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Clinical Trial to Evaluate the Efficacy of Umbilical Cord Blood to Improve Outcomes for Children with Autism Spectrum Disorder

In this interview, Dr Joanne Kurtzberg explains how cord blood is being used in a Stage 2 clinical trial for autism and her hopes for the future. 

Joanne Kurtzberg is an internationally renowned expert in pediatric hematology/oncology, pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation, and novel applications of cord blood in the emerging fields of cellular therapies and regenerative medicine.

Along with a team of researchers at the Duke University Medical Center, she has been conducting a program of research to evaluate the efficacy of autologous and allogeneic cord blood for improving outcomes of individuals with autism spectrum disorder.  

The first study examined the safety and efficacy of using autologous cord blood to treat young children with autism spectrum disorder and assessed the feasibility of various outcome measures to determine which measures can be used as primary and secondary endpoints for a randomized phase 2 clinical trial (DukeACT) that is currently underway.

Cord blood cells can be collected from the placenta and stored for future usage in cord blood banks for cellular therapies or blood stem cell transplantation.

Previous research has shown that cord blood cells can help reduce inflammation and signal cells to help repair damaged brain areas. The goal of this study was to investigate whether similar success will be shown in children with ASD


About Dr Kurtzberg

Dr. Kurtzberg pioneered the use of umbilical cord blood as an alternative stem cell source for unrelated hematopoietic stem cell transplantation (HSCT).

Over the last two decades Dr. Kurtzberg has established an internationally known pediatric transplant program at Duke which treats children with cancer, blood disorders, immune deficiencies, hemoglobinopathies and inherited metabolic diseases.

 In 2010, Kurtzberg established the Julian Robertson Cell and Translational Therapy Program (CT2) at Duke. CT2 focuses on translational studies from bench to bedside with a focus on bringing cellular therapies in regenerative medicine to the clinic. Recent areas of investigation in CT2 include the use of autologous cord blood in children with autism spectrum disorder, neonatal brain injury and cerebral palsy, as well as preclinical studies manufacturing oligodendrocyte-like cells from cord blood to treat patients with acquired and genetic brain diseases. Studies of autologous bone marrow ALDHbright cells in adults with stroke and radiation induced brain injury are also underway.



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