Case Western Reserve University receives $1.5 million grant from Foundation Fighting Blindness to test possible new treatment for inherited retinal disease

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Case Western Receive University (CWRU) has received a 3-year, $1.5 million grant from the Fo0undation Fighting Blindness to research therapeutic options for inherited retinal disease (IRD).

Currently, there is a single FDA-approved therapy for an inherited retinal disease, and dozens of retinitis pigmentosa (RP) genes for which no therapy is available.

With the grand, Shigemi Matsuyama, PhD, an associate professor of ophthalmology and visual sciences at the Case Western Reserve University School of Medicine, will conduct research seeking a potential breakthrough drug that can be taken orally—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,” Matsuyama said in a news release.

The disease, and promising science

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 to 2020, 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.

According to the CWRU news release, the proposed studies, which include toxicology evaluation and drug-formulation testing—for both oral drugs and eye drops—are aimed at advancing the technology to FDA-regulated clinical trials.

“Our primary objective is bringing an CPSC-based therapeutic to RP patients who are suffering from the fear of blindness,” Matsuyama said in the news release.

Moreover, researchers noted that Bax-induced cell death is also a major cause of various neurodegenerative conditions, including glaucoma, ischemia-reperfusion injury, amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease.

“Therefore,” he concluded, “there is significant potential to expand the application of our Bax-inhibiting therapeutic for additional uses.”

Chad Jackson, senior director of preclinical translational research at Foundation Fighting Blindness, pointed out the foundation is pleased to support Matsuyama's research.

“Through this award, we continue our commitment to advancing scientific discoveries that have the potential to realize tangible solutions for those affected by inherited retinal diseases,” Jackson concluded.

Reference:

1. Case Western Reserve University receives $1.5M grant from Foundation Fighting Blindness to test possible new treatment for inherited retinal disease. EurekAlert! Accessed July 12, 2024. https://www.eurekalert.org/news-releases/1050251