The U.S. Food and Drug Administration has approved the start of a mid-stage clinical trial of an adult stem cell treatment for patients with amyotrophic lateral sclerosis (ALS) according to its developer, BrainStorm Cell Therapeutics Inc. The Phase II clinical trial will be launched initially at UMass Medical School and Massachusetts General Hospital in Boston.
BrainStorm, an Israel-based biotech company developing stem cell technology to provide treatments for currently incurable neurodegenerative diseases, has been working with Robert H. Brown Jr., DPhil, MD, the Leo P. and Theresa M. LaChance Chair in Medical Research and chair and professor of neurology, to bring their stem cell treatment, NurOwn, to the U.S. for clinical trials (…)
The Phase II trial for NurOwn is designed to evaluate the safety and efficacy of transplantation of the stem cells in 48 ALS patients. The cells will be administered via intramuscular and intrathecal injection. Patients will be followed monthly for three months before transplantation and for six months following transplantation.
Earlier clinical trials have shown that treatment with NurOwn cells was well tolerated and safe. This will be the first Phase II double-blinded stem cell study to be conducted for ALS, according to BrainStorm.
The NurOwn technology, based on discoveries at Tel Aviv University, processes adult human stem cells in bone marrow that can self-renew and differentiate into many cell types. BrainStorm believes the adult stem cells can differentiate into cells that can release neurotrophic factors, including glial-derived neurotrophic factor, a small protein that may promote survival of many types of neurons. In 2011, BrainStorm was granted an Orphan Drug Designation for NurOwn by the FDA (…)
(…) “Today’s announcement represents the most significant milestone BrainStorm has achieved to date,” said Mr. Chaim Lebovits, President. “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” (…)
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(TM)) in 48 ALS patients. The NurOwn(TM) 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 (…)