FDA Grants Approval To U.S. Phase-II Trials For ALS Disease Treatment

On Monday, the FDA announced that it had approved Phase II clinical trials for BrainStorm Cell Therapeutics’ stem cell treatment for patients with amyotrophic lateral sclerosis, or ALS.

ALS is a progressive neurodegenerative disorder that adversely affects the central nervous system. It is fairly rare, but researchers believe that 5,000 new cases are diagnosed in the United States each year. The disease slowly kills the central nervous system’s motor neurons, leaving the brain without the ability to send signals to muscles throughout the body. The e average patient survives about 3 to 5 years after their initial diagnosis. Eventually, respiratory failure causes death.

BrainStorm focuses on developing stem cell technologies for treating neurodegenerative diseases. Recently, the company focused its attentions on bringing its clinical trials to the United States. The Phase II clinical trials for ALS will be starting at the UMass Medical School and Massachusetts General Hospital in Boston. The United States study will include 48 male and female patients from ages 18 to 75, all of whom suffer from ALS.  Stem cells will be injected into each patient’s spinal fluid. Researchers expect the following evaluation process to take about a year.

BrainStorm’s president, Chaim Lebovits, released a statement regarding the FDA’s approval. “Today’s announcement represents a significant milestone BrainStorm has achieved to date,” said Lebovits. “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 and build upon the positive indications of clinical benefit that we observed in earlier studies.”