Autism spectrum disorder (ASD) is a group of conditions in children that involve affected behaviour, language and communication. The intellect may be extremely poor or the other extreme-highly intelligent. According to the World Health Organization, ASD is seen in one in 160 children. The main aspects of this disease include a “repetitive” behaviour or activity or interest shown by the affected child along with highly challenged social communication. Other aspects of the disease include aggression, depression, irritability, compulsive behaviour, moodiness, sleep issues and gastrointestinal issues. A major challenge with the disease is also the social stigma associated.
There are many approaches to treat the disease: psychotropic drugs, social training and behavior therapy. However, the side effects associated with the drugs used such as mood stabilizers, antipsychotics or anticonvulsants are many. There are alternative treatments such as music therapy, hyperbaric oxygen treatment, and nutritional supplements that all require further studies in terms of efficacy.
As ASD is a disease where the nervous system is affected and the formation of neurons is limited, the use of stem cells is promising. The ability of stem cells to regenerate damaged cells and secrete factors to repair damaged tissue has been reported in many animal models, preclinical and clinical trials. A few examples in the case of autism are discussed below:
A “proof of concept” study published in Stem cells international in 2013 by Sharma and team reported encouraging results when autologous stem cells were administered to 32 patients with autism. The process involved the transplantation of each patient’s bone marrow stem cells followed by occupational therapy, speech therapy and diet control. Follow up over 26 months showed that total ISAA (Indian Scale for Assessment of Autism) scores improved in 91% of the patients while the Clinical Global Impression (CGI-I) scores that reflect how a disease is progressing was lowered in 62% of the patients showing lowered disease severity. Close to 94% of patients showed no adverse effects on the CGI-III scale. While 91% of the patients improved in social relationships, 78% showed an improvement in speech and communication. An interesting finding was the PET-CT (Positron Emission Tomography-Computed Tomography) scan that showed an improvement in balanced metabolism in the patients before and after 6 months of transplantation.
A phase I, open‐label trial was published by Dawson and team in 2017 in Stem cells translational medicine. A single dose of autologous umbilical cord blood (source of stem cells) was given to 25 children with ASD (age between 2 to 6). A range of assessment tests was conducted before the infusion and at 6-months and 12-months after the treatment. The treatment was termed as safe as no adverse side effects were seen in the children. As reported by the parents, the children improved in autism symptoms and communication skills. There was an improvement in clinical scores that record the ability of children to focus on an external signal showing improved attention.
Thus, the benefits offered by preclinical and clinical trials to improve the quality of life in autistic kids are promising so that children can live a free and joyful life without a label of “autistic” or “special”.
Siniscalco, D., Kannan, S., Semprún-Hernández, N., Eshraghi, A. A., Brigida, A. L., & Antonucci, N. (2018). Stem cell therapy in autism: recent insights. Stem cells and cloning: advances and applications, 11, 55–67. doi:10.2147/SCCAA.S155410.
Sharma A, Gokulchandran N, Sane H, et al. Autologous bone marrow mononuclear cell therapy for autism: an open label proof of concept study. Stem Cells Int. 2013;2013:623875.
Dawson, G., Sun, J. M., Davlantis, K. S., Murias, M., Franz, L., Troy, J., … Kurtzberg, J. (2017). Autologous Cord Blood Infusions Are Safe and Feasible in Young Children with Autism Spectrum Disorder: Results of a Single-Center Phase I Open-Label Trial. Stem cells translational medicine, 6(5), 1332–1339. doi:10.1002/sctm.16-0474.