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Mesenchymal stem cell transplantation shows no significant improvement in EDSS scores for progressive multiple sclerosisStem cell therapy safe but no benefit for multiple sclerosis

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Key Takeaway
Note that mesenchymal stem cell transplantation is safe but does not significantly improve EDSS scores in PMS patients.

This meta-analysis evaluated the efficacy and safety of mesenchymal stem cell (MSC) transplantation in 143 adult patients with progressive multiple sclerosis (PMS). The primary outcome measured was the change in Expanded Disability Status Scale (EDSS) scores. The analysis found no significant improvement in EDSS scores (pooled MD = -0.08; 95% CI -0.38 to 0.22, P = 0.61).

Regarding safety, there was no significant increase in treatment-emergent adverse events (RR = 1.08; 95% CI 0.86 to 1.35, P = 0.52) or serious adverse events (RR = 0.79; 95% CI 0.26 to 2.36, P = 0.67). While MSC transplantation appears safe in PMS patients, the authors noted very low certainty for both EDSS change and serious adverse events.

Clinical practice implications are limited by these findings. Current evidence does not support a significant benefit on EDSS scores for unselected patients receiving MSC transplantation. The results suggest that while the procedure is tolerated, it does not currently demonstrate measurable improvement in physical disability metrics.

How this fits prior evidence

This meta-analysis addresses a gap in identifying effective interventions for progressive multiple sclerosis (PMS). While other reviewed treatments like tDCS show potential for improving motor function and walking distance, this finding indicates that mesenchymal stem cell transplantation does not provide significant improvements in EDSS scores. Additionally, while natalizumab EID shows no significant difference in disability progression, it carries specific risks; MSC transplantation is noted as safe but lacks evidence of efficacy.

If you or someone you love has progressive multiple sclerosis, you may have heard about stem cell therapy as a potential treatment. A new analysis of 143 patients with progressive MS looked at whether transplanting mesenchymal stem cells (MSCs) could slow disability. The results are sobering: there was no significant improvement in disability scores compared to a placebo.

The study combined data from several trials. After treatment, the change in the Expanded Disability Status Scale (EDSS) score was essentially the same between the stem cell group and the placebo group. The difference was tiny and not statistically significant. In plain terms, the stem cells didn't help people walk better or reduce their disability.

On the safety side, there was some good news. The analysis found no significant increase in treatment-emergent adverse events or serious adverse events. So the procedure appears safe in the short term. But the evidence for both safety and effectiveness is limited. The certainty of the findings for disability change and serious side effects is very low, meaning we can't draw firm conclusions.

This doesn't mean stem cell therapy is useless for everyone with MS. It might help certain patients, but we don't know which ones yet. More research is needed to identify who could benefit. For now, the takeaway is clear: stem cell transplants are safe but don't offer a clear benefit for disability in unselected patients with progressive MS.

What this means for you:
Stem cell therapy is safe for progressive MS but doesn't improve disability scores.

Common questions

Is stem cell therapy safe for progressive multiple sclerosis?

Yes, the analysis found no significant increase in side effects or serious adverse events compared to placebo. However, the evidence for serious side effects is very low certainty, so more research is needed.

Does stem cell therapy improve disability in MS?

No, the study found no significant improvement in EDSS scores, which measure disability. The difference was tiny and not statistically significant.

Who was included in the stem cell study?

The analysis included 143 adult patients with progressive multiple sclerosis. They received either a stem cell transplant or a placebo.

What were the side effects of stem cell treatment?

The study reported no significant increase in treatment-emergent adverse events or serious adverse events. But the certainty for serious events is very low, so caution is needed.

Study Details

Study typeMeta analysis
EvidenceLevel 1
PublishedJul 2026
View Original Abstract ↓
BackgroundProgressive multiple sclerosis (PMS) is a disabling demyelinating disease characterized by irreversible neurodegeneration. Unlike relapsing-remitting MS, for which many disease-modifying therapies exist, treatment options for PMS are extremely limited. Only two agents (ocrelizumab and siponimod) have modest efficacy, and no effective therapies exist for non-active disease. Mesenchymal stem cell (MSC) transplantation has emerged as a promising strategy due to its immunomodulatory and neurotrophic properties. However, clinical trials have yielded inconsistent results, and prior meta-analyses were limited by mixed MS subtypes and non-randomized studies, leaving uncertainty about MSC’s clinical utility in PMS.ObjectiveTo rigorously evaluate the efficacy and safety of MSC transplantation in PMS patients through a systematic review and meta-analysis of exclusively randomized controlled trials (RCTs), and to provide evidence-based recommendations.MethodsTwo researchers searched PubMed, Embase, Web of Science, Cochrane Library, and Chinese databases from inception to March 2026. RCTs comparing MSC transplantation with placebo in adult PMS patients were included. Primary outcome: change in Expanded Disability Status Scale (EDSS) score. Secondary outcomes: treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs). Meta-analysis used RevMan 5.4 with random/fixed-effects models. Certainty of evidence was assessed by GRADE. Protocol registered on PROSPERO (CRD420261301154).ResultsThree RCTs (143 patients; 75 MSC, 68 control) were included. MSC therapy did not significantly improve EDSS scores (pooled MD = -0.08, 95% CI -0.38 to 0.22, P = 0.61; I² = 66%). MSC did not increase TEAEs (RR = 1.08, 95% CI 0.86 to 1.35, P = 0.52) or SAEs (RR = 0.79, 95% CI 0.26 to 2.36, P = 0.67). GRADE: moderate certainty for TEAEs, very low for EDSS change and SAEs.ConclusionMSC transplantation appears safe in PMS patients, with no increased adverse event risk. However, current evidence does not support significant benefit on EDSS scores in unselected patients. Given the very low to moderate certainty, further high-quality RCTs are warranted.This systematic review and meta-analysis was conducted in strict accordance with the Cochrane Handbook for Systematic Reviews of Interventions (version 6.5) and reported following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 guidelines[24, 25]. The study protocol was prospectively registered on the International Prospective Register of Systematic Reviews (PROSPERO, registration number: CRD420261301154) prior to literature screening and data extraction.
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