amyotrophic lateral sclerosis

The drug's failure is a disappointment for patients and advocates, who pressed the FDA and other federal agencies to fund and approve more experimental therapies for the fatal muscle-wasting disease.

The FDA panel voted 17-1 that drugmaker Brainstorm’s stem cell-based treatment has not been shown effective for patients with amyotrophic lateral sclerosis. One panel member abstained from voting.

The agency reiterated its longstanding position that a lone study by drugmaker Brainstorm doesn't provide convincing evidence that its stem cell-based therapy helps patients with amyotrophic lateral sclerosis.

After Brian Wallach was diagnosed with ALS, he and his wife Sandra Abrevaya threw themselves into advocating for patients. Everyone up to President Biden took notice.

The approval came despite a drug-makers' study that failed to meet the company goal of significantly slowing ALS. The drug did make a difference on lab measures of the disease, but the FDA is requiring additional research.

Patients with insurance coverage say the cost set by drugmaker Amylyx Pharmaceutical is fueling insurance delays or denials, and sometimes exorbitant out-of-pocket expenses.

In March, experts who advise the FDA questioned the efficacy of an experimental new drug for ALS. In September, they voted to approve it anyway.

The vote by the agency's health advisers is a potential setback for patient groups who have lobbied for the medication’s approval for more than a year.

Scientists say new drugs are on the way for patients with ALS. The latest is a two-drug combo that appears to slow the progression of the fatal nerve disease with a modest but meaningful benefit.

Structures inside healthy brain cells nimbly move from one state to the next to perform different functions. But in certain degenerative brain diseases, scientists now think, that process gets stuck.