Friedreich's ataxia: clinical pilot trial with recombinant human erythropoietin.
To determine the role of recombinant human erythropoietin as a possible treatment option in Friedreich's ataxia, we performed an open-label clinical pilot study. Primary outcome measure was the change of frataxin levels at week 8 versus baseline. Twelve Friedreich's ataxia patients received 5,000 units recombinant human erythropoietin three times weekly subcutaneously. Frataxin levels were measured in isolated lymphocytes by enzyme-linked immunosorbent assay. In addition, urinary 8-hydroxydeoxyguanosine and serum peroxides, were measured. Treatment with recombinant human erythropoietin showed a persistent and significant increase in frataxin levels after 8 weeks (p < 0.01). All patients showed a reduction of oxidative stress markers.
Boesch
Sylvia
S
Department of Neurology, Innsbruck Medical University, Innsbruck, Austria.
Sturm
Brigitte
B
Hering
Sascha
S
Goldenberg
Hans
H
Poewe
Werner
W
Scheiber-Mojdehkar
Barbara
B
D000293:Adolescent; D000328:Adult; D004921:Erythropoietin / Q000627:therapeutic use*; D005260:Female; D005621:Friedreich Ataxia / Q000097:blood / Q000188:drug therapy*; D006801:Humans; D033862:Iron-Binding Proteins / Q000097:blood; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D011994:Recombinant Proteins; D000096923:Frataxin
D004921:Erythropoietin; D033862:Iron-Binding Proteins; D011994:Recombinant Proteins; D000096923:Frataxin