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Clinical trial to test gene therapy for LHON
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A trial scheduled to start this month will analyze the efficacy of gene therapy for patients who suffer from Leber's hereditary optic neuropathy, according to a press release.
John Guy, MD, professor of ophthalmology and director of the ocular gene therapy laboratory at the Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine, is leading a multidisciplinary team for the 5-year study.
As detailed in the release, trial patients who experience vision loss as a result of LHON will receive an injection of a mitochondrial gene into the vitreous of the eye. The LHON clinical trial is among the first to use gene therapy to target a disease produced by a faulty gene in the mitochondria, according to the release.
“A wide range of other conditions, including aging, cancer, and Parkinson’s disease, are also caused by mutations in the mitochondria,” Guy said in the release. “This novel approach shows the vast potential for genetic therapy applications, while helping to address a significant cause of blindness.”
Guy developed a method to introduce a normal ND4 gene into retinal ganglion cells via a virus, according to the release. Preclinical testing has shown that the approach is safe, effective and could restore visual function.
“Other research studies have shown that LHON patients who have lost their vision still have some sensitivity to light,” Guy said in the release. “This indicates that if you can restore the functioning of those cells through gene therapy, those patients could see again.”
The clinical trial is being funded by a $6 million agreement from the National Eye Institute (NEI). Guy has conducted preclinical research since 2007 and has received grants from the National Institutes of Health and NEI totaling $6.1 million.