Introduction

At the forefront of stem cell research, Duke Medicine’s Paediatric Blood and Marrow Transplant Programme was the first centre in the world to perform a transplant using umbilical cord blood from an unrelated donor in 1993. Cord blood has also been noted as a potential treatment option for more than 80 diseases, from types of malignancies and blood disorders, to inherited metabolic disorders and immune system deficiencies. Now, Duke researchers are investigating the efficacy of umbilical cord blood in the treatment of diseases such as autism.

Founder of the Paediatric Blood and Marrow Transplant Programme, Duke Professor of Paediatrics and Pathology and umbilical cord blood transplantation pioneer, Dr Joanne Kurtzberg, explains that a small percentage of cells in umbilical cord blood are blood stem cells that can rescue damaged bone marrow and can therefore act as a source of stem cells for transplant patients who do not have an appropriately matched donor. Dr Kurtzberg and her Duke teams’ investigations into the use of in umbilical cord blood has most recently focused on its possible use in treating a variety of inherited metabolic diseases, hematologic cancers, cerebral palsy and autism. Having completed an initial safety trial, findings in this regard have been positive and presents an optimistic view for future use for children with autism.

What about those who do not have stored cord blood available?

While initially those who did not have their stem cells stored at birth could not undergo treatments requiring stem cells sourced from umbilical cord blood, neuroscientists and neurosurgeons around the world are developing ways to generate a person’s own stem cells to repair the brain. Studies of this new approach, which involves the culturing of a specific type of brain stem cell – called Doublecortinpositive cells, provides widespread optimism of future access to such treatment.

What’s next?

Phase II of the clinical trials studying autism spectrum disorder (co-led by Kurtzberg and Geraldine Dawson, director of the Duke Centre for Autism and Brain Development and Professor of Psychiatry and Behavioural Sciences) is currently underway with diagnostic and clinical assessments, as well as examinations on the effect of the therapy on brain activity, are taking place. The team at Duke are also entering into a randomised phase II trial to test if cord blood can be used to treat children with cerebral palsy.

Determined as safe to use when infused intravenously, Dr Kurtzberg has highlighted that stem cells might not be the only components of umbilical cord blood that trigger a response for body repair and has identified that some of the most active cells that induce repair are believed not to be stem cells.
The Chief of The Paediatric Neurology Division and Professor of Neurobiology, Mohamad Mikati – who has worked with umbilical cord blood as a potential treatment for cerebral palsy – has added that his group have been investigating the safety and efficacy of this treatment in a very controlled and comprehensive study which he hopes will answer the current questions surrounding its potential.

Following current trials and investigations, Dr. Kurtzberg and Prof. Mikati will be conducting further tests to confirm their findings and look into other neurological diseases for which umbilical cord blood may have potential in treating.