New Grant Awarded to Mark Payne, MD – Could a potential treatment for Duchenne Muscular Dystrophy (DMD) also treat the cardiac disease in Friedreich's ataxia (FA)?

Could a potential treatment for Duchenne Muscular Dystrophy (DMD) also treat the cardiac disease in Friedreich's ataxia (FA)?

FA can often give rise to cardiac complications and a reduction in heart function. When the heart has severe fibrosis (scar tissue), cardiac function decreases, leading to heart failure and early death.

In patients with DMD, the heart also develops fibrosis. An investigational drug called Ifetroban has been shown to improve cardiac function and increase lifespan in DMD mice. Ifetroban is currently in clinical trials for DMD.

We are pleased to award a grant to Dr. Mark Payne at Indiana University School of Medicine to study the potential use of Ifetroban in FA. Dr. Payne’s lab will first test whether Ifetroban decreases fibrosis in the hearts of an FA mouse model. They will then test whether Ifetroban increases the lifespan of these mice.

This work will study the mechanism of fibrosis in the FA heart and test a potential treatment for it. If successful, this will provide the preliminary data needed to support a clinical trial in humans with FA-related cardiac disease.