Stem cell transplantation may be a safe and effective treatment for autism, according to a new study. Researchers tested two types of stem cell therapies from human umbilical cord stem cells -- one using mononuclear cells called "CBMNCs" and one using mesenchymal cells called "UCMSCs." The study included 37 children with autism.
The researchers said they conducted the study because "stem cell transplantation may offer a unique treatment strategy for autism" because of the disorder's inherent "dysregulation" of the immune system and brain.
The children were divided into three groups:
- Fourteen received CBMNC transplantation as well as standard rehabilitation therapy for their autism.
- Nine received both CBMNC and UCMSC transplantation as well as standard rehabilitation therapy.
- Fourteen received only rehabilitation therapy (control group).
Participants in the transplant groups received a total of four stem cell infusions through intravenous and intrathecal injections once weekly. (Intrathecal injections are delivered into the fluid-filled space between the layers of tissue that cover the brain and spinal cord.) They were evaluated for adverse effects through examination of their blood in a laboratory and by clinical assessments. The researchers reported no "significant safety issues related to treatment."
Researchers used standard autism testing to determine if there was improvement in participants' autism symptoms. Tests used were the Childhood Autism Rating Scale (CARS), Clinical Global Impression (CGI) scale, and the Aberrant Behavior Checklist (ABC). The children were tested before and after treatment.
"Statistically significant differences were shown on CARS, ABC scores and CGI evaluation in the two treatment groups compared to the control [group] at 24 weeks post-treatment," the researchers wrote.
They concluded that transplantation of CBMNCs was effective when compared to the control group. However, the combination treatment of CBMNCs and UCMSCs showed "larger therapeutic effects" than the CBMNC transplantation alone.
(Four more National Institutes of Health studies on stem cell transplants in autism are planned or in progress. For more information, go to ClinicalTrials.gov.)