Friedreich's ataxia: clinical pilot trial with recombinant human erythropoietin

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Abstract
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. Ann Neurol 2007