Human umbilical cord blood-derived mononuclear cell transplantation: case series of 30 subjects with hereditary ataxia.

Abstract

BACKGROUND:

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.

METHODS:

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.

RESULTS:

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.

CONCLUSION:

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.

• Authors: Wan-Zhang Yang, Yun Zhang, Fang Wu, Min Zhang, S C Cho, Chun-Zhen Li, Shao-Hui Li, Guo-Jian Shu, You-Xiang Sheng, Ning Zhao, Ying Tang, Shu Jiang, Shan Jiang, Matthew Gandjian, Thomas E Ichim, Xiang Hu
• Journal: Journal of translational medicine (J Transl Med) Vol. 9 Pg. 65 (May 16 2011) ISSN: 1479-5876 [Electronic] England
• PMID: 21575250 (Publication Type: Clinical Trial, Journal Article)

Chemical References

• Immunoglobulins

Topics

• Adult
• Aged
• Cell Transplantation
• Female
• Fetal Blood (cytology)
• Humans
• Immunoglobulins (immunology)
• Leukocytes, Mononuclear (transplantation)
• Male
• Middle Aged
• Spinocerebellar Degenerations (immunology, therapy)
• T-Lymphocyte Subsets (immunology)
• Treatment Outcome
• Young Adult