FDA remains unsure about potential ALS treatment
- Federal regulators will be meeting to discuss an experimental ALS treatment
- They have been skeptical of this treatment for years
- But advocates say it is worth letting patients try
WHITE OAK, MD – JULY 20: A sign for the Food And Drug Administration is seen outside of the headquarters on July 20, 2020 in White Oak, Maryland. (Photo by Sarah Silbiger/Getty Images)
Testing widget old system
(NewsNation) — Federal regulators at the Food and Drug Administration are expected to meet this week to discuss an experimental treatment for Lou Gehrig’s disease, also known as ALS (amyotrophic lateral sclerosis).
But the treatment, called NurOwn, faces deep skepticism in the FDA.
Documents that were disclosed ahead of Wednesday’s meeting on the matter paint a picture of regulators who appeared to be unpersuaded by the treatment’s manufacturer, BrainStorm Cell Therapeutics.
The FDA wrote in those documents that the company’s Biologics License Application, which companies send to the FDA to seek approval to introduce a new product to the market, was “scientifically incomplete to demonstrate substantial evidence of effectiveness, and that the manufacturing information was grossly deficient to ensure adequate product quality.”
During the Wednesday hearing, the FDA will hear from its own scientists, the company behind the drug and patients. This will lead to a nonbinding vote about the treatment’s effectiveness; the final decision about the therapy will be made later in the year.
While the FDA remains skeptical about NurOwn, advocates for ALS patients have been campaigning for its approval.
Brian Wallach, co-founder of the organization I AM ALS, argued that it should still be available even if the treatment only helps a few patients.
“We do not want the perfect to be the enemy of the good,” said Wallach. “The key is to have treatments that make it possible to turn ALS into more of a chronic disease and to allow all patients to live longer and hopefully see a cure.”
