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CBA Foundation Supports Autism Treatment Study

Issued:  Nov. 10, 2020

The Cord Blood Association Foundation has awarded a $200,000 grant for further study of the therapeutic benefits of autologous or sibling cord blood infusions for young children with autism.

Logo of the Cord Blood Association Foundation

The two-year, multi-center research project has two objectives:

  • assess the outcomes of infusions of autologous or sibling cord blood for treating young patients with autism spectrum disorder (ASD)
  • develop standardized methods for remote monitoring of those treatment outcomes

Duke University Medical Center is the coordinating center for the study. Other participating centers will include Rutgers New Jersey Medical School and MD Anderson Cancer Center.

The research is being conducted under an IND from the FDA under its “expanded access program” that permits use of an investigational treatment for a serious disease or condition outside of a clinical trial when no comparable or satisfactory alternative therapies are available.

Recent Progress

In a recently completed study, Duke researchers tested whether a single infusion of a unit of a child’s own cord blood, or donor cord blood, could improve social communication skills in children ages 2 through 7 diagnosed with ASD. 

Although a cord blood infusion was not associated with improved social communications skills for the treated group as a whole, a subgroup without an intellectual disability showed improvements in communication, the ability to sustain attention measured by eye tracking, and enhanced brain function measured by increased alpha and beta electroencephalogram (EEG) power. The findings in the double-blind placebo-controlled trial were published in May in The Journal of Pediatrics.

“The theory supporting the treatment is that cord blood contains immune modulating cells called monocytes,” said Geraldine Dawson, PhD, a principal investigator for the recently completed and the upcoming studies. “In the laboratory, these cells calm down a type of brain inflammation that can be seen in children with autism. Clinical studies are testing whether cord blood infusion can lessen symptoms in children with autism.” 

Outcomes Measurement

The new study will monitor treatment outcomes in 150 children with ASD who received cord blood infusions, but do not qualify or are unable to participate in an active clinical trial. The investigators will develop web-based methods for remote monitoring and digital measurement of treatment progress. 

“Autism spectrum disorder is a complex neurological disease,” said Kenneth Giacin, president of the CBA Foundation. “Infusion of autologous cord blood is a promising treatment of autism and other brain injuries in children. This research will provide additional information about the effectiveness of cord blood for treating autism by developing sensitive, objective and reliable methods for measuring efficacy in clinical trials.”

About the Foundation

The CBA Foundation is a non-profit subsidiary of the Cord Blood Association that promotes both public and family cord blood banking and accelerates the use of cord blood and perinatal tissues to benefit patients and advance medicine. Its projects include clinical research for the use of cord blood and cord tissue in hematopoietic therapies and regenerative medicine, as well as other scientific, educational and philanthropic activity. Among its programs is a Research Scholar Award that supports young investigators.