An open-label trial in Friedreich ataxia suggests clinical benefit with high-dose resveratrol, without effect on frataxin levels

E.M. Yiu, G. Tai, R.E. Peverill, K.J. Lee, Kevin Croft, Trevor Mori, B. Scheiber-Mojdehkar, B. Sturm, M. Praschberger, A.P. Vogel, G. Rance, S.E.M. Stephenson, J.P. Sarsero, C. Stockley, C.Y.J. Lee, A. Churchyard, M.V. Evans-Galea, M.M. Ryan, P.J. Lockhart, L.A. CorbenM.B. Delatycki

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© 2015, Springer-Verlag Berlin Heidelberg. Friedreich ataxia (FRDA) is due to a triplet repeat expansion in FXN, resulting in deficiency of the mitochondrial protein frataxin. Resveratrol is a naturally occurring polyphenol, identified to increase frataxin expression in cellular and mouse models of FRDA and has anti-oxidant properties. This open-label, non-randomized trial evaluated the effect of two different doses of resveratrol on peripheral blood mononuclear cell (PBMC) frataxin levels over a 12-week period in individuals with FRDA. Secondary outcome measures included PMBC FXN mRNA, oxidative stress markers, and clinical measures of disease severity. Safety and tolerability were studied. Twenty-four participants completed the study; 12 received low-dose resveratrol (1 g daily) and 12 high-dose resveratrol (5 g daily). PBMC frataxin levels did not change in either dosage group [low-dose group change: 0.08 pg/μg protein (95 % CI −0.05, 0.21, p = 0.21); high-dose group change: 0.03 pg/μg protein (95 % CI −0.10, 0.15, p = 0.62)]. Improvement in neurologic function was evident in the high-dose group [change in Friedreich Ataxia Rating Scale −3.4 points, 95 % CI (−6.6, −0.3), p = 0.036], but not the low-dose group. Significant improvements in audiologic and speech measures, and in the oxidative stress marker plasma F2-isoprostane were demonstrated in the high-dose group only. There were no improvements in cardiac measures or patient-reported outcome measures. No serious adverse events were recorded. Gastrointestinal side-effects were a common, dose-related adverse event. This open-label study shows no effect of resveratrol on frataxin levels in FRDA, but suggests that independent positive clinical and biologic effects of high-dose resveratrol may exist. Further assessment of efficacy is warranted in a randomized placebo-controlled trial.
Original languageEnglish
Pages (from-to)1344-1353
JournalJournal of Neurology
Issue number5
Publication statusPublished - 2015


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