Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy in Hereditary Spinocerebellar Ataxia

Author(s): Jia-Li Jin, Zhuo Liu, Zhen-Juan Lu, De-Ning Guan, Chong Wang, Zhi-Bin Chen, Jun Zhang, Wei-Yun Zhang, Jia-Yong Wu, Yun Xu

Journal Name: Current Neurovascular Research

Volume 10 , Issue 1 , 2013

Become EABM
Become Reviewer


Hereditary spinocerebellar ataxia (SCA) is a devastating, incurable disease. Stem-cell-based therapies represent new promise for clinical research in neurology. The objectives of this study were to assess the feasibility, efficacy, and potential toxicity of human umbilical cord mesenchymal stem cells (UCMSCs) therapy in patients with SCA. Sixteen genomically diagnosed SCA patients were enrolled and received intravenous and intrathecal infusion of UCMSCs. Clinical, laboratory, and radiographic evaluations were conducted to assess the safety of UCMSC therapy. Efficacy was evaluated by the Berg Balance Scale (BBS) and International Cooperative Ataxia Rating Scale (ICARS) scores. Among the 16 cases, there were no serious transplant-related adverse events happened in 12 months follow-up. The majority of patients showed improved BBS and ICARS scores continuing for at least 6 months which indicated UCMSC therapy could alleviate SCA symptoms. This study suggested that UCMSC transplantation was safe and might delay the progression of SCA. This may represent a new therapeutic strategy for SCA and other genetic neurological diseases.

Keywords: Berg Balance Scale, Efficacy, International Cooperative Ataxia Rating Scale, Safety, Spinocerebellar Ataxia, Umbilical Cord Mesenchymal Stem Cell

Rights & PermissionsPrintExport Cite as

Article Details

Year: 2013
Page: [11 - 20]
Pages: 10
DOI: 10.2174/1567202611310010003

Article Metrics

PDF: 180