic cancers. 100% of all public funds raised directly support research and programs. For more information, visitacgtfoundation.org, call (203) 358-5055,or join the Alliance for Cancer Gene Therapy community onFacebook,Twitter,LinkedIn, Instagram andYouTube @acgtfoundation.

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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ACGT Scientific Advisory Council Chair Michael T. Lotze, MD, honored with Lifetime Achievement Award from Society for Immunotherapy of Cancer -...

Recommendation and review posted by Bethany Smith

PALO ALTO, Calif., Nov. 3, 2021 /PRNewswire/ --BridgeBioPharma, Inc. (Nasdaq: BBIO),a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the first patient has been dosed in CANaspire, its Phase 1/2 clinical trial of BBP-812, an investigational adeno-associated virus (AAV) 9 gene therapy for the treatment of Canavan disease. Canavan disease is an ultra-rare and fatal disease that affects approximately 1,000 children in the United States and European Union. There are currently no approved therapies for the condition.

"Dosing the first patient in our Canavan disease trial is a significant achievement for our gene therapy team and we would not be here without the support of our collaborative partners in the patient, medical and scientific communities," said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy. "We are committed to the Canavan community and will work to advance this trial and the other programs in our gene therapy portfolio because we believe deeply in the potential of gene therapy to improve and save lives."

"Based on the efficacy and safety data we've observed in our preclinical studies, we are hopeful that our investigational gene therapy can become a meaningful treatment option for children living with Canavan. Right now, these children only have access to supportive care because there are currently no approved therapies to treat this devastating disease," added Adam Shaywitz, M.D., Ph.D., chief medical officer at BridgeBio Gene Therapy.

The Phase 1/2 open-label study is designed to evaluate the safety, tolerability, and pharmacodynamic activity of the company's AAV9 gene therapy, BBP-812, in pediatric patients with Canavan disease. In the initial dose-finding phase of the study, each patient will receive a single intravenous (IV) infusion of BBP-812. The primary outcomes of the study are safety, as well as change from baseline of urine and central nervous system N-acetylaspartate (NAA) levels. Motor function and development will also be assessed. Preclinical proof-of-concept data have shown the approach restores survival and normal motor function.

"Gene therapy is designed to treat diseases at their source, which for Canavan disease would be an extremely beneficial treatment option. Through this trial, we hope to provide evidence that this investigational therapy could represent a promising treatment option for Canavan patients and their families. We are grateful to the first family for participating," said Florian Eichler, M.D., director of the Leukodystrophy Service and principal investigator at Massachusetts General Hospital, the first clinical site open in the CANaspire trial.

BridgeBio's gene therapy was originally developed by Guangping Gao, Ph.D., and Dominic J. Gessler, M.D., Ph.D., at the University of Massachusetts Medical School. Dr. Gao, a pioneer in AAV gene therapy, was also the first person to clone the ASPA gene, which in its mutated form causes Canavan disease. Dr. Gao has been working on developing a cure for Canavan disease for more than 25 years.

"Throughout my career, I've been passionate about connecting with families in the Canavan community to understand how to help them through their journey. After decades of research and work, I'm thrilled that we have dosed the first patient in this trial. I'm hopeful that this represents a meaningful turning point for the Canavan community," said Dr. Gao, co-director of the Li Weibo Institute for Rare Diseases Research, director of the Horae Gene Therapy Center and Viral Vector Core, and professor at University of Massachusetts Medical School.

BridgeBio's investigational AAV9 gene therapy for Canavan disease is one of the Company's 14 programs that are in the clinic or commercial setting for patients living with genetic diseases and genetically-driven cancers. An initial Phase 1/2 data readout for Canavan disease is expected in 2022. For more information about the CANaspire trial, visit TreatCanavan.com or ClinicalTrials.gov (NCT04998396).

About BBP-812BBP-812 is an investigational AAV9 gene therapy for Canavan disease. Using AAV gene therapy, BridgeBio seeks to deliver functional copies of the ASPA gene throughout the body and into the brain, potentially correcting the disease at its source. Preclinical proof-of-concept results in Canavan disease models have shown the approach restores survival and normal motor function. BBP-812 was granted Fast Track Designation, Rare Pediatric Drug Designation, and Orphan Drug Designation by the U.S. Food and Drug Administration. BBP-812 was also granted Orphan Drug Designation by the European Medicines Agency.

About Canavan DiseaseAffecting approximately 1,000 children in the United States and European Union, Canavan disease is an ultra-rare, disabling and fatal disease with no approved therapy. Most children are not able to meet developmental milestones, are unable to crawl, walk, sit or talk, and pass away at a young age. The disease is caused by an inherited mutation of the ASPA gene, which codes for aspartoacylase, a protein that breaks down a compound called N-acetyl-L-aspartic acid (NAA). Deficiency of aspartoacylase activity results in accumulation of NAA, and ultimately results in toxicity to myelin in ways that are not well understood. Myelin insulates the nerves, and without it, neurons are unable to send and receive messages as they should. The current standard of care is limited to supportive therapy.

About BridgeBio Pharma, Inc.BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio's pipeline of over 30 development programs ranges from early science to advanced clinical trials, and its commercial organization is focused on delivering the company's first two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers, and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.comand follow us onLinkedInandTwitter.

BridgeBio Pharma, Inc. Forward-Looking StatementsThis press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the "Securities Act"), and Section 21E of the Securities Exchange Act of 1934, as amended (the "Exchange Act"), which are usually identified by the use of words such as "anticipates," "believes," "estimates," "expects," "intends," "may," "plans," "projects," "seeks," "should," "will," and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to the timing and success of BridgeBio's Phase 1/2 clinical trial of BBP-812 for the treatment of Canavan disease, expectations, plans and prospects regarding BridgeBio's regulatory approval process for BBP-812, the ability of BBP-812 to treat Canavan disease in humans, and the timing and success of initial top-line Phase 1/2 date of BBP-812, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, BridgeBio's ability to continue and complete its Phase 1/2 clinical trial of BBP-812 for the treatment of Canavan disease, past data from preclinical studies not being indicative of future data from clinical trials, BridgeBio's ability to advance BBP-812 in clinical development according to its plans, the ability of BBP-812 to treat Canavan disease, the ability of BBP-812 to retain Fast Track Designation, Rare Pediatric Drug Designation, and Orphan Drug Designation from the U.S. Food and Drug Administration and Orphan Drug Designation from the European Medicines Agency, and potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and clinical trials, supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; as well as those set forth in the Risk Factors section of BridgeBio's most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent SEC filings, which are available on the SEC's website atwww.sec.gov.Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

BridgeBio Media Contact: Grace Rauh[emailprotected](917) 232-5478

BridgeBio Investor Contact: Katherine Yau[emailprotected](516) 554-5989

SOURCE BridgeBio

https://bridgebio.com/

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Recommendation and review posted by Bethany Smith

Updates from OTL-201 Clinical Proof-of-Concept Study in MPS-IIIA and OTL-204 Preclinical Study for GRN-FTD at ESGCT Showcase Potential for HSC Gene Therapy in Multiple Neurodegenerative Disorders

Launch Activities for Libmeldy Across Key European Countries, including Reimbursement Discussions, Progressing in Anticipation of Treating Commercial Patients

Frank Thomas, President and Chief Operating Officer, to Step Down Following Transition in 2022; Search for a Chief Financial Officer Initiated

Cash and Investments of Approximately $254M Provide Runway into First Half 2023

BOSTONandLONDON, Nov. 04, 2021 (GLOBE NEWSWIRE) --Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today reported financial results for the quarter ended September 30, 2021, as well as recent business updates and upcoming milestones.

This quarter, we are pleased by the progress demonstrated by our investigational neurometabolic HSC gene therapy programs with promising preclinical and clinical updates at ESGCT, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With follow-up in OTL-201 for MPS-IIIA patients now ranging between 6 and 12 months, biomarker data remain highly encouraging, showing supraphysiological enzyme activity and corresponding substrate reductions in the CSF and urine. The launch strategy for Libmeldy is also advancing in Europe with momentum building on reimbursement discussions and patient finding activities.

Recent Presentations and Business Updates

Data presentations at ESGCT

Clinical and pre-clinical data from across the companys investigational hematopoietic stem cell (HSC) gene therapy portfolio were featured in two oral and seven poster presentations at the European Society of Gene & Cell Therapy Congress (ESGCT) on October 19-22. Highlights from key presentations are summarized below:

R&D Investor Event Summary

In September, Orchard hosted an R&D investor event highlighting its discovery and research engine in HSC gene therapy, including an update on the OTL-104 program in development for NOD2 Crohns disease (NOD2-CD) and potential new applications in HSC-generated antigen-specific regulatory T-cells (Tregs) and HSC-vectorization of monoclonal antibodies (mAbs).

The discussion also covered the differentiated profile of Orchards HSC gene therapy approach, which has exhibited favorable safety, long-term durability and broad treatment applicability.

Libmeldy (atidarsagene autotemcel) launch in Europe

Orchard is providing an update on the following key launch activities for Libmeldy in Europe:

Executive organizational update

The company also announced that Frank Thomas will step down from his role as president and chief operating officer, following a transition in 2022. A search for a chief financial officer is underway. Mr. Thomas other responsibilities will be assumed by existing members of the leadership team in commercial and corporate affairs. Orchard recently strengthened the executive team with the appointments of Nicoletta Loggia as chief technical officer and Fulvio Mavilio as chief scientific officer and the promotion of Leslie Meltzer to chief medical officer.

I want to extend my gratitude to Frank Thomas for his immense contributions to Orchard, said Gaspar. During his tenure, Frank oversaw the transition of the organization to a publicly traded company and has managed operations with a focus on cross-company innovation, including his role as a key architect in creating and executing the focused business plan we rolled out in 2020. Along with the entire board of directors and leadership team, I appreciate Franks commitment to facilitate a smooth transition during this time.

Gaspar continued, Our search is focused on a CFO to lead the broad strategic planning efforts necessary to capitalize on the full potential of our hematopoietic stem cell gene therapy platform.We have a strong team in place to aid Orchards success in this next phase of growth and are well capitalized through the anticipated completion of several value-creating milestones.

Upcoming Milestones

In June 2021, Orchard announced several portfolio updates following recent regulatory interactions for the companys investigational programs in metachromatic leukodystrophy (MLD), Mucopolysaccharidosis type I Hurler syndrome (MPS-IH) and Wiskott-Aldrich syndrome (WAS).

Third Quarter 2021 Financial Results

Revenue from product sales of Strimvelis were $0.7 million for the third quarter of 2021 compared to $2.0 million in the same period in 2020, and cost of product sales were $0.2 million for the third quarter of 2021 compared to $0.7 million in the same period in 2020. Collaboration revenue was $0.5 million for the third quarter of 2021, resulting from the collaboration with Pharming Group N.V. entered into in July 2021. This revenue represents expected reimbursements for preclinical studies and a portion of the $17.5 million upfront consideration received by Orchard under the collaboration, which will be amortized over the expected duration of the agreement.

Research and development (R&D) expenses were $20.8 million for the third quarter of 2021, compared to $14.7 million in the same period in 2020. The increase was primarily due to higher manufacturing and process development costs for the companys neurometabolic programs and lower R&D tax credits as compared to the same period in 2020. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and development milestone payments under the companys agreements with third parties, and personnel costs to support these activities.

Selling, general and administrative (SG&A) expenses were $13.0 million for the third quarter of 2021, compared to $13.0 million in the same period in 2020. SG&A expenses are expected to increase in future periods as the company builds out its commercial infrastructure globally to support additional product launches following regulatory approvals.

Net loss was $36.4 million for the third quarter of 2021, compared to $20.3 million in the same period in 2020. The increase in net loss as compared to the prior year was primarily due to higher R&D expenses as well as the impact of foreign currency transaction gains and losses. The company had approximately 125.5 million ordinary shares outstanding as of September 30, 2021.

Cash, cash equivalents and investments as of September 30, 2021, were $254.1 million compared to $191.9 million as of December 31, 2020. The increase was primarily driven by net proceeds of $143.6 million from the February 2021 private placement and $17.5 million in upfront payments from the July 2021 collaboration with Pharming Group N.V., offset by cash used for operating activities and capital expenditures. The company expects that its cash, cash equivalents and investments as of September 30, 2021 will support its currently anticipated operating expenses and capital expenditure requirements into the first half of 2023. This cash runway excludes an additional $67 million that could become available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers granted by the FDA following future U.S. approvals.

About Libmeldy / OTL-200

Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.

Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

About Orchard

At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S. headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us onTwitterandLinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (TwitterandLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, the therapeutic potential of Libmeldy (OTL-200) and Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchards financial condition and cash runway into the first half of 2023. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2021, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaBenjamin NavonDirector, Corporate Communications+1 857-248-9454Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent Business Updates - BioSpace

Recommendation and review posted by Bethany Smith

NEW YORK and BASEL, Switzerland, Nov. 4, 2021 /PRNewswire/ --Aruvant Sciences ("Aruvant"), a private company focused on developing gene therapies for rare diseases, announced that an abstract demonstrating the clinical benefit of the company's lead product candidate ARU-1801 has been published online and will be the subject of a poster presentation at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting will take place in Atlanta, Georgia fromDecember 11 to 14, 2021.Punam Malik, M.D., Director of the Cincinnati Comprehensive Sickle Cell Center and Program Leader of the Hematology and Gene Therapy Program at the Cincinnati Children's Hospital Medical Center, will present the data at6:00 to 8:00 PM ESTonDecember 13, 2021.

"Clinical data from our ongoing MOMENTUM study has shown 100 percent resolution of vaso-oclusive events (VOE) in our recently treated SCD patients at 18 and 12 months of follow up," said Will Chou, MD, Aruvant chief executive officer. "In addition, we are excited to be sharing additional clinical data at ASH that demonstrates how the unique anti-sickling potency of ARU-1801 translates to these robust clinical outcomes."

Dr. Malik will present data from theongoing MOMENTUM study, an open label Phase 1/2 clinical trial examining ARU-1801 as a one-time potentially curative gene therapy for individuals with sickle cell disease (SCD). The MOMENTUM study examines ARU-1801, an autologous lentiviral cell therapy with a modified, highly potent gamma globin payload, in individuals with severe SCD. Unlike investigational gene therapies that require fully myeloablative conditioning, ARU-1801 is given with reduced intensity conditioning (RIC), which is a lower dose chemotherapy. ARU-1801 is designed to address the limitations of current curative allogeneic transplant options, such as low donor availability, the risk of graft-versus-host disease and toxicity from myeloablative chemotherapy. The data to be presented at ASH highlights clinically meaningfulreduction in participants' VOEs and the unique attributes that contribute to the potency of ARU-1801.

"The emerging clinical data shows that ARU-1801 holds promise for achieving durable responses in patients with severe SCD using only reduced intensity conditioninga key differentiator from other investigational gene therapy regimens," said Dr. Malik. "Given the advantage for patients, providers and payers a reduced conditioning regimen offers, ARU-1801 has the potential to be an important option for SCD patients seeking gene therapy, including those in low resource settings."

Abstract and Poster Presentation Information

Title: Safety and Efficacy of ARU-1801 in Patients with Sickle Cell Disease: Early Results from the Phase 1/2 MOMENTUM Study of a Modified Gamma Globin Gene Therapy and Reduced Intensity Conditioning

Publication Number: 3970Session Name: 801. Gene Therapies: Poster IIIDate:Monday, December 13, 2021Presentation Time:6:00 to 8:00 PM ESTLocation: Georgia World Congress Center, Hall B5 or online through the ASHwebsite.

About Aruvant SciencesAruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talentedteamwith extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an activeresearchprogram with a lead product candidate, ARU-1801, in development for individuals suffering fromSCD. ARU-1801, an investigational lentiviral gene therapy, is being studied in aPhase 1/2 clinical trial,the MOMENTUM study, as a one-time potentially curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of reduced intensity chemotherapy. The company's second product candidate, ARU-2801, is in development to cure hypophosphatasia, a devastating, ultra-orphan disorder that affects multiple organ systems and leads to high morbidity and mortality when not treated. Data from pre-clinical studies with ARU-2801 shows durable improvement in disease biomarkers and increased survival. For more information on the ongoing ARU-1801 clinical study, please visit http://www.momentumtrials.com,and for more on the company, pleasevisitwww.aruvant.com. Follow Aruvant on Facebook, Twitter @AruvantSciencesand on Instagram @Aruvant_Sciences.

SOURCE Aruvant Sciences

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Aruvant Announces ARU-1801 Data to be Presented at the 63rd American Society of Hematology (ASH) Annual Meeting - PRNewswire

Recommendation and review posted by Bethany Smith

BRISBANE, Calif.--(BUSINESS WIRE)-- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today reported third quarter financial results and provided business and clinical highlights.

We are delighted to share clinical data and business updates across several programs demonstrating that Sangamo has three important assets progressing toward late-stage development. Our gene therapy portfolio is advancing with accumulating safety and efficacy data in our Fabry and hemophilia A programs, and preliminary proof-of-concept data demonstrate the clinical potential of our zinc finger genome engineering technology in sickle cell disease. These data readouts show the progression of our first-generation genomic medicine pipeline and potentially pave the way for new treatments. Our next generation programs focus on genome regulation and allogeneic CAR-Treg cell therapy, where we have a robust preclinical pipeline in neurological and autoimmune diseases. We are energized by this momentum and look forward to continued execution of our corporate strategy, said Sandy Macrae, Chief Executive Officer of Sangamo.

Recent Clinical and Business Highlights

Fabry Disease First four patients dosed exhibited above normal -Gal A activity; Phase 3 planning initiated

Sickle Cell Disease Preliminary-proof-of-concept data will be presented at ASH as clinical program advances

Hemophilia A Four patients at highest dose experienced mean FVIII activity of 30.9% at week 104

Renal Transplant First patient enrolled, expect two patients to be dosed by mid-2022

Research, Manufacturing, and Corporate Updates

Third Quarter 2021 Financial Results

Consolidated net loss attributable to Sangamo for the third quarter ended September 30, 2021 was $47.7 million, or $0.33 per share, compared to a net loss attributable to Sangamo of $1.6 million, or $0.01 per share, for the same period in 2020.

Revenues

Revenues for the third quarter ended September 30, 2021, were $28.6 million, compared to $57.8 million for the same period in 2020, a decrease of $29.2 million.

The reduction in revenue was primarily due to a $39.3 million decrease related to our giroctocogene fitelparvovec and C9ORF72 collaboration agreements with Pfizer, resulting from the completion of our activities in 2020, and a $2.3 million decrease related to our collaboration agreement with Sanofi. These decreases were partially offset by higher revenues of $11.5 million and $1.3 million related to our collaboration agreements with Novartis and Biogen, respectively.

GAAP and Non-GAAP operating expenses

2021

2020

2021

2020

$

62.5

$

45.3

$

179.0

$

128.3

14.5

16.2

47.1

50.2

77.0

61.5

226.1

178.5

(7.9

)

(6.7

)

(24.9

)

(19.1

)

$

69.1

$

54.8

$

201.2

$

159.4

Total operating expenses on a GAAP basis for the third quarter ended September 30, 2021 were $77.0 million compared to $61.5 million for the same period in 2020. Non-GAAP operating expenses, which exclude stock-based compensation expense, for the third quarter ended September 30, 2021 were $69.1 million compared to $54.8 million for the same period in 2020.

The increase in total operating expenses on a GAAP basis was primarily driven by our higher clinical and manufacturing supply expenses along with our increased headcount to support the advancement of our clinical trials and our ongoing collaborations.

Cash, cash equivalents and marketable securities

Cash, cash equivalents and marketable securities as of September 30, 2021 were $519.0 million compared to $692.0 million as of December 31, 2020.

Revised Financial Guidance for 2021

We are revising our full-year operating expense guidance initially provided on February 24, 2021 and reiterated most recently on August 5, 2021 as follows:

$285 to $305

$300 to $310

$255 to $275*

$265 to $275**

Conference Call

Sangamo will host a conference call today, November 4, 2021, at 9:15 a.m. Eastern Time, which will be open to the public. The call and live Q&A will be webcast.

The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 5178059. Participants may access the live webcast via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. Call replay will be available for one week following the conference call. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 5178059.

About Sangamo Therapeutics

Sangamo Therapeutics is a clinical-stage biopharmaceutical company with a robust genomic medicines pipeline. Using ground-breaking science, including our proprietary zinc finger genome engineering technology and manufacturing expertise, Sangamo aims to create new genomic medicines for patients suffering from diseases for which existing treatment options are inadequate or currently dont exist. For more information about Sangamo, visit http://www.sangamo.com.

Forward-Looking Statements

This press release contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to the therapeutic and commercial potential of our product candidates, the anticipated plans and timelines of Sangamo and our collaborators for screening, enrolling and dosing patients in and conducting our ongoing and potential future clinical trials and presenting clinical data from our clinical trials, the anticipated advancement of our product candidates to late-stage development including potential future Phase 3 trials, anticipated implementation of a protocol amendment for the Phase 3 AFFINE clinical trial of giroctocogene fitelparvovec and the resumption of the dosing of additional patients in the trial; our revised 2021 financial guidance related to GAAP and non-GAAP total operating expenses and stock-based compensation; our continued execution of our corporate strategy; the anticipated completion of our in-house cell therapy manufacturing facility in Valbonne, France; and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to the effects of the evolving COVID-19 pandemic and the impacts of the pandemic on the global business environment, healthcare systems and business and operations of Sangamo and our collaborators, including the initiation and operation of clinical trials; the research and development process, including the enrollment, operation and results of clinical trials and the presentation of clinical data; the uncertain timing and unpredictable nature of clinical trials and clinical trial results, including the risk that any protocol amendment for the Phase 3 AFFINE trial of giroctocogene fitelparvovec may not be accepted by the relevant review bodies in a timely manner, or at all, or that the FDA may not lift its clinical hold on the Phase 3 AFFINE trial in a timely manner, or at all, each of which could further delay or preclude further patient dosing in the trial as well as the risks that therapeutic effects observed in clinical trial results will not be durable in patients and that final clinical trial data will not validate the safety and efficacy of our product candidates; reliance on results of early clinical trials, which results are not necessarily predictive of future clinical trial results; our limited experience manufacturing biopharmaceutical products, including the risks that we may be unable to maintain compliant manufacturing facilities, build additional facilities and manufacture our product candidates as intended; and our ability to achieve expected future financial performance.

There can be no assurance that we and our collaborators will be able to develop commercially viable products. Actual results may differ materially from those projected in these forward-looking statements due to the risks and uncertainties described above and other risks and uncertainties that exist in the operations and business environments of Sangamo and our collaborators. These risks and uncertainties are described more fully in our Securities and Exchange Commission filings and reports, including in our Annual Report on Form 10-K for the year ended December 31, 2020 as supplemented by our Quarterly Report on Form 10-Q for the quarter ended September 30, 2021. Forward-looking statements contained in this announcement are made as of this date, and we undertake no duty to update such information except as required under applicable law.

Non-GAAP Financial Measure

To supplement our financial results and guidance presented in accordance with GAAP, we present non-GAAP total operating expenses, which exclude stock-based compensation expense from GAAP total operating expenses. We believe that this non-GAAP financial measure, when considered together with our financial information prepared in accordance with GAAP, can enhance investors and analysts ability to meaningfully compare our results from period to period and to our forward-looking guidance, and to identify operating trends in our business. We have excluded stock-based compensation expense because it is a non-cash expense that may vary significantly from period to period as a result of changes not directly or immediately related to the operational performance for the periods presented. This non-GAAP financial measure is in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. We encourage investors to carefully consider our results under GAAP, as well as our supplemental non-GAAP financial information, to more fully understand our business.

2021

2020

Read more:
Sangamo Therapeutics Reports Recent Business and Clinical Highlights and Third Quarter 2021 Financial Results - BioSpace

Recommendation and review posted by Bethany Smith

SAN DIEGO, Nov. 02, 2021 (GLOBE NEWSWIRE) -- Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced the establishment of its Cell Therapy Scientific Advisory Board (SAB). The Cell Therapy SAB is comprised of industry and academic leaders in the cell therapy field, covering important areas of expertise including cutting edge research, preclinical development, manufacturing, and clinical development.

The Cell Therapy SAB will play an important role in advising and guiding the companys efforts to develop safe and effective cell therapies targeting receptor-tyrosine kinase-like Orphan Receptor 1 (ROR1), leveraging our deep expertise on ROR1 and the single chain variable fragment (scFv) of our ROR1 antibodies, including cirmtuzumab. ROR1 is highly expressed by many solid tumors as well as hematological malignancies and confers both an aggressive phenotype and survival advantage to the tumor cells. Cirmtuzumab binding to ROR1 on leukemia and lymphoma cells decreases tumor cell proliferation and survival by blocking Wnt5a-induced activation, while it does not bind to normal adult tissues. Cirmtuzumab has also demonstrated encouraging safety and efficacy results in its ongoing Phase 1/2 study in combination with ibrutinib for the treatment of patients with mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL) as well as in a Phase 1b study in combination with paclitaxel for the treatment of patients with Her2-negative breast cancer.

We are pleased to welcome our newly appointed scientific advisors, whose deep expertise in cell therapy research and development will help us bring safe and effective ROR1 targeted cell therapies to patients faster, said James Breitmeyer, M.D., Ph.D., Oncternals President and CEO. We believe that ROR1 is an ideal target for next generation cell therapies due to its proven role in tumor progression and its wide expression in many cancer types with significant unmet needs.

The members of the Oncternal Cell Therapy SAB include:

Michael Wang, MD, Endowed Professor in the department of Lymphoma & Myeloma at MD AndersonDr. Wang has published more than 200 peer-reviewed papers and has presented his work at meetings nationally and internationally. He is the Director of the Mantle Cell Lymphoma (MCL) Program of Excellence and Co-Director of the B-Cell Lymphoma Moon Shot Program at the University of Texas MD Anderson Cancer Center. The Wang Laboratory at MD Anderson research program aims to elucidate the mechanisms underlying therapeutic resistance in B-cell lymphoma and to translate these findings to the clinic to improve patient outcomes. Dr. Wang obtained his M.D. from Shandong Medical University and M.S. from Beijing University Medical School, and completed his clinical training as a resident at Norwalk Hospital,Norwalk, Conn., and as a Fellow in Oncology and in Hematology at MD Anderson.

Angela Shen, MD, MBA, Clinical and Translational Market Sector Leader Mass General BrighamDr. Shen has unique, deep knowledge of the cell and gene therapy landscape having provided clinical, regulatory, and strategic leadership for autologous and allogeneic CAR-T cell therapies, NK cell therapies, and other novel cell therapy programs across industry. Dr. Shen currently hold a position at Mass General Brigham (formerly known as Partners HealthCare), an affiliate of Harvard Medical School and serves a part-time CMO at Walking Fish Therapeutics, Inc. Previously, she held Chief Medical Officer (CMO) positions at multiple biotech companies, including Arcellx, NKarta, Arvinas, and acting CMO of Tizona. Dr. Shen led the clinical team at Novartis responsible for designing and launching the industrys first multi-site, registration CAR-T cell therapy trial supporting the approval of Kymriah (CTL019, CART-19). She received a BS through Rensselaers accelerated biomedical program, and holds an MD from Albany Medical College in New York and MBA from New York University Stern School of Business.

Marcela V. Maus, MD, PhD, Associate Professor, Medicine, Harvard Medical School, Director of Cellular Immunotherapy, Cancer Center, Massachusetts General HospitalDr. Maus is a translational physician-scientist in the field of cancer immunology. Her laboratory focuses on the design, generation, and use of innovative forms of immune cell engineering, including chimeric antigen receptors and investigates basic mechanisms of human immunology to design and test novel immune-based therapeutic interventions in vitro, in mouse models, and in patients. Dr. Maus received her S.B. from the Massachusetts Institute of Technology, and her M.D. and Ph.D. degrees from the University of Pennsylvania. Dr. Maus trained in internal medicine at University of Pennsylvania and in hematology and medical oncology at Memorial Sloan Kettering, and is board-certified in these three disciplines. Her laboratory research training was focused on gene and cell therapies, and occurred in the laboratories of Dr. Katherine High, Dr. Michel Sadelain, and Dr. Carl June.

Sadik Kassim, PhD, Chief Technology Officer at Vor BiopharmaDr. Kassim is a cell and gene therapy bioprocessing and translational research expert. Dr. Kassim served as Executive Director at Kite Pharma where he led the development of manufacturing processes for autologous CAR-T and TCR-based cell therapies. He and his team at Kite led the BLA and MAA filing efforts for Kites X-19 product, which is a CD19 CAR-T therapy for Mantle Cell Lymphoma. Before Kite, Dr. Kassim served as Chief Scientific Officer at Mustang Bio, where he was the first employee and oversaw the foundational build-out of the companys preclinical and manufacturing activities. Earlier in his career, Dr. Kassim was Head of Early Analytical Development for Novartis Cell and Gene Therapies Unit, where he and his team contributed to the BLA and MAA filings for Kymriah. Dr. Kassim earned his BS in cell and molecular biology from Tulane University and received his PhD in microbiology and immunology from Louisiana State University. After receiving his PhD, he was a research fellow in the lab of Dr. James Wilson at the University of Pennsylvanias Gene Therapy Program.

About Oncternal Therapeutics

Oncternal Therapeutics is a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies for the treatment of cancers with critical unmet medical need. Oncternal focuses drug development on promising yet untapped biological pathways implicated in cancer generation or progression. The clinical pipeline includes cirmtuzumab, an investigational monoclonal antibody designed to inhibit the ROR1 pathway, a type I tyrosine kinase-like orphan receptor, that is being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of patients with mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL) and in an investigator-sponsored, Phase 1b clinical trial in combination with paclitaxel for the treatment of women with HER2-negative metastatic or locally advanced, unresectable breast cancer, as well as a Phase 2 clinical trial of cirmtuzumab in combination with venetoclax, a Bcl-2 inhibitor, in patients with relapsed/refractory CLL. Oncternal is also developing a chimeric antigen receptor T cell (CAR-T) therapy that targets ROR1, which is currently in preclinical development as a potential treatment for hematologic cancers and solid tumors. The clinical pipeline also includes TK216, an investigational targeted small-molecule inhibitor of the ETS family of oncoproteins, that is being evaluated in a Phase 1/2 clinical trial for patients with Ewing sarcoma alone and in combination with vincristine chemotherapy. More information is available at https://oncternal.com.

Contact Information:

InvestorsRichard VincentChief Financial Officer858-434-1113rvincent@oncternal.com

MediaCorey DavisLifeSci Advisors212-915-2577cdavis@lifesciadvisors.com

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Oncternal Therapeutics Announces Formation of Cell Therapy - GlobeNewswire

Recommendation and review posted by Bethany Smith

Many people develop Alzheimers or other forms of dementia as they get older. However, others remain sharp well into old age, even if their brains show underlying signs of neurodegeneration.

Among these cognitively resilient people, researchers have identified education level and amount of time spent on intellectually stimulating activities as factors that help prevent dementia. A new study by MIT researchers shows that this kind of enrichment appears to activate a gene family called MEF2, which controls a genetic program in the brain that promotes resistance to cognitive decline.

The researchers observed this link between MEF2 and cognitive resilience in both humans and mice. The findings suggest that enhancing the activity of MEF2 or its targets might protect against age-related dementia.

Its increasingly understood that there are resilience factors that can protect the function of the brain, says Li-Huei Tsai, director of MITs Picower Institute for Learning and Memory. Understanding this resilience mechanism could be helpful when we think about therapeutic interventions or prevention of cognitive decline and neurodegeneration-associated dementia.

Tsai is the senior author of the study, which appears today in Science Translational Medicine. The lead authors are recent MIT PhD recipient Scarlett Barker and MIT postdoctoral fellow and Boston Childrens Hospital physician Ravikiran (Ravi) Raju.

Protective effects

A large body of research suggests that environmental stimulation offers some protection against the effects of neurodegeneration. Studies have linked education level, type of job, number of languages spoken, and amount of time spent on activities such as reading and doing crossword puzzles to higher degrees of cognitive resilience.

The MIT team set out to try to figure how these environmental factors affect the brain at the neuronal level. They looked at human datasets and mouse models in parallel, and both tracks converged on MEF2 as a critical player.

MEF2 is a transcription factor that was originally identified as a factor important for cardiac muscle development, but later was discovered to play a role in neuron function and neurodevelopment. In two human datasets comprising slightly more than 1,000 people all together, the MIT team found that cognitive resilience was highly correlated with expression of MEF2 and many of the genes that it regulates.

Many of those genes encode ion channels, which control a neurons excitability, or how easily it fires an electrical impulse. The researchers also found, from a single-cell RNA-sequencing study of human brain cells, that MEF2 appears to be most active in a subpopulation of excitatory neurons in the prefrontal cortex of resilient individuals.

To study cognitive resilience in mice, the researchers compared mice who were raised in cages with no toys, and mice placed in a more stimulating environment with a running wheel and toys that were swapped out every few days. As they found in the human study, MEF2 was more active in the brains of the mice exposed to the enriched environment. These mice also performed better in learning and memory tasks.

When the researchers knocked out the gene for MEF2 in the frontal cortex, this blocked the mices ability to benefit from being raised in the enriched environment, and their neurons became abnormally excitable.

This was particularly exciting as it suggested that MEF2 plays a role in determining overall cognitive potential in response to variables in the environment, Raju says.

The researchers then explored whether MEF2 could reverse some of the symptoms of cognitive impairment in a mouse model that expresses a version of the tau protein that can form tangles in the brain and is linked with dementia. If these mice were engineered to overexpress MEF2 at a young age, they did not show the usual cognitive impairments produced by the tau protein later in life. In these mice, neurons overexpressing MEF2 were less excitable.

A lot of human studies and mouse model studies of neurodegeneration have shown that the neurons become hyperexcitable in early stages of disease progression, Raju says. When we overexpressed MEF2 in a mouse model of neurodegeneration, we saw that it was able to prevent this hyperexcitability, which might explain why they performed cognitively better than control mice.

Enhancing resilience

The findings suggest that enhancing MEF2 activity could help to protect against dementia; however, because MEF2 also affects other types of cells and cellular processes, more study is needed to make sure that activating it wouldnt have adverse side effects, the researchers say.

The MIT team now hopes to further investigate how MEF2 becomes activated by exposure to an enriching environment. They also plan to examine some of the effects of the other genes that MEF2 controls, beyond the ion channels they explored in this study. Such studies could help to reveal additional targets for drug treatments.

You could potentially imagine a more targeted therapy by identifying a subset or a class of effectors that is critically important for inducing resilience and neuroprotection, Raju says.

The research was funded by the Glenn Center for Biology of Aging Research, the National Institute of Aging, the Cure Alzheimers Fund, and the Eunice Kennedy Shriver National Institute of Child Health and Human Development.

Follow this link:
Study links gene to cognitive resilience in the elderly - MIT News

Recommendation and review posted by Bethany Smith

MIT researchers have discovered a gene linked to cognitive resilience in the elderly. Environmental enrichment, they find, appears to activate the MEF2 protein, which controls a genetic program in the brain that promotes resilience to declines related to Alzheimers and age-related dementia. Credit: MIT News, iStockphoto

The findings may help explain why some people who lead enriching lives are less prone to Alzheimers and age-related dementia.

Many people develop Alzheimers or other forms of dementia as they get older. However, others remain sharp well into old age, even if their brains show underlying signs of neurodegeneration.

Among these cognitively resilient people, researchers have identified education level and amount of time spent on intellectually stimulating activities as factors that help prevent dementia. A new study by MIT researchers shows that this kind of enrichment appears to activate a gene family called MEF2, which controls a genetic program in the brain that promotes resistance to cognitive decline.

The researchers observed this link between MEF2 and cognitive resilience in both humans and mice. The findings suggest that enhancing the activity of MEF2 or its targets might protect against age-related dementia.

Its increasingly understood that there are resilience factors that can protect the function of the brain, says Li-Huei Tsai, director of MITs Picower Institute for Learning and Memory. Understanding this resilience mechanism could be helpful when we think about therapeutic interventions or prevention of cognitive decline and neurodegeneration-associated dementia.

Tsai is the senior author of the study, which was published on November 3, 2021, in Science Translational Medicine. The lead authors are recent MIT PhD recipient Scarlett Barker and MIT postdoctoral fellow and Boston Childrens Hospital physician Ravikiran (Ravi) Raju.

A large body of research suggests that environmental stimulation offers some protection against the effects of neurodegeneration. Studies have linked education level, type of job, number of languages spoken, and amount of time spent on activities such as reading and doing crossword puzzles to higher degrees of cognitive resilience.

The MIT team set out to try to figure how these environmental factors affect the brain at the neuronal level. They looked at human datasets and mouse models in parallel, and both tracks converged on MEF2 as a critical player.

MEF2 is a transcription factor that was originally identified as a factor important for cardiac muscle development, but later was discovered to play a role in neuron function and neurodevelopment. In two human datasets comprising slightly more than 1,000 people all together, the MIT team found that cognitive resilience was highly correlated with expression of MEF2 and many of the genes that it regulates.

Many of those genes encode ion channels, which control a neurons excitability, or how easily it fires an electrical impulse. The researchers also found, from a single-cell RNA-sequencing study of human brain cells, that MEF2 appears to be most active in a subpopulation of excitatory neurons in the prefrontal cortex of resilient individuals.

To study cognitive resilience in mice, the researchers compared mice who were raised in cages with no toys, and mice placed in a more stimulating environment with a running wheel and toys that were swapped out every few days. As they found in the human study, MEF2 was more active in the brains of the mice exposed to the enriched environment. These mice also performed better in learning and memory tasks.

When the researchers knocked out the gene for MEF2 in the frontal cortex, this blocked the mices ability to benefit from being raised in the enriched environment, and their neurons became abnormally excitable.

This was particularly exciting as it suggested that MEF2 plays a role in determining overall cognitive potential in response to variables in the environment, Raju says.

The researchers then explored whether MEF2 could reverse some of the symptoms of cognitive impairment in a mouse model that expresses a version of the tau protein that can form tangles in the brain and is linked with dementia. If these mice were engineered to overexpress MEF2 at a young age, they did not show the usual cognitive impairments produced by the tau protein later in life. In these mice, neurons overexpressing MEF2 were less excitable.

A lot of human studies and mouse model studies of neurodegeneration have shown that the neurons become hyperexcitable in early stages of disease progression, Raju says. When we overexpressed MEF2 in a mouse model of neurodegeneration, we saw that it was able to prevent this hyperexcitability, which might explain why they performed cognitively better than control mice.

The findings suggest that enhancing MEF2 activity could help to protect against dementia; however, because MEF2 also affects other types of cells and cellular processes, more study is needed to make sure that activating it wouldnt have adverse side effects, the researchers say.

The MIT team now hopes to further investigate how MEF2 becomes activated by exposure to an enriching environment. They also plan to examine some of the effects of the other genes that MEF2 controls, beyond the ion channels they explored in this study. Such studies could help to reveal additional targets for drug treatments.

You could potentially imagine a more targeted therapy by identifying a subset or a class of effectors that is critically important for inducing resilience and neuroprotection, Raju says.

Reference: MEF2 is a key regulator of cognitive potential and confers resilience to neurodegeneration by Scarlett J. Barker, Ravikiran M. Raju, Noah E.P. Milman, Jun Wang, Jose Davila-Velderrain, Fatima Gunter-Rahman, Cameron C. Parro, P. Lorenzo Bozzelli, Fatema Abdurrob, Karim Abdelaal, David A. Bennett, Manolis Kellis and Li-Huei Tsai, 3 November 2021, Science Translational Medicine.DOI: 10.1126/scitranslmed.abd7695

The research was funded by the Glenn Center for Biology of Aging Research, the National Institute of Aging, the Cure Alzheimers Fund, and the Eunice Kennedy Shriver National Institute of Child Health and Human Development.

Originally posted here:
MIT Researchers Discover Gene Linked to Cognitive Resilience in the Elderly - SciTechDaily

Recommendation and review posted by Bethany Smith

Vitamin D is an essential nutrient thought to reduce the risk of chronic diseases, including cancer and cardiovascular disease. Observational studies have consistently found that vitamin D deficiency increases the risk of developing these conditions and of death from any cause.

Scientists do not know how exactly vitamin D levels might influence multiple health conditions. Some have suggested it may protect against cancer by regulating cadherins, which are proteins involved in cell-cell adhesion responsible for maintaining tissue architecture and regulation of cell growth and differentiation.

Despite this, several large randomized trials have not been able to find evidence that vitamin D supplementation for vitamin D deficiency has any benefit for cancer or cardiovascular disease.

This has made researchers question whether vitamin D levels really play a part in reducing the risk of these diseases or whether healthier people have naturally higher levels of the vitamin for other reasons.

Researchers led by the University of Cambridge in the United Kingdom conducted a study to see whether inherited genetic variants predisposing people to have higher vitamin D levels play a role in overall health.

Our investigation provides intriguing new evidence that suggests raising vitamin D levels may reduce the risk of major disease and mortality, but only for people who have low vitamin D levels, says Dr. Stephen Burgess, lead author of the study.

Maintaining adequate levels of vitamin D is important, but there appears to be no benefit in supplementation beyond a threshold level, he adds.

The study appears inThe Lancet Diabetes & Endocrinology.

The researchers initially gathered data from the UK Biobank, the European Prospective Investigation into Cancer and Nutrition Cardiovascular Disease study, and 31 studies from the Vitamin D Studies Collaboration.

Altogether, they analyzed health data from 386,406 middle-aged individuals with European ancestry. Each was followed for an average of 9.5 years, underwent 25-hydroxyvitamin D (25(OH)D) measurements, and did not have cardiovascular disease at baseline.

25(OH)D is the major circulating form of vitamin D in the body. It is measured via blood test to indicate vitamin D supply in the body from sunlight and nutrition.

Over the study period, 33,546 people developed coronary heart disease, 18,166 people had a stroke, and 27, 885 people died.

To understand the role of vitamin D levels in these health outcomes, the researchers investigated the participants inherited genetic variants predisposing them to higher vitamin D levels and analyzed their 25(OH)D measurements.

Similarly to authors of other studies, they found no link between genetic predisposition to higher vitamin D levels and coronary heart disease, stroke, or death.

However, among those with vitamin D deficiency, defined as lower than 25 nanomoles per liter (nmol/l), the researchers identified a strong link between genetic predisposition to higher vitamin D levels and a lower mortality risk.

They also found a link between a genetic predisposition to 10 nmol/l higher levels of 25(OH)D and a 30% lower all-cause mortality risk. Their analyses suggested similar effects on cardiovascular and cancer mortality.

These effects, however, were only evident in individuals with extremely low vitamin D levels, or those below 40 nmol/l.

Previous [] studies assumed a linear association between genetically predicted 25(OH)D, the main marker of vitamin D status, and cardiovascular disease, despite the evidence from cohort studies of observed 25(OH)D having a nonlinear association with cardiovascular disease, Prof. Robert Scragg, head of the School of Population Health at the University of Auckland in New Zealand, who was not involved in the study, told Medical News Today.

However, the current study [] shows a nonlinear association for genetically predicted 25(OH)D [and cardiovascular disease]. [] To my knowledge, this is the first study to do this type of analysis to see if the association is nonlinear. Interestingly, the study also shows no association when you combine all participants, which is what previous [] studies reported. The key difference is the stratification by baseline 25(OH)D level.

The fact that the beneficial effect is mainly in people with 25(OH)D levels below 25 nmol/l explains why the recent clinical trials observed null results as they did not have enough participants with very low 25(OH)D levels. In the ViDA study, we only had 91, while the VITAL study has been estimated to have had only about 500 not enough to show any effect.

The researchers highlight several potential mechanisms underlying the effects of vitamin D.

For example, animal studies have shown that vitamin D could regulate cardiac function due to its action on cardiac metalloproteinases, a group of enzymes that break down proteins, and fibroblasts, which are the most common type of cell in connective tissue.

Vitamin D also plays a role in endothelial cell function. These cells form the lining of blood vessels and regulate exchanges between blood and surrounding tissues. Via its effects on endothelial cells, vitamin D modulates:

The researchers add that vitamin D status also affects gene expression related to cell division and programed cell death, which could affect the growth of neoplasms that is, excessive growth and division of cells as well as DNA repair and immunomodulation in cancer.

The authors of the study conclude that genetic evidence suggests a causal relationship between 25(OH)D concentrations and mortality in people with low vitamin D levels.

However, they note some limitations to their results. For instance, they highlight that their analysis only included middle-aged individuals with European ancestry, meaning their results may not apply to people with different skin tones. Further analysis is needed, as people with dark skin often have lower vitamin D levels.

This study has addressed an important question of whether taking vitamin D will have an impact on reducing the risk of chronic diseases, such as cardiovascular diseases and cancer, Prof. Vimal Karani, professor in nutrigenetics and nutrigenomics at the University of Reading in the U.K., who was not involved in the study, told MNT. However, the findings can be generalized only to the European populations, given that the study has focused on participants of European ancestries.

Moreover, the effect has been observed only in those who have low vitamin D concentrations, which could be partly attributed to the choice of genetic instrument. [] The genetic risk score was generated using 21 genetic variants from four different gene regions, of which two genes are involved in the synthesis of [25(OH)D], and the other two are involved in the metabolism of vitamin D, he added.

Previous studies have shown that the variation in [25(OH)D] concentrations explained by synthesis genes is lesser than the variation explained by the metabolism genes. Even though the authors had performed a sensitivity analysis to see if the results were driven by a single gene region, the study did not look at the individual impact of synthesis and metabolism genetic risk scores, respectively, on the risk of major disease and mortality, and this might partly explain the effects seen only in those who have low vitamin D levels, he explained.

However, given that nearly 40% of Europeans are vitamin D deficient, and 13% are severely deficient, this study has significant public health implications in suggesting the need for taking vitamin D supplementation to overcome the risk of chronic diseases.

Prof. Vimal Karani

This study will rekindle the debate about vitamin D and cardiovascular disease, said Prof. Scragg. I think most researchers thought the matter was resolved after the findings of null results from two clinical trials of vitamin D supplementation the VITAL study from the [United States] and the ViDA study from New Zealand particularly in the light of previous [] studies, which showed no association between genetic markers of vitamin D status and cardiovascular disease.

Given that there is only a moderate beneficial effect in a small proportion of participants in the current study, it would take a huge clinical trial to [make similar observations for] vitamin D supplementation. I dont think a further clinical trial now will ever be done, particularly since participants in a trial can always purchase their own vitamin D if they believe they have been allocated to the placebo group, and thus weaken any effect from vitamin D, he explained.

Consequently, this study is likely to provide the strongest and most definitive evidence on whether vitamin D protects against cardiovascular disease and all-cause mortality, he concluded.

See the article here:
Vitamin D and mortality risk: Is there a causal link? - Medical News Today

Recommendation and review posted by Bethany Smith

Embargoed until 7 a.m. CT / 8 a.m. ET Wednesday, Nov. 3, 2021

DALLAS, Nov. 3, 2021 The American Heart Association will present its 2021 Distinguished Scientist in Basic Cardiovascular Sciences to Northwestern Universitys Elizabeth M. McNally, M.D., Ph.D., FAHA. The Association designates Distinguished Scientist awards in several categories to members who have significantly advanced the understanding of cardiovascular, stroke or brain health. The six 2021 Distinguished Scientist awardees will be honored during the AssociationsScientific Sessions 2021, which will be fully virtual, Saturday, Nov. 13 through Monday, Nov. 15, 2021.

Dr. McNally was selected for this honor because of her multiple discoveries around the heredity of musculoskeletal and cardiovascular disorders. Her work impacts both scientific research and patient care. Additionally, Dr. McNally discovered new techniques for identifying and mapping genetic modifiers for inherited cardiovascular and myopathic disorders.

Thank you and congratulations to Dr. Elizabeth McNally, said Association President Donald M. Lloyd-Jones, M.D., Sc.M., FAHA. Her work continues to help us develop a deeper understanding of how genetic mutations exert their effects on cardiovascular disease. She leads an incredible team that is using these genetic signals to take the critically important step of developing of new therapies, particularly for inherited cardiac conditions such as cardiomyopathies.

Dr. McNally directs the Center for Genetic Medicine at Northwestern Universitys Feinberg School of Medicine in Chicago and is the Elizabeth J. Ward Professor of Genetic Medicine - a cardiologist with expertise in cardiovascular genetics. As a clinician, she developed one of the first cardiovascular genetics clinics in the nation, integrating genetic testing into cardiovascular care for patients and families.

Her research team at Northwestern discovers genetic causes of cardiac disorders and then works to define the mechanisms of how these genetic variants cause disease. By developing a deeper understanding as to how these genetic mutations exert their effects, she is using these genetic signals to drive the development of new treatments for cardiovascular disease. She has a special interest in neuromuscular genetic diseases like muscular dystrophy since these disorders often have accompanying cardiovascular complications.

Genetic treatments are becoming a reality, said Dr. McNally. As a physician scientist, its amazing to see some of these treatments beginning to make it to patients. In Duchenne Muscular Dystrophy, there are now multiple ongoing trials of gene therapy, and this will also be gene therapy that treats the heart. We also know about all the newly developing gene editing tools, and how these can be adapted to treat patients with genetic cardiovascular diseases and one day to also change genes to treat even more common forms of heart disease.

Dr. McNallys translational accomplishments have been recognized through an award from the Burroughs Wellcome Foundation and as a recipient a Distinguished Clinical Scientist Award from the Doris Duke Charitable Foundation. She serves on the Board of Directors for the Muscular Dystrophy Association and is currently the chair of the Associations Council on Basic Cardiovascular Sciences. She is a past president of the American Society for Clinical Investigation and currently president of the Association of American Physicians. Earlier this year, she was elected to the American Academy of Arts and Sciences.

Additional Resources:

The Association receives funding primarily from individuals; foundations and corporations (including pharmaceutical, device manufacturers and other companies) also make donations and fund specific Association programs and events. The Association has strict policies to prevent these relationships from influencing the science content. Revenues from pharmaceutical and biotech companies, device manufacturers and health insurance providers and the Associations overall financial information are availablehere.

The American Heart AssociationsScientific Sessions 2021is a premier global exchange of the latest scientific advancements, research and evidence-based clinical practice updates in cardiovascular science for health care professionals worldwide. The three-day meeting will feature more than 500 sessions focused on breakthrough cardiovascular basic, clinical and population science updates in a fully virtual experience Saturday, Nov. 13 through Monday, Nov. 15, 2021. Thousands of leading physicians, scientists, cardiologists, advanced practice nurses and allied health care professionals from around the world will convene virtually to participate in basic, clinical and population science presentations, discussions and curricula that can shape the future of cardiovascular science and medicine, including prevention and quality improvement. During the three-day meeting, attendees receive exclusive access to more than 4,000 original research presentations and can earn Continuing Medical Education (CME), Continuing Education (CE) or Maintenance of Certification (MOC) credits for educational sessions. Engage in Scientific Sessions 2021 on social media via#AHA21.

About the American Heart Association

The American Heart Association is a leading force for a world of longer, healthier lives. With nearly a century of lifesaving work, the Dallas-based association is dedicated to ensuring equitable health for all. We are a trustworthy source empowering people to improve their heart health, brain health and well-being. We collaborate with numerous organizations and millions of volunteers to fund innovative research, advocate for stronger public health policies, and share lifesaving resources and information. Connect with us onheart.org,Facebook,Twitteror by calling 1-800-AHA-USA1.

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Elizabeth M. McNally, M.D., Ph.D., FAHA, named the American Heart Association's 2021 Distinguished Scientist in Basic Cardiovascular Sciences -...

Recommendation and review posted by Bethany Smith

Sponsored programs at ASH 2021 - preliminary clinical data to be presented at ASH 2021 for product candidate UCART22 and preclinical data from Cellectis gene therapy product candidate TALGlobin01

Partnered programs at ASH 2021 ALPHA2 and UNIVERSAL abstracts selected for oral presentations, ALPHA abstract selected for poster presentation

Preclinical data forRAG1deficiencySevere Combined Immunodeficiency (SCID) andSTAT3 forHyperIgEsyndrome presented at ESGCT 2021

Preclinical data supporting anti-tumor activity of UCARTMESO to be presented at SITC 2021 on November 12, 2021

Qualification of facility equipment and systems completed for

Raleigh GMP manufacturing site; Paris GMP manufacturing site now operational, focusing on the production of starting and raw materials for Cellectis UCART product candidates

Donald A Bergstrom, M.D., Ph.D., appointed as a Board Observer on Cellectis Board of Directors

Cash position1 of $216 million as of September 30, 2021

NEW YORK, Nov. 04, 2021 (GLOBE NEWSWIRE) -- Cellectis S.A. (NASDAQ: CLLS EURONEXT GROWTH: ALCLS) (the Company), a gene-editing company with clinical-stage immuno-oncology programs using allogeneic chimeric antigen receptor (CAR)-T cells and gene therapy programs for genetic diseases, today announced results for the three-month and nine-month periods ending September 30, 2021.

Cellectis will hold a conference call for investors on Friday, November 5, 2021, at 8:00 AM ET / 2:00 PM CET. The call will include the Companys third quarter results, and an update on business activities.

________________________1Cash position includes cash, cash equivalents and current financial assets and restricted cash. Restricted cash was $6 million as of September 30, 2021.

The live dial-in information for the conference call is:

US & Canada only: +1 877-407-3104

International: +1 201-493-6792

In addition, a replay of the call will be available until November 19th, by calling +1 877-660-6853 (Toll Free US & Canada); +1 201-612-7415 (Toll Free International).

Conference ID: 13724432

2021 has been a productive year thus far for Cellectis. We have made great progress during Q3 with both our clinical trials and preclinical product candidate pipeline, and are eager to share additional preliminary data from our BALLI-01 clinical trial and disclose initial pre-clinical data from TALGlobin01, at ASH this year, said Dr. Andr Choulika, Chief Executive Officer of Cellectis. With regard to our preclinical UCART pipeline focusing on solid tumors, we have made notable progress with UCARTMESO, targeting mesothelin - expressing solid tumors, and are excited to share new pre-clinical data that support anti-tumor activity at the Society for Immunotherapy of Cancer (SITC) Annual Meeting later this month.

GMP production remains on track for Cellectis Manufacturing site in Raleigh, NC, where qualification of facility equipment and systems was completed during Q3. Cellectis also continues to expand its internal manufacturing capabilities with its Paris site, which is now operational. Cellectis continues to leverage its expertise in gene editing and clinical development to transform the lives of patients with cancer and rare genetic diseases, and we look forward to continuing this effort in Q4, into 2022, and beyond.

Allogeneic CAR-T Cell Development Programs

Sponsored Phase 1 Studies

Cellectis continues to make progress in its proprietary programs enrolling patients in its three sponsored Phase 1 dose escalation trials:

UCART22 is an allogeneic CAR-T cell product candidate targeting CD22 being evaluated in patients with relapsed or refractory B cell acute lymphoblastic leukemia (r/r B-ALL) in the BALLI-01 Phase 1, multi-center dose-escalation clinical study.

UCART123 is an allogeneic CAR T-cell product candidate targeting CD123 being evaluated in patients with relapsed or refractory acute myeloid leukemia (r/r AML) in the AMELI-01 Phase 1, multi-center dose-escalation clinical study.

UCARTCS1 is an allogeneic CAR T-cell product candidate targeting CS1 being evaluated in patients with relapsed or refractory multiple myeloma (r/r MM) in the MELANI-01 Phase 1, multi-center dose-escalation clinical study.

Cellectis to present updated clinical data on BALLI-01 investigating UCART22 product candidate in R/R B-ALL at the 2021 American Society of Hematology Annual meeting

Wholly-controlled UCART Preclinical Programs

UCART20x22, is in development as the first allogeneic dual CAR-T cell product candidate which is being developed for patients with B-cell Non-Hodgkin lymphoma.

UCARTMESO, is an allogeneic CAR-T cell product candidatetargeting mesothelin,which is being developedforpatients withmesothelin expressing solidtumors.

Gene Therapy Programs

.HEAL is a gene therapy platform for genetic diseases developed by Cellectis. The platform leverages the power of TALEN gene editing technology to perform genome surgery resulting in highly efficient and precise gene inactivation, insertion, and correction in hematopoietic stem cells (HSCs). Cellectis has announced programs in sickle cell disease, lysosomal storage disorders and primary immunodeficiencies.

Sickle Cell Disease

TALGlobin01, is an autologous ex vivo TALEN-edited CD34+ HSC therapy for the treatment of SCD

TALGlobin01 is developed using both TALEN technology to induce a double strand DNA break in the SCD-causing hemoglobin subunit beta (HBB) gene and adeno-associated virus (AAV) particles containing a DNA repair template designed to correct the faulty HBB gene via endogenous homology directed repair.

Cellectis to present preclinical data on TALGlobin01 for the treatment of Sickle Cell Disease at the 2021 American Society of Hematology Annual meeting

Primary Immunodeficiencies

In collaboration with professor ToniCathomen,Scientific Director at the Center for Chronic Immunodeficiency, Medical Center - University of Freiburg, Germany, Cellectis is developing two gene edited HSC product candidates to address primary immunodeficiencies.

The authors presented encouraging preclinical data for RAG1 for Severe Combined Immunodeficiency (SCID) and STAT3 for HyperIgEsyndrome, at the European Society of Gene and Cell Therapy (ESGCT) Congress held on October 19-22, 2021.

RAG1 Severe Combined Immunodeficiency (SCID)

HyperIgEsyndrome

Licensed Allogeneic CAR-T Cell Development Programs

ALLOGENE/SERVIER: ALLO-501 and ALLO-501A in patients with relapsed/refractory non-Hodgkin lymphoma (r/r NHL)

CD19 AlloCAR T program utilizes Cellectis technologies. ALLO-501 and ALLO-501A are being jointly developed under a collaboration agreement between Servier and Allogene based on an exclusive license granted by Cellectis to Servier. Servier grants to Allogene exclusive rights to ALLO-501 and ALLO-501A in the U.S., while Servier retains exclusive rights for all other countries.

ALLOGENE: ALLO-715 in patients with relapsed/refractory multiple myeloma (r/r MM)

The anti-BCMA AlloCAR T program, which utilize the Cellectis TALEN technologies, are licensed exclusively from Cellectis by Allogene and Allogene holds global development and commercial rights to these AlloCAR T programs.

Manufacturing Facility

Paris Starting Materials Manufacturing Facility

Raleigh GMP Manufacturing Facility

New appointment

Board appointment

Financial Results

The interim condensed consolidated financial statements of Cellectis, which consolidate the results of Calyxt, Inc. of which Cellectis is a 64.2% stockholder (as of September 30, 2021), have been prepared in accordance with International Financial Reporting Standards, as issued by the International Accounting Standards Board (IFRS).

We present certain financial metrics broken out between our two reportable segments Therapeutics and Plants in the appendices of this Q3 2021 and First Nine Months 2021 financial results press release. Third Quarter and First Nine Months 2021 Financial Result.

Cash: As of September 30, 2021, Cellectis, including Calyxt, had $216 million in consolidated cash, cash equivalents, current financial assets and restricted cash of which $201 million are attributable to Cellectis on a stand-alone basis. This compares to $274 million in consolidated cash, cash equivalents, current financial assets and restricted cash as of December 31, 2020, of which $244 million was attributable to Cellectis on a stand-alone basis. This net decrease of $58 million primarily reflects (i) $92 million of net cash flows used in operating, investing and lease financing activities of Cellectis, (ii) $15 million of net cash flows used in operating, capital expenditures and lease financing activities of Calyxt and (iii) $6 million of unfavorable FOREX impact which was partially offset by (iv) $45 million of net equity proceeds raised from sales under the Companys At-The-Market (ATM) program in April 2021 and (v) $10 million of proceeds from stock options exercises at Cellectis. Based on the current operating plan, Cellectis excluding Calyxt anticipates that the cash, cash equivalents, and restricted cash of $201 million as of September 30, 2021 will fund its operations into early 2023.

Revenues and Other Income: Consolidated revenues and other income were $11 million for the three months ended September 30, 2021 compared to $9 million for the three months ended September 30, 2020. Consolidated revenues and other income were $53 million for the nine months ended September 30, 2021 compared to $67 million for the nine months ended September 30, 2020. 50% of consolidated revenues and other income was attributable to Cellectis in the first nine months of 2021. This decrease between the nine months ended September 30, 2021 and 2020 was mainly attributable to a $28 million upfront payment received in March 2020 and the recognition of $19 million of other previously-received upfront and milestone payments on the five released targets based on the March 2020 amendment of the License, Development and Commercialization Agreement signed with Servier as well as a decrease in licenses revenue. That was partially offset by (i) the recognition of $15 million in Cytovia stock or an upfront non-cash payment of $15 million if certain conditions are not met by December 31, 2021, (ii) the recognition of a $5 million milestone payment from Allogene related to the Phase 1 clinical study for ALLO-316, in advanced or metastatic clear cell renal cell carcinoma, (iii) $15 million from higher high oleic soybean revenues and by (iv) $1.5 million from the PPP Loan forgiveness at Calyxt.

Cost of Revenues: Consolidated cost of revenues were $9 million for the three months ended September 30, 2021 compared to $8 million for the three months ended September 30, 2020. Consolidated cost of revenues was $29 million for the nine months ended September 30, 2021 compared to $18 million for the nine months ended September 30, 2020. This increase was primarily explained by the cost of products sold during the period by Calyxt.

R&D Expenses: Consolidated R&D expenses were $34 million for the three months ended September 30, 2021 compared to $20 million for the three months ended September 30, 2020. Consolidated R&D expenses were $97 million for the nine months ended September 30, 2021 compared to $64 million for the nine months ended September 30, 2020. 91% of consolidated R&D expenses was attributable to Cellectis in the first nine months of 2021. The $33 million increase between the first nine months of 2021 and 2020 was primarily attributable to (i) higher wages and salaries and social charges on stock option grants of $12 million, to (ii) higher purchases, external and other expenses of $19 million and to (iii) higher non-cash stock-based compensation expenses of $2 million.

SG&A Expenses: Consolidated SG&A expenses were $10 million for the three months ended September 30, 2021 and 2020. Consolidated SG&A expenses were $28 million for the nine months ended September 30, 2021 compared to $31 million for the nine months ended September 30, 2020. 59% of consolidated SG&A expenses was attributable to Cellectis in the first nine months of 2021. The $3 million decrease was attributable to lower non-cash stock-based compensation expenses of $5 million which was partially offset by higher wages and salaries and social charges on stock option grants of $1 million and higher other expenses of $1 million.

Net Income (loss) Attributable to Shareholders of Cellectis: The consolidated net loss attributable to shareholders of Cellectis was $37 million (or $0.82 per share) for the three months ended September 30, 2021, of which $33 million was attributed to Cellectis, compared to $30 million (or $0.71 per share) for the three months ended September 30, 2020, of which $25 million was attributed to Cellectis. The consolidated net loss attributable to Shareholders of Cellectis was $89 million (or $2.00 per share) for the nine months ended September 30, 2021, of which $75 million loss was attributed to Cellectis, compared to a loss of $42 million (or $0.98 per share) for the nine months ended September 30, 2020, of which $21 million was attributable to Cellectis. This $48 million increase in net loss between first nine months 2021 and 2020 was primarily driven by a decrease in revenues and other income of $13 million and by an increase in operating expenses of $39 million partially offset by $7 million increase in net financial gain.

Adjusted Net Income (Loss) Attributable to Shareholders of Cellectis: The consolidated adjusted net loss attributable to shareholders of Cellectis was $32 million (or $0.71 per share) for the three months ended September 30, 2021, of which $29 million is attributed to Cellectis, compared to a net loss of $27 million (or $0.63 per share) for the three months ended September 30, 2020, of which $22 million was attributed to Cellectis. The consolidated adjusted net loss attributable to Shareholders of Cellectis was $80 million (or $1.79 per share) for the nine months ended September 30, 2021, of which $66 million loss was attributable to Cellectis, compared to a loss of $30 million (or $0.72 loss per share) for the nine months ended September 30, 2020, of which $13 million was attributable to Cellectis. Please see "Note Regarding Use of Non-GAAP Financial Measures" for reconciliation of GAAP net income (loss) attributable to shareholders of Cellectis to adjusted net income (loss) attributable to shareholders of Cellectis.

We currently foresee focusing our cash spending at Cellectis for the Full Year of 2021 in the following areas:

CELLECTIS S.A.(unaudited)STATEMENT OF CONSOLIDATED FINANCIAL POSITION($ in thousands, except per share data)

CELLECTIS S.A.STATEMENT OF CONSOLIDATED OPERATIONS Third quarter(unaudited)($ in thousands, except per share data)

CELLECTIS S.A.STATEMENT OF CONSOLIDATED OPERATIONS First nine months(unaudited)($ in thousands, except per share data)

CELLECTIS S.A.DETAILS OF KEY PERFORMANCE INDICATORS BY REPORTABLE SEGMENTS Third Quarter(unaudited) - ($ in thousands)

CELLECTIS S.A.DETAILS OF KEY PERFORMANCE INDICATORS BY REPORTABLE SEGMENTS First nine-months(unaudited) - ($ in thousands)

Note Regarding Use of Non-GAAP Financial Measures

Cellectis S.A. presents adjusted net income (loss) attributable to shareholders of Cellectis in this press release. Adjusted net income (loss) attributable to shareholders of Cellectis is not a measure calculated in accordance with IFRS. We have included in this press release a reconciliation of this figure to net income (loss) attributable to shareholders of Cellectis, which is the most directly comparable financial measure calculated in accordance with IFRS. Because adjusted net income (loss) attributable to shareholders of Cellectis excludes Non-cash stock-based compensation expensea non-cash expense, we believe that this financial measure, when considered together with our IFRS financial statements, can enhance an overall understanding of Cellectis financial performance. Moreover, our management views the Companys operations, and manages its business, based, in part, on this financial measure. In particular, we believe that the elimination of Non-cash stock-based expenses from Net income (loss) attributable to shareholders of Cellectis can provide a useful measure for period-to-period comparisons of our core businesses. Our use of adjusted net income (loss) attributable to shareholders of Cellectis has limitations as an analytical tool, and you should not consider it in isolation or as a substitute for analysis of our financial results as reported under IFRS. Some of these limitations are: (a) other companies, including companies in our industry which use similar stock-based compensation, may address the impact of Non-cash stock- based compensation expense differently; and (b) other companies may report adjusted net income (loss) attributable to shareholders or similarly titled measures but calculate them differently, which reduces their usefulness as a comparative measure. Because of these and other limitations, you should consider adjusted net income (loss) attributable to shareholders of Cellectis alongside our IFRS financial results, including Net income (loss) attributable to shareholders of Cellectis.

RECONCILIATION OF GAAP TO NON-GAAP NET INCOME Third Quarter(unaudited)($ in thousands, except per share data)

(1)When we have adjusted net loss, in accordance with IFRS, we use the Weighted average number of outstanding shares, basic to compute the Diluted adjusted net income (loss) attributable to shareholders of Cellectis ($/share). When we have adjusted net income, in accordance with IFRS, we use the Weighted average number of outstanding shares, diluted tocompute the Diluted adjusted net income (loss) attributable to shareholders of Cellectis ($/share)

RECONCILIATION OF GAAP TO NON-GAAP NET INCOME First nine-months(unaudited)($ in thousands, except per share data)

(1)When we have adjusted net loss, in accordance with IFRS, we use the Weighted average number of outstanding shares, basic to compute the Diluted adjusted net income (loss) attributable to shareholders of Cellectis ($/share). When we have adjusted net income, in accordance with IFRS, we use the Weighted average number of outstanding shares, diluted tocompute the Diluted adjusted net income (loss) attributable to shareholders of Cellectis ($/share)

About CellectisCellectis is a gene editing company, developing first of its kind therapeutic products. Cellectis utilizes an allogeneic approach for CAR-T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients, and a platform to make therapeutic gene editing in hemopoietic stem cells for various diseases. As a clinical-stage biopharmaceutical company with over 21 years of expertise in gene editing, Cellectis is developing life-changing product candidates utilizing TALEN, its gene editing technology, andPulseAgile, its pioneering electroporation system to harness the power of the immune systemin order totreat diseases with unmet medical needs.As part of its commitment to a cure, Cellectis remains dedicated to its goal of providing lifesaving UCART product candidates for multiple cancers including acute myeloid leukemia (AML), B-cell acute lymphoblastic leukemia (B-ALL) and multiple myeloma (MM)..HEALis a new platform focusing on hemopoietic stem cells to treat blood disorders, immunodeficiencies andlysosomialstorage diseases.Cellectis headquarters are in Paris, France, with locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS).

For more information, visitwww.cellectis.com Follow Cellectis on social media: @cellectis, LinkedIn and YouTube.

For further information, please contact:

Media contacts:Margaret Gandolfo, Senior Manager, Communications, +1 (646) 628 0300Pascalyne Wilson,Director,Communications,+33776991433, media@cellectis.com

Investor Relation contact:EricDutang, Chief Financial Officer,+1 (646) 630 1748,investor@cellectis.com

Forward-looking Statements

This presentation contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by words such as at this time, anticipate, believe, expect, on track, plan, scheduled, and will, or the negative of these and similar expressions. These forward-looking statements, which are based on our managements current expectations and assumptions and on information currently available to management, include statements about our research and development projects and priorities, our pre-clinical project development efforts and the timing of our presentation of data. These forward-looking statements are made in light of information currently available to us and are subject to numerous risks and uncertainties, including with respect to the numerous risks associated with biopharmaceutical product candidate development as well as the duration and severity of the COVID-19 pandemic and governmental and regulatory measures implemented in response to the evolving situation. With respect to our cash runway, our operating plans, including product development plans, may change as a result of various factors, including factors currently unknown to us. Furthermore, many other important factors, including those described in our Annual Report on Form 20-F and the financial report (including the management report) for the year ended December 31, 2020 and subsequent filings Cellectis makes with the Securities Exchange Commission from time to time, as well as other known and unknown risks and uncertainties may adversely affect such forward-looking statements and cause our actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.

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Cellectis Provides Business Update and Reports Financial Results for Third Quarter and First Nine Months 2021 - GlobeNewswire

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass., Nov. 04, 2021 (GLOBE NEWSWIRE) --Intellia Therapeutics (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology bothin vivoandex vivo, today reported financial results for the third quarter ended September 30, 2021, and recent operational highlights.

Intellia continues to make important progress toward advancing its full-spectrum genome editing pipeline. Today, we are pleased to share that we remain highly encouraged by the safety profile, consistency of TTR reduction and ongoing effect of NTLA-2001 in the Phase 1 study. We believe we are closing in on identifying the recommended therapeutic dose for NTLA-2001 in patients with ATTR amyloidosis with polyneuropathy for further evaluation in Part 2, a single-dose expansion cohort. Additionally, while it has long been our plan to develop NTLA-2001 for all forms of ATTR amyloidosis, we have decided to accelerate the evaluation of NTLA-2001 in patients with ATTR amyloidosis whose primary clinical manifestation is cardiomyopathy. Based on the strength of our interim dataset, we are now seeking regulatory feedback for inclusion of the ATTR-CM patient population in our current Phase 1 study. We look forward to sharing updates from this program in Q1 2022, said Intellia President and Chief Executive Officer John Leonard, M.D. Beyond our lead program, we remain focused on advancing NTLA-2002 for HAE and NTLA-5001 for AML. We received regulatory clearance for both programs to initiate first-in-human studies and we expect those studies to begin later this year. Finally, we nominated two gene insertion development candidates NTLA-3001, our wholly owned AATD program, and a Factor 9 gene insertion candidate for Hem B in collaboration with Regeneron. Our rapidly expanding pipeline is yet one more example of the benefits of our modular platform in generating novel and potentially curative treatment options for the patients we aim to serve.

Third Quarter 2021 and Recent Operational Highlights

In Vivo Program Updates

Ex Vivo Program Updates

Research and Corporate Updates

Upcoming Events

The Company will participate in the following events during the fourth quarter of 2021:

Upcoming Milestones

The Company has set forth the following for pipeline progression:

Third Quarter 2021 Financial Results

Conference Call to Discuss Third Quarter Earnings

The Company will discuss these results on a conference call today, Thursday, November 4, at 8 a.m. ET.

To join the call:

A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellias website at http://www.intelliatx.com, beginning on November 4 at 12 p.m. ET.

About Intellia TherapeuticsIntellia Therapeutics, a leading clinical-stage genome editing company, is developing novel, potentially curative therapeutics using CRISPR/Cas9 technology. To fully realize the transformative potential of CRISPR/Cas9, Intellia is pursuing two primary approaches. The companys in vivo programs use intravenously administered CRISPR as the therapy, in which proprietary delivery technology enables highly precise editing of disease-causing genes directly within specific target tissues. Intellias ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases. Intellias deep scientific, technical and clinical development experience, along with its robust intellectual property portfolio, have enabled the company to take a leadership role in harnessing the full potential of CRISPR/Cas9 to create new classes of genetic medicine. Learn more at intelliatx.com. Follow us on Twitter @intelliatweets.

Forward-Looking Statements This press release contains forward-looking statements of Intellia Therapeutics, Inc. (Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its: ability to complete clinical studies for NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis pursuant to its clinical trial applications (CTA), including submitting additional regulatory applications in other countries; clinical data from the ongoing single-ascending dose portion of the Phase 1 study evaluating NTLA-2001; expectations on regulatory approval to expand the Phase 1 trial population to include patients with ATTR with cardiomyopathy (ATTR-CM); expectations of being able to initiate clinical trials for NTLA-2002 for the treatment of hereditary angioedema (HAE) and NTLA-5001 for the treatment of acute myeloid leukemia (AML); ability to enroll patients in its Phase 1/2 study for NTLA-2002 for the treatment of hereditary angioedema (HAE) in New Zealand and the United Kingdom; its IND-enabling activities for NTLA-3001 for the treatment of alpha-1 antitrypsin deficiency; nomination and continued preclinical development of new development candidates; expectations for a new collaboration with Regeneron for a Factor 9 (F9) development candidate for its Hemophilia B program; expectations for a new collaboration with SparingVision for the treatment of ocular diseases; advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as its ability to maintain and expand its related intellectual property portfolio; statements regarding the timing of regulatory filings regarding its development programs; plans to attend and present data at scientific conferences later this year; and our expectations regarding our use of capital and our ability to fund operations beyond the next 24 months.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain its intellectual property position; risks related to Intellias relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; the risk that regulatory approval to expand the Phase 1 patient population to include patients with ATTR-CM will be denied; risks related to the COVID-19 pandemic including the effects of the Delta variant; and the risk that Intellias collaborations with Regeneron, SparingVision or its other collaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent quarterly report on Form 10-Q as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with the Securities and Exchange Commission (SEC). All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Investors:Ian KarpSenior Vice President, Investor Relations and Corporate Communications+1-857-449-4175ian.karp@intelliatx.com

Lina LiDirector, Investor Relations+1-857-706-1612lina.li@intelliatx.com

Media:Matt CrensonTen Bridge Communications+1-917-640-7930media@intelliatx.commcrenson@tenbridgecommunications.com

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Intellia Therapeutics Announces Third Quarter 2021 Financial Results and Highlights Recent Company Progress - BioSpace

Recommendation and review posted by Bethany Smith

Julie Fisher, MD: Hello, and welcome to Cure Expert Connections on HER2-positive breast cancer. I'm Dr Julie Fisher and I'm a medical breast oncologist at the Levine Cancer Institute in Charlotte, North Carolina. I'd like to welcome one of my patients, Jocelyn Muller, who's here today to share the story of her journey with HER2-positive breast cancer. Welcome, Jocelyn. We're going to spend the next 60 minutes walking you through her diagnosis, treatment, and follow-up from each of our perspectives and our continued communication throughout the process.

Let me just go ahead and open with a brief overview of HER2-positive amplified breast cancer. This is referring to the human epidermal growth factor 2 receptor. This is a protein that is expressed on the surface of all breast cancer cells. It is overexpressed or present in excess in roughly 15% of cases of breast cancer. This is diagnosed via biopsy, so if a patient presents with an abnormal finding on her routine mammogram or if she's able to palpate an area of abnormality and is then referred for a biopsy, that's the next thing that happens. That is typically done these days in our radiology departments. They numb the skin, place a hollow bore needle in the tumor itself, and obtain some tissue. Our colleagues in pathology then look at the tissue under the microscope and can ascertain if it is indeed breast cancer. Anytime someone is diagnosed with an invasive breast cancer, the pathologists first test for the presence of the estrogen and progesterone receptors. They also assess this HER2/neu protein to determine: is this one of the cases that is overexpressed and has excess protein? Or is this indeed a case where there is the standard amount of HER2/neu protein? Again, the roughly 15% of cases that overexpress HER2/neu are referred to as HER2/neu-amplified or HER2/neu-positive; this is very relevant for the physician team taking care of the patient with HER2-amplified breast cancer because it has significant implications for both prognosis and treatment. We know that HER2-positive breast cancer has the potential to behave more aggressively, and it has a higher risk of returning post-treatment. Historically, that was something that we could only lament, but thankfully that changed dramatically in the early part of the 2000s with the advent of drugs that specifically target that HER2/neu protein. I tell my patients, "We use those drugs in conjunction with chemotherapy." I think of those specially target drugs, such as snipers, and they're going to specifically home-in on that overexpressed HER2/neu protein. That has again dramatically changed the landscape of how we treat HER2-amplified breast cancer. It's changed this from a disease that we previously thought was very high risk, into something that we have wonderful tools for treating. Again, these account for about 15% of breast cancers. Most HER2-amplified breast cancers will be hormone receptor-positive, meaning sensitive to either estrogen, progesterone, or both. The minority of those cases will be insensitive to those hormones but overexpress the HER2/neu protein. That has an effect on subsequent treatment recommendations as well. How do we test for this? There are 2 main ways that our pathology colleagues assess HER2/neu positivity. One is a simple stain: it's a test referred to as immunohistochemistry. Another scientific method for determining HER2/neu status is what we call FISH, which stands for fluorescence in situ hybridization. Either of those 2 tests can garner a HER2-positive breast cancer diagnosis.

Transcript edited for clarity.

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An Overview of HER2-positive Breast Cancer - Curetoday.com

Recommendation and review posted by Bethany Smith

Lanita Bennett is a local breast cancer survivor. Photo by Stephen Gross/The Anniston Star

Lanita Price Bennett believes in doing everything she can to prevent bad health issues.

I have taken every vaccine available; you name it Ive taken it, explained Bennett. Ive gotten my yearly mammogram for 40 years.

In 2020, her yearly mammogram was scheduled for May 10, but it had to be rescheduled to later that June.

In early July, the doctor said Bennett needed a repeat mammogram and a sonogram because something had shown up. She got a biopsy in mid-July and went back to get the results in late July.

My husband, Stan, and I would regularly deliver meals for Meals on Wheels, and we thought wed go in that morning for the appointment and head on to our route, Bennett said.

The doctor told Bennett she had a very aggressive triple-negative invasive ductal carcinoma. With the surge of COVID-19 at the time, her doctor was not certain how long surgery would be an option, but he did have an opening at 6 a.m. the next morning. Bennett decided to get her surgery then.

Because of COVID restrictions, my husband couldnt come in with me. He has been by my side for 41 years, so him not being allowed to come in was tough, said Bennett.

Dr. Michael Hall performed the surgery, and Bennett was home later the same day. At a follow-up appointment, Dr. Hall referred her to Dr. Aasim Sehbai, who told Bennett that hormone treatments were not going to work because of her type of cancer.

Bennett received chemotherapy treatments on Sept. 1, Sept. 21, Oct. 13 and Nov. 3 of 2020.

She was informed that her surgery increased her rate of survival by 75 percent and receiving chemotherapy increased her rate of survival by 15 percent.

I lost my hair shortly after the first treatment, and I had some taste issues. Water didnt even taste right, and I had some fatigue, Bennett said.

In mid-January of 2021, Bennett had what she assumed was an allergy. A chest x-ray and a scan of her lungs revealed she had pneumonia caused by the chemotherapy.

Dr. Raul Magadia informed her that the good news was she had the immune system of a 20-year-old.

Unfortunately, the bad news was that since it wasnt related to my immune system, there is not much the doctors could do, Bennett said. Antibiotics every day can cause resistance, as well as lung and kidney damage.

Bennett has a prescription for an antibiotic, and she typically makes it about 12 or 14 days before experiencing shortness of breath.

It isnt painful, just aggravating. There is no surgery that will correct it, Bennett said.

Bennett is looking into getting a percussion vest like some COVID or cystic fibrosis patients use.

Even after cancer and while dealing with pneumonia, Bennett never stays in bed all day.

I get out of bed and get dressed everyday. We just are not doing Meals on Wheels because I cannot risk exposure, she said.

The Bennetts regularly provide meals for St. Michaels clinic in Anniston, where Lanitas niece is a physicians assistant. They also regularly provide meals for the RMC COVID unit meal train.

I would go crazy if I just sat here. I just feel so fortunate that they caught it early. We dont have to go to Birmingham. We have a fantastic hospital right here, Bennett concluded.

Faith Dorn is a freelance writer in Anniston. Contact her at faith.h.dorn@gmail.com.

More:
Local survivor still dealing with after effects of breast cancer treatment - Anniston Star

Recommendation and review posted by Bethany Smith

BATON ROUGE, Louisiana - A new study will investigate how the brain and body fat communicate to control the production and release of leptin, a feedback hormone that helps regulate appetite and the number of calories burned.

The project is part of the National Institutes of Healths new effort focusing on interoception, the ways in which organisms sense and regulate signals within their bodies. Heike Muenzberg-Gruening, Ph.D., Professor and Director, Central Leptin Signaling Laboratory at Pennington Biomedical Research Center, is the principal investigator on this project.

Fat tissue plays an important role in our health. It stores and breaks down fat but also secretes hormones like leptin to impact energy expenditure, food intake and blood sugar levels, Dr. Muenzberg-Gruening said.

Fat cells are controlled by the sympathetic nervous system, which sets off the bodys fight or flight response and impacts leptin levels. However, this brain-to-fat circuit has yet to be fully explored.

Dr. Muenzberg-Gruening will use cutting-edge techniques to identify new components of the neural circuits to brown and white fat tissue. One of those tools is immunolabeling-enabled three-dimensional imaging of solvent-cleared organs (iDISCO), which allows researchers to create three-dimensional images of structures deep inside the brain and fat samples.

Dr. Muenzberg-Gruening will also study how various physiological conditions high and low body temperature, fasting and fed states influence interactions between fat tissues, the spinal cord and the brain that are involved in temperature control and metabolic regulation.

She plans to generate a circuit model that can predict how the body adapts the amount of energy it uses under different physiological conditions.

The new project is one of seven awards involving interoception, a new research focus for NIH. Interoception is not well understood, but if the process is not working properly, a person may not sense whether they are hungry, full, cold, hot or thirsty.

Gaining a better understanding of how brain and fat tissue communicate represents an important advance, one that could help researchers find better ways to treat obesity, said Pennington Biomedical Executive Director John Kirwan, Ph.D.

This 5-year project is supported by the National Center for Complementary & Integrative Health of the National Institutes of Health under Award Number AT011676-01. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

###

About the Pennington Biomedical Research Center

The Pennington Biomedical Research Center is at the forefront of medical discovery as it relates to understanding the triggers of obesity, diabetes, cardiovascular disease, cancer and dementia. The Center conducts basic, clinical, and population research and architected the Obecity, USA awareness and advocacy campaign to help solve the obesity epidemic by 2040. The Center is affiliated with Louisiana State University. The research enterprise at Pennington Biomedical includes over 480 employees within a network of 40 clinics and research laboratories, and 13 highly specialized core service facilities. Its scientists and physician/scientists are supported by research trainees, lab technicians, nurses, dietitians, and other support personnel. Pennington Biomedical is located in state-of-the-art research facilities on a 222-acre campus in Baton Rouge, Louisiana. For more information, see http://www.pbrc.edu.

Pennington Biomedical Research Center

6400 Perkins Road

Baton Rouge, LA 70808

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

See more here:
NIH will fund study of how the brain and fat tissue communicate - EurekAlert

Recommendation and review posted by Bethany Smith

Sometime in the distant past, well before humans walked the Earth, the ancestors of modern-day elephants evolved their iconic tusks. Elephants use their bleach-white incisors theyre technically giant teeth, like ours but longer to dig, collect food, and protect themselves.

Then Homo sapiens arrived, and elephant tusks became a liability. Poachers kill the massive animals for their tusks, which are worth about $330 a pound wholesale as of 2017. Hunters slaughter roughly 20,000 elephants a year to supply the global ivory trade, according to the World Wildlife Fund.

But just as tusks evolved because they provide a number of benefits, a striking new study shows that some populations of African elephants have rapidly evolved to become tuskless. Published in the journal Science, the papers authors found that many elephants in a park in Mozambique, which were heavily hunted for their ivory during a civil war a few decades ago, have lost their tusks presumably because tuskless elephants are more likely to survive and pass the trait on to their offspring.

While scientists have known about this trend for a while its not uncommon to see tuskless elephants in places with lots of poaching the study provides strong evidence that the trait is rooted in genetics, something previous research failed to do, said Andrew Hendry, an evolutionary biologist at McGill University who was not involved in the research. In other words, the study shows evolution in action.

The results also offer a vivid example of how animals can quickly adapt under human pressures such as poaching and climate change. Past research has shown that creatures can evolve new colors, shapes, and even behaviors to better tolerate the increasingly inhospitable world weve created for them. The problem is that even rapid evolution has its limits and many species are already on the brink.

Social conflict and the decline of wildlife are often closely linked, the authors of the Science study write. Few locations reveal a clearer picture of this than Gorongosa National Park, a protected area in central Mozambique where Shane Campbell-Staton, an evolutionary biologist at Princeton University, led the research.

During a 16-year civil war that began in 1977, poachers on both sides of the conflict slaughtered a huge number of elephants in the park for their ivory, which they sold to finance their efforts, according to the study. Over that period, the number of large herbivores (like elephants) at Gorongosa fell by more than 90 percent.

Thats not all that changed in the park. Between 1970 and 2000 a period that encompassed much of the impact of the long-running war the portion of female elephants without tusks nearly tripled. The researchers best guess was that it had something to do with genetics: A trait visible only in females suggests it might be associated with changes to genes on the X chromosome. (Female elephants have two X chromosomes, whereas males have an X and a Y chromosome.)

This study all but proved it. The first bit of evidence was that female calves born from tuskless mothers were often themselves tuskless, indicating that the trait is passed on from one generation to the next. A heritable trait is pretty strong evidence of a genetic basis, said Robert Pringle, a biology professor at Princeton and a co-author of the study.

The authors also identified a couple of regions in the animals DNA that appear to be associated with a lack of tusks. Sure enough, There is strong evidence for mutations on a particular region of the X chromosome, Pringle said. Mutations, or variations in an organisms DNA, are an important engine of evolution. If they result in traits that are beneficial such as tusklessness, for certain populations of female elephants theyre more likely to get passed to the next generation and drive evolution.

Remarkably, one of the genes associated with tusklessness is also present in humans, where its linked to a condition that limits the growth of our lateral incisors. These are essentially the same teeth that, in elephants, evolved into tusks millions of years ago.

What makes this study so fascinating is that it offers evidence of rapid evolution in an animal that has a pretty long lifespan 50 or 60 years in the wild, said Hendry and Fred Allendorf, a professor emeritus at the University of Montana who was not involved in the research.

Studies of elephants rarely can say anything about the genetic basis of tusklessness, Hendry said. For years, researchers assumed that rapid evolution was common only in small species with short life cycles. Given these results, Nobody can argue that evolution isnt occurring, even in the biggest and longest-lived species, he added.

In theory, its advantageous to be born without tusks in areas where poachers are active, Hendry said. But tusklessness also has its downsides. Elephants need their tusks to dig, lift objects, and defend themselves. The hulking incisors are not useless appendages.

The genes that seem to make female elephants tuskless also appear to prevent mothers from giving birth to male calves thats why all the tuskless elephants in the park are female, Pringle said. (Some mothers did give birth to males with tusks, who likely didnt inherit the gene.) Over time, a shift in the sex of elephants could have consequences for population growth.

There are also potential costs to African grasslands, which are among the rarest and most biodiverse ecosystems on Earth, the study authors write. By turning over soil in search of food and minerals and gouging trees with their tusks, African savanna elephants prevent forests from growing too dense and help maintain grasslands. Thats why theyre considered engineers of the ecosystem. If they lose their tusks, a whole web of plants and animals may feel the impact.

This evolutionary change could have massive cascading ecological influences, Hendry said.

Humans have shaped the environment around them for centuries, down to the very genetics of wild plants and animals. The tuskless elephants in this study are just one example in a long list of species that have adapted in response to the pressures weve placed on them.

Human-induced changes are creating conditions for fast biological evolution so rapid that its effects can be seen in only a few years or even more quickly, a team of scientists wrote in a landmark intergovernmental biodiversity report in 2019.

One of the earliest and most famous examples is the peppered moth in the UK. Before the Industrial Revolution, most of the moths flitting about England were white with speckles of black, which helped them blend in with lichen and tree bark. Then, in the mid- to late-1800s, coal-fired power plants and mills started belching dark soot that blackened trees in parts of the country. White moths stood out against the newly dark background and were more likely to be eaten by birds, whereas the once-rare black ones were camouflaged and survived. In a matter of years, some populations of peppered moths inverted from white to mostly black. The phenomenon was deemed industrial melanism.

Scientists have measured similar changes in recent decades. One study from 2003, for example, found that bighorn sheep in Alberta, Canada, evolved smaller horns in roughly 30 years. The reason? Trophy hunters tend to target rams with larger horns. Another study, published in November 2020, suggests that a type of lily found in the mountains of China is evolving less-colorful leaves so it doesn't stand out in regions where its harvested as a traditional herb.

Rising temperatures from climate change also appear to be making some animals, including birds and mammals, smaller, as I previously reported. Smaller bodies cool off more easily than larger ones, so shrinking could be an adaptive response in warming environments (though its not yet clear whether these particular changes are genetic).

Then there are species changing in less conspicuous ways. In Japan, populations of mamushi snakes that are heavily hunted for their perceived medicinal and nutritional benefits seem to be better at evading predators, compared to snake populations that hunters have ignored. Many species, including plants and insects, have evolved resistance to pesticides, which is why farmers often use several at once and chemical companies must constantly develop new solutions.

Theres something like hope behind the idea of rapid evolution. Humans are deforesting, polluting, and exploiting the Earth at an alarming pace, yet in some cases, animals are adapting to live another day. Theres even a term for this resilience, Hendry said: evolutionary rescue.

Still, this evolution, as fast-tracked as it may be, still often isnt quick enough to overcome the many threats species face. And because adaptations can also come with drawbacks, there are untold and unpredictable consequences for the ecosystem at large.

Plus, not all species can adapt their way out of crisis. Consider rhinoceroses, which poachers kill for their horns. Three of the five rhino species have been hunted almost to extinction, yet none appear to have evolved hornlessness.

In Gorongosa National Park, the ecosystem has largely recovered from the war, Pringle said. Poaching has subsided, but tusks havent bounced back. After the war, the park successfully rebuilt its infrastructure, ramped up law enforcement, and put social development programs into place. The presence of tuskless elephants is now akin to a scar from an injury thats healed, Pringle said. So while evolution may have helped these creatures survive, the real remedy is putting an end to the underlying forces that triggered it in the first place.

Original post:
Hunting alters animal genetics. Some elephants are even losing tusks. - Vox.com

Recommendation and review posted by Bethany Smith

As I travel around the country and go from farm to farm, I see a good trend beginning to take hold as dairy producers are understanding the value of carrying extra heifer inventory.

Since the inclusion of sexed semen into dairy breeding programs, dairies can easily be heavy on heifer numbers. This is an amazing technology that allows us to produce more heifers than may be needed, but when we look at the drain these animals can put on the dairy economy, several things come into view. Replacement cost, calf value, production standards, genetic progress and labor concerns are just a few of the items that need to be processed to make an informed decision on your heifer inventory choices.

To begin this discussion, we do have to get a little philosophical. For the known history of dairy production in North America, management has dictated that heifers be cherished and treasured as the lifeblood of sustainability for your dairy. I have heard over and over, Yeah, growing up I knew I was going to be in trouble if we lost any calves. Make no mistake, I am in no way suggesting we should not have good husbandry for our youngstock. In fact, I would suggest we care for and manage them even better than before but only the ones we choose to retain.

Keep in mind, there is a ratio of first-lactation animals in your herd that is the most productive. Heifers by physiology are about 80% to 85% of the size and the production value of a periparturient cow. So just adding first-lactation animals into the herd because we have them may in fact cost us production in the herd. So what are some ideas to think about that will allow us to capitalize on this untapped potential in our herds?

In the last 10 years, beef on dairy or dairy beef phrases have taken off like wildfire as a way to make some extra income from drop calves or to capitalize on higher prices and better growth rates for feeders or finish cattle. I think we need to bring in another technique from our friends in the beef world and start looking at only retaining the heifers we need. Beef producers do not cull mother cows unless they absolutely must, as they are the breadwinners of the herd. Heifers will be inserted into the management chain only as needed to fulfill a need, whether for space or replacement. If we investigate doing this similarly in dairy management, we need to reverse our thinking and start from the end of a heifers life cycle.

To begin, we need to decide on the number of heifers we would ideally like to insert into the herd every month to maintain current population but not overwhelm the lactation ratio we choose. Dr. Jud Heinrichs from Penn State University has published equations to help us figure it out.

First, we calculate the heifers we need: (herd size[total]) x (cull rate) x (age at first calving 24) x (1 + non-completion rate for heifers). The non-completion rate being heifers that are born alive but never make it to the milk barn. Second, we calculate the number we produce: (herd size[total]) x (12 calving interval) x (percent female calves) x (1 calf mortality rate) x (24 age at 1st calving). Calf mortality counts deaths in first 48 hours after birth.

These two equations allow us to get an idea of our heifer surplus. These are the animals we carry in inventory that are costing us money, space or potential. Opinions vary, but I have seen operations as low as 33% and as high as 45% with first-lactation animals in the herd. If we are going to limit ourselves to raising fewer heifers than we are producing, where can we find advantages? Do we raise dairy beef or do we look at exclusively sexed semen, or embryo transfer for high genetic potential, or should we have cows that only milk and dont get pregnant? What can we do with the extra cow womb space now that we dont need every cow to deliver a calf into the herd?

There are a few items we need to investigate to realize the financials for this management change:

Economically, every dairy is different in lots of ways. Whether you sell extra feed, raise beef for sale, buy less feed, this analysis and change in philosophy may be of benefit. There will be considerable effort required to make change happen and for it to work.

First steps to get started are to use the two equations above, add 5% to 10% for safety and look at exactly how many heifers you need every month to maintain your herd. We cannot automatically say this will work for you, as this discussion is much more in-depth than an article can address. As with almost everything on a dairy operation, quality management and a team approach can make these scenarios achievable.

PHOTO:To maximize production, farms should evaluate their replacement heifer numbers and only raise the ones they need. Dairy beef may be a great option to fill extra cow womb space.Photo by Mike Dixon.

Original post:
Old heifer-raising ideals may not be the way of the future - Progressive Dairy

Recommendation and review posted by Bethany Smith

Abstract

The results of multiple studies have shown that a substantial proportion of men with advanced prostate cancer carry germline DNA repair mutations. Germline testing in prostate cancer may inform treatment decisions and consideration for clinical trials. There are 2 FDA approved PARP inhibitors (PARPi), olaparib (Lynparza) and rucaparib (Rubraca), for the treatment of advanced prostate cancer with DNA repair deficiency. Increasing demand for germline testing in prostate cancer and a shortage of genetic counselors have created a need for alternative care models and encouraged oncologists to take a more active role in performing germline testing. This article summarizes recommendations for germline testing in prostate cancer and describes care models for providing counseling and testing.

Genetic testing in men with prostate cancer has become more widespread since the discovery that men with metastatic prostate cancer are more likely to carry germline DNA repair gene mutations and the approval of PARP, or poly adenosine diphosphate-ribose polymerase, inhibitors (PARPi) for prostate tumors with DNA repair deficiency. The resulting substantial increase in men with prostate cancer who are eligible for germline testing, with time-sensitive treatment implications, challenges the traditional in-person, time- and resource-intensive cancer genetics care delivery model, and calls for alternative approaches. Urologists, oncologists, and other medical providers are encouraged to take a more active role in delivering germline testing, and they should be aware of current guidelines and optimal pretest and posttest counseling components. This article focuses on the implementation of germline testing in the care of patients with prostate cancer.

Germline genetic testing evaluates for inherited mutations (otherwise known as pathogenic or likely pathogenic variants) that are found in virtually all cells of the body and are derived from the fundamental DNA of an individual. DNA from no cancerous, healthy cells (eg, leucocyte or saliva/buccal swab cells) are used for germline genetic testing. The goals of germline genetic testing are to evaluate for an inherited cancer syndrome; to inform individual and family cancer risks; and to guide cancer prognosis and treatment decisions. Germline testing should be distinguished from recreational and somatic (tumor-specific) testing. Direct-to-consumer recreational genetic testing consists of an at-home test that is advertised to help understand the customers ancestry. Recreational genetic panels look for inherited variants in saliva/buccal swab cells to inform genealogy, and they are not primarily intended to guide medical decisions as they lack gene coverage and clinical-grade precision. None of the recreational genetic tests include a comprehensive assessment of the BRCA1/2 or other DNA damage repair genes and are inadequate for medical purposes. Somatic testing panels are designed to identify alterations in a tumors DNA. A somatic test may occasionally identify mutations expected to be germline, in which case follow-up dedicated germline tests are needed. Examples of somatic panels that report germline mutations include Tempus and UW-Oncoplex. However, many somatic panels use bioinformatics algorithms that may filter out, miss, and/or choose not to report germline mutations. Thus, in general, somatic panels should not be considered adequate for germline conclusions; at most, they should prompt confirmatory germline testing. This articlefocuses on dedicated clinical-grade germline testing.

Germline testing in men with prostate cancer is being performed more often since an important number of prostate cancer cases have a heritable component.1,2 Germline mutations in DNA repair genes, such as BRCA1/2, contribute to hereditary prostate cancer risk and are present in up to 11.8% of men with metastatic prostate cancer,3 compared with 4.6% among men with localized prostate cancer and 2.7% in persons without a known cancer diagnosis.3,4

Germline BRCA1/2 mutations are associated with increased risk of prostate cancer: up to a 3.8-fold increase with BRCA1 and an 8.6-fold increase with BRCA2 mutations.5 Men who carry germline BRCA1/2 mutations are not only at increased risk of developing prostate cancer but are also at risk of a more aggressive prostate cancer phenotype. In their study, Castro et al found that patients with prostate cancer with germline BRCA1/2 mutations at the time of diagnosis were more likely to have higher Gleason score (8) and more advanced stage (T3/4, nodal involvement, and metastases) compared with noncarriers. Men with germline BRCA1/2 mutations also had shorter cancer-specific survival (CSS) than noncarriers (15.7 vs 8.6 years; P=.015).6 Men with localized prostate cancer and germline BRCA1/2 mutations have worse outcomes after definitive treatment with surgery or radiation compared with noncarriers: 5-year metastasis-free survival, 72% vs 94%; P <.001; 5-year CSS, 76% vs 97%; P <.001.7 The prospective PROREPAIR-B study found that germline BRCA2 status is an independent prognostic factor for CSS in patients with metastatic castration-resistant prostate cancer (mCRPC; 17.4 vs 33.2 months; P = .027).8

Based on the study results above and others, the current National Comprehensive Cancer Network (NCCN) guidelines for prostate cancer (version 1.2022)9 recommend germline testing for the subsets of patients with prostate cancer who are more likely to have germline DNA repair mutations (Figure 1).

The NCCN guidelines recommend offeringgermline testing to the following groups of patients with prostate cancer9:

I. Men with node positive, high-risk or very highrisk localized prostate cancer

II. Men with metastatic prostate cancer

III. Men meeting family history criteria (Table 1)

NCCN recommends considering germline testing for men with personal history of prostate cancer and:

I. intermediate risk prostate cancer and intraductal/cribriform histology

II. personal history of exocrine pancreatic, colorectal, gastric, melanoma, pancreatic, upper tract urothelial, glioblastoma, biliary tract or small intestinal cancers

Several commercial vendors provide germline testing panels, including Invitae, Color, and Ambry. Further details and information on available panels can be found on the vendors websites. Panel sizes vary from dedicated BRCA1/2 testing to 91-gene panels. The NCCN guidelines for prostate cancer9 recommend that germline testing panels include genes associated with Lynch syndrome (MLH1, MSH2, MSH6, PMS2) and homologous recombination genes (BRCA1/2, ATM, PALB2, CHEK2).9,10 Broader panels might be appropriate for men with mCRPC, especially if clinical trial participation is being considered. Average turnaround time for germline testing is between 10 and 30 days, which varies depending on the particular panel. The cost of germline testing varies depending on insurance coverage. Some companies offer provide testing for a flat out-of-pocket fee (eg, $250), and a benefit of participating in certain research studies may be no-cost testing.

NCCN guidelines recommend germline testing for a large subset of patients with prostate cancer, but the best care model to offer education and testing is unclear. The traditional clinical care delivery model for cancer genetics includes 2 in-person visits with a genetic counselor, the first for pretest risk assessment and education and the second to discuss the results. This is the most established pathway and, historically, has been utilized the most. However, broadening recommendations for germline testing create great demand that cannot be currently met in a timely fashion by the approximately 4000 genetic counselors in the United States.11,12 Therefore, oncologists and other providers are increasingly performing pretest counseling, ordering genetic testing, and providing posttest counseling for their patients, or following hybrid models (Table 2).13

The provider-led germline testing model has been tested in breast and ovarian cancer but is new in prostate cancer.14-18 Scheinberg et al reported results of a multicenter prospective study evaluating provider-led germline testing for men with prostate cancer. Twelve oncologists received training about the role of germline testing and in counseling patients, and then offered germline testing to patients with mCRPC in their practice. Those patients who accepted germline testing received pretest counseling and educational materials, and later discussed test results in the oncologists office. If a germline mutation was identified, the patient was referred to a genetic counselor to discuss the further implications of the results and to initiate cascade testing. Most patients (63 of 66; 95%) accepted the germline testing and high satisfaction rates were achieved among both oncologists and patients.19 A provider-led germline testing model in the Veterans Affairs health care system was also evaluated. Patients with metastatic prostate cancer were offered germline testing by their oncologists during regular clinic visits. Pretest counseling was provided by oncologists and study coordinators and saliva for the test was collected in the clinic. Posttest counseling sessions with genetic counselors were provided over the phone by the testing panel company. Again, most patients (190 of 227 approached veterans; 84%) accepted testing, and the test completion rate was 80% (182/227).20 Results of early studies suggest that provider-led germline testing in prostate cancer could be effective and satisfactory for both patients and providers.

The need to streamline germline testing also calls for the utilization of new technologies, such as video- or phone-based counseling. The EMPOWER study (NCT04598698) assessed mens preference of in-person genetic counseling vs video-based genetic education21; results indicated that in-person genetic counseling was preferred by men with less education and higher anxiety levels, and it resulted in greater improvement of cancer genetics knowledge. The rates of genetic testing uptake were similar for video-based and in-personcounseling groups.21 Video-based counseling was also evaluated by Tong et al, who compared 2 models of streamlined germline testing in prostate cancer: (a) a take-home genetic kit provided by an oncologist, followed by referral to a genetic counselor if subsequent results are concerning; and (b) a genetic testing station, at which the patient participated in a video call from a genetic counseling assistant for genetics education and collection of family history, which was followed by saliva sample collection and, later, referral to a genetic counselor if any mutation was identified. The latter approach resulted in a lower rate of incomplete tests and a higher rate of follow-up with genetic counselors for positive results. Authors suggested that utilization of video education and involvement of genetic counselor assistants may improve access to germline testing among patients with prostate cancer.22 Several studies are ongoing to evaluate other care models to provide genetic testing in prostate cancer (eg, NCT02917798, NCT03076242, NCT03328091, NCT03503097).23

Oncologists who choose to perform germline testing need to be comfortable with several aspects of genetic counseling and to remain current on the ethics of informed consent and posttest counseling for germline testing (Figure 2). The 2019 Philadelphia Prostate Cancer Consensus Conference suggests that optimal pretest consent should include discussion of the purpose of testing, types of possible results (ie, pathogenic/likely pathogenic; benign/likely benign; variant of unknown significance; no variants identified), the possibility of identifying hereditary cancer syndrome and/or other cancer risks, testings potential cost, the importance of cascade family testing, and the Genetic Information Nondiscrimination Act (GINA) law.12 The GINA law protects against discrimination based on genetics in employment and health insurance; however, it is not applicable to life insurance, long-term care disability insurance, Indian Health services, and patients enrolled into federal employee, Veterans Administration, and US military health benefit plans.23,24 These gaps in protection by GINA law are important to discuss with patients, who may need to consider them before proceeding with the germline testing. Providers should also consider discussing the different panels available for testing, the privacy of genetic tests, and genetic laboratories policies related to sharing and selling of data.12

Providers ordering germline tests also must accept responsibility to follow up with patients if reclassification occurs of a variant of (currently) unknown significance (VUS). VUS are reported in about 30% of men with prostate cancer who undergo germline testing.4 VUS results do not change clinical recommendations, and the majority of them end up being reclassified as benign.25,26 In the Find My Variant Study, 38 of 63 VUS (61%) were reclassified: 32 of 38 (84%) as benign/likely benign and 6 of 38 (16%) as pathogenic/likely pathogenic.27,28 In the rare case when a VUS is reclassified as pathogenic or likely pathogenic, the provider who ordered the test is notified and they are responsible for disclosing the reclassification to the patient. Regardless of the model used, genetic counselor referral is recommended if a patient has a germline mutation identified and/or if clinical suspicion is high for an inherited cancer predisposition. Collaborative efforts are needed to educate oncology providers on aspects of germline testing counseling and to create shared printed and video resources for patients to facilitate informed consent.

Germline testing in men with prostate cancer can potentially benefit not only the patient but also family members. If a germline mutation is identified in a patient, testing for the same mutation in family members (cascade testing) should be performed. For instance, identifying family members with BRCA1/2 mutations could inform potentially lifesaving risk-reducing interventions, eg, prophylactic salpingo-oophorectomy for female BRCA1 mutation carriers. The IMPACT study (Identification of Men with a Genetic Predisposition to Prostate Cancer: Targeted screening ingBRCA1/2mutation carriers and controls) evaluated the utility of prostate-specific antigen (PSA) screening in men aged 40 to 69 years with germline BRCA1/2 mutations compared with its utility in noncarriers.29,30 The study enrolled 3027 men with no personal history of prostate cancer: 919 BRCA1 carriers, 902 BRCA2 carriers, 709 BRCA1 noncarriers, and 497 BRCA2 noncarriers. Preliminary results, reported after 3 years of follow-up, showed that BRCA2 mutation carriers, compared with noncarriers, have a higher incidence of prostate cancer and a younger age of diagnosis. The results for BRCA1 carriers were not definitive, and further investigation is needed. The results from IMPACT suggest annual PSA screening for BRCA2 mutation carriers aged between 40 and 69 years, using PSA cutoff of 3.0 ng/ml.30 Studies evaluating the predictive value of lower PSA cutoff and prostate MRI are ongoing (eg, NCT03805919, NCT01990521).

Advanced disease

PARPi. Patients with DNA repair mutations have higher responserates toPARPiand platinum chemotherapy.31,32 In 2020, two PARPi received FDA approval for treatment of mCRPC with germline or somatic DNA damage repair gene mutations. Rucaparib was approved based on the phase 2 TRITON2 (NCT02952534) study; it reported a 51% (50/98) radiographic response rate among men with mCRPC and BRCA1/2 alterations.33 The benefit for men with non-BRCA DNA repair mutations was less clear, and rucaparib is currently approved only for carriers of BRCA1/2 mutations. 33-35 The olaparib label includes a larger number of mutated genes eligible for treatment (BRCA1, BRCA2, ATM, BRIP1, BARD1, CDK12, CHEK1, CHEK2, FANCL, PALB2, RAD51B, RAD51C, RAD51D, RAD54L), based on results of the phase 3 ProFOUND study (NCT02987543). ProFOUND compared olaparib with enzalutamide or abiraterone and showed improved radiographic progression-free survival (5.8 months vs 3.5 months) with olaparib. 36 Several other ongoing studies are evaluating the efficiency of PARPi monotherapy and combined therapies in mCRPC. Table 3 summarizes study results reporting response rates to PARPi in prostate cancer. 37

Platinum chemotherapy. Historically, platinum chemotherapy has been used to treat tumors, such as ovarian or pancreatic cancer, that have a high frequency of DNA repair mutations.38,39 Early data suggest that platinum chemotherapy is also effective in prostate tumors with DNA repair deficiency.40-43 A retrospective case series by Cheng et al showed that 3 of 3 patients with prostate cancer who had biallelic inactivation of BRCA2 had an exceptional response to platinum chemotherapy after progressing on several therapies.40 The results of a larger retrospective study supported this observation, reporting that 75% (6/8) of patients with mCRPC and withgermline BRCA2 mutations had a PSA50 response (ie, decline of prostate-specific antigen by 50% from baseline) to platinum chemotherapy compared with 17% (23/133) of mCRPC patients without gBRCA2 mutations.41 Mota et al reported a 53% (8/15) PSA50 response to platinum chemotherapy among men with mCRPC and DNA damage repair mutations (ie, BRCA2, BRCA1, ATM, PALB2, FANCA, and CDK12).43

NCCN guidelines recommend considering DNA repair mutation status when discussing the possibility of active surveillance. Germline mutations in BRCA1/2 or ATM are associated with a higher likelihood of grade reclassification among men undergoing active surveillance.44 Mutation carriers should be closely monitored; they could potentially benefit from an earlier definitive treatment approach.

BRCA1/2 carriers have worse outcomes with conventional definitive therapies. Castro et al evaluated the response of BRCA1/2 carriers with localized prostate cancer to 2 radical treatmentsdefinitive radiation and radical prostatectomyand reported that BRCA status is an independent prognostic factor for metastasis-free survival (HR, 2.36; P = .002) and CSS (HR, 2.17; P = .016).7 New treatment approaches in earlier disease stages are being evaluated in clinical trials for patients with prostate cancer and DNA repair deficiency. Targeted therapies, such as PARPi, are being actively investigated in the biochemically recurrent stage of prostate cancer (eg, NCT03047135, NCT03810105, NCT04336943, NCT0353394) and as neoadjuvant therapy in localized disease (eg, NCT04030559).

Germline testing is becoming more commonplace with advances in precision oncology and expanding treatment implications of the results of this testing. The NCCN prostate cancer guidelines recommend germline testing for men with high-risk or very highrisk localized prostate cancer; men with metastatic prostate cancer; patients with intraductal histology of the prostate; and patients meeting family history criteria. These recommendations have created a need for germline testing of many prostate cancer patients, which calls for a change in the traditional cancer genetics delivery model to meet the new demand.45 Oncologists are encouraged to take a more active role in performing germline testing, but the optimal approach is unclear. Until the results of larger trials focusing on various testing delivery models are available, joint efforts are needed to build collaborative relationships between oncologists and genetic specialists. Further efforts are required to create dedicated resources to support providers in this new era of genetic testing and precision oncology in prostate cancer, which is marked by near-constant change.

ACKNOWLEDGMENTS: We gratefully acknowledge support from the Institute for Prostate Cancer Research, NIH/NCI CCSG P30CA015704, NIH SPORE CA097186, NCI T32CA009515 award, Congressional Designated Medical Research Program (CDMRP) award W81XWH-17-2-0043, and the Prostate Cancer Foundation.

Conflict of interest/disclosures: AOS has no conflicts to disclose; HHC receives research funding to her institution fromClovis Oncology, Color Genomics, Janssen Pharmaceuticals, Medivation, Inc. (Astellas Pharma Inc), Phosplatin Therapuetics, and Sanofi S.A., and has a consulting or advisory role withAstraZeneca.

Sokolova is from the Division of Medical Oncology at Oregon Health Science University (OHSU) and the OHSU Knight Cancer Institute.

Cheng is from the Division of Medical Oncology at the University of Washington and the Division of Clinical Research at Fred Hutch Cancer Research Center.

1. Mucci LA, Hjelmborg JB, Harris JR, et al; Nordic Twin Study of Cancer (NorTwinCan) Collaboration. Familial risk and heritability of cancer among twins in Nordic countries. JAMA. 2016;315(1):68-76. doi:10.1001/jama.2015.17703

2. Hjelmborg JB, Scheike T, Holst K, et al. The heritability of prostate cancer in the Nordic Twin Study of Cancer. Cancer Epidemiol Biomark Prev. 2014;23(11):2303-2310. doi:10.1158/1055-9965.EPI-13-0568

3. Pritchard CC, Mateo J, Walsh MF, et al. Inherited DNA-repair gene mutations in men with metastatic prostate cancer. N Engl J Med. 2016;375(5):443-453. doi:10.1056/NEJMoa1603144

4. Nicolosi P, Ledet E, Yang S, et al. Prevalence of germline variants in prostate cancer and implications for current genetic testing guidelines. JAMA Oncol. 2019;5(4):523-528. doi:10.1001/jamaoncol.2018.6760

5. Giri VN, Beebe-Dimmer JL. Familial prostate cancer. Semin Oncol. 2016;43(5):560-565. doi:10.1053/j.seminoncol.2016.08.001

6. Castro E, Goh C, Olmos D, et al. Germline BRCA mutations are associated with higher risk of nodal involvement, distant metastasis, and poor survival outcomes in prostate cancer. J Clin Oncol. 2013;31(14):1748-1757. doi:10.1200/JCO.2012.43.1882

7. Castro E, Goh C, Leongamornlert D, et al. Effect of BRCA mutations on metastatic relapse and cause-specific survival after radical treatment for localised prostate cancer. Eur Urol. 2015;68(2):186-193. doi:10.1016/j.eururo.2014.10.022

8. Castro E, Romero-Laorden N, Del Pozo A, et al. PROREPAIR-B: a prospective cohort study of the impact of germline DNA repair mutations on the outcomes of patients with metastatic castration-resistant prostate cancer. J Clin Oncol. 2019;37(6):490-503. doi:10.1200/JCO.18.00358

9. NCCN Clinical Practice Guidelines in Oncology. Prostate cancer, version 1.2022. https://www.nccn.org/professionals/physician_gls/pdf/prostate.pdf. Accessed 9/10/2021

10. Cheng HH, Sokolova AO, Schaeffer EM, Small EJ, Higano CS. Germline and somatic mutations in prostate cancer for the clinician. J Natl Compr Cancer Netw. 2019;17(5):515-521. doi:10.6004/jnccn.2019.7307

11. Abacan MA, Alsubaie L, Barlow-Stewart K, et al. The global state of the genetic counseling profession. Eur J Hum Genet. 2019;27(2):183-197. doi:10.1038/s41431-018-0252-x

12. Giri VN, Knudsen KE, Kelly WK, et al. Implementation of germline testing for prostate cancer: Philadelphia Prostate Cancer Consensus Conference 2019. J Clin Oncol. 2020;38(24):2798-2811. doi:10.1200/JCO.20.00046

13. Giri VN, Hyatt C, Gomella LG. Germline testing for men with prostate cancer: navigating an expanding new world of genetic evaluation for precision therapy and precision management. J Clin Oncol. 2019;37(17):1455-1459. doi:10.1200/JCO.18.02181

14. George A, Riddell D, Seal S, et al. Implementing rapid, robust, cost-effective, patient-centred, routine genetic testing in ovarian cancer patients. Sci Rep. 2016;6:29506. doi:10.1038/srep29506

15. Yoon SY, Bashah NSAhmad, Wong SW, et al. LBA4_PR. Mainstreaming genetic counselling for genetic testing of BRCA1 and BRCA2 in ovarian cancer patients in Malaysia (MaGiC study). Ann Oncol. 2017;28(suppl 10):x187. doi:10.1093/annonc/mdx729.004

16. Enomoto T, Aoki D, Hattori K, et al. The first Japanese nationwide multicenter study of BRCA mutation testing in ovarian cancer: CHARacterizing the cross-sectionaL approach to Ovarian cancer geneTic TEsting of BRCA (CHARLOTTE). Int J Gynecol Cancer. 2019;29(6):1043-1049. doi:10.1136/ijgc-2019-000384

17. Kemp Z, Turnbull A, Yost S, et al. Evaluation of cancer-based criteria for use in mainstream BRCA1 and BRCA2 genetic testing in patients with breast cancer. JAMA Netw Open. 2019;2(5):e194428. doi:10.1001/jamanetworkopen.2019.4428

18. Colombo N, Huang G, Scambia G, et al. Evaluation of a streamlined oncologist-led BRCA mutation testing and counseling model for patients with ovarian cancer. J Clin Oncol. 2018;36(13):1300-1307. doi:10.1200/JCO.2017.76.2781

19. Scheinberg T, Goodwin A, Ip E, et al. Evaluation of a mainstream model of genetic testing for men with prostate cancer. JCO Oncol Pract. 2021;17(2):e204-e216. doi:10.1200/OP.20.00399

20. Sokolova A, Cheng HH, Montgomery B. Implementation of systematic germline genetic testing (GT) for metastatic prostate cancer (mPC) patients at the Puget Sound VA Prostate Oncology Clinic. J Clin Oncol. 2020;38(15 suppl):abstr 1578. doi:10.1200/JCO.2020.38.15_suppl.1578

21. Giri VN, Bowler N, Hegarty S, et al. Video vs. in-person genetic counseling for men considering germline prostate cancer testing: a patient-choice study. J Clin Oncol. 2020;38(15 suppl):abstr 1577. doi:10.1200/JCO.2020.38.15_suppl.1577

22. Tong B, Borno H, Alagala F, et al. Streamlining the genetics pipeline to increase testing for patients at risk for hereditary prostate cancer. J Clin Oncol. 2021;39(6 suppl):abstr 66. doi:10.1200/JCO.2021.39.6_suppl.66

23. Paller CJ, Antonarakis ES, Beer TM, et al; PCCTC Germline Genetics Working Group. Germline genetic testing in advanced prostate cancer; practices and barriers: survey results from the Germline Genetics Working Group of the Prostate Cancer Clinical Trials Consortium. Clin Genitourin Cancer. 2019;17(4):275-282.e1. doi:10.1016/j.clgc.2019.04.013

24. Genetic discrimination. National Human Genome Research Institute. Updated September 16, 2020. Accessed April 27, 2021. https://www.genome.gov/about-genomics/policy-issues/Genetic-Discrimination

25. Mersch J, Brown N, Pirzadeh-Miller S, et al. Prevalence of variant reclassification following hereditary cancer genetic testing. JAMA. 2018;320(12):1266-1274. doi:10.1001/jama.2018.13152

26. Slavin TP, Manjarrez S, Pritchard CC, Gray S, Weitzel JN. The effects of genomic germline variant reclassification on clinical cancer care. Oncotarget. 2019;10(4):417-423. doi:10.18632/oncotarget.26501

27. Tsai GJ, Raola JMO, Smith C, et al. Outcomes of 92 patient-driven family studies for reclassification of variants of uncertain significance. Genet Med. 2019;21(6):1435-1442. doi:10.1038/s41436-018-0335-7

28. Sokolova AO, Shirts BH, Konnick EQ, et al. Complexities of next-generation sequencing in solid tumors: case studies. J Natl Compr Canc Netw. 2020;18(9):1150-1155. doi:10.6004/jnccn.2020.7569

29. Page EC, Bancroft EK, Brook MN, et al; IMPACT Study Collaborators. Interim results from the IMPACT study: evidence for prostate-specific antigen screening in BRCA2 mutation carriers. Eur Urol. 2019;76(6):831-842. doi:10.1016/j.eururo.2019.08.019

30. Eeles RA, Bancroft E, Page E, Castro E, Taylor N. Identification of men with a genetic predisposition to prostate cancer: targeted screening in men at higher genetic risk and controlsthe IMPACT study. J Clin Oncol. 2013;31(6 suppl):abstr 12. doi:10.1200/jco.2013.31.6_suppl.12

31. Athie A, Arce-Gallego S, Gonzalez M, et al. Targeting DNA repair defects for precision medicine in prostate cancer. Curr Oncol Rep. 2019;21(5):42. doi:10.1007/s11912-019-0790-6

32. Carlson AS, Acevedo RI, Lim DM, et al. Impact of mutations in homologous recombination repair genes on treatment outcomes for metastatic castration resistant prostate cancer. PLoS ONE. 2020;15(9)e0239686. doi:10.1371/journal.pone.0239686

33. Abida W, Bryce AH, Balar AV, et al. TRITON2: an international, multicenter, open-label, phase II study of the PARP inhibitor rucaparib in patients with metastatic castration-resistant prostate cancer (mCRPC) associated with homologous recombination deficiency (HRD). J Clin Oncol. 2018;36(6 suppl):abstr TPS388. doi:10.1200/JCO.2018.36.6_suppl.TPS388

34. Abida W, Campbell D, Patnaik A, et al. Non-BRCA DNA damage repair gene alterations and response to the PARP inhibitor rucaparib in metastatic castration-resistant prostate cancer: analysis from the phase II TRITON2 study. Clin Cancer Res. 2020;26(11):2487-2496. doi:10.1158/1078-0432.CCR-20-0394

35. Sokolova AO, Yu EY, Cheng HH. Honing in on PARPi response in prostate cancer: from HR pathway to gene-by-gene granularity. Clin Cancer Res. 2020;26(11):2439-2440. doi:10.1158/1078-0432.CCR-20-0707

36. de Bono J, Mateo J, Fizazi K, et al. Olaparib for metastatic castration-resistant prostate cancer. N Engl J Med. 2020;382(22):2091-2102. doi:10.1056/NEJMoa1911440

37. Sokolova AO, Cheng HH. Genetic testing in prostate cancer. Curr Oncol Rep. 2020;22(1):5. doi:10.1007/s11912-020-0863-6

38. Alsop K, Fereday S, Meldrum C, et al. BRCA mutation frequency and patterns of treatment response in BRCA mutation-positive women with ovarian cancer: a report from the Australian Ovarian Cancer Study Group. J Clin Oncol. 2012;30(21):2654-2663. doi:10.1200/JCO.2011.39.8545

39. Chetrit A, Hirsh-Yechezkel G, Ben-David Y, Lubin F, Friedman E, Sadetzki S. Effect of BRCA1/2 mutations on long-term survival of patients with invasive ovarian cancer: the national Israeli study of ovarian cancer. J Clin Oncol. 2008;26(1):20-25. doi:10.1200/JCO.2007.11.6905

40. Cheng HH, Pritchard CC, Boyd T, Nelson PS, Montgomery B. Biallelic inactivation of BRCA2 in platinum-sensitive, metastatic castration-resistant prostate cancer. Eur Urol. 2016;69(6):992-995. doi:10.1016/j.eururo.2015.11.022

41. Pomerantz MM, Spisk S, Jia L, et al. The association between germline BRCA2 variants and sensitivity to platinum-based chemotherapy among men with metastatic prostate cancer. Cancer. 2017;123(18):3532-3539. doi:10.1002/cncr.30808

42. Mota JM, Barnett E, Nauseef J, et al. Platinum-based chemotherapy in metastatic prostate cancer with alterations in DNA damage repair genes. J Clin Oncol. 2019;37(15 suppl):abstr 5038. doi:10.1200/JCO.2019.37.15_suppl.5038

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45. Carlo MI, Giri VN, Paller CJ, et al. Evolving intersection between inherited cancer genetics and therapeutic clinical trials in prostate cancer: a white paper from the Germline Genetics Working Group of the Prostate Cancer Clinical Trials Consortium. JCO Precis Oncol. 2018;2018: PO.18.00060. doi:10.1200/PO.18.00060

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Germline Testing in Prostate Cancer: When and Who to Test - Cancer Network

Recommendation and review posted by Bethany Smith

A generation ago, clinical wisdom strongly suggested that ADHD rarely existed in girls, much less adult women. Researchon ADHD almost exclusively focused on boys and men.

However, research now shows that ADHD in women and girls simply presentsdifferently. This is why the myth that only males have ADHD has persisted for decades.

ADHD in boys is often fairly noticeable because they're disruptive at home and at school. ADHD symptoms in boys often affectothers.

They "act out"aggressively, are hyperactive, restless, and interruptive. Their problems are very noticeable to teachers and parents, so they're more likely to be assessed, diagnosed, and treated.

RELATED:6 Common But Often Overlooked Symptoms Of ADHD In Adults

In some cases, this is also mixed with hyperactivity.

Generally, these girls tend to turn their emotions and behaviors inward that inward vs. outward focus is apparent in many of the behaviors, reactions, and disorders that affect both men and women.

A leading ADHD expert in womens and girls ADHD, Stephen Hinshaw Ph.D.,says that girls', and later womens, symptoms include that they "tend to take their problems out on themselves rather than others. Compared with boys who have the disorder, as well as with girls without it, girls with ADHD suffer more mood disorders such as anxiety and depression as well."

Due to these differences in presentation, teachers and parents often believe girls are just daydreamers, absent-minded, "dumb,"or socially awkward.

Since they often turn inward, they "act out" less and are not as disruptive as boys. Thus parents and teachers often miss these more subtle clues and their significance.

Or they focus solely on concerns about the girls' home lifeor depressive and anxious behaviors, missing the ADHD part of the picture altogether.

Recent research posits that potential adult-onset cases of ADHD are more likely to exist in females.

Research has not been conclusive so far however as to whether this is due to the factors explained above that it's just not seen or assessed due to presenting differently or if it's truly absent before adolescence or young adulthood in more females than males.

There's evidence that genetics plays a large role. Statistics indicate levels of genetic impact align with disorders such as schizophrenia or bipolar disorder.

Genetic factors are thought to contribute up to 40 percent of causes when it comes to these disorders.

Whether it's geneticsor another factor such as nurture orsituational aspects or all of these factors together in some measure, young, older onset, and ongoing adult female ADHD has been found to have a profound impact in womens lives.

The bad news is thatgirlsand young women with undiagnosed ADHDcan become women with higher risk for multiple social and emotional problems,intimate partner violence, self-harm (NSSI, "nonsuicidal self-injury,"harming themselves through cutting, etc.) as well as have a greater incidence of actual suicide attempts.

Females with ADHD have been shown to have impulsive behaviors that lead to factors likestrikingly high rates of unplanned pregnancies, substance abuse, and other "impulse control"problems.

Research has shown that40 percent of women with ADHD encounter these life events versus just 10 percent of young women without ADHD.

Obviously, these effects can come from many factors besides ADHD including situational factors likeexposure to trauma, poverty, or societal/community harms.

RELATED:Im A Lost Girl Of ADHD

Women with ADHD often site their ability to focus for long periods of timeusually called "hyperfocus" as a benefit of ADHD.

Some feel the eclectic point of view ADHD can bring allows them to be more creative. Many have an artistic bent and believe they are able to think outside of the box more easily due to ADHD.

While self-report tests can try to answer the question, "Do I have ADHD?", most professionals believe such tests are only indicators that consultation with an expert is needed.

Assessment and diagnosis are very important. Most women report a sense of relief and clarity upon being diagnosed. They experience a boost in self-esteem and improved functioning when they understand their diagnosis and prognosis.

Another benefit towomen with ADHD and symptoms of ADHD is understanding that no matter the cause of their ADHD,it is treatable.

Some believe medication is the answer.

Medications likeAdderall (mixed amphetamine salts) greatly improve focus for some and lessen anxiety. The majority of therapists and doctors agree that the most beneficial treatment includes a blend of medication and talk therapy.

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A combination of Cognitive Behavioral Therapy (CBT) and Relational Multicultural Theory (RMT)-based therapycan be the most helpful to women experiencing ADHD.

Research backs up this view.

While Cognitive Behavioral Therapy provides a structured way to challenge ones automatic thoughts and choose new ones that better fit our current life circumstances and beliefs, RMT can provide an important addition, especially for women.

Relational Multicultural Theory (RMT) addresses the social anxiety and sense of self or self-esteem aspects of ADHD in women.

RMT was developed by the Stone Center at WellesleyCollege which bases its longitudinal research on girls and women and how they develop and grow.

Their research shows that most teen, young adult, and adult women with ADHD benefit from discussion, education, and therapy experiences that focus on their unique needs as females.

Therapy should include assistance with interactions in relationships, realistic and aspirational relational and personal expectations, and a connection with a therapist that is positive and supportive.

RMT addresses these concerns to help women with ADHD see themselves, their own needsand others' needs, and their path forward in a clearer and more positive light.

This blend of CBT, RMT, and improved research and treatment can help women with this disorder improve their lives and regain their sense of agency in the world.

Though ADHD in women and girls receives less attention than ADHD in men and boys, there are viable options for individuals to lead successful, healthy lives.

RELATED:How Adult ADD & ADHD Can Impact Healthy Relationships

Tracy Deagan is a psychotherapist with a strong background in working with those that experience PTSD, Dissociative disorders, and Healthy Multiplicity from traumatic experiences.

Read more:
The Different Ways That ADHD Symptoms Present in Women And How It Affects Everyday Life - YourTango

Recommendation and review posted by Bethany Smith

Dr. William N. Kelley, MACP, MACR, Professor of Medicine at the University of Pennsylvania, was recently chosen to be featured in the Top 50 Fearless Leaders Publication by the International Association of Top Professionals (IAOTP).

While being selected to be published in IAOTPs Top 50 Fearless Leaders Publication, is an honor in itself, only 50 of the worlds most brilliant, courageous, inspirational professionals are selected for this distinction.

These special honorees are hand selected to share their stories of perseverance, resilience, passion and strength. They have made outstanding contributions to society; they have impacted their industries and are respected in their trades. A chapter will be dedicated to each honoree and the book is anticipated to be released in the 4th quarter of 2021.

Dr. Kelley has exemplary brilliance in medical research and education. He has dedicated more than fifty years of his career as a Physician Scientist, Medical Doctor, and Educator. He will receive his IAOTP recognition from 2020 as Top Professor of the Year in Medicine at their Annual Awards Gala being held at the Plaza Hotel in NYC this December. Furthermore, Dr. Kelley will be inducted into IAOTPs exclusive Hall of Fame on stage at this ceremony.

Dr. Kelley earned his Doctor of Medicine at Emory University in Atlanta, GA, in 1963 and subsequently served an internship and residency in Medicine at the Parkland Memorial Hospital in Dallas, TX. He completed his senior residency in Medicine at Massachusetts General Hospital in Boston. Dr. Kelleys other titles have included Clinical Associate in Human Biochemical Genetics with the National Institutes of Health, Educator to Fellow of Medicine at Harvard University, and Macy Faculty scholar at the University of Oxford in England. Later in his career, he received an honorary Master of Arts from the University of Pennsylvania.

His impressive repertoire of roles has included Dean of the Perelman School of Medicine, CEO of the University of Pennsylvania Medical Center, and Founding CEO of the Penn Health System (now known as Penn Medicine). Prior appointments included Professor of Medicine, Associate Professor of Biochemistry, and Chief of Rheumatic and Genetic Diseases at Duke University, followed by Professor of Biological Chemistry and Internal Medicine, and Chair of Internal Medicine with the Medical School at the University of Michigan in Ann Arbor.

In the early 1990s at PENN, Dr. Kelley, in his role as Dean of the Medical School and CEO of the Health System (the combination now known as PENN Medicine), began to build a broad research program focused on the creation of gene-based medicine and vaccines as a new method for preventing and curing human disease. While the road was a rocky one over the last three decades, he is proud to note that PENN Medicine is now the global leader in this new field. This includes the two recently FDA approved mRNA vaccines (Moderna and Biontech/Pfizer) to prevent COVID-19 which came from the PENN Medicine research laboratories of Doctors Katalin Kariko and Drew Weissman. Dr. Kelley is noted for developing the first fully integrated university-based academic health system in the country at the University of Pennsylvania and expanding the Medical Centers regional footprint by acquiring hospitals and private practices, including Pennsylvania Hospital and Penn Presbyterian Medical Center.

Dr. Kelley was known for his breakthrough research and leadership of academic medical programs at Duke and the University of Michigan when he arrived at Penn. During Dr. Kelleys Tenure, the Perelman School became a research powerhouse moving the school into the top 3 rankings for NIH funding. There is now a Professorship named in his honor at the Perelman School of Medicine.

Throughout his illustrious career, Dr. Kelley has received many awards, accolades and has been recognized worldwide for his outstanding leadership and commitment to the profession. This year he will be considered for an exclusive interview on TIP Radio and for the 2022 Doctor of the Decade award. In 2018 he received the Albert Nelson Marquis Lifetime Achievement Award. In 2005, Dr. Kelley was presented with the Kober Medal by the Association of American Physicians and the Emory Medal in 2000 from his alma mater, Emory University. He was the recipient of the David E. Rogers Award from the Association of American Medical Colleges, the John Phillips Award of the American College of Physicians, the Gold Medal Award from the American College of Rheumatology, the Robert H. Williams Award from the Alliance for Academic Internal Medicine, and the National Medical Research Award from the National Health Council. Dr. Kelley has been featured in many magazines and publications, including Whos Who in America, Whos Who in Medicine and Healthcare, and Whos Who in the World.

In addition to his successful career, Dr. Kelley is a sought-after lecturer, speaker, and contributor to numerous professional journals and chapters to books. He was the co-inventor of a Viral-Mediated Gene Transfer System, now the most used method today for in vivo gene therapy. Dr. Kelley founded and edited numerous early editions of Kelley and Firesteins Textbook of Rheumatology and Kelleys Textbook of Internal Medicine. He was also editor-in-chief for Essentials of Internal Medicine and co-editor of Arthritis Surgery and Emerging Policies for Bio-Medical Research. Dr. Kelley has served on the Board of Directors for many public companies such as Beckman Coulter, Inc. and Merck & Co., Inc, and has been involved with many committees and subcommittees with the National Institutes of Health. He is a member of the National Academy of Medicine, The American Academy of Arts & Sciences, and the American Philosophical Society.

Looking back, Dr. Kelley attributes his success to his perseverance, his education, his mentors as well as outstanding students and trainees he has had along the way. When not working, he enjoys traveling and spending time with his family. For the future, he hopes that his contributions, including support for those many outstanding faculty he helped to succeed, will continue to improve human health worldwide.

For more information on Dr. Kelley please visit: http://www.iaotp.com

Watch his video: https://www.youtube.com/watch?v=6uhxBnYVY54

About IAOTP

The International Association of Top Professionals (IAOTP) is an international boutique networking organization that handpicks the worlds finest, most prestigious top professionals from different industries. These top professionals are given an opportunity to collaborate, share their ideas, be keynote speakers, and to help influence others in their fields. This organization is not a membership that anyone can join. You have to be asked by the President or be nominated by a distinguished honorary member after a brief interview.

IAOTPs experts have given thousands of top prestigious professionals around the world, the recognition and credibility that they deserve andhave helped in building their branding empires.IAOTP prides itself to bea one of a kind boutique networking organization that hand picks only the best of the best and creates a networking platform that connects and brings these top professionals to one place.

For More information on IAOTP please visit: http://www.iaotp.com

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Award Winning Dr. William Kelley selected to be featured in IAOTPs Top 50 Fearless Leaders Publication - PRUnderground

Recommendation and review posted by Bethany Smith

A mutation in the BRCA gene can exacerbate the risk of developing hereditary breast cancer

This rise in breast cancer all over the world can be attributed to lifestyle choices such as sedentary living, alcohol or tobacco abuse, stress, increased oestrogen exposure due to use of contraceptive pills, delayed child birth etc. On the other hand, genetics also play a significant role. For instance, a mutation in the BRCA gene can exacerbate the risk of developing hereditary breast and ovarian cancer. It's also reported that constant factors such as polluted air and water can be attributed to the rise of cancer in people.

Early detection is the key

Though extremely serious, it must be noted that breast cancer can be treated and swiftly bridged to remission if a patient has early detection. It is advised to start mammography screenings as indicated by guidelines, women aged between 45-54 every year and women aged 55 and older once in 2 years. Furthermore, self-examinations are also helpful in detecting superficial lumps or undulations. Early education and awareness is critical, especially since breast cancer when occurring in young women is fairly aggressive. Any unusual discharge or lumps must be checked out at once.

While there is no definitive method of preventing breast cancer, early detection provides the best chance of effective treatment. When detected early, tumors can be surgically removed to eliminate the chances of metastasis. This offers excellent chances of complete recovery and increased survival rates. Alternatively, a patient will be less reliant on chemotherapy drugs which can eliminate or reduce chemotherapy-induced toxic effects resulting in improved quality of life. Early detection will reduce a patient's financial stress; along with emotional and psychological trauma associated with chemotherapy treatment. Since late detection of breast cancer will require aggressive forms of treatment along with surgery, radiation therapy and chemotherapy.

Up until a few decades ago, breast cancer was managed by mastectomy followed by several rounds of chemotherapy. Thanks to the technological and medical advancements that have opened a plethora of treatment options for these patients.

Myths busted!

In addition to a customized treatment plan for individual patients, there needs to be awareness about breast cancer, especially among young people who believe they're unlikely to develop this disease. Here are some other popular misconceptions-

Myth- If no one in the family has had cancer, that means you are risk-free

Only about 5-10% of all cancers are inherited or genetic. Majority of cancers (90-95%) are caused by DNA mutations that could develop over a person's life as a natural progression influenced by age, environmental factors (air pollution, cigarette smoke, tobacco) and encounters with carcinogens. It is advised to receive regular screenings and mammograms even if there's no suspected family history of cancer.

Myth- A cancer diagnosis is always fatal

Indeed, a cancer diagnosis used to be considered terminal. However, survivability rates today are higher than ever especially with early detection and appropriate treatment. With so much global investment in cancer research, certain variants of the disease are entirely curable, and most patients can live a long and healthy life once treated. Breast cancer patients for instance, have a 90% survival rate if detected in early stages (I and II).

Myth- One treatment suits all

We've come a very long way from having limited treatment options. Plans depend on stage of detection, size of lump, age, lifestyle, etc. Patients today can choose between full breast removal (mastectomy) or removal of only the malignant lump (breast conservation surgery). Radiation therapy could be applied depending on the type of surgery and lymph node involvement. Chemotherapy too could be entirely avoided.

In fact, cancer treatment is being customized based on molecular testing: mutations in cancer cells may help guide a patient's treatment. In the last few years, companies have been investing in the development of prognostic tests that can analyses the molecular make-up of a patient's cancer stage and estimate the probability of relapse. These tests are widely available today, even in India.

(Dr Rohan Khandelwal isLead Consultant & Head, The Breast Cancer Centre atCK Birla Hospital)

Disclaimer: The opinions expressed within this article are the personal opinions of the author. NDTV is not responsible for the accuracy, completeness, suitability, or validity of any information on this article. All information is provided on an as-is basis. The information, facts or opinions appearing in the article do not reflect the views of NDTV and NDTV does not assume any responsibility or liability for the same.

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Breast Cancer Patients, Here's What An Oncologist Wants You To Know - NDTV Doctor

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The month of October is observed as Breast Cancer Awareness Month

Breast cancer is now the most common form of cancer and has surpassed cervical cancer. Breast cancer is also now more common in the younger age group with more women in their thirties and forties being now diagnosed with breast cancer. This can also be attributed to increasing awareness on the disease but genetic, lifestyle and environmental factors are also at play. We often hear that early diagnosis is important. So how can we maximize the chance of catching breast cancer early? Here are some points to note.

Risk for breast cancer for women is high if there is family history. If a woman has a mother or a sister who has had breast cancer or family members on either her maternal or paternal side who have had breast or ovarian cancer, her risk for developing cancer is higher and therefore it is important for her to screen more often. Inherited gene mutations, such as BRCA1 and BRCA2 increase risk of breast cancer. Women who have inherited these genetic changes are a greater risk. The BRCA gene test is a blood test that helps determine if you have mutations in your DNA that increase the risk of breast cancer.

Family history of breast cancer increases the risk of an individualPhoto Credit: iStock

A self - breast examination along with other diagnostic tools can be powerful in detecting breast cancer early. Breast self-exam is no cost and convenient and helps understand your body better and most importantly if there are any changes, you may catch it early. A breast self-exam is recommended for all women above 20 years of age. There are many guides that help in a detailed self-breast examination and this usually does not take more than 15 minutes. A week after the menstrual cycle is the best time to do a self - breast exam.

A clinical breast exam is recommended annually for those between 20 and 30 years, half yearly for those between 30 and 60 years and annually for those above 60 years. A clinical breast exam happens at the doctor's office.

Mammograms are nothing but low dose x-rays of breast. A mammogram can detect breast changes so early that it may be years before physical symptoms develop and therefore for a women who is at average risk, a mammogram is a good diagnostic tool, especially when ordered after a clinical breast examination when any changes are noted.

Mammograms are not perfect and therefore may miss some cancers and may also lead to over-diagnosis. It is possible that someone is diagnosed with cancer which is caught during screening which may not have caused any problems at all. There is also 3D Mammogram that is available now that is especially found to be useful in women with dense breasts. Your doctor will help you understand the risk and limitations of a mammogram and accordingly provide medical guidance.

The Mammogram Guidance for a women with average risk as per the American Cancer Society is as below. Average risk is defined if a woman she doesn't either have a personal history of breast cancer of a family history or a genetic mutation like the BRCA gene or has not undergone chest radiation therapy before the age of 30.

To improve survival rates and treatment outcomes, it is important for women and men to be fully aware of breast cancer, understand one's own risk and take steps to increase the chances of diagnosing breast cancer early.

(Dr Simi Bhatia fromSRL Diagnostics, Mumbaihas almost 30 years of experience in laboratory medicine)

Disclaimer: The opinions expressed within this article are the personal opinions of the author. NDTV is not responsible for the accuracy, completeness, suitability, or validity of any information on this article. All information is provided on an as-is basis. The information, facts or opinions appearing in the article do not reflect the views of NDTV and NDTV does not assume any responsibility or liability for the same.

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Breast Cancer Awareness Month: All You Need To Know About Early Detection Of Breast Cancer - NDTV

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A mother and daughter from Blair, Nebraska, are in remission after their simultaneous battles with breast cancer. If you look through the branches of Amanda Nelson's family tree, you'll find a long history of breast cancer. So it was no surprise to her when she found out she carries the BRCA-2 gene, which makes her more susceptible to the disease."I knew without a doubt just from that history that the risk was very high for me," said Nelson. "There really never was a question of if I would get breast cancer, it was just always a matter of when is it going to happen," said Nelson.Nelson stayed on top of her breast health, scheduling annual mammograms and breast MRIs."So with that breast MRI, it does take a deeper dive so-to-speak out of that breast tissue, just to see what's going on," said Nelson. "It's a better picture from what you're going to get with a mammogram."Then in 2019, that MRI caught a tumor deep in her breast tissue."I believe is what saved my life," said Nelson.Things were already hard for Amanda, who was taking care of her mom, Terry Wulf, after she received a diagnosis of her own, a rarer form of breast cancer known as triple-negative. "It was scary. It was really, really scary to get hers," said Nelson.The mother and daughter's treatments were very different. Amanda underwent a double mastectomy, while her mom was put on several rounds of chemotherapy and intensive oral medications. "That was probably so hard on my body, that that is when I truly thought I wasn't going to live any longer, that that was going to kill me," said Wulf. Dr. Katie Honz is a reconstructive surgeon with Methodist Health System. She followed the two on their journey and performed Amanda's reconstructive surgery. Honz and a team of doctors meet each week for a tumor conference. These experts analyze cancer masses and come up with options for each patient. "These patients need a lot of care, even down to their physical therapy and support teams," said Honz. Wulf's battle with cancer would continue. To her doctors' surprise, Wulf was diagnosed with another form of cancer in her fallopian tube. But, with her daughter by her side, they never gave up, and after long, arduous battles, they both went into remission. Now, Terry and Amanda both ask others to keep a close eye on their breast health. Amanda hopes women can find groups for support and the right doctor to make everything more manageable. "I just want to think, 'I made it through it. And now I just want to live my life,'" said Wulf.

A mother and daughter from Blair, Nebraska, are in remission after their simultaneous battles with breast cancer.

If you look through the branches of Amanda Nelson's family tree, you'll find a long history of breast cancer. So it was no surprise to her when she found out she carries the BRCA-2 gene, which makes her more susceptible to the disease.

"I knew without a doubt just from that history that the risk was very high for me," said Nelson. "There really never was a question of if I would get breast cancer, it was just always a matter of when is it going to happen," said Nelson.

Nelson stayed on top of her breast health, scheduling annual mammograms and breast MRIs.

"So with that breast MRI, it does take a deeper dive so-to-speak out of that breast tissue, just to see what's going on," said Nelson. "It's a better picture from what you're going to get with a mammogram."

Then in 2019, that MRI caught a tumor deep in her breast tissue.

"I believe [the MRI] is what saved my life," said Nelson.

Things were already hard for Amanda, who was taking care of her mom, Terry Wulf, after she received a diagnosis of her own, a rarer form of breast cancer known as triple-negative.

"It was scary. It was really, really scary to get hers," said Nelson.

The mother and daughter's treatments were very different. Amanda underwent a double mastectomy, while her mom was put on several rounds of chemotherapy and intensive oral medications.

"That was probably so hard on my body, that that is when I truly thought I wasn't going to live any longer, that that was going to kill me," said Wulf.

Dr. Katie Honz is a reconstructive surgeon with Methodist Health System. She followed the two on their journey and performed Amanda's reconstructive surgery. Honz and a team of doctors meet each week for a tumor conference. These experts analyze cancer masses and come up with options for each patient.

"These patients need a lot of care, even down to their physical therapy and support teams," said Honz.

Wulf's battle with cancer would continue. To her doctors' surprise, Wulf was diagnosed with another form of cancer in her fallopian tube. But, with her daughter by her side, they never gave up, and after long, arduous battles, they both went into remission.

Now, Terry and Amanda both ask others to keep a close eye on their breast health. Amanda hopes women can find groups for support and the right doctor to make everything more manageable.

"I just want to think, 'I made it through it. And now I just want to live my life,'" said Wulf.

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Mother and daughter survive simultaneous battles with breast cancer - KETV Omaha

Recommendation and review posted by Bethany Smith

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MELVILLE, N.Y., Oct. 26, 2021 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (BioRestorative or the Company) (OTC: BRTX), a life sciences company focused on stem cell-based therapies, today announced the nomination of two new independent members to its Board of Directors with industry and medical device experience: Patrick F. Williams, Chief Financial Officer at STAAR Surgical, and David Rosa, President and Chief Executive Officer at NeuroOne. Their election to the Board will take effect in the event the Companys pending registration statement becomes effective.

Our new board member nominations represent qualified and diverse executives who bring new perspectives, relevant expertise and leadership experience, positioning BioRestorative to fulfill our mission of bringing cell therapies to patients said Lance Alstodt, Chief Executive Officer of BioRestorative. The addition of Patrick and David is part of a strategic effort to add meaningful leadership experience to BioRestoratives Board of Directors to support the companys focus on driving future growth, enhancing its corporate governance, and creating additional shareholder value.

Patrick F. Williams

Patrick F. Williams has more than 20 years of experience across medical device, consumer product goods and technology sectors. Appointed as Chief Financial Officer of STAAR Surgical Company in July 2020, Mr. Williams is responsible for optimizing the financial performance of STAAR and ensuring the scalability of various functions to support high growth expansion. From 2016 to 2019, he served as the Chief Financial Officer of Sientra, Inc. before transitioning to General Manager for its miraDry business unit. From 2012 to 2016, Mr. Williams served as Chief Financial Officer of ZELTIQ Aesthetics, Inc., a publicly-traded medical device company that was acquired by Allergan. Previously, he served as Vice President in finance, strategy and investor relations roles from 2007 to 2012 at NuVasive, Inc., a San-Diego based medical device company servicing the spine sector. He has also held finance roles with Callaway Golf and Kyocera Wireless. Mr. Williams received an MBA in Finance and Management from San Diego State University and a Bachelor of Arts in Economics from the University of California, San Diego.

David Rosa

DavidRosa has served as the Chief Executive Officer, President and a director of NeuroOne Medical Technologies Corporation, or NeuroOne (Nasdaq: NMTC), since July2017 and served as Chief Executive Officer and a director of NeuroOne, Inc., formerly its wholly-ownedsubsidiary, from October2016 until December2019, when NeuroOne, Inc. merged with and into NeuroOne. NeuroOne is committed to providing minimally invasive and hi-definition solutions for EEG recording, brain stimulation and ablation solutions for patients suffering from epilepsy, Parkinsons disease, dystonia, essential tremors, chronic pain due to failed back surgeries and other related neurological disorders that may improve patient outcomes and reduce procedural costs. From November2009 to November2015, Mr.Rosa served as the Chief Executive Officer and President of Sunshine Heart, Inc., n/k/a Nuwellis, Inc. (Nasdaq: NUWE), a publicly-heldearly-stagemedical device company. From 2008 to November2009, he served as Chief Executive Officer of Milksmart, Inc., a company that specializes in medical devices for animals. From 2004 to 2008, Mr.Rosa served as the Vice President of Global Marketing for Cardiac Surgery and Cardiology at St. Jude Medical, Inc. He serves as a director on the board of directors of Biotricity Inc (Nasdaq: BTCY) and is Chairman of the Board at Neuro Event Labs, a privately held AI-based diagnostics company in Finland.

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat chronic lower back pain arising from degenerative disc disease.

Metabolic Program (ThermoStem): We are developing a cell-based therapy candidate to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in animals may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company's latest Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:Email: ir@biorestorative.com

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BioRestorative Therapies Announces Nomination of Two New Members to the Board of Directors - StreetInsider.com

Recommendation and review posted by Bethany Smith

VANCOUVER, BC, Oct. 21, 2021 /PRNewswire/ -- The global culture media market size was USD 5.43 billion in 2020 and is expected to register a CAGR of 9.3% between 2021 and 2028. Steady market revenue growth is driven by rising need for monoclonal antibodies, growing emphasis on personalized medicine, increasing prevalence of infectious diseases, rising investment in research & development of innovative cell culture products, rising awareness about vaccines based on cell culture, and high demand for single-use technologies.

Drivers: Increased Investment in Research & Development of Innovative Cell Culture Products

Increased investment in research & development of innovative cell culture products is a key factor driving culture media market revenue growth. Cell culture media is an important component in producing cultivated meat. Cell culture media is necessary to keep the cells healthy and alive. Currently, most of these media are very expensive and oftentimes deliver inconsistent outcomes. A limited number of species-specific formulations of commercial culture media exists in case of cultivated meat firms dealing with fish species. For instance; in September 2020, GFI announced providing a two-year grant to a research project focused on development of a serum-free, high-quality fish cell culture media, which is an essential move in making cultivated seafood to reach market. Researchers at Virginia Tech are developing a formulation for open-source media improved for growing fish cells. This research project deploys artificial neural networks and Response Surface Methodology (RSM) to optimize cell culture media for better thriving of fish cells.

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Restraints: Cost Prohibitive Culture Media and Contamination Risks

Cost prohibitive culture media and contamination risks may hamper market revenue growth over the forecast period. Culture media comprise various ingredients such as serum and nutrients for cell growth, which makes the product very costly. Also, issues associated with specificity, variability, and standardization may also impact market revenue growth negatively. Sometimes, ingredients procured from poor sources can lead to contamination risks of cell culture media. This factor would also restrain demand for culture media.

Growth Projections

The global culture media market size is expected to reach USD 11.10 billion in 2028 and register a revenue CAGR of 9.3% over the forecast period, attributed to growing population, especially geriatric population, and rising prevalence of infectious diseases. Increasing prevalence of infectious diseases and rising need for development of more efficient drugs to combat resulting conditions are driving market revenue growth. Infectious diseases are considered to be the foremost cause of mortalities across the globe, particularly in young children living in low-income countries. As per the World Health Organization (WHO), diarrheal diseases and lower respiratory infections were included in the top 10 leading causes of death worldwide in 2019. Culture technologies are considered crucial for identification of infectious diseases, despite significant increase in molecular testing, as pathogenic organisms causing disease are required to be separated from other microbes in mixed cultures. In addition, occurrence of an organism is necessary for assessing the probability that a specific organism is responsible for a said disease, unlike a culture.

COVID-19 Direct Impacts

COVID-19 pandemic has boosted demand for culture media, as many biotechnology firms are conducting in-vitro R&D for vaccines and antivirals. In-vitro assessment of vaccines normally requires a culture media for identifying and analyzing the response and growing targeted microbes. Increasing emphasis on research & development of vaccines by various pharmaceutical companies to curb spread of COVID-19 virus is also propelling market revenue growth.

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Current Trends and Innovations

Increasing trend of single-use technologies plays a pivotal role in driving market revenue growth. In the biotechnology industry, use of single-use technologies has become a common practice. Engineers and researchers are utilizing plastic components as an alternative to stainless steel items in biomanufacturing processes. In cell culture production, adoption of single-use is quite essential and these cell growth systems may be wave-type bioreactors, plastic bioreactors, or plastic linings present in stainless-steel support. Reusable or disposable probes are present in all systems that protrude through an interior sleeve or attach to the outside. Majority of the connections depend on separate systems having aseptic/plastic connectors. Single-use systems are pre-cleaned and pre-sterilized, generally via gamma irradiation. Hence, there is no requirement for cleaning, sterilization, or sanitization steps. It saves money on use of chemicals for cleaning, as well as power and equipment needed to produce pure water and steam.

Geographical Outlook

Culture media market in Asia Pacific is expected to register fastest revenue CAGR during the forecast period, attributed to increasing geriatric population in countries such as Japan and China and increase in prevalence of chronic diseases. In addition, increasing prevalence of contagious diseases, high demand for personalized medication, and presence of biotechnology firms such as Daiichi Sankyo Company Limited and large population base in countries in the region are also contributing to market growth.

Strategic Initiatives

In December 2018, Fujifilm acquired IS Japan (ISJ) and Irvine Scientific Sales Company (ISUS). Both companies have expertise and technological know-how on cell culture media. Irvine Scientific Sales Company distributes its products mostly in Europe and the US, whereas IS Japan distributes its products primarily in Japan and various other Asian countries. Both of these firms offer culture media to bio-ventures, pharmaceutical companies, and academia. Fujifilm is a photography and imaging firm in Japan. It has entered into stock purchase contract worth approximately USD 800.0 million.

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Emergen Research has segmented global culture media market on the basis of type, research type, application, end-use, and region:

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Culture Media Market Size to Reach USD 11.10 Billion in 2028 | Increased Investment in Research & Development of Innovative Cell Culture Products...

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