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World J Stem Cells. Apr 26, 2014; 6(2): 173-178
Published online Apr 26, 2014. doi: 10.4252/wjsc.v6.i2.173
Mesenchymal stem cells in treating autism: Novel insights
Dario Siniscalco, James Jeffrey Bradstreet, Nataliia Sych, Nicola Antonucci
Dario Siniscalco, Department of Experimental Medicine, Second University of Naples, 16-80138 Napoli, Italy
Dario Siniscalco, Centre for Autism, La Forza del Silenzio, 81036 Caserta, Italy
Dario Siniscalco, Cancellautismo, 50132 Florence, Italy
James Jeffrey Bradstreet, Brain Treatment Center, Buford, GA 30518, United States
Nataliia Sych, Clinical Department, Cell Therapy Center EmCell, 04073 Kiev, Ukraine
Nicola Antonucci, Biomedical Centre for Autism Research and Treatment, 70126 Bari, Italy
Author contributions: Siniscalco D designed the paper; Siniscalco D and Bradstreet JJ wrote the paper; Sych N contributed to the paragraph on MSCs; Antonucci N contributed to the paragraph on autism; Bradstreet JJ edited the English language.
Correspondence to: Dario Siniscalco, ChemD, PhD, Department of Experimental Medicine, Second University of Naples, Via S Maria di Costantinopoli, 16-80138 Napoli, Italy. dariosin@uab.edu
Telephone: +39-81-5665880 Fax: +39-81-5667503
Received: November 11, 2013
Revised: December 19, 2013
Accepted: March 17, 2014
Published online: April 26, 2014
Abstract

Autism spectrum disorders (ASDs) are complex neurodevelopmental disorders characterized by dysfunctions in social interactions, abnormal to absent verbal communication, restricted interests, and repetitive stereotypic verbal and non-verbal behaviors, influencing the ability to relate to and communicate. The core symptoms of ASDs concern the cognitive, emotional, and neurobehavioural domains. The prevalence of autism appears to be increasing at an alarming rate, yet there is a lack of effective and definitive pharmacological options. This has created an increased sense of urgency, and the need to identify novel therapies. Given the growing awareness of immune dysregulation in a significant portion of the autistic population, cell therapies have been proposed and applied to ASDs. In particular, mesenchymal stem cells (MSCs) possess the immunological properties which make them promising candidates in regenerative medicine. MSC therapy may be applicable to several diseases associated with inflammation and tissue damage, where subsequent regeneration and repair is necessary. MSCs could exert a positive effect in ASDs through the following mechanisms: stimulation of repair in the damaged tissue, e.g., inflammatory bowel disease; synthesizing and releasing anti-inflammatory cytokines and survival-promoting growth factors; integrating into existing neural and synaptic network, and restoring plasticity. The paracrine mechanisms of MSCs show interesting potential in ASD treatment. Promising and impressive results have been reported from the few clinical studies published to date, although the exact mechanisms of action of MSCs in ASDs to restore functions are still largely unknown. The potential role of MSCs in mediating ASD recovery is discussed in light of the newest findings from recent clinical studies.

Keywords: Autism spectrum disorders, Autism treatment, Cell therapy, Mesenchymal stem cells

Core tip: Autism spectrum disorders are still untreatable pathologies. Mesenchymal stem cells possess the immunological properties which make them promising candidates as a novel therapeutic option.