International Foundation for Optic Nerve Disease

LHON viral vector treatment trials

IFOND scientific members, coordinating with colleagues in North America and Europe, are involved in human LHON 11778 viral vector treatment trials using technology pioneered by Dr. John Guy's team at Bascom Palmer Eye Institute at the University of Miami Miller School of Medicine in Miami Florida.

Preliminary human clinical safety and efficacy trial reports have so far shown only modest benefits in restoring central vision in LHON. The technique used in the studies is to inject only one eye with active agent in a double blinded fashion per individual trial subject. Interestingly both treated and non treated eyes have similar outcomes so far. Best results were obtained in patients with early disease.

In December 2024 lead by our scientific board member, Patrick Yu-Wai-Man, world leading Leber Hereditary Optic Neuropathy (LHON) researchers in the LHON Study Group including a majority of our scientific board have published [open access] a review of outcomes five years post single eye intraocular gene therapy in LHON. The Best Corrected Visual Acuity results suggest a lasting clinically significant therapeutic effect in both eyes in 41 of 62 participants (66%). Quality of life and adverse events were the other end points of the study. Both were favourable. An analysis and critique of the study is found in the accompanying editorial [paywalled].

See:

Yu-Wai-Man P, Newman NJ, Biousse V, et al. Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy. JAMA Ophthalmol. Published online December 19, 2024. doi:10.1001/jamaophthalmol.2024.5375

Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease However, FDA approval isn't expected soon due to quirk in earlier results

Zhang Y, Tian Z, Yuan J, Liu C, Liu HL, Ma SQ, Li B. The Progress of Gene Therapy for Leber's Optic Hereditary Neuropathy. Curr Gene Ther. 2017;17(4):320-326. doi: 10.2174/1566523218666171129204926. PMC5902861

Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)

www.gensight-biologics.com

Wan et al. Efficacy and Safety of rAAV2-ND4 Treatment for Leber’s Hereditary Optic Neuropathy. Sci Rep. 2016; 6: 21587. Also at http://www.nature.com/articles/srep21587

Newman NJ, Yu-Wai-Man P, Carelli V, et al. Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset. Ophthalmology. 2021 Jan. DOI: 10.1016/j.ophtha.2020.12.012.

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