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

Wan-Zhang Yang1, Yun Zhang2, Fang Wu1, Min Zhang1, SC Cho3, Chun-Zhen Li1, Shao-Hui Li1, Guo-Jian Shu1, You-Xiang Sheng1, Ning Zhao1, Ying Tang1, Shu Jiang2, Shan Jiang2, Matthew Gandjian4, Thomas E Ichim4* and Xiang Hu2*

Author Affiliations

1 Department of Rehabilitation Medicine, Nanshan Affiliated Hospital of Guangdong Medical College, Shenzhen, China

2 Shenzhen Beike Cell Engineering Research Institution, Shenzhen, China

3 Department of Neurology and Neurosurgery, Stanford University, Stanford, CA, USA

4 Medistem Inc, San Diego, CA, USA

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Journal of Translational Medicine 2011, 9:65  doi:10.1186/1479-5876-9-65

Published: 16 May 2011



The 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.


30 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.


A 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.


The 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.