Identifying candidate drugs in mitochondrial cardiomyopathies: From mouse to human. 2021-2025, AFM-Strategic Project, Partner Véronique Paquis-Flucklinger.

The MITODRUG project aims to understand the mechanisms of action and in vivo proof of concept for a selection of 5 FDA-approved drug candidates identified by our consortium (researchers ranging from basic science to the clinic). Cardiac damage is present in 80% of adults with mitochondrial diseases. The experimental strategy will combine both mouse models and iPSC-derived cardiomyocytes, targeting 3 pathologies associated with mitochondrial cardiomyopathies (Barth’s syndrome, CHCHD10 and complex I-associated diseases). We will pursue the analysis of these drugs and test their effectiveness and mode of action especially targeting cardiac cells. This study will open new treatment avenues for mitochondrial disorders and paved the way for future clinical trials.