CASE REPORT article
Front. Med.
Sec. Ophthalmology
Volume 12 - 2025 | doi: 10.3389/fmed.2025.1609941
This article is part of the Research TopicOptic Neuropathies: Etiologies, Diagnosis, and TreatmentsView all 9 articles
Metformin May Alter the Course of Leber's Hereditary Optic Neuropathy (LHON): A Case Report
Provisionally accepted
Shenoda Yousef Azmy Abd Elmaseh1,2*
Danielle Gauthier1,2
Maryam Golmohammadi1,2
Nutsa Pargalava2
Valerio Carelli3
Alfredo A. Sadun1,2- 1University of California, Los Angeles, Los Angeles, United States
- 2UCLA Stein Eye Institute, Los Angeles, California, United States
- 3Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Emilia-Romagna, Italy
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Leber's Hereditary Optic Neuropathy (LHON) is a rare inherited mitochondrial disease due to mtDNA variants exclusively transmitted along the maternal line. The disease predominantly affects young males and presents with progressive bilateral vision loss. Idebenone is a well-studied drug that modestly improves mitochondrial function and visual acuity in many LHON patients. In the present paper, we report a 48-year-old woman affected with LHON (m.11778G>A/MT-ND4) and type 2 diabetes mellitus who experienced visual field improvement following metformin treatment after 17.5 months of progressive vision loss unresponsive to idebenone, NAD+, and hormone replacement therapy. Our findings are intriguing for LHON management but require more investigations, particularly on the molecular effect of metformin on the mitochondria of LHON patients.
Keywords: Metformin, LHON, idebenone, NAD+, Vision Loss, Mitochondrial dysfunction
Received: 11 Apr 2025; Accepted: 28 Jul 2025.
Copyright: © 2025 Abd Elmaseh, Gauthier, Golmohammadi, Pargalava, Carelli and Sadun. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Shenoda Yousef Azmy Abd Elmaseh, University of California, Los Angeles, Los Angeles, United States
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