Erythropoietin in Friedreich’s ataxia (FA)

Frataxin production may be stimulated through the use of erythropoietin, a naturally occurring protein. In 2010, an Austrian research team reported results of a small “proof-of-concept” trial in which 12 people with FA received erythropoietin injections three times a week for eight weeks. Eight of the 10 trial participants showed stable increases in frataxin levels, with individual increases ranging from 15 to 63 percent. Ataxia severity showed a 6 percent improvement in study participants. Carbamylated erythropoietin, a derivative of erythropoietin, has been shown to increase frataxin levels in FA-affected cells without the unwanted added effect of stimulating blood-cell production, as erythropoietin does, making it a potential alternative for drug development for FA.