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Early Stem Cell Study Offers Hope for Children With Autism

Early Stem Cell Study Offers Hope for Children With Autism

Umbilical cord blood improved autism symptoms in small, preliminary study.

Autism, a developmental disorder that affects about one out of 68 U.S. children, typically involves communication and learning difficulties. It’s a spectrum disorder, meaning it can come with a wide range of possible symptoms and sometimes, special talents. For many kids and families, autism presents challenges at school and home that interfere with daily life.

But a small, preliminary study from Duke University may offer some hope for these families. Researchers gave 25 children with autism infusions of their own umbilical cord blood. They wanted to see if the stem cells within the blood would have any effect on the kids’ symptoms, and to find out if the infusions were safe.

Over two-thirds of the children did experience improvements. Symptom changes were reported by parents and observed by researchers during physical, behavioral and reasoning tests.

Since the treatment didn’t cause any major side effects, it will move on to the next phase of research; a larger study to be overseen by the Food and Drug Administration (FDA).

Room for optimism, but still a long way to go
Despite these positive results, researchers caution that it’s far too soon to get excited or jump to conclusions about what this study could mean in the future.

“We don’t want to mislead people and claim it’s working before we have definitive proof,” Joanne Kurtzberg, MD, one of the lead researchers and head of the Robertson Clinical and Translational Cell Therapy Program at Duke University in Durham, North Carolina, told CNN.

The study, published in the journal Stem Cells Translational Medicine, is just one small step towards understanding the potential of this treatment. There are limitations to the study because:

  • It only included 25 participants, so it’s impossible to predict whether the same results will be seen among the population in general.
  • It was an open-label study, so parents and doctors knew that every child was receiving the actual treatment, and not just a placebo. That knowledge could have affected their assessments of the children’s behavior.
  • It took place over a full year, but autism symptoms may improve somewhat around preschool age, so the results could have come from natural growth and development.
  • Scientists still don’t know exactly why the treatment had the effects it did. One possible explanation is that stems cells reduce brain inflammation, which may play a role in autism.

Despite these limitations, the stories of family members have sparked optimism. CNN reports that one child’s learning has accelerated to the point that she’s now able to attend a mainstream, rather than a specialized school. She also has far fewer tantrums and welcomes hugs from family members. (Many people with autism have touch aversion due to sensory processing issues and trouble regulating their emotional responses.)

Cases like this are a source of hope, but again, more research is needed to confirm that similar outcomes are possible among the general population.

What’s next?
Phase II research will include 165 children with autism between ages two and eight. Kids may receive their own umbilical cord blood, cord blood from a donor or a placebo. After receiving the treatment, they’ll undergo a series of behavioral and reasoning tests, along with brain imaging.

Six months later, they’ll return for another round of treatment. Except this time, they’ll receive the placebo if they got cord blood initially, and cord blood if they first received the placebo. Researchers chose this type of study, called a crossover trial, because they wanted every child to experience any positive outcomes—but they still need to compare the treatment against placebo to determine if it’s truly working. 

To learn more about phase II testing, call 844-800-CORD or email cordbloodtherapyinfo@dm.duke.edu or autismresearch@duke.edu. Read more about autism.

Medically reviewed in May 2018.

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