Revolutionary Procedure to Potentially Change Landscape of HLHS Treatment
Breakthrough Research will Test Resident Cardiac Stem Cells that Repair damaged Heart Tissue and Improve Ability of Diseased Hearts to Pump Blood throughout the Body
In the U.S., approximately 1,000 babies per year are born with hypoplastic left heart syndrome (HLHS) and currently have a mortality rate of approximately 33 percent within the first year of life. Despite surgical advancements, about half of those children make it to their 5th birthday. The poor clinical outcomes are predominantly due to right ventricular dysfunction leading to heart failure. Presently, heart transplantation is the only clinically viable option even though there is not a significant improvement in long term survival rates. Innovative, adjunctive therapies to regenerate and remodel the right ventricle have become a crucial, yet unmet medical need in these challenging patients.
However, experts at the Children's Heart Program at University of Maryland Children's Hospital are currently studying a breakthrough procedure that will test cardiac stem cells that can repair damaged heart tissues and improve the ability of diseased hearts to pump blood throughout the body. This study will test how safe and effective autologous cardiac stem cell therapy is for managing HLHS. It is hypothesized that by injecting these stem cells into the right ventricle of the heart, there will be better right heart pumping function as the child gets older.
The primary objective is to overlay a novel cell therapeutic strategy on the two-stage surgical procedures that HLHS patients typically undergo in the first year of life:
- Stage I Norwood operation in the neonatal period
- Stage II BDCPA / GLENN procedure operation at approximately 4 months of age
To accomplish this, cardiac stem cells are grown from heart tissue collected during the Stage I Norwood operation. Usually, this tissue is discarded after the surgery, however for this trial, it will be collected and shipped to the University of Miami Interdisciplinary Stem Cell Institute. There the tissue will be processed, cultured, and expanded to grow cardiac stem cells. The culture and expansion process can take up to 8 to 10 weeks. Upon completion, cardiac stem cells will be cryopreserved and tested for quantity, quality and safety as mandated by FDA for an Investigational New Drug (IND). Upon completion of these tests, cells are shipped back to the clinical center for administration back to the patient during the Stage II BDCPA / GLENN Procedure operation.
For more information on cardiac research at University of Maryland Children's Hospital visit umm.edu/cardiacresearch.