Appelt Pablo Andrei, Comella Kristin, de Souza Luciane Aparecida Pascucci Sande, Luvizutto Gustavo José
Master student in Physical Therapy of Federal University of Triângulo Mineiro (UFTM), Uberaba, Minas Gerais, Brazil.
Chief Scientific Officer of US Stem Cell Clinic, Weston, Florida, USA.
Cerebellum Ataxias. 2021 Feb 25;8(1):8. doi: 10.1186/s40673-021-00130-8.
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.
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.
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.
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.
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.
脊髓小脑共济失调是一种遗传性神经退行性疾病，其特征为平衡、运动和运动协调能力发生改变。目前正在研究干细胞疗法作为延缓该疾病进展的替代方法，并且一些实验研究已探讨了干细胞治疗对脊髓小脑共济失调的影响。
本综述的目的是研究干细胞的应用是否对脊髓小脑共济失调患者的功能恢复产生影响。
本综述纳入的研究调查了从脐带和脂肪组织中提取的间充质干细胞应用方案的疗效和安全性。两项研究采用鞘内注射途径给药，一项研究采用静脉注射途径给药。
研究显示，国际合作共济失调评定量表（ICARS）、日常生活活动量表（ADL）、伯格平衡量表（BBS）和共济失调评估与分级量表（SARA）的评分有临床改善，但缺乏统计学意义。
在脊髓小脑共济失调患者中推荐干细胞治疗的证据不足，且在改善患者功能恢复方面未观察到统计学差异。需要采用不同设计、更大样本量和安慰剂对照进行进一步研究，以全面了解脊髓小脑共济失调细胞治疗的预期结果。
