Experimental treatment pushed by ALS patients gets day before FDA, but agency unconvinced it works

The Food and Drug Administration meets this week to consider approval of an experimental treatment for Lou Gehrig’s disease, the culmination of a yearslong lobbying effort by patients with the fatal neurodegenerative disease.

Those advocates still face one giant hurdle: FDA regulators say the treatment hasn’t been shown to work.

In documents posted Monday, the FDA 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 ALS, or amyotrophic lateral sclerosis.