Adcomm Votes Down ALS Stem Cell Therapy, Debates Clinical Data vs. Personal Data

October 2, 2023

Despite strong testimony and comments from patients and clinicians, the FDA advisory committee reviewing NurOwn, an investigational treatment for amyotrophic lateral sclerosis (ALS), voted against recommending the drug, citing a lack of consistent data and ill-defined biomarkers.

The Cellular, Tissue and Gene Therapies Advisory Committee voted almost unanimously in its rejection — one person voted yes and one abstained — after a discussion about trial design and how much weight to give personal data.

Consumer representative and founder of ALS Connect, Kathleen O’Sullivan-Fortin, asked the committee to consider both the scientific data and the personal data from patients and others who took the time to submit their views on paper. The FDA received an extraordinary 2,000 public comments ahead of the meeting in support of the therapy, many noting the severe lack of effective treatments available for this deadly disease.

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