title: 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.
  
  
  CONCLUSIONS
  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.affiliation: Shenzhen Beike Cell Engineering Research Institution, Shenzhen, China.
authors.forename: Wan-Zhang
authors.initials: WZ
authors.lastname: Yang
authors.forename: Yun
authors.initials: Y
authors.lastname: Zhang
authors.forename: Fang
authors.initials: F
authors.lastname: Wu
authors.forename: Min
authors.initials: M
authors.lastname: Zhang
authors.forename: S C
authors.initials: SC
authors.lastname: Cho
authors.forename: Chun-Zhen
authors.initials: CZ
authors.lastname: Li
authors.forename: Shao-Hui
authors.initials: SH
authors.lastname: Li
authors.forename: Guo-Jian
authors.initials: GJ
authors.lastname: Shu
authors.forename: You-Xiang
authors.initials: YX
authors.lastname: Sheng
authors.forename: Ning
authors.initials: N
authors.lastname: Zhao
authors.forename: Ying
authors.initials: Y
authors.lastname: Tang
authors.forename: Shu
authors.initials: S
authors.lastname: Jiang
authors.forename: Shan
authors.initials: S
authors.lastname: Jiang
authors.forename: Matthew
authors.initials: M
authors.lastname: Gandjian
authors.forename: Thomas E
authors.initials: TE
authors.lastname: Ichim
authors.forename: Xiang
authors.initials: X
authors.lastname: Hu
mesh_terms: D000328:Adult; D000368:Aged; D017690:Cell Transplantation; D005260:Female; D005312:Fetal Blood / Q000166:cytology*; D006801:Humans; D007136:Immunoglobulins / Q000276:immunology; D007963:Leukocytes, Mononuclear / Q000637:transplantation*; D008297:Male; D008875:Middle Aged; D013132:Spinocerebellar Degenerations / Q000276:immunology / Q000628:therapy*; D016176:T-Lymphocyte Subsets / Q000276:immunology; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D007136:Immunoglobulins