Cell Therapy for ALS Associated with Increased Survival at 5 Years

Nine out of 10 people with amyotrophic lateral sclerosis (ALS) treated with NurOwn (autologous mesenchymal stem cells [MSC]–neurotrophic factors [NTF]; BrainStorm Cell Therapeutics, New York, NY) survived more than 5 years after symptom onset. These results from an analysis of survival data from an Expanded Access Program (EAP) exceeded population-level survival expectations for those diagnosed with ALS, indicating a clinically meaningful observation, according to a statement released by the company.

The 10 participants enrolled in the EAP received access to NurOwn, with analysis assessing survival data. Eligibility was limited to those who had previously completed a phase 3 clinical trial (NCT03280056), which evaluated the safety and efficacy of repeated administration of NurOwn in those diagnosed with ALS. This phase 3 trial did not achieve its primary end point of a 1.25 point per month increase in Revised ALS Functional Rating Score (ALSFRS-R) slope from pretreatment to posttreatment. In the EAP, survival data were compiled from the onset of symptoms through both EAP treatment periods and extended using public sources. At the start of the EAP, participants had a mean ALSFRS-R score of 31.4.

Key findings from the EAP include the following:

• 90% of participants survived for more than 5 years postsymptom onset, while only approximately 10% of the overall ALS population reaches this milestone.
• The median survival was 6.8 years (range: 6 to 7 years).
• One death occurred within 5 years, which resulted from elective euthanasia.
• Six of 10 participants (60%) remain alive >7 years after the onset of symptoms.

“The fact that 9 out of 10 EAP participants surpassed five years of survival from first symptom—in contrast with the very modest expected survival pattern typically seen in ALS—represents a clinically meaningful observation,” said Bob Dagher, MD, Chief Medical Officer of BrainStorm Cell Therapeutics. “Furthermore, 6 out of 10 patients are still alive, even post seven years from symptom onset. These data further strengthen the scientific rationale to pursue our pivotal Phase 3b trial of NurOwn in ALS, under full alignment with the FDA guidance.”