Paediatrics (RCH) - Research Publications

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End date: 2017 × Author: Corben, Louise × Author: Ryan, Monique ×

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    An open-label trial in Friedreich ataxia suggests clinical benefit with high-dose resveratrol, without effect on frataxin levels
    Yiu, EM ; Tai, G ; Peverill, RE ; Lee, KJ ; Croft, KD ; Mori, TA ; Scheiber-Mojdehkar, B ; Sturm, B ; Praschberger, M ; Vogel, AP ; Rance, G ; Stephenson, SEM ; Sarsero, JP ; Stockley, C ; Lee, C-YJ ; Churchyard, A ; Evans-Galea, MV ; Ryan, MM ; Lockhart, PJ ; Corben, LA ; Delatycki, MB (SPRINGER HEIDELBERG, 2015-05)
    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.
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    AN OPEN LABEL CLINICAL PILOT STUDY OF RESVERATROL AS A TREATMENT FOR FRIEDREICH ATAXIA
    Yiu, EM ; Tai, G ; Peverill, R ; Lee, K ; Croft, K ; Mori, T ; Scheiber-Mojdehkar, B ; Sturm, B ; Praschberger, M ; Vogel, A ; Rance, G ; Stephenson, S ; Lockhart, P ; Sarsero, J ; Stockley, C ; Churchyard, A ; Evans-Galea, M ; Ryan, MM ; Corben, L ; Delatycki, M (WILEY-BLACKWELL, 2013-06)