Duke receives $15M to study potential autism treatment using umbilical cord blood

 In Stem Cell News

Duke Medicine received $15 million for research that explores the use of umbilical cord blood cells to treat autism, stroke, cerebral palsy and related brain disorders.

The award from The Marcus Foundation, an Atlanta-based philanthropic organization, will fund the first two years of a planned five-year, $41 million project by Dr. Joanne Kurtzberg, chief scientific and medical officer of Duke’s Robertson Cell and Translational Therapy Program, and Geraldine Dawson, director of the Duke Center for Autism Diagnosis and Treatment.

Duke university, and Kurtzberg specifically,has been a trailblazer in studying the benefits of umbilical cord blood. In 1993, for example, it performed the world’s first successful transplant of unrelated cord blood. In 2012, Duke University School of Medicine received approval from the U.S. Food and Drug Administration to market DUCORD, a stem cell product, making it only the third bank in the United States to receive that designation.

“I am excited about this unprecedented opportunity,” said Dr. Victor Dzau, chancellor for health affairs and president and CEO of Duke University Health System. “Joanne Kurtzberg has done groundbreaking work on cord blood transplantation at Duke, and Geri Dawson brings an enormous wealth of knowledge and experience of autism. Together they will explore innovative approaches to treating these challenging brain disorders. This research holds the promise of truly transformational discovery, and we are deeply grateful to The Marcus Foundation for making it possible.”

Kurtzberg and Dawson hope to develop cell-based therapies that can potentially restore brain function in people with the disorders, for which there currently are no cures. If successful, the study could identify therapies for further evaluation in clinical trials to potentially decrease disabilities and improve the quality of life for millions of children and adults.

The project will consist of a series of clinical trials using umbilical cord blood cells to treat a total of 390 children and adults with autism, 100 children with cerebral palsy and 90 adults with stroke. Based on previous research, Kurtzberg and Dawson hypothesize that cord blood may promote repair of dysfunctional or damaged areas of the brain.

“Funding for this type of research is very scarce, so the only way we can truly make progress is with support from private philanthropic organizations like The Marcus Foundation,” said Dr. Nancy Andrews, dean of Duke University School of Medicine. “With the foundation’s help, we hope to give untold numbers of people with autism and related disorders hope for a better outcome.”

There are some 2 million people in the United States with autism spectrum disorder, a group of conditions affecting social communication and behavior. Stroke kills an average of nearly 130,000 in the U.S. every year, while cerebral palsy currently affects an estimated 764,000 children and young adults.

“The whole program has enormous potential,” said Kurtzberg, who is also director of the Pediatric Blood and Marrow Transplant Program and the Carolinas Cord Blood Bank. “Autism, stroke and cerebral palsy are all neurologic conditions that impair function and quality of life for these children and adults. If we can make that better, it will have a huge personal and societal impact.”

Cord blood cells are collected without risk to the mother or baby from the placenta, which is otherwise discarded as medical waste after a baby is born. After collection, the cells can be frozen and stored for future use in blood stem cell transplantation or cellular therapies.

Kurtzberg’s previous research has shown that cord blood cells can reduce inflammation and signal normal cells to repair damage in areas of the brain affected by inherited pediatric brain diseases. A recently published Duke study indicates that treating children with their own cord blood cells can have similarly beneficial results in cases of hypoxic ischemic encephalopathy.

Building on those research results, the new study will try to determine whether cord blood cells will have the same effect in cases of autism and stroke. Using methods developed by Dawson, the study will examine whether the therapy not only improves behavioral outcomes in children and adults with autism, but also reshapes the patterns of brain activity.

Source: Triangle Business Journal


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