July 31, 2024
“White Coats” is an AABB News series that interviews the experts that are transforming the fields of transfusion medicine and biotherapies. Join AABB today to read the rest of this month’s issue.
Joanne Kurtzberg, MD, the Jerome S. Harris Distinguished Professor of Pediatrics, a professor of pathology, part of the core faculty in Innovation & Entrepreneurship, a member of the Duke Cancer Institute and an affiliate of the Duke Regeneration Center, 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 and birthing tissues in the emerging fields of cellular therapies and regenerative medicine. Kurtzberg serves as the director of the Marcus Center for Cellular Cures (MC3), founder of the Pediatric Transplant and Cellular Therapy Program, director of the Carolinas Cord Blood Bank and co-director of the Stem Cell Transplant Laboratory at Duke University.
Kurtzberg performed the world's first unrelated cord blood transplant in 1993 at Duke University. Her research focuses on translational studies from bench to bedside, seeking to develop transformative clinical therapies using cells, tissues, molecules, genes and biomaterials to treat diseases and injuries that currently lack effective treatments.
Recent areas of investigation in the MC3 include clinical trials investigating the safety and efficacy of autologous and allogeneic cord blood in children with neonatal brain injury – hypoxic ischemic encephalopathy (HIE), cerebral palsy (CP) and autism. In the past, Kurtzberg has developed novel chemotherapeutic drugs for acute leukemias, assays enumerating ALDH bright cells to predict cord blood unit potency, methods of cord blood expansion, potency assays for targeted cell and tissue-based therapies. Kurtzberg currently holds several INDs for investigational clinical trials from the FDA.
AABB News spoke to Kurtzberg about her career path in cord blood banking, new developments and lessons learned.
I’ve always been drawn to working with children and challenged by complex diseases with unmet medical needs. During medical school and my pediatric residency (where Frank Oski, MD, was our chair), I was drawn to treating children with refractory hematological malignancies. I participated in the treatment of a teenager with refractory T-cell acute lymphoblastic leukemia (ALL) who was treated with a novel therapeutic and whose cells converted from a lymphoid to myeloid phenotype over five days.
That sparked my interest in hematopoietic stem cell biology, and in studying hematopoietic stem and progenitor cells, I got to know Hal Broxmeyer, PhD, and became interested in cord blood stem cells. I also cared for the first patient who had the first cord blood transplant for Fanconi anemia (by Eliane Gluckman, MD, PhD) in 1988.
When I started the pediatric transplant program at Duke in 1990, we focused on cord blood transplantation. In 1996, when the National Heart, Lung and Blood Institute (NHLBI) offered a request for proposal to establish additional public cord blood banks, Duke was awarded one of three contracts and established the Carolinas Cord Blood Bank.
The most exciting area of research relates to non-transplant applications where cord blood is being used as source material for the manufacturing of immune effector cell, induced pluripotent stem cells (IPSCs) and cells that are helpful in tissue repair in the field of regenerative medicine.
We are launching a phase three registration trial testing cord blood infusion in children with cerebral palsy. That trial will open in the next couple of months. If the trial is presented as positive and shows a treatment benefit, it will expand the indication for use of publicly banked alginate cord blood to include both transplant and treatment of cerebral palsy.
We’re going to see an explosion of biomaterials and cell therapies that will come from cord blood, and I believe public banks—which are FDA-licensed facilities that understand how to operate and procure cord blood and birthing tissues under Good Manufacturing Practice (GMP)—will be the providers of these materials.
In some cases, cord blood banks will expand their scope and collaborate with small biotech or other academic centers to make products. For example, there are companies making allogeneic and natural killer (NK) cell therapies and allogeneic mesenchymal stromal cells (MSCs), and the NK cells and MSCs are coming from donor cord blood or placenta.
There are also companies making allogeneic chimeric antigen receptor (CAR) T-cell therapies so that patients whose cells are not healthy enough to make their own CAR T cells can use a donor-derived product. Cord blood is also being used as a source material for manufacture of those cells.
There are not enough cord blood units being selected for hematopoietic cell transplantation. The decline is unfortunate and threatens business models for all cord blood banks. Cord blood banks aren't supplying enough units to sustain the operation of the banks and ongoing collection. It also means that transplanters aren't thinking about cord blood as much as they should because there are thousands of patients in the United States every year who don't receive a transplant, and in some of those patients, it's because people don't think they have a viable donor.
Cord blood transplant outcomes are much better now than they were in the early 90s for many, many reasons. Transplanters are gun shy or scared about getting involved with cord blood, which is depriving some patients of access to a transplant that would be successful for them and give them better options for treatment of their disease. We as cord blood bankers did not market ourselves as well as we should have. There needs to be another look at the modern outcomes of cord blood, which are equivalent to other alternative donor sources, as well as reconsideration and retraining of programs to perform cord blood transplants again.
As a transplanter for more than 30 years, I know that cord blood works. Patients I have transmitted as babies or young children are now in their 20s and 30s. They were transplanted for diseases they otherwise would have died from as young children. Knowing that these patients survived and are healthy and doing well brings me a lot of satisfaction. I also know that in some of the newer applications where newborn screening is being implemented, such as using cord blood to treat children with leukodystrophies, cord blood is very successful in both saving these children's lives and extending the quality of their life.
Cord blood is a necessity because they need a very fast transplant, and cord blood is essentially off the shelf. I'm also always energized by moms who donate their baby’s cord blood. We know cord blood would otherwise be medical waste. Moms really enjoy donating and giving back, and they get immense satisfaction knowing not only have they birthed a baby, but they may help someone else with a serious disease survive due to their donation.
I’m most proud of all the patients I’ve treated throughout the past 40+ years who have essentially become ‘my kids’ and part of my extended family.
I’ve learned to never give up hope and to really keep trying to solve the most complicated problems and think of ways to help the sickest patient, because you can make progress over time. I would love to fix things quickly, but change doesn’t happen overnight. I’ve learned some of the bigger advances take a lot of time, but it's worth it in the end.
I am an avid knitter and also love bike riding. Otherwise, I spend as much time as I can with my three grandchildren Bella, age 3; Sophie, age 1; Eve, age 1.
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