Hypoplastic Left Heart Syndrome

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 rare and complex form of congenital heart disease in which the left side of a child's heart is severely underdeveloped. Today, treatment normally involves a three-stage surgery to reroute blood flow in the heart.

Though effective, these surgeries do not eliminate the chance that a child with HLHS may need a heart transplant in the future.

In the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome, a diverse group of Mayo Clinic physicians and researchers led by Timothy J. Nelson, M.D., Ph.D., at Mayo Clinic's campus in Rochester, Minnesota, is launching clinical trials that bring the latest advances in cell therapy to people with HLHS.

Within the program, teams are:

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

Visit the HLHS Cause to Cure blog for the latest research developments, patient stories and other information from Mayo Clinic researchers, physicians and staff.

More information about the program is on the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome website.