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LHON EPI-743 treatment trials
IFOND scientific members are involved in new LHON treatment trials using the Vitamin E metabolite substance,
Alpha-tocotrienol-quinone or Vatiquinone, coded EPI-743,
a potent antioxidant.
A review published in 2017 summarises progress in the therapeutic trials and uses of EPI-743.
An update of the ongoing open label compassionate use study published as an ARVO presentation abstract in June 2015 is here. The abstract concludes: "... EPI-743 treatment improved vision by these criteria in 56% of treated eyes. Visual improvement was noted up to 4 years following initiation of therapy suggesting that there is a large therapeutic window for recovery with EPI-743. There were no significant drug-related adverse events or clinical laboratory abnormalities. EPI-743 is a safe and effective treatment for LHON for most patients..."
Clinical Trials in Mitochondrial Disease: An Update on EPI-743 and RP103 Gregory M. Enns MB, ChB, Bruce H. Cohen, MD. Journal of Inborn Errors of Metabolism and Screening. Sage Journals First Published October 6, 2017 Research Article. doi: 10.1177/2326409817733013 https://doi.org/10.1177/2326409817733013
α-Tocotrienol quinone modulates oxidative stress response and the biochemistry of aging William D Shrader 1 , Akiko Amagata, Adam Barnes, Gregory M Enns, Andrew Hinman, Orion Jankowski, Viktoria Kheifets, Ryo Komatsuzaki, Edgar Lee, Paul Mollard, Katsuyuki Murase, Alfredo A Sadun, Martin Thoolen, Kieron Wesson, Guy Miller. Bioorg Med Chem Lett. 2011 Jun 15;21(12):3693-8. doi: 10.1016/j.bmcl.2011.04.085. Epub 2011 Apr 24. PMID: 21600768 http://doi.org/10.1016/j.bmcl.2011.04.085
Effect of EPI-743 on the Clinical Course of the Mitochondrial Disease Leber Hereditary Optic Neuropathy Alfredo A. Sadun, MD, PhD; Carlos Filipe Chicani, MD, PhD; Fred N. Ross-Cisneros, BA; et al Piero Barboni, MD; Martin Thoolen, PhD; William D. Shrader, PhD; Kenneth Kubis, MD; Valerio Carelli, MD, PhD; Guy Miller, MD, PhD Author Affiliations Arch Neurol. 2012;69(3):331-338. doi:10.1001/archneurol.2011.2972 https://doi.org/10.1001/archneurol.2011.2972
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