ALS patients advocate for experimental treatment, but FDA remains skeptical

The Food and Drug Administration (FDA) is skeptical of an experimental treatment for ALS, or Lou Gehrig's disease, called NurOwn. Despite a yearslong lobbying effort by patient advocates, the FDA maintains that the stem cell-based therapy has not been proven effective. The FDA has expressed major concerns about the evidence provided by drugmaker Brainstorm, stating that a single study does not provide convincing results. However, under pressure from ALS patients and Congress, the FDA has emphasized the urgent need for new ALS treatments and pledged to use regulatory flexibility. At a meeting this week, federal advisers will hear from FDA scientists, company researchers, and patients before taking a non-binding vote on NurOwn's effectiveness. The FDA will make the final decision on the therapy later this year.