Effect of stem cell treatment on functional recovery of spinocerebellar ataxia: systematic review and meta-analysis

• Main Authors: Pablo Andrei Appelt, Kristin Comella, Luciane Aparecida Pascucci Sande de Souza, Gustavo José Luvizutto
• Format: Article
• Language: English
• Published: BMC 2021-02-01
• Series: Cerebellum & Ataxias
• Subjects: Spinocerebellar ataxia; Stem cells; Hereditary; Mesenchymal
• Online Access: https://doi.org/10.1186/s40673-021-00130-8

Abstract Background Spinocerebellar ataxia is a hereditary neurodegenerative disease characterized by changes in balance, locomotion and motor coordination. Stem cell therapies are currently being investigated as an alternative to delay the evolution of the disease, and some experimental studies have investigated the effect of stem cell treatment on spinocerebellar ataxia. Objectives The aim of this review was to investigate whether the application of stem cells produced an effect on functional recovery in individuals with spinocerebellar ataxia. Methods The studies included in this review investigated the efficacy and safety of a protocol for the application of mesenchymal stem cells extracted from umbilical cord and adipose tissue. Two studies used intrathecal route for application and one study used intravenous route. Results Studies have shown clinical improvement in the scores of the ICARS (International Cooperative Ataxia Rating Scale), ADL (Activities of Daily Living Scale), BBS (Berg Balance Scale) and SARA (Scale for the Assessment and Rating of Ataxia), but lacked statistical significance. Conclusions There was low evidence for recommending stem cell therapy in individuals with spinocerebellar ataxia, and no statistical difference was observed for improving functional recovery of patients. Further studies are needed with different designs, largest sample sizes and placebo control, to fully understand anticipated outcomes of cellular therapy for spinocerebellar ataxia.