Written by Whitney L.J. Howell

It’s a real-life idea-sketched-on-a-cocktail-napkin story – only with an academic twist. A chance meeting between two researchers from different departments at Duke University opened the door for the greatest advancement in treating glaucoma in nearly half a century.

David Epstein, M.D., the most recent chair of Duke’s ophthalmology department, spent his research career searching for the elusive cure to this disease that causes irreversible vision loss. Over time, glaucoma – the second leading cause of blindness worldwide – increases the pressure within the eye, damaging the optic nerve.

Traditionally, doctors have treated glaucoma with eye drops, but Epstein wanted to find a better solution for relieving symptoms. As it happened, discovering the right chemical combination wasn’t the sticking point. Epstein identified an existing diuretic used in patients with kidney disease that could also effectively reduce eye pressure. However, the medication only worked when injected directly into the eye. The stumbling block was getting the drug to its target more easily.

And, jumping that hurdle led to the launch of the start-up company Aerie Pharmaceuticals. When Epstein met Duke chemistry professor Eric Toone, Ph.D., it was the chemist’s knack for translating bench-side discoveries into clinical medications that led to success.

“[Eric] told me to draw a picture of the structure, and he said, ‘This is a piece of cake,'” Epstein said. “Basically, we started a collaboration of making compounds that might penetrate the eye. That really was the origin of it.”

Together, they developed once-daily eye drops called AR-13324 that actively prompt fluid to drain from the eye. The medication, a Rho Kinase (ROCK) inhibitor scheduled for two Phase 3 clinical trials in mid-2014, induces fluid to leave the eye and controls the amount coming in. ROCK is an enzyme that regulates two proteins – actin and myosin – that control cellular contraction, as well as promotes the production of extracellular matrix proteins that hold cells in place. When used correctly ROCK inhibitors block cell contraction and reduce the extracellular matrix, making it easier for fluid to leave the eye and relieve eye pressure.

In addition, ROCK inhibitors prevent the production of norepinephrine transporter (NET) protein, effectively diminishing the amount of eye fluid produced.

Based on anticipated positive clinical trial results, Epstein said Aerie will file a new drug application with the Food & Drug Administration in mid-2016.

Although the drug discovery and development were integral to creating Aerie Pharmaceuticals, the launch could never have been possible outside the Duke environment, he said. Only at Duke were he and Toone able to bring together the knowledge of medical faculty, business and engineering students, as well as undergraduates, to create a truly entrepreneurial venture.

“It’s likely this collaboration couldn’t have happened anywhere else. Duke is so collegial,” he said. “It’s the home of the most inquisitive clinicians and faculty. It’s the most intellectual and idealistic place I’ve ever been – a primordial soup for innovations.”

In that vein, he laid the groundwork for a program called Duke Biosolutions. This initiative would bring together faculty and students from all disciplines in pursuit of discoveries that will positively impact human health.

Sadly, Epstein will not see his glaucoma discovery or his faculty-student project come to fruition. The 22-year ophthalmology chair died suddenly on March 4. But his strong legacy at Duke will live on. His innovative and collaborative spirit will remain strong among the cadre of researchers with whom he worked throughout the University.

To learn more about Aerie Pharmaceuticals, visit its website.

To read about more Duke Entrepreneurs, click here.

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