Abstract

Even if there is sparse evidence of efficacy of stem cell administration for amyotrophic lateral sclerosis (ALS) in preclinical studies, the clinical use of cell-based therapy is yet to be defined. To assess the efficacy, feasibility and safety of cell-based therapy in people with ALS/MND, compared with placebo or no treatment. A Cochrane Review on the topic was summarized with comments. Two randomized controlled trials met the selection criteria, but only one provided data useful for the analysis. It compared autologous bone marrow-mesenchymal stem cells, combined with riluzole, to riluzole only, in 64 people with ALS. The ALS Functional Rating Scale-Revised score slightly improved 6 months after the intervention, though the change was not clinically meaningful. Respiratory function, overall survival and the risk of total adverse events or serious adverse events were not different in the two groups. The available evidence does not support the use of bone marrow-mesenchymal stem cells to treat people with ALS/MND.

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