FDA Panel Says No to Experimental ALS Drug

THURSDAY, Sept. 28, 2023 (HealthDay News) -- An advisory panel to the U.S. Food and Drug Administration on Wednesday voted resoundingly against recommending a stem cell-based experimental treatment for amyotrophic lateral sclerosis (ALS).

Although the FDA is not bound by the votes of its advisory panels, agency scientists have already penned a scathing review of the drug, called NurOwn. The application from Brainstorm, the company that developed the treatment, was "scientifically incomplete" and "grossly deficient," FDA staff scientists wrote in the review.

Meanwhile, the advisory panel voted 17-1 against the drug to treat ALS. Only a panelist representing patients voted for the medication, while one adviser abstained from voting, the Associated Press reported.

"Creating false hope can be considered a moral injury and the use of statistical magic or manipulation to provide false hope is problematic," said Lisa Lee, Ph.D., a bioethics and research integrity expert from Virginia Tech, who voted against the treatment, the AP reported.

The FDA agreed to convene the advisory panel in response to a 30,000-signature petition from ALS patients and advocates. But a study from Brainstorm involving 200 patients did not show that NurOwn extended life, slowed disease, or improved patient mobility, the AP reported. Not only that, but the Brainstorm application was missing details on manufacturing and quality control.

"It really is a disease that needs a safe and effective treatment and there are a lot of other prospects out there that we need to encourage. Approving one like this would get in the way of that," said Kenneth Fischbeck, M.D., a distinguished investigator in the Hereditary Neurological Diseases branch of the National Institutes of Neurological Disorders and Stroke, the AP reported.

Associated Press Article