Increasing evidence suggests that lipid peroxidation, a damaging chain reaction driven by iron and oxidative stress, contributes to the development of cardiomyopathy in Friedreich’s ataxia. By damaging the fatty membranes that surround cells, lipid peroxidation can impair energy production and promote cell death, making it a promising target for therapeutic intervention.
This project will evaluate CBM-01, a novel drug designed to stop lipid peroxidation at its source. Researchers will test whether CBM-01 can improve heart function and survival in a mouse model of FA cardiomyopathy. The study will also examine whether combining CBM-01 with omaveloxolone provides additional benefit and will assess biomarkers to confirm that the drug is engaging its intended target.
If successful, this work could validate lipid peroxidation as a therapeutic target and support development of a new treatment strategy for cardiomyopathy. The findings may also help guide future clinical trials of CBM-01 in Friedreich’s ataxia.