Leber hereditary optic neuropathy: new and emerging therapies.
Journal
Current opinion in ophthalmology
ISSN: 1531-7021
Titre abrégé: Curr Opin Ophthalmol
Pays: United States
ID NLM: 9011108
Informations de publication
Date de publication:
01 Nov 2022
01 Nov 2022
Historique:
pubmed:
7
9
2022
medline:
12
10
2022
entrez:
6
9
2022
Statut:
ppublish
Résumé
To review recent therapeutic advances in Leber hereditary optic neuropathy (LHON). Idebenone, a synthetic analog of ubiquinone (Coenzyme Q10) is an antioxidant and component of the mitochondrial electron transport chain. Since the initial approval of the drug in 2015 in Europe, recent trials have evaluated its role as prolonged treatment in LHON. Gene therapy has recently emerged as a promising alternative for the treatment of LHON. Among several investigations, RESCUE and REVERSE are two phase 3 clinical trials of gene therapy in patients with LHON in early stages. Results in these trials have shown a bilateral visual acuity improvement with unilateral intravitreal injections at 96 weeks and sustained visual improvement after 3 years of treatment. The most recent REFLECT phase 3 clinical trial in LHON has shown significant improvement of vision after bilateral intravitreal injections compared with the group that received unilateral injections. Historically, LHON has been considered an untreatable disease, but recent developments show that new pharmacological and gene therapy approaches may lead to visual recovery. Further studies are needed to support these data.
Identifiants
pubmed: 36066375
doi: 10.1097/ICU.0000000000000891
pii: 00055735-202211000-00016
doi:
Substances chimiques
Antioxidants
0
DNA, Mitochondrial
0
Ubiquinone
1339-63-5
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
574-578Informations de copyright
Copyright © 2022 Wolters Kluwer Health, Inc. All rights reserved.
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