Treatment of Leber's Hereditary Optic Neuropathy

Curr Pharm Des. 2017;23(4):624-628. doi: 10.2174/1381612823666170125164856.


Leber's hereditary optic neuropathy (LHON) is a rare mitochondrial disease of complex I of the respiratory chain. Patients typically present with subacute vision loss in one eye followed by the loss of vision in the second eye approximately 4-8 weeks later, ultimately leading to blindness. Therapeutic interventions have so far failed to prevent this vision loss; however, there has been recent interest in new managements for this prototypic mitochondrial disease. A review of the literature was performed and articles were identified using PubMed (the search terms LHON and treatment were used). These publications were paired to the clinical trials listed on the website. There are eighteen studies including 5 phase II/III clinical trials in LHON listed on the website and they vary from oral medication to eye drops and intravitreal injections. In this review, we discuss failed and active therapies and the future of clinical trials in LHON.

Keywords: LHON; Mitochondria; gene therapy; optic neuropathy; retinal ganglion cell; treatment.

Publication types

  • Review

MeSH terms

  • Humans
  • Ophthalmic Solutions / administration & dosage
  • Ophthalmic Solutions / therapeutic use*
  • Optic Atrophy, Hereditary, Leber / genetics
  • Optic Atrophy, Hereditary, Leber / metabolism
  • Optic Atrophy, Hereditary, Leber / therapy*
  • Reactive Oxygen Species / metabolism
  • Stem Cell Transplantation*
  • Stem Cells


  • Ophthalmic Solutions
  • Reactive Oxygen Species