Comment: Autologous mesenchymal stem cells: Hope for patients with multiple system atrophy?

Abstract

Multiple system atrophy (MSA), a rapidly progressive neurodegenerative disorder with parkinsonism, cerebellar ataxia, and autonomic failure, has few effective therapies, so disease-modifying therapies remain a critical need. So far, randomized controlled trials (RCTs) testing a variety of drug candidates largely failed.1 The exception was a small-scale interventional RCT of sequential intra-arterial and IV administration of mesenchymal stem cells (MSC) in patients with cerebellar-type MSA2 that found a small reduction in the increase of severity rating scores (Unified MSA Rating Scale [UMSARS]) over 1 year, as compared to placebo.