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. 1. Introduction Autism spectrum disorders (ASD) are a group of heterogeneous neurodevelopmental disorders characterized by deficits in verbal and nonverbal communication, social interaction, and presence of stereotypical repetitive behavior. The genetic, environmental, and immunological factors have been attributed as underlying causes, though its exact etiology is unknown. The incidence of autism has increased to a great extent, which may be due to increased awareness leading to an early and accurate diagnosis or due to perinatal complications, genetic factors, environmental factors, and lifestyle changes. Presently, the worldwide incidence is 12 per 1000 children [1]. Despite its increasing rate, currently autism remains untreatable. The available options of behavioral therapy and pharmacological and supportive nutritional treatments are only palliative. Medical therapy is directed towards the neuropsychiatric disorders associated with ASDs. Commonly prescribed medicines are selective serotonin reuptake inhibitors, antipsychotics, mood stabilizers, and psychostimulants. Methylphenidate may be used to treat attention
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