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New data published in the Journal Science Translational Medicine shows encouraging results in a worldwide clinical trial for patients diagnosed with the neuro-degenerative blinding eye disease; Leber Hereditary Optic Neuropathy (LHON.
The disease is an inherited type of vision loss estimated to affect 1 in 30,000 to 50,000 individuals. LHON most often afflicts individuals with rapid onset of blindness in the prime of their life – men and women in their teens or twenties. Males are about four to five times more likely than females to be influenced by the condition.
We are pleased that we’ve seen an improvement to this extent in our patients dealing with this devastating vision loss – especially those at such a young age. The data shows sustained visual improvement in both eyes over the 96-week follow-up period. That underscores the importance of this research and the continuing important scientific work to bring it to market.”
Robert C. Sergott, MD, Chief of Wills Eye Neuro-Ophthalmology Service, Wills Eye Hospital
REVERSE is a randomized, double-masked, sham-controlled, multi-center, phase III clinical trial that assessed the effectiveness of a single intravitreal injection of ND4 in areas with visual loss from LHON.
The gene therapy trial, which took place at seven centers worldwide including at Wills Eye Hospital in Philadelphia, involved treating patients over age 15 with LUMEVOQ® (Gensight Biologics, Paris, France) gene therapy for a mutation at the ND4 mitochondrial gene.
One eye was injected with the gene and the other with a sham injection.
So as to explain the unexpected bilateral improvement of visual function, this paper also reports the results of a parallel non-human primate study. After unilateral injection of the gene treatment, evidence of viral vector DNA was found in the anterior segment, retina, and optic nerve of the contralateral uninjected eye.
This supports”a plausible mechanistic explanation for the unexpected bilateral improvement in visual function seen in LHON subjects treated with a unilateral injection of the ND4 gene therapy vector” according to this study.
The patients’ other eye, which received a sham injection, experienced an average visual acuity advantage over baseline of +13 letters equal.
Mark L. Moster, MD, Principal Investigator of this REVERSE trial in Wills Eye, said”Most striking to me was an average greater than a 5-line improvement of visual acuity in the patients’ worst eyesight (nadir), which is well beyond the natural history of healing in patients with ND4 LHON. From a standpoint of the patients’ wellbeing, there were significant improvements on the well-established National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25). Many who entered the study legally blind were no longer legally blind at the end of the study.”
The gains were most impressive when comparing enhanced vision against the worst vision in patients’ experience throughout their condition. 81 percent of patients showed a clinically relevant recovery (CRR) from the worst point of vision loss in one or both eyes.
One of these patients in the study was 27-year older Haley Hampton of Philadelphia. Ms. Hampton was diagnosed at age 22 at Wills’s Eye after undergoing an unexplained, sudden, and rapidly deteriorating vision.
“All of a sudden, my vision became very fuzzy. It prevented me from reading, getting around, and working in my career area as a fashion stylist. I had difficulty seeing colors and several essential details of close-up work,” she said.
“Once enrolled in the trial and I got the injection, I noticed a visual improvement about two to three weeks afterwards. I was reading more letters each time I had a follow-up appointment with the physicians at Wills,” said Hampton.
Julia A. Haller, MD, Wills Eye Ophthalmologist-in-Chief, a retina specialist, and a treating doctor in the Wills Eye trial, said”How inspiring and exciting to yet again see Ophthalmology at the tip of the spear in pioneering medical invention. This landmark study provides hope for the brave young people battling this blinding condition. Gene therapy is no longer a futuristic dream — it does and can provide help and hope in the dynamically evolving field of vision recovery.”
“It’s amazing,” added Hampton. “I wasn’t expecting anything, but this remedy is working out for me and, for that I am truly blessed,” she said.
As of December 2020, the clinical trial at Wills Eye is complete and information has been submitted for FDA approval consideration and availability.
Wills Eye Hospital
