Does the key to improving the lives of children with autism lie in their own umbilical cord blood? The answer may be yes, according to a new study by Duke researchers.

Scientists conducted an investigation to determine whether infusing an autistic child with blood from the umbilical cord would reduce symptoms of autism. The research has since received national media attention following its release in April. The small-scale experiment involved 25 child participants aged four to six. After giving them a battery of tests, researchers determined that certain symptoms of their autism decreased following the treatment, wrote Geraldine Dawson, director of the Duke Center for Autism and Brain Development, in an email.

“We measured the children’s social and communication abilities using various tests and parent questionnaires,” Dawson said. “We found that the infusion was safe and many children showed improvements in their social and language skills.”

Dr. Joanne Kurtzberg, professor in the department of pediatrics, added that the approach taken by this study was “entirely new.”

Dawson explained that in the investigation, the children were infused with blood from their own umbilical cord that contained stem cells. After a year had elapsed, the children were then called in for evaluation.

However, this pilot study is only preliminary, Dawson added, as it was not enough to guarantee that the umbilical cord blood treatment was the determining factor leading to the improvement in symptoms.

“Because there was no comparison group in this study, we don’t yet know whether the improvements we observed were because of the cord blood,” she wrote. 

In other words, no control group of children without cord blood infusions was included in the investigation. Kurtzberg also explained that in this "Phase I" study, both the evaluators and families knew that their child had been given umbilical cord blood as the treatment, which could have biased the results. 

“As much as we’re hopeful that this is helping, we don’t want to prematurely make claims that haven’t been confirmed,” she said.

Dawson and Kurtzberg said that they have already begun working on a second phase of the study, which should be complete in less than two years. The second phase will include a blind control group—children will receive a placebo infusion. In addition, the second phase, which will be randomized and larger in scale, testing 165 children with autism, Kurtzberg added.

She noted that children will be split into two groups—one group will receive a treatment of umbilical cord blood for six months, and the other will be given a placebo. After six months, the groups will switch and receive the opposite treatment. Kurtzberg explained that this particular setup will potentially give the researchers a better idea whether the cord blood treatment is truly effective.

The investigators have also developed a hypothesis detailing how umbilical cord blood might be acting to improve autistic symptoms. Kurtzberg explained it as a possible “suppression of microglial activation,” referring to a class of immune cells within the nervous system.

“There is a remodeling of certain abnormal brain connections [by the microglia], which then results in decreasing symptoms of autism,” she said.

Although the initial phase of the study appears to have been successful, Dawson and Kurtzberg cautioned against being too optimistic about the isolated trial—definitive results will have to wait for now.

“At the conclusion of [the Phase II] study, we’ll be able to answer the question about whether cells are effective,” Kurtzberg said.