Hypoplastic Left Heart Syndrome

Transcript
Timothy J. Nelson, M.D., Ph.D.
  • Director
  • Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome

Timothy J. Nelson, M.D., Ph.D.: The Hypoplastic Left Heart Research Program is focused on many aspects. One of which is regenerative strategies, and how can we use the knowledge of stem cells to try to understand this particular disease and how we can ultimately design new therapeutics that we could use to treat this, treat patients with this syndrome. Hypoplastic left heart syndrome is a congenital heart disease. Babies are born with this condition, with half of their heart missing. And one strategy that we're aiming to understand is what causes this congenital birth defect in these children. And one hypothesis is that the stem cells may be defective. So our goal is to understand how the stem cells may not be functioning properly in these children. And by understanding that, we may be able to find ways to reverse it and to have a therapeutic that we could offer.

One of the problems with hypoplastic left heart is that the heart is too weak to do what we're asking it to do. The right side of the heart isn't designed to be strong enough to do the workload. So we're aiming to understand how we can use stem cells and how we can plant the right stem cell into these patients to make the right heart bigger, better and stronger. If we can identify the right stem cell and we can transplant it into the patient at the right time, this may be one strategy to make these patients live a better life and have a stronger heart function.

Hypoplastic left heart syndrome (HLHS) is a complex and rare heart defect present at birth in which the left side of a child's heart is severely underdeveloped.

Treatment usually involves a three-stage surgery to reroute blood flow in the heart. While surgery is lifesaving and effective, it does not eliminate the possibility that a person with hypoplastic left heart syndrome may need a heart transplant in the future.

The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome, is a diverse group of physicians and researchers led by Timothy J. Nelson, M.D., Ph.D., at Mayo Clinic's campus in Rochester, Minnesota. The overall goal of the program is to delay or even prevent heart failure for people with hypoplastic left heart syndrome. The program takes a multifaceted research approach that includes imaging and outcomes, human genetics, and regenerative medicine.

Within the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome, regenerative strategies include:

  • Studying how regenerative therapies can strengthen the hearts of patients with HLHS and delay or even prevent heart failure
  • Developing better ways to assess right ventricular function in HLHS and predict outcomes
  • Determining the genetic basis for HLHS
  • Building a comprehensive HLHS biorepository

The program has launched clinical trials using a patient's own stem cells with the goal of strengthening the heart. One of the clinical trials uses stem cells collected from a baby's own umbilical cord blood banked at no cost with the program. The stem cells are injected into the baby's heart during the second surgery.

A second clinical trial uses stem cells collected from the patient's own bone marrow that are injected back into the body with the goal of strengthening the heart. Learn more about these clinical trials on ClinicalTrials.gov and Mayo Clinic's clinical trials page, respectively:

For more information about no-cost umbilical cord blood banking, email HLHS@mayo.edu.

The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome has also built a hypoplastic left heart syndrome consortium — a network of institutions working together across the U.S. to delay and prevent heart failure for people with this congenital heart defect. The consortium makes it possible for patients to participate in the research without traveling long distances.

Visit the HLHS blog for the latest research developments, patient stories and other information related to hypoplastic left heart syndrome and follow the program on Facebook, Twitter or Instagram. To learn more about how to participate, email HLHS@mayo.edu.

Strengthening the HLHS Heart With Regenerative Medicine