A paper just out from the Shenzen Beike Cell Engineering Research Institute in Guangdong, China presents the first results of a proof-of-concept, safety trial of stem cell (SC) injections in 37 autistic children. The justification for this intervention is primarily that animal studies of various inflammatory conditions can be ameliorated by SC therapies, and there is good evidence of immune dysregulation in autism (as well as Rett, OCD, PANDAS and Tourette). It is not suggested that the SCs will populate the brain and participate in the circuitry.
Two types of cells were used: human cord blood mononuclear cells and umbilical cord-derived mesenchymal SCs. The first group of patients initially received the cord blood cells by intravenous infusion and then, at 5-7 day intervals, they received 3 more injections by the intrathecal (spinal) route. They also received behavioral therapy. The second group of patients two cord blood intravenous and intrathecal injections each, followed by two umbilical SC intrathecal injections. This group is termed the combination group. They also received behavioral therapy. The third group of patients received only behavioral therapy.
The drawbacks in this study are that it was non-randomized, open-label (everyone knew who received which treatment), conducted at a single center, primarily by people who were employees at the company that produces these SCs (Beike Cell Engineering Research Inst). Thus, we can’t take the autism-related results too seriously, but we can probably rely on the results related to safety – they report that none of the 23 subjects that received multiple injections of these (foreign) cells displayed signs of toxicity or changes in blood biochemistry over the 6 months of the study. This bodes well for future studies of this kind.
Regarding the behavioral data, they did not use the gold standard ADOS method of scoring autism symptoms. Rather, they used the descriptive CARS, ABC Chinese Version, and the CGI (global) scales. In all of these 3 measures, the combination treatment group showed improvement over the control group. The single, cord blood treatment group was somewhat intermediate in efficacy. The large grain of salt is, as mentioned above, the folks doing the evaluations were aware of the treatment used on each patient. Plus, the number of subjects in each group was small (but not minuscule!). No doubt, these results will push this group and other SC labs and companies to move forward with further testing, hopefully double-blind!