There's only one U.S. Food and Drug Administration-approved therapy for an inherited retinal disease, and dozens of retinitis pigmentosa (RP) genes for which no therapy is available.
With a new three-year, $1.5 million grant from the Foundation Fighting Blindness, Shigemi Matsuyama, an associate professor of ophthalmology and visual sciences at the Case Western Reserve University School of Medicine, will test a possible breakthrough drug that can be taken by mouth-;one that may address many RP disease manifestations, regardless of the underlying genetic mutation. We believe it can serve as the basis of an oral medicine to prevent blindness in RP patients, regardless of the underlying gene mutations.
" Shigemi Matsuyama, associate professor of ophthalmology and visual sciences, Case Western Reserve University School of Medicine RP causes blindness when cells in the retina that respond to light-;called photoreceptors-;die. In a previous foundation-funded drug discovery program from 2017-20, Matsuyama and his research team developed a series of novel orally-active cell-death inhibitors-;known as Cytoprotective Small Compounds (CPSCs)-;which work by blocking the activation of Bax, a protein that contributes to cell death. Their lead compound prevented retinal cell death and vision loss in four mouse models of inherited retinal disease.
The proposed studies, which include toxicology evaluation and drug-formulation testing-;for both oral drugs and eye drops-;are aimed at advancing t.