The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome is a collaborative network of specialists that are bonded by the focused vision of delaying or preventing transplant for individuals affected by congenital heart defects. As pioneering surgery and evolving medical practices have dramatically changed the outlook for children with HLHS in the past two decades, we now have many young adults that have successfully navigated the challenges of reconstructive heart surgeries. The emerging challenges, resulting from the huge successes of our past, now fuel our team with a new focus and purpose-to transform the treatment options with cell-based regenerative strategies and delay or prevent transplantation.
To accelerate regenerative applications for the purpose of congenital heart disease, our team was recruited to address basic questions that would unlock the therapeutic potential of emerging technologies from clinical imaging to molecular genetics to regenerative applications. The ultimate success of this integrative approach was interdependent on individual success of clinical practice, academic research, and entrepreneurial spirit of providing innovative products and services. This multidisciplinary collaboration was bold and surely fraught with unforeseen challenges, yet was aligned with the tradition, culture, and expectations of Mayo Clinic staff. With the right team focused on the right questions, we have come to expect the unexpected and strive to translate idealism into practice.
The first question, what are the predictors of right ventricular failure and how do we anticipate the needs of our patients that may develop heart failure? To address these critical questions, we have engineered a collection process of cardiac imaging studies from individuals across the spectrum of this syndrome to test the hypothesis that emerging technologies offer superior resolution of right ventricular function. Ultimately, answers to these questions will lead to practice guidelines that allow cardiologist to proactively track and follow patients through high-risk periods.
The second question, what is the genetic underpinning of this congenital heart defect and how do we apply this knowledge to utilize the most effective treatments for individuals from traditional drug therapy to innovative cell-based treatments? To address these unknown questions, we have implemented a comprehensive pipeline that identifies the most valuable patient samples for the purposes of genetic studies, generates the list of potential candidate genes, and tests the functional significance of these genetic variances. This process generates new knowledge that will guide and prioritize future clinical diagnostics and therapeutic options.
The third question, what is the stem cell-based strategy that will be optimal for cardiac repair in this congenital heart disease and how do we establish protocols that lead to the most practical, safe, and effective experimental treatments required to launch clinical trials? To address these novel questions, we have re-purposed expertise in cardiac regeneration towards pediatric congenital heart disease models. By comparing a wide variety of stem cells and progenitor cells, we aim to accelerate our discoveries and focus the planning of clinical trials using the optimized strategies that have been validated in pre-clinical studies.
As our business plan highlights, our most valuable collaborating partners are the families and individuals affected by this syndrome. Furthermore, our ultimate customer of the products and services we aim to foster are the same families and individuals living with this syndrome. Our program spans across clinical, research, and educational components available to our collaborative team and that are mandated by the complexity of our patients. Our unique focus on HLHS provides the constant reminder of what we do and why we do it.