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.
Note: the posting or linking of research, news, information or forum articles on this site does not mean that any proposed treatment methods are accepted or safe. Material posted here is FOR INFORMATION ONLY. The information is NOT COMPREHENSIVE and NOT NECESSARILY CURRENT. INFORMATION CONTAINED THEREIN IS NOT AN ENDORSEMENT BY IFOND. If you seek treatment or preventative guidance, discuss this with your personal health professional.
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