Duke University recently conducted a first-of-its-kind study that sought to determine whether a transfusion of umbilical cord blood containing rare stem cells could help treat autism. The study included 25 children diagnosed with autism and the results were optimistic; two-thirds of the children, ranging from ages 2 to 6, reported improvements.
The safety trial began over a year and a half ago, which found that umbilical cord blood was not only safe, but 70% of the 25 participants had behavioral advances as described by their parents and tracked by the Duke researchers. The children traveled to Duke three times over the course of a year, and each visit included a series of evaluations including MRIs, EEGs, and autism assessments. The children received the umbilical cord blood infusion on the first visit. Each child received 1 to 2 billion cells, given through an IV in their arms or legs. The subsequent visits occurred six months and one year after the infusion.
Dr. Joanne Kurtzberg and Dr. Geraldine Dawson spearheaded the study. Kurtzberg, head of the Robertson Clinical and Translational Cell Therapy Program, and Dawson, director of the Duke Center for Autism and Brain Development, each saw the intense need for advancements in autism treatments.
Kurtzberg has been studying for years the effects of umbilical cord blood treatments on several disorders, including inherited metabolic disorders and cerebral palsy. In children with cerebral palsy who also exhibited autistic tendencies, their symptoms of autism improved with the treatments. The improvements among children with cerebral palsy gave Kurtzberg the idea to test cord blood specifically for autism.
While both doctors were encouraged by the results of the initial study, they cautioned that plenty of additional research is necessary in order to definitively state whether or not umbilical cord blood treatments improve autism symptoms. Dawson noted the study did not have a comparison group, which she stated is “very important in establishing whether a treatment is actually effective” (Drash & Sanjay, 2017).
Next up is a definitive trial — a double-blind, placebo-controlled trial involving 165 children with autism, ranging in age from 2 to 8. During this secondary phase, the children will receive either their initial umbilical cord blood infusion during their first visit to Duke, or a placebo. A battery of tests, including brain monitoring, will follow.
After six months, the children will receive a second infusion with whatever preparation they did not receive the first time. Known as a crossover trial, subjects receive both a treatment and the placebo, but in a different order. The order of the infusion is not known and researchers say it would be practically impossible to find participants if their parents knew that their children might not receive an infusion.
If this second phase of research shows that umbilical cord blood is effective in improving autism symptoms, primarily social behaviors, Dawson said the finding would be “game-changing”. Again, before any optimism can be confirmed, Kurtzberg reiterated, “we don’t want to mislead people and claim it’s working before we have definitive proof” (Drash & Sanjay, 2017).
And while such definitive proof is still up-in-the-air, these studies should, at the very least, spark hope for the future of autism research. When dealing with research concerning any disorder that lacks a known cause or cure, it is vital to remember that there may be several dead-ends before progress is actually made. Although the Duke study is extremely preliminary, it demonstrates the constant push for more knowledge surrounding autism spectrum disorder. It is this type of research that one day may uncover a breakthrough that could alter the lives of millions of people affected by autism.
Drash, Wayne, and Sanjay Gupta, Dr. “Stem cells offer hope for autism.” CNN. Cable News Network, 05 Apr. 2017. Web. 09 Apr. 2017.