Stem Cells for ALS: Anxiously Awaiting a Clear Signal of Efficacy

In a phase II trial, 64 patients with mild-to-moderate amyotrophic lateral sclerosis (ALS; ALS Functional Rating Scale-Revised [ALSFRS-R] scores, 31–46) were randomized to receive standard treatments including riluzole, with (33 patients) or without (31 patients) 2 intrathecal injections of bone marrow–derived mesenchymal stem cells (BM-MSCs) 1 month apart, and were followed for 4 and 6 months postinjection.

The mean changes in the ALSFRS-R from baseline to 4 and 6 months, the primary endpoint, were significantly smaller in the MSC group than the control group; the between-group difference was 2.98 (−1.69 with MSC vs. −4.67 in controls) at 4 months and 3.38 (−3.10 with MSC vs. −6.48 in controls) at 6 months. There were no serious adverse eve…