BrainStorm's NurOwn: ALS Patients Petition FDA for Reconsideration Amid New Survival Data

BrainStorm Cell Therapeutics' journey with its amyotrophic lateral sclerosis (ALS) treatment NurOwn has taken a new turn as patients and advocates push for FDA reconsideration. The stem cell therapy, which faced regulatory setbacks in recent years, is now at the center of a Citizens' Petition backed by compelling survival data from an expanded access program.

Patient-Led Initiative Seeks FDA Review

Last week, several individuals living with ALS submitted a Citizens' Petition to the FDA, requesting approval of NurOwn based on "new evidence and totality of evidence." The petition, supported by BrainStorm Cell Therapeutics, aims to provide a critical opportunity to reaffirm the therapy's potential in treating ALS.

The petitioners, including lead petitioner Nick Warack, point to real-world data and a 90% survival rate in an expanded access program (EAP) as key factors supporting their case. According to the petition, nine out of ten Phase III participants who entered the EAP survived more than five years from ALS symptom onset, significantly exceeding the typical 10% five-year survival rate for ALS patients.

Clinical Data and Regulatory History

NurOwn's path through clinical trials and regulatory review has been complex. In its Phase III trial, 34.7% of NurOwn-treated patients met the primary endpoint of a 1.5 point per month improvement on the ALS Functional Rating Scale – Revised (ALSFRS-R), compared to 27.7% in the placebo group. While this difference did not reach statistical significance, BrainStorm has consistently argued that a "floor effect" in severely progressed patients may have masked the therapy's true efficacy.

The FDA previously issued a Refusal to File Letter for NurOwn's Biologics License Application (BLA) in 2022, followed by a 17-1 vote against approval by an advisory committee in 2023. These setbacks led BrainStorm to withdraw its BLA and pivot towards planning a Phase IIIb trial.

Patient Advocacy and Real-World Evidence

The petition's supporters, including neurologist Danielle Geraldi-Samara and several trial participants' family members, emphasize the importance of real-world evidence and clinical outcomes in evaluating NurOwn's efficacy. They cite the 21st Century Cures Act, which encourages the FDA to consider such data in accelerating drug development.

Patients who received NurOwn, including Matt Bellina, Matt Klingenberg, and Kade Simons, have reported significant improvements in their condition. These range from increased muscle strength and improved respiratory function to unprecedented gains in mobility.

Looking Ahead: Regulatory Landscape and Industry Impact

As the FDA reviews the Citizens' Petition, the outcome could have far-reaching implications for ALS treatment and the broader field of stem cell therapies. BrainStorm continues to pursue its planned Phase IIIb trial, with an estimated completion date of May 2029, pending funding.

The petition also draws comparisons to cancer drug approvals, arguing that NurOwn's survival data exceeds that of many cancer therapies granted accelerated approval. This comparison highlights ongoing debates about approval standards for treatments targeting rare diseases with critical unmet needs.