2026-07-27
“Can Stem Cells Cure ALS?” — What a Phase 3 Trial That Missed Its Primary Endpoint Still Taught Us
A phase 3 trial in ALS of autologous mesenchymal stem cells induced to secrete high levels of neurotrophic factors (NurOwn). The primary endpoint was not met (33% vs 28%, OR=1.33, P=.45). The signal in the less-impaired subgroup was not statistically significant and is only hypothesis-generating, yet the CSF biomarkers showed a clear biological effect. A piece for reading expectation and reality apart.
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