Human umbilical cord blood-derived mononuclear cell transplantation: case series of 30 subjects with Hereditary Ataxia

Journal of Translational Medicine

Research

SC Cho¹  Ning Zhao²  You-Xiang Sheng²  Guo-Jian Shu²  Ying Tang²  Wan-Zhang Yang²  Shao-Hui Li²  Fang Wu²  Min Zhang²  Chun-Zhen Li²  Matthew Gandjian³  Thomas E Ichim³  Shan Jiang⁴  Xiang Hu⁴  Shu Jiang⁴  Yun Zhang⁴ 

[1] Department of Neurology and Neurosurgery, Stanford University, Stanford, CA, USA;Department of Rehabilitation Medicine, Nanshan Affiliated Hospital of Guangdong Medical College, Shenzhen, China;Medistem Inc, San Diego, CA, USA;Shenzhen Beike Cell Engineering Research Institution, Shenzhen, China;

关键词: Amyotrophic Lateral Sclerosis;    Spinocerebellar Ataxia;    Human Umbilical Cord;    Berg Balance Scale;    Rehabilitation Training;   

DOI  :  10.1186/1479-5876-9-65

 received in 2011-01-30, accepted in 2011-05-16,  发布年份 2011

来源: Springer

【 摘 要 】

BackgroundThe differential diagnosis for hereditary ataxia encompasses a variety of diseases characterized by both autosomal dominant and recessive inheritance. There are no curative treatments available for these neurodegenerative conditions. This open label treatment study used human umbilical cord blood-derived mononuclear cells (CBMC) combined with rehabilitation training as potential disease modulators.Methods30 patients suffering from hereditary ataxia were treated with CBMCs administered systemically by intravenous infusion and intrathecally by either cervical or lumbar puncture. Primary endpoint measures were the Berg Balance Scale (BBS), serum markers of immunoglobulin and T-cell subsets, measured at baseline and pre-determined times post-treatment.ResultsA reduction of pathological symptoms and signs was shown following treatment. The BBS scores, IgG, IgA, total T cells and CD3+CD4 T cells all improved significantly compared to pre-treatment values (P < 0.01~0.001). There were no adverse events.ConclusionThe combination of CBMC infusion and rehabilitation training may be a safe and effective treatment for ataxia, which dramatically improves patients' functional symptoms. These data support expanded double blind, placebo-controlled studies for these treatment modalities.

【 授权许可】

Unknown   
© Yang et al; licensee BioMed Central Ltd. 2011. This article is published under license to BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.