FDA advisers reject experimental stem cell treatment for ALS

A panel of outside experts for the Food and Drug Administration on Wednesday voted 17-1 that a treatment for Lou Gehrig’s disease has not been shown to be effective.

Drugmaker BrainStorm Cell Therapeutics this month tried to get the treatment, called NurOwn, approved for use in patients with mild to moderate Lou Gehrig’s, also called ALS, or amyotrophic lateral sclerosis.

The FDA in 2022 rejected an earlier approval application from BrainStorm. The company’s 200-patient study did not show that the therapy extended life, slowed the disease, or improved patient mobility. The FDA convened Wednesday’s panel after ALS patients and advocates submitted a 30,000-signature petition seeking a public meeting.

What other treatments exist for ALS? The FDA in the last year has approved two new drugs to treat the disease. Advocacy groups have increased lobbying efforts in recent years to push the FDA to approve experimental treatments for terminal illnesses like ALS.

This story originally appeared in WORLD. © 2023, reprinted with permission. All rights reserved.