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

• Published in: Journal of translational medicine Vol. 11; no. 1; p. 196
• Main Authors: Lv, Yong-Tao, Zhang, Yun, Liu, Min, Qiuwaxi, Jia-na-ti, Ashwood, Paul, Cho, Sungho Charles, Huan, Ying, Ge, Ru-Cun, Chen, Xing-Wang, Wang, Zhao-Jing, Kim, Byung-Jo, Hu, Xiang
• Format: Journal Article
• Language: English
• Published: England BioMed Central Ltd 27.08.2013; BioMed Central
• Subjects: Autism; Autistic Disorder - blood; Autistic Disorder - therapy; B cells; Behavior; Care and treatment; Cell culture; Child; Child, Preschool; Children & youth; Complications and side effects; Cord Blood Stem Cell Transplantation; Engineering research; Female; Hepatitis; Hospitals; Humans; Leukocytes, Mononuclear - transplantation; Male; Medical research; Medicine, Experimental; Mesenchymal Stem Cell Transplantation - adverse effects; Rehabilitation; Risk factors; Stem cells; Transplantation; Treatment Outcome; Umbilical Cord - cytology; China
• ISSN: 1479-5876; 1479-5876
• DOI: 10.1186/1479-5876-11-196

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. 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. 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). 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. ClinicalTrials.gov: NCT01343511, Title "Safety and Efficacy of Stem Cell Therapy in Patients with Autism".