Results from the study show that umbilical cord blood infusion were safe and well tolerated by the 180 children participating in the clinical trial. Cord blood was also associated with improvements in communication skills in children with autism spectrum disorder (ASD) without intellectual disability (ID).
What did the study involve?
The study set out to evaluate whether umbilical cord blood infusions were safe and associated with improved social and communication abilities in children with autism. 180 children diagnosed with autism participated in the study and were evaluated 6 months after receiving the cord blood infusion.
Children between the ages of 2 and 7 with autism received a single IV autologous (a transfusion of their own cord blood) or allogenic cord blood infusion (a transfusion of cord blood from a donor).
Clinical outcomes were assessed using validated measures, including The Vineland Adaptive Behaviour Scale, which assessed the adaptive functioning, social and communication skills, daily living skills and motor skills of participants prior to the study, and at 6 months and 12 months after the infusion had been made.
The study also evaluated the participant’s eye-tracking. Children with ASD tend to have shorter look duration and often avoid eye contact.
What were the results?
Overall, the results of the study suggest that a single infusion of cord blood was not associated with improvements in communication, social skills, and other autism symptoms of the group as a whole. However, further analysis revealed that the cord blood infusion improved communication skills within a subgroup of the study’s population.
After receiving the umbilical cord blood transfusion, children with autism who were not diagnosed with intellectual disability (ID) saw significant improvements in social cognitive abilities as well as sustained attention and changes in brain activity. Children with autism without ID showed significantly greater improvements in communication skills when treated with cord blood in comparison to those treated with the placebo.
6 months after receiving the cord blood treatment, participants without ID showed significant increases in sustained attention and eye contact during the assessment.
Previous studies have suggested that children with autism typically exhibit shorter look durations and minimal eye contact when looking at complex audio-visual stimuli. However, the findings of this study suggest cord blood treatment for autistic children without ID may enhance attention to complex stimuli.
people in the UK are autistic
are affected by autism spectrum disorder
people with ASD also have learning disabilities
What are the outcomes of this study?
Whilst the results from this clinical trial are promising, it’s important to emphasise that this is a phase II study. This study calls for more research to be carried out to determine whether cord blood infusion is an effective treatment for children with autism.
Are there other studies using cord blood to treat autism?
In 2017, Duke University released results from a Phase I clinical trial which demonstrated that the use of autologous cord blood infusions was safe. The FDA then approved an ‘Expanded Access Protocol’ shortly after for umbilical cord blood infusions at Duke University.
This protocol enables children with neurologic conditions, such as autism and other related brain injuries, and who have their own or a sibling’s cord blood stored to receive cord blood therapy through the program.
Many other clinical trials across the world are also showing positive results in the use of cord blood for autism. Studies to date have shown significant improvements in overall motor function and social skills. However, more studies are needed to add to this growing evidence.
What is Intellectual Disability (ID)?
Intellectual Disability is the most common developmental disability which results in significant difficulties in both intellectual functioning, i.e. communicating, learning, problem solving, and adaptive behaviour, i.e. everyday social and practical skills.
Intellectual Disability is characterised by below average intelligence or mental ability, with children with ID typically scoring less than 70 on an IQ (intelligence quotient) test. There can be varying degrees of intellectual disability ranging from mild to severe.
Duke University (2020), “Cord Blood Transplant: Umbilical Cord Blood Infusion Therapy for Brain Injuries and Disorders”, Duke Health, available at:
ClinicalTrials.gov (2019), “Expanded Access Protocol: Umbilical Cord Blood Infusions for Children with Brain Injuries”, U.S. National Library of Medicine, available:
Dawson et al., (2020), “A Phase II Randomized Clinical Trial of the Safety and Efficacy of Intravenous Umbilical Cord Blood Infusion for Treatment of Children with Autism Spectrum Disorder”, The Journal of Pediatrics, available at:
Surabhi Verma (2016), “Autism and intellectual disability: The big difference”, The Pioneer, available at:
Emily Sohn (2020), “The blurred line between autism and intellectual disability”, Spectrum, available at:
MentalHealth.org (2020), “Autism Spectrum Disorder Key Facts Summary”, Mental Health, available at:
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Sharma, A., Gokulchandran, N., Sane, H., Nagrajan, A., Paranjape, A., Kulkarni, P., Shetty, A., Mishra, P., Kali, M., Biju, H., & Badhe, P. (2013). Autologous bone marrow mononuclear cell therapy for autism: an open label proof of concept study. Stem cells international, 2013, 623875. https://doi.org/10.1155/2013/623875
Nguyen Thanh, L., Nguyen, H. P., Ngo, M. D., Bui, V. A., Dam, P. T. M., Bui, H. T. P., Ngo, D. V., Tran, K. T., Dang, T. T. T., Duong, B. D., Nguyen, P. A. T., Forsyth, N., & Heke, M. (2021). Outcomes of bone marrow mononuclear cell transplantation combined with interventional education for autism spectrum disorder. Stem cells translational medicine, 10(1), 14–26. https://doi.org/10.1002/sctm.20-0102
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Nguyen, L. T., Nguyen, P. H., & Hoang, D. M. (2021). A phase II randomized clinical trial of the safety and efficacy of intravenous umbilical cord blood infusion for treatment of children with autism spectrum disorder. The Journal of pediatrics, 230, 271–272. https://doi.org/10.1016/j.jpeds.2020.11.063
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