Abstract
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
Current Neurovascular Research
Title:Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy in Hereditary Spinocerebellar Ataxia
Volume: 10 Issue: 1
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 and Yun Xu
Affiliation:
Keywords: Berg Balance Scale, Efficacy, International Cooperative Ataxia Rating Scale, Safety, Spinocerebellar Ataxia, Umbilical Cord Mesenchymal Stem Cell
Abstract: 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.
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Jin Jia-Li, Liu Zhuo, Lu Zhen-Juan, Guan De-Ning, Wang Chong, Chen Zhi-Bin, Zhang Jun, Zhang Wei-Yun, Wu Jia-Yong and Xu Yun, Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy in Hereditary Spinocerebellar Ataxia, Current Neurovascular Research 2013; 10 (1) . https://dx.doi.org/10.2174/1567202611310010003
DOI https://dx.doi.org/10.2174/1567202611310010003 |
Print ISSN 1567-2026 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5739 |
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