Research is vital to advancing CHD therapies and improving outcomes. Dr. Joy Lincoln and the Herma Heart Institute at Children’s Wisconsin are committed to that mission of advancement. In this week’s blog, read Dr. Lincoln’s article about the work they are doing to identify ways in which treatment can be tailored to the individual, and learn how you or someone you know might be able to participate in the process.
In 2006, Chris and Stephanie Wolfe were excited to be first-time parents of twins. During a standard ultrasound visit with their obstetrics team, one of the babies was diagnosed with a rare double outlet right ventricle, single ventricle variant. After the diagnosis, the Wolfe family put Caleb’s life in the hands of Michael Mitchell, MD, medical director of cardiothoracic surgery at the Herma Heart Institute at Children’s Wisconsin and professor and chief of congenital heart surgery at the Medical College of Wisconsin. When Caleb was 3 months old, Dr. Mitchell performed the first of Caleb’s surgeries, a pulmonary artery banding. Four months later, Caleb underwent his Glenn procedure with Dr. Mitchell, followed by the Fontan at 3 years old.
In the summer of 2009, Stephanie was once again pregnant. Again, the baby, was diagnosed in utero with a single ventricle disease that was later classified as ‘true’ hypoplastic left heart syndrome after birth. After another high-risk pregnancy for the Wolfe family, while also dealing with Caleb’s Fontan procedure, Emma was born in January of 2010. Over the next 5 years of Emma’s life, Dr. Mitchell performed six major surgeries including her Norwood at 3 days of age, Glenn at 6 months, coarctation of the aorta repair at 8 months, aortic reconstructions at 10 months and 2 and half years, then her final Fontan at age 5.
Thanks to the advancements in our understanding of single ventricle disease and surgical approaches, Caleb, Emma, and their unaffected brother Tyler (Caleb’s twin) are thriving today and living life as normal as they can. Caleb and Emma both play percussion in their school band and enjoy baseball, volleyball and dance. The Wolfes continue to be actively engaged in congenital heart disease research at the Herma Heart Institute at Children’s Wisconsin and both Caleb and Emma look forward to their week at residential heart camp with Camp Odayin each summer.
Thomas Jefferson once said, “All men are created equal….” But in this blog post, I would like to respond to that as a biologist with, “All are not created equal” as approximately 1 in 100 are born with a heart defect. Furthermore, the cause of why Emma and Caleb developed single ventricles is likely very different from why other affected children develop the same disease-type. The reason for this is not clear, but we are learning that the patient’s individual genetic make-up, environment and lifestyle all contribute to problems with heart structures being formed correctly in utero. As the etiology of congenital heart disease is unique to the patient, why should they be commonly treated the same way?
This is where precision medicine comes in — a revolutionary approach that helps your clinical care providers determine your unique disease risks, and the best treatment strategy that works best for YOU.
Here at the Herma Heart Institute at Children’s Wisconsin, we are working to identify person-to-person and population differences in children with congenital heart disease to further develop individualized cellular-based therapies according to the patient-specific profiles. Our ultimate goal is to achieve the best therapeutic results and outcomes. For this approach, the Herma Heart Institute welcomes participation of families with a history of familial congenital heart disease or recent diagnosis in utero to enroll into our new Cord Blood Program.
What does this entail? Expecting families at risk, or pre-diagnosed with congenital heart disease have the opportunity to meet with our team to discuss the program and enroll into the Cord Blood Program, which will involve the collection of umbilical material (blood, tissue) at the time of your delivery. Following the birth of your child, your clinical care team will ship your sample to our clinical grade storage facility, where it will be stored free of charge until utilized for research studies or clinical trials.
Why umbilical material? The umbilical cord tissue and blood is an enriched source for stem cells that have the unique ability to develop into specialized cell types depending on the environment that they are in. This includes cardiac cell types that make up the vessels, valves, myocardium (muscle), conduction system and other structures.
How can this help my child? As for Caleb and Emma, many children born with congenital heart defects require reconstructive surgery to correct their structural malformation. While this can be effective, there are major limitations, including the burden of anti-coagulation and immunosuppressive therapies and failure to adapt with the growing child should foreign (mechanical, bioprosthetic, material) devices be implanted. To overcome these limitations, the field has turned to the utilization of patient’s own cells (autologous), in this case umbilical-derived stem cells, to engineer cardiovascular structures (myocardial patches, heart valves, vessels, etc.) that can be implanted back into the same child during reconstructive surgeries. In utilizing autologous cells, rejection risks are low and the engineered structures become integrated into the heart’s native environment, allowing them to grow in size as the patient goes through life, thereby avoiding reoperations.
How can I get involved? If you are pregnant, or thinking about becoming pregnant (with or without risk for congenital heart disease) and are interested in enrolling into the Cord Blood Program at the Herma Heart Institute at no cost to you or your family, please fill out the contact us form on the Cord Blood Program website.
Joy Lincoln, PhD is a Professor of Pediatrics at the Medical College of Wisconsin, Director of Cardiovascular Research at The Herma Heart Institute and Associate Section Chief of Pediatric Cardiology at Children’s Wisconsin. She completed her PhD at The University of Durham in the United Kingdom, and then moved to the US for her post-doctoral training at Cincinnati Children’s Hospital in 2002. She began her faculty career at The Miller School of Medicine, University of Miami and in 2011 relocated to Nationwide Children’s Hospital in Columbus, Ohio. She was recruited to Milwaukee in 2019 where she continues her translational research program examining the etiology of congenital heart disease.