Breakthrough Stem Cell Treatment for HLHS Patients

The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome at Mayo Clinic has successfully demonstrated a new treatment therapy using umbilical cord blood for children with hypoplastic left heart syndrome (HLHS).

“This is the first report of a personalized cell-based strategy to rebuild hearts in children with congenital heart disease (CHD) at the time of surgery,” said Dr. Tim Nelson, co-author of the study and director of the program. “This approach could eventually transform the way we surgically repair CHD by preemptively strengthening the heart muscle before it has time to fail.”

The HLHS program at Mayo Clinic is the first in the U.S. to receive permission from the FDA for a clinical trial utilizing autologous umbilical cord blood (UCB) to test this potential therapeutic option for patients with HLHS. The current care for children with HLHS has improved using a series of open heart surgeries, but despite the many advances, there is still a possible risk of right ventricle failure, requiring the need for a heart transplant.

“This landmark study is key to developing complementary therapies for patients with CHD. It is important to remember that current surgical palliation offers only part of the solution for complete long-term management of these patients. Having the ability to finally take basic research in stem cell therapy and adding it to the surgical management of these single ventricle patients is truly exciting and only begins to touch on the numerous future possibilities of combining surgery and regenerative therapies,” said Dr. Harold Burkhart, a pediatric cardiothoracic surgeon, and co-author of the study.

The case study involves an HLHS newborn whose umbilical cord blood was collected at birth. The umbilical cord blood was then processed to achieve mononuclear cells and preserved at below zero temperatures until the time of delivery. At four days old, the infant underwent the Stage I Norwood procedure, allowing the right ventricle to pump blood to the lungs and the body. Then, at four months old, the patient underwent Stage II surgery, also known as the Glenn procedure, and the stem cells were injected into the baby’s right ventricle. During the one and three month follow ups, the child’s right ventricle function had improved. “The study tells us that our goal of delaying and preventing heart transplants by using stem cell therapy is not just a dream but is an achievable target in the near future. These exciting results fuel the passion of our team to work harder and faster toward our goal. It gives optimism to families and physicians caring for children with HLHS that transplant is not the only option when the single ventricle starts deteriorating in performance,” said Dr. Yasir Qureshi, co-author of the study and pediatric cardiologist.

“The study tells us that our goal of delaying and preventing heart transplants by using stem cell therapy is not just a dream but is an achievable target in the near future. These exciting results fuel the passion of our team to work harder and faster toward our goal. It gives optimism to families and physicians caring for children with HLHS that transplant is not the only option when the single ventricle starts deteriorating in performance,” said Dr. Yasir Qureshi, co-author of the study and pediatric cardiologist.

The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) is a collaborative network of specialists bonded by the vision of delaying or preventing heart failure for individuals affected by congenital heart defects including HLHS. The specialized team is addressing the various aspects of these defects by using research and clinical strategies ranging from basic science, to diagnostic imaging to regenerative therapies.