title: Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism.
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.
  
  
  BACKGROUND
  ClinicalTrials.gov: NCT01343511, Title "Safety and Efficacy of Stem Cell Therapy in Patients with Autism".
authors: Shenzhen Beike Cell Engineering Research Institute, 2/F, Yuanxing Technology Building, #1 Songpingshan Street, Nanshan District, Shenzhen, Guangdong 518057, China. publication@beikebiotech.com.
authors: Yong-Tao
authors: YT
authors: Lv
authors: Yun
authors: Y
authors: Zhang
authors: Min
authors: M
authors: Liu
authors: Jia-na-ti
authors: JN
authors: Qiuwaxi
authors: Paul
authors: P
authors: Ashwood
authors: Sungho Charles
authors: SC
authors: Cho
authors: Ying
authors: Y
authors: Huan
authors: Ru-Cun
authors: RC
authors: Ge
authors: Xing-Wang
authors: XW
authors: Chen
authors: Zhao-Jing
authors: ZJ
authors: Wang
authors: Byung-Jo
authors: BJ
authors: Kim
authors: Xiang
authors: X
authors: Hu
mesh_terms: D001321:Autistic Disorder / Q000097:blood / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation; D005260:Female; D006801:Humans; D007963:Leukocytes, Mononuclear / Q000637:transplantation*; D008297:Male; D045164:Mesenchymal Stem Cell Transplantation / Q000009:adverse effects; D016896:Treatment Outcome; D014470:Umbilical Cord / Q000166:cytology*
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