Protein May Increase Frataxin in Friedreich’s Ataxia

The protein erythropoietin, known mostly for its ability to boost red blood cell production, has been found to have another, previously unsuspected action. It increases levels of the protein frataxin, which is deficient in people with Friedreich’s ataxia (FA), and might provide a direction for future drug development for that disease. Erythropoietin is now used to treat patients with kidney failure who are undergoing dialysis.

Barbara Scheiber-Mojdehkar, a chemist and MDA grantee at the Medical University of Vienna (Austria), headed a research team that administered erythropoietin to white blood cells taken from people with FA, and to cardiac muscle cells, connective tissue cells and nerve cells obtained from other sources. In all cases, increased frataxin levels were seen.

The study’s authors, who published their results in the November 2005 issue of the European Journal of Clinical Investigation, write that there is now a “scientific basis for examining the effectiveness of this agent for the treatment of Friedreich’s ataxia patients.”