Abstract
BackgroundAutism 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.Methods37 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.ResultsThere 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).ConclusionsTransplantation 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 registrationClinicalTrials.gov: NCT01343511, Title “Safety and Efficacy of Stem Cell Therapy in Patients with Autism”.
Highlights
Autism spectrum disorders (ASD) are heterogeneous neurodevelopmental disorders [1]
Enrolled subjects were divided into three groups: the Control group, which received only rehabilitation therapy; the cord blood mononuclear cells (CBMNCs) group, which received CBMNC transplantation and rehabilitation therapy; and the Combination group, which received combined CBMNC and umbilical cord-derived mesenchymal stem cells (UCMSCs) transplantation and rehabilitation therapy
Immunological reactions or other serious adverse events were observed at the time of injection or during the whole follow-up period in the two groups receiving stem cell transplantation
Summary
With the support from accumulated clinical experience of our exploratory stem cell treatments [11,12,16], the subjects recruited in the CBMNC and Combination groups received four cell transplantations at an interval of 5 ~ 7 days. Safety and efficacy measure Treatment safety was evaluated with: documentation of physical examination, vital signs and adverse events; complete blood count, liver and renal function, serum glucose, lipid profile, immunology testing (including immunoglobulin (Ig) A/G/M, complement C3/C4 and T-cell subsets) at baseline (pre-treatment) and 4 (4w), 8 (8w), and 24 (24w) weeks after the first cell transplantation. Subjects were assessed with CARS, CGI scale, and Aberrant Behavior Checklist (ABC) at baseline and 4w, 8w, 16w and 24w after the first cell transplantation for efficacy. A p-value
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