Health Professional News

Collaborating on new therapies for hypoplastic left heart syndrome

Children’s Minnesota and Mayo Clinic recently announced a formal collaboration focused on developing new treatments to prevent heart failure for hypoplastic left heart syndrome (HLHS), a rare form of congenital heart disease in which the left side of a child’s heart is severely underdeveloped.

The HLHS consortium, founded by Mayo Clinic’s Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome, also includes experts from Children’s Hospital of Los Angeles and Children’s Hospital of Philadelphia. With a national network of researchers, the consortium widens access to clinical trials for patients regardless of location and enables the nation’s top pediatric cardiovascular programs to pool scientific knowledge. The collaboration will speed the development of new treatments, moving them quickly from the research stage to clinical application.

Delaying or preventing heart failure is the focus of the consortium’s research. Among the most promising developments is cell-based regenerative therapy, which may help restore the right side of the heart’s pumping ability when it begins to decline, or may even help prevent the decline altogether. Using different types of stem cells from various sources — including cells from the patient’s own body, such as stem cells from cord blood — the therapy stimulates the regeneration of defective heart tissue, leaving new, healthy tissue in its place.

The team is also researching advanced imaging to improve diagnostics and uncover predictive factors for HLHS, as well as studying the genes of individuals with HLHS and their family members to better understand the genetic underpinnings of the defect.

Leading Children’s in this collaboration is David Overman, MD, chief of cardiovascular surgery. “We’re excited to discover the next innovation in treating HLHS,” he says. “Children’s has a long history with this condition, as the first Midwest team to perform the Norwood procedure,” he notes, adding that Children’s brings to the collaboration a deep understanding of the condition from decades of experience treating the region’s largest volume of complex HLHS patients.

“Even though the early surgeries save lives, we still face the likelihood that the child will eventually have ventricular failure. That’s why these new approaches — the therapies we’re studying as part of this consortium — could have such a positive impact on the lives of these patients. If we can restore the function of the single ventricle, we could delay all kinds of complications related to gradual failure, and cardiac transplantation may not be necessary for several additional decades.

Alissa Gunderson