For the clinician-scientists at Wilmer, seeing patients has two benefits: making a difference in people’s lives and creating a guide for lab research. “Because I treat patients in the clinic, I know the really pressing problems, the unmet needs that patients have,” says Peter Campochiaro, M.D., the George S. and Dolores Doré Eccles Professor of Ophthalmology and Neuroscience. “That has helped me to focus my laboratory work.”

One of those needs is controlling a protein called vascular endothelial growth factor (VEGF), which plays a critical role in causing a number of retinal diseases, from neovascular age-related macular degeneration to diabetic macular edema. Since 2006, however, a doctor has been able to meet this need with anti-VEGF injections, which contain antibodies and proteins that bind to VEGF, in the vitreous of a patient’s eye. “These proteins act like a sponge and soak up the VEGF,” says Campochiaro.

While the anti-VEGF treatments are very effective compared with previous options, they are not without drawbacks. For many patients, especially those with macular degeneration, the therapy is usually effective for only four to six weeks at a time; thus, such patients must come in for shots frequently. Campochiaro is striving to eliminate this burden.

Rather than injecting the anti-VEGF directly into the eye, his strategy is to inject something that will produce the antiVEGF within the cells of the eye—a virus.

“Viruses are very good at getting into cells,” says Campochiaro. “We engineer the viruses so that we take out all of their inside machinery but we use the outside called the capsid, which is able to penetrate cells. And we replace the bad viral genes with good genes that produce the therapeutic protein we now continually inject in the eye.”

The therapy, which involves a 30-minute procedure, is now in a phase I/II clinical trial to test for both safety and efficacy. “We have a lot of indications that this treatment will produce the protein at a good level, for a very prolonged period of time,” says Campochiaro—which could be life-changing for patients. “We could be able to reduce or eliminate injections in the majority of patients.”

Campochiaro has worked on this solution as it has progressed from the lab to the clinical trial and has enjoyed the discoveries along the way. “When you find out something new that you can add to the body of scientific knowledge, it’s really exciting,” he says. “And then when you see how those new pieces of knowledge can add up to something that can help patients, then it really becomes rewarding.”