FDA Approves Adult Stem Cell Trial for People With Autism

Bioethics   |   Rebecca Taylor   |   Aug 27, 2012   |   12:00PM   |   Washington, DC

Autism is as mysterious as it is devastating. Does it have a genetic cause? Is it environmental? Or is there some complex combination of both that derails normal development of communication and social skills.

The treatments for autism are as varied as the patients, since it is often the patient’s needs that dictate which treatments are most effective. Some parents have brought their children overseas for stem cell treatments that claim to improve the symptoms of autism.

The FDA has approved a ground-breaking adult stem cell trial in the United States to see if stem cells can in fact help those with autism. Unlike treatments in other countries, this trial will be using cord blood stem cells saved from a child’s own umbilical cord.

From Scientific American:

A clinical trial being conducted by the Sutter Neuroscience Institute in Sacramento, California to address this situation began recruiting participants today for a highly experimental stem cell therapy for autism. The institute plans to find 30 autistic children between ages 2 and 7 with cord blood banked at the privately-run Cord Blood Registry, located about 100 miles west of the institute….

A major difference between the Sutter trial and those in China is that his will use the child’s own stem cells, rather than those from a donor. Chez hypothesizes that one way autologous stem cell infusion might work is by reducing inflammation within the body’s immune system. This would answer previous research that suggests that autism may be an autoimmune disease. “One of our exploratory goals will be to look at inflammatory markers in cells,” he says.



The study’s primary goal, however, will be assessing changes in patients’ speaking and understanding of vocabulary. For each individual, researchers will create a baseline benchmark that establishes current skill levels. The group will be evenly divided, with one initially receiving an infusion of their own, unmodified cord blood stem cells and the other a placebo treatment of saline injection. Six months later, all of the children will be tested on their ability to comprehend and form words. The groups will then be switched. In the course of the 13-month-long study, both groups will receive only one stem cell therapy infusion.

It is much too early to suggest that an autologous stem cell transplant will be a cure for autism. Hopefully, the participants in this study will see some improvement. That would spark more research into a stem cell treatment. It will also shed some light on whether it is efficacious for parents to be engaging in stem cell tourism on behalf of their children.