Autism – promising results from stem cell clinical trial

Promising results from a Phase I clinical trial evaluating the safety and feasibility of intravenous infusion of own umbilical cord blood for children with autism were published recently.

In this small, early stage clinical trial, 25 children ages 2 to 5, (median age 4,6 years) with a confirmed diagnosis of autism spectrum disorder, had undergone the new experimental treatment proposed by the scientists at Duke Health.

All the children enrolled in the clinical trial had available their own umbilical cord blood stored in family banks, according with parents decision to collect the blood at their birth.

72%  of enrolled children had moderately severe or severe autism symptoms. 

The primary endpoint of the clinical trial was safety, similar with the prior clinical trial evaluating the infusion of autologous (own) cord blood in 184 children with neurologic disorder (cerebral palsy, hydrocephalus, other acquired  neurologic disorder).

The assessment of safety was done during the infusion and post-infusion, through phone interviews with participants’ parent or guardian at 7–10 days, 3 months, and 9 months after infusion. Also the possible related or unrelated adverse effects were assessed in person at the baseline, 6- and 12-month clinic visits.

Only 12 adverse events were considered to be related with infusion, the most common being allergic reaction (urticartia and or/cough occurring on the day of infusion). These AE were considered expected and resolved without sequelae.

The safety of the single infusion of own cord blood was confirmed by the assessment of reported AE during the 12 months period.

Multiple assessments scales were used during this clinical trial to determine the utility as an endpoint for future clinical trials, which would focus on evaluating the efficacy of the treatment for children with autism.

Applying a wide range of outcome measures (multiple parent-rated and clinician rated measures ) improvements were shown in more than 2/3 of the treated children.

"Most of the observed behavioral changes occurred during the first 6 months and were sustained between 6 and 12 months postinfusion∗".

Even if  observational behavioral  changes were reported as improvements in speech, socialization and eye contact, it's important to highlight the limitation of this early stage clinical trial.

"As an uncontrolled open-label study,it is not possible to determine whether the observed behavioral changes were due to the treatment or reflect the natural course of development during the preschool period∗".

The use of own cord blood stem cells represent a new therapeutic approach. The medical team which run this clinical trial hypothesized that autism symptoms may be alleviated by modulating inflammatory processes in the brain driven by umbilical cord blood derived cell.

Here you can watch a presentation of Geraldine Dawson PhD about autism and infusion of own umbilical cord blood, as new potential therapy.

A larger phase II , controlled clinical trial has been initiated to verify if the observational results can be replicated and if there is or not, a correlation between these results and the infusion of own cord blood.

“After we’ve concluded the Phase II trial, we will have much more information and should be able to better characterize the potential for this therapy.” Geraldine Dawson, PhD, director of the Duke Center for Autism and Brain Development


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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