September 2016
Volume 57, Issue 12
Open Access
ARVO Annual Meeting Abstract  |   September 2016
EPI-743 (Quinone) therapy for Leber’s Hereditary Optic Neuropathy: the Brazil experience.
Author Affiliations & Notes
  • Supanut Apinyawasisuk
    Ophthalmology, Doheny Eye Institute, Los Angeles, California, United States
  • Amitha Ganti
    Ophthalmology, University of Southern California, Los Angeles, California, United States
  • Rustum Karanjia
    Ophthalmology, Doheny Eye Institute, Los Angeles, California, United States
  • Edward R Chu
    Ophthalmology, University of Southern California, Los Angeles, California, United States
  • Tana Wagschal
    Visual Field Reading Center, University of Iowa, Coralville, Iowa, United States
  • Rubens Belfort
    Ophthalmology, Universidade Federal de Sao Paulo, Sao Paulo, Brazil
  • Milton N de Moraes-Filho
    Ophthalmology, Universidade Federal de Sao Paulo, Sao Paulo, Brazil
    Centro Universitario do Espirito Santo, Colatina, Brazil
  • Solange Rios Salomao
    Ophthalmology, Universidade Federal de Sao Paulo, Sao Paulo, Brazil
  • Guy Miller
    Edison Pharmaceuticals, Mountain View, California, United States
  • Alfredo A Sadun
    Ophthalmology, Doheny Eye Institute, Los Angeles, California, United States
  • Footnotes
    Commercial Relationships   Supanut Apinyawasisuk, None; Amitha Ganti, None; Rustum Karanjia, Edison Pharmaceuticals (F), Stealth Biotechnology (F); Edward Chu, edison Pharmaceuticals (F); Tana Wagschal, None; Rubens Belfort, None; Milton de Moraes-Filho, None; Solange Salomao, None; Guy Miller, Edison Pharmaceuticals (E); Alfredo Sadun, Edison Pharmaceuticals (F), Stealth Biotechnology (F)
  • Footnotes
    Support  None
Investigative Ophthalmology & Visual Science September 2016, Vol.57, 5084. doi:
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      Supanut Apinyawasisuk, Amitha Ganti, Rustum Karanjia, Edward R Chu, Tana Wagschal, Rubens Belfort, Milton N de Moraes-Filho, Solange Rios Salomao, Guy Miller, Alfredo A Sadun; EPI-743 (Quinone) therapy for Leber’s Hereditary Optic Neuropathy: the Brazil experience.. Invest. Ophthalmol. Vis. Sci. 2016;57(12):5084.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : To determine the degree of visual improvement, using markers of visual function, in a homogeneous Brazilian cohort of Leber’s Hereditary Optic Neuropathy (LHON) 11778 receiving up to 52 months of therapy with a novel quinone.

Methods : As part of an open label trial, six Brazilian, LHON patients experiencing significant visual loss were offered an experimental therapeutic, alpha-tocotrienol quinone (EPI-743). Two acute patients receiving EPI-743 during acute visual decline and four patients had chronic disease (>5 years) at the start of therapy. Snellen visual acuity (VA) in logMAR represented best correct vision. We utilized an algorithm developed by the University of Iowa Visual Field Reading Center that derives mean deviations (MD) from raw numerical data of STIM III and STIM V Humphrey Visual Fields (HVFs) to obtain participants’ visual field MDs. A change of +5 db was considered an improved HVF. Retinal nerve fiber layer (RNFL) thickness was measured using time (2007-2009) or spectral (2010-2015) domain Optical Coherence Tomography (OCT).

Results : The two patients who were treated at time of conversion showed continued bilateral progressive loss of vision for the first 6 months, consistent with the natural history of LHON. VA declined or stabilized in the first year of therapy in all patients. Due to issues with agent access, one acute and two chronic patients chose to withdraw from the study. The observed improvement in VA was stable for up to 52 months of EPI-743, though there was a small VA decline after the withdrawal of therapy in those patients who discontinued therapy. HVF improved (>5 dB) in one chronic and both acute patients, while three chronic patients remained stable. HVF remained stable after the withdrawal of therapy in those patients who chose to withdraw from the study. As expected, all eyes showed reductions in RNFL on OCT that eventually stabilized on therapy, not inconsistent from the natural history of the disease.

Conclusions : The visual acuity improvements begun in the first 12 months of EPI-743 described in our previous ARVO abstract, continued for 36-52 months of treatment; remarkably this included even in subjects who had converted over 5 years prior to the initiation of treatment.

This is an abstract that was submitted for the 2016 ARVO Annual Meeting, held in Seattle, Wash., May 1-5, 2016.

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