Abstract Details

Title Safety and Efficacy of Mesenchymal Stem Cell Therapy in Multiple System Atrophy: Systematic Review

Topic Movement Disorders

Presentation(s) P8 - Poster Session 8 (11:45 AM-12:45 PM)

Poster/Presentation
Number 16-015

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.

Authors/Disclosures
Sharvani Gurupadayya Salimath, MD PRESENTER	Dr. Salimath has nothing to disclose.
Noon Elimam, MBBS	Dr. Elimam has nothing to disclose.
Shams S. Albarari	Shams S. Albarari has nothing to disclose.
Yara Shaalan (Misr University for Science and Technology)	Ms. Shaalan has nothing to disclose.
Shazaa M. Elsheikh, MD	Mrs. Elsheikh has nothing to disclose.
Ainaa A. Alzamari, MD	Dr. Alzamari has nothing to disclose.
Nourhan M. Elmekkawi III, MD	Dr. Elmekkawi has nothing to disclose.
Rahaf Mogahed, MD	Dr. Mogahed has nothing to disclose.
Razan H. Alghuweiri	Dr. Alghuweiri has nothing to disclose.
Sara Nihro Ibrahim	Sara Nihro Ibrahim has nothing to disclose.

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