Safety and Efficacy of Mesenchymal Stem Cell Therapy in Multiple System Atrophy: Systematic Review
Noon Elimam1, Shams Albarari2, Yara Shaalan3, Shazaa Elsheikh4, Ainaa A.Alzamar5, Nourhan Elmekkawi6, Rahaf Mogahed7, Razan Alghuweiri8, Sara N. Ibrahim9
1Faculty of Medicine, Bogomolets National Medical University, Kyiv, Ukraine, 2Faculty of Medicine, Tbilisi State Medical University, Tbilisi, Georgia, 3Faculty of Medicine, Misr University for Science and Technology, 6th of October City, Egypt, 4Clinical Pharmacology, Benha Faculty of Medicine, Benha, Egypt, 5Palestinian medical complex, Faculty of Medicine, Al-Quds University, Palestine, 6Faculty of Medicine, Zifta General Hospital, Egypt, 7Faculty of Medicine, 6th of October University, 6th of October city, Egypt, 8Faculty of Pharmacy, Jordan University of Science and Technology, Irbid, Jordan, 9Faculty of Medicine, Ahfad University for Women, Khartoum, Sudan
Objective:
To systematically evaluate the efficacy and safety of mesenchymal stem cell (MSC) therapy for patients with Multiple System Atrophy (MSA) by synthesizing evidence from clinical trials.
Background:
MSA is a rapidly progressive and fatal neurodegenerative disorder for which no disease-modifying therapies exist. MSC therapy has emerged as a potential treatment, with proposed mechanisms of action centered on neuroprotection and disease modification through anti-inflammatory and trophic effects, rather than direct cell replacement.
Design/Methods:
We systematically searched PubMed, Scopus, the Cochrane Library, and Web of Science for studies on mesenchymal stem cell (MSC) therapy in adults with probable or confirmed multiple system atrophy (MSA). Eligible studies included single-arm trials or comparisons with placebo or usual care. The primary outcome was safety and tolerability, assessed by the type, frequency, and severity of adverse events. Secondary outcomes included the rate of disease progression measured by UMSARS total, part I, and part II scores. Risk of bias was assessed using the Cochrane RoB 2 tool.
Results:
Seven studies (n = 123) met the inclusion criteria. Participants with probable MSA (predominantly MSA-C) received various MSC types via intra-arterial, intravenous, intrathecal, or cisterna magna routes. Adverse events occurred in ~65–70%, mainly fever or headache. Dose-limiting toxicity appeared only at the highest intra-arterial dose. No serious adverse effects occurred. MSC therapy, in all studies, slowed progression of UMSARS scores versus baseline or placebo (e.g. +5.15 ± 1.46 vs +10.90 ± 1.26; p = 0.006).
Conclusions:
MSA therapy demonstrates potential for disease modification in MSA, effectively slowing the progression of neurological deficits. The intervention was well-tolerated in the studied population, supporting the need for larger, definitive clinical trials.
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