Archive for the ‘Bone Marrow Stem Cells’ Category

The Phase 3 clinical trial testing BrainStorm Cell Therapeutics cell therapy candidateNurOwn inamyotrophic lateral sclerosis (ALS) patients is continuing as planned after a second safety assessment by the trials independent Data Safety Monitoring Board (DSMB) found no reasons to stop, the company announced.

The DSMBs recommendation comes after a pre-specified interim analysis of the first 106 ALS patients treated repeatedly with NurOwn in this randomized, placebo-controlled clinical trial.

After reviewing all of the safety data as of September 30th, the DSMB has recommended the study continue without any changes in the protocol. We did not identify any significant safety concerns, Carlayne Jackson, MD, a professor of Neurology and Otolaryngology UT Health San Antonioand the DSMB chairperson, said in a press release.

DSMBs consist of research experts who monitor the progress of a clinical trial and review safety and efficacy data while the study is ongoing. This panel can recommend that a trial be stopped early because of safety concerns or evidence a therapy is not working as intended, or if the trials main goals have already been reached.

NurOwn consists of mesenchymal stem cells (MSCs; stems cells able to generate various cell types) collected from a patients bone marrow. These MSCs are expanded and matured into a specific cell type called MSC-NTF by growing them under conditions that induce them to secrete high levels of neurotrophic factors (NTFs) that support the growth, survival, and maturation of nerve cells.

MSC-NTF cells also deliver immune system regulating cytokines, small proteins important in cell signaling or messaging, to sites of damage, BrainStorm reports. It is thought this will help to slow or stabilize disease progression.

The double-blind Phase 3 trial (NCT03280056),fully enrolledat its six U.S. sites, is investigating use of NurOwn in 200 ALS patients whose symptoms became evident within two years of the studys start. Patients are randomized 1:1 to either NurOwn or placebo, given via intrathecal (spinal canal) injection every two months.

The studysprimary measures of safety and efficacy are being determined using the ALS functional rating scale score (ALSFRS-R; a score of abilities like swallowing, speech, handwriting, walking, etc.) in patients after 28 weeks of treatment compared to placebo.

A secondary goal is assessing how biomarkers, such as cell-secreted neurothrophic factors, inflammatory agents, andcytokines, change in the blood and cerebrospinal fluid (the liquid surrounding the brain and spinal cord) after treatment with NurOwn.

BrainStorm is expecting to have topline trial data by the end of 2020, which will potentially support the submission of a Biologics License Application (BLA) to theU.S. Food and Drug Administration requesting approval.

We are very pleased with the DSMB recommendation that the Phase 3 clinical trial continue without any protocol modification. This represents an important clinical trial advancement for BrainStorm and for the development of NurOwn as an innovative cellular therapy approach for ALS patients, added Ralph Kern MD, BrainStorms chief operating officer and chief medical officer.

This clinical trial is being funded by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989), and other types of investment.

NurOwn was given orphan drug status by both theFDA and the European Medicines Agency (EMA) as apotential ALS treatment.

Iqra holds a MSc in Cellular and Molecular Medicine from the University of Ottawa in Ottawa, Canada. She also holds a BSc in Life Sciences from Queens University in Kingston, Canada. Currently, she is completing a PhD in Laboratory Medicine and Pathobiology from the University of Toronto in Toronto, Canada. Her research has ranged from across various disease areas including Alzheimers disease, myelodysplastic syndrome, bleeding disorders and rare pediatric brain tumors.

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Ins Martins holds a BSc in Cell and Molecular Biology from Universidade Nova de Lisboa and is currently finishing her PhD in Biomedical Sciences at Universidade de Lisboa. Her work has been focused on blood vessels and their role in both hematopoiesis and cancer development.

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Phase 3 Trial of ALS Cell Therapy, NurOwn, Gets Thumbs Up in Safety Review by Monitoring Board - ALS News Today

Mr. McManushas over 20 years of leadership experience in the biopharmaceutical industry that includes leading the transformation of several biotech companies through strategic partnerships and execution of value-based strategies.Prior to joining PTx, John was the CEO of Aeolus Pharmaceuticals, where he managed a $100+ million BARDA contract for the advanced development of a radiation medical countermeasure.Before joining Aeolus, John served in strategic and financial roles at Spectrum Pharmaceuticals where he focused on oncology and NeoTherapeutics where he focused on Alzheimer's, Parkinson's, ALS and spinal cord injury.John holds a B.S. in International Finance and Business Economics from the University of Southern California.

"We are very pleased to welcome John to our team to lead our business development activities," said Bob Mulroy, PTx's Chief Executive Officer. "His breadth of experience as a biotech executive and business leader will be tremendous assets for our team as we continue to build and grow the opportunity for Leukine to help patients in need. We also will benefit from John's extensive experience working with the U.S. government to advance our ability to serve as a partner for a wide range of important government programs."

"I am excited to join the PTx team in strengthening and expanding the Leukine franchise and identifying additional products that would benefit from the Company's development and clinical expertise," said Mr. McManus. "Leukine is a critical drug in the treatment of adults and children facing life-threatening diseases and a key medical countermeasure for improving survival after lethal levels of radiation exposure.I am especially excited about working to support the development of Leukine in new indications like melanoma and diseases of the central nervous system like Alzheimer's and Parkinson's and expanding its use as a medical countermeasure through development partnerships with third parties including the U.S. government.I see tremendous potential for Leukine to help patients across a number of difficult-to-treat diseases."

PTx acquired the global rights to develop, manufacture, and commercialize Leukine in 2018.

Leukine is a multi-lineage immune-stimulant that has been demonstrated to promote growth and activation of monocytes, macrophages, neutrophils and dendritic cells.It is the only FDA-approved recombinant human granulocyte-macrophage colony stimulating factor (GM-CSF). It is currently indicated for the treatment of AML in older adults to reduce the incidence of severe and life-threatening infections resulting in death; use in the treatment of allogeneic bone marrow transplants to reduce the incidence of bacteremia and other culture positive infections and shorten the median duration of hospitalization; to prolong the survival of patients who are experiencing bone marrow transplant failure or delay; and to increase survival in adult and pediatric patients from birth to 17 years of age acutely exposed to myelosuppressive doses of radiation (Hematopoietic Syndrome of Acute Radiation Syndrome [H-ARS]).

About Leukine(sargramostim)

Leukine is a yeast-derived recombinant humanized granulocyte-macrophage colony stimulating factor (rhuGM-CSF) and the only FDA approved GM-CSF.GM-CSF is an important leukocyte growth factor known to play a key role in hematopoiesis, effecting the growth and maturation of multiple cell lineages as well as the functional activities of these cells in antigen presentation and cell mediated immunity3.

Important Safety Information for LEUKINE (sargramostim)

Contraindications

Warnings and Precautions

Adverse Reactions

Adverse events occurring in >10% of patients receiving LEUKINE in controlled clinical trials and reported in a higher frequency than placebo are:

Please see full Prescribing Information for LEUKINE at http://www.leukine.com

Indications and Usage

LEUKINE (sargramostim) is a leukocyte growth factor indicated for the following uses:

About Partner Therapeutics, Inc.:

PTx is an integrated commercial-stage biotech company focused on the development and commercialization of therapeutics that improve health outcomes in the treatment of cancer. PTx's development focus spans the entire range of cancer therapy from primary treatments to supportive care. The company believes in delivering great products with the purpose of creating the best possible outcomes for patients and their families.

SOURCE Partner Therapeutics, Inc.

https://www.partnertx.com/

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Partner Therapeutics (PTx) Announces the Appointment of John McManus as Chief Business Officer - PRNewswire

MONTREAL, Nov. 06, 2019 (GLOBE NEWSWIRE) -- ExCellThera Inc., an advanced biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, announced today the publication of full data from the first clinical trial using ECT-001 (single UM171-expanded cord blood) in patients with haematological malignancies. The data were published in the peer-reviewed medical journal, The Lancet Haematology.

The clinical trial findings indicate that ECT-001 cell therapy is feasible, safe (as suggested by the low transplant-related mortality, low incidence of severe acute graft-vs-host disease (GVHD), and absence of moderate to severe chronic GVHD) and allows for the use of small cords without compromising engraftment. In addition, ECT-001 has shown potential to overcome the disadvantages of unexpanded cord blood transplants while maintaining their benefits of low risk of chronic GVHD and relapse. The Lancet Haematology paper provides the first detailed analysis of the study results presented at the 60th American Society of Hematology Annual Meeting (ASH 2018) in December 2018 and supports the recent advancement of the ECT-001 clinical program.

Were pleased that these important results from the first clinical trial using ECT-001 in haematological malignancies are now fully available to the broader bone marrow transplant community, said Dr. Guy Sauvageau, CEO and founder of ExCellThera, and co-senior author of the paper. These results indicate that ECT-001 transplants combine the advantages of conventional grafts using bone marrow (low treatment-related mortality), peripheral blood (fast engraftment) and cord blood (greater accessibility, low relapse and chronic GvHD) in a single, low cost, easy to produce 7-day culture product, which could lead to a paradigm shift in bone marrow transplantation.

The FDA granted ECT-001 Orphan Drug Designation for the prevention of graft-versus-host disease in 2018 and Regenerative Medicine Advanced Therapy Designation in the treatment of hematologic malignancies in 2019. ECT-001 is currently being used for the treatment of blood disorders in other ongoing and approved clinical trials in the United States and Canada. ExCellThera also plans to initiate a European clinical trial as well as a pivotal, multi-centre clinical trial in the coming months.

About ExCellThera Inc.

ExCellThera is an advanced clinical stage biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for use in novel one-time curative therapies for patients with hematologic malignancies, autoimmune and other diseases. ExCellTheras lead solution combines a proprietary small molecule, UM171, and an optimized culture system. In pursuit of better treatments for patients, the company is building out its portfolio of products, as well as supporting best-in-class clinical trials. excellthera.com

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ExCellThera announces publication in The Lancet Haematology highlighting excellent clinical results of ECT-001 in patients with haematological...

Antineoplastic is a class of medicine to restrain or kill tumor cells in the body. It plays an important role in comprehensive cancer treatment, particularly for disseminated tumor and leukemia that cannot be treated with surgery. Most current antineoplastics work by inhibiting DNA or RNA synthesis, or even damaging the structure of DNA. Antineoplastics can be divided into CCNSA (Cell cycle nonspecific drug) and CCSA (Cell Cycle Specific agents) according to their different effect on tumor cells in different stages of generation cycles. Both of them have a killing effect on bone marrow hematopoietic stem cells and other cells with a short growth cycle, as well as an inhibiting effect on the bodys immune response.

Factors such as growing incidence of cancer across the globe and demand for cost-effective treatment options are believed to play crucial role for the growth of global anti-neoplastic agents market. Rapid innovation in the field of personalized medicine presents a huge opportunity to manufacturers of anti-neoplastic agents. However, heterogeneous nature of cancer and high development cost of neoplastic agents are the factors limiting the growth of global anti-neoplastic agents market.

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The global Anti-Neoplastic Agents market is valued at xx million US$ in 2018 is expected to reach xx million US$ by the end of 2025, growing at a CAGR of xx% during 2019-2025.

This report focuses on Anti-Neoplastic Agents volume and value at global level, regional level and company level. From a global perspective, this report represents overall Anti-Neoplastic Agents market size by analyzing historical data and future prospect. Regionally, this report focuses on several key regions: North America, Europe, China and Japan.

Key companies profiled in Anti-Neoplastic Agents Market report are Hoffmann-La Roche, Amgen, Bristol-Myers Squibb Company, Baxter, Boehringer Ingelheim, Bayer, Teva Pharmaceutical Industries, Johnson & Johnson, Merck, Pfize, Accord Healthcare, Lundbeck, Abbvie and more in term of company basic information, Product Introduction, Application, Specification, Production, Revenue, Price and Gross Margin (2014-2019), etc.

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Table of Content

1 Anti-Neoplastic Agents Market Overview

2 Global Anti-Neoplastic Agents Market Competition by Manufacturers

3 Global Anti-Neoplastic Agents Production Market Share by Regions

4 Global Anti-Neoplastic Agents Consumption by Regions

5 Global Anti-Neoplastic Agents Production, Revenue, Price Trend by Type

6 Global Anti-Neoplastic Agents Market Analysis by Applications

7 Company Profiles and Key Figures in Anti-Neoplastic Agents Business

8 Anti-Neoplastic Agents Manufacturing Cost Analysis

9 Marketing Channel, Distributors and Customers

10 Market Dynamics

11 Global Anti-Neoplastic Agents Market Forecast

12 Research Findings and Conclusion

13 Methodology and Data Source

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Global Anti-Neoplastic Agents Market: Development History, Current Analysis and Estimated Forecast to 2025 - Market Research Reporting

Many for-profit stem cell clinics advertise therapies that are not backed by science and may actually cause harm.

For-profit stem cell clinics have popped up around California in recent years, advertising that they can treat everything from arthritis to Alzheimers, without FDA approval.

They claim that injections of stem cells (naturally occurring blank slate cells that can grow into any type of cell) can help alleviate pain or illness by replacing or regenerating diseased tissue claims that are not supported by existing research. The procedures can cost thousands of dollars out-of-pocket, and regulators have warned that patients have developed tumors, suffered infections and even lost eyesight after unapproved procedures.

No one knows how many clinics there are, but California reportedly has more than any other state. We asked Arnold Kriegstein, MD, PhD, director of the UC San Francisco Developmental & Stem Cell Biology Program, about whats real and whats not in stem cell medicine.

How do these clinics operate?

There has been an explosion of so-called clinics offering stem cell treatments for a wide range of ailments, none of which have been shown to be effective. They are largely unregulated. Many clinics claim that they can treat untreatable illnesses like Alzheimer's disease, autism, muscular dystrophy, or stroke. The list is quite extensive.

The majority are using fat tissue for their stem cells, obtained through liposuction. These are usually autologous cells, which means that they are taking the patient's own tissue and extracting cells to re-administer to the same patient, usually through an intravenous route. In addition to fat cells, some clinics administer bone marrow stem cells or umbilical cord or placental stem cells, which come from unrelated donors.

The clinics often advertise through testimonials from patients who've received their therapies. Many of the conditions that the testimonials address are the kinds that normally improve or fluctuate over time, such as joint pain, low back pain, arthritis, or multiple sclerosis.

The problem is that patients will receive a treatment, and then, within a month or two, they'll notice that the aches and pains in the joints are improving, and they will attribute the improvement to the stem cell therapy, when in fact it would've happened regardless.

What is the risk of trying an unproven stem cell treatment?

Reports of physical harm have included infections and the development of tumors. When using cells that are not the patients own, umbilical cord cells for example, immune responses can occur often triggering inflammatory conditions.

In cases where stem cells have been delivered into the eye, blindness has been reported, and when they have been delivered to the central nervous system through lumbar puncture (spinal tap), adverse outcomes including serious infections of the central nervous system and tumors have occurred.

Then there's the emotional cost associated with raising false hope, and the financial loss that comes from exorbitant fees charged for ineffective, potentially harmful therapies.

Why arent there more legitimate stem cell therapies available?

Stem cells have been in the news so much over the last decade or so that I think it has created the impression that therapies are already on the market. The reality is that it is very early days for the science. The most interesting, most promising animal studies are only now beginning to be translated into clinical trials, and the process for approval of therapies takes many years and very few are likely to succeed.

Unfortunately, the public needs to be patient, but the good news is that potential treatments are progressing along the pipeline.

What are some examples of proven stem cell therapies?

For the last 50 years or so, there have been countless patients successfully treated with hematopoietic stem cells, commonly known as bone marrow transplants. This remains the prototype for how a stem cell therapy can work. Other successful examples include corneal stem cell grafts for certain eye conditions, and skin grafts for burn victims.

There are efforts to see if stem cells could successfully treat diseases like Parkinson's and diabetes, particularly type 1 diabetes. There are clinical trials testing whether stem cell therapy might work against macular degeneration, a blinding disease that is very common as people age. There are also early stage clinical trials for nervous system disorders including stroke, spinal cord injury, and ALS (Lou Gehrigs disease).

All of these examples are still at a very early stage, where the primary goal is to make sure that the approaches are safe. To determine if they are effective will require large, well-controlled, relatively long-term clinical trials.

What will it take to advance stem cell therapy into more real treatments?

This is where basic research comes in. The field is evolving quickly, there's much to be done, and there's still a huge amount of promise in stem cell therapies down the road. But it's going to take a lot of very careful and very laborious research before we get there.

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Stem Cell Therapy: What's Real and What's Not at California's For-Profit Clinics - UCSF News Services

Three researchers at theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLAhave received awards totaling more than $18 million from the California Institute for Regenerative Medicine, the states stem cell agency.

The recipients are Dr. Sophie Deng, professor of ophthalmology at the UCLA Stein Eye Institute;Yvonne Chen, a UCLA associate professor of microbiology, immunology and molecular genetics; and Dr. Caroline Kuo, a UCLA assistant clinical professor of pediatrics. The awards were announced at a CIRM meeting today.

Dengs four-year, $10.3 million award will fund a clinical trial for a blinding eye condition called limbal stem cell deficiency. Limbal stem cells are specialized stem cells in eye tissue that help maintain the health of the cornea. Because of genetic defects or injuries caused by infections, burns, surgeries or other factors, some people do not have enough limbal stem cells, which results in pain, corneal scarring and blindness.

The approach she is testing involves extracting a small number of limbal stem cells from a persons eye, multiplying them in a lab, and then transplanting them back into the eye, where they could regenerate the cornea and restore vision. The research will be conducted in collaboration with theUCLAUCI Alpha Stem Cell Clinic, a partnership between UCLA and UC Irvine.

The grants awarded to Chen and Kuo are for projects that are heading toward the FDAs investigational new drug application process, which is required by the agency before a phase 1 clinical trial the stage of testing that focuses on a treatments safety.

Chens two-year, $3.2 million award will fund efforts to create a more effectiveCAR T cell therapyfor multiple myeloma, a blood cancer that affects white blood cells. The research will evaluate a specialized form of CAR T therapy that simultaneously targets two markers, BCMA and CS1, commonly found on multiple myeloma cells. CAR T therapies that target BCMA alone have been effective in clinical trials, but the presence of BCMA on multiple myeloma cells is not uniform.

Previous research has shown that the marker CS1 is present in around 90% of multiple myeloma cells. A CAR T therapy that targets both markers could potentially help more patients and reduce the likelihood of a cancer relapse.

Kuos 2 1/2-year, $4.9 million award, will support the development of a stem cell gene therapy for a deadly immunodeficiency called X-linked hyper IgM syndrome, or XHIM.

The syndrome, which is caused by a mutation in the CD40LG gene, results in invasive infections of the liver, gastrointestinal tract and lungs. Currently, the only potential cure is a bone marrow transplant from a matched donor, which carries life-threatening risks and is often less effective for XHIM patients than patients with other forms of immune deficiency. Even with current treatments, only 30% of people with the syndrome live to age 30.

Kuo will evaluate a stem cell gene therapy that corrects the genetic mutation that causes XHIM. After removing blood-forming stem cells from a person with the syndrome, the therapy would use a genetic engineering technique called CRISPR to insert a correct copy of the affected gene into the DNA of the stem cells. The corrected blood-forming stem cells would be infused back into the patient, where they could regenerate a healthy immune system.

She will collaborate with Dr. Donald Kohn, a UCLA distinguished professor of microbiology, immunology and molecular genetics who has successfully treated two other immune deficiencies bubble baby disease and X-linked chronic granulomatous disease with a similar therapy.

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Three UCLA scientists receive grants totaling more than $18 million - UCLA Newsroom

Saving the life of a fellow Canadian can be as easy as checking a box online or saying yes to being an organ donor when you renew your drivers license. But, thats just the beginning for those wanting to make a difference.

Deceased donations

In Alberta, individuals over the age of 18 can register their intent to become an organ or tissue donor when they die by using the Alberta Organ and Tissue Donation Registry. (Go to myhealth.alberta.ca online and search organ donation registry.) As well, agents and provincial registries are required to ask the donor question when clients are renewing a drivers licence or identification card.

For those who have Alberta Health Cards issued prior to 2018, the back of the card can be signed (with a witness) to declare their intention to donate.

The Alberta registry has been integrated into the provinces health care system through the use of donor co-ordinators. If a person has declared his or her intent to donate and is in a position to be considered for organ or tissue donation, a co-ordinator will discuss it with family members, who ultimately make the final decision.

Each deceased donor can provide up to eight organs (both lungs, both kidneys, liver, heart, pancreas, intestines), while donated tissues can benefit up to 75 individuals.

Living donations

The vast majority of living organ donors spares one of their two functioning kidneys to a person in need, though living liver donations also occur to a lesser extent.

In most cases, family members or acquaintances donate a living organ if theyre healthy enough to safely act as a donor. Once a viable donor is found, transplant programs in both Calgary and Edmonton perform the surgeries for kidneys, while live liver transplants are only performed in Edmonton.

Theres also been a rise in so-called altruistic donors, who are willing to share their organs with a stranger. Both the Kidney Foundation of Canada and Canadian Blood Services can advise prospective living donors on where to turn, while Alberta Health can connect donors to local living donor programs.

Canadian Blood Services also operates the Kidney Paired Donation Program, an inter-provincial initiative that maintains prospective donors in a registry if they arent a compatible match for their intended recipient. Since January 2009, some 500 living donors across Canada have entered the KPD program, including 90 anonymous donors who joined the program without a specific recipient in mind. Non-directed, anonymous donations are responsible for more than two-thirds of the transplants in the KPD program, and all patients with a match have received a transplant in less than a year.

The Living Donor Services Program Edmonton: Phone 780-407-8698; toll free 1-866-253-6833; email: livingdonors@ahs.ca.

Southern Alberta Transplant Program Calgary: Phone 403-944-4635.

More information on kidney health is available from the Kidney Foundation of Canada: http://www.kidney.ca; 780-451-6900 or 403-255-6108.

More information on liver health is available from the Canadian Liver Foundation: http://www.liver.ca; 403-276-3390 or 1-800-563-5483.

Details about Green Shirt Day and Logan Boulet are at greenshirtday.ca.

Stem cell donations

Stem cell transplants replace a patients unhealthy stem cells with a donors healthy ones, and can be used to treat cancers and other diseases. The three sources of stem cells are from bone marrow, peripheral (circulating) blood and umbilical cord blood.

Prior to any donation, the donor will undergo a comprehensive health assessment before undergoing the procedure. Peripheral blood stem cell donation only requires blood to be drawn from a needle in hospital following five days of under-the-skin injections to boost the number of blood cells in the bloodstream.

Bone marrow donations are performed under anesthesia, with hollow needles used to withdraw stem cells from bone marrow in the back of pelvic bones. The procedure lasts between 45 to 90 minutes and the marrow replenishes itself in four to six weeks.

Those who wish to become a stem cell donor can call Canadian Blood Services at 1-888-2-DONATE (1-888-236-6283) or by visiting the agencys website at blood.ca.

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Organ donations: What you can do to help save a life - Calgary Herald

The research provides in-depth study and analysis on Stem Cell Banking market. This report also gives complete overview of the global market, covering the different aspects such as product definitions along with leading market players. To get better perspectives of global market, relevant chart and graphs are included in the report.

Stem cell banking or preservation is a combined process of extraction, processing and storage of stem cells, so that they may be used for treatment of various medical conditions in the future, when required. Stem cells have the amazing power to get transformed into any tissue or organ in the body. In recent days, stem cells are used to treat variety of life-threatening diseases such as blood and bone marrow diseases, blood cancers, and immune disorders among others.

Stem Cell Banking market report gives the reasonable picture of the current industry situation which incorporates authentic and anticipated market estimate in terms of esteem and volume, technological advancement, macroeconomic and governing factors in the market. The report provides detailed statistics and strategies of the best key players in the industry. The report additionally gives a broad study of the distinctive market sections and areas.

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Some of the leading key players profiled in this study:Cordlife, ViaCord (A Subsidiary of PerkinElmer), Cryo-Save AG, StemCyte India Therapeutics Pvt. Ltd., Cryo-Cell International, Inc., SMART CELLS PLUS, Vita 34, LifeCell, Global Cord Blood Corporation, CBR Systems, Inc

What the report features:-

Global analysis of Stem Cell Banking market from 2017 2027 illustrating the progression of the market.

Forecast and analysis of Stem Cell Bankingmarket by Dosage, Route of Administration and Application from 2017 2027

Forecast and analysis of Stem Cell Banking market in five major regions, namely; North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South & Central America

The market of stem cell banking is anticipated to grow with a significant rate in the coming years, owing to factors such as, development of novel technologies for stem cell preservation and processing, and storage; growing awareness on the potential of stem cells for various therapeutic conditions. Moreover, increasing investments in stem cell research is also expected to propel the growth of the stem cell banking market across the globe. On other hand rising burden of major diseases and emerging economies are expected to offer significant growth opportunities for the players operating in stem cell banking market.

The Global Stem Cell Banking Market is characterized by the presence of a large number of global, regional, and local players and is highly-competitive. These international players are increasingly focusing on expanding their geographical presence and they have huge production facilities located across the world. Several vendors are increasingly competing against each other based on factors such as innovations, price, and quality of the product. Vendors with better financial and technological resources can withstand changes in different market conditions when compared to their competitors.

The various factors supporting the markets growth and those posing threat are studied in detail in this report. Additionally, the market study segments the Global Stem Cell Banking Market based on end-users, verticals, and size. In these sections, it shields various factors impelling the markets trajectory across the segments. Furthermore, it recognizes the most lucrative of them all to help investors take the well-informed decision.

The Global Stem Cell Banking Market Analysis to 2025 is a specialized and in-depth study of the biotechnology industry with a focus on the global market trend. The stem cell banking market report aims to provide an overview of global stem cell banking market with detailed market segmentation by source, service type, application, and geography. The global stem cell banking market is expected to witness high growth during the forecast period. The stem cell banking market report provides key statistics on the market status of the leading market players and offers key trends and opportunities in the market.

Relating to the latest hierarchy in the global Stem Cell Banking market, the report summaries some of the crucial players operative in the market. Discriminating information about the significant players including their revenue, business segmentation, product portfolio, and financial overview has been integrated in the report. Latest improvements in the industry have been taken into concern while anticipating the future perspective of the market. The report also exemplifies the various marketing channels prevailing in the global market and conveys information about few of the critical distributors functioning in the market. The report assists as a helpful guide for the new as well as prevailing players in the market.

Market Segmentation:

The global stem cell banking market is segmented on the basis of source, service type, and application. The source segment includes, placental stem cells (PSCS), dental pulp-derived stem cells (DPSCS), bone marrow-derived stem cells (BMSCS), adipose tissue-derived stem cells (ADSCS), human embryo-derived stem cells (HESCS), and other stem cell sources. Based on service type the market is segmented into, sample processing, sample analysis, sample preservation and storage, sample collection and transportation. Based on application, the market is segmented as, clinical applications, research applications, and personalized banking applications.

This report includes several arrangements, definitions, the chain assembly of the industry in one piece, and the various uses for the global market. This section also integrates an all-inclusive analysis of the different enlargement plans and government strategies that influence the market, its cost assemblies and industrialized processes. The second subdivision of the report includes analytics on the Global Stem Cell Banking Market based on its size in terms of value and volume.

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The most crucial key factors in the businesses have been elaborated to get ample and accurate data of market dynamics. Rising needs and popularity of Stem Cell Banking Market sector is driving the flow of the market towards progress. In addition to this, it lists the factors which are restraining the growth of the market.

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Stem Cell Banking Market Anticipated to Grow at a Significant Pace by 2028 - Weekly Spy

How isleukaemia treated?

The treatment of leukaemia varies depending on the patient and type of leukaemia they have.

Acute leukaemia (fast developing) is usually curable with standard treatments, such as chemotherapy.

Chronic leukaemia (slow developing), is often incurablebut treatable. For CLL (a form of chronic leukaemia) some patients are not given treatmentstraight away;however if they do require treatment it will often involve chemotherapy.

The main treatments for leukaemia are:

Chemotherapy: This treatment involves theuse ofdrugs.Chemotherapy drugs either kill cancerous cells or stop them from dividing; they can also kill normal blood cells as a side effect.The type of leukaemia you have will depend on the amount and strength of chemotherapy you are offered, along with other factors such as your age and fitness.

Radiation therapy:Similar to chemotherapy, radiation therapy can be used to destroy the cancerous cells but using radiation waves rather than drugs.Again, the type of leukaemia you have will determine what treatment you're offered. External beam radiation therapy (EBRT) is often used for CLL.It is a fast, painless procedure which usually lasts just a few minutes.

Targeted therapy:Drugs are used to block the growth of cancer cells by disturbing specific molecules in the cells. Targeted therapy can also kill cancer cells by stimulating the patient's immune system to recognise the cells as a threat and consequently kill them.

Biological therapy:This treatment does not target the cancer cells directly, but instead helps to stimulate the body's immune system to act against the cancer. It is also often referred to as "immunotherapy". It is often usedfor patients with CML.

Stem cell or bone marrow transplant: Transplants for stem cells or bone marrow are commonly carried out for patients withacute leukaemia,if chemotherapy does not prove effective.By undergoing a stem cell or bone marrow transplant it can help replenish the healthy bone marrow in patients, and stimulate new growth that restores the immune system. It is usually given to younger, or more healthy patients.

Leukaemia Care, which provides support to individuals and families affected by blood cancer, is one ofthree charities supported by this years Telegraph Christmas Charity Appeal. Our two other charities are Wooden Spoon, which works with Britains rugby community to raise money for sick, disabled and disadvantaged children; and The Silver Line, a 24-hour helpline and support service for lonely elderly people. To make a donation, visit telegraph.co.uk/charity or call 0151 284 1927

See the article here:
Leukaemia: what is it, how to spot the warning signs and who is at risk? - The Telegraph

Stem cells from the patients body when isolated and administered at an appropriate time and at the right place, with the right dose, is expected to help the patient in various ways

Stem Cells In The Body

All humans are born and develop from a small tiny structure called an egg. The cells in the egg have a tremendous potential to develop, multiply and form different cells that are functional in the body. These cells are called mother cells or in scientific terms, they are called stem cells. And all human beings have these stem cells preserved in the body. It is these cells that help us in every day wear and tear and also for tissue repair.

The Body Can Heal Itself

Most of the cells in our body have a definite lifespan that need to be replaced by new cells. The stem cell reserves in the body make up for this and it is done without our knowledge! In fact, any cut or injury, external or internal is healed by the bodys innate mechanism. Our intelligent body recognises the signal of injury and recruits the required stem cells. These stem cells transform themselves into the cells that are required for the repair of the injury and it is always many types of cells in various permutations and combinations.

Where Stem Cells Reside

Bone marrow can be considered as the manufacturing unit of stem cells as it is continuously making blood cells and keeps our circulatory system working perfect all the time. Circulating blood is another source of stem cells, because it works as a courier, carrying cells and other essential enzymes, hormones from one organ to the other in the body. The body converts all the extra material into fat which gets accumulated around the belly. This fatty tissue works like a fixed deposit of stem cells.

Stem cells either from the donor (allogenic) or from the patient (autologous) are being used for more than 50 years and especially for treatment. Blood cancers and other blood-related diseases can be cured using a perfect matched donor stem cells obtained from bone marrow. Patients suffering from organ cancers like breast cancer etc. are given autologous stem cells as a supportive treatment along with chemotherapy and/or radiation.

Protocols for these treatments are standardised globally and considered as standard-of-care. In recent years, umbilical cord blood derived stem cells are being used as an alternative to bone marrow, especially in the paediatric age group. People fall victim to numerous degenerative diseases which occur, as the repairing stem cell system from the body fails slowly with age. Stem cells from the patients body when isolated and administered at an appropriate time and at the right place, with the right dose, is expected to help the patient in various ways. It may also replace, rejuvenate or restore the damaged tissues.

Our body carnes its own repairing kit in the form of stem cells and the body tries its level best to make use of these stem cells to ward off diseases. However, it is possible that with age, the bodys power to recruit and make use of the stem cells diminishes slowly. This is when dreadful degenerative diseases like diabetes, arthritis, Parkinsons disease and heart problems, set in. Heres what the clinical applications of regenerative medicine have found novel mechanisms of:

It is increasingly observed that this kind of autologous therapy takes care of the root cause of disease and offers benefits to patients to whom there is no further solution in other modalities of treatment.

Since each tissue and organ of our body is made up of cells that are derived from the egg cell, any disease which is due to derangement or degeneration of cells can be cured using autologous cellular therapy. And though the list can be endless, here are some examples where there have been very promising results:

Read the original post:
How Stem Cells Can Heal The Body - Version Weekly

This level of disease stabilization has not been observed to this date in approved or investigational ALS therapies.

- Mr. Chaim Lebovits, CEO, Brainstorm Cell Therapeutics

In May of this year, I published an article on Brainstorm Cell Therapeutics (BCLI). This small company is developing a mesenchymal stem cell product called NurOwn, which is in late phase 3 trials targeting amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease. My article was bearish, deploring not only the company's cash position but also phase 2 trial data. The article can be read here.

That article received a lot of critical comments from the ALS community. That made me realize that a fair overview of the issues could be best addressed by going through the comments, as well as my own coverage, and by asking BCLI management, specifically its CEO, Chaim Lebovits, to clarify some of these issues. So, that's what I did. I emailed a set of 11 questions to Mr. Lebovits, and he was kind enough to respond to them in great detail. The entire interview, sans any edits, is available to Total Pharma Tracker members.

Mr. Lebovits has been with BCLI for well over 12 years, joining in 2007 as president and also becoming the CEO in 2015. He has helped develop NurOwn through its preclinical stage to its current stage and is, therefore, just the right person to talk to if we want to understand NurOwn and BCLI.

I began by asking him to locate NurOwn in the ALS therapy space and where it stands with respect to competitors. What's its mechanism of action, and how does that MOA distinguish it from the competition?

Mr. Lebovits said that there are "currently 4 products active in phase 3 ALS clinical trials (Brainstorm (NurOwn, autologous MSC-NTF cells secreting neurotrophic factors), Orion (levosimendan, muscle troponin calcium sensitizer), Orphazyme (arimochlomol, heat shock protein enhancer), and Biogen (SOD1, antisense oligonucleotide)." Top-line data from these ALS phase 3 trials is expected in 2020 (Q4 2020 for Brainstorm) and Orion, 2021 (Orphazyme), and 2022 (Biogen). He discussed a number of earlier-stage compounds as well as various stem cell therapies. He said that what distinguishes NurOwn among ALS therapies is that it "confers both neuroprotection and immunomodulation by delivering neuronal survival factors and immune regulatory molecules, including microRNA directly to the CNS compartment at or near the site of disease, and therefore directly addresses two important ALS disease mechanisms."

Among stem cell therapies, Mr. Lebovits said that NurOwn distinguishes itself by being autologous and because it can produce high levels of neurotrophic factors. Moreover, unlike most stem cell competitors, it's delivered directly into the spinal fluid through bimonthly lumbar punctures, unlike others that need an invasive surgical procedure "that carries considerable morbidity."

This feature it shares with a competing product from Corestem. However, it's differentiated from Corestem because "NurOwn is more convenient than the Corestem product as a single bone marrow cell harvest due to validated cryopreservation, whereas the Corestem product requires repeat bone-marrow aspiration for each treatment."

My next question was a technical question about pharmacoresistance. I wanted to know how NurOwn is managing to cross the blood-spinal cord barrier despite the strong pharmacoresistance (body's resistance to drugs) seen in ALS, specifically for disease-modifying neurotrophic factors. What was it about NurOwn's delivery mechanism that the company thinks is overcoming this natural resistance. So I asked: "Talking about MOA, pharmacoresistance is a disease driving mechanism in ALS. Can you discuss NurOwn's delivery mechanism vis-a-vis the inability of neurotrophic factors to effectively cross the blood-brain barrier, or, specifically, the blood-spinal cord barrier (BSCB)? Please correlate that discussion regarding the observed increase in CSF NTFs post-treatment as seen in the phase 2 trial."

Mr. Lebovits explained this with great clarity - for his entire response, take a look at the complete interview. Broadly, what he said was that NurOwn, being delivered through lumbar puncture directly into the spinal fluid, has an advantage. Moreover, the cells secrete neuronal survival factors as well as molecules that regulate the immune system, so that they are able to survive and overcome the pharmacoresistance. Systemically administered NTFs are unable to do that.

As he said, "In the phase 2 trial, CSF biomarkers obtained just prior to treatment and two weeks afterward demonstrated that MSC-NTF cell-secreted neurotrophic factors were significantly increased post-treatment and correlated with the reduction in inflammatory biomarkers, consistent with the proposed mechanism of action."

My third and fourth questions related to aspects of the phase 2 study. One, comparison of safety and efficacy data with competitors, and two, the relevance of the reported caspase-3 reduction of 60% in responders versus 30% in non-responders.

Mr. Lebovits said that although the phase 2 study was not powered for efficacy, it exhibited a "level of disease stabilization (that) has not been observed to this date in approved or investigational ALS therapies." About the ongoing phase 3 study, he said the following:

Those who read my original article will recall I was particularly puzzled by the increased occurrence of serious adverse events in active-treatment groups than in placebo groups. 8/36 or 22.2% patients in the treatment arm had an SAE compared to only one out of 12 placebo patients, or 8.3%. Most SAEs were related to the progression of the underlying ALS, most commonly dysphagia. No SAEs were related to study treatment. So I asked Mr. Lebovits how this data could be interpreted in the most positive way.

According to him, this decline was not an effect of treatment itself and simply indicated the need for repeat dosing in this patient group. His exact response was as follows:

The MSC-NTF treated group had a slightly more rapid rate of decline compared to the placebo group in the three-month run-in period and most ALS disease progression in the treated group was seen toward the end of the clinical trial, long after a single transplantation. In fact, the bulbar subscale, that includes assessment of swallowing, was the subscale most improved after MSC-NTF treatment in rapid progressors, suggesting that the late decline in motor function was not an adverse effect of treatment per se. Hence the need for repeated dosing.

Last week, the DSMB recommended continuation of the phase 3 trial without any modification. This was major good news, so we asked him about this. Mr. Lebovits said that this was a second interim safety review, and there were no significant safety concerns. Therefore, the DSMB recommended no modification in protocol and no other interim analysis is planned. Phase 3 data will be available by mid-2020 according to this interviewer's reading of the press release.

Now we moved on to another critical aspect of our analysis - funds, or rather, the lack of it. Since this is an important issue, here's the exact exchange we had.

Dr. Ashok Dutta: How does the company plan to fund its operations through the next couple years until the lead development candidate is approved and commercialized? Given the weak financial position, does Brainstorm see the possibility for ATM operations, or thinks about selling rights in regions like China, Japan or Europe to increase the financial condition?

CEO Chaim Lebovits: As you are aware we do receive proceeds from the hospital exemption pathway and also receive grant funding from CIRM and IIA. These avenues have allowed to fund and continue with our trials over the years with non-dilutive financing. From a business standpoint as our ALS phase 3 trial is now fully enrolled, the management team continues to hold high level conversation with some of the leading global pharmaceutical and biotechnology companies. We are actively engaged in strategic partnering and collaboration discussions and although we cannot disclose the details of our conversations due to NDAs we signed with them... we are exploring several opportunities with key interested parties to advance the opportunities for NurOwn development and commercialization. As you have rightly pointed out, we have a $20mm ATM facility in place with Raymond James. We may activate the ATM as required and raise up to $20mm by selling our stock "at the market" only if the prices are attractive to us. So far as of end of Q3'19, we have not activated the ATM. If the need arises and the prices are attractive to us, we may employ this tool to raise capital.

This is reassuring that the company intends to focus on non-dilutive financing. The ATM facility, coupled with the grants, should ideally see them through the approval phase. We still wonder how they will manage marketing and sales. Perhaps those commercialization NDAs they have signed will help.

Next, we discussed market potential and a question about a recent patent grant. The CEO's detailed responses can be found in the complete interview material.

The strong involvement of the ALS community impressed us previously, so we now asked the CEO about the recent roundtable convention they had with ALS advocacy groups. Since this will be important for the ALS community as a whole, here's Mr. Lebovits' entire response on the question:

Finally, we asked him what we ask everyone: Give us three simple and straightforward reasons why investors would be interested. Here's what he said:

Thanks to the ALS community for inspiring us to conduct this interview, and to Mr. Chaim Lebovits, CEO of Brainstorm Cell Therapeutics, for answering our questions.

Thanks for reading. At the Total Pharma Tracker, we interview management of important small biotech doing disruptive work in healthcare. Our members are given exclusive access to these interviews, which helps them with additional primary resource in doing DD on their investments. Sometimes, extracts from these interviews may be published for everyone; but TPT members always get the exclusive view.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: General Disclaimer - This is to confirm that Avisol Capital Partners has neither requested, nor been offered, any monetary compensation for conducting this interview, by any party other than Seeking Alpha.

Also to be noted, this was an emailed questionnaire, and certain editorial material is present in this version, which may or may not reflect BCLI or its CEO's position on the issues discussed.

View original post here:
Interview With Chaim Lebovits, CEO Of Brainstorm Cell Therapeutics - Seeking Alpha

MULTIPLE sclerosis campaigners have hailed a huge step forward for patients in Scotland after a stem cell therapy was recommended for use on the NHS for the first time.

Haematopoietic stem cell transplantation (HSCT) has been described as a game-changer for MS after an international clinical trial showed that it could reboot patients immune systems and halt the progress of the disease.

Some patients who had been in wheelchairs prior to treatment said their condition improved so dramatically it was like they had never been diagnosed with MS.

READ MORE: Scots MS patients 'missing out' on pioneering stem cell treatment available in England

The Scottish Health Technologies Group (SHTG) said there is now sufficient evidence for it to recommend making HSCT available on the NHS in Scotland to MS patients who have the relapsing-remitting form of the disease, and who were not responding to drug treatments.

Iain Robertson, chairman of the SHTG, said: Our committee members were able to advise that this treatment should be considered for those with this particular type of MS who have not responded to treatment with disease-modifying therapies.

We hope that our advice will be of use in helping decide the best course of treatment for these patients.

The SHTG also stressed that patients must be made aware of the demands, risks and uncertainties of the treatment, which uses chemotherapy to wipe out patients' 'faulty' immune systems before replenishing it with a transplant of stem cells harvested from their own bone marrow.

It puts patients at high risk from infections, which can be fatal, but the theory is that the treatment works by enabling patients to 'reset' their immune system to stop it attacking the central nervous system as is the case in MS.

READ MORE: Anger of Scots MS patients travelling abroad for stem cell therapy available to some on NHS England

HSCT is not considered an effective treatment for patients with the progressive form of MS, however, as stem cells cannot regrow nerves or repair damaged myelin - the protective sheath which coats nerves.

It will also be unavailable to patients with relapsing-remitting MS who no longer show signs of inflammation on an MRI brain scan.

Scotland has one of the highest rates of MS in the world, but until now Scottish patients seeking HSCT have had to travel overseas to Mexico, Russia and Israel and bankroll their own private treatment at a cost of around 40-60,000.

It has also been available privately in London since 2017, but with a 100,000 price tag.

A small number of MS patients in England have been able to access the treatment on the NHS, however, because there are clinical trials into HSCT taking place at NHS hospitals in Sheffield and London.

Morna Simpkins, director of MS Society Scotland, said: The decision from SHTG to approve HSCT for the treatment of MS is good news and could help in the development of a clear pathway, for people who could potentially benefit, to access it.

We will push to ensure that this decision leads to real change for people with MS by continuing to engage with other groups to offer the treatments, including HSCT, which are right for them.

READ MORE: Stem cells help mother with MS make 'remarkable' recovery

The SHTG said eligible patients must have equal access to the procedures regardless of where they live, but it is unlikely all health boards will be able to provide it.

The MS Society wants a centre, or centres, of excellence set up where patients from across Scotland can be referred.

Lucy Clarke from the Scottish HSCT Network said the recommendation was "a huge step forward" for people in Scotland living with MS.

Ms Clarke underwent HSCT in Russia and credits it with substantially reversing her disability.

She added: This important decision supports HSCT as a treatment option where other treatments have failed. We will continue to push so that this treatment is available to people in Scotland who need it.

A Scottish Government spokeswoman said: We are grateful to the Scottish Health Technologies Group for this important work.

"NHS Boards are expected to consider their advice on technologies in the planning and provision of its services and clinicians are expected to follow their professional judgement, working within the management structure of their Board.

We will work closely with MS Society Scotland, other third sector bodies and the clinical community to consider what the Technologies Groups findings means for provision in Scotland, including the information that needs to be available to people about eligibility and risks.

Here is the original post:
Stem cell therapy approved for MS patients in Scotland - HeraldScotland

In September end, a good news greeted the biomedical world when a team led by Takanori Takebe at Cincinnati Children's Hospital Medical Center succeeded at growing a connected set of three organs: the liver, pancreas and biliary ducts, in the lab, from human stem cells. The findings were published in journal Nature.

While human organoids already provide a sophisticated tool for research, the connected set of three organs, for the first time, allow scientists to study how human tissues work in concert. This was dubbed as a significant step forward.

In October, another news came. At the annual meeting of the Society for Neuroscience, researchers said that brain cell clusters prepared in the lab- a type of organoid- show abnormal behaviour as compared to the normal brain cells.

They said that the cells in these clumps had ambiguous identities and made more stress molecules than cells taken directly from human brains. However, these abnormalities were found to be alleviated a little bit when the implanted into a more hospitable environment - a mouses brain.

What are organoids?

With the available technology, scientists can grow a group of cells in laboratories into three-dimensional, miniature structures that mimic the cell arrangement of a fully-grown organ.

This is done using stem cells.

Stem cells are special human cells that have the ability to develop into many different cell types, from muscle cells to brain cells.

The embryonic stem cells that are derived from unused embryos (These are created from an in vitro fertilization procedure and used for scientific research) are pluripotent, meaning, they can turn into any type of cell.

On the other hand are adult stem cells. They are derived from fully developed tissues, like the brain, skin, and bone marrow. These cells often have capability of turning into only certain types of cells. For example, a stem cell derived from the liver will only generate more liver cells.

However, the adult stem cells can be manipualted in the laboratory to act like embryonic stem cells. These are called induced pluripotent stem cells. (The technique was developed in 2006). However, scientists are yet to find adult pluripotent stem cells that can develop every kind of cell and tissue.

When scientists create right environment in the laboratory for them, these stem cells follow their own genetic instructions to develop into tiny structures that resemble miniature organs composed of many cell types.

Using these, researchers have been able to produce organoids that resemble the brain, kidney, lung, intestine, stomach, and liver etc.

For example, in the three-organ research mentioned above, Dr Takebe started with stem cells from human skin cells and then guiding and prodding those stem cells to form two very early-stage "spheroids" of cells loosely termed the foregut and the midgut (In human embryos, these form late in the first month of gestation. Over time, they merge and morph into the organs that constitute the digestive tract).

The spheroids were first placed next to each other in a lab dish suspended in a gel used to support organoid growth, then placed on top of a thin membrane that covered a carefully mixed batch of growth medium.

From this point on, the cells knew what to do, and 70 days later, the mini organoids began processing bile acids as if they were digesting and filtering food.

Why are organoids important?

The technique to develop organoids was named by The Scientist as one of the biggest scientific advancements of 2013.

Organoids are an excellent tools to study biological processes like uptake of nutrients, drug transport, secretion of hormones and enzymes etc. This way, diseases related to malabsorption of nutrients, and metabolism-related diseases like obesity, diabetes, insulin resistance can be studied at the cellular-level.

Recently, scientists at the at Memorial Sloan Kettering created a tumor organoid to develop a more accurate rectal cancer model.

In the case of the human brain, organoids opens a window to understand some of the most complicated and hidden aspects of our own biology. They can be used to study neuropsychiatric or neurodevelopmental diseases like schizophrenia or autism spectrum disorder, which are uniquely human diseases that affect the whole human genome.

Organoids also provide a window into how cells interact with each other and their environment. They can be used to create cellular models of human disease, which can be studied in the laboratory to better understand the causes of disease and identify possible treatments. The effects of different drugs and be tested.

Scientists have even used gene editing techniques (CRISPR-Cas9) on the stem cells to to introduce targeted mutations in genes corresponding to two different kidney diseases. When these modified pluripotent cells grew into human kidney organoids, they exhibited the diseases.

Using such organoids relieves the scientific community from experimenting on human and animal subjects. Also, certain treatments that would be unethical to administer on the latter, can be tested on the organoids.

With organoids, researchers can produce a limitless supply of tissue from each patient. This will also be extremely useful for the study of rare diseases, where the number of patients on which to conduct research and test treatments is limited.

Organoids are also being used to develop personalised and precision medicine.

For example, it was found that repairing the CFTR protein could give relief to a patient suffering from non-cystic fibrosis, an inherited disease caused due to a gene mutation. Using the Intestinal organoids grown from a patients stem cells, the doctors could quantify the patients response to the CFTR modulating therapy.

Organoids can have significant therapeutic applications. For example, pluripotent stem cells derived from a diabetes patient could be transformed into insulin-producing beta-like cells.

Organoids also offer an incredible opportunity to study developmental biology. Using them, for example, we can learn more about how organs are formed in embryonic stages and associated disorders.

Visit link:
Growing Human Organs In A Lab: As Scientists Develop Pathbreaking Three-Organ System, Heres All You Need To Know - Swarajya

WHEN Laura Farmer-Maia's daughter suddenly became clingy and unhappy, she initially brushed it off as nothing but "a phase".

And given little Beatriz was just three-years-old, the last thing to cross her mind was cancer.

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Yet, months after doctors repeatedly dismissed the symptoms as clinginess, the diagnosis was confirmed - Beatriz had an aggressive childhood cancer known as a neuroblastoma.

The horrifying news came after Beatriz's father Tiago Maia refused to leave the hospital after discovering the potential diagnosis himself on Google.

Shocked, Laura, 39, and Tiago, 40, are now urging all parents to be vigilant and check their kids for signs of the disease.

The mum, who works in advertising, first suspected something was wrong last July, when Beatriz's behaviour dramatically changed.

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She said: "Before she was diagnosed, Beatriz was quite naughty but when she reached two, she suddenly became clingy and picky with her food, and had a fever all the time.

"We took her to the GP who believed it was a virus and after recurrent visits they gave her some antibiotics to cover for a potential bacterial cause, which didnt have any effect.

"Beatriz started to complain that her legs hurt so we took her to A&E, where they did some more tests and still said it might be a virus.

"Its hard to get a diagnosis right when a child is too young to explain how theyre feeling, but in the back of our minds we knew it was something bad.

"We want to spread awareness of the difficulty of diagnosing cancer in young children - if your child doesnt seem right, you should push for further tests."

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Tiago pushed the GP for more tests, and blood tests showed something was wrong, so Beatriz was sent urgently to hospital.

It was there medics finally discovered a lump above her kidney and diagnosed her with neuroblastoma in September last year.

The cancer is aggressive and has a 40 per cent chance of long-term survival.

Everything moved so quickly and we all felt frightened as they carried out the tests

Tiago, originally from Portugal, added: "At the hospital, they twice said it was likely to be a virus and I refused to leave until I saw a specialist.

"I waited for three hours until a more senior doctor was free, and then Beatriz was examined by different specialists who admitted her to do all kinds of tests and observations including X-rays and ultrasounds - it was the last one that confirmed there was a lump.

"When my fears from Google turned out to be true, it was very strange because even though my life had just flipped upside down, I was almost relieved to be right - it was weird and confusing."

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Beatriz was referred straight to Great Ormond Street Hospital in London, where they carried out further tests including scans, blood tests and biopsies.

She began chemotherapy just a week after being diagnosed and underwent eight gruelling rounds of chemo over the next 18 months.

Doctors then carried out a stem cell transplant to regenerate bone marrow destroyed by high dose chemo, which meant Beatriz couldnt leave the hospital for eight weeks.

Tiago, a design director, said: "I was quite scared when Beatriz was diagnosed because my mum and dad had only recently died from cancer I thought of the worst.

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"Everything moved so quickly and we all felt frightened as they carried out the tests.

"Doctors found that the cancer had spread across her body, so she began chemotherapy just weeks after being diagnosed.

"We were told the treatment would last 18 months which was a massive shock to us.

"A week after Beatriz started chemotherapy she massively improved, but it was tricky being in hospital at first.

"Now, she still has periods of discomfort but sometimes shes happy to be in hospital because she has toys and people to come and play with her."

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Laura added: "The stem cell transplant was a hard time for us all because we had to spend a lot of time apart from our other daughter, Clara, six.

"Beatriz was diagnosed in Claras first week of school and it was difficult because that was supposed to be an exciting time for her."

After more scans and hopes of an all-clear, doctors found more metastatic growths still remaining in Beatrizs head, which meant that the cancer hadnt fully cleared up and she had relapsed.

The brave youngster is now undergoing immunotherapy and is due to start a six month medical trial on the NHS at Great Ormond Street Hospital, called the Beacon Trial.

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What is neuroblastoma?

Neuroblastoma is a type of cancer that most commonly afflicts babies and young children.

The disease develops from special nerve cells, known as neuroblasts, which get left behind from the child's development in the womb.

It mostly begins in the sufferer's adrenal glands located above the kidneys but can occur in the nerve tissue that runs along the spinal cord in the neck, chest, abdomen or pelvis.

The vicious illness can then spread to other organs like the bone, bone marrow, lymph nodes and skin.

Neuroblastoma afflicts around 100 children a year in the UK but the cause of the disease is still not known.

Its symptoms can include:

It is uncertain whether the trial will work and, even if Beatriz goes into remission, relapse rates are high but her parents are determined to do everything they can to stop the cancer from returning.

Laura and Tiago are now trying to raise 200,000 to help get their daughter into remission or to keep the cancer away if her treatment goes well.

The money is hoped to go towards further treatment, or if Beatriz gets the all-clear, a special vaccine in New York which helps keep the disease away.

Laura said: "After the stem cell transplant, the end was almost in sight but then she relapsed.

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"Were afraid that the cancer will get worse and worse and want to raise money to help get her into remission the ideal outcome is that the trial works and clears the disease.

"Luckily, compared to other two-year-olds, Beatriz has suffered less side effects with treatment and despite losing her curly hair shes powering through."

You can donate on Beatriz's JustGiving page here.

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Here is the original post:
Docs said our toddler was just clingy but we learned the truth on Google it was cancer - The Sun

PEORIA, Ill. November is National Bone Marrow Awareness Month.

The Greater Peoria Family YMCA is hoping you take the time to see if youre a match and it comes with a local tie.

Marsha Krone has been a teacher in the Peoria area for over 30 years.

Despite being diagnosed with a rare bone marrow cancer in 2016, shes yet to find a match.

You never know when youre going to be the link to saving someones life and its very simple. You come out, fill out a little paperwork online. You swab the inside of your cheek. You put it back on the cart and its sent back.

You can join the donor registry for bone marrow & blood stem cells this Wednesday, Nov. 6 from 3 p.m. 7 p.m.

It would be awesome to find a match for me, but if I dont, there are other people that their lives can be saved through that, said Krone . Their joy would certainly not be pain for me. I would be happy for them to find their cure and what a gift to be able to give someone.

The YMCAs is located 7000 North Fleming Lane, Peoria, IL 61614. The registry will be held in the upstairs lounge.

See the original post:
You can be the match, help local people in need Wednesday - CIProud.com

The proposal was nothing fancy. Shawn Russell arrived at the pub, placed a pint on the table in front of Sarah Hodgetts and asked her to marry him.

It was out of the blue, with no ring, nothing. And I thought: Yeah, thats a really good idea, we should just get married, says Sarah. Id been in love with him for ages.

Their story began seven years earlier, in 2009, also in a pub in north London. He was sitting in his flat cap, good Yorkshireman that he was, and was reading the sports pages. I had just got off the Tube after work. From my perspective, it was love at first sight, says Sarah.

But dating wasnt easy. Shawn, a boarding school-educated boy from an Army family, who had lost his mother to leukaemia when he was two, worked as a picture editor at The Telegraph. Although Sarah was down the road in Westminster, where she worked as a civil servant (and still does), their hours were long and their schedules largely incompatible. They managed just six months initially.

It was a disaster trying to date, so we ended up with a firework display of an argument and decided there was no way we could, says Sarah over coffee near her office.

Their split didnt last, however; they had far too much in common. Besides his ridiculous sense of humour, Shawn was so into news and politics, and I worked in politics, so we couldnt not communicate. We realised we were incredibly good friends, says Sarah. Plus, she adds with a smile, he was a very attractive, 6ft 4in blue-eyed man who I was completely smitten with.

Over time, they got back together in a non-committal way, sharing weekends when their busy lives permitted. It was during this period, three-and-a-half years ago, that Shawn, then 44, proposed. The following week, he moved in with Sarah, then 41, and her son Eddy, 10, from a previous relationship, in Kings Cross, and, like all newly engaged couples, they started making plans for their future. A pair of recovering workaholics, they were going to transform their lives.

Originally posted here:
I lost my fianc to leukaemia, but in my dreams hes just working the night shift - Telegraph.co.uk

SAN FRANCISCO--(BUSINESS WIRE)--

Imago BioSciences, Inc., a clinical-stage biotechnology company developing innovative treatments for malignant and life-threatening diseases of the bone marrow, today announced the appointment of James D. Watson as Chief Business Officer.

James has a long history of success in biotech financing, business development, and commercial planning. That experience will guide the strategic direction and growth of Imago, said Hugh Young Rienhoff, Jr., M.D., Chief Executive Officer of Imago BioSciences. His experience in corporate development and financings will help ensure the advancement of bomedemstat (IMG-7289) through clinical development in myelofibrosis and other indications.

Mr. Watson most recently served as Chief Business Officer at Sigilon Therapeutics where he closed a $473 million strategic partnership with Eli Lilly for a treatment for Type 1 diabetes. Prior to Sigilon, Mr. Watson was Chief Business Officer for Alvine Pharmaceuticals and led strategy, corporate development, new product planning, and finance during which he closed a transaction giving AbbVie the right to acquire Alvine for $345 million. Previously, Mr. Watson was CEO of a San Francisco-based, boutique investment bank focused on mergers, acquisitions, partnering, and raising capital for life science companies.

I am excited to join Imago BioSciences at this important stage of growth, helping the company realize its full potential. Bomedemstat has great promise for the day-to-day management of myelofibrosis and it offers the additional possibility of altering the course of this disease. Furthermore, its broader myeloproliferative neoplasm platform and expertise in life-threatening diseases of the bone marrow represent an opportunity to address areas of high unmet clinical need and to build a valuable company. said Mr. Watson.

About Bomedemstat (IMG-7289)

Bomedemstat is a small molecule discovered by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme essential for production and normal function of megakaryocytes and for self-renewal of malignant hematopoietic stem or progenitor cells. Megakaryocytes are the primary producer of growth factors and cytokines that drive myelofibrosis pathogenesis.

In non-clinical studies, bomedemstat demonstrated robust in vivo efficacy as a single agent and in combination with other therapeutics across a range of myeloid malignancy models including the myeloproliferative neoplasms encompassing myelofibrosis, essential thrombocythemia and polycythemia vera. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to bomedemstat for the treatment of myelofibrosis which is currently being studied in an international Phase 2b study. Additional clinical studies in hematologic disorders will begin in 2020.

About Imago BioSciences

Imago BioSciences is a clinical-stage, private therapeutics company focused on malignant and life-threatening diseases of the bone marrow. The initial clinical focus is on myeloproliferative neoplasm (MPN) disorders including myelofibrosis, essential thrombocythemia and polycythemia vera. Investors in Imago include a fund managed by Blackstone Life Sciences, Frazier Healthcare Partners, Omega Funds, Amgen Ventures, MRL Ventures Fund, HighLight Capital, Pharmaron, Greenspring Associates and Xeraya Capital.

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Imago BioSciences Further Expands Executive Team with Appointment of James D. Watson as Chief Business Officer - Yahoo Finance

Aplastic anemia is a medical condition that damages stem cells in a person's bone marrow. These cells are responsible for making red blood cells, white blood cells, and platelets, which are vital to human health.

Doctors believe various conditions can cause aplastic anemia, while the disease itself ranges in severity from mild to life threatening.

Medical advancements mean that aplastic anemia is more treatable than ever. In this article, learn more about this rare medical disorder.

When a person has aplastic anemia, their bone marrow does not create the blood cells it needs. This causes them to feel ill and increases their risk of getting infections.

Doctors also call aplastic anemia bone marrow failure.

Doctors do not know exactly how many people in the United States have aplastic anemia.

According to the National Organization for Rare Disorders (NORD), doctors diagnose approximately 500 to 1,000 cases every year. It is most common in older children, teenagers, and young adults.

Researchers believe that most cases of aplastic anemia are due to the immune system attacking healthy bone marrow cells, according to NORD.

Doctors have also identified some of the possible causes of this immune system response, including:

However, doctors usually cannot pinpoint the underlying cause in most aplastic anemia cases.

When the cause is unknown, doctors refer to the condition as idiopathic aplastic anemia.

Symptoms of aplastic anemia include:

These symptoms may be severe. Some people may have heart-related symptoms, such as chest pain.

A doctor will start by asking about a person's symptoms and their medical history.

They will usually use a blood test known as a complete blood count (CBC) to evaluate a person's red blood cells, white blood cells, and platelets. If all three of these components are low, a person has pancytopenia.

A doctor may also recommend taking a sample of bone marrow, which comes from a person's pelvis or hip.

A laboratory technician will examine the bone marrow. If a person has aplastic anemia, the bone marrow will not have typical stem cells.

Aplastic anemia can also have similar symptoms as other medical conditions, such as myelodysplastic syndrome and paroxysmal nocturnal hemoglobinuria. A doctor will want to rule out these conditions.

Sometimes, a person with other medical conditions can develop aplastic anemia. These conditions include:

If a person has these conditions, a doctor will recognize that they are more likely to get aplastic anemia.

Doctors usually have two goals when treating aplastic anemia. The first is to reduce the person's symptoms, and the second is to stimulate the bone marrow to create new blood cells.

People with aplastic anemia can receive blood and platelet transfusions to correct low blood counts.

A doctor may also prescribe antibiotics as a person needs white blood cells to fight infections. Ideally, these drugs will prevent infections until a person can build more new white blood cells.

Doctors usually recommend a bone marrow transplant to stimulate new cell growth in the long term.

For this, a doctor may first prescribe chemotherapy medications to kill off abnormal bone marrow cells that are affecting a person's overall bone marrow function.

Next, a doctor performs a bone marrow transplant by injecting the bone marrow into a patient's body.

Ideally, the individual will receive bone marrow from a close family member. However, even a sibling donor is only a match in 2030% of cases.

People can also receive bone marrow from someone who is not related to them if doctors can find a compatible donor.

Some people cannot tolerate bone marrow transplants, especially older adults, and those having difficulty recovering from chemotherapy. Others may not be able to find a donor that matches their bone marrow. In these instances, a doctor can prescribe immunosuppressive therapy.

Immunosuppressive medicines suppress the immune system, which ideally stops it from attacking healthy bone marrow cells. Examples of these medications include antithymocyte globulin (ATG) and cyclosporine.

According to NORD, an estimated one-third of people with aplastic anemia do not respond to immunosuppressive drugs.

If this is the case, doctors may consider other treatments, such as hematopoietic stem cell transplantation and a medication called eltrombopag (Promacta).

Those with aplastic anemia may face complications due to their disease as well as their treatment.

Sometimes, a person's body rejects a bone marrow transplant. Doctors call this graft-versus-host disease or GVHD.

GVHD can make a person feel extremely ill and can cause symptoms that include:

According to 2015 research, about 15% of aplastic anemia patients who receive immunosuppressive therapy will develop myelodysplastic syndromes or acute myeloid leukemia.

These conditions can develop years after a person's initial diagnosis.

Some people do not respond to aplastic anemia treatments. When this is the case, they are more vulnerable to infections that can be life threatening.

The outlook for a person with aplastic anemia depends on many factors, including:

A doctor will discuss a person's treatment outlook when considering the various therapies.

Aplastic anemia damages stem cells in a person's bone marrow. The bone marrow makes red blood cells, white blood cells, and platelets, which are all essential for the body.

A person with aplastic anemia may experience severe anemia symptoms. Treatment may include chemotherapy, stem cell transplants, and immunotherapy.

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What is aplastic anemia? Symptoms, causes, and treatment - Medical News Today

JenniferDelgado grew up in St. Louis, Missouri. She attended Webster University, whereshe received her Bachelor of Arts in Media Communications. She then went to MississippiState University, where she received a Bachelor of Science in Geosciences witha concentration in Broadcast Meteorology.

In 2006,Jennifer Delgado worked as a morning and noon meteorologist for WTVR-TV inRichmond, Virginia. Then in 2008, she began working at CNN International inAtlanta, Georgia, as their primary meteorologist, as well as a fill-inmeteorologist on all CNN networks. In 2010, she won a Peabody Award for CNNscoverage on the Deepwater Horizon oil spill in the Gulf of Mexico.

In 2013,Delgado was hired as a co-host of AMHQ (Americas Morning Headquarters) at TheWeather Channel. She anchored continuous coverage of breaking news and weatherevents, including live interviews with state and local officials, experts andresidents. She was also their fill-in co-host of Wake-Up with Al.

JenniferDelgado began freelancing as a meteorologist/anchor for WXIA-TV in 2017. Shepresented weathercasts every six minutes during a two-hour morning newscast andproduced weathercasts for radio, web, and the 24-hour weather channel.

Two yearsago, Jennifer Delgado was diagnosed with blood cancer. She underwent treatmentand received a bone marrow/stem cell transplant. Since the transplant, she hasbeen receiving treatment at the Emory Winship Cancer Institute and advocatingfor cancer awareness and more bone marrow donors.

No one is ever prepared tohear the words, you have cancer. It literally blew up my world. I had to stopworking because beating cancer became my full-time job. I knew something waswrong for months based on my symptoms. I was tiredall the time, my bones were aching, had migraines, vertigo andconfusion. Dealing with any illness is stressful, especially if you arent ableto work. Some people say cancer changed their life for the better; however, Idont want to credit cancer for anything positive. It was a wake-up call. Lifeis short, and you have to enjoy every moment.

I immediately went into adeep depression. I hid and only shared the news with my close friends andfamily. I was trying to hide the awful chemo port in my chest and made excuses for my appearanceand fatigue. It was very stressful. I think anyone dealing with a seriousmedical condition should reach out to people going through the same battle. I got some amazing tips from fellow blood cancersurvivors on Instagram and Facebook support groups. I have formed many closebonds and when I am feeling down they completely understand. Cancer patients caneasily go through their savings in a short amount of time. I was lucky to haveamazing health insurance but not everyone is that fortunate. There is a lot of grant money out there forpeople struggling financially. The Leukemia & Lymphoma Society is anamazing organization and helps patients with everything from financial help,information on clinical trials etc.

If you are strong enough, Isay its important to be your own health advocate. You know your body best. Ialso suggest if you have one, reaching out to a friend or family member whoworks in medicine (nurse, PA, doctor) to be your medical advocate. The advocatecan come to your appointments or even join a conference call during yourappointments when you need help understanding your treatment options. I waslucky to have both my mom and one of my best friends to help me interpreteverything. Never be afraid to ask your doctor questions, and dont forgetabout the physicians assistant, who often has more availability.

I was going back and forthto the doctor for nearly a year, and they keep dismissing my symptoms. At onepoint, one doctor told me to take probiotics. I finally decided it was time toget a second opinion when I was having trouble walking. Luckily, I found Dr.Drew Freilich, whom I credit with saving my life. He recognized that mysymptoms were severe and insisted that I needed an MRI. Thats how theydiscovered I had a blood cancer that was attacking my bones. I could havebecome disabled if I had waited longer to get help. If you know something iswrong, you have to be persistent about getting answers.

I know it sounds clich, butmy friends, family, and neighbors. They all took excellent care of me. Theydrove me to the hospital for chemotherapy or bone marrow biopsies. My friends were great and woulddrop by to bring me food or help clean up myhouse.

I know it may sound sillybut my dogs really helped keep my spirits up. Quite often, it was just me and the dogs and duringisolation. I truly believe that pets are healing, and studies show that havingone improves your mental health. There were several weeks when I had to be awayfrom my dogs because my immune system was too weak. I was lucky enough to havegreat friends watch my fur babies. I even tried to convince my friends to driveby Emory Hospital so that I could see them.

I would say you have to bepositive. It seems like its a long way away, and you wonder at times whetheror not everything you did is going to pay off when you finally get toremission. So, I think you have to be positive because you get very paranoid. Ibelieve positive thinking can be healing and improve your health. Keeping inmind that everyones journey is different, I think its also important to see apsychologist or therapist. Sometimes its easier to share your real concernswith a stranger. We always try and put on a brave face for family and friends.

Aftereverything, I felt like I had to give back to the cancer community and EmoryWinship Cancer Center. I got my dogs certified to be Happy Tails therapydogs, and now we visit patients battling cancer while they are getting chemo.Its amazing and emotional all at the same time. Many times, patients will say,your puppy made my day.

Iam also trying to raise awareness for the need of more bone marrow donors.Right now, the majority of donors come from Europe. It would be awesome if morepeople would register to be a bone marrow donor. Its a simple swab test. Ithink its a small price to pay, considering more than 170,000 people arediagnosed with blood cancer every year. Check out Be The Match or The Leukemia& Lymphoma Society.

I am not going to sugarcoatit, staying motivated is extremely challenging and a daily battle. I thinkevery cancer survivor questions, why did this happen to me? Is it gone? How longwill I stay in remission? It can be quite depressing, but you have to live forthe day and stick to a routine. I try to remind myself that there is a reasonwhy I am still alive, and I want to give back to others who are struggling.

Everything. I had months ofchemo to get my cancer level down enough to collect my stem cells for thetransplant. I wondered constantly, will I be in remission? And then once Iwas in remission, how long will I stay in remission before I relapse? Whenyoure dealing with blood cancers, most have no cure. So, theres always thatchance of relapse, and youre always worrying about it.

I did six rounds of chemobefore I was even ready to get a transplant. The stem cell transplant wassomething I was dreading because of the high dose of chemotherapy and losing myhair. That can be a very difficult experience, especially for women. After thosesix rounds, they collected my stem cells, which is not a fun process. Then theyprepped me, and I had the transplant.

After, I was in isolation atthe hospital for three weeks. Then I went home, and I was still under isolationfor another 100+ days. I felt like I was ready to lose my mind. During thistime, your white blood cells are regenerating, which means you dont have animmune system, and you suffer from extreme fatigue and pain. Walking up a shortflight of stairs would wipe me out. I couldnt eat salads, fruits, basicallyanything raw. When I left the house, Id have to wear a mask to protect myimmune system. I really hated that because everyone would stare and pretty muchknew I had cancer.

However, to put a positivespin on it, because of my time in isolation at home, I really felt my creativejuices start to flow. I began brainstorming and thinking of a lot of differentthings because life is short, and the cancer was my wake-up call.

So, my best advice duringthat period is to make a reading list and binge-watch shows on Netflix. I readthe Game of Thrones series. Iliterally had a calendar counting down to 100 days. Thats also the time whenyour hair finally starts to grow back!

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A Discussion With Jennifer Delgado on Life After Cancer and Weathering the Storm - Thrive Global

Doting dad Stephen Armstrong knows all too well what its like to be waiting for a transplant donor.

His son Jacob was diagnosed at two years old with a rare blood disorder and called on the public to donate stem cells to find him a match.

He then set out to raise as much money as he could for the blood cancer charity Anthony Nolan in a bid to save lives.

And now, after raising over 20,000, his efforts have been recognised by the charity as they honour him at an awards ceremony held at the Tower of London in November.

Stephen, 33, of Wallsend, North Tyneside, has been shortlisted for the Individual Fundraiser of the Year Award at the Anthony Nolan Supporter Awards 2019.

The prestigious awards are back for their seventh year and will recognise the outstanding achievements of the volunteers, fundraisers and campaigners who help the pioneering blood cancer charity save lives.

Stephens nomination is in recognition of his incredible fundraising efforts, leading a group of 19 friends and family in a series of physical challenges, all while his son was undergoing treatment.

After Jacob was diagnosed in 2017, Stephen set out to find a matching stem cell donor, as well as raise awareness of the need for more people on the register.

From here Jacobs Journey was born, and through a series of challenges including the Great North Run, the Great North Bike Ride and climbing Ben Nevis, Stephen has helped raise over 20,000 for the charity.

Jacob, who turns four in November, and his family have been told he does not need a transplant, but Stephen and his family want to continue raising awareness for others who arent so lucky.

When Jacob was diagnosed, we were stunned by how few people were on the stem cell donor register. I couldnt believe how a stranger in the street could potentially save our little boys life, said Stephen, an assistant manager for Dixons Carphone.

Anthony Nolan helped us massively while Jacob was ill and provided a great support network. I feel very proud to be nominated for an award, and I hope it can help build even more awareness for the cause.

Stephen and mum Kirsty, 28, received the news in December 2017 that Jacob was suffering from bone marrow failure, which affects between 30 and 40 children each year.

They first became concerned about his health when they went abroad to get married and noticed he was getting bruised easily. The marks would take weeks to disappear, so when the couple returned to the UK they decided to take Jacob to the doctor for a check up.

After tests he was then diagnosed and was treated at the Great North Childrens Hospital in Newcastle, where he received two blood transfusions.

Stephen added: When we were told Jacob did not need the transplant it was the best news in the world, a total relief. He still needs check ups every three months and his consultants is keeping an eye on him. There are so few people on the stem cell donor register so I just wanted to create a ripple effect with awareness and get more people on it.

Stephen, who has raised a further 8,000 for other smaller charities, has also been nominated for our Chronicle Champions Award in the Champion Fundraiser category.

Henny Braund, Chief Executive of Anthony Nolan, said: It is remarkable to see how many people support our work to find a match for those in need of a stem cell transplant. Without them, none of our lifesaving work would be possible.

Stephen has shown tremendous commitment to Anthony Nolan by continually going above and beyond in his fundraising efforts.

Henny added: We want to extend a huge congratulations to Stephen and look forward to celebrating with him at the awards.

The awards take place on Thursday 28 November at the Tower of London, and all winners will be revealed on the night.

Anthony Nolan is the charity that finds matching stem cell donors for people with blood cancer and blood disorders and gives them a second chance at life. It also carries out ground-breaking research to save more lives and provide information and support to patients after a stem cell transplant, through its clinical nurse specialists and psychologists, who help guide patients through their recovery.

To see the full shortlist, and find out more about the charity visit http://www.anthonynolan.org/awards

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Dad who called on the public for stem cells for his son is up for an award - Chronicle Live

MELVILLE, N.Y., Oct. 23, 2019 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (the Company") (BRTX), a life sciences company focused on stem cell-based therapies, today announced that the Australia Patent Office has issued a Certificate of Grant for the Companys patent application titled Human Brown Adipose Derived Stem Cells and Uses.

This is the second patent issued for the Companys brown fat technology in Australia and adds to three other patents related to BioRestoratives metabolic program (ThermoStem Program) that have previously been issued to the Company in the United States and other countries.

This patent will allow for the protection of a specific isolated human brown adipose tissue stem cell line capable of differentiating into multiple cell types. This particular cell line possesses strong characteristics applicable for potential therapeutic uses for treating a wide range of degenerative and metabolic disorders, including diabetes, hypertension, cardiac deficiency and obesity.

This patent, granted by the Australian Patent Office for our metabolic program, adds to our growing family of IP surrounding and protecting our brown fat metabolic cell program, said Mark Weinreb, CEO of BioRestorative Therapies. In particular, our invention relates to an isolated brown fat stem cell line that we expect to be used in our development of cell-based therapies to treat metabolic disorders.

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. 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 persistent lower back pain due to painful degenerative discs.

Metabolic Program (ThermoStem): We are developing a cell-based therapy 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 the body 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, whether the Company will be able to consummate the private placement and the satisfaction of closing conditions related to the private placement and those set forth in the Company's 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.

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CONTACT:Email: ir@biorestorative.com

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BioRestorative Therapies Receives A Second Patent in Australia For Its Metabolic Program - Yahoo Finance

United Therapeutics received a building permit Tuesday for a $9.5 million build-out of its cell therapy facility on the second floor of Mayo Clinics Discovery and Innovation Building.

The 21,843-square-foot space will house an automated stem cell manufacturing site, which is one of the first of its kind in the country. The Whiting-Turner Contracting Co. is the project contractor.

The technology, approved by the FDA in 2018, allows the Mayo Clinic Center for Regenerative Medicine to produce cells from the bone marrow of a stem cell donor in large enough quantities to be used as treatments in clinical trials. It allows for the treatment of multiple patients at the same time.

Construction began in 2017 on the $32.4 million building at 14221 Kendall Hench Drive. It held a grand opening in August.

The first floor houses three ex-vivo lung perfusion surgical suites used for lung restoration, another form of regenerative medicine. It turns donor lungs, which previously would have previously been unusable, into viable transplant organs. United Therapeutics also collaborates with Mayo Clinic on lung restoration.

The third floor houses the Life Sciences Incubator for biotech entrepreneurs, which offers coworking space, wet labs, business resources, networking and entrepreneurial training.

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United Therapeutics receives permit for cell therapy facility build-out at Mayo - Jacksonville Daily Record

NEW YORK, Oct. 25, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, today announced that it will be presenting at the Dawson James Securities 5th Annual Small Cap Growth Conference, being held on October 28-29, 2019 at the Wyndham Grand Hotel in Jupiter, Florida.

Preetam Shah, PhD, MBA, Chief Financial Officer is scheduled to present on Tuesday, October 29th at 3:40 p.m. Eastern Time, in Track 2 - Preserve Ballroom B, with one-on-one meetings to be held throughout the conference.

Chaim Lebovits, President and CEO of BrainStorm said, We are pleased to have the opportunity to have Dr. Shah present at the Dawson James Small Cap Growth Conference. Dr. Shah, joined BrainStorm in September 2019, and we look forward to having him present the Companys growth strategy and future to a wide audience of accreditied investors.

About NurOwn NurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

AboutBrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

Safe-Harbor Statements Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PR Phone: +1.646.677.1839sean.leous@icrinc.com

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BrainStorm Cell Therapeutics to Present at the Dawson James Securities 5th Annual Small Cap Growth Conference - GlobeNewswire

The study, published in the Journal of Cannabis Research, looked at a number of studies covering cannabis use and the immune system, noting that little is known on circulating white blood cell counts and cannabis use.

The researchers looked at the National Health and Nutrition Examination Survey (20052016), a survey designed to be nationally representative of United States non-institutionalised population, and found that there was a modest association between heavy cannabis use and higher white blood cell count but that neither former nor occasional cannabis use was associated with total or differential WBC counts.

White blood cells are the cells in our body that function mainly as immune cells originating in the bone marrow.

Today, it is known that cigarette smoking generates several chemicals that are implicated in oxidative stress pathways and systemic inflammation and elevated white blood cell count in tobacco cigarette smokers have been well documented, whereas tobacco abstinence is associated with sustained decrease in white blood cell count.

The study highlights how cannabis is able to mediate its effects through the cannabinoid-1 (CB1) and cannabinoid-2 (CB2) receptors.

CB2 receptors can be found in numerous parts of the body related to the immune system, including bone marrow, thymus, tonsils and spleen. CB1 receptors are present in the central nervous system, and at lower levels in the immune system.

The effects of cannabinoids on hematopoiesis, and immune cell proliferation using animal and cell based models has been widely demonstrated and a number of studies have examined the association of cannabis use and white blood cell counts in human immunodeficiency virus (HIV).

The studies have shown a higher white blood cell count in HIV positive men who used cannabis.

Last year a study discovered certain cannabinoids that enhance the immunogenicity of tumour cells, rendering them more susceptible to recognition by the immune system. This discovery is important because the leading class of new cancer fighting agents, termed checkpoint inhibitors, activates the immune system to destroy cancer cells.

Enhancing recognition of cancer cells with cannabinoids may greatly improve the efficacy of this drug class. The Pascal study was the first to identify a mechanism in which cannabinoids may provide a direct benefit in immunotherapy.

When looking at white blood cell counts the study noted that: Several of the important study limitations merit attention. The observational nature of the study constrained causal inferences. Even though NHANES collects blood and urine specimens, drug testing is not conducted, and cannabis use was self-reported which may lead to non-differential misclassification bias. There was no available information on the route of administration of cannabis (smoking, ingestion, etc.) or cannabis preparation/potency.

In addition, the study is based on fairly recent NHANES surveys (200516) which might be more representative of the increasing cannabis potency compared to NHANES III (19881994) surveys.

A number of laboratory studies have reported suppression of immune responses with cannabinoid administration, and some epidemiological studies found lower levels of inflammatory biomarkers such as fibrinogen, C-reactive protein and interleukin-6 in adult cannabis users.

The study also noted that the reported anti-inflammatory effects of cannabis were greatly attenuated when body weight is controlled for and suggests that the inverse cannabis-body weight association might explain the lower levels of circulating inflammatory biomarkers in adult cannabis users.

The study highlights that these alterations of immune responses by cannabis use might be associated with increased susceptibility to infections and hence the higher white blood cell count, however, it notes that it is possible that the elevated white blood cell count and suboptimal health status contributed to cannabis use rather than cannabis use caused suboptimal health.

The study states: This hypothesis, though, cannot be tested as NHANES does not collect information on cannabis use motives. Another potential mechanism can be through the effect of cannabinoids on stem cells. Pre-clinical studies suggest that cannabinoids stimulate hematopoiesis and hence this stimulation to bone marrow tissues can be associated with increased circulating white blood cell count in cannabis users.

Positive associations between heavy cannabis use, and total white blood cell and neutrophil counts were detected. Clinicians should consider heavy cannabis use in patients presenting with elevated white blood cell count.

Research on cannabis use and the immune system is lacking and the study suggests further research is needed to understand the immune related effects of different modes of cannabis use.

The study noted: Research on heavy cannabis use and cardiovascular health is needed as systemic inflammation, increased cardiovascular risk and increased mortality risk have been all associated with white blood cell elevation within the normal physiologic range.

Studies with repeated measures are needed to study immunomodulatory changes in cannabis users, and whether the mode of cannabis use can differentially affect immune responses.

Additional research is needed to understand the immune related effects of different modes of cannabis use and to elucidate the role of proinflammatory chemicals generated from smoking cannabis.

Originally posted here:
Cannabis use and the immune system: white blood cell count - Health Europa

Nicole Duhaney couldnt believe her luck when she learned she was having identical twins.

I felt like had won the lottery, Duhaney, 21, told TODAY Parents. "It was the happiest surprise."

After being pregnant for what felt like an eternity, Duhaney and her boyfriend, Niles Liburd, finally welcomed sons Emre pronounced Em-ree" and Elijah on Dec. 23, 2018.

Our life seemed perfect, the mom from Huddersfield, England, said.

But just three weeks later, Elijah developed a lump on his cheek, and both babies developed colds they couldnt seem to kick. Suddenly, they were projectile vomiting.

Trending stories,celebrity news and all the best of TODAY.

At just 4 months old, Emre and Elijah were both diagnosed with childhood acute myeloid leukemia. The disease, also known as AML, is a type of cancer in which the bone marrow makes a large number of abnormal white blood cells, according to the National Cancer Institute.

Myeloid leukemia is the second most common pediatric blood cancer, but it's still relatively rare. In the United States there are roughly 500 children a year between the ages of 0 and 14 that are diagnosed with AML, according to Dr. Richard Aplenc, a physician-scientist within the Division of Oncology at Children's Hospital of Philadelphia.

Aplenc said it is not surprising that Emre and Elijah were diagnosed at the same time.

"If the twins are identical, then they share the same placenta and the same blood supply, so that leukemic cell goes to the other twin," Aplenc explained. "We know that if leukemia is diagnosed before a year or so, there is 100 percent chance that the other twin will develop it."

Tragically, 10-month-old Elijah passed away at home in Tuesday. Doctors allowed Emre, who is currently undergoing chemotherapy, to leave the hospital so he could say goodbye to his brother.

The love they had for each other was just unbreakable, Duhaney noted. "They didn't like to be separated."

She recalled how Elijah pulled his brother in for a kiss after a recent stem cell transplant.

Elijah was beautiful. Every person he met, he touched their heart," Duhaney said. There were times when I cried and he rubbed my tears away. I wish God took me instead of him.

As Duhaney and Liburd, 26, make funeral arrangements a GoFundMe has been set up to help the couple with expenses they are finding comfort in knowing Elijah took his final breaths at home.

He spent six months of his life in a hospital, Duhaney told TODAY Parents. His final night he was where he wanted to be, with the people who loved him him the most.

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Baby dies from AML, the same cancer his identical twin has - TODAY