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Antioxidants and other pharmacological treatments for Friedreich ataxia
Cochrane Reviews, 10/09/09

Kearney M et al. – Recent studies have suggested that a synthetic antioxidant, idebenone, may help the most frequent heart abnormality, enlargement of the left ventricle. Antioxidants occur naturally in foods but do not reach a level that would be considered necessary to alter the progress of Friedreich ataxia...A review of the medical literature revealed that there was only one small randomised controlled trial (RCT) with 29 participants which used idebenone for a sufficient period, 12 months. The included RCT showed that idebenone did not help the ataxia but did have a significant effect on the heart abnormality. No research on clinical relevance of this heart change has been done. Other pharmacological treatments being considered for Friedreich ataxia include deferiprone, erythropoietin, pioglitazone and histone deacetylase (HDAC) inhibitors but no RCTs have been completed using these treatments (www.curefa.org). A collaborative project using gene therapy in Friedreich ataxia mouse models and human brain cells is also in progress.

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