Previous studies had shown that intervention while only a single eye is affected had little impact on spread of the condition to the second eye, “which was very disappointing,” said Dr. Newman answered by noting another surprise from the research. Rost also wondered if it would be possible to capture patients earlier in their disease process, in the hopes of countering degeneration before it becomes severe enough to impact vision. Newman, who is a professor of ophthalmology and neurology at Emory University, Atlanta.ĭr. 01), “which suggest that this effect is maintained,” said Dr. At 4 years, there was a difference of 16.5 Early Treatment of Diabetic Retinopathy Study (ETDRS) letters equivalent between treated patients and natural history controls ( P <. Newman responded that ongoing data from earlier studies are also encouraging regarding the long-term effect of the treatment.
The question is, are there early indications that this improvement in vision will have a lasting effect?”ĭr. Natalia Rost, MD, who chairs the AAN Science Committee, commented after the presentation: “We’re quite impressed with advances in gene therapy. Those treated with the drug had more ocular inflammation, as would also be expected, but all were easily treated with topical medications,” said Dr. There were no adverse events other than what we would expect from injecting eyes. “Those patients who had both eyes injected with the drug did better than in those who had one eye injected with drug and one eye injected with placebo, suggesting some sort of dose effect. Newman.ĭespite the problem with placebo, the results were encouraging. That’s something that we will forward,” said Dr. We do not have naive controls here that did not receive any injection at all in any eye. And that was a mistake, because it turns out there is a does appear to be second eye effects. “This was not a case-control study by person, it was by eye. Unfortunately, the phase 3 REFLECT study was designed before that process was understood. “This would imply some kind of transport within retrograde up the opposite optic nerve after crossing in the chiasm to the eye, but this is going to take a fair bit of work to know exactly how that happens,” said Dr.
This was noted in previous studies, called RESCUE and REVERSE, and follow-up studies in monkeys found viral vector in the unaffected eye 3-6 months after an injection. Newman also noted a surprise finding: Visual improvement also occurred in the placebo-control eye. Newman, MD, during a press conference held March 29 in advance of the 2022 annual meeting of the American Academy of Neurology.ĭr.
But for people whose vision is devastated by this disease, it certainly is a first step,” said Nancy J. “This is not hitting it out of the ballpark. The results in the treated eye were encouraging, though modest. The study protocol called for injection of the therapy into one eye and a placebo into the other, using the patient as his or own placebo control. Once synthesized, a mitochondria-targeting sequence facilitates transport of the protein to the mitochondria. Researchers believe that the injected viral vector gets taken up retinal ganglion cells, where the mutated gene interferes with vision. The condition often starts with blindness in one eye, accompanied or followed shortly by blindness in the second eye. LHON is a rare, maternally inherited mitochondrial mutation that can cause blindness, most commonly in young men, though it does not happen in all individuals with the mutation. The therapy, delivered by intravitreal injection, uses an adeno-associated virus vector to deliver a corrected copy of the mutated ND4 mitochondrial gene.
MODEST EYES TRIAL
SEATTLE – The latest data from a phase 3 clinical trial shows that gene therapy can counter visual degeneration associated with Leber hereditary optic neuropathy (LHON).
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