Role of the Thromboxane-Prostanoid Receptor in FRDA Cardiomyopathy

This project will test if the drug ifetroban, currently in clinical trials for other diseases that affect the heart, improves the survival of a mouse model of FA. The long-term goal of this project is to improve the heart function in people who have Friedreich ataxia. It is known that the heart in this disease develops significant thickening and has extensive fibrosis that develops over time. This markedly decreases the function of the heart and leads to early death. This project will determine if a drug called ifetroban will stop or decrease fibrosis in the heart of the Friedreich ataxia mouse, thus extending its life. This drug is targeted to a specific receptor called the thromboxane-prostanoid receptor, which, in the heart, stimulates the production of fibrosis. By studying the effect of ifetroban in the FA mouse model, Dr. Payne hopes to generate enough data to apply for a grant from NIH or FDA to support a clinical trial of ifetroban in FA patients.