Monday, 28 April 2014

BrainStorm's stem cell trial for ALS approved by the FDA

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BrainStorm Cell Therapeutics announced today that the US Food and Drug Administration (FDA) has approved commencement of its Phase II clinical trial with NurOwn™ in patients with Amyotrophic Lateral Sclerosis (ALS). 

The trial will be launched initially at the Massachusetts General Hospital (MGH) in Boston, MA and the University of Massachusetts Memorial (UMass) Hospital in Worcester, MA following Institutional Review Board (IRB) approvals. Dana-Farber Cancer Institute's Connell O'Reilly Cell Manipulation Core Facility will manufacture the NurOwn™ cells for these two clinical sites. The trial will also be conducted at the Mayo Clinic in Rochester, Minnesota.
"Today's announcement represents the most significant milestone BrainStorm has achieved to date. More importantly, in our view, it has positive implications for the entire ALS community, indicating that the FDA recognizes the significant clinical potential of transplantation with our autologous, differentiated mesenchymal stem cells. We are excited to begin the US trial and are optimistic that the Phase II data will confirm the positive indications of clinical benefit we observed in earlier studies. This trial will be the first Phase II double-blinded stem cell study to be conducted for ALS." said Mr. Chaim Lebovits, President.  

"Following successful completion of the technology transfer process for BrainStorm's NurOwn cells, we are looking forward to collaborating on this multi-center study as the cell production facility for the Massachusetts clinical sites." added Professor Jerome Ritz, Department of Medicine, Harvard Medical School, and Director, Connell O'Reilly Cell Manipulation and Gene Transfer Laboratory, Dana-Farber Cancer Institute.

BrainStorm’s Phase II trial is a randomized, double-blind, placebo controlled multi-center study designed to evaluate the safety and efficacy of transplantation of Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors ("MSC-NTF" or NurOwn™) in 48 patients with amyotrophic lateral sclerosis.

The NurOwn™ cells will be administered via combined intramuscular and intrathecal injection. Patients will be followed monthly for approximately three months before transplantation and six months following transplantation. 

The primary objective of the study will be safety and tolerability, as defined by a measure of the number of patients with adverse events. Secondary endpoints will measure a change in the Amyotrophic Lateral Sclerosis (ALS) Functional Rating Scale (ALS-FRS) and a change in Slow Vital Capacity (SVC) slopes. For further details, please visit http://1.usa.gov/1l5XWkW.

Earlier clinical trials have shown that treatment with NurOwn™ cells was well tolerated and safe. According to the final clinical study report for BrainStorm's Phase I trial in Israel, the study successfully met its primary and secondary endpoints. 

Moreover, there were encouraging signals of efficacy in individuals receiving NurOwn treatment, in which a dramatic improvement was observed during more than two consecutive visits following treatment in one or more of their secondary efficacy endpoints (ALSFRS-R score, total neurological examination score, FVC, and CMAP). Initial observations of the company's Phase IIa trial data in Israel appear to reinforce these findings. An interim update is expected to be released later this quarter.


Amyotrophic lateral sclerosis (ALS) - also referred to as motor neurone disease (MND), and sometimes referred to as Lou Gehrig's disease in the United States - is a neurodegenerative disease with various causes. It is characterised by rapidly progressive weakness due to muscle atrophy and muscle spasticity, difficulty in speaking (dysarthria), swallowing (dysphagia), and breathing (dyspnea). ALS is the most common of the five motor neuron diseases.

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