FDA skeptical of experimental ALS treatment pushed by patient advocates

For free real time breaking news alerts sent straight to your inbox sign up to our breaking news emails Sign up to our free breaking news emails Sign up to our free breaking news emails SIGN UP I would like to be emailed about offers, events and updates from The Independent. In documents posted Monday, the FDA reiterated its longstanding position that drugmaker Brainstorm's lone study doesn't provide convincing evidence that its stem cell-based therapy helps patients with ALS, or amyotrophic lateral sclerosis. “FDA is the 800-pound gorilla here and if they’re convinced that the drug doesn’t work it’s very hard to change their minds,” said Marc Scheineson, a former associate FDA commissioner who now consults for drugmakers. “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.” Still, there’s little consensus on NurOwn among the normally tight-knit ALS community. “I don’t want to lose a potential treatment for ALS, but I also don’t want to foist on the public an expensive treatment that doesn’t work,” said Dr. Terry Heiman-Patterson of Temple University.