Encouraging start for cord blood therapy in autism spectrum disorder

Autism spectrum disorder is one of the diseases for which many advances in early diagnosis and behavioral therapies have been done in the last years. But still, researchers is trying to find a more effective therapy.

Infusion of cord blood stem cells is an innovation therapeutic approach for autism spectrum disorder. 

According to current hypothesis therapeutic effect could be related with suppression of inflammation processes and altering brain connectivity.

Noah Barcolos a 4 year old little boy is one of the 180 children enrolled in the phase II clinical trial taking place at Duke University Hospital, in USA.

At 2 year after he was diagnosed with autism he underwent the cord blood infusion.

This groundbreaking treatment had been already proven to be safety and feasible by the results of phase I clinical trial published in April, this year. 25 children ages 2 to 5, (median age 4,6 year) with a confirmed diagnosis of autism spectrum disorder were infused with their own cord blood.

Observational behavioral changes were reported as improvements in speech, socialization and eye contact in 2/3 of the treated children.

“Generally we saw behaviours improve at 6 months compared to their baseline study,” prof.dr. Joanne Kurtzberg told CNN

Based on the outcome of this early stage clinical trial, a new phase II clinical study has been initiated this year.

In comparison with previous one, the objective of this clinical trial is to evaluate the efficacy of intravenous umbilical cord blood infusion as cell therapy for children with autism spectrum disorder.

Children ages 2-7 years, who meets criteria for enrollment  in this clinical trial are undergoing intravenous infusion of own cord blood, or  donated unrelated cord blood, for those who doesn’t have available own cord blood.

Children are randomly assigned in 2 groups: Sequence A and Sequence B. Those included in Sequence A receive cord blood infusion at baseline and placebo, at 6 months. Children from Sequence B receive placebo at baseline and cord blood infusion, at 6 months.

Assessment of all patients will be done at 6 months from baseline infusion and at 12 months.

Noah Barcolos is one of the children taking part in this clinical trial. His parents already seen remarkable improvements:

“He gets upset. We got more eye contract, interaction, facial expressions,” said Noah’s dad, Michael.

“Now he makes the expressions at the right moments,” said Sammy. “He’s also calling me mommy. He’s never done that.”

Such positive outcome is very encouraging, both for parents and investigators medical team.

But until the clinical trial will be finished and results assessed from statistical point of view, no clear conclusions can be drawn.

It’s much too early and currently no statistically supported, as modern “evidence-based medicine” requires for systematic research.

More details about Noah Barcolos improvements you can find out watching the video.



Geraldine Dawson, Jessica M.Sun & al. " Autologous Cord Blood Infusions Are Safe and Feasible in Young Children with Autism Spectrum Disorder: Results of a Single-Center Phase I Open-Label Trial"  Stem Cells Translational Medicine

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