In a newly planned trial, recently approved by the FDA, researchers will examine whether stem cells obtained through umbilical cord blood at birth may be an effective treatment for children with autism.The 30 children that will participate in the placebo controlled trial, ages 2-7, had umbilical cord blood banked at birth, as part of the Cord Blood Registry, a highly organized and well known stem cell bank. The goal of the trial will be to evaluate whether stem cell therapy has any effect on behavior and language difficulties commonly experienced by children with autism. There is other ongoing research evaluating stem cells for autism in Europe; however, this is the first trial to use a child’s own umbilical cord as a source for stem cells.

The rationale for pursuing stem cell therapy in autism is based on encouraging results of unpublished data examining stem cell therapy in children with cerebral palsy, a group of disorders that affect not only movement, but learning, behavior and cognition. The potential dysfunction of the immune system as a driver for abnormalities in the nervous system has researchers interested in a possible link.

Throughout this study, inflammatory markers from each child’s blood will be checked to assess any changes in chemistry due to the therapy, along with EEGs to measure electrical activity. Parental and physician observations of behavior, especially socialization and irritability will be closely evaluated according to investigators involved with this study.

Based on data from the CDC, 1 in 88 children are diagnosed with autism,with five times as many boys as girls afflicted. The cause is unknown -presumed to be multifactorial- with signs noted in early childhood related to abnormalities in behavior, language, and social development.

Environmental factors along with genetics and unknown immunologic factors are believed to play a role in the development of autism. Autism is actually more accurately described as a spectrum disorder, (Autism Spectrum Disorder) with some children demonstrating more severe symptoms than others. While some theories suggest that connections between neurons or nerve cells may be to blame for autism, it is unclear if stem cell therapy may help to restore such connections.

While stem cells have been used to treat cancer, Parkinson’s disease, and certain immunologic disorders, the role and relationship of the immune system and the nervous system in the development of autism is something that has been an area of recent interest. Research in autism has indicated abnormalities in spinal fluid and chemical signaling in the brain- anecdotal reports of children undergoing stem cell therapy have indicated some potentially encouraging results.

It should be cautioned that research examining the role of the immune system in development of autism is preliminary, and it is unclear if there is any immunologic factor that may be involved. The goal of this research is to establish whether there may be any role at all for stem cell therapy in autism spectrum disorder. This will likely take several years before we have  preliminary results to guide us.

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