ALS Stem Cell Trial: Efficacy and Safety of NurOwn in Early & Moderate ALS (ENDURANCE STUDY)
Brainstorm Therapeutics is back, and they’ve learned a lot from their previous trials. If you don’t know the history of Nurown, their ALS stem cell candidate, it showed early promise in Phase 2 with some slowed disease progression. In Phase 3, it failed to meet its primary endpoint, however, a post-hoc analysis suggested potential benefit […]
Brainstorm Therapeutics is back, and they’ve learned a lot from their previous trials. If you don’t know the history of Nurown, their ALS stem cell candidate, it showed early promise in Phase 2 with some slowed disease progression.
In Phase 3, it failed to meet its primary endpoint, however, a post-hoc analysis suggested potential benefit in a subgroup of patients with less advanced ALS. Despite mixed results, the company and a significant chunk of the ALS community has continued to advocate for it, citing the lack of ALS treatments available.
The ALS Foundation issued this statement on those previous trials:
https://www.als.org/research/als-research-topics/drug-development/our-position-supporting-approval-experimental/our-position-nurown
This study will evaluate if Debamestrocel – MSC-NTF (autologous bone marrow-derived stem cells with high secretions of neurotophic factors, a.k.a. NurOwn) can help participants with early symptomatic Amyotrophic Lateral Sclerosis (ALS) and moderate disease presentation.
Researchers will administer NurOwn cells suspended in Dulbecco Modified Eagle Medium (DMEM), comparing it to placebo. Participants will receive either NurOwn or placebo every 8 weeks for 24 months, followed by an open-label extension where all participants receive NurOwn every 8 weeks for an additional 24 months.
The primary outcome will measure changes in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) total score from baseline to Week 24. This clinician-administered questionnaire consists of 12 items, each scored from 0 to 4, with higher total scores indicating better outcomes. Secondary outcomes include evaluating changes in respiratory function from baseline to Week 24, measured by slow vital capacity (SVC).
Approximately 200 participants will enroll in this Phase 3 clinical trial sponsored by Brainstorm-Cell Therapeutics. The study team will conduct visits approximately every 8 weeks for checkups and tests. They expect to finish the trial in May of 2029.
Link to trial:
https://clinicaltrials.gov/study/NCT06973629
