TRPV2 agonism for improved cardiac function in patients with single ventricle physiology

Doctor's Name: 
Jack Rubinstein
Hospital/Institution: 
University of Cincinnati

Collaboratively awarded through the CHF and AHA Congenital Heart Defect Research Awards

(Total Grant Amount $154,000; CHF portion = $83,160)

Children with single ventricle physiology are now able to live longer due to improved surgical techniques. Unfortunately, a large percentage will develop heart failure that will lead to an early death. Our laboratory has discovered that an old and safe drug (Probenecid) targets a recently discovered receptor in the heart and improves cardiac function in animal models and in adults. This study will test the effects of this drug in children.

We are seeking to answer the question, does Probenecid therapy improve cardiac function and symptoms in children and young adults with single ventricle physiology? We will study the effect in patients with single right and single left ventricles. We will test the drug in a double blind, placebo control, crossover design fashion, and will measure heart function directly via echocardiography and indirectly via exercise testing.

Improving the outcome of patients with single ventricle physiology will not only have a huge impact on their lives, but may also lead to the use of this and related medications in children and adults with cardiomyopathies from other etiologies.

Award Date 1: 
2017
Award Amount 1: 
$83,160