An Old Drug May Offer a New Treatment for Young Heart Patients

A University of Cincinnati heart researcher hopes an old drug may offer a new cure for treating children born with only one functioning ventricle in the heart.

Jack Rubinstein, MD, associate professor in the Division of Cardiovascular Health and Disease and UC Health cardiologist, has received a $154,000 grant from the American Heart Association (AHA) and The Children’s Heart Foundation (CHF) to study how the use of Probenecid, a medication used to treat gout, might improve cardiac function and symptoms in children and young adults with a single functioning pumping chamber in the heart.

Most babies are born with two separate pumping chambers called ventricles, though some are born with a single ventricle, explains Rubinstein. These babies frequently undergo several surgeries that improve the flow of blood to the lungs and the rest of the body resulting in a configuration known commonly as "Fontan circulation.” 

Rubinstein, a member of the UC Heart, Lung and Vascular Institute, is working with co-investigators, Gruschen Veldtman, MD, professor of pediatrics at UC and director of adult congenital heart disease at Cincinnati Children’s Hospital Medical Center, and Jessica Woo, PhD, associate professor of pediatrics at UC and a Cincinnati Children’s researcher.

The research team will run a clinical trial that is randomized, placebo-controlled, double-blinded cross over designed to learn how Probenecid may help children with single ventricles. They hope to start enrolling in the trial by late summer. Probenecid has been in clinical use for decades starting in World War II to extend limited supplies of penicillin and subsequently to treat gout and gouty arthritis.

"We will measure how they feel and how their heart function works before and after receiving Probenecid,” explains Rubinstein. "They will then have another month of the opposite treatment of either the placebo or the Probenecid, depending on what they had the first time.”

"Our participants will not know, neither will their parents as to what they received first and then later for us to determine without bias if the drug worked,” says Rubinstein.

The study has been under development for the past two years to ensure safety for everyone involved.

"We had to be very careful to make sure the study was well-designed and safe. If positive this could really change the management of kids with this disease,” says Rubinstein.

The award Rubinstein received is one of five offered nationally during this round of grants totaling $561,798 in funding from the AHA and CHF. 

"We are honored and excited to continue our research funding partnership with AHA,” explains William Foley, executive director of The Children’s Heart Foundation. "Through this collaboration and our ongoing commitment to research focused on congenital heart defects, we strive to make a lasting impact in the lives of those with CHD. This $550,000 of new research will help bring innovative solutions to CHD survival rates and care”

The American Heart Association has been a past supporter of Rubinstein’s laboratory.

"We are committed to working with The Children’s Heart Foundation to fund these important research projects, to secure healthier futures for babies born with CHD,” says Nancy Brown, CEO of the American Heart Association. "Innovations in research drive our work to improve the health of all Americans.”