About the Program

A dedicated, multidisciplinary team of Mayo Clinic physicians, researchers and other study staff members in the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) are working together to discover and apply innovative solutions for HLHS.

HLHS is a rare, complex form of congenital heart disease in which the left side of a child's heart is severely underdeveloped. The cause of HLHS is unknown, and treatment generally requires a series of surgeries. Though post-surgery outlook is good and many people with HLHS live nearly normal lives, there is always the chance that a heart transplant may be needed in the future.

Vision and goal

Toward a vision of delaying or preventing heart transplantation for people with HLHS, the program's overall goal is to initiate clinical trials that bring the latest advances in cell therapy to people with HLHS.

Objectives

To accomplish this goal and achieve the program's vision, Mayo physicians and scientists are collaboratively working on four strongly connected objectives:

• Human genetics: Uncovering the molecular basis of HLHS. By investigating the genetic underpinnings of HLHS, researchers hope to identify the genetic mutation or mutations that cause HLHS.
• Imaging and outcome: Predicting the need for a heart transplant. Mayo investigators are working to understand how a patient's medical history, the medical history of the patient's family, and ultrasound and MRI images of the patient's heart relate to one another — and how they can be used to predict whether a patient will need a heart transplant.
• Regenerative strategies: Creating patient-specific stem cell-based HLHS interventions. Using state-of-the-art technology — including induced pluripotent stem cells — Mayo researchers are looking to not only create new kinds of cell therapies, but also to determine from whom these therapeutic cells can originate and when best to deliver them for maximum benefit.
• Biorepository: Setting up a system for continued learning about HLHS. To study HLHS and explore new treatment technologies, investigators are creating a large repository of patient data and tissue samples, called a biorepository, which can be drawn from in the future.

Team approach

Finding solutions for a disease as complex as HLHS requires teamwork, which is why the Program for HLHS takes a highly integrated approach.

Led by a comprehensive core group of Mayo Clinic physicians and scientists — including specialists in pediatric cardiology, cardiovascular diseases and regenerative medicine — the program also has a fully dedicated staff with expertise in biomedical informatics, biospecimens management, information technology, and family education and subject participation.

Additional support comes from Mayo research staff members who dedicate some portion of their time to the program, such as postdoctoral fellows, laboratory personnel, genomics experts, sonographers and administrators.