NIH funding bolsters rare diseases research collaborations | National Institutes of Health – National Institutes of Health

News Release

Thursday, October 3, 2019

New grants aimed at better understanding diseases, moving potential treatments closer to the clinic.

Of an estimated 6,500 to 7,000 known rare diseases, only a fraction maybe 5% have U.S. Food and Drug Administration-approved treatments. To increase that percentage, the National Institutes of Health has awarded approximately $31 million in grants in fiscal year 2019 to 20 teams including five new groups -- of scientists, clinicians, patients, families and patient advocates to study a wide range of rare diseases. An additional $7 million has been awarded to a separate data coordinating center to support these research efforts.

The grants, which support consortia that together form the Rare Diseases Clinical Research Network (RDCRN), are aimed at fostering collaborative research among scientists to better understand how rare diseases progress and to develop improved approaches for diagnosis and treatment. This is the fourth five-year funding cycle for the RDCRN, which is supported by multiple NIH Institutes and Centers and led by NIHs National Center for Advancing Translational Sciences (NCATS) and the NCATS Office of Rare Diseases Research.

Individually, most rare diseases affect only a few hundred to several thousand people; collectively, rare diseases affect more than 25 million Americans. Many rare diseases are life-threatening and about half of those affected are children.

Because rare diseases affect a small number of people, they can be extremely challenging to study. Scientists often lack basic information about a rare diseases symptoms and biology, and the ways a disease can affect people over time. Research funding can be scarce.

Over the years, RDCRN scientists have partnered with patients and advocates to develop new insights into the causes and progression of and potential therapies for rare diseases that were simply not receiving the attention they deserved, said NCATS Director Christopher Austin, M.D. Their pioneering work in discerning underlying clinical differences and commonalities in hundreds of rare conditions has already changed the rare disease landscape in immeasurable ways.

Established by Congress under the Rare Diseases Act in 2002, the RDCRN has included more than 350 sites in the United States and more than 50 in 22 other countries. To date, they have encompassed 237 research protocols and included more than 56,000 participants in studies ranging from immune system disorders and rare cancers to heart and lung disorders, brain development diseases and more.

Each RDCRN member is a consortium of clinical and scientific experts and patient groups who study a group of rare diseases. Each consortium must study three or more diseases, partner with rare disease patient advocacy groups, provide rare disease research training to investigators and perform natural history studies that chart the course and progression of diseases. The primary focus of the RDCRN is clinical research, and the network does not generally support clinical care outside of research activities.

A key component of the RDCRN is the Data Management and Coordinating Center (DMCC), which was awarded to the Cincinnati Childrens Hospital Medical Center. The DMCC manages shared resources and data from the RDCRN research studies. The DMCC emphasizes the standardization of data, increased data sharing and broad dissemination of research findings.

The RDCRN consortia have a rich history of accomplishment. For example, Lysosomal Disease Network scientists led crucial natural history studies and gene editing research that provided a foundation for first-in-human genome editing clinical studies for a rare metabolic disease. Primary Immune Deficiency Treatment Consortium members showed the advantage of early stem cell transplants for patients with a rare immune system disorder, severe combined immunodeficiency, and the groups work contributed to advances in gene therapy-based treatments for the disease.

New groups, new emphasis

The five new consortia are:

According to ORDR director Anne Pariser, M.D., an important focus of the latest group of awards is on clinical trial readiness.

Some of the RDCRN research groups have been working together for 10 or 15 years and have gathered important data and developed a good understanding of the diseases they study, in addition to new potential therapies. Were emphasizing the need to be prepared to conduct clinical trials, Pariser said.

Were trying to get the drug candidates closer to be ready for clinical testing and de-risk the processes that lead to a successful clinical trial, said RDCRN program officer Tiina Urv, Ph.D. To get funding to conduct trials, they need to have strong natural history studies that show how the disease progresses, ways to measure outcomes of treatments and biomarker studies that provide indicators of how a drug is working in patients.

Collaboration is key. Consortia can involve numerous partner research teams from different sites, along with rare disease patients and advocacy groups. Scientists from different institutions come together to pool patients, data, experience and resources.

Scientists cant work alone. They wouldnt have enough patients, and they wouldnt have adequate resources and information about the diseases, Urv said. Patients and families help scientists decide what is important to study, test and treat.

To read more about the five new consortia, 15 continuing consortia and the DMCC, see: https://ncats.nih.gov/rdcrn/consortia

In addition to NCATS, other NIH funding support comes from the National Institute of Allergy and Infectious Diseases, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Institute of Neurological Disorders and Stroke, the National Heart, Lung, and Blood Institute, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute of Diabetes and Digestive and Kidney Diseases, the National Institute of Dental and Craniofacial Research, the National Institute of Mental Health and the Office of Dietary Supplements.

About the National Center for Advancing Translational Sciences (NCATS):NCATS conducts and supports research on the science and operation of translation the process by which interventions to improve health are developed and implemented to allow more treatments to get to more patients more quickly. For more information about how NCATS is improving health through smarter science, visithttps://ncats.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

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NIH funding bolsters rare diseases research collaborations | National Institutes of Health - National Institutes of Health

Stem Cell Therapy Market worth USD 4759.27 Million By 2024 – Global Market News 24

Zion Market Research published a new 110+ pages industry researchStem Cell Therapy Market by Type (Allogenic SCs and Autologous SCs) by Therapeutic Application (Musculoskeletal Disorders, Wounds & Injuries, Cardiovascular Diseases, Gastrointestinal Diseases, Immune System Diseases, and Others), by Cell Source (Adipose Tissue-Derived Mesenchymal SCs, Bone Marrow-Derived Mesenchymal SCs, Embryonic SCs, and Other Sources), and by End User (Hospitals and ASCs): Global Industry Perspective, Comprehensive Analysis and Forecast, 2017 2024.

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What is the current CAGR of the Global Stem Cell Therapy Market?

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Tip Sheet: How a failed Alzheimer’s drug boosts CAR T-cell therapy; a new type of anti-CRISPR; and how GVHD starts in the gut – Newswise

MEDIA CONTACT

Available for logged-in reporters only

Immunotherapy

Nanotech turns pro-tumor immune cells into cancer-killing triple agents Nanotechnology could be the key to redirecting specialized immune cells to attack and shrink tumors. In anew studypublished last month in Nature Communications, scientists at Fred Hutch showed in mice that miniscule, dissolving polymer particles can ferry genetic instructions that temporarily rewire certain immune-suppressing cells into cancer fighters without causing body-wide toxicities. The researchers demonstrated that once reprogrammed, these cells called macrophages help orchestrate an anti-cancer immune response that can shrink, and even clear, tumors in mice with ovarian, brain and skin cancers.Media contact: Molly McElroy, mwmcelro@fredhutch.org, 206.667.6651

Failed Alzheimers drug boosts CAR T-cell therapyAnew studyin the journal Blood describes how an experimental drug called gamma secretase inhibitors can keep multiple myeloma visible to the immune system. Scientists at Fred Hutch have found that GSIs disable cancers cloaking device and make them easily spotted by CAR T cells, which are programmed to attack. The researchers have now launched aclinical trialto test this combination of CAR T and GSIs. Their paper details promising but early results in three multiple myeloma patients, noted Dr. Margot Pont, a postdoctoral researcher at Fred Hutch and the lead scientist on the analysis. Media contact: Molly McElroy, mwmcelro@fredhutch.org, 206.667.6651

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Human microbiome

New type of viral anti-CRISPR found in human microbiome Theres a war going on in your gut, and it has nothing to do with what you just ate. Viruses and bacteria are engaged in an age-old, life-or-death game of rock-paper-scissors (minus the paper). Bacteria attack with DNA-snipping CRISPR scissors and viruses block with anti-CRISPR rocks. Hutch scientists recently reported the discovery of a new type of anti-CRISPR. Insights into this battle could help researchers improve gene therapy or potentially find new solutions to antibiotic resistance. Media contact: Molly McElroy, mwmcelro@fredhutch.org, 206.667.6651

Mystery solved: How graft-vs.-host disease starts in the gut A new studypublished in the journal Immunity identifies the complex chain of events that triggers GVHD in the gut. It involves a large cast of cells and molecules, including some from a surprising source: the trillions of tiny organisms that live in and on us known as the microbiome. Fred Hutch researchers Dr. Geoffrey Hill , scientific director of the Immunotherapy Integrated Research Center at Fred Hutch, and Dr. Motoko Koyama also found a promising clue as they traced the diseases complicated pathway.One of the key players in that pathway is a chemical signal called interleukin-12. By snuffing out that signal, the researchers could prevent the disease from happening in mice. They are now applying for funding to test this approach in transplant patients via a clinical trial. Media contact: Molly McElroy, mwmcelro@fredhutch.org, 206.667.6651

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Clinical Research

The link between babys cells and moms disease risk Fred Hutch researchers have for the first time identified how the two-way traffic between mom and fetus known as microchimerism can sometimes harm mom years later. During this exchange in pregnancy, cells flow back and forth between mother and child. They can take up residence in the others body for decades. In the Proceedings of the National Academy of Sciences, the researchers report how those fetal immigrants can contribute to moms risk of rheumatoid arthritis, a painful autoimmune disease where the bodys immune system attacks its own joints. It turns out some of these lingering fetal cells can trigger moms immune system to go on the offensive, much like a transplant recipients body might reject a donor organ.Media contact: Molly McElroy, mwmcelro@fredhutch.org, 206.667.6651

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Prevention

How do we get teens to stop vaping? Vaping, the wildly popular new way to consume nicotine and marijuana has turned out to be riskier than expected with at least a dozen deaths and nearly 1,000 sickened. Washington Gov. Jay Inslee and public health officials announced anew, science-based smartphone appto help teens and young adults kick the vaping habit. The app is based on research done by Fred Hutch smoking cessation expertDr. Jonathan Bricker. His research group at theFred Hutch HABIT Laband collaborators have since conducted studies and mobile interventions to help people stop other unhealthy behaviors such as overeating and excessive drinking and tailored their smoking cessation apps for high-risk groups such as cancer patients. Media contact: Tom Kim,tomkim@fredhutch.org, 206.667.6240

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September Recognitions

Researchers at Fred Hutch are often recognized for their work. Were proud to celebrate their achievements and grateful to the awarding organizations.

Dr. Zhe Ying receives award to study how head and neck cancers overcome newly discovered tumor-blocking mechanism

# # #

AtFred Hutchinson Cancer Research Center, home to three Nobel laureates, interdisciplinary teams of world-renowned scientists seek new and innovative ways to prevent, diagnose and treat cancer, HIV/AIDS and other life-threatening diseases. Fred Hutchs pioneering work inbone marrow transplantationled to the development ofimmunotherapy, which harnesses the power of the immune system to treat cancer. An independent, nonprofit research institute based in Seattle, Fred Hutch houses the nations first National Cancer Institute-funded cancer prevention research program, as well as the clinical coordinating center of the Womens Health Initiative and the international headquarters of theHIV Vaccine Trials Network.

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Tip Sheet: How a failed Alzheimer's drug boosts CAR T-cell therapy; a new type of anti-CRISPR; and how GVHD starts in the gut - Newswise

Global CAR T-Cell Therapy Market 2019 Trends, lucrative Opportunities, Analysis to 2024 – Markets Gazette

Fior Markets latest research publication offering titled Global CAR T-Cell Therapy Market Growth (Status and Outlook) 2019-2024 assesses historical, latest values, and current changes to forecast market way for upcoming years from 2019 to 2024. Each segment are studied in detail and covered and market estimates and forecasts at the regional and country level are provided in this report. The segment analysis will help to know the probable opportunities and growth areas of the market.

The report serves necessary information regarding the market key participants including both established companies and new market entries. This global market has been progressing at a faster pace with the advance inventive systems and growth tendency. The detailed segmentation by product type, application, and various processes and systems has been given in the report. It has mentioned strategically significant competitor information and insights to develop effective R&D strategies as well as identify emerging players with a product portfolio to formulate effective counter-strategies. Additionally, the report figures out the product price, specification, financial and technical details, and research methodologies that will help businesses expand their market operations.

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Market competition by top manufacturers, with production, revenue (value) and market size for each manufacturer; the key players including as follow: Celgene (Juno Therapeutics), Novartis, Gilead (Kite Pharma), Pfizer, CARsgen Therapeutics, Autolus Therapeutics, Aurora BioPharma, Sorrento Therapeutics, Mustang Bio, Bluebird Bio, Collectis, Allogene Therapeutics, Celyad

The CAR T-Cell Therapy market report mainly throws light on dominant players in the regions of Americas (United States, Canada, Mexico, Brazil), APAC (China, Japan, Korea, Southeast Asia, India, Australia), Europe (Germany, France, UK, Italy, Russia, Spain), Middle East & Africa ( Egypt, South Africa, Israel, Turkey, GCC Countries). The estimates of market size and revenue depend on the demand of major segment within the diverse category.

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Market by product type: Allogeneic, Autologous

Market by applications: Hospitals, Cancer Research Centers, Others

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Global CAR T-Cell Therapy Market 2019 Trends, lucrative Opportunities, Analysis to 2024 - Markets Gazette

CBMG: Initiating Coverage of Cellular Biomedicine Group; Multiple Cell-Based Therapies to Treat Cancer and Degenerative Diseases – Yahoo Finance

By David Bautz, PhD

NASDAQ:CBMG

READ THE FULL CBMG RESEARCH REPORT

Initiating CoverageWe are initiating coverage of Cellular Biomedicine Group, Inc. (NASDAQ:CBMG) with a valuation of $24.00. Cellular Biomedicine Group is a biopharmaceutical company developing cell-based therapies for cancer and degenerative diseases, with a focus on the Chinese market. In addition to a full pipeline of development products comprised of CAR-T, TIL, and TCR, along with stem-cell based therapies for the treatment of osteoarthritis, Cellular Biomedicine Group signed a collaboration agreement last year with Novartis for the production of Kymriah in China. CAR-T products targeting multiple myeloma and non-Hodgkin lymphoma are currently being evaluated in clinical trials and we anticipate additional clinical trials initiating in other indications over the next six to twelve months.

Robust Pipeline with Multiple Cell-Therapy Development ProductsCellular Biomedicine Group has a robust pipeline of products derived from in-house discovery and strategic in-licensing including CAR-T, TIL, TCR, and stem cell products for which we anticipate at least six modalities being in Phase 1 clinical trials by the end of 2019.

Investigator-Initiated Trials to Speed-Up Development TimelinesCellular Biomedicine Group has begun investigator-initiated trials in China for its CAR-T products in patients with multiple myeloma and non-Hodgkin lymphoma, with additional trials starting later in 2019 and early 2020. These trials are intended to quickly evaluate safety and proof-of-concept in a cost-effective manner before moving to larger trials in China and the U.S. We anticipate initial data readouts from the ongoing trials at the end of 2019 or early 2020.

Collaboration with Novartis for Kymriah in ChinaIn 2018, Cellular Biomedicine Group signed a collaboration agreement with Novartis to manufacture and supply Kymriah in China. Included in the deal was a $40 million equity purchase from Novartis, which represents a 9% equity stake in the company. Cellular Biomedicine Group will be responsible for manufacturing the product while receiving an escalating single-digit royalty on net product sales of Kymriah in China, while Novartis will be responsible for distribution, regulatory, and commercialization efforts in China. This deal validates Cellular Biomedicine Groups cell manufacturing technology and we believe puts them at the forefront of cell therapy companies in China.

First-Mover Advantage in ChinaThe company is leveraging its expertise in the Chinese regulatory environment to establish itself as the leader in the Chinese cell therapy market. Very few other companies have the requisite expertise to handle the various compliance, quality assurance, and regulatory issues inherent in the Chinese market while simultaneously working to improve manufacturing timelines for cell therapy clinical trials and commercial launch.

Proprietary Manufacturing Processes for Cellular TherapiesThrough collaborations with GE Healthcare Life Sciences and ThermoFisher Scientific, Cellular Biomedicine Group has developed an automated, closed, integrated cell manufacturing system with complete control of the chain-of-custody. The company maintains manufacturing facilities totaling 100,000 ft2 in Shanghai and Wuxi that meet the quality management systems requirements of ISO 9001:2015, and the facilities of Shanghai and Wuxi sites meet local regulatory standards.

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Brooks Koepka had stem cell treatment on knee during offseason – ESPN

LAS VEGAS -- Brooks Koepka spent the majority of his short offseason recovering from stem cell treatment on his left knee, he said Wednesday.

Koepka, the No. 1 player in the world golf ranking, returns to competition this week in the Shriners Hospitals for Children Open in Las Vegas.

After his pro-am round Wednesday, Koepka said he had treatment on his patella on Aug. 25, the day after the PGA Tour season ended at the Tour Championship. He said he was limping for a few days because the treatment was painful, but he was excited about finally feeling whole.

"My knee was bugging me from about March on," Koepka said, adding that the final four months were a "struggle."

Koepka won three titles last season, including the PGA Championship -- his fourth major.

He is the third player from the top 10 in the world golf ranking to have work done on his knee during the offseason, joining Dustin Johnson and Tiger Woods. None was too serious, as Woods plans to return in three weeks at the new tournament in Japan. Johnson is likely to return at the Hero World Challenge in the Bahamas the first week of December, followed by the Presidents Cup in Australia.

Koepka never talked about injury during the season. He said his knee made it difficult to walk down hills and that it kept him from practicing. He returned to practice a little over a week ago and feels this is the most prepared he has felt going into a tournament in some time.

With all that, he still managed to have three wins, lead the PGA Tour money list and join Jack Nicklaus, Woods and Jordan Spieth as the only players to finish among the top four in every major. Koepka was runner-up at the Masters and U.S. Open.

It just wasn't good enough to be voted PGA Tour player of the year. That award, voted on by players, went to Rory McIlroy, whose three victories included The Players Championship and the Tour Championship, which gave him the FedEx Cup for the second time.

"I don't play for awards," Koepka said Wednesday. "I play to win tournaments."

He also used an NBA reference, suggesting everyone knows that LeBron James has been the best in basketball for the last decade or more.

"LeBron has won only four MVPs," Koepka said. "I'm pretty sure he's been the best player."

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Brooks Koepka had stem cell treatment on knee during offseason - ESPN

Early clinical trial for ALS sufferers shows biotech firm ‘on the right track’ – The Times of Israel

Kadimastem Ltd., a biotechnology firm that develops cell therapies, said it has received promising interim results from a first group of patients treated with its new therapy for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrigs disease, a fatal neurodegenerative condition that causes the loss of muscle control.

Looks like we are on the right track, said Rami Epstein, the CEO of Kadimastem, in a phone interview. The interim results are important because they demonstrate that we have managed to inject the live cells into a human body without any treatment-related significant adverse effect, while also showing a measurable therapeutic effect. Our cell therapy managed to significantly slow down the disease progression and halt deterioration of the disease.

The cell therapy aims to slow or even halt the progression of the disease and improve patients quality of life and life expectancy, he said.

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ALS leads to muscle weakness, loss of motor function, paralysis, breathing problems, and eventually death. The average life expectancy of ALS patients is two to five years. According to the ALS Therapy Development Institute, there are approximately 450,000 ALS patients worldwide, 30,000 of them in the US. According to the ALS Foundation for Life, the annual average healthcare costs of an ALS patient in the US are estimated at US$ 200,000. Thus, the annual healthcare costs of ALS patients in the US alone amount to $6 billion.

Reserachers at Kedimastems Good Manufacturing Practice (GMP)-compliant production facilities in Nes Ziona, Israel (Hagit Stavinsky)

In a filing to the Tel Aviv Stock Exchange last week, the firm said that the treatment administered to one group of patients in a Phase 1/2a clinical trial held in Israel apparently caused a significant slowdown in the progress of the disease and was found to be safe.

ALS is a devastating disease and there is a critical need for new treatments to more effectively slow down or halt its rapid progression, said Dr. Marc Gotkine of the Department of Neurology at Hadassah Medical Center, Jerusalem, and the principal investigator of the trial. These preliminary results on a small subset of patients are encouraging, as the treatment seems to be safe, at this lower dose.

The initial data analysis appears to demonstrate a slowing down of the reported deterioration rate over the three months following treatment, he said.

This is a positive, but preliminary results and the final results should be awaited, analysts Frost & Sullivan said in an investor note filed with the Tel Aviv Stock Exchange.

Should the final results expected at the end of 2019 for this phase 1/2a trial also prove positive, this will position Kadimastem as a potential major player in the ALS market and will have positive effect also on firms value, the analysts said. This will also serve as proof of the potential of the companys platform which can be used to treat additional indications in the future.

Prof. Michel Revel, the CSO and founder of Kadimastem (Courtesy)

The company, whose shares are traded on the Tel Aviv Stock Exchange, was founded by Prof. Michel Revel, who serves as the companys chief scientific officer. Revel is an emeritus faculty member of the Weizmann Institute of Science and a winner of the Israel Prize, the nations greatest accolade. He is also the creator of Rebif, a multiple sclerosis drug now marketed by Merckand selling for billions of euros globally.

Revel founded Kadimastem in 2009 with Yossi Ben-Yossef, based on cell therapy research Revel had conducted at the Weizmann Institute. Such research focuses on live cells transplanted into the body to replace malfunctioning, disease-causing cells.

In the cell therapy methodology developed and implemented by Kadimastem, we take embryonic stem cells, expand them into large quantities, and differentiate them into specializing cells, he said. Our cells are injected into the patient, replacing and compensating for the biological activity of the damaged cells.

A researcher working in the lab of Kadimastem, in Nes Ziona, Israel (Hagit Stavinsky)

The firm operates in two main spheres: neurological diseases, with the aim of replacing nervous system cells that have stopped functioning; and type 1 diabetes, in which cells that secrete the insulin needed to absorb sugar stop functioning. We develop cells that secrete insulin and regulate blood sugar levels, he said.

In the neurological sphere, the firm has started clinical trials, still at very early stages and on a small group of patients, to replace astrocyte cells,which are responsible for supporting neurons that enable muscle activity. In ALS patients, as a result of dysfunctional astrocytes, the neurons degenerate and muscle control deteriorates as the disease progresses.

All of the ALS patients die eventually, said Epstein, as there is no known cure for the disease. As the disease progresses, patients eventually lose control of their respiratory muscles, including the diaphragm, causing severe respiratory complications, the most common cause of death for ALS patients.

In their work at Kadimastem, the researchers differentiated embryonic stem cells into astrocytes. Scientific literature has indicated that astrocyte cells play a substantial role in supporting malfunctioning cells in ALS and other neurodegenerative diseases, said Revel in a statement issued by the firm.

Kadimastems lab in Nes Ziona, Israel (Hagit Stavinsky)

The patients had these cells injected into their spinal cord, where they secreted various factors that act as goodies, said Epstein. In animal trials, these cells created a protective layer around damaged neurons preventing further deterioration and maintaining and supporting the remaining neuron and muscle activity.

The cells were also able to secrete factors that remove toxic materials accumulated on the damaged neurons, thus acting as a garbage truck, taking toxic and damaging materials away from the neurons, Epstein said.

Now, the Phase 1/2a clinical trial conducted in humans is the first one ever, worldwide, in which live astrocyte cells, our own proprietary AstroRx cells, have been transplanted into humans, he said.

The trial, which started last year, is being conducted at the Hadassah Ein Kerem Medical Center in Jerusalem on 21 patients divided into four groups.

Each group receives our AstroRx cells, Epstein explained, saying the firm is very excited about the interim results of the first group, which got the lowest dose of the cells some 100 million cells injected into the spinal cord fluid of each patient.

The results are very significant for ALS patients and for us, he said. We hope that in the other groups we will see improved or prolonged efficacy and also safety. Kadimastem has advanced the quest to find a treatment for ALS but has also shown that the company has the ability to differentiate cells into clinical grade functional cells, fit for injection and also effective in humans.

A second cohort of patients, forming part of the same ongoing Phase 1/2a clinical trial, will be getting 250 million cells; group three will get two doses of 250 million cells each, to see if repeated treatments will yield enhanced results; and the fourth group is expected to receive a dose of 500 million AstroRx cells.

The journey ahead is still very long, Epstein emphasized, and it will be years before a drug will be made available to patients. The current Phase 1/2a trial is expected to conclude at the end of 2020, and then an additional trial will start, hopefully with a larger group of patients and in a number of medical centers.

Shares of the company have declined 2.1% in the past 12 months, bringing its market value to NIS 74 million ($21.2 million), as of end of day Wednesday.

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Early clinical trial for ALS sufferers shows biotech firm 'on the right track' - The Times of Israel

"I have talked about this treatment for so long… I finally gave in and set up the GoFundMe page. It killed me to have to do it." – Echo…

AN ordinary man with ordinary interests, Patrick Keane loves life, even though it has become more of a challenge than ever before.

He cannot play a round of golf or go for a walk by the beach any more. But he doesnt want pity and exudes positivity. So he enjoys soaking in the sea air around Clonea or Garryvoe without stretching his legs.

But reality has a way of crashing through the faade at times, no matter how strong you are.

For Patrick, it can be when he wakes up and cannot feel his legs, or loses his train of thought, or misses his mouth when trying to eat because he cannot feel the spoon in his hand. Or if he gets out of bed and falls and screams at himself, Just get up will ya, ya fool!

But the man known as Pa to his friends, or Patch to his mother Margaret, brother Paul and sisters Yvonne and Elaine, dusts himself down, drags himself back to an upright position and goes about taking on the day in as fearless a fashion as possible, given what he must encounter now and the knowledge of what lies ahead.

Multiple sclerosis affects the central nervous system, the brain and the spine, and while the rate of degeneration differs for all, there is no escaping the degeneration. Ireland has 8,000 sufferers with apparently no cure.

Patrick, who was diagnosed in January, 2009, is not without hope however. A doctor, Denis Federenko, has been providing stem cell transplants to people from all over the world at the AA Maximov Hospital in Moscow, with some success, by wiping out the faulty immune system with drugs used to treat cancer and replacing it with stem cells taken from the patient.

Irish comedian, Stephen Garland is one who underwent treatment in November, 2016, having been told he was around six months away from being confined to a wheelchair.

He returned home just before Christmas that year and has thrived since, even writing a show about his journey to Russia and back.

Garland brought his creation, Post-Disposed, back to the world-renowned Edinburgh Fringe Festival in August, confirming his continuing improved health.

The treatment costs between 50,000 and 60,000 including aftercare and other expenses. It can be hard to ask for help but so many people have extended a hand to Patrick, without ever being asked. People Patrick doesnt know from Adam or Eve have even contributed to his cause and it moves him to tears.

Time is against the Corkman however, because if his condition exceeds 6.5 on a scale of 0-8, he will not be taken on. At present, he is between 5.5 and 6, and has been accepted, but clearly the treatment must take place sooner rather than later.

To that end, a Breakfast With The Stars event is taking place at The Park Hotel, Dungarvan on Thursday, October 10. Tipperarys All-Ireland-winning manager, Liam Sheedy and multiple champion jockey Davy Russell will regale patrons with stories from their careers, prompted by MC Marty Morrissey.

All proceeds will go towards funding Patricks treatment and ancillary expenses. Ticket purchasing details are below.

Here is more about Patricks story...

************

LAYLA walks into the family home in Ballinroad, just outside Dungarvan. It was Patrick who named her, after the Eric Clapton song that he happened to be listening to when the family were discussing what to call the now 12-year-old dog.

Music is a boon, a real infusion of energy, pumping the blood, making him feel like he could jump out of his skin and dance like the old days. He cant but that burst of adrenaline is a godsend.

I have the car adapted with hand controls, Patrick explains. I dont use my feet for driving. Push and pull the lever in the car.

On a Sunday morning I love nothing better, especially when I am down at home. Head down the coast road to Tramore, listen to a bit of music.

I would listen to absolutely anything. I am influenced by dads taste in music. All the older stuff, 70s, 80s rock music kind of stuff. You could find me listening to dance music two minutes later. Once it has a beat I couldnt care less.

Patricks dad, Richie Keane, was a hard worker, who was brilliant with his hands and especially with cars. And all his life he had a smaller shadow. The son looked up to the father like he was Superman.

When he was sick and on his last legs, talking through the window from the house he taught me how to take the sump off my car because it was cracked and leaking oil. He was able to guide me through it without looking at it, word for word.

I restored a Jeep that he left when he passed away. It was a 1983, same age as myself, Mercedes jeep. Very rare, like hens teeth. Restored it to about 25,000 to 30,000 worth.

I had to sell it. I couldnt drive it anymore because the leg was so bad. That was hard. That was the last connection. It was something I had to remember him by. The day I saw that go out the drive was tough.

Richie died in November, 2008, around the time of Patricks 25th birthday. Richies mother passed away two weeks later. Patrick was diagnosed with MS in January, 2009. Already a celiac and diabetic, he was accustomed to restrictions and putting up with things. This was a different stratosphere though.

It began with losing his balance and the development of numbness down his right side.

I went to the doctor, and I think, by the look on his face, he knew there was something not right. He sent me to Ardkeen (Hospital) and I got checked out. They had me in isolation for about a week. Then I was told it was MS.

I didnt know what it was but it was something I heard from a conversation when I was younger, That poor fella has MS. I didnt know I would end up the way I am now.

I have friends since that have been diagnosed after me that I have gotten to know from reaching out. There are a couple of them in a wheelchair. As it progresses, that is where you are heading. But there is a gentleman up the road and he must have it for 20-something years. Unless someone told you, you wouldnt know he has MS.

Part of the difficulty of dealing with MS is that no two cases are alike. Keeping active, having physiotherapy regularly and working are advantages and Patrick has not declined as quickly as others because of that.

But he has hit the secondary stage, where there are cognitive problems and, in particular, his short-term memory is affected.

People say to me, do you get pain with it? I dont know what new pain or old pain is. I just get on with. I have my days where I whinge and moan and cry. God knows I have them. But there is people out there worse off. I have what I have.

It could get worse. Now that it is gone to progressive MS, it probably will. When or how long? Who knows?

It is a horrible disease, there is no two ways. But you get up, get on with it, and do what you can.

He went to Australia not long after the diagnosis. It was a real gesture of defiance, one that probably scared his mother but he knew too that it might well be a case of now or never.

A blocklayer by trade, Patrick eventually had to give that up as his coordination worsened and he nearly fell off a roof. He is on crutches now for three years and would not be able to catch a football if you threw it at him. He wears a leg cast too, to reduce the instances of tripping himself up, without eradicating them totally. He has a mobility scooter.

A 35-year-old man doesnt dream about a mobility scooter, he wants a flash sports car. But it allowed me to go down to the Greenway for the first time. That was nice, to be on the Greenway, to be out, and see the whole lot of it, it was lovely. But watching other people cycle off down killed me.

My two nephews were out the front playing soccer. I used to do the exact same thing. They were saying Patrick, are you coming out to play? I said I would love to but that gets you. Its the simple little things.

Katie, my partner, I dont know where I would be without her. My mam, the girls and my brother, they are fantastic. They have been with me since the start of it.

I decided I want to be independent. If I didnt have the car I dont know what I would be doing. That is my freedom. I can get into the car, go to the shop for coffee. I will get there. I will get the same place as anyone else will, but it will take me longer.

He lives in Cork with Katie and her son Aaron, and works for Voxpro, who have been tremendously supportive. If he were housebound, he would wither.

I am an outgoing guy. I would chat away, waffle on about anything for hours on end. But you take that away I dont know have I changed since the diagnosis.

I have tried to remain as positive as possible but sometimes its hard. Simple little things you take for granted. Just run out there to get the clothes off the line. Now I have had to get handrails put into the house so that I can get up when I trip and fall.

I would wake in the mornings and the legs are like jelly. They shake, you cant control it, you let them shake out and that could be for 20 or 30 seconds. Real spasticity and stiffness in the legs. With the heat during the summer, I sat in the car with the air con on. I would be good for doing weather forecasta. I know what its like when I wake up in the morning, ever before the curtains are pulled. I feel it in my body.

He has been trawling for potential treatments, along with his medical team. Dr Federenkos work stands up to inspection. Stopping the MS in its tracks without the need for further medication would be a tantalising prospect.

Reversing the effects is something he dare not even contemplate, though the treatment has had that effect in Garland and many others.

Being given the green light, after Dr Federenko reviewed his case and medical records, was like an infusion in itself. Patrick details every step of the treatment and though it sounds daunting, it isnt compared to the alternative.

He set up the GoFundMe page (https://www.gofundme.com/f/stop-ms-progression-with-stem-cells-transplant) and was staggered by the reaction. Meanwhile, the local community has rallied, as have his work colleagues and friends, organising fund-raiser after fund-raiser. People he didnt know had events. He finds it hard to process.

To ask for someones help, it is a sign that you are not able. I have talked about this treatment for so long and I got so bad in my legs and balance and everything, I finally gave in and set up the GoFundMe page. It killed me to have to do it.

There was a donation yesterday on GoFundMe from Jamaica. I dont know the person but they found it in their heart to say, There you go. People said to me, Sorry I cant give you too much, I want to give you more. If it is a euro or 20 cent, it could be that 20 cent or euro that gets me over the line.

Whatever happens, he will not give up because it is what he learned when he was Richies shadow: It is instilled in me from dad, I would always have looked up to him. Even now, since he has passed away, I would always say to myself, What would he do? Would he have approved of that? He would always say to stick at something until you get it. If you are going to start something, do it. Just dont walk away from it.

Breakfast With The Stars, featuring Tipperary manager Liam Sheedy, one of the all-time greats of National Hunt racing Davy Russell and RTs Marty Morrissey, takes place at The Park Hotel on Thursday, October 10. To book a table of 10 for 1000 contact Michael Ryan (087 2585299) or The Local Bar (058 41854).

Donations can also be made to Patrick Keanes GoFundMe pagehttps://www.gofundme.com/f/stop-ms-progression-with-stem-cells-transplant

Continued here:
"I have talked about this treatment for so long... I finally gave in and set up the GoFundMe page. It killed me to have to do it." - Echo...

Global Cell Therapy Technologies Market Industry Analysis and Forecast (2018-2026) – Markets Gazette

Global Cell Therapy Technologies Market was valued US$ 12 billion in 2018 and is expected to reach US$ 35 billion by 2026, at CAGR of 12.14 %during forecast period.

The objective of the report is to present comprehensive assessment projections with a suitable set of assumptions and methodology. The report helps in understanding Global Cell Therapy Technologies Market dynamics, structure by identifying and analyzing the market segments and projecting the global market size. Further, the report also focuses on the competitive analysis of key players by product, price, financial position, growth strategies, and regional presence. To understand the market dynamics and by region, the report has covered the PEST analysis by region and key economies across the globe, which are supposed to have an impact on market in forecast period. PORTERs analysis, and SVOR analysis of the market as well as detailed SWOT analysis of key players has been done to analyze their strategies. The report will to address all questions of shareholders to prioritize the efforts and investment in the near future to the emerging segment in the Global Cell Therapy Technologies Market.

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Global Cell Therapy Technologies Market: OverviewCell therapy is a transplantation of live human cells to replace or repair damaged tissue and/or cells. With the help of new technologies, limitless imagination, and innovative products, many different types of cells may be used as part of a therapy or treatment for different types of diseases and conditions. Celltherapy technologies plays key role in the practice of medicine such as old fashioned bone marrow transplants is replaced by Hematopoietic stem cell transplantation, capacity of cells in drug discovery. Cell therapy overlap with different therapies like, gene therapy, tissue engineering, cancer vaccines, regenerative medicine, and drug delivery. Establishment of cell banking facilities and production, storage, and characterization of cells are increasing volumetric capabilities of the cell therapy market globally. Initiation of constructive guidelines for cell therapy manufacturing and proven effectiveness of products, these are primary growth stimulants of the market.

Global Cell Therapy Technologies Market: Drivers and RestraintsThe growth of cell therapy technologies market is highly driven by, increasing demand for clinical trials on oncology-oriented cell-based therapy, demand for advanced cell therapy instruments is increasing, owing to its affordability and sustainability, government and private organization , investing more funds in cell-based research therapy for life-style diseases such as diabetes, decrease in prices of stem cell therapies are leading to increased tendency of buyers towards cell therapy, existing companies are collaborating with research institute in order to best fit into regulatory model for cell therapies.Moreover, Healthcare practitioners uses stem cells obtained from bone marrow or blood for treatment of patients with cancer, blood disorders, and immune-related disorders and Development in cell banking facilities and resultant expansion of production, storage, and characterization of cells, these factors will drive the market of cell therapy technologies during forecast period.

Do Inquiry Before Purchasing Report Here: https://www.maximizemarketresearch.com/market-report/global-cell-therapy-technologies-market/31531/#inquiry

On the other hand, the high cost of cell-based research and some ethical issue & legally controversial, are expected to hamper market growth of Cell Therapy Technologies during the forecast period

AJune 2016, there were around 351 companies across the U.S. that were engaged in advertising unauthorized stem cell treatments at their clinics. Such clinics boosted the revenue in this market.in August 2017, the U.S. FDA announced increased enforcement of regulations and oversight of clinics involved in practicing unapproved stem cell therapies. This might hamper the revenue generation during the forecast period; nevertheless, it will allow safe and effective use of stem cell therapies.

Global Cell Therapy Technologies Market: Segmentation AnalysisOn the basis of product, the consumables segment had largest market share in 2018 and is expected to drive the cell therapy instruments market during forecast period at XX % CAGR owing to the huge demand for consumables in cell-based experiments and cancer research and increasing number of new product launches and consumables are essential for every step of cell processing. This is further expected to drive their adoption in the market. These factors will boost the market of Cell Therapy Technologies Market in upcoming years.

On the basis of process, the cell processing had largest market share in 2018 and is expected to grow at the highest CAGR during the forecast period owing to in cell processing stage,a use of cell therapy instruments and media at highest rate, mainly in culture media processing. This is a major factor will drive the market share during forecast period.

Global Cell Therapy Technologies Market: Regional AnalysisNorth America to held largest market share of the cell therapy technologies in 2018 and expected to grow at highest CAGR during forecast period owing to increasing R&D programs in the pharmaceutical and biotechnology industries. North America followed by Europe, Asia Pacific and Rest of the world (Row).

Browse Full Report with Facts and Figures of Cell Therapy Technologies Market Report at: https://www.maximizemarketresearch.com/market-report/global-cell-therapy-technologies-market/31531/

Scope of Global Cell Therapy Technologies Market

Global Cell Therapy Technologies Market, by Product

Consumables Equipment Systems & SoftwareGlobal Cell Therapy Technologies Market, by Cell Type

Human Cells Animal CellsGlobal Cell Therapy Technologies Market, by Process Stages

Cell Processing Cell Preservation, Distribution, and Handling Process Monitoring and Quality ControlGlobal Cell Therapy Technologies Market, by End Users

Life Science Research Companies Research InstitutesGlobal Cell Therapy Technologies Market, by Region

North America Europe Asia Pacific Middle East & Africa South AmericaKey players operating in the Global Cell Therapy Technologies Market

Beckman Coulter, Inc. Becton Dickinson and Company GE Healthcare Lonza Merck KGaA MiltenyiBiotec STEMCELL Technologies, Inc. Terumo BCT, Inc. Thermo Fisher Scientific, Inc. Sartorius AG

MAJOR TOC OF THE REPORT

Chapter One: Cell Therapy Technologies Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Cell Therapy Technologies Market Competition, by Players

Chapter Four: Global Cell Therapy Technologies Market Size by Regions

Chapter Five: North America Cell Therapy Technologies Revenue by Countries

Chapter Six: Europe Cell Therapy Technologies Revenue by Countries

Chapter Seven: Asia-Pacific Cell Therapy Technologies Revenue by Countries

Chapter Eight: South America Cell Therapy Technologies Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Cell Therapy Technologies by Countries

Chapter Ten: Global Cell Therapy Technologies Market Segment by Type

Chapter Eleven: Global Cell Therapy Technologies Market Segment by Application

Chapter Twelve: Global Cell Therapy Technologies Market Size Forecast (2019-2026)

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Global Cell Therapy Technologies Market Industry Analysis and Forecast (2018-2026) - Markets Gazette

Cancer cells to be tested in zero gravity on Chinese Space Station – E&T Magazine

Cancer cells are to be transported into space to see if weightlessness can stop their growth, in one of nine research projects destined for the new China Space Station (CSS) in 2022.

Upon completion, the CSS will include a cancer research project called Tumours in Space, headed by a Canadian researcher based in Norway. The project will examine the roles of both microgravity and cosmic radiation in tumour growth and development. The project is one of just nine selected by the UN Office for Outer Space Affairs (UNOOSA) and the China Manned Space Agency (CMSA) under their programme to provide scientists from all over the world with the opportunity to fly experiments on the CSS.

The plan is to send three-dimensional stem cell organoids from both healthy and cancer tissue from the same person into space. Here we will study mutations and look at how the cells DNA is affected by weightlessness and cosmic radiation, said the projects principal investigator Tricia L. Larose.

The experiment will rely on three-dimensional cancerous tumours, called organoids. These organoids are grown from adult human stem cells, which are a kind of cell that can divide indefinitely and create different types of cells in doing so. Researchers have perfected their ability to grow organoids so they actually form tiny structures that mimic different organs. Larose theorises that the cancer organoid growth will slow or stop when they are not affected by Earths gravity. Previous research on two-dimensional cells has shown that weightlessness has an influence on gene expression linked to tumour development.

When we look at mutational signatures in cancer cells, there is a lot of noise. The noise is something we simply do not know a lot about, she said. Part of my experimental process is identifying new causes of that noise, and some of that might be gravity

Her theory is that some of the unknown noise in the cancer cells is there as a result of gravity. Since both healthy cells and cells with cancer are affected by gravity, the researchers should be able to detect this in the fingerprints in all our cells.

Im looking for the molecular fingerprint for the gravitational force, she said; this could help explain the meaning of some of the noise in the cancer cells.

She added that the mutational signature of gravity has never been studied or even proposed as a concept. The experiment will also test how cosmic radiation affects the DNA of the healthy organoids and whether this leads to mutations and cancer. The various causes of cancer, such as smoking, UV radiation and ionizing radiation, also leave mutational signatures. Identifying mutational signatures from cancer-causing exposures can be used for risk prediction, eventually leading to better diagnostics and therapeutics.

My ground-based research with ionising radiation will also help us understand the side effects of radiation therapy for cancer patients on Earth, she said.

The studies of cosmic radiation will also help with understanding the cancer risk for astronauts on long-duration missions in the space station, or longer journeys, such as to Mars.

The biggest challenge with human spaceflight and exploration for long-duration missions to Mars and beyond, is the cancer risk for crew due to exposure of cosmic radiation. By identifying the mutational signature of cosmic radiation and comparing that to the known signature of ionising radiation, we may be better able to predict risk and protect crew on a long-duration space mission Larose said.

It is thought that astronauts on a mission to Mars would be exposed to at least 60 per cent of the total radiation dose limit recommended for their career during the journey alone to and from the Red Planet.

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Cancer cells to be tested in zero gravity on Chinese Space Station - E&T Magazine