On December 14th, BrainStorm cell therapeutics announced an expanded access program (EAP) for the recent phase three trial of their investigational cell-based therapy NurOwn in Amyotrophic Lateral Sclerosis (ALS). This means that some participants in the trial will be able to continue receiving the treatment as the FDA continues to review the results.
In order to qualify for the program, an individual must have participated in and completed the trial, as well as meeting certain additional requirements. The EAP will initially only enroll those who are “less affected by ALS” according to their ALSFRS-r scores. While this will be up to the discretion of the treating physician, it will likely mean those with an ALSFRS-r score of 35 points or more.
The Phase 3 Trial for NurOwn, which published results in November of 2020, missed its primary endpoints, failing to produce clinically significant results in participants with rapidly progressing ALS. However, investigators did report potentially promising biomarker data in some participants with more recent diagnoses and higher ALSFRS-r scores. The results of the trial are currently under review by the FDA – whose approval was also required for the EAP.
"We are pleased to have the opportunity to treat additional patients with NurOwn through this Expanded Access Program, which was strongly advocated for by members of the ALS advocacy community,” said BrainStorm CEO Chaim Leibowitz in a press release. “We recognize the urgency with which people with ALS want and need access to new potential treatments. This EAP is an important next step to providing an immediate option for some patients. We remain committed to rapidly advancing NurOwn through clinical development and regulatory review in the hope that the greatest number of people living with ALS may benefit."
Investigators at the sites where the trial occurred will be reviewing participant data and contacting those who are eligible for the EAP. Brainstorm will cover the cost for these participants. They will also continue collecting data about those enrolled in the program, and may later expand to others who participated in the original trial.
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