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# FDA Reviewers Dismiss ALS Therapy Benefit
- URL: https://www.fdaweb.com/fda-reviewers-dismiss-als-therapy-benefit/
- Published: 2023-09-25T12:00:00.000Z
- Updated: 2026-09-14T18:58:09.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5155404

FDA reviewers say clinical data submitted in a BrainStorm Cell Therapeutics BLA for NurOwn, a stem cell therapy for treating amyotrophic lateral sclerosis (ALS and also known as Lou Gehrig’s Disease), do not demonstrate efficacy and that survival was worse at study completion for subjects who received the therapy. The reviewers' assessment is in a just-released [briefing document](https://www.fda.gov/media/172403/download?ref=fdaweb.com) released in advance of a 9/27 Cellular, Tissue, and Gene Therapy Advisory Committee meeting that will review the data.

BrainStorm refiled the BLA earlier this year under the agency’s “file-over-protest procedure” after the agency issued an 11/10/2022 refusal to file letter. A [file-over-protest submission](https://www.google.com/url?sa=t&rct=j&q=&esrc=s&source=web&cd=&cad=rja&uact=8&ved=2ahUKEwjnj9Xr-vz9AhW4kIkEHTWvDo8QFnoECAoQAQ&url=https%3A%2F%2Fwww.fda.gov%2Fmedia%2F108943%2Fdownload&usg=AOvVaw0VcmZgmOamVqfxY2Aexqhi) is not commonly used, and a recent *JAMA* [article](https://jamanetwork.com/journals/jamainternalmedicine/fullarticle/2775955?ref=fdaweb.com) noted that it has been used just 10 times over the past decade and no approvals were granted for those submissions.

According to the briefing document, the agency now appears to be sticking to earlier concerns raised in an unprecedented 2021 notice ([see story](https://www.fdaweb.com/nurown-als-phase-3-trial-doesnt-support-benefit-fda/)), saying that despite high hopes in the ALS community for NurOwn, Phase 3 trial data did not support its proposed clinical benefit. None of the trial’s primary or secondary endpoints were met, the notice said, with no statistically significant difference between the active and placebo groups.

BrainStorm says the NurOwn technology platform (autologous MSC-NTF cells) “represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders.” MSC-NTF cells are described as “autologous, differentiated bone-marrow-derived mesenchymal stem cells (MSC) that secrete neurotrophic factors (NTFs), regulatory miRNAs that demonstrate neuroprotection and immunomodulation in preclinical models.”