Exploring Better Treatment Options for Rare Congenital Heart Disease

Mayo Clinic’s Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome and Children’s Hospital of Philadelphia are collaborating to delay and prevent heart failure for hypoplastic left heart syndrome, a rare and complex form of congenital heart disease in which the left side of a child's heart is severely underdeveloped.

“We are very excited to be working with Children’s Hospital of Philadelphia to explore better treatment options for patients with hypoplastic left heart syndrome,” says Timothy Nelson, M.D., Ph.D., director, Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome. “By entering into this collaboration, we are making it possible for all children with hypoplastic left heart syndrome to be able to participate in cell-based treatments, no matter their location. This new hypoplastic left heart syndrome consortium significantly expands the reach of hypoplastic left heart syndrome research.”

“Children’s Hospital of Philadelphia has a long-standing history of caring for children with hypoplastic left heart syndrome, and this exciting collaboration with Mayo Clinic offers promising opportunities to develop new ways to give patients an even better quality of life,” says Robert Shaddy, M.D., chief of Cardiology, Children’s Hospital of Philadelphia. “Lifesaving palliative surgery reroutes a child’s blood flow, but patients may have significant health problems, as they grow up with a unique circulation. Cell-based therapy offers us another potential option – beyond conventional medical treatments, ventricular assist devices or transplants – for a child or young adult with a failing heart.”

The collaboration is part of a consortium across the nation and will allow for a decrease in the amount of time from research and discovery to the clinical application of innovative cell-based therapies.

The consortium aligns regional centers into a nationwide collaboration led by the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome at Mayo Clinic to accelerate innovation on hypoplastic left heart syndrome, discovery sciences, and clinical expertise by investing local resources back into research. The program seeks to work with five to seven regional centers across the U.S. to fund the development of cell-based innovative research opportunities to transform the lives of people living with hypoplastic left heart syndrome.