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

Yong-Tao Lv1, Yun Zhang2, Min Liu1, Jia-na-ti Qiuwaxi2, Paul Ashwood3, Sungho Charles Cho4, Ying Huan1, Ru-Cun Ge1, Xing-Wang Chen1, Zhao-Jing Wang2, Byung-Jo Kim5 and Xiang Hu2*

Author Affiliations

1 Shandong Jiaotong Hospital, Jinan, Shandong, China

2 Shenzhen Beike Cell Engineering Research Institute, 2/F, Yuanxing Technology Building, #1 Songpingshan Street, Nanshan District, Shenzhen, Guangdong 518057, China

3 Department of Medical Microbiology & Immunology, University of California Davis, Davis, CA, USA

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

5 Department of Neurology, Korea University Anam Medical Center, Seoul, Korea

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Journal of Translational Medicine 2013, 11:196  doi:10.1186/1479-5876-11-196

Published: 27 August 2013

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”.

Keywords:
Autism; Cord blood mononuclear cell; Umbilical cord-derived mesenchymal stem cell; Cell transplantation