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International Foundation for Optic Nerve Disease

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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

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

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

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The information contained on this website should not be considered medical guidance or professional advice. IFOND is not responsible for errors or omissions in information provided on this site or actions resulting from its use. IFOND does not publish all information from all available sources on optic nerve disease. IFOND is not responsible for the validity of the studies or reviews nor is it an advocate of studies or reviews mentioned on or linked from the IFOND web site. IFOND does not endorse or recommend participation in any particular clinical trial or treatment protocol which may be mentioned on this site. Direct any questions concerning your personal health to your appropriate health care professional.