ALS

Israel's BrainStorm says stem cell drug benefits ALS patients

Israel's BrainStorm Cell Therapeutics said a mid-stage clinical trial of its adult stem cell treatment showed a "statistically significant" effect in patients with amyotrophic lateral sclerosis (ALS).

According to the ALS Association, 5,600 people in the United States are diagnosed each year with the neurodegenerative disease, also known as Lou Gehrig's Disease, which has severely disabled British physicist Stephen Hawking.

New data to be presented at the American Academy of Neurology annual meeting on Tuesday show that six months after a single administration of the stem cell treatment called NurOwn there was a statistically significant improvement in the rate of decline in Forced Vital Capacity, which measures the amount of air a person can dispense from the lungs.

There was also improvement in the rate of decline in the ALS Functional Rating Score, which tests 12 different functions.

In addition, patients who received NurOwn through an intramuscular injection showed an improvement in the rate of decline in muscle mass in the right arm, the site of NurOwn administration, as compared to the left arm.

"Given the nature of ALS and lack of effective therapies, a treatment that could even modestly slow progression would be welcomed by patients and physicians," said Dimitrios Karussis of Hadassah University Medical Center, the principal investigator for the study.

In October the U.S. Food and Drug Administration designated NurOwn as a "fast-track" product to treat ALS.

BrainStorm, which is also conducting clinical trials at three sites in the United States, plans to move to a study in the next few months to see if the results can be amplified with repeated doses.