Archive for September, 2019

Its a real bummer when Aunt Flo arrives unexpectedly, but it can be even more panic-inducing when she doesnt show up at all. Every woman has a late or missed period at some point and the first step to figuring out why is getting familiar with your period before its missed. In order to determine your normal menstrual cycle it is important to keep track of when you have your period through a calendar or app on your phone, says Shannon Schellhammer, M.D., F.A.C.O.G., an obstetrician/gynecologist at Orlando Health.

Essentially, every womans body is different and works at its own speed. In fact, despite the popular belief that there are 28 days in a menstrual cycle, a 2019 study of more than 600,000 women in the journal Nature found that only 13% of women have 28-day cycles, and 29.3 days is actually the average length. On top of that, the International Federation of Gynecology and Obstetrics reports that a normal cycle can range anywhere from 24 to 38 days.

Your period is late if you are more than five days past the expected start date of your period, says Jacqueline M. Walters, M.D., an obstetrician/gynecologist and author of the forthcoming book The Queen V: Everything You Need to Know About Sex, Intimacy, and Down There Health Care. Your period is considered missed if you have gone more than one to two weeks past the expected start date. Our bodies are so complex that the potential reasons why you might miss a period are endless, but here are a few common ones.

1. Pregnancy

The number one cause for a missed period is pregnancy! says Dr. Schellhammer. She advises taking a home pregnancy test if you are sexually active, regardless of your age and form of birth control. Once you rule out pregnancy, it is safe to wait for the next month to see if you have a normal period. However, if you are concerned or do not have a clear cause of why you missed your period, I recommend you call your OB/GYN or primary care doctor to be seen. This is especially important if you have not been seen within the last year for an annual well-woman exam.

2. Stress

Stress whether its from something great like planning a wedding, starting a job, or moving to a new house, or something not-so-great like job loss, a bad relationship, or depression can press pause on your period. The chemistry required to develop and produce a normal cycle can be influenced and delayed by high levels of cortisol and/or adrenaline, which increase in the body as a result of chronic, daily stress, says Hector O. Chapa, M.D., F.A.C.O.G., clinical assistant professor of obstetrics and gynecology at Texas A&M University College of Medicine. This delayed cycle may be your bodys way of reminding you to take care of yourself. If stress is the cause for your missed period, it should resume when things in your life calm down.

3. Polycystic Ovarian Syndrome (PCOS)

"Without a doubt, PCOS is the most common hormone imbalance in women that leads to delayed or missed cycles in women aged 18 to 44, excluding pregnancy, says Dr. Chapa. The condition occurs when a woman produces more male hormones than normal. While blood tests and a vaginal ultrasound may support a diagnosis, the diagnosis may be considered if a woman has two of these three symptoms: excess facial or male-pattern hair growth, irregular periods and ovaries that appear to have little cysts. Theres no cure, but your OB/GYN can help you find the right treatment to get your cycle back on track. If PCOS isnt the culprit, your physician can help you figure out if another hormonal imbalance caused by thyroid or pituitary problems is at play.

4. Excess exercise

Exercise is necessary for our well-being, but too much of itwithout the caloric supportcan take a toll on our bodies and trigger missed periods. The female athlete triad occurs when a female athlete has low energy intake or disordered eating, menstrual delays or absence and low bone mass, says Dr. Chapa. Along the same lines, a bodyweight thats more than 10% below the ideal weight for your height can mess with your menstrual cycle.

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5. Medications

When you put a chemical into your body (including herbal supplements), it can cause a chain reaction that spreads across different systems. Birth control, antipsychotics, antidepressants, and chemotherapy can all cause a missed period, says Dr. Walters. If you recently started a new medication and started skipping periods, bring it up with your physician to see if its a normal side effect. For instance, with some contraceptives including IUDs, the shot, and certain birth control pills your period may stop altogether.

6. Perimenopause

Unless the ovaries are surgically removed, menopause doesnt just happen, says Dr. Chapa. The interval leading up to menopause is called the perimenopause and is marked by hot flashes, mood changes, and of course, changes in the menstrual cycle called skips and delays. While the average age of menopause in the U.S. is 51, Dr. Chapa says these changes can start anytime in your 40s.

The bottom line: A missed period rarely causes immediate harm to a womans health, but it does signify something underlying, says Dr. Walters. If the pattern continues, it can affect a womans ability to get pregnant, cause bone loss, lead to ovarian cysts, and cause excessive male-pattern hair growth thinning or balding on the head and more hair on the face, chest and abdomen. No one knows your body better than you so the best thing you can do is pay attention and dont hesitate to give your OB/GYN a call if something feels off.

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6 Reasons for a Missed Period Why Your Period is Late, According to Doctors - GoodHousekeeping.com

September 25, 2019 Whole breast radiation and partial breast radiation following a lumpectomy yield similar cosmetic outcomes for women diagnosed with early stages of cancer who wish to preserve their breasts. Findings of the new analysis from a phase III clinical trial were presented at the 61st Annual Meeting of the American Society for Radiation Oncology (ASTRO), Sept. 15-18, 2019,in Chicago.

Partial breast radiation is a good choice. Thats what Im going to say to certain subsets of my patients, said Julia White, M.D., FASTRO, a professor of radiation oncology at The Ohio State University Comprehensive Cancer Center and lead author on the abstract.

Women diagnosed with early stages of cancer (stage 2 or earlier) who undergo lumpectomies typically follow their surgery with radiation therapy to lower the risk of having the cancer return. Partial breast radiation can be an appealing treatment option, because the radiation is delivered over several days to a smaller area around the surgical site instead of 3 to 4 weeks to the entire breast.

Previously published results from this phase III trial, NRG Oncology/NSABP B39-RTOG 0413, found partial breast radiation to be only slightly less effective than whole breast radiation at reducing the risk of cancer recurring in the breast. There were no significant differences between treatment groups in overall survival rates, the time patients lived without the cancer coming back or the time patients lived without the cancer spreading to another site. In the current analysis, White and her team focused on cosmetic outcomes to help guide patients for whom both treatment choices would be equally or nearly equally effective for breast-conserving therapy.

In this analysis of 900 women with early-stage breast cancer who received either partial breast radiation (n=477) or whole breast radiation (n=423), both the patients and their physicians were asked to rate the cosmetic outcome of the treated breast, in comparison to the untreated side, as either excellent, good, fair or poor at three different times (baseline, 12 months after treatment and three years after treatment). Patients also rated their satisfaction with the outcome. Additionally, digital photos of the womens breasts acquired at each time point were rated by two teams of three physicians each who were blinded to which treatment the women received, when the photos were taken or which breast was treated.

We found that whether the women received whole breast radiation or partial breast radiation, there was an equivalent cosmetic outcome from the patients' perspective, said White. This pattern held whether the patient also received chemotherapy as well as radiation and lumpectomy. In addition, patients' satisfaction with their treatment and cosmetic outcome were equivalent for whole breast and partial breast radiation. Three years after completing radiation therapy, 81 percent and 86 percent of patients said they were totally satisfied with partial breast or whole breast radiation, respectively; 14 percent and 11 percent were somewhat satisfied; 2 percent and 3 percent were neither satisfied nor dissatisfied; 1 percent and 2 percent were somewhat dissatisfied; and less than 1 percent of patients in each group said they were totally dissatisfied with their treatment.

Treating physicians rated cosmetic outcomes from partial and whole breast radiation as equivalent at one year after treatment (p=1.00), but outcomes from partial breast radiation were considered worse at three years after treatment (p=0.001).

The physicians who reviewed digital photos without knowing if or when the breast received radiation rated cosmetics outcomes as equivalent for partial and whole breast radiation at one year (p=0.99) and three years (p=0.99) after treatment. There were differences between the ratings for patients who did or did not receive chemotherapy, however, with outcomes after partial breast treatment rated slightly worse among patients who received chemotherapy and outcomes after whole breast radiation rated slightly worse in the non-chemotherapy group.

Agreement between the patient and physician ratings was notably stronger when patients had better cosmetic outcomes after treatment. On average, patients who scored their outcomes as "excellent" or "good" (versus those who rated them as "fair" or "poor") agreed with their doctors' ratings 89 percent (vs. 45 percent) of the time and agreed with physicians making photo-based ratings 85 percent (vs. 32 percent) of the time.

White said the findings were important for women facing difficult choices after being diagnosed with breast cancer. If a patient chooses breast conservation for her treatment, she generally wants the breast to feel and look as normal as possible. It is difficult to have to say to a patient that she can have a shorter, more convenient treatment but will have to accept a cosmetic outcome that is not as nice as she would get with a longer treatment. We were relieved to find out the cosmetic outcomes are equivalent.

Understanding the physicians perspective on cosmetic outcomes as well as the patients was important, said White, because how the physician regards the results of treatment will help guide improvements in care. I am happy that the cosmetic outcomes based on the patients assessment and physician review of the digital photos were in agreement, she said. On the other hand, doctors who treat patients will ask themselves what they could have done differently to reach a better outcome or start cataloging what they did to reach the positive one, perhaps leading to innovations that improve outcomes.

The results of NSABP B39-RTOG 0413 and other randomized trials indicate that patients with Stage 0 or 1 breast cancer, who are over the age of 50 and have hormone-sensitive disease, would benefit equally well from partial or whole breast radiation for breast-conserving therapy, consistent with the ASTRO Consensus Statement for Partial Breast Irradiation. This subset of patients with breast cancer accounts for roughly 25-30 percent of new cases treated, or about 30,000 women each year, said White.

View additional coverage of ASTRO 2019

For more information: http://www.astro.org

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Partial and Whole-Breast Radiotherapy After Lumpectomy Provide Equally Satisfying Cosmetic Results - Imaging Technology News

Now, in addition to the nightmare of cancer, I had the nightmare of having to make a decision.

Two weeks post lumpectomy, the bruising and swelling were slowly going away. I stopped worrying about recovery and started agonizing about what lay ahead. If there were any benefit to doing chemo to decrease my chances of becoming metastatic, I should take it. I want to be alive when my kids get married and spoil my future grandchildren, even if that means torturing my body for a few months.

My husband, Kevin, a family practice physician, tried to talk me down. One-point-six percent is insignificant compared to how it could weaken your immune system, cause permanent nerve damage, osteoporosis it could even give you another cancer or kill you. You should trust your doctor. And your husband.

I wanted to. But the market researcher in me trusted information. I needed to collect qualitative and quantitative data and analyze which choice I would regret more. Which would allow me to live most fully and take care of the people I love?

At restaurants, waiters try to hide their irritation as I scroll through Yelp reviews on my phone to ensure the most popular dishes are being ordered. My notes on choosing a preschool were so robust I turned them into a how-to book. My newfound research project gave me the opportunity to feel as if I were actively doing something to heal myself.

Each night, once I heard Kevin snoring, I sat up against the headboard, turned on my iPad and lowered the screen brightness. I hurled myself down the rabbit hole of news articles, breast cancer websites and community forums. The TAILORx study was so new, however, there wasnt much information and no concrete answer.

In my support group, most of the conversation revolved around tips for getting through chemo the depression, mouth sores, numb hands and feet, and what to do when hair that fell out never grew back. I couldnt relate and didnt want to, yet somehow I envied them for being fighters.

One day I read about another test called MammaPrint. At our next appointment, my doctor happily showed me that those results also supported the argument against chemo. Time had run out and that was the final data point I needed. We arranged to start my month of radiation treatment the following week, followed by five years of daily hormone blocker pills. I apologized for second-guessing. She gave me an understanding smile and replied, Knowledge is power. She hugged me, said congratulations, and I was on my way. Decision made.

Originally posted here:
Is Everyone Doing Chemo Without Me? - The New York Times

NEW YORK--(BUSINESS WIRE)--Pfizer Inc. (NYSE: PFE) today announced the presentation of four IBRANCE (palbociclib) real-world analyses. The studies support the effectiveness of IBRANCE combination therapy in everyday clinical practice and provide additional insights on its use in certain patients with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) metastatic breast cancer (MBC). The posters will be presented at the European Society for Medical Oncology (ESMO) Congress 2019 in Barcelona, Spain on Sunday, September 29.

Among the data, Pfizer will share the first real-world comparative analysis of a CDK 4/6 inhibitor in combination with an aromatase inhibitor compared to an aromatase inhibitor alone.

We have an opportunity to make positive changes in cancer care by incorporating learnings from real-world data in addition to data gathered from clinical trials, said Chris Boshoff, M.D., Ph.D., Chief Development Officer, Oncology, Pfizer Global Product Development. We are pleased to share this view of the impact IBRANCE has had on patients treated outside of traditional clinical studies, as it continues to add to the body of evidence for IBRANCE and provides insights into the patient experience.

About the Real-World Comparative Analysis

In this retrospective analysis (Abstract #329P: Comparative effectiveness of palbociclib plus letrozole vs. letrozole for metastatic breast cancer in U.S. real-world clinical practices), treatment with IBRANCE plus letrozole demonstrated a statistically significant improvement in real-world progression-free survival (rwPFS) compared to letrozole alone: 24.5 months (95% CI = 20.7 32.7) versus 17.1 months (95% CI = 13.7 19.8) (HR = 0.68, 95% CI = 0.56 0.84, p = 0.0003).

To help deliver the best care to our patients, it is critical that physicians have compelling evidence of a medicines benefit on patients who resemble those who they treat every day, said Rachel Layman, M.D., Associate Professor, Department of Breast Medical Oncology at The University of Texas MD Anderson Cancer Center. The real-world evidence presented at ESMO provides a more robust understanding of the effectiveness of IBRANCE in patients who may not be reflected in the randomized trials.

The analysis compared 906 matched patients with HR+, HER2- MBC who started IBRANCE plus letrozole as initial endocrine-based therapy in the metastatic setting (n=453) or letrozole alone (n=453) from February 2015 to August 2018. rwPFS was measured by the treating physicians clinical assessment of source evidence, such as radiographic scans or pathology from the Flatiron Health longitudinal database. The most recent update for this database includes de-identified electronic health records from more than 280 cancer clinics representing more than 2.2 million cancer patients in the U.S.

The additional real-world posters at ESMO examining the use of IBRANCE in patients with HR+, HER2- MBC are:

To further educate the global oncology community about the importance of real-world data at ESMO, Pfizer is sponsoring a satellite symposium, Real World Data in Oncology: Its Growing Role in Research and Patient Care. The symposium will take place on Friday, September 27, from 6:00 8:00 pm CEST at Fira Gran Via in the Alicante Auditorium (Hall 3).

About IBRANCE (palbociclib) 125 mg capsules

IBRANCE is an oral inhibitor of CDKs 4 and 6,1 which are key regulators of the cell cycle that trigger cellular progression.2,3 In the U.S., IBRANCE is indicated for the treatment of adult patients with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-)advanced or metastatic breast cancer in combination with an aromatase inhibitor as initial endocrine based therapy in postmenopausal women or in men; or with fulvestrant in patients with disease progression following endocrine therapy.

The most common adverse reactions (incidence 10%) associated with IBRANCE are neutropenia, infections, leukopenia, fatigue, nausea, stomatitis, anemia, alopecia, diarrhea, thrombocytopenia, rash, vomiting, decreased appetite, asthenia, and pyrexia. Today in the U.S., IBRANCE is the most prescribed FDA-approved oral combination treatment for HR+, HER2- metastatic breast cancer.

IBRANCE currently is approved in more than 90 countries and has been prescribed to more than 230,000 patients globally.

The full U.S. Prescribing Information for IBRANCE can be found here.

IMPORTANT IBRANCE (palbociclib) SAFETY INFORMATION FROM THE U.S. PRESCRIBING INFORMATION

Neutropenia was the most frequently reported adverse reaction in PALOMA-2 (80%) and PALOMA-3 (83%). In PALOMA-2, Grade 3 (56%) or 4 (10%) decreased neutrophil counts were reported in patients receiving IBRANCE plus letrozole. In PALOMA-3, Grade 3 (55%) or Grade 4 (11%) decreased neutrophil counts were reported in patients receiving IBRANCE plus fulvestrant. Febrile neutropenia has been reported in 1.8% of patients exposed to IBRANCE across PALOMA-2 and PALOMA-3. One death due to neutropenic sepsis was observed in PALOMA-3. Inform patients to promptly report any fever.

Monitor complete blood count prior to starting IBRANCE, at the beginning of each cycle, on Day 15 of first 2 cycles and as clinically indicated. Dose interruption, dose reduction, or delay in starting treatment cycles is recommended for patients who develop Grade 3 or 4 neutropenia.

Severe, life-threatening, or fatal interstitial lung disease (ILD) and/or pneumonitis can occur in patients treated with CDK4/6 inhibitors, including IBRANCE when taken in combination with endocrine therapy. Across clinical trials (PALOMA-1, PALOMA-2, PALOMA-3), 1.0% of IBRANCE-treated patients had ILD/pneumonitis of any grade, 0.1% had Grade 3 or 4, and no fatal cases were reported. Additional cases of ILD/pneumonitis have been observed in the post-marketing setting, with fatalities reported.

Monitor patients for pulmonary symptoms indicative of ILD/pneumonitis (e.g. hypoxia, cough, dyspnea). In patients who have new or worsening respiratory symptoms and are suspected to have developed pneumonitis, interrupt IBRANCE immediately and evaluate the patient. Permanently discontinue IBRANCE in patients with severe ILD or pneumonitis.

Based on the mechanism of action, IBRANCE can cause fetal harm. Advise females of reproductive potential to use effective contraception during IBRANCE treatment and for at least 3 weeks after the last dose. IBRANCE may impair fertility in males and has the potential to cause genotoxicity. Advise male patients to consider sperm preservation before taking IBRANCE. Advise male patients with female partners of reproductive potential to use effective contraception during IBRANCE treatment and for 3 months after the last dose. Advise females to inform their healthcare provider of a known or suspected pregnancy. Advise women not to breastfeed during IBRANCE treatment and for 3 weeks after the last dose because of the potential for serious adverse reactions in nursing infants.

The most common adverse reactions (10%) of any grade reported in PALOMA-2 for IBRANCE plus letrozole vs placebo plus letrozole were neutropenia (80% vs 6%), infections (60% vs 42%), leukopenia (39% vs 2%), fatigue (37% vs 28%), nausea (35% vs 26%), alopecia (33% vs 16%), stomatitis (30% vs 14%), diarrhea (26% vs 19%), anemia (24% vs 9%), rash (18% vs 12%), asthenia (17% vs 12%), thrombocytopenia (16% vs 1%), vomiting (16% vs 17%), decreased appetite (15% vs 9%), dry skin (12% vs 6%), pyrexia (12% vs 9%), and dysgeusia (10% vs 5%).

The most frequently reported Grade 3 adverse reactions (5%) in PALOMA-2 for IBRANCE plus letrozole vs placebo plus letrozole were neutropenia (66% vs 2%), leukopenia (25% vs 0%), infections (7% vs 3%), and anemia (5% vs 2%).

Lab abnormalities of any grade occurring in PALOMA-2 for IBRANCE plus letrozole vs placebo plus letrozole were decreased WBC (97% vs 25%), decreased neutrophils (95% vs 20%), anemia (78% vs 42%), decreased platelets (63% vs 14%), increased aspartate aminotransferase (52% vs 34%), and increased alanine aminotransferase (43% vs 30%).

The most common adverse reactions (10%) of any grade reported in PALOMA-3 for IBRANCE plus fulvestrant vs placebo plus fulvestrant were neutropenia (83% vs 4%), leukopenia (53% vs 5%), infections (47% vs 31%), fatigue (41% vs 29%), nausea (34% vs 28%), anemia (30% vs 13%), stomatitis (28% vs 13%), diarrhea (24% vs 19%), thrombocytopenia (23% vs 0%), vomiting (19% vs 15%), alopecia (18% vs 6%), rash (17% vs 6%), decreased appetite (16% vs 8%), and pyrexia (13% vs 5%).

The most frequently reported Grade 3 adverse reactions (5%) in PALOMA-3 for IBRANCE plus fulvestrant vs placebo plus fulvestrant were neutropenia (66% vs 1%) and leukopenia (31% vs 2%).

Lab abnormalities of any grade occurring in PALOMA-3 for IBRANCE plus fulvestrant vs placebo plus fulvestrant were decreased WBC (99% vs 26%), decreased neutrophils (96% vs 14%), anemia (78% vs 40%), decreased platelets (62% vs 10%), increased aspartate aminotransferase (43% vs 48%), and increased alanine aminotransferase (36% vs 34%).

Avoid concurrent use of strong CYP3A inhibitors. If patients must be administered a strong CYP3A inhibitor, reduce the IBRANCE dose to 75 mg. If the strong inhibitor is discontinued, increase the IBRANCE dose (after 3-5 half-lives of the inhibitor) to the dose used prior to the initiation of the strong CYP3A inhibitor. Grapefruit or grapefruit juice may increase plasma concentrations of IBRANCE and should be avoided. Avoid concomitant use of strong CYP3A inducers. The dose of sensitive CYP3A substrates with a narrow therapeutic index may need to be reduced as IBRANCE may increase their exposure.

For patients with severe hepatic impairment (Child-Pugh class C), the recommended dose of IBRANCE is 75 mg. The pharmacokinetics of IBRANCE have not been studied in patients requiring hemodialysis.

About Pfizer Oncology

At Pfizer Oncology, we are committed to advancing medicines wherever we believe we can make a meaningful difference in the lives of patients. Today, Pfizer Oncology has an industry-leading portfolio of 22 approved innovative cancer medicines and biosimilars across more than 30 indications, including breast, prostate, kidney and lung cancers, as well as leukemia and melanoma. Pfizer Oncology is striving to change the trajectory of cancer.

Pfizer Inc.: Breakthroughs that change patients lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.pfizer.com. In addition, to learn more, please visit us on http://www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube, and like us on Facebook at Facebook.com/Pfizer.

DISCLOSURE NOTICE: The information contained in this release is as of September 24, 2019. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about IBRANCE (palbociclib), including its potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of IBRANCE; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when drug applications may be filed in any additional jurisdictions for IBRANCE for potential HR+/HER2- metastatic breast cancer indications or in any jurisdictions for any other potential indications for IBRANCE; whether and when any such other applications may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether such product candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of IBRANCE; and competitive developments.

A further description of risks and uncertainties can be found in Pfizers Annual Report on Form 10-K for the fiscal year ended December 31, 2018 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors That May Affect Future Results, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

____________________________1 IBRANCE (palbociclib) Prescribing Information. New York. NY: Pfizer Inc: 2019.2 Weinberg, RA. pRb and Control of the Cell Cycle Clock. In: Weinberg RA, ed. The Biology of Cancer. 2nd ed. New York, NY: Garland Science; 2014:275-329.3 Sotillo E, Grana X. Escape from Cellular Quiescence. In: Enders GH, ed. Cell Cycle Deregulation in Cancer. New York, NY: Humana Press; 2010:3-22.

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Pfizer Presents New Evidence of IBRANCE (palbociclib) Effectiveness in HR+, HER2- Metastatic Breast Cancer Patients in Four Real-World Studies at ESMO...

Newman Center offering classes in bioethicsFILE PHOTO | THE SPECTRUMCatholic-specific courses held through NDSU.

Saint Pauls Catholic Newman Center, home of the bisonCatholics, has introduced a new course this semester. The ethics course, advertised for students going into the health professions, is meant to teach bioethics through the lens of Catholic dogma. The source of the course material as well as the introduction of religious ethical teachings sets an unusual, and perhaps concerning, precedent for classes at NDSU.

The course is listed as CHP 391: Fundamentals of Catholic Healthcare Ethics and Bioethics. The term Catholic bioethics and the idea of a course directed at health professionals seems like a bit of a paradox considering the churchs historical relationship with science and medicine. The full course takes place over three semesters and results in a certification from the National Catholic Bioethics Center (NCBC).

In an email sent out to bisonCatholics using NDSU logos, the course was described as follows: The National Catholic Certification Program in Health Care Ethics has been developed at the request of many bishops and administrators of health care facilities to provide a credible and systematic formation so that dioceses, hospitals and ethics committees will have advisors better qualified to apply the Catholic moral tradition to challenging contemporary issues in healthcare. For a three-semester course, which provides students with nine NDSU credits, the description is especially vague.

The class is taught by the Monsignor, Gregory Schlesselmann, using course modules developed by the NCBC; so, the question is: What is the National Catholic Bioethics Center? According to the website, the NCBC, Conducts research, consultation, publishing and education to promote human dignity in health care and the life sciences, and derives its message directly from the teachings of the Catholic Church. Again, this is a fairly vague description, but you can probably imagine what these messages from the Catholic Church might be.

The NCBC uses legislation and outreach to promote Catholic values within medical professions. Their stances on abortion and assisted-suicide can be easily imagined: strongly opposed. However, it is their views on topics such as gender identity and same-sex marriage that cause surprise and concern.

NCBC share regular updates, one such update stated: NCBC joins medical associations in advising the US Supreme Court that reinterpreting in federal law the term sex as gender creates a subjective and unworkable means of categorization. This ideology leaks into their work trying to legislate for policies that would protect medical professionals who do not want to assist patients trying to get gender reassignment surgery or hormone therapy.

They elaborate on this by saying, We believe that the best therapies for gender dysphoria will seek to make patients comfortable in their own bodies, rather than take unnecessary medical risks to attempt the impossible and make their bodies reflect their feelings. They are essentially saying that individuals who wish to go through gender reassignment surgery should instead receive psychological treatment to accept their bodies.

Another disturbing take from NCBC has to do with the medical professionals obligations to respect the marital status of same-sex couples. Their exact words on the topic were, There is a fundamental difference between the marital relationship of one man and one woman, with the ensuing rights recognized for the well-being of children and society, and committed relationships between two persons of the same sex.

With all of this information in mind, you may ask: Why should we care? Sure, its no secret how the Catholic church feels about abortion, same-sex marriage, gender identity and physician-assisted suicide. However, when a course promoting Catholic views on all these topics is being taught through NDSU, these feelings go beyond religious expression into the muddied waters of possible discrimination.

NDSU anti-discrimination policy specifically states that programs cannot discriminate on the basis of sexual orientation or gender identity; however, CHP 391 is being taught through an organization who is fighting for legislation that will discriminate on those bases specifically. The course seems to provide future medical professionals with legal tools to avoid treating certain patients. Its difficult to see how this course work will not directly contradict NDSUs own policies.

The real concern is how instituting religion-specific ethics courses, or any other courses directly in favor of a specific religion will affect the NDSU community. NDSU is at its most beautiful and progressive when multiple religions and diverse backgrounds are present. If courses are to be taught that support Catholic doctrine, where can we draw the line from stopping courses being taught which encourage other religious teachings, which propagandize certain political ideologies or that blatantly encourage the discrimination of identities held by NDSU students?

While I am not Catholic, I can say with absolute certainty that I would not want courses held at NDSU which only serve to encourage my personal beliefs. Individuals come to college to broaden their perspective and learn about disparate points of view. As of now, no rules may technically have been broken. However, the possibility that an individual may find themselves in the hands of an NDSU health professions graduate and not be served due to identity or spiritual belief should concern both the students and the administration. A precedent has been set and the resulting dangers could be substantial.

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Religion in the health professions - NDSU The Spectrum

MedicalResearch.com Interview with:Adrian E. Bostrm MD, on behalf of the authorsDepartment of NeuroscienceUppsala University, Sweden

MedicalResearch.com: What is the background for this study?

Response: While prevalence estimates vary, literature indicates that hypersexual disorder (HD) affects 3-6% of the population. However, controversy surrounds the diagnosis and little is known about the neurobiology behind it.

Hypersexual disorder has not previously been investigated with regards to epigenomic and transcriptomics in a hypothesis-free study approach and little is known about the neurobiology behind this disorder.We investigated whether there were any epigeneticchanges that affect gene activity and expression in hypersexual disorder (HD)patients and identified a dysregulated microRNA that is believed to influence the mechanism of action of the hormone oxytocin in brain.

Oxytocin is known to have wide-rangingbehavioral influences. To the bestofour knowledge, no previous study provided evidence for an association between DNA methylation, microRNA activity andoxytocin inhypersexual disorder.Our findings merits further research in the role of MIR4456 and especially Oxytocin in hypersexual disorder. Further studies are needed to confirm the role of Oxytocin in HDand to investigate whether treatment with oxytocin antagonist drug therapy could have beneficial effects for patients suffering from hypersexual disorder.

MedicalResearch.com: What are the main findings?

Response: In this study we investigated over 8000 different DNA methylation sequenced in a hypothesis-free and thereby unbiased manner. Therefore, we were intrigued and surprised to identify a strongly dysregulated microRNA targeting genes primarily expressed in brain and that are involved in major neuronal molecular mechanisms thought to be relevant for hypersexual disorder, e.g. the oxytocin signaling pathway. This microRNA alsoappears to be evolutionary conserved throughout primates, which is also an interesting and unexpected finding.

MedicalResearch.com: What should readers take away from your report?

Response: Hypersexual disorder incorporates different pathophysiological mechanisms including impulsivity, compulsivity, sexual desire dysregulation and sexual addiction. This can be interpreted such that hypersexual disorder contains addictive elements, but is not to be seen as exclusively an addiction. Our findings, in light of the crossover with alcohol dependence, suggest that MIR4456 and the oxytocin signaling pathway may be primarily involved with the addictive component of hypersexual disorder. Further studies are needed to fully confirm this.

MedicalResearch.com: What recommendations do you have for future research as a result of this work?

Response: Our results motivates further research in the efficacy of, for example, oxytocinregulating drug therapy in hypersexual disorder whichcould contribute to novel treatment options to improve the clinical outcome of those affected. In addition, we identify a specific microRNA (miRNA)for whichfuture potentialmiRNAregulating drugs could be tested in hypersexual disorder.

MedicalResearch.com: Is there anything else you would like to add?

Response: Our DNA is genetic code for genes that translate into different sequences of amino acids calledproteins. Proteins, in turn, constitute a main definingelement of all living things. Our DNA is inherited and does not change over time. This study, however, pertained to epigenetics, which arechanges that affect gene activity and expression. These epigenetic activitieschange over time and can be dysregulated in certain ailments. There are different epigenetic mechanisms.

In this study, we studied DNA methylation (a process known to influence gene expression, that is, the quantityof a gene that is translated into a protein)and microRNA activity(short non-coding gene segments that can influence the translation of several hundred different genes).

Comparing patients with hypersexual disorder to healthy volunteers, we identified a DNA methylation sequence to be significantly altered in hypersexual disorder. To ascertain the significance of this finding, the same DNA sequence wasfurther demonstrated to be dysregulated in subjects with alcohol dependence, suggesting it could be primarily associated with the addictive component of hypersexual disorder. The identifiedDNA methylation sequence was associated to a microRNA called (microRNA 4456; MIR4456), and further analysis showed that this DNA methylationsequence influence the quantity of MIR4456 that is produced.Furthermore, in the same study group, we demonstrate that MIR4456 exists in significantly lower quantity in hypersexual disorder as compared to healthy volunteers, strongly suggesting that altered DNA methylation patterns in hypersexual disorder influence and contributes to explaining the observed dysregulation of MIR4456.As microRNA:s theoretically are abletotarget several hundred different genes, we used computer algorithms to reveal that MIR4456 targets genes that arepreferentially expressed in brain and that are involved in major neuronal molecular mechanisms thought to be relevant for HD, e.g., the oxytocin signaling pathway. Our findings merits further research in the role of MIR4456 and especially Oxytocin in hypersexual disorder. Further studies are needed to confirm the role of Oxytocin in HDand to investigate whether treatment with oxytocin antagonist drug therapy could have beneficial effects for patients suffering from hypersexual disorder.

Yet unpublished data intended for a separate follow-up study show a highly significant increase in Oxytocin levels in patients with hypersexual disorder as compared to controls, and a significant reduction in oxytocin levels after Cognitive Behavior Therapy treatment, strongly implying a causal role of Oxytocin in hypersexual disorder andmaking the claims presented in this study much stronger. These preliminary results have been presented as a late breaking poster in Society of Biological Psychiatry meeting in May 2019 and also submitted as a poster in ACNP in December 2019.

Citation:

Adrian E. Bostrm et al, Hypermethylation-associated downregulation of microRNA-4456 in hypersexual disorder with putative influence on oxytocin signalling: A DNA methylation analysis of miRNA genes,Epigenetics(2019).DOI: 10.1080/15592294.2019.1656157

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Epigenetic Changes Linked to Hypersexual Disorder and Addictive Behaviors - MedicalResearch.com

BALTIMORE With violent crime in Baltimore approaching an all-time high, its no secret that much of the malfeasance gripping the city is drug-related. But should law-abiding citizens who apply for a Maryland State Police Handgun Wear and Carry Permit be required to list anxiety, hormone, erectile dysfunction, and breast cancer medications as part of the application process? And what about banking? Should the way you handle your cash invalidate your right to self-protection?

To understand the troubling intimidation factor that intrusive medical and money questions bring to Marylands Wear and Carry Permit application process, we spoke with Post-Examiner contributor Brian Bissett. Bissett is currently appealing his second consecutive restricted Maryland concealed carry permit and has asked the Maryland State Police to open their files as a part of that appeal process.

This is the second part of a two-part interview. The first part of this interview may be found here.

BPE: Your comments in the previous article about the politics surrounding gun legislation were most enlightening. But Id like to get back to the carry permit application process with a couple of oft-asked questions. First, do you have any idea how many gang members annually apply for a carry permit?

Bissett: I have no idea, but I cant imagine it would be many.

BPE: Do you have any numbers on how many felons have attempted to lawfully purchase a gun?

Bissett: No, I dont have any statistics for those things either.

BPE: I realize it may appear that I just set up a couple of straw men for you to knock down, but dont questions such as these help us get around to the thorny issue at hand that is what kind information the State Police should and should not be tracking in the permit process?

Bissett: I believe that they have been tracking all this information for a while, and Ive been told they have a database with all of this information.

Certain information on those applications is what Im looking for.

In fairness to the Maryland State Police, it may not go back as far as I want. But they most certainly have a database for all of this permit information going back the last twenty years.

They could easily query the information Ive requested.

BPE: What sort of information are you requesting?

Bissett: Id like to look at the racial breakdown of approvals and denials, as well as the kind of personal information which has been mined and stored.

One of the things I found troubling when I first went through the permit process is, I was asked on my interview to put down every single prescription drug I am taking.

I was pretty taken aback by that. What if you are a man taking Viagra or a woman taking Avasitin for breast cancer? Should that be included on any sort of application? Come on you could never get a judge to sign a search warrant for that.

Race is one of the questions an applicant must answer on the Maryland State Police Concealed Carry Permit application.

BPE: How does the State Police application on this point compare to what you underwent for a Federal Government security clearance?

Bissett: In the Federal process, they are only allowed to ask a physician one question in regard to mental health: If the person under investigation has a condition that could impair judgment, reliability, or ability to properly safeguard classified national security information?

If the answer is No, no further questions can be asked. If the answer is Yes, only then they may ask additional questions.

Do questions about embarrassing medical or mental health conditions dissuade people from applying for a permit?

The question here is, why arent the Maryland State Police only asking, If the person under investigation has a condition that could impair judgment, reliability, or ability to properly and safely carry a firearm in public?

The fishing expedition of requiring everyone to give personal medical information including all prescription medication they are taking and any mental health diagnosis is in my opinion a violation of HIPAA and done to dissuade and intimidate those with any embarrassing medical or mental health conditions from applying for a permit.

Is it their business if someone has breast cancer, erectile dysfunction, or general anxiety disorder? Quite frankly, its amazing nobody has sued them over this issue.

BPE: Isnt it reasonable, though, to ask if the applicant is on any psychotropic medications?

Bissett: I would say no, in most cases, unless the person has a history of schizophrenia or is bi-polar. People who have some type of mental disorder tend to be victims of violent crime more than they are perpetrators.

Dont get me wrong. I believe that people who have been forcibly institutionalized because they present a danger to themselves probably shouldnt be allowed to obtain a permit. But if you are taking a prescription medication to help you relax when you fly? There they want a note from your doctor, and what doctor can predict a persons mental condition once they leave the office?

Look, people go through problems all of their life. They get divorced; they lose a loved one to death. They may be treated for depression and then they recover. Should an incident such as profound grief mean they should be denied the right to own a firearm in perpetuity?

BPE: Doesnt the Federal Background Instant Check system look at that sort of thing, too?

Bissett: Thats a very difficult question to answer, and the only reason I know this much about it is because Im presently taking a Department of Defense class.

There is not a single database or repository that a law enforcement agency can go to that will necessarily pick up every arrest and/or crime a person may have committed. The records are siloed to a certain degree. Then when it comes to mental health, youre in a whole other ball game, because those records have also been siloed. Then there is HIPPA and the Privacy Act. Most organizations will err on the side of caution because they are potentially liable for millions of dollars if they disclose certain information.

The thing that disturbs me and I know others feel this way as well is that they are effectively ending mental health treatment in this country.

When you have something like the New York Safe Act, where a mental health professional must report anybody who might be a risk, they report everyone, no matter how minute the risk, and that discourages people from seeking the treatment they need.

If you drive people underground, and they dont seek treatment, you create the potential for even greater problems.

BPE: As we are speaking, Baltimore City is quickly approaching the 250 murder mark for the year. Its also gearing up for a major election. Is anyone in the mix for political office there taking a pro-gun or a pro-self defense position?

Bissett: I wouldnt know, to be honest with you, because I dont follow city politics to that degree. The one thing I can tell you is that I often listen to an African-American talk radio station, and whenever the subject of gun ownership comes up, four out of five callers express outrage that they cannot either purchase a firearm or get a permit to carry one.

There are no shooting ranges in Baltimore City. (Wikimedia Commons)

When the state passed the Firearms Safety Act of 2013, they created a process that was much more complicated for your average person to buy a firearm. They have to take a safety course and fire one bullet out of a handgun. But there are no ranges in Baltimore City. So, you are not going to be able to fulfill that requirement using public transportation.

There is a requirement to get fingerprinted, and I dont know how many places do that in Baltimore City. Then there is the requirement to complete the application online using a credit card.

Believe it or not, there is a class of people in this country who do not use credit cards.

BPE: That would bring us back to the hypothetical case of the house-keeper or the carpenter who just deals in cash?

Bissett: Yes. Then they have to use a scanner to enter their documents. Brian Frosh says they can just go to a library, but here you have individuals who are not technically savvy, using equipment which may be on lock-down or not adequately secure. It really puts city residents at a disadvantage to buy a gun, not to mention trying to obtain a carry permit.

BPE: Wouldnt such technical requirements particularly discriminate against African-American and Hispanic city residents?

Bissett: I believe they do. Given everything we have talked about here, African-Americans and Hispanics are much less likely than a white person to be able to navigate the handgun permit process. And yet, they are more likely to be the victim of a crime.

Lets face it: The State Police have created a process that is so intricate, so complicated, so convoluted, that a recent immigrant or an uneducated person doesnt stand a chance.

Maryland Attorney Gen. Brian Frosh.

If you compare a person who went to Harvard to someone who went to Baltimore City Public Schools, the city resident is much less likely to navigate that process.

BPE: Yet the Harvard graduate isnt the one living in a dangerous neighborhood?

Bissett: Exactly, and that is where people like Civil Rights Attorney Dwight Pettit contend that this system is inherently racist.

BPE: You did a pretty thorough job of charting the correlation between strict gun laws and an increase in crime in Baltimore City. Have you looked at other Maryland Counties?

Bissett: I have not because, frankly, getting the numbers together for Baltimore and similar cities was hard enough. I knew Id be crucified for any mistake, and to date, nobody has challenged the validity of the numbers I published in the graphs. Its always been, Well, even though there may be a mathematical correlation, I dont believe this is the cause.

Thats the reason I went through the Bradford-Hill causality theorem. It shows that, more likely than not, restricting an honest persons access to a firearm is going to result in more crime. By restricting I mean going beyond checking a persons background at the point of the sale.

I support the instant background checks, which determine that this person is who they say they are; that this person is not a felon, and is qualified to own a gun. However, I dont think that stops anyone who wants to own a gun from obtaining a gun. What it does do is it prevents them from getting the gun as quickly as theyd like to. But thats a topic for another conversation.

BPE: Where are Americans today on the right to carry?

Bissett: Well, following the Florida example, weve gone from states enacting Shall Issue laws to the Constitutional Carry which means you dont need a permit to carry your firearm as long as you are a law-abiding citizen. I believe there are now thirteen states which allow you to strap it on your hip. They said the same thing about that that they did about concealed carry permits, but by and large that hasnt happened.

BPE: So, in your opinion, Maryland lags behind on idea of a Constitutional Right to carry?

Bissett: I believe the county is moving in that direction. And not just conservatives.

I have a good friend at work, and his wife has been a social worker in Baltimore City for the past 25 years. Interestingly enough, this guy is very liberal, but he and I are very good friends.

I put this friend down as a reference, when I applied for my carry permit. I was later told that when the investigator called him, he said, Let me be perfectly clear: I dont like guns, I dont own guns, I dont believe in guns. And I think the world would be a better place if there were no guns. But if anybody should be carrying a gun, its this guy.

BPE: Wow!

Bissett: Yeah, the investigator said he had never heard a reference like that before. Anyway, as I said, this mans wife has been a social worker for a long time in Baltimore City. She works with a lot of disadvantaged youth, and they have all told her the same. If youd like a gun, I can have one for you in an hour.

BPE: What does it cost to buy such a handgun in Baltimore City?

Bissett: She said the answer from all of them is also the same. That depends on how many people have been killed with it. If no one has been killed with the gun, then thats a clean gun. Those guns cost top dollar. The more crimes that can be tied to a gun, the cheaper it becomes.

(This is the second part of a two-part interview. The first part may be found here.)

Here is the original post:
Concealed Carry: Does Maryland State Police Need to Know if Permit Applicant is Taking Viagra? - Baltimore Post-Examiner

Three new animal-model tests of a potential gene therapyfor oculopharyngeal muscular dystrophy (OPMD), known as BB-301, are planned, Benitec Biopharmaannounced. Results are expected tosupport a request for a Phase 1 clinical trial in patients.

OPMD results from a faulty PABPN1 gene, leading to a protein that can form insoluble clumps linked with muscle weakness.

BB-301 uses a DNA-directedRNA interference(ddRNAi) strategy. Delivered via a modified, harmless adeno-associated virus (AAV), the therapy is intended to silence and replace the mutant protein. It suppresses its production and provides a source of normal, or wild-type, protein to target cells.

A 2017 study showedthat this approach significantly reduced the amount of PABPN1 aggregates, restored muscle strength and eased muscle fibrosis (scarring) in mice. Subsequent experiments in cells from OPMD patients confirmed these results.

The preclinical research, to be conducted in a canine disease model, will help to optimize methods of administration, confirm the efficiency of the AAV vector in introducing a healthy gene into key muscle cells, assess optimal dosing, and further characterize toxicological data necessary for regulatory filings and clinical trial design.

According to the company, these three planned dog studies will support the submission of an Investigational New Drug (IND) application, an essential step in getting regulatory approval to start the trial. Pre-IND application meetings with the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and Health Canada took place in 2017.

Through our continued focus on BB-301 for the treatment of [OPMD], our team has an unprecedented opportunity to develop a novel genetic medicine that could facilitate clinically meaningful patient benefit, Jerel A. Banks, MD, PhD, Benitecs executive chairman and CEO, said in a press release.

The preclinical studies will be done in partnership with a medicine and surgery team with several decades of experience in OPMD treatment, Benitec added.

The FDA granted orphan drug status to BB-301 as a potential OPMD treatment in January 2018. The EMA awarded it similar status in early 2017.

Jos is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimers disease.

Total Posts: 307

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

Read more from the original source:
Final Tests Planned into Potential OCMD Gene Therapy, Could Support Trial in Patients - Muscular Dystrophy News

RESEARCH TRIANGLE PARK Pfizer is investing $19 million to create a new gene therapy production facility in RTP and could add up to 120 scientists and staff to operate it.

Information provided by Pfizer on Thursday says the company will move operations from a related facility in Chapel Hill that it acquired as part of a deal to buy Bamboo Therapeutics in 2016.

The new location is near the intersection of I-40 and I-540 in RTP.

According to the drug giant, an existing building will be retrofitted to accommodate production. It will provide facilities for drug substances, and related products as well as testing labs.

The news follows the recent announcement that Pfizer would invest $500 million and add some 300 jobs related to gene therapy at its campus in Sanford.

Construction will begin in 2020 with completion expected in 2022.

Pfizer adding 300 jobs, investing $500M at Sanford campus

Read this article:
Pfizer to build gene therapy facility in RTP, could add up to 120 scientists, staff - WRAL Tech Wire

Until the day she dies, Sandy Morris is hunting for a comma and a clause.

In May 2018, the 53-year-old mother of three learned she has ALS, a diagnosis made even more devastating by a somber directive to get her affairs in order period. The finality speaks to the absence of an effective treatment, yet alone a cure, for the neurodegenerative disease also known as Lou Gehrig's disease.

Since the diagnosis, Morris has sought to give future ALS patients some measure of optimism, amending the 'get your affairs in order' instruction to add 'but, there's something worth looking into.' While medical advances in areas like gene therapy show potential to change the course of other potentially fatal diseases, a disease-modifying treatment in ALS has proved elusive.

"In cancer, you have a shot," Morris said in an interview. "Not always a great shot, but you have one. In this land, everybody is just like, 'We don't have anything.'"

But now, Morris said she has reason for hope, stemming from a first-of-its-kind study in ALS that will test five experimental drugs simultaneously in a platform trial. Five drugmakers selected for the trial were announced last week.

After years of work, the broader promise of these adaptive trials, which allow more flexibility to make changes throughout, is beginning to be put into action. This trial aims to accelerate the pace of ALS research and may prove to be a template for other rare diseases as well, experts said.

The design offers advantages for each key stakeholder in drug development: patients have a lower probability of receiving placebo due to the study's shared control arm; companies get quicker topline results at lower cost; and researchers talk of the study as an "endpoint engine" that could advance their scientific understanding of the disease.

Formidable challenges remain in the way of broader adoption, particularly among leaders of the drug industry. The ALS study uses experimental treatments from small biotechs instead of industry giants, which can afford to prioritize control and pay for their own studies instead.

Even so, it's an exciting advancement in ALS research. Morris, who is also a leader of the patient group I Am ALS, said the trial can bring hope to future patients, even if it comes too late to help her.

"It's not going to save me. I'm not in time for any of this," Morris said. "But, dammit, we have to make it better for the rest."

Merit Cudkowicz understands speed. After 25 years specializing in ALS research, Cudkowicz is fully aware of how ALS changes a patient's life, shortening their life expectancy from decades to just years. The disease has a roughly three-year median survival.

Merit Cudkowicz

Massachusetts General Hospital

That life-changing diagnosis came in May 2018 for Sean Healey, then the CEO of an asset management firm worth more than $8 billion. After resigning from that post, Healey connected with Cudkowicz as he searched for treatment options and realized there was an opportunity to accelerate the pace of research.

Just six months from his diagnosis, Healey raised $40 million to launch a research center at Massachusetts General Hospital. Now, slightly less than a year after being established, the Sean M. Healey & AMG Center for ALS Research is aiming to start the platform trial in the first few months of 2020.

And earlier this month, the Healey Center announced the first five therapies to be tested in the study, including drugs from Biohaven Pharmaceutical and Ra Pharmaceuticals.

Cudkowicz, who leads the study, as well as the Healey Center and Mass General's neurology department, said in an interview the trial aims to have 160 patients for each of five treatments. The primary outcome will be whether or not the drug boosted a functional rating score for ALS after six months. One placebo group will be shared for all treatment arms, and more drugs can be added as the study continues. (Cudkowicz cautioned the plan is nearing finalization with the Food and Drug Administration, so specifics could still change.)

Moving quickly is the trial's aim. Cudkowicz called the study "an endpoint engine" that can help create better outcome measurements, including future surrogate markets, pushing ALS research forward. She estimated topline results to come 12 to 18 months after the study begins, depending on enrollment speed.

But those goals are steps toward the actual mission: developing a cure, Healey wrote in an email to BioPharma Dive.

"Of course, we all understand that the most meaningful measure of success will be the development of effective treatments and ultimately a cure," Healey wrote. "I am convinced that the Platform Trial, along with other initiatives we are supporting, will substantially accelerate the achievement of this ultimate goal."

For the first five biotechs, the decision to participate was a no-brainer. The center and other groups are footing the majority of the cost, leaving the companies' primary expense to simply provide their drug.

Out of about 30 applicants, the Healey Center selected five drugs for the study, most notably Biohaven's verdiperstat and Ra Pharma's zilucoplan. The trial also will test therapies from Implicit Bioscience, Prilenia Therapeutics and Clene Nanomedicine.

Cudkowicz said the typical applicants were small biotechs that "have great ideas but not deep pockets."

The expense of clinical trials limits the ability for biotechs to run multiple studies simultaneously across a range of indications. Instead, companies typically focus on a lead indication, with others following in succession.

"As a small company, we live and die by being able to run efficiently and test our hypotheses," said Irfan Qureshi, Biohaven's vice president of neurology.

For instance, Biohaven's verdiperstat is in Phase 3 testing for multiple system atrophy, and Ra Pharma's zilucoplan is focused on a different neuromuscular disorder called generalized myasthenia gravis.

"We wanted to do the study anyway, but to be honest, we probably wouldn't have gotten to it for years if this had not come along," Ra Pharma CEO Doug Treco said in an interview.

While smaller biotechs have bought into the platform trial and its efficiencies, missing from the list of initial participants are industry leaders. With a market value of about $2 billion, Biohaven is the largest company involved.

Cudkowicz noted larger drugmakers have shown interest in roundtable discussions that shaped the trial's design, and there's always potential to add additional therapies after the trial starts.

"We're talking to the other ones like Biogen and Sanofi," she said. "They are interested and came to these meetings, but they have the money to do it on their own."

But the main hang-up for these companies is ceding control, said Scott Berry, a senior statistical scientist and co-founder of Berry Consultants, which worked on the ALS platform trial.

"All these companies have people that this is what they do for a living they run trials, and they know how to do it," Berry said. "To hand your drug to somebody else and you don't have control over making sure that happens is uncomfortable for companies, and it's different than what they usually do."

Additionally, with lengthy protocols and many moving parts, the trials are complex and typically require extensive consulting to get off the ground. Running a multi-arm study also brings statistical pitfalls that can make it harder to interpret results.

Beyond ALS, adaptive trials have been started in breast cancer, Alzheimer's disease and glioblastoma. Experts say they anticipate other rare diseases as logical future targets for these studies.

"As these start to get developed and people see them, in most rare diseases there will be people jumping on board," predicted Berry.

Biohaven's Qureshi added that once companies are willing to experiment beyond typical drug development, these trials could be particularly attractive for rare diseases by easing enrollment concerns where there are "not patients growing on trees," he said.

But the ultimate test to get the industry's full attention, Qureshi said, would be such a study yielding an approved drug.

In the meantime, platform trials appear here to stay. Janet Woodcock, the long-time leader of the FDA's drug review center, has been an influential supporter for these study designs. Just this month, the agency finalized guidance on ALS research that advised companies to consider adaptive trial designs.

Earlier this year, Woodcock told BioPharma Dive she believes these types of studies will gain ground as patients become more vocal about how trials are conducted.

Morris said the platform trial has struck her with its compassion. While no patient wants to be put on placebo, it's of particular importance in a disease that progresses as rapidly and severely as ALS, leaving most patients the time to try one study in their life.

She already took her shot, enrolling in a clinical trial that required a three-month observation period before receiving treatment, which then carried a 50% chance of being placebo. "We are humans," she said. "We aren't zebrafish."

Now, she wants to take the baton from "the voices in the graveyard" and pass it onto the next generation. Platform trials may allow her to cover a bit more ground before that hand-off, eventually reaching a day when people might be able to live with ALS, like HIV.

"ALS's day is coming," Morris said.

See the article here:
First-of-its-kind trial in ALS spurs hope for brutal disease - BioPharma Dive

Modern retroviral therapies have done a good job of turning HIV/AIDS into a treatable chronic illness, but except in a few rare experimental cases using stem cell transplants, there is no cure. Cure, in this case, meaning that the HIV virus is completely eradicated in the patient. Retroviral therapies cause the virus to become inactive, but they still remain in the body and may potentially reactivate if treatment ends.

Researchers at the University of California, San Diego (UCSD) School of Medicine have now identified a key switch that has the potential to eliminate dormant HIV reservoirs. They published their research in the journal mBio.

This is one of the key switches that the HIV field has been searching for three decades to find, said Tariq Rana, professor of pediatrics and genetics at UCSF. The most exciting part of this discovery has not been seen before. By genetically modifying a long noncoding RNA, we prevent HIV recurrence in T-cells and microglia upon cessation of antiretroviral treatment, suggesting that we have a potential therapeutic target to eradicate HIV and AIDS.

Rana and fellow researchers utilized genome-wide expression analysis of long noncoding RNA (lncRNA) in specialized immune cells called macrophages that had been infected with HIV. Macrophages promote inflammation, stimulate the immune system and remove foreign matter. Usually, lncRNAs dont behave the way other RNAs do, which is to say, RNAs deliver DNA codes for proteins. The lncRNAs are involved in controlling which genes are switched on or off in a cell.

The researchers focused on a single lncRNA called HIV-1 Enhanced LncRNA (HEAL) that is found in higher amounts in HIV patients. The researchers believe this is a recently emerged gene that regulates HIV replication in immune cells, including macrophages, microglia and T-cells.

They ran experiments that silenced HEAL or removed it using CRISPR-Cas9 gene editing, which resulted in HIV no longer recurring when antiretroviral treatment was halted. The researchers expect to continue to confirm the data in animal models.

Our results suggest that HEAL plays a critical role in HIV pathogenesis, Rana said. Further studies are needed to explain the mechanism that leads to HEAL expression after an individual is infected by HIV, but this finding could be exploited as a therapeutic target.

This appears to be a major finding, but there have been others recently as well. In August, researchers from the University of Texas Medical Branch at Galveston discovered that the protein BRD4 has a significant role in regulating the production of new copies of the HIV gene. The research group, led by Haitao Hu, assistant professor of microbiology and immunology, designed, synthesized and studied several small molecules to selectively program BRD4 to suppress HIV. They then picked a lead compound, ZL-580, which significantly delayed the reactivation of dormant HIV after antiretroviral therapy was halted.

Like the research published by the UCSD team, this appears to have the potential to be a drug therapy to eradicate the virus, or at least better deal with resistant strains of the disease.

HIV still affects about 37 million people worldwide and approximately 1 million people die annually from HIV-related causes. Treatment typically involves a cocktail of antiretroviral therapy which HIV patients take their entire lives.

Read the original here:
New Research Suggests a Cure for HIV Could be on the Horizon - BioSpace

WASHINGTON--(BUSINESS WIRE)--Greenleaf Health, Inc., a leading Food and Drug Administration (FDA) regulatory consulting firm, today announces that the firm has expanded its portfolio of services to guide companies developing cell and gene therapy products. Greenleafs team of regulatory experts will be led by Karen Midthun, M.D., former Director of the FDAs Center for Biologics Evaluation and Research (CBER), and John Taylor, former FDA Counselor to the Commissioner and Principal Deputy Commissioner.

REGULATORY LANDSCAPE: Cell & Gene Therapy

The rapidly evolving fields of cell and gene therapy offer the possibility of novel treatments, and perhaps ultimately cures, for devastating and intractable illnesses. In response to what the FDA has called a "turning point in the development of these technologies and their application to human health, new policies have been introduced to address the development of safe and effective cell and gene therapies.

With innovation often comes uncertainty. In the case of cell and gene therapy products, the FDA has raised concerns about developers operating outside of the existing regulatory paradigm. To prevent this, the FDA has clarified the regulatory framework for regenerative medicine products and announced near-term enforcement actions aimed at ensuring compliance by companies developing and manufacturing cell and gene therapies.

Greenleafs expanded services support companies striving to introduce cell and gene therapy products to patients. The firms team of experts has a robust blend of technical skill and FDA institutional knowledge that spans all therapeutic areas and quality, manufacturing, and compliance systems. By working cross-functionally, Greenleaf ensures that clients have the comprehensive, specialized support needed to understand and navigate the complex regulatory landscape for cell and gene therapies.

FULL-SERVICE SUPPORT

Members of Greenleafs Drug and Biological Products Team work together with the firms Product Quality, Manufacturing, and Compliance Team to deliver guidance on cell and gene therapy products.

Product Development & Review

With the expert direction of Karen Midthun, M.D., Greenleafs team of advisors assists sponsors of cell and gene therapies by optimizing FDA interactions and submissions to support development and regulatory review. Greenleaf also helps sponsors understand and respond to the FDA requirements applicable to various cellular products, and provides guidance to sponsors of cell and gene therapies to treat rare and ultra-rare diseases on ways to maximize trial design using appropriate clinical endpoints and natural history study data to aid efficient product development.

Quality, Manufacturing & Compliance

Greenleafs Product Quality, Manufacturing, and Compliance Team, led by John Taylor and supported by the firms network of independent compliance experts, offers credible, informed guidance to help manufacturers of cell and gene therapies comply with the FDAs multiple current GXP regulations. Greenleaf experts provide strategic and technical support for establishing manufacturing and quality controls; pre- and postapproval inspection readiness; compliance assessments; evaluating and responding to FDA regulatory correspondence; and engaging with CBERs Advanced Technologies Team.

UNMATCHED EXPERTISE

Greenleaf is comprised of experts with a combined total of more than 250 years of FDA experience. The firms team of advisors demonstrates unmatched levels of skill in its specialties of drug and biological products and product quality, manufacturing, and compliance. Greenleafs Cell and Gene Therapy Team, led by Dr. Karen Midthun and John Taylor, is guided by decades of regulatory experience in senior FDA positions, global public health organizations, academia, and industry.

Karen Midthun, M.D.Principal, Drug & Biological Products

Dr. Midthun contributes specialized insight informed by her regulatory, research, and clinical experience to FDA-regulated entities developing cell and gene therapies. Dr. Midthun joined Greenleaf following a distinguished 28-year career in public health, of which 22 years were dedicated to the FDA.

An infectious disease physician by training, Dr. Midthun most recently served as the Director of the FDAs Center for Biologics Evaluation and Research (CBER). During her FDA tenure, Dr. Midthun played a critical role in facilitating policy and technology development in the areas of cell, tissue, and gene therapies, blood products, and vaccines.

John Taylor, J.D.President, Greenleaf Health, and Principal, Compliance & Regulatory Affairs

Taylor has held many high-profile positions at the FDA, as well as senior leadership roles within industry. Taylors wealth of regulatory experience, robust technical skills, and unique strategic perspective are unmatched. Clients working with Greenleafs Product Quality, Manufacturing, and Compliance Team benefit from Taylors vast FDA institutional knowledge.

Taylor joined Greenleaf following a distinguished 20-year career at the FDA, where he served in multiple leadership positions, including as the FDAs Acting Deputy Principal Commissioner, FDA Counselor to the Commissioner, Acting Deputy Commissioner for Global Regulatory Operations and Policy, and Associate Commissioner for Regulatory Affairs.

ABOUT GREENLEAF

Greenleaf Health provides strategic and technical guidance to pharmaceutical, biotechnology, and medical device companies researching, developing, and manufacturing innovative solutions to pressing global public health challenges.

The firm includes former leaders and regulatory professionals from the FDA, Capitol Hill, top global pharmaceutical and medical device companies, leading law firms, and the top U.S. biotechnology trade organization. Greenleafs blend of former FDA officials and industry experts provides a unique set of capabilities when advising entities regulated by the FDA.

For more information on Greenleafs cell and gene therapy services and Greenleaf Health, visit greenleafhealth.com.

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Greenleaf Health Expands Services to Support Cell & Gene Therapy - Business Wire

By Murielle Gonzalez 26-Sep-2019

Design and Build | Pharmaceuticals

Expansion follows the success of first six cleanrooms built at the large-scale manufacturing centre in Stevenage

The centre has been fully operational since April 2019 and achieved MHRA licensure in August 2019

The UK's Cell and Gene Therapy Catapult (CGT Catapult) has completed six additional modules of cleanroom space at its manufacturing centre in Stevenage. The first collaborators are expected to start benefitting from this new space at the beginning of 2020.

CGT Catapult said the centre has seen increasing demand, which accelerated the planned build-out of the expansion phase with an additional six cleanrooms in the already constructed space on the second floor of the building.

The centre has been fully operational since April 2019 and achieved MHRA licence in August 2019. Depending on the process, each module can accommodate 20 parallel autologous cell processes or a bioreactor process up to 1,000L bioreactors for allogeneic cell or viral vector manufacture.

Dr Ian Campbell, Interim Executive-Chair, Innovate UK, for UK Research and Innovation, commented: In its first year and a half, the Cell and Gene Therapy Catapult manufacturing centre has gone from strength to strength and has established itself as a crucial part of the UKs medicines infrastructure.

Each module can accommodate 20 parallel autologous cell processes or a bioreactor process up to 1,000L bioreactors for allogeneic cell or viral vector manufacture

"This world-leading centre is bringing manufacturing of pioneering treatments closer to patients and will complement the new Advanced Therapy Treatment Centres. By bridging the gap between scientific research and commercialisation of the medicines of the future, this centre, which we are proud to support, will play an ever-greater role in this vital sector.

At the centre, CGT Catapult provides the infrastructure and expertise to enable companies to develop their manufacturing capabilities and systems for large scale, commercial cell and gene therapy supply.

Companies currently collaborating at the centre are Adaptimmune, Autolus, Cell Medica, Freeline Therapeutics and TCR2 Therapeutics.

Located within the rapidly growing European cell and gene therapy cluster in Stevenage, the manufacturing centre, alongside wider CGT Catapult initiatives, reinforces the UK Governments ambition to support the rapidly growing global cell and gene therapy industry in the United Kingdom.

The centre is backed by over 75 million of funding, including investment from the UK Governments Industrial Strategy Challenge Fund; the department of Business Energy and Industrial Strategy, from Innovate UK, the UKs innovation agency, and from the European Regional Development Fund.

CGT Catapult was awarded an additional 3.36m in funding from the European Regional Development Fund (ERDF) and 12m Medicines Manufacturing Industrial Strategy Challenge Fund to further support this expansion.

We are very pleased to be able to double the existing capacity of the CGT Catapult manufacturing centre with help from our partners Innovate UK and the European Regional Development Fund," said Keith Thompson, CEO, Cell and Gene Therapy Catapult.

Thompson pointed out that the growth of this centre is integral to the success of the industry in the UK. "We can now collaborate potentially with up to a further six companies to support them in developing their GMP manufacturing processes, preparing for large scale and commercial supply," he said.

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Cell and Gene Therapy Catapult doubles capacity at UK site - Cleanroom Technology

RESEARCH TRIANGLE PARK Fennec Pharmaceuticals Inc. has appointed Jodi Cook to its board of directors the company announced in a statement Tuesday.

Cook is currently the head of gene therapy for PTC Therapeutics Inc., a global biopharmaceutical company. Before beginning her work at PTC, she helped found Agilis Biotherapeutics, a clinical-stage company focused on gene therapies, where she also served as chief operating officer.

Jodi Cook

On behalf of the board of directors, we welcome theaddition of Jodi Cook, saidKhalid Islam, Fennecs chairman, in a press release. She brings extensive scientific, clinical and executive business experience to the Company. Her background and track record of success will enhance our team as we further advance the commercialization and development strategy of PEDMARK.

PEDMARK is a therapy intended to help children with hearing loss as a result of platinum-based chemotherapy. There is currently no preventative measure for this type of hearing loss. Fennec aims to complete its NDA submission for the treatment in early 2020 with a potential commercial launch of PEDMARK in the second half of 2020.

I am delighted to be joining Fennecs board of directors at this key point of development for PEDMARK, said Cook in the press release. Cisplatin induced hearing loss is a significant unmet medical need, a solution to which has evaded medicine for decades.

This story is from the North Carolina Business News Wire, a service of UNC-Chapel Hills School of Media and Journalism.

Excerpt from:
Fennec Pharma names gene therapy exec to its board - WRAL Tech Wire

WEST CHESTER Nathanael Ogden is your average 13-year-old. He has a younger brother, Jackson. He likes to read and play Dungeons & Dragons, enjoys board games and logic puzzles, and is running for vice president of his eighth-grade class at Peirce Middle School.

He also has something the majority of his peers dont have: a white cane. Nathanael has lost 95 percent of his vision over the last 10 years. But theres one more important thing he has: the spirit of a fighter.

Before he was born, Nathanael was diagnosed with Bardet Biedl Syndrome (BBS,) a rare genetic disorder.

Never heard of Bardet Biedl Syndrome? You're not alone. It is a rare syndrome that affects about 1 in 250,000 people in North America. According to bardetbiedl.org, people with BBS have a defect in the way their cells communicate with each other. It's a complex syndrome with a wide range of symptoms and a lot of variation from person-to-person. However, usually, people with BBS have low muscle tone, impaired kidney function, hampered senses, and vision loss.

Nathanael's parents, Tim and Catherine Ogden, first became aware that something was wrong with their baby during a 26-week ultrasound. The scan revealed that his kidneys were three times larger than they should have been, which Tim says is also indicative of a fatal kidney disease.

"We were told he wasn't going to live," said Tim Ogden.

The couple went to Children's Hospital of Philadelphia for a follow-up and doctors concluded that the baby most likely had BBS.

"They couldn't say definitively, but they were like 'Yeah, we think we know what this is.' Honestly, it was a huge blessing because most people will spend years trying to get a diagnosis for their child. It made a big difference."

Enlarged kidneys weren't the only symptom of BBS for Nathanael.

"Cilia are essentially the radio antennas of our cells. They are responsible for sending and receiving signals from cell to cell," explained Tim.

"When they break down, messages get garbled, or they don't get passed and, his kidneys didn't stop developing. He was also born with 24 digits for the same reason. His body made a pinky but didn't receive the message to stop, so it made another one. He was born with six toes on each foot, and seven fingers on his left hand."

Nathanael also had to deal with poor muscle development, speech problems, and dietary issues. All things he works on with pure grit and determination, and the aid of occupational, physical, and speech therapists. Nathanael has also learned to read braille and how to use a cane to help him navigate while walking.

"You never find him feeling sorry for himself or discouraged. It's always just 'This is what I have to do. This is my next task'" Tim says of Nathanael.

And, preparing for the next task he is. On Sunday, Sept. 29, Nathanael and Tim will tackle the third annual Rocky Ride for Bardet Biedl Syndrome. The trek begins at Uptown Worthington in Malvern, and winds through the Chester Valley Trail to the Schuylkill River Trail concluding at the Philadelphia Museum of Art. And, of course, the journey would not be complete without a triumphant run up the famous art museum steps.

The money raised from the Rocky Ride goes to support the Clinical Registry Investigating Bardet Biedl Syndrome (CRIBBS.) The registry consists of slightly over 500 people in the world diagnosed with BBS and helps researchers to develop effective and targeted treatment for those with the syndrome.

In the first year, the Rocky Ride raised $25,000. In year two, it raised nearly $35,000.

Funds raised from the Rocky Ride also support gene therapy research at the University of Iowa.

"Researchers have identified 23 genes associated with BBS. When research first began, there was only one. Four of them account for roughly 80 percent of people with symptoms of BBS. Those four genes are now part of a standard genetic testing panel which allows for a much earlier diagnosis. The earlier the diagnosis, the earlier services can be secured, and a plan can be put in action," said Tim.

"All of the research that has been done is exciting. When Nathanael was born, it felt like there was nothing and now, what we've been able to do over 13 years ... It feels like there's a lot of hope for people now."

Preparing for the task of riding 30 plus miles is a challenge. Tim and Nathanael spent a good part of the summer in training. They use a tandem bike for each of their training rides, which last anywhere from 15 to 25 miles per trip. Physical activity is a crucial part of Nathanael's care. Another symptom of BBS is the body's inability to recognize when it is full from food, so his parents keep a close eye on his diet and physical activity. Nathanael takes medicine to control his appetite.

"We've ridden a total of 165 miles since August in preparation for the Rocky Ride," said Tim.

For Nathanael, he enjoys the time with his father and feels a strong sense of accomplishment. "I like being able to do the bike ride itself. It's nice to be able to do exercise such as this," said Nathanael.

One hundred sixty-five miles is a lot of ground to cover. How does he find the energy?

"I don't know," he laughed.

All of Nathanael's senses are degenerating to some degree, with vision loss being the most severe.

According to his father, in first grade, he could see. Nathanael is now legally blind. He does the majority of his schoolwork on a computer screen with assistive technology.

"All of it has to be magnified and high contrast. It gets harder and harder, and then the question is - at what point is it more work to have him be able to see the screen as opposed to transitioning him to braille?" said Tim.

The Ogdens are humbled by the community support they have received.

"The biggest thing for anyone in the community to do is just to be there. A big part of having a child with a rare disease is the isolation. The feeling that no one knows about this, that no one understands," said Tim.

Families from across the West Chester Area School District are joining them for the ride.

"We're going to be barreling down the Schuykill River Trail - all 30 of us in red T-shirts to show that Nathanael doesn't have to do this alone. It's a big deal."

One repeat rider is Joe DiAntonio, Nathanael's principal at Peirce Middle School. DiAntonio will be riding with his son, who is a seventh-grader at Stetson Middle School.

"Nathanael is so special to our school," said DiAntonio. "He is everything that is right about Peirce. We want to support him in any way that we can. His attitude is so positive and the way he interacts with people. You just want to be around him."

Despite Nathanael's positive attitude, do Tim and Catherine ever question why this happened to their child?

"Absolutely. You can't get away from that. He lost the lottery. It was this totally bizarre thing that my wife and I had these genes that we didn't know we had. It's hard as a parent. You always want the best for your child."

For Tim, it was important to find something that he could do with his son and for other families with BBS. He helped start and is currently the president of the Bardet Biedl Syndrome Foundation, which provides support for families affected by the syndrome in North America and around the world.

"We're not really raising money for Nathanael. We are raising money that's going to benefit people in the future who have this syndrome and so he gets the feeling that there is something he can do. It's not about what he can't do, but what he can do to make the world a better place and help others," said Tim.

Tim and Nathanael drew inspiration for the Rocky Ride from the Rocky movies. The main character's attitude of determination is ever-present in Nathanael.

"Rocky doesn't win that first fight, but he still does it," said Tim. "That's the message that I want for Nathanael."

Nathanael's moto? "Keep pushing through it and try to find ways to be happy."

For more information on Bardet Biedl Syndrome, or to donate to the Rocky Ride, visit http://www.bardetbiedl.org/rockyride.

See original here:
Eighth-grader fights for himself and others with rare genetic syndrome - Daily Local News

RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--Synteract, an innovative CRO partnering with biotech and pharma companies to bring new medicines to market, has hired two experienced leaders to further strengthen its Centers of Development in Oncology and Rare/Orphan diseases. Given the enhanced strategic solutions that often result from the intersecting expertise of Synteracts centers of development, clients will derive further benefit from the therapeutic knowledge and clinical development acumen gained from these key additions.

Hassan Aly, M.D., Ph.D., has joined Synteract as senior medical director supporting Synteracts Oncology Center of Development and Derek Ansel has joined as director of Rare and Orphan Disease Development. Both are clinical strategists with multidisciplinary experience in developing and operationalizing global therapeutic strategies.

Rare disease, oncology, and hematology clinical trials pose unique challenges that require the ability to think broadly while applying very specific trial strategies, said Steve Powell, Synteract CEO. Both Hassan and Derek bring a wealth of knowledge, expertise, and creativity, coupled with a passion to help patients and families, that will help us to further advance trials in the most complex and acute areas of need.

Dr. Aly is a certified hematologist with over 20 years experience in oncology/hematology medical research and patient treatment, including nine plus years in the clinical research and pharmaceutical industry. He brings deep therapeutic expertise across all phases of oncology/hematology drug development, having consulted on complex study design, conduct, monitoring, and submission. Prior to joining Synteract, he worked at AstraZeneca, Boehringer-Ingelheim, and leading CROs.

In addition, Dr. Aly has authored or co-authored 105 papers on the treatment and biology of malignant hematological diseases in peer-reviewed medical periodicals and made more than 70 presentations at British and European conferences.

Prior to joining Synteract, Derek Ansel was a clinical strategy lead at the Center for Rare Diseases and a co-founder of the Gene Therapy Working Group at a leading CRO. Ansel has a breadth of clinical research experience spanning from the laboratory through clinical monitoring and project management, across Phases I-IV. He will work in a multidisciplinary, cross therapeutic team, where he will apply extensive gene and cell therapy expertise developed from his work in several liver-targeted gene therapy programs.

Ansel frequently speaks at industry events. He is a member of the ACRP and holds a Certified Clinical Research Associate (CCRA) credential. He is an IRB member at the Thomas Jefferson University in Philadelphia and is also a contributing member of Cochrane, a non-profit organization that promotes evidence-based decision making in healthcare.

Dr. Frank Santoro, chief medical officer for Synteract, commented, Hassan and Derek bring unique skillsets to our centers of development with a combination of medical, therapeutic, and operational know-how related to running complex trials. This expertise, along with their crossover experience in multiple therapeutic areas, offers exceptional insight to our clients looking to partner with Synteract for their clinical development needs.

About Synteract

Bringing Clinical Trials to Life represents Synteracts commitment to engage with drug developers, patients, investigators, and regulatory experts to bring insights to action and make better therapies a reality. Synteract supports biotech and pharma companies across all phases of drug development to help bring new medicines to market. Synteract has conducted nearly 4,000 studies on six continents and in more than 60 countries. It has contributed to more than 240 product approvals. Synteract offers notable depth of therapeutic expertise in oncology, dermatology, general medicine, neuroscience, pediatrics, and rare and orphan diseases. Connect on LinkedIn and Twitter.

Original post:
Synteract Augments Leadership Team for Oncology and Rare Disease Centers of Development - Business Wire

NetworkNewsWire Editorial Coverage

NEW YORK, Sept. 24, 2019 /PRNewswire/ -- Advances in biotechnology look to deliver promising new treatment options against breast cancer. Independent researchers have found that Genprex Inc.'s (NASDAQ: GNPX) (GNPX Profile) TUSC2 prevented tumor growth against triple-negative breast cancer. Eli Lilly and Company (NYSE: LLY) is focusing on those patients with the greatest need and, in the process, has developed a new breast-cancer treatment option. Roche Holding (OTCQX: RHHBY) is expanding the use of its biotech to both identify and treat patients with challenging breast cancers. AbbVie Inc. (NYSE: ABBV) has been rapidly increasing its research, targeting more than 15 different types of cancer. AstraZeneca (NYSE: AZN) has seen hopeful results against metastatic breast cancer as it works in collaboration with a Japanese firm.

To view an infographic of this editorial, click here.

Battling Breast Cancer

Breast cancer is one of the most widely prevalent cancers in the world. Accounting for a quarter of all cancers in women worldwide, as well as a number of cases in men, it kills over half a million people every year.

Treatment for breast cancer varies, based on both the patient's circumstances and type of cancer. The development of treatments to suppress or even eliminate cancer has led to a high survival rate in wealthy countries, with around 85% of patients in the United States and United Kingdom surviving for at least five years from diagnosis. But even in these countries, survival depends upon the exact form of the cancer, how far it has progressed and whether a treatment has been developed for that particular form. Cancer is a difficult disease to defeat, and survival can depend upon the ability of scientists to counter a specific genetic defect in a specific set of cells.

Good News for Triple-Negative Breast Cancer Patients

Because cancers are so varied and challenging to tackle, every individual win is worth celebrating. That's why recent news relating to triple-negative breast cancer (TNBC) has brought excitement both for independent researchers and for Genprex Inc. (NASDAQ:GNPX), a clinical-stage, gene-therapy company whose tumor suppressor candidate 2 (TUSC2) was found to prevent tumor growth in TNBC.

TNBC covers a variety of cancers that do not express three specific types of receptor proteins. Most hormone therapies for breast cancer target one of these receptors, so tackling a cancer that doesn't feature any of those receptors is more difficult. TNBC is an extremely aggressive subtype of breast cancer associated with poor prognosis and high mortality rates. The lack of targeted treatment for triple-negative breast cancer makes it a particularly feared diagnosis. Because up to 20% of breast-cancer patients are fighting TNBC, finding therapies that effectively fight these forms of cancer is essential.

Recently TNBC patients have heard good news, thanks to research published in Nature. This research shows that TUSC2 prevented the growth of tumors in TNBC. And with Genprex already working on cancer treatments using TUSC2, including its lead drug candidate, Oncoprex(TM) immunogene therapy, the new research appears to validate the company's focus and direction.

Currently Genprex is conducting clinical and preclinical research to evaluate the effectiveness of TUSC2 when combined with targeted therapies and immunotherapies for non-small cell lung cancer. Existing preclinical data also suggest that TUSC2 may be effective against glioblastoma, head and neck cancer, kidney cancer and soft-tissue sarcomas. This new independent study raises the possibility that TUSC2 expression may also be used to treat this most aggressive subset of breast cancer.

"The results of the study evaluating TUSC2 for the treatment of triple-negative breast cancer are encouraging," said Genprex chairman and CEO Rodney Varner. "We believe that the data reported in this Nature article by independent researchers supports our belief that TUSC2 may be effective to treat a variety of cancers, including some of the most-deadly types of cancer."

This new report is a particularly encouraging moment for Genprex, as the study doesn't come from the company's own research work but instead verifies its value through an independent source. With multiple teams and researchers in multiple locations all evaluating and studying TUSC2 as a potential source for cancer treatment, the evidence is mounting that this approach may provide something new and valuable for doctors and patients.

Gene Therapy to Tackle Breast Cancer

Like many of the most promising cancer treatments, Genprex's TUSC2 treatment is a form of gene therapy.

A technology less than 50 years old, gene therapy remains on the cutting edge of modern medicine. The approach involves delivering new genetic material into the patient's body, where it is absorbed by cells. This rewrites the code of those cells, changing how they grow, reproduce, spread and die.

The growth and death of cells is fundamental to the challenge of cancer. Damaged genetic material leads cancer cells to develop in harmful ways and spread through the body, sometimes at a rapid rate. Treatments such as Genprex's Oncoprex can be used to write over the harmful DNA code and introduce a new gene. The result is that cancer itself is rewritten to reduce its harm.

Genetic treatments vary in the way they affect the body's cells. Some directly destroy cancerous cells. Others slow their reproduction, thereby reducing the spread of unhealthy tissue throughout the body. Other treatments make cells more susceptible to forms of treatment that might otherwise be ineffective.

TUSC2 treatment falls into both categories, helping cancer dells to die and preventing the growth of cancerous tumors. If successfully used, this approach could halt the growth of existing cancers and their spread through the body. TUSC2 is a vital element of treatment if cancer is to be prevented from running rampant through patients.

Genprex's Oncoprex therapy delivers cancer-fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles. The nanovesicles are then administered intravenously. They flow through the bloodstream until they locate and are absorbed by the cancer cells. The TUSC2 gene then expresses proteins that are missing or in short supply, bringing the damaged cells closer to normal behavior. By rewriting the very fundamentals of the body, such treatments may save lives.

Disruptive Technology for the Medical Sector

"Disruptive" isn't a term that's often applied to medicine, where the aim is to avoid disrupting the health of the human body. But technology such Genprex's Oncoprex immunogene therapy is disruptive precisely because it could improve doctors' ability to preserve health. It may also change the course of cancer, as new technology often opens up new possibilities.

Since its first successful use in the 1980s, gene therapy has rightly been presented as a disruptive technology. It deals with health on a basic level, altering the building blocks of life. This is why Genprex was among the companies presenting at the 4th Annual Disruptive Growth Conference in New York this September. Oncoprex's multimodal mechanism allows it to tackle cancer in a number of different ways, reducing the cancer's spread, encouraging the death of cancer cells and modulating the response of the immune system to fight cancer. It can block mechanisms that cause resistance to other anti-cancer drugs and so increase the effectiveness of a broader course of treatment.

As both treatments and the business models of companies behind them develop, the battle against cancer is being transformed.

Founded more than 140 years ago, Eli Lilly and Company (NYSE:LLY) is one of the oldest players in the field of cancer treatment, a company that has spent more than a century making medicines to help people around the world. Lilly uses predictive and prognostic biomarkers to work out how a tumor is likely to behave and how it will respond to potential therapies. This approach allows more targeted treatment designed to tackle the patient's specific cancer, thereby increasing the likelihood of success in any given case. LLY's Verzenio treatment has been shown to be effective in cancer treatment, encouraging cell death and reducing the spread of tumors. The company's strategy is a targeted one, concentrating on those cancer patients with the greatest need, and so providing the greatest possible impact from its medicines.

Another long-established company, Roche Holding (OTC:RHHBY) was one of the first to provide targeted treatments such as those offered by gene therapy. The world's largest biotech company, Roche has poured a huge amount of resources into biopharmaceuticals and is a global leader in cancer treatments. The company recently announced the expanded use of its technology to identify TNBC patients and so to provide them with the targeted treatment they need to fight their cancer. The company's support for cancer patients extends beyond better medicine for those in wealthy countries. Roche is a supporter of a health-care approach in South Africa that takes treatment to the rails, providing mobile health-care facilities and services that include breast cancer screening for people who might otherwise lack access.

A research-driven biopharmaceutical company, AbbVie Inc. (NYSE:ABBV) is working on new treatments for more than 15 different cancers, including forms of breast cancer. The company's wide-ranging work means that insights from one area can affect work in another, allowing greater progress in the overall understanding of cancer and the development of specific, targeted treatments. AbbVie's oncology research has increased dramatically over the past six years, often through partnerships with other companies, allowing a rich cross-fertilization of ideas.

Based in the United Kingdom, AstraZeneca (NYSE:AZN) benefits from its proximity to some of the finest minds in the world and an elite recruitment pool. Like AbbVie, AstraZeneca has been making breakthroughs in a wide range of cancer types. Research in collaboration with a Japanese company has recently led to promising results for the treatment of metastatic breast cancer, as the company works to improve survival rates and quality of life for breast cancer patients.

With the attention of so many big players in the biopharmaceutical industry, promising treatments for cancer are developing fast.

For more information about Genprex, please visit Genprex Inc. (NASDAQ:GNPX).

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Gene Therapies with Potential to Conquer Tough-to-Tackle Breast Cancers content= - PR Newswire UK

-- Webcast conference call to be held on Friday, Oct. 4, 2019 at 8:00 a.m. Eastern Time --

CAMBRIDGE, Mass., Sept. 25, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Friday, Oct. 4, 2019 at 8:00 a.m. Eastern Time (ET), it will host a webcast and conference call to present the 9-month functional results from the SRP-9003 gene therapy trial to treat Limb-girdle muscular dystrophy Type 2E, or beta-sarcoglyanopathy.

The presentation will include slides and will be webcast live under the investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-and-presentations and will be archived there following the call for one year. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. The conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers. The passcode for the call is 4986359. Please specify to the operator that you would like to join the "Sarepta hosted LGMD results call."

AboutSarepta TherapeuticsSarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for Limb-girdle muscular dystrophy diseases (LGMD), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, please visit http://www.sarepta.com.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics to Announce 9-Month Functional Results from the SRP-9003 Gene Therapy Trial to Treat Limb-girdle Muscular Dystrophy Type 2E, or...

For the latest events and activities happening in Greenwich, turn to For the record. To have your event included, submit a description, date, time, price and contact information. Photos are welcome. Drop us an email about your latest goings-on at gtcitydesk@scni.com.

Try Tai Chi

Tai Chi is a relaxing exercise that can loosen joints, improve balance and teach graceful movements to music. Fun and no pressure classes are held at 9 a.m. Thursdays in the auditorium at the First Congregational Church Auditorium on Sound Beach Avenue in Old Greenwich. The cost is $10 per one-hour session. Newcomers welcome. For info, call Joe at 203-504-4678.

Special Education parent forum

The Stamford JCC will present a Special Education Parent Forum: Pursuing a Specialized School Placement at 7 p.m. Thursday at 1035 Newfield Ave., Stamford. Free and open to the public. Registration is recommended with Liza Fahey at lfahey@stamfordjcc.org or 203-487-0946. Workshop speakers will include Lawrence Berliner, special education law attorney in Westport; and Clay Kaufman, head of school, and Sue Rappaport, assistant director of admissions and placement, at Eagle Hill School in Greenwich.

Farm to Table Dinner

Chef Geoff Lazlo brings his Farm to Table Dinner series to his hometown of Greenwich at the Greenwich Botanical Center, 130 Bible St., Cos Cob. The elegant four-course dinner kicks off with a signature cocktail and passed hors doeuvres as guests are treated to highlights of the propertys magnificent history, followed by a seated dinner featuring key ingredients of herbs and vegetables locally grown. Dinners will be from 6 to 9 p.m. on Sept. 26 and Oct. 3. For more information and tickets, visit greenwichbotanicalcenter.org.

Qigong at the GBC

Qigong expert Donna Bunte teaches classes at 10 a.m. Fridays at the Greenwich Botanical Center, 130 Bible St., Cos Cob. With roots in Chinese medicine, philosophy and martial arts, qigong is viewed as a practice to cultivate and balance qi (chi), translated as life energy. Qigong practice involves moving meditation, coordinating slow flowing movement, deep rhythmic breathing, and a calm meditative state of mind. For information, visit greenwichbotanicalcenter.org.

Abilis Has Talent Show

The third annual Abilis Has Talent Show is a star-studded evening fun for all ages. The event is held at the First Presbyterian Church at 1 W. Putnam Ave., Greenwich, on Friday. Tickets are $25 with advance reservation or $30 at the door. Register at abilis.us/calendar. Abilis is a nonprofit organization that supports more than 700 individuals with special needs and their families annually from birth through adulthood.

Enjoy Oktoberfest

Enjoy beer and brats for Oktoberfest on Friday at the St. Lawrence Club. Its one of the favorite Family Night Dinners as Chef Tony prepares an authentic German-style feast with beer & cheese soup, schnitzel, sauerkraut, potato salad and strudel. Cost is $25 for adults, $10 for kids. No tax, no tip. Society members get a free drink. The St. Lawrence Club is at 86 Valley Road, Cos Cob. To RSVP, visit http://www.stlawrencesociety.com/events or call 203-618-9036.

Bruce closes temporarily

The Bruce Museum will then be closed through Friday to facilitate work in the main galleries. When it reopens Saturday, admission will be free to all visitors through Jan. 31 while the main gallery spaces are renovated. During the renovation phase, the Permanent Science Gallery will remain open, as will the Bantle Lecture Gallery, Education Workshop, and Museum Store. The galleries will reopen Feb. 1, with the installation of major new art and science exhibitions. Visit BruceMuseum.org for more information.

Farmers Market

The Greenwich Farmers Market runs every Saturday from 9:30 a.m. to 1 p.m. into the fall in the Arch Street commuter lot. Enjoy fresh Connecticut-grown produce all season. Over a dozen farm vendors will be in attendance. The parking lot at Arch Street and Horseneck Lane is off Exit 3 of I-95. For more information, visit http://www.greenwichfarmersmarketct.com/.

Cocktails & Comedy

There will be a cocktails and comedy fundraiser to venefit The Undies Project from 7 to 10 p.m. Saturday at St. Catherine of Siena in Riverside. To purchase tickets and for more info, visit e.givesmart.com/events/dWy/.

Greenwich Symphony Orchestra opens season

The Greenwich Symphony Orchestra will feature five concerts during its 62nd season, beginning Saturday and Sunday. The opening concerts will feature Bartok, Divertimento for Strings; Dittersdorf, Concerto for Harp, with Barbara Allen on harp; Wieniawski, Violin Concerto No. 2, with Edita Orlinyte on violin; and Debussy, Iberia. Concerts are at the Greenwich High School Performing Arts Center, 10 Hillside Road, at 8 p.m. on Saturdays and 4 p.m. on Sundays. Tickets are $40 per person, $10 students. For more information, call 203-869-2664 or visit http://www.greenwichsymphony.org.

Puttin on the Dog

Puttin On The Dog, the popular annual fundraising event for Armonk-based Adopt-A-Dog, will be held from 10 a.m. to 4:30 p.m. Sunday in Roger Sherman Baldwin Park in Greenwich. Dogs and cats will be available adoption that day. The event will also feature a silent auction, childrens activities, pet demonstrations, vendors and food trucks as well as canine competitions. Dogs are welcome, as well as people of all ages. For more information about the organization, visit adopt-a-dog.org, call 914-273-1674 or email adoptions@adoptadog.org.

Springsteen at Seventy

The C. Parker Gallery presents Growin Up Bruce Springsteen at 70, a 70th birthday celebration featuring rare photographs, artwork, gold records and signed memorabilia showcasing The Boss. The exhibit will run through Sept. 29 at the gallery at 409 Greenwich Ave. Highlights include rare unpublished 1972 to 1975 photographs from Bruces earliest days performing in the Philadelphia area from the late Phil Ceccola. Debra Rothenberg, author of Bruce Springsteen In Focus 1980 2012, will showcase her photographs. Free. All works are available for purchase. To learn more, visit http://www.cparkergallery.com.

Archery open shoot

The Cos Cob Archers will hold an Open Shoot from 8 a.m. to 1 p..m. Sept. 29 as they open their club to the public and invite all to attend. Open Shoots are held once a month, generally on the last Sunday of each month; bad weather cancels. The club provides loaner compound bows, arrows and instruction for beginners. The cost $20 for shooting adults, $5 for kids under 16, and $10 for non-shooting adults includes lunch of grilled hamburgers, hot dogs and soft drinks. The Clubs trails cover 23 wooded acres and there are more than 40 shooting stations, with both paper and 3D targets. Cos Cob Archers is located at 205 Bible St. For more information, visit http://www.CosCobArchers.com.

Meters for a Cure ERG Challenge

Swim Across America Fairfield County and Greenwich Crew are teaming for the second annual charity rowing event Meters for a Cure ERG Challenge with proceeds supporting Swim Across America Fairfield County and its local beneficiary, the Alliance for Cancer Gene Therapy, the nations only foundation dedicated exclusively to funding cancer gene therapy research. On Sept. 29, hundreds of crew members, their families and friends, will come together to help raise money for cancer research at the Greenwich Water Club at 49 River Road, Cos Cob, and relay against each other on ERG machines (a rowing machine also known as an ergometer). Teams are divided by age group and each participant is asked to raise at least $100. Open to all. Register at swimacrossamerica.org/gwc2019 or on-site that day starting at 7:30 a.m.

Choral Evensong

Join Christ Church Greenwich in celebrating Saint Michael and All Angels with music by Rose, Sumsion, and Bainton sung by the Choir of Men & Boys with members of the St. Cecilia Choir of Girls. The concert will be at 5 p.m. Sept. 29 at the church at 254 E. Putnam Ave. Admission is free. Visit christchurchgreenwich.org/music/ for more information.

Duplicate Bridge Games

Weekly open duplicate Bridge games are held at 12:15 p.m. Mondays at the Greenwich YWCA. The games are sanctioned by the American Contract Bridge League, with masterpoint awards to top finishers. The card fee to play one session is $12. For more information, contact Steve Becker at 203-637-8927.

Exhibit of Bird Mobiles

The Greenwich Art Society Gallery will present a show called Bird Mobiles: Suspension Of Time through Sept. 30 at the Arts Council Building at 299 Greenwich Ave. The mobiles were created by local artist Susan McHale, who creates her art with discarded branches found on the beach. The process of making my Bird Mobiles is like catching an ephemeral moment suspended in time, the hanging flock fly by and seem to continue their journey, she says. Call 203-629-1533 for more information.

Error art installation

The Brant Foundation Art Study Center is presenting Urs Fischer: Error, a solo exhibition of works by Fischer from the last two decades, through Oct. 1 at 941 North St. Featuring some of the artists most notable large-scale sculptures and paintings from the Brant Collections, Error celebrates The Brant Foundations 10th anniversary at its Greenwich space with Urs Fischer, the first artist to present a solo exhibition at there in 2010. To book a tour, visit brantfoundation.org/exhibitions/error/.

ASOG Fall Art Show

As summer fades and fall beckons, the Art Society of Old Greenwich is preparing for its 2019 ASOG Fall Art Show. The exhibit takes place at the Greenwich Botanical Center in Cos Cob from Oct. 1 to Oct. 28, with an artists reception on Oct. 6 from 3 to 5 p.m. Visitors can view the artwork, mingle with the artists, listen to live music and sample refreshments. The Greenwich Botanical Center is open 9 a.m. to 4 p.m. weekdays.Free parking on premises. For more information, contact artsocietyoldgreenwich@gmail.com or visit http://www.asogct.com/fall-show.

Abilis Gardens & Gifts

Starting in the fall, Abilis Gardens & Gifts will be selling holiday gifts and its greenhouse will be full of holiday wreaths and greenery. It is must-shop store offering handcrafted candles, bath products, childrens gifts, jewelry, housewares and gifts, with fresh flowers, plants and micro greens in the greenhouse. Prices range from $5 to $40 and proceeds support Abilis programs. Abilis Gardens & Gifts provides job training for adults with special needs. Hours are 10 a.m. to 5 p.m. Mondays, Wednesdays and Fridays; 10 a.m. to 3p.m. Tuesdays; 10 a.m. to 7 p.m. Thursdays; and 10 a.m. to 2 p.m. Saturdays for October, November and December. Abilis Gardens & Gifts is at 50 Glenville St. and can be reached at 203-531-GIFT (4438). For more info, visit http://www.abilis.us.

An American Story

The Greenwich Historical Society will present a new exhibition on the immigrant experience called An American Story: Finding Home in Fairfield County from Oct. 2 to Jan. 6. The exhibit will include poignant stories of the grit and resilience of immigrants and refugees, including 12 who found home in Greenwich. An American Story is a celebration of the determination, resilience and courage of the human spirit. The stories illuminated in this timely exhibition reach across the world from five continents, shining a light on the ways that refugees and asylum seekers find hope and persevere in the face of daunting challenges for creating new lives in Fairfield County. The landmark exhibition is presented in partnership with the Connecticut Institute for Refugees and Immigrants. The Greenwich Historical Society is located at 47 Strickland Road, Cos Cob. For more information, visit greenwichhistory.org.

Talk on The Bottom of the World

The Bruce Museum Presents, a new series of monthly public programs featuring thought leaders in the fields of art and science, showcases experts on compelling subjects of relevance and interest to members and visitors to the Bruce Museum. The series continues with an event presented in cooperation with Columbia Universitys Lamont-Doherty Earth Observatory called From the Bottom of the World: The Art and Science of Antarctica from 6 to 8:30 p.m. Oct. 3. It will convene experts in the art and science of this unique and fragile continent. Two research scientists from Lamont-Doherty, Jonny Kingslake and Kirsty Tinto, will reveal their new findings, while photographer Rick Sammon, who has twice visited and documented the Antarctic landscape, will remind us of its beauty. Leonard Jacobs moderates. Doors open at 6 p.m. for a reception with light bites and beverages, followed by the panel discussion and Q&A from 7 to 8:30 p.m. Seats are $30 for museum members, $45 for nonmembers. To reserve a seat, visit brucemuseum.org or call 203-869-0376.

Breast Cancer Awareness

To kick off Breast Cancer Awareness Month, Greenwich Town Hall will raise the Breast Cancer Alliances flag at 9 a.m. Oct. 3 with First Selectman Peter Tesei; Dr. Barbara Ward, director of breast care services at Greenwich Hospital, and Mary Jeffery, president of the BCA. The event will continue as Richards of Greenwich hosts the BCAs GoForPink breakfast called Love, Loss and Cancer: An Intimate Conversation with authors Lee Woodruff and Allison Gilbert at 10 a.m. Oct 3. The organizations annual luncheon, which takes place at 11 a.m. Oct. 21 at the Hyatt Regency Greenwich, will feature celebrity chef and restaurateur Todd English. For more info, go to http://www.BCAgoforpink.org, contact Info@breastcanceralliance.org or call 203-861-0014.

Dinner With a Cause

The Social Justice Committee of the Parish of St. Catherine of Siena and St. Agnes is sponsoring its Dinner with a Cause from 6:30 to 8:30 p.m. Oct. 3 at the Church Hall. The featured guest is Cathleen Caron, founder of Justice in Motion, a nonprofit dedicated to service the needs of migrant workers. Come for a light dinner at 6:30, presentation at 7:15 followed by Q & A. Free will offering to benefit Justice in Motion. For information on the nonprofit, visit https://justiceinmotion.org/about-us. RSVP to parkerelizabeth@sbcglobal.net or 203-637-8092.

Outdoor Arts Festival

More than 80 new and returning artists will showcase their work on the grounds of the Bruce Museum on Oct. 5 and Oct. 6, as the Greenwich museum celebrates the 38th edition of its annual Outdoor Arts Festival. The event will feature juried works, including paintings, mixed media in 2D and 3D, drawings and graphics such as digital media, sculpture and photography. There will be food-truck offerings and free art activities for children. Free parking offered in all nearby municipal lots. Festival admission is free to Bruce Museum members and children less than 5 years old; entry is $10 for non-members. Festival-goers can also tour the museum. Fore more info, visit BruceMuseum.org.

YWCAs walk against domestic violence

On Oct. 6, YWCA Greenwich is hosting a communitywide walk to raise awareness and funds for YWCA Greenwich Domestic Abuse Services and education. The family-friendly event takes place at Bruce Park. Registration will open at 8:30 a.m. Members of the community are encouraged to form teams, come with friends and family, and even dogs are welcome. To preregister, go to ywcagrn.org/walkthewalk. Proceeds will support the work of YWCA Greenwich Domestic Abuse Services. October is Domestic Violence Awareness and Prevention Month. YWCA is the state-designated provider of domestic abuse services for victims of domestic violence in Greenwich. If you or someone you know is a victim of domestic abuse, call the YWCA 24/7 hotline at 203-622-0003. For more information about the walk, contact Jackie Stam at 203-869-6501 x102 or j.stam@ywcagreenwich.org.

Teddy Bear Clinic

The Teddy Bear Clinic Greenwich Hospitals largest community event will celebrate its 21st anniversary from noon to 3 p.m. Oct. 6 at 49 Lake Ave. in the Greenwich Medical Building parking lot behind the hospital. Geared for children ages 3 through 12, this popular free event will take place rain or shine. Children and their families are invited to experience the friendly face of medicine at this event, staged as a mini-hospital under the tents. Visit with Greenwich Hospital doctors, nurses, technicians and other staff during this fun, interactive day of learning. Peer through a microscope, climb aboard an ambulance, check out the X-ray machine and test your balance by negotiating an obstacle course. Families can learn about nutritious food, strong bodies, sun protection, sports safety and other healthy habits. Children are encouraged to bring a favorite stuffed animal or doll for an exam. Depending on the diagnosis, dolls, bears and other fuzzy patients may receive splints, stitches, surgery or X-rays. Free parking in the hospitals staff lot on Lake Avenue. For information, call 203-863-3627.

First Sunday bird walks

A First Sunday Bird Walk will be held at Greenwich Point Park from 9 to 11:30 a.m. on Oct. 6, Oct. 13 and Oct. 20 to see the migrating hawks and raptors. Bring binoculars and meet near the main concession stand at the south end of the beach. The Bird Walks are a series of free, year-round, monthly bird walks providing friendly and informative birding. Guides are knowledgeable local naturalists and birders. Everyone from beginner to expert, of all ages is welcome. No fees or registration are required. For more information, visit birdwalk.home.blog/. Co-sponsored by Wild Wings and Friends of Greenwich Point.

Lecture on Classical Americana

The Greenwich Decorative Arts Society will present Classical Americana: The Life and Legacy of Richard Hampton Jenrette, a lecture by Peter M. Kenny, co-president of the Classical American Homes Preservation Trust. The event will be from 1:15 to 3 p.m., with refreshments to follow, on Oct. 7 at the Bruce Museum. Insights will be offered into the numerous houses he restored, his approach to their interior decoration and his role as a collector of classical American furniture from the workshops of New Yorks premier cabinetmakers of the early 19th century. Kenny was the Ruth Bigelow Wriston Curator of American Decorative Arts and Administrator of the American Wing at the Metropolitan Museum. Admission for nonmembers is $25. Space is limited. Reservations required for members and guests by Sept. 30. For reservations and information: email greenwichdecorativearts@gmail.com or visit http://www.greenwichdecorativearts.com.

Blood drives

The American Red Cross urges people of all races and ethnicities to give blood or platelets to help increase the diversity of the blood supply. The vast majority of blood types fall into one of the major blood groups. However, for patients with rare blood types or those who receive regular blood transfusions, blood must be matched closely - beyond the primary A, B, O and AB blood types - to reduce the risk of developing complications from transfusion therapy. The best match may be someone of the same racial or ethnic group. That includes patients who need blood transfusions to treat complications from sickle cell disease, a genetic disease that is most common among people of African descent or Latino descent. Local blood drives will be held from 1:30 to 6:30 p.m. Oct. 7 at Christ Church Greenwich, 254 E. Putnam Ave., and from 9 a.m. to 2 p.m. Oct. 8 at the Boys & Girls Club of Greenwich, 4 Horseneck Lane. Appointments can be made by downloading the free Red Cross Blood Donor App, visiting RedCrossBlood.org, or calling 1-800-RED CROSS (1-800-733-2767). As a thank-you, donors who give blood or platelets during October will be entered for a chance to win one of five $500 gift cards.

Outdoor movie night

As part of the Greenwich Reads Together program, there will be an outdoor screening of the movie Fahrenheit 451 at 7:30 p.m. Oct. 12 on the front lawn of the Greenwich Arts Council at 299 Greenwich Ave. Legendary French director Franois Truffaut brings to life a terrifying care-free future in which Guy Montag (Oskar Werner) questions his actions after meeting Clarisse (Julie Christie) and begins to rebel against society. Free and open to all, but registration is encouraged at http://www.greenwichlibrary.org. For more information, call 203-862-6750 or visit greenwichartscouncil.org. Co-sponsored by Alliance Francaise of Greenwich, The Avon Theater, The Commission on Aging, Greenwich Arts Council, Greenwich Library, and Sebass Events & Entertainment.

Honoring Columbus

The St. Lawrence Society will celebrate Columbus Day on Oct. 14 with an event honoring First Selectman Peter Tesei. Festivities begin with a 9 a.m. flag raising at Town Hall, followed by a breakfast at The Club and ending with cocktails, presentation of awards and an elegant Italian dinner back at the Club at 6:30 p.m. The cost for dinner is $100 per person. The St Lawrence Club is 86 Valley Road, Cos Cob. To RSVP, visit http://www.stlawrencesociety.com/events or call 203-618-9036.

Lush Landscapes benefit

Landscape designer James Doyle will present a program on Dream Locations and Special Places at 11:30 a.m. Oct. 17 at the Belle Haven Club as a benefit for Community Centers Inc., a Greenwich-based social service agency. Doyle, founder and principal of Greenwich-based Doyle Herman Design Associates, has developed an award-winning design business with projects in the United States, Europe and the Middle East. His work combines his horticultural expertise with strong design philosophy to bring to life unique and innovative landscapes. Projects range from small courtyards to large country estates. CCI, which has been serving the community for 65 years, is dedicated to building skills that empower clients to overcome educational, social and economic barriers. Tickets may be purchased on the CCI website at ccigreenwich.org.

Walk/Run for Abilis

Abilis will host its 15th annual Walk/Run for Abilis on Oct. 20 at Greenwich Point Park. This fun family-friendly event includes childrens activities, a Bubble Bus, music, arts & crafts, a 1-mile wheelchair and stroller-accessible walk and a 5k run. This event is one of Abilis largest fundraiser events of the year and attracts hundreds of participants for a morning of fun. Registration is $40 for adult runners; $20 for child runners (ages 11 to 17) and free for children under 11. There is no cost for the walk portion of the event. To register in advance, donate and or volunteer, visit abilis.us/walkrun. Abilis is a nonprofit organization that supports more than 700 individuals with special needs and their families annually from birth throughout adulthood in the area.

Model sailboat regatta

The Old Greenwich-Riverside Community Center will hold its popular annual Model Sailboat Regatta from 1 to 4 p.m. Oct. 20 at Binney Park. Model sailboat enthusiasts of all ages are welcome, with categories for homemade boats, motorized and remote-controlled boats spread out throughout the afternoon. Hundreds of families are expected to attend. Many families set up a picnic for the day as kids run around and enjoy Binney. The OGRCC provides arts and crafts activities and music. The regatta raises money for the Scholarship Program that supports over 70 families in the community each year for everything from soccer to childcare to summer camps. To buy a ticket, visit myogrcc.org. Walk-ins are welcome. For more info, call 203-637-3659.

TAGs grand prix fundraiser

The Transportation Association of Greenwich will hold its fall fundraiser, the Greenwich Grand Prix, from 3 to 5 p.m. Oct. 27 at the RPM Raceway in Stamford. The proceeds of the go-kart racing event will be used to purchase a new 20-passenger vehicle with wheelchair lift. The go-kart racing teams are from Greenwichs top car dealers, who will be competing for the Greenwich Grand Prix Championship. The community is invited to cheer on the teams and support TAG to raise these much-needed funds. Tickets are on sale now. For more information on tickets, teams, donations and sponsorship, visit ridetag.org/greenwich-grand-prix.

Nostalgia dining

A Remember Maneros Steak Dinner will be held at 6:30 p.m. Nov. 1 at The St. Lawrence Club, 86 Valley Road, Cos Cob. Join the club and reminisce over the beloved and sorely missed Greenwich landmark restaurant. Diners can linger with family and friends over a mouth-watering meal of Steak Tid-bits, garlic bread, fried onions, gorgonzola salad and more. Theres no tax or tip, and members of the St. Lawrence Society get a free drink. Cost is $40 for members, $45 for guests and $25 for kids. To RSVP, visit http://www.stlawrencesociety.com/events or call 203-618-9036.

Celebrating documentaries on artists

The Bruce Museum Presents, a new series of monthly public programs featuring thought leaders in the fields of art and science, showcases experts on compelling subjects of relevance and interest to members and visitors to the Bruce Museum. The series continues with Rewind/Fast-Forward: Celebrating the Artist Documentaries of Olympia Stone from 6 to 8:30 p.m. Nov. 7. Stone is an independent producer of documentary films about art and artists. Her first documentary, The Collector, explored the five-decade career of her father, famed NYC gallery owner and art collector Allan Stone. For the event, join Stone and two of her artist subjects James Grashow (The Cardboard Bernini) and Elizabeth King (Double Take: The Art of Elizabeth King) for a retrospective of these films, and their maker. Moderated by Leonard Jacobs. Doors open at 6 p.m. for a reception with light bites and beverages, followed by the panel discussion and Q&A from 7 to 8:30 p.m. Seats are $30 for museum members, $45 for nonmembers. To reserve a seat, visit brucemuseum.org or call 203-8690376.

Bacon Brothers perform for charity

The Bacon Brothers, a band started by the award-winning actor Kevin Bacon and his brother Michael, an Emmy-winning composer, will perform a rare intimate concert in Greenwich on Nov. 9 at a fundraiser for Fairfield Countys Inner-City Foundation for Charity & Education. The event, the 28th annual fall gala to benefit The Inner-City Foundation, will be held at the Hyatt Regency in Old Greenwich. It is the largest fundraiser of the year for the Inner-City Foundation, which provides support to local Fairfield County programs and organizations that provide education, food, shelter, help for addiction, domestic violence and much more to the neediest in Fairfield County. The band also includes Paul Guzzle (bass, backing vocals), Joe Mennonna (keyboards, accordion), Tim Quick (lead guitar, mandolin and backing vocals) and Frank Vilardi (drums). For more information and tickets, tables or sponsorships, visit innercityfoundation.org or call 203-416-1363.

Sip & Shop Art Show

Stop by the Holiday Sip & Shop Art Show at Abilis Gardens & Gifts from 5 to 8 p.m. Dec. 4. Enjoy wine, cheese and other refreshments while shopping with a 20 percent discount in the entire store. The Art Show will feature paintings and digital art created by adults who are supported by Abilis. To learn more, visit abilis.us/calendar. Abilis is a nonprofit organization that supports more than 700 individuals with special needs and their families annually from birth throughout adulthood in the area.

Talk on art and climate change

The Bruce Museum Presents is a new series of monthly public programs featuring thought leaders in the fields of art and science. It will showcase experts on compelling subjects of relevance and interest to members and visitors to the Bruce Museum. The program continues with Can Art Drive Change on Climate Change? An Evening with Alexis Rockman from 6 to 8:30 p.m. Dec. 5. Among current American artists profoundly motivated by nature and its future from the specter of climate change to the implications of genetic engineering Rockman holds a high place of honor. Join the Bruce for a night of discussion and debate featuring Rockman and a panel of thought leaders in contemporary art and science. Doors open at 6 p.m. for a reception with light bites and beverages, followed by the panel discussion and Q&A from 7 to 8:30 p.m. Seats are $30 for Museum members, $45 for nonmembers. To reserve a seat, visit brucemuseum.org or call 203-869-0376.

SLS golf tourney

The St. Lawrence Society will hold its 29th annual Charity Golf Tournament on May 11. Enjoy a perfect day of golf, food and fun. Get your foursomes together and join the fun. Cost is $175 for everything: the luncheon, cart, golf, cocktail hour and dinner. For cocktails and dinner only, its $100. Starts at 11:30 a.m. at E. Gaynor Brennan Golf Course, with a 12:45 p.m. shotgun start, scramble format. Golf is followed by cocktail hour, antipasti and a prime rib dinner back at the Club. To RSVP, visit http://www.stlawrencesociety.com/events or call 203-618-9036.

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Greenwich Symphony Orchestra concerts and other things to do - CT Insider

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Newswise The Childrens Cancer Institute at the Joseph M. Sanzari Childrens Hospital at Hackensack Meridian Health Hackensack University Medical Center and the John Theurer Cancer Center have announced they are participating in a multicenter Phase I/II clinical trial of an investigational gene therapy from bluebird bio, Inc. This trial is specifically for adolescents and adults with severe sickle cell disease (SCD) who cannot be effectively treated using standard therapies such as antibiotics, vitamins, blood transfusions or any pain relieving medications. The study is evaluating the safety and effectiveness of LentiGlobin for sickle cell disease, a gene therapy produced using the patients own modified stem cells to treat their sickle cell disease.

By using the patients own cells to produce functional hemoglobin that can prevent sickling of their red blood cells, LentiGlobin for SCD offers patients the opportunity to treat their disease without the need to have a matched bone marrow donor. The John Theurer Cancer Center is one of a limited number of centers internationally, and the Joseph M. Sanzari Childrens Hospital is the only pediatric site in New Jersey, where the study, which is enrolling patients age 12-50, is taking place.

Sickle cell affects 100,000 Americans. It affects one in every 365 African American births and one in every 16,000 Hispanic American births, said Alfred P. Gillio, M.D., director, Childrens Cancer Institute and section chief, Pediatric Stem Cell Transplantation and Cellular Therapy Program, Joseph M. Sanzari Childrens Hospital at Hackensack University Medical Center. This trial is for patients who have severe sickle cell disease and seek advanced treatment options but do not have a well-matched stem cell donor. Only 15% of sickle cell patients have a matched sibling donor and only 25 percent of patients have a matched unrelated volunteer donor.

Sickle cell affects every organ in a patients body, said Stacey Rifkin-Zenenberg, D.O., FAAP, pediatric hematologist/oncologist, Childrens Cancer Institute, and section chief, Pain and Palliative Care, Joseph M. Sanzari Childrens Hospital at Hackensack University Medical Center. This disease really has a tremendous effect not only on the patient, but also the family.

Sickle cell disease is an inherited disease caused by a mutation in the beta-globin gene, resulting in abnormal hemoglobin and sickle-shaped red blood cells. Symptoms and complications of the disease include anemia, infections, stroke, poor quality of life and early death. To date, the only cure for sickle cell disease is receiving a stem cell transplant from a matched donor, but this is not a therapeutic option for many patients. Supportive care including hydroxyurea and blood transfusions can ameliorate symptoms of the disease. To date, without a marrow donor, there has been no alternate curative therapy. Life expectancy of a person with sickle cell disease is 20 to 40 years of age. In some cases, patients using disease modifying medications can live to 50 or 60.

This therapy may be a major advance for sickle cell patients and so far, the results look very promising, said Scott D. Rowley, M.D., FACP, hematologist, medical director, Stem Cell Transplantation and Cellular Therapy and medical director, BMT Cell Lab, John Theurer Cancer Center, Hackensack Meridian Health Hackensack University Medical Center, who is enrolling adult patients. This investigational treatment, which is a one-time therapy, may be an option for our patients who have no other treatment options.

The results from early clinical studies are encouraging, said Dr. Gillio. With this treatment, the patient is their own donor and we are modifying their own cells to add copies of a functional beta globin gene.

In the current study:

About Hackensack Meridian Health Hackensack University Medical Center

Hackensack Meridian Health Hackensack University Medical Center, a 781-bed nonprofit teaching and research hospital located in Bergen County, NJ, is the largest provider of inpatient and outpatient services in the state. Founded in 1888 as the countys first hospital, it is now part of the largest, most comprehensive and truly integrated health care network in New Jersey, offering a complete range of medical services, innovative research and life-enhancing care, which is comprised of 34,100 team members and more than 6,500 physicians. Hackensack University Medical Center is ranked #2 in New Jersey and #59 in the country in U.S. News & World Reports 2019-20 Best Hospital rankings and is ranked high-performing in the U.S. in colon cancer surgery,lung cancersurgery,COPD, heart failure, heart bypass surgery, aortic valve surgery,abdominal aortic aneurysm repair, knee replacement and hip replacement. Out of 4,500 hospitals evaluated, Hackensack is one of only 57 that received a top rating in all nine procedures and conditions. Hackensack University Medical Center is one of only five major academic medical centers in the nation to receive Healthgrades Americas 50 Best Hospitals Award for five or more years in a row. Beckers Hospital Review recognized Hackensack University Medical Center as one of the 100 Great Hospitals in America 2018. The medical center is one of the top 25 green hospitals in the country according to Practice Greenhealth, and received 26 Gold Seals of Approval by The Joint Commission more than any other hospital in the country. It was the first hospital in New Jersey and second in the nation to become a Magnet recognized hospital for nursing excellence; receiving its sixth consecutive designation in 2019. Hackensack University Medical Center has created an entire campus of award-winning care, including: John Theurer Cancer Center, a consortium member of the NCI-designated Georgetown Lombardi Comprehensive Cancer Center; the Heart & Vascular Hospital; and the Sarkis and Siran Gabrellian Womens and Childrens Pavilion, which houses the Joseph M. Sanzari Childrens Hospital and Donna A. Sanzari Womens Hospital, which was designed with The Deirdre Imus Environmental Health Center and listed on the Green Guides list of Top 10 Green Hospitals in the U.S. Hackensack University Medical Center is the Hometown Hospital of the New York Giants and the New York Red Bulls and is Official Medical Services Provider to THE NORTHERN TRUST PGA Golf Tournament. It remains committed to its community through fundraising and community events especially the Tackle Kids Cancer Campaign providing much needed research at the Childrens Cancer Institute housed at the Joseph M. Sanzari Childrens Hospital. To learn more, visit http://www.HackensackUMC.org.

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Joseph M. Sanzari Childrens Hospital and John Theurer Cancer Center Launch Clinical Trial Evaluating Gene Therapy for Severe Sickle Cell Disease in...

A study in Molecular Therapy describes how neuroD1-based gene therapy turns glial cells abundant support cells in the brain into neurons, repairing damage that results from stroke and significantly improving motor function in mice.

Once further developed, this NeuroD1-based gene therapy could potentially be used to treat stroke, the researchers suggest.

The current treatment for stroke has a narrow time window, typically within a few hours after the occurrence of stroke, says lead author Yuchen Chen, a postdoctoral fellow at Penn State, in a media release.

Many patients cannot receive the treatment in time and as a result, often suffer from permanent disability caused by irreversible neuronal loss. There is an urgent need to develop a new therapy to regenerate new neurons and restore lost brain functions among stroke patients.

The human brain has approximately 86 billion neurons. While mini-strokes can be tolerated, moderate stroke involving the loss of billions of neurons leaves detrimental effects that do not spontaneously recover.

So, the critical question that is still unanswered in the neuroregeneration field is how can we regenerate billions of new neurons in a patients brain after stroke? said Gong Chen, professor of biology and Verne M. Willaman Chair in Life Sciences at Penn State and leader of the research team. The biggest obstacle for brain repair is that neurons cannot regenerate themselves. Many clinical trials for stroke have failed over the past several decades, largely because none of them can regenerate enough new neurons to replenish the lost neurons.

Gong Chen and his team pioneered a new approach to regenerate functional neurons using glial cells, a group of cells surrounding every single neuron in the brain that provide essential support to neurons. Unlike neurons, glial cells can divide and regenerate themselves, especially after brain injury.

I believe that turning glial cells that are already present in the brain into new neurons is the best way to replenish the lost neurons, Gong Chen states. These glial cells are the neighbors of the dead neurons in the brain and are likely to share the same ancestral cellular lineage.

Gong Chens team previously reported that a single genetic neural factor, NeuroD1, could directly convert glial cells into functional neurons inside mouse brains with Alzheimers disease, but the total number of neurons generated was limited. The research team believed that this limited regeneration was due to the retroviral system used to deliver NeuroD1 to the brain. In the current study, the research team used the AAV viral system, which is now the first choice for gene therapy in the nervous system, to deliver NeuroD1 into mouse motor cortex that had suffered from stroke.

Many neurons die after stroke but surviving glial cells can proliferate and form a glial scar in the stroke areas. The AAV system was designed to express NeuroD1 preferentially in the glial cells that form these scars, turning them directly into neuronal cells. Such direct glia-to-neuron conversion technology not only increased neuronal density in the stroke areas, but also significantly reduced brain tissue loss caused by the stroke.

Interestingly, the newly converted neurons showed similar neuronal properties to the neurons that were lost after stroke. This suggests a potential impact of the local glial lineage on the converted neuronal identity, the release explains.

The most exciting finding of this study is to see the newly converted neurons being fully functional in firing repetitive action potentials and forming synaptic networks with other preexisting neurons, Gong Chen adds. They also send out long-range axonal projections to the right targets and facilitate motor functional recovery.

A separate collaborative work led by Gregory Quirk, professor at the University of Puerto Rico, further tested the NeuroD1-based gene therapy in a rat stroke model. Quirk and colleagues also found that this direct glia-to-neuron conversion technology can rescue cognitive functional deficits induced by stroke.

Because glial cells are everywhere in the brain and can divide to regenerate themselves, our study provides the proof-of-concept that glial cells in the brain can be tapped as a fountain of youth to regenerate functional new neurons for brain repair not only for stroke but also for many other neurological disorders that result in neuronal loss, Yuchen Chen comments.

Our next step is to further test this technology and ultimately to translate it into clinically effective therapies to benefit millions of patients worldwide.

[Source(s): Penn State, Science Daily]

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Gene Therapy Could Help Improve Functional Recovery After Stroke - Physical Therapy Products

WASHINGTON, D.C., Sept. 24, 2019 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE -- The Alliance for Regenerative Medicine (ARM) and the National Association of Managed Care Physicians (NAMCP) Medical Directors Institute announced today the joint release of their recent study of medical director and manufacturer perspectives on value demonstration and reimbursement for cell- and gene-based regenerative and advanced therapies.

The study, entitled Roadmap for Navigating Cell and Gene Therapy Value Demonstration and Reimbursement in U.S. Managed Care, characterizes step-by-step considerations for achieving appropriate patient access to transformative and potentially curative therapies in the U.S. managed care setting. The findings identify key issues relevant to value demonstration and access to potentially curative therapies at a pivotal time for the industry, as several products have reached the market, with dozens more in late-stage clinical trials.

The initial wave of cell and gene therapies has launched into an environment that was not built with transformative or curative therapies in mind, said Eric Faulkner, Vice President, Precision and Transformative Medicine at Evidera and lead author for the publication. Its crucial for payers, providers, patients, and other stakeholders to align on expectations on value demonstration to ensure sustainable access.

In the next two to three years, the sector expects the number of marketed cell and gene therapies to more than triple, said Janet Lambert, CEO for the Alliance for Regenerative Medicine. We will improve patient lives if we ensure patients have timely access to these treatments and value assessment and reimbursement processes have been structured with these unique therapies in mind.

Appropriate reimbursement and access to transformative therapies is critical to constantly improve managed care practice, appropriate patient access and outcomes. This effort is one step to achieving that goal by identifying and acknowledging the key acceptance requirements and stakeholder perspectives as these therapies enter the marketplace, said Bill Williams, MD, Executive Vice President of the NAMCP Medical Directors Institute.

The study concludes with several key learnings for therapeutic developers and payers:

The study results are available to download here.

About The Alliance for Regenerative MedicineThe Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 350 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visithttp://www.alliancerm.org.

About the NAMCP Medical Directors InstituteThe NAMCP Medical Directors Institute is a non-profit membership association, which was established to provide tools, education, and resources to medical directors, practicing physicians, and other healthcare professionals. NAMCP Medical Directors Institutes mission is to help Medical Directors from purchasers, health plans, and provider systems make effective and informed decisions, respond to opportunities and challenges in managed care, while helping improve healthcare outcomes, and ultimately the lives of members and patients. To learn more about the NAMCP Medical Directors Institute or to become a member, visit http://www.namcp.org

Lyndsey Scull202-213-7086lscull@alliancerm.org

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ARM and NAMCP Publish Recommendations to Increase Patient Access in Joint Study, "Roadmap for Navigating Cell and Gene Therapy Value...

PARIS--(BUSINESS WIRE)--

Regulatory News:

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190922005072/en/

GenSight Biologics (SIGHT.PA) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported the first set of results from Week 96 of the RESCUE Phase III clinical trial. The trial evaluated the efficacy and safety of a single intravitreal injection of GS010 (rAAV2/2-ND4) in 39 subjects whose visual loss due to 11778-ND4 Leber Hereditary Optic Neuropathy (LHON) commenced up to 6 months prior to study treatment. Week 96, which marks the time when individual patient profiles can be analyzed, is the last of the scheduled readouts for the RESCUE trial and completes the data collection from GS010s pivotal trials in Europe.

The results point to continued efficacy of GS010 two years past injection, with best-corrected visual acuity (BCVA) sustaining a clinically meaningful improvement over nadir. Having been treated early in the course of the disease, RESCUE patients vision initially deteriorated to a worst point, or nadir, before beginning to recover.

Note: A mixed model of analysis of covariance (ANCOVA) was used with change from baseline as the response, and subject, eyes of the subject as random factor, treatment and the baseline LogMAR value as covariates in the model.

When visual acuity is measured from the post-baseline nadir, the visual acuity of GS010-treated eyes in fact recovered significantly from the worst BCVA reading post-baseline.

Table 1: BCVA, Change from Nadir*, RESCUE

Week 48

Week 72

Week 96

N

Mean (SD)

N

Mean (SD)

N

Mean (SD)

All-GS010 Eyes

36

+12.8 (17.9)

34

+20.6 (26.3)

34

+24.9 (3.8)

All-Sham Eyes

36

+11.8 (15.6)

33

+21.7 (25.1)

34

+22.3 (3.8)

Note: *Nadir defined as worst BCVA measured in LogMAR after baseline, up to the week of interest; baseline reading was excluded from consideration. Mean change was calculated using observed values (no data imputation).

GS010-treated eyes regained more than two-thirds of the initial loss occurring in the most acute phase of the disease. This improvement from nadir (-0.498 LogMAR mean improvement, or +24.9 ETDRS letters equivalent) corresponds to 5 lines of Snellen acuity and is far above the 3-line threshold commonly accepted as a clinically meaningful level of visual improvement. Such recovery of vision is unprecedented in any gene therapy trial. Moreover, these results demonstrate the durability of improvement seen in earlier readouts of this trial.

Vision in sham-treated eyes evolved in parallel fashion, continuing the bilateral improvement already observed in earlier Phase III readouts. The picture that has emerged is one of durable, bilateral recovery from the effects of the acute phase in LHON.

The BCVA results from RESCUE show a remarkable correspondence with those from the REVERSE trial, which studied the treatment of subjects at 6 to 12 months after onset of vision loss.

The visual evolution in RESCUE appears to be a phase shift of the REVERSE curves, with an additional impact from the acute phase. This coherence will be more rigorously explored in a meta-analysis of the pooled data from the two trials, which is planned for this year.

In previous analyses of these trials, LHON clinical experts confirmed that the visual recovery seen in these trials are at odds with and much superior to their observations from clinical practice. That natural history of vision in untreated LHON patients stands in strong contrast to acuities seen in both RESCUE and REVERSE.

A natural history study conducted by Santhera1 provides another way of assessing the results in RESCUE. In that study, 28% of subjects who had the 11778A mutation achieved the following definition of spontaneous clinically relevant recovery (CRR) from nadir in at least one eye:

By comparison, 58% of RESCUE subjects achieved this definition of CRR in at least one eye at Week 96, with GS010-treated eyes as likely to achieve this as sham-treated eyes (58% vs. 45%, p = 0.0956).

The results from the RESCUE study are encouraging and convincing, particularly because we are seeing a similar pattern to the REVERSE study results, said Dr. Mark L. Moster, Neuro-Ophthalmology, Wills Eye Hospital, Professor of Neurology and Ophthalmology at Thomas Jefferson University, Philadelphia, PA, and Principal Investigator in the RESCUE and REVERSE trials. Patients in RESCUE were treated before the nadir so, as expected, they continued to worsen early on. But then from week 48 until week 96 they experienced a recovery from the nadir. That is much better than the natural history in any prior studies.

Story continues

Examination of other visual functions and biomarkers, including Contrast Sensitivity, show that these measures stabilized at Week 96. Based on preliminary analysis of the safety data, GS010 was well-tolerated after 96 weeks. There were no ocular serious adverse events or discontinuations that were due to ocular events. The ocular adverse events most frequently reported in the therapy group were mainly related to the injection procedure, except for the occurrence of intraocular inflammation (accompanied by elevation of intraocular pressure in some patients) that is likely related to GS010, and which was responsive to conventional treatment and without sequelae. There were no systemic serious adverse events or discontinuations that were related to study treatment or study procedure.

These results are remarkable, showing the durable difference that GS010 can make for patients who would otherwise go blind due to the onset of LHON, commented Bernard Gilly, Co-founder and Chief Executive Officer of GenSight. These findings, which we will be discussing at the meetings we have planned with regulatory authorities, form a compelling core for the clinical and non-clinical data that support our marketing authorization application in Europe. GenSight is excited to have reached this milestone in GS010s clinical development and energized by the prospect of pulling it all together for our European dossier.

GenSight is planning to schedule a pre-submission meeting with the EMA in early 2020 and expects to submit application for marketing approval in Europe in the third quarter of 2020.

An End of Phase II meeting with the U.S. Food and Drug Administration (FDA) has been requested and is expected for November 2019.

GenSight will host a conference call today, September 23, 2019, at 10:30am CEST in French, and at 2.30pm CEST (8.30am EST) in English, to discuss in greater detail these results and the roadmap to submission.

Webcast & Conference call in French

Dial-in numbers:

United States: +1 212 999 6659 France: +33 (0) 1 7037 7166 United Kingdom: +44 (0) 20 3003 2666 Password: GenSight

Webcast link: https://channel.royalcast.com/webcast/gensightbiologicsfr/20190923_1/

Webcast & Conference call in English

Dial-in numbers:

United States: +1 212 999 6659 France: +33 (0) 1 7037 7166 United Kingdom: +44 (0) 20 3003 2666 Password: GenSight

Webcast link: https://channel.royalcast.com/webcast/gensightbiologicsen/20190923_1/

A replay of the calls and webcasts will be available by using the above links.

Reference:

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, GS010, is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

About GS010

GS010 targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function.

About Leber Hereditary Optic Neuropathy (LHON)

Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1st eye, with the 2nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of patients have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. The estimated incidence of LHON is approximately 1,400 to 1,500 new patients who lose their sight every year in the United States and Europe.

About RESCUE and REVERSE

RESCUE and REVERSE are two separate randomized, double-masked, sham-controlled Phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene.

The primary endpoint will measure the difference in efficacy of GS010 in treated eyes compared to sham-treated eyes based on BestCorrected Visual Acuity (BCVA), as measured with the ETDRS at 48 weeks post-injection. The patients LogMAR (Logarithm of the Minimal Angle of Resolution) scores, which are derived from the number of letters patients read on the ETDRS chart, will be used for statistical purposes. Both trials have been adequately powered to evaluate a clinically relevant difference of at least 15 ETDRS letters between treated and untreated eyes adjusted to baseline.

The secondary endpoints will involve the application of the primary analysis to bestseeing eyes that received GS010 compared to those receiving sham, and to worseseeing eyes that received GS010 compared to those that received sham. Additionally, a categorical evaluation with a responder analysis will be evaluated, including the proportion of patients who maintain vision (< ETDRS 15L loss), the proportion of patients who gain 15 ETDRS letters from baseline and the proportion of patients with Snellen acuity of >20/200. Complementary vision metrics will include automated visual fields, optical coherence tomography, and color and contrast sensitivity, in addition to quality of life scales, biodissemination and the time course of immune response. Readouts for these endpoints are at 48, 72 and 96 weeks after injection.

The trials are conducted in parallel, in 37 subjects for REVERSE and 39 subjects for RESCUE, in 7 centers across the United States, the UK, France, Germany and Italy. Week 96 results were reported in 2019 for both trials, after which patients were transferred to a long-term follow-up study that will last for three years.

ClinicalTrials.gov Identifiers: REVERSE: NCT02652780 RESCUE: NCT02652767

About REFLECT

REFLECT is a multi-center, randomized, double-masked, placebo-controlled study to evaluate the safety and efficacy of bilateral injections of GS010 in subjects with LHON due to the NADH dehydrogenase 4 (ND4) mutation.

The trial planned to enroll 90 patients with vision loss up to 1 year in duration and will be conducted in multiple centers in Europe and in the US.

In the active arm, GS010 will be administered as a single intravitreal injection to both eyes of each subject. In the placebo arm, GS010 will be administered as a single intravitreal injection to the first affected eye, while the fellow eye will receive a placebo injection.

The primary endpoint for the REFLECT trial is the BCVA reported in LogMAR at 1-Year post-treatment in the secondaffected/notyetaffected eye. The change from baseline in secondaffected/notyetaffected eyes receiving GS010 and placebo will be the primary response of interest. The secondary efficacy endpoints include: BCVA reported in LogMAR at 2-Years post-treatment in the secondaffected/notyetaffected eye compared to both placebo and the firstaffected eye receiving GS010, OCT and contrast sensitivity and quality of life scales. The first subject was treated in March 2018, and enrolment was completed in July 2019, ahead of schedule.

ClinicalTrials.gov Identifiers: REFLECT: NCT03293524

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GenSight Biologics Reports Sustained Efficacy and Safety at 96 Weeks in RESCUE Phase III Clinical Trial of GS010 for the Treatment of Leber Hereditary...

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Global Gene Therapy Market with Indepth Analysis, Competitive Players, Future Trends and Forecast to 2025 : Novartis, Kite Pharma, Inc.,...

YORBA LINDA, Calif. (PRWEB) September 26, 2019

LabRoots, the leading scientific social networking website offering premier, interactive virtual events and webinars, will host its 2nd Annual CRISPR Virtual Event, on October 3, 2019. Since the first conference was held in 2018, research has continued to advance rapidly, however many questions remain about the science, applications, and the use of biotechnological techniques for editing genomes.

CRISPR 2019 resumes the global dialogue on these issues by bringing together a broad range of participants including biomedical researchers, academia and industry, and renowned scientists to explore topics such as CRISPR Biology, CRISPR in Gene Editing Applications, and Ethical and Regulatory dimensions and concerns of Gene Editing.

CRISPR has evolved into various technologies for basic research with new applications. Now, in its 2nd year, this premier event focused on the discovery and classification of CRISPR systems, gene therapy, animal model development for medical research, agriculture and food security will feature eleven prominent speakers, and a keynote delivery by Dr. Marie-Christine Birling, Head Associate of the Genetic Engineering and Model Validation Department at the Institut Clinique de la Souris on generating rodent model by CRISPR/Cas9 genome editing.

CRISPR generates a high level of interest, said Dimitri Perrin, Senior Lecturer in Data Science, Queensland University of Technology and member of the organizing committee. Research in this area intersects with a number of disciplines, from the basic biology of CRISPR systems to applications in gene editing (for instance in model organisms, health or agriculture) and reflections on how to ethically use and adequately regulate these technologies. It has been a pleasure to work with LabRoots to bring you an exciting program of excellent researchers across these domains, added Perrin.

Our inaugural conference was a huge success, said Greg Cruikshank, Chief Executive Officer of LabRoots. CRISPR 2019 returns highlighting momentum and recent progress made in gene editing and biotechnology and its potential moving forward. LabRoots continues to be dedicated to developments in life sciences using its unique stage for sharing data and research critical to the scientific community.

LabRoots interactive platform allows attendees to watch, learn and connect seamlessly across all desktop and mobile devices. By joining this online event, viewers can earn one Continuing Education credit per presentation for a maximum of 14 credits.

To register for the event and for more information, click here. Participants can follow the conversation online by using #LRcrispr.

About LabRoots LabRoots is the leading scientific social networking website, and primary source for scientific trending news and premier educational virtual events and webinars and more. Contributing to the advancement of science through content sharing capabilities, LabRoots is a powerful advocate in amplifying global networks and communities. Founded in 2008, LabRoots emphasizes digital innovation in scientific collaboration and learning. Offering more than articles and webcasts that go beyond the mundane and explore the latest discoveries in the world of science, LabRoots users can stay atop their field by gaining continuing education credits from a wide range of topics through their participation in the webinars and virtual events.

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LabRoots Announces Second Annual Conference on CRISPR Technologies - PR Web