Research Horizons


Mayo Clinic, Cincinnati Children’s to collaborate on rare congenital heart defect

Mayo Clinic’s Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) and Cincinnati Children’s Hospital Medical Center announce their collaboration within the nationwide HLHS Consortium to provide solutions for patients with hypoplastic left heart syndrome.

Hypoplastic left heart syndrome is a rare and complex form of congenital heart disease in which the left side of the heart is severely underdeveloped. Infants born with HLHS undergo a series of three surgeries to support the right side of the heart, which must work doubly hard to pump blood to the lungs and the rest of the body. The consortium’s regenerative research continues to look for safe and effective new therapies to further strengthen these young patients’ hearts, with the hope of delaying or eliminating the need for a heart transplant later in life.

The consortium aligns regional medical centers of excellence and advocacy groups with the shared goal of finding solutions for people affected by congenital heart disease, including HLHS. The consortium, which was developed by Mayo Clinic’s Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome, works to sustain a continuous pace of research and innovation by bringing clinical trials and expertise to patients across the country.

With a 135-year history serving pediatric patients, Cincinnati Children’s Hospital Medical Center is positioned as one of the oldest and most distinguished pediatric hospitals in the nation. Cincinnati Children’s will participate in future HLHS Consortium clinical trials under the guidance of James Tweddell, MD, executive co-director of the Heart Institute and director of cardiothoracic surgery at Cincinnati Children’s.

“We’re excited to collaborate with Dr. Tweddell and the team at Cincinnati Children’s,” says Tim Nelson, MD, PhD, director of the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome. “They are not only a center of excellence for the early-stage HLHS surgeries, but they also provide high-quality, comprehensive care for teens and adults with congenital heart disease. We’re proud to work together with them to find new ways to strengthen the hearts of people with HLHS.”

“A large portion of the patients that we care for have a single pumping chamber, and decreased function continues to plague a subset of our patients with single ventricle anatomy,” says Tweddell. “Dr. Nelson and the Wanek family have developed a cutting-edge research program into the use of autologous stem cells for maintenance and improvement of single ventricle function.”

Nelson and his colleagues have developed techniques to isolate and amplify stem cells from umbilical cord blood, Tweddell explains. Then the cells are injected into the myocardium of single ventricle patients at the time of the second staged surgery.

“Preliminary studies have shown the stem cell injections to be safe, and future studies will build on this experience while looking at the benefits of stem cell therapy. We are excited to collaborate with Mayo Clinic and the Wanek family on this important new strategy to improve the lives of some of our most challenging patients,” says Tweddell.

Cincinnati Children’s is the ninth member of the HLHS Consortium, joining Mayo Clinic, Children’s Hospital of Philadelphia, Children’s Hospital Los Angeles, Children’s Minnesota, Children’s Hospital Colorado, The Children’s Hospital at OU Medicine, and Ochsner Hospital for Children, as well as the advocacy group Sisters by Heart.