Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism

Yong Tao Lv, Yun Zhang, Min Liu, Jia na ti Qiuwaxi, Paul Ashwood, Sungho Charles Cho, Ying Huan, Ru Cun Ge, Xing Wang Chen, Zhao Jing Wang, Byung Jo Kim, Xiang Hu

Research output: Contribution to journalArticle

70 Scopus citations

Abstract

Background: Autism is a pervasive neurodevelopmental disorder. At present there are no defined mechanisms of pathogenesis and therapy is mostly limited to behavioral interventions. Stem cell transplantation may offer a unique treatment strategy for autism due to immune and neural dysregulation observed in this disease. This non-randomized, open-label, single center phase I/II trial investigated the safety and efficacy of combined transplantation of human cord blood mononuclear cells (CBMNCs) and umbilical cord-derived mesenchymal stem cells (UCMSCs) in treating children with autism.Methods: 37 subjects diagnosed with autism were enrolled into this study and divided into three groups: CBMNC group (14 subjects, received CBMNC transplantation and rehabilitation therapy), Combination group (9 subjects, received both CBMNC and UCMSC transplantation and rehabilitation therapy), and Control group (14 subjects, received only rehabilitation therapy). Transplantations included four stem cell infusions through intravenous and intrathecal injections once a week. Treatment safety was evaluated with laboratory examinations and clinical assessment of adverse effects. The Childhood Autism Rating Scale (CARS), Clinical Global Impression (CGI) scale and Aberrant Behavior Checklist (ABC) were adopted to assess the therapeutic efficacy at baseline (pre-treatment) and following treatment.Results: There were no significant safety issues related to the treatment and no observed severe adverse effects. Statistically significant differences were shown on CARS, ABC scores and CGI evaluation in the two treatment groups compared to the control at 24 weeks post-treatment (p < 0.05).Conclusions: Transplantation of CBMNCs demonstrated efficacy compared to the control group; however, the combination of CBMNCs and UCMSCs showed larger therapeutic effects than the CBMNC transplantation alone. There were no safety issues noted during infusion and the whole monitoring period.Trial registration: ClinicalTrials.gov: NCT01343511, Title " Safety and Efficacy of Stem Cell Therapy in Patients with Autism" .

Original languageEnglish (US)
Article number196
JournalJournal of Translational Medicine
Volume11
Issue number1
DOIs
StatePublished - Aug 27 2013

Keywords

  • Autism
  • Cell transplantation
  • Cord blood mononuclear cell
  • Umbilical cord-derived mesenchymal stem cell

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)
  • Medicine(all)

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    Lv, Y. T., Zhang, Y., Liu, M., Qiuwaxi, J. N. T., Ashwood, P., Cho, S. C., Huan, Y., Ge, R. C., Chen, X. W., Wang, Z. J., Kim, B. J., & Hu, X. (2013). Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism. Journal of Translational Medicine, 11(1), [196]. https://doi.org/10.1186/1479-5876-11-196