TEL AVIV BrainStorm Cell Therapeutics said the U.S. Food and Drug Administration approved the start of a mid-stage clinical trial of its adult stem cell treatment for patients with amyotrophic lateral sclerosis (ALS).
The Phase II trial will be launched initially at Massachusetts General Hospital in Boston and the University of Massachusetts Memorial Hospital in Worcester.
Dana-Farber Cancer Institute's Connell O'Reilly Cell Manipulation Core Facility will manufacture BrainStorm's NurOwn cells for these two clinical sites. The trial will also be conducted at the Mayo Clinic, the Israel-based company said on Sunday. The trials are expected to start soon.
"Today's announcement represents the most significant milestone BrainStorm has achieved to date," the company's president, Chaim Lebovits, said.
This trial will be the first Phase II double-blinded stem cell study to be conducted for ALS, he added.
ALS, also known as Lou Gehrig's Disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord.
BrainStorm's Phase II trial 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.
Last week, Cytokinetics Inc said its experimental treatment for Lou Gehrig's Disease failed the main goal in a mid-stage trial.
(Reporting by Tova Cohen)