Investigation of Novel Biomarkers and Treatment Targets for Pediatric Heart Failure
Abstract
The most common birth defect, congenital heart diseases (CHDs), such as congenital cardiomyopathy and structural diseases, often lead to heart failure. Unlike the zebrafish, which has heart regeneration ability, human hearts are unable to self-repair due to their very limited endogenous regenerative capacity. Thus, mortality rates of heart failure are extremely high. Indeed, pediatric heart failure (PHF) is the leading non-trauma related cause of death for infant, child, or adolescent in the United States. Many children with PHF are treated by inserting a pump into the heart to help circulate blood. The pumping device is known as a Left Ventricular Assist Device (LVAD) implantation. Many children require heart transplantation, and these treatments have important value. However, most patients do not respond to LVAD and require heart transplantation. Unfortunately, heart transplantation is severely limited by the scarcity of donor hearts. Obviously, it would be of great clinical value if we could find a way to predict the PHF patient response to LVAD treatment, to help with clinical care and physicians’ decisions on whether heart transplantation will be needed. Our study will develop methods based on gene expression signatures for predicting whether PHF patients are LVAD treatment responders or non-responders. Moreover, identification of biomarkers in blood samples will provide a novel non-invasive method to understand if the heart is improving on LVAD treatment. Importantly, if we could find a way to facilitate endogenous cardiac regeneration, then survival rates of PHF would greatly improve. Our study will use cutting-edge techniques to gain important insights into the molecular mechanisms that stimulate endogenous cardiac regeneration and to develop potential novel therapeutic approaches.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Oct 29, 2018
- Source ID
- W81XWH1710418
Entities
People
- James S. Martin
Organizations
- Baylor College of Medicine
- United States Army