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Clinical Trials for Rare Childhood Disorders

Clinical Trials for Rare Childhood Disorders

Duke University
Durham, NC Dr. Joanne Kurtzberg

In North Carolina, the National Stem Cell Foundation helped fund a collaboration between the Institute for Cellular Therapeutics (ICT) at the University of Louisville and Dr. Joanne Kurtzberg, Director of the Pediatric Transplant and Cellular Therapy (PTCT) program at Duke University.

The collaboration was focused on the further development of an outpatient “mini” bone marrow transplant to halt the progression of otherwise fatal inherited childhood disorders and permanently treat inherited red blood cell disorders such as sickle cell disease and thalassemia.


Dr. 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.

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 ALDH bright cells in adults with stroke and radiation induced brain injury are also underway.

In 1998 Dr. Kurtzberg established the Carolinas Cord Blood Bank, one of the world’s largest unrelated donor cord blood banks. The bank has a current inventory of more than 40,000 units and has provided cord blood units to over 2,200 patients undergoing unrelated donor HSCT over the past 10 years.

Dr. Kurtzberg’s lab has developed novel assays to predict cord blood potency from segments attached to cryopreserved cord blood units, and is performing translational research testing cord blood expansion, cellular targeted therapies and tissue repair and regeneration.

In 2012, under the direction of Dr. Kurtzberg, the Carolinas Cord Blood Bank received FDA approval for DuCord, a stem cell product derived from umbilical cord blood, for use in transplants between unrelated donors and recipients. Dr. Kurtzberg currently holds several INDs for investigational clinical trials.

Dr. Kurtzberg has published over 300 peer-reviewed papers, chapters and scientific reviews. She is a member of the American Society of Hematology, the American Association of Blood and Marrow Transplantation, the American Society of Pediatric Hematology/Oncology, the International Society of Cellular Therapies, the Pediatric Blood and Marrow Transplant Consortium (PBMTC), and other organizations.

She serves on the Board of the Foundation of Accreditation of Cellular Therapies, co-chairs the National Marrow Donor Program’s Cord Blood Advisory Group and is a member of the Advisory Council of Blood Stem Cell Transplantation to Health and Human Services. Dr. Kurtzberg was awarded a Lifetime Achievement Award from the PBMTC in 2012.

November 2, 2017

“A child’s own umbilical cord blood may ease cerebral palsy”

FierceBiotech, November 2, 2017

Dr. Joanne Kurtzberg discusses a recent study using stem cells from banked cord blood to improve motor skills in children with cerebral palsy.

Read more.