Efficacy and safety of hematopoietic and mesenchymal stem cells in multiple sclerosis: a meta-analysis

Objective Currently available treatment options for multiple sclerosis (MS) have limited efficacy and/or safety concerns. Various mesenchymal stem cells and hematopoietic stem cells have been evaluated in recent clinical trials. The present meta-analysis was conducted to assess the safety and efficacy of stem cell therapy in multiple sclerosis.Methods PubMed/MEDLINE, EMBASE, Cochrane Database, Scopus, and clinical trial registries were searched, and 11 RCTs were included. Quality assessment was performed using the risk-of-bias assessment 2 tool, and the random-effects model was used to estimate the effect size. Subgroup analysis, sensitivity analysis, and meta-regression were performed as applicable. PRISMA reporting guidelines were followed for reporting.Results A total data of 691 patients from 11 RCTs were included, and it showed that there was a greater change in EDSS score from baseline in the stem cell therapy arm as compared to the control arm (MD:-0.35; 95%CI: -0.79 to 0.08; p = 0.104), but was not significant statistically. The change in number of T2 lesions was also not statistically significant between the groups (MD:-0.75; 95%CI: −4.16 to 2.66; p = 0.619). Number of patients with at least one TEAE were comparable between the groups (OR:0.99; 95%CI: 0.61 to 1.62; p = 0.977).Conclusion Stem cell therapy is safe; however, it is no better than control (mostly placebo, mitoxantrone and conventional disease-modifying therapy) in the treatment of MS. Well-designed, adequately powered randomized controlled trials focusing on standardized stem cell types, routes, and dosing are essential to clarify their therapeutic role in multiple sclerosis.Prospero registration number CRD42023457808