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Mesenchymal stem cells in autism spectrum and neurodevelopmental disorders: pitfalls and potential promises

Abstract Objectives. In this conceptual review, the authors discuss the promises and pitfalls in the use of mesenchymal stem cells as a potential experimental therapeutic for autism spectrum and other neurodevelopmental disorders. Methods. The relevant literature in autism spectrum disorders and other neurodevelopmental disorders regarding immune dysregulation and neuroinflammation and relevant therapeutics with mesenchymal stem cell infusion is reviewed. The relevant literature pertaining to mesenchymal stem cells and their clinical applications is also reviewed. Results. It is proposed that immune dysregulation and neuroinflammation play a role in the aetiology of autism spectrum disorders. Mesenchymal stem cells have been shown to have immune-modulating capabilities and are neuroprotective. There are three international studies that have utilized mesenchymal stem cell infusions as a treatment for children with autism spectrum disorders, all of which demonstrated improvement in autism rating scale scores, although each study has limitations which are described. Conclusions. Mesenchymal stem cell transplantation for the treatment of autism spectrum disorders is a novel approach that deserves further investigation, however substantial methodological and theoretical challenges and pitfalls remain before this can be considered a viable therapeutic option.