The US Food and Drug Administration met this week to consider the approval of an experimental treatment for Lou Gehrig’s disease, the culmination of years of lobbying by patients with the deadly disease. neurodegenerative disease.
But those advocates still face a big hurdle: FDA regulators say the treatment hasn’t been proven to work.
In documents released Monday, the FDA reiterated its long-standing position that a study by drugmaker Brainstorm did not provide strong evidence that stem cell-based therapy could help patients with amyotrophic lateral sclerosis. (ALS).
This is the same message the FDA conveyed to company executives in early 2021 when they first shared data on the treatment, called NurOwn. And again last November, when the FDA refused to accept the company’s request for review.
But with the support of thousands of ALS patients, Brainstorm took the unusual step of “filing a protest,” forcing the FDA to make a decision.
“The FDA here is an 800-pound gorilla, and if you’re convinced that a drug doesn’t work, it’s very difficult to change your mind,” said Marc Scheineson, a former FDA associate commissioner who now advises the drug manufacturers.
Still, ALS patients find reason to be optimistic.
Under pressure from the ALS community and Congress, FDA officials recently emphasized the “urgent need” for new treatments for the disease and pledged to use the highest “availability regulatory flexibility” in its review. The FDA approved two new ALS drugs last year, though neither of them met the agency’s traditional approval standards.
NurOwn is the clearest evidence of how far the agency is willing to compromise to get a new drug approved for a rare and deadly disease with few treatment options.
ALS gradually destroys the nerve connections necessary for basic movements and functions, including breathing. Most people die within five years of diagnosis.
At Wednesday’s meeting, federal advisers will hear from FDA scientists, company researchers and patients before taking a no vote on NurOwn’s effectiveness. The FDA will make a final decision on the therapy later this year.
The meeting was scheduled after ALS advocates submitted 30,000 signatures calling for a public investigation into the treatment.