Could a drug used to treat high cholesterol be repurposed to treat eye disorders?

The connection between the two isn’t as far-fetched as it sounds, and Qingguo Xu, Ph.D., an associate professor in the Department of Pharmaceutics at the School of Pharmacy, is on a path to making it work.

His lab has formulated a pharmaceutical using fenofibrate, an FDA-approved oral drug used to treat high cholesterol, to hopefully someday treat certain eye diseases.

Fenofibrate activates a protein in the body known as peroxisome proliferator-activated receptor alpha (PPAR-alpha), which Xu’s lab is studying for its ability to improve the body’s “good” cholesterol, decrease triglycerides and reduce inflammation. Those factors cause diabetic retinopathy and speed up macular degeneration, which can impair vision and even cause blindness.

The current treatment for those ocular conditions is a class of drugs, known as anti-VEGF, that slow growth of abnormal blood vessels in the eye. The drugs are injected into the numbed eye, typically once a month, by an ophthalmologist. While the medication can stabilize most patients’ vision, it improves eyesight in only one-third of them.

Xu’s fenofibrate formulation is also injected into the eye but releases the medication slowly over time. Patients would need only one or two injections a year. And, he said, activating PPAR-alpha is likely more effective than targeting VEGF, the substance that stimulates vessel formation in the eye.

“We’re repurposing a drug that has known safety, efficacy and is used routinely, and patients will be more compliant because they don’t have to get as many treatments over the course of a year,” Xu says.

He and his research partner, Jian-Xing Ma, M.D., Ph.D., the chair and a professor of biochemistry at Wake Forest University, were granted a patent on the formulation in June 2022 with the guidance of VCU TechTransfer and Ventures. Xu was one of six recipients of the fall 2022 round of TechTransfer and Ventures’ Commercialization Fund Awards.