A recent study has found that an anti-viral drug, currently in use as a therapy for HIV may be a potential future candidate for Friedreich’s ataxia (FA) therapy. FA is caused by mutation of the frataxin gene, leading to decreased frataxin expression, mitochondrial dysfunction and oxidative stress. Since frataxin levels influence disease severity, the main goal of therapy for FA is to try and increase frataxin levels.

The study searched a library of drugs which had already been approved by the food and drug administration (FDA) to determine which ones might be able to increase frataxin levels. They found that etravirine can lead to a significant increase in frataxin levels in cells derived from FA patients, which was comparable to the levels in cells unaffected by FA.

The researchers commented: ‘Considering its excellent safety profile along with its ability to increase frataxin levels and correct some of the disease‐related defects, etravirine represents a promising candidate as a therapeutic for Friedreich's ataxia.’

For more information view the study abstract here.

Posted on 25/01/2019