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Stem Cells International
Volume 2013, Article ID 623875, 13 pages
http://dx.doi.org/10.1155/2013/623875
Clinical Study

Autologous Bone Marrow Mononuclear Cell Therapy for Autism: An Open Label Proof of Concept Study

1Department of Medical Services and Clinical Research, NeuroGen Brain and Spine Institute, Surana Sethia Hospital and Research Centre, Sion Trombay Road, Chembur, Mumbai 400071, India
2Department of Research & Development, NeuroGen Brain and Spine Institute, Surana Sethia Hospital and Research Centre, Sion Trombay Road, Chembur, Mumbai 400071, India
3Department of NeuroRehabilitation, NeuroGen Brain and Spine Institute, Surana Sethia Hospital and Research Centre, Sion-Trombay Road, Chembur, Mumbai 400071, India

Received 4 May 2013; Revised 24 June 2013; Accepted 7 July 2013

Academic Editor: Chen Lin

Copyright © 2013 Alok Sharma et al. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

Abstract

Cellular therapy is an emerging therapeutic modality with a great potential for the treatment of autism. Recent findings show that the major underlying pathogenetic mechanisms of autism are hypoperfusion and immune alterations in the brain. So conceptually, cellular therapy which facilitates counteractive processes of improving perfusion by angiogenesis and balancing inflammation by immune regulation would exhibit beneficial clinical effects in patients with autism. This is an open label proof of concept study of autologous bone marrow mononuclear cells (BMMNCs) intrathecal transplantation in 32 patients with autism followed by multidisciplinary therapies. All patients were followed up for 26 months (mean 12.7). Outcome measures used were ISAA, CGI, and FIM/Wee-FIM scales. Positron Emission Tomography-Computed Tomography (PET-CT) scan recorded objective changes. Out of 32 patients, a total of 29 (91%) patients improved on total ISAA scores and 20 patients (62%) showed decreased severity on CGI-I. The difference between pre- and postscores was statistically significant ( ) on Wilcoxon matched-pairs signed rank test. On CGI-II 96% of patients showed global improvement. The efficacy was measured on CGI-III efficacy index. Few adverse events including seizures in three patients were controlled with medications. The encouraging results of this leading clinical study provide future directions for application of cellular therapy in autism.