TEL AVIV (Reuters) - Data from the first ALS patients in a clinical trial treated with BrainStorm Cell Therapeutics adult stem cell therapy did not show significant side effects and the treatment has so far proven to be safe, the company said on Tuesday.
Israel-based BrainStorm is developing NurOwn for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease, a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord.
The U.S. Food and Drug Administration last year granted orphan drug designation to NurOwn. The FDA’s orphan drug program encourages the development of treatments for rare diseases by providing financial incentives.
“There have been no significant side effects in the initial patients we have treated with BrainStorm’s NurOwn technology,” said Dimitrios Karussis, the head of Israel’s Hadassah Medical Center’s Multiple Sclerosis unit, who is leading the clinical trial.
“In addition, even though we are conducting a safety trial, the early clinical follow-up of the patients treated with the stem cells shows indications of beneficial clinical effects, such as an improvement in breathing and swallowing ability as well as in muscular power.”
Patients in the trial are transplanted with stem cells derived from their own bone marrow and treated with the NurOwn stem cell technology.
The initial phase of the study is designed to establish the safety of NurOwn and will later be expanded to assess efficacy.
“Based on the interim safety report, the hospital ethical and safety committee granted the company approval to proceed with treating the next patients,” said Chaim Lebovits, president of BrainStorm.
According to the ALS Association, 5,600 people in the United States are diagnosed with the disease each year. It is estimated that as many as 30,000 Americans may have the disease -- which has severely disabled British physicist Stephen Hawking -- at any given time.
Reporting by Tova Cohen; Editing by Helen Massy-Beresford