Etravirine (Brand name: Intelence) – Dr. Roberto Testi and his team at the University of Rome and Fratagene recently reported the results of a drug screening study. They developed a cell-based assay to conduct high-throughput screening of 853 FDA-approved drugs in search of drugs that could increase frataxin protein levels in the assay. Among the potentially interesting drugs identified as apparently able to increase frataxin protein levels in the cell-based assay, the team focused on Etravirine for the next step. The team then introduced the drug to some fibroblasts (skin cells) and lymphoblasts (blood cells) derived from F A patients.
In those cells, it seemed able to increase frataxin protein levels to some extent and restore the activity of an iron-sulfur-cluster-containing enzyme, aconitase, that is decreased with frataxin deficiency. Etravirine appeared also able to protect F A patient-derived cells from oxidative stress. Initial studies to clarify the mechanism of action, also indicated that Etravirine might be able to increase the translation of frataxin messenger RNA (mRNA) into frataxin protein. The team reported to the F A patient community at a recent meeting but has not yet published the fact that it then gave the drug orally to one of the FA mouse models. In those mice, the drug appeared to increase frataxin protein levels to some extent in skeletal muscle and heart.