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 (P < 0.001) 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.