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以色列的成體幹細胞與中樞神經治療公司--BrainStorm,與麻州總醫院(MGH)和麻州醫學院合作,利用骨髓的間葉幹細胞,治療肌萎縮性脊髓側索硬化症(漸凍人)。

在美國,每年有5600人診斷為漸凍人,平均一天有15個案例。BrainStorm的間葉幹細胞治療技術-NurOwn,以孤兒藥(罕見疾病藥物)方式獲得美國食品藥物管理局(FDA)核准,目前正申請美國的人體臨床試驗,希望可以提供漸凍人新的治療方式。

BrainStorm的NurOwn技術是第一個成功將間葉幹細胞經體外培養,誘導分泌神經滋養因子,如膠質細胞神經滋養因子(GDNF),促進神經細胞的分化,可望修復漸凍人壞死的中樞運動神經。

MGH, UMass Medical form ALS clinical trial deal with BrainStorm 

BrainStorm Cell Therapeutics Inc., an adult stem cell technology and central nervous system therapeutics company, said it will collaborate with Massachusetts General Hospital and the University of Massachusetts Medical School on a human clinical trial for its NurOwn experimental treatment for amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease).

The memorandum of understanding between the Massachusetts entities and BrainStorm, of Israel and New York, leads up to BrainStorm’s applying for U.S. Food and Drug Administration (FDA) approval to start ALS human clinical trials in the United States. This U.S. trial will be conducted after FDA approvals and in parallel with trials BrainStorm is running in Israel with the Hadassah Medical Center.

ALS affects nerve and muscle functions and eventually leads to death. About 5,600 Americans are diagnosed with the disease each year, which translates into about 15 new cases a day, according to the ALS Association. Up to 30,000 Americans have the disease at any given time. Current treatments are aimed at slowing the disease’s progression and prolonging life.

BrainStorm has received FDA orphan drug designation for NurOwn. The collaboration is the next step towards including more ALS patients in clinical trials by extending them to U.S. medical centers under FDA regulations, according to a company press release.

NueOwn technology, based on discoveries at Tel Aviv University, processes adult human mesenchymal stem cells in bone marrow that can self-renew and differentiate into many cell types. BrainStorm claims the research team is among the first to have achieved in vitro differentiation of adult bone marrow cells into cells that can release neurotrophic factors, including glial-derived neurotrophic factor, a small protein that may promote survival of many types of neurons.

http://www.masshightech.com/stories/2011/07/18/daily7-MGH-UMass-Medical-form-ALS-clinical-trial-deal-with-BrainStorm.html

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