Duke researchers are investigating the ability of umbilical cord blood to treat a number of diseases, including autism.

In 1993, the Pediatric Blood and Marrow Transplant Program of Duke Medicine became the first center in the world to perform a transplant using umbilical cord blood from an unrelated donor source for patients who did not have a matching donor in their family or in the adult donor registries. To date, they have performed more transplants of this kind than any other center in the world. Dr. Joanne Kurtzberg—professor of pediatrics and pathology, founder of the PBMT program and pioneer of umbilical cord blood transplantation—has been conducting clinical trials to investigate whether umbilical cord blood can help treat a variety of inherited metabolic diseases, hematologic cancers, cerebral palsy and, recently, autism. 

“[Umbilical cord blood] comes from the leftover blood in the placenta or afterbirth, and it used to be discarded as medical waste," Kurtzberg said. “It can be collected without any risk to the mother or baby."

Kurtzberg explained the theory behind her approach—that a low proportion of cells in umbilical cord blood are blood stem cells that can rescue damaged bone marrow. Umbilical cord blood can thus act as an alternative source of stem cells for those who require transplants, but do not have an appropriately matched family or unrelated donor. Cord blood is not a controversial stem cell source, she noted.

Kurtzberg's team has been conducting clinical trials to better understand the efficacy of umbilical cord blood as a regenerative treatment for many years now, and her findings have made her optimistic about the future of this potential therapy, she said.

“We are encouraged by our results with children in autism, although we’ve just completed a safety trial and are about to open a randomized phase II trial," she explained. “We’ve just completed a randomized phase II trial to test whether there is a benefit of cord blood in children with cerebral palsy. We will be publishing that data soon, but it looks encouraging.”

So far, Kurtzberg's group has demonstrated that umbilical cord blood is safe to use when infused through an IV. She noted that stem cells might not be the only components of umbilical cord blood that trigger a response for body repair, adding that some of the most active cells that induce repair are not believed to be stem cells.

The phase II clinical trials studying autism spectrum disorder, which began last month, are being co-led by Kurtzberg and Geraldine Dawson, director of the Duke Center for Autism and Brain Development and professor of psychiatry and behavioral sciences. Dawson's team is currently conducting the diagnostic and clinical assessments and examining how the therapy affects brain activity.

Mohamad Mikati, chief of the pediatric neurology division and professor of neurobiology, has worked specifically with umbilical cord blood as a potential treatment for cerebral palsy, and it is hypothesized that umbilical cord blood could facilitate neural cell repair in the brain. He noted that his group has been investigating the safety and efficacy of umbilical cord stem cells in treating the disease.

“We have been conducting a very well-controlled, comprehensive study that we believe will help answer the question of whether stem cell therapy is useful in [treating] cerebral palsy," he said. “It will be a very thorough evaluation of the therapy.”

The next steps for Kurtzberg and Mikati will be to identify the patient subgroups who are helped by the therapy, and subsequently to do further testing to confirm their findings. They also hope to look into other neurological diseases, including those in adults, that umbilical cord blood might prove useful in treating.

“There are other developmental and neurological diseases that may potentially be helped by cell therapy, and these would be the next steps that one would consider,” Mikati said.