Heart Institute

Translational Research

Translational research – the move from bench to bedside – is a core part of research at the Heart Institute at Cincinnati Children’s. Most of the work we do is patient-centric; the questions raised at the bedside motivate research in the lab, which we then translate into care, returning our findings to the bedside.

Our current translational research includes work to identify characteristics and features of heart / muscle disease and cardiomyopathy. By identifying these earlier in patients, we can improve their outcomes with faster, more effective treatment. We are also working on a genetic level to understand the mechanisms that turn a gene mutation into a heart condition. By understanding these very basic mechanisms, we move closer to early diagnostics and gene therapies for these diseases.

The Pediatric Heart Network

Cincinnati Children’s is one of eight core centers chosen to become part of the Pediatric Heart Network (PHN), an organization created by the National Institutes of Health. The PHN is a select group of hospitals in the United States and Canada that conducts research studies with children who have congenital and acquired heart defects.

The Heart Institute is a core site for the PHN study, “Marfan Trial of Randomized Beta Blocker Therapy (atenolol) vs. Angiotensin II Receptor Blocker (ARB) Therapy (losartan).”

The trial compares the effect of these two drugs on the rates of aortic growth and progression of aortic regurgitation in patients with Marfan syndrome (MFS). MFS is a disorder of connective tissue due to mutation in the Fibrillin-1 gene. Cardiovascular pathology, including aortic root dilation, dissection and rupture, is the leading cause of mortality in patients with MFS. 

Studies have shown that beta-blockers reduce the rate of aortic growth, but these effects have been small. Recent animal studies demonstrated that treatment with losartan normalized aortic root growth and architecture, preventing aortic aneurysms and premature death. Preliminary data from a small pilot trial suggest a potential benefit of losartan use in humans with MFS.

Faculty Conducting Translational Research in the Heart Institute

 

Faculty Focus of Research
Jeffery Molkentin, PhD Cardiac signaling and transcriptional mechanisms in disease
Transitional Research.