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Gene therapy, stem cells show promise for AMD in early studies
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NEW ORLEANS — Gene vector therapy and embryonic stem cell transplantation may one day be effective therapies for rehabilitating the retinas of patients with age-related macular degeneration, according to speakers here. Both therapies are under investigation for promoting survival of photoreceptors and/or retinal pigment epithelial cells during choroidal neovascularization (CNV), said two presenters.
“Gene vector therapy was well tolerated, with few adverse reactions, in patients with severe AMD,” Eric R. Holtz, MD, told attendees of the retina subspecialty day at the American Academy of Ophthalmology meeting.
He presented outcomes of a compassionate use study of Genvec, a gene product containing an adenovector and pigment epithelium-derived factor (AdPEDF). In animal studies, Genvec was found to inhibit progression of CNV and promote survival of photoreceptors and RPE cells. Further study is needed to prove the efficacy of Genvec in the human population, he said.
Another investigation, presented by Kourous A. Rezaei, MD, found that stem cells implanted into the retinas of primates and rats effectively convert to retinal pigment epithelium (RPE) cells.
“After transplantation into the subretinal space, [the stem cells] promoted the survival of photoreceptors in dystrophic rats,” Dr. Rezaei said. Studies are under way to convert human embryonic stem cells into RPE cells for transplantation for the treatment of AMD, he said.