SCHEIE VISION | ANNUAL REPORT 2020
RESEARCHERS DISCOVER TWO DRUGS THAT CAN PROTECT AGAINST GLAUCOMA IN PRECLINICAL MODELS By Kristen Mulvihill
Recent studies led by Qi N. Cui, MD, PhD and Joshua Dunaief, MD, PhD discovered two drugs can protect retinal ganglion cells (RGCs) in a mouse model of glaucoma. Glaucoma is an eye disease that slowly damages the optic nerve and RGCs. It is the leading cause of irreversible blindness worldwide, predominantly affecting individuals over the age of 60. By 2040, approximately 112 million people around the globe are predicted to be living with glaucoma. This neurodegenerative disease can cause permanent loss of
the visual field, usually due to high pressure inside the eye, or intraocular pressure (IOP). There are limited therapeutic mechanisms to effectively slow disease progression; all available treatments reduce IOP. However, glaucoma can continue to progress even in patients who achieve normal IOP levels after treatment, so it is necessary to investigate novel glaucoma therapies. “All available treatments for glaucoma target IOP control, which is not sufficient to prevent vision loss in a significant number of patients,� explained Dr. Cui. The teams of Dr. Cui, Assistant Professor of Ophthalmology, and Dr. Dunaief, Adele Niessen Professor of Ophthalmology, sought to examine new treatments with the potential to target other risk factors in glaucoma. They found that the drug deferiprone (DFP) and the compound NLY01 can protect RGCs in a mouse model of glaucoma. DFP is an orally-administered iron chelator approved by the FDA to treat patients with iron overload. The drug has been used to treat mouse models of macular degeneration and retinal degeneration, both of which are associated with oxidative stress. Oxidative stress, often produced by excess iron, likely plays a role in the development of glaucoma. To test whether DFP could be protective in glaucoma, the team used microbead injections to induce elevated IOP in a group of mice. The researchers found that administering DFP was protective against RGC and optic nerve loss. These results, published in Experimental Eye Research, suggest
Dr. Qi Cui.
Dr. Joshua Dunaief presenting a lecture at the 2019 Macula Society Meeting.
