The program's goal is to transform patient care guidelines by identifying genetic causes of HLHS (led by Dr. Olson), predicting outcomes (led by Dr. O'Leary), developing a cell-based therapy (led by Dr. Nelson) and creating a system for continuous learning (led by Dr. Terzic).
For people born with a single ventricle heart, options are scarce. They must either undergo a palliative series of open heart surgeries, ending with the Fontan, or receive a heart transplant. As the first Fontan patients reach adulthood, emerging data suggests that all Fontans will eventually fail. It is no longer a matter of if, but when. There are several complex and individualized factors that determine when and how failure will present, but once it does, the only option becomes a heart transplant. While heart transplants are lifesaving, they come with their own risks and another timeline for eventual failure. In 2000, while at Stanford University, Dr. Mark Rodefeld began rethinking the way Fontan patients should be treated. Guided by the belief that there must be a better approach toward stronger outcomes for single ventricle heart patients, he began his research. He sought to develop a treatment option for Fontan patients that would extend the life of the Fontan repair and put off, or eliminate, the need for a heart transplant.
The HLHS program’s team of physicians, scientists, and engineers have a combined average of nearly 30 years of experience across their respective disciplines and have focused this expertise on treatments for the most severe forms of CHD, thereby furthering advanced research in genetics, stem cell biology, clinical trials, and cell-based manufacturing. The team developed a platform that enables multiple cell-based clinical trials to be conducted simultaneously in collaboration with a consortium of world class medical institutions. HeartWorks will expand the HLHS program product pipeline and grow its ecosystem of partners to scale up development of new CHD therapies, clinical trials, and product launches.
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