Etravirine as a potential therapeutic for Friedreich's ataxia
Ataxia UK
University of Rome Tor Vergata
Friedreich’s ataxia is the most common inherited form of ataxia and is caused by a mutation in the frataxin gene, which decreases the production of frataxin protein. This group recently showed that etravirine, an anti-retroviral drug currently in use as a therapeutic for HIV-positive patients, is able to upregulate frataxin in different cell types derived from FA patients. Frataxin upregulation induced by etravirine could be therapeutically relevant, as it results in increased aconitase activity and protection from oxidative stress in cells derived from patients (Alfedi et al., 2019). In this project, they plan to test different etravirine analogues for their ability to increase frataxin levels in cells, with the aim to define the pharmacophore portion of the molecule that confers frataxin up-regulating properties. This could potentially lead to the identification of already existing and approved etravirine analogues with better efficacy and potency and inspire the design of new derivatives.
5.1 Pharmaceuticals