A randomized trial of mesenchymal stem cells in multiple system atrophy

Area of Science:

• Neurology
• Regenerative Medicine
• Neurodegenerative Diseases

Background:

• Multiple system atrophy (MSA) is a rapidly progressing neurodegenerative disorder with a poor prognosis.
• Effective neuroprotective or regenerative treatments for MSA are urgently needed.

Purpose of the Study:

• To evaluate the efficacy of autologous mesenchymal stem cells (MSCs) in patients with MSA-cerebellar type (MSA-C).

Main Methods:

• Thirty-three probable MSA-C patients received MSCs (4 × 10^7 cells/injection) via intra-arterial and intravenous routes or placebo.
• Outcomes included changes in the Unified MSA Rating Scale (UMSARS) scores, cerebral glucose metabolism, gray matter density, and cognitive function over 360 days.

Main Results:

• The MSC group exhibited a significantly smaller increase in total and part II UMSARS scores compared to the placebo group (p = 0.047 and p = 0.008).
• Placebo group showed greater decline in cerebral glucose metabolism, gray matter density, and frontal cognition.
• No serious adverse effects were directly linked to MSC treatment, though intra-arterial infusion caused minor ischemic lesions.

Conclusions:

• Mesenchymal stem cell therapy demonstrated potential in delaying neurological deficit progression in MSA-C patients.
• MSC therapy represents a potential treatment candidate for multiple system atrophy.