Highlights in Vision Research: BrightFocus Reports from ARVO

And They’re Off

ORLANDO, FL.—How fitting that the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) began on the same day as the Kentucky Derby. With some 3,300 MDs and 3,200 PhDs in attendance, plus more than 1,000 MD-PhD scientists and several thousands of additional researchers and exhibitors, this  thoroughbred  gathering represents one of the world’s best marriages of clinical expertise with advanced research directed towards  preventing, treating, and curing  vision diseases.

ARVO’s reach is global—about 45 percent of  registrants have traveled here from outside the U.S.—in fact, from 75 other countries.

BrightFocus researchers are prominent here and their contributions to vision science are being shared in dozens of presentations. Many of them are being honored with awards. More details to be provided.

And more research is being planned! Everywhere one looks in this vast Orlando convention hall, there are clutches of scientists sharing their discoveries and ideas. This is definitely where the action is.

It’s a different kind of race from the Kentucky Derby, bu t it is a race nonetheless, and just being here makes one sense the urgency behind translating research results into clinically useful therapies and potential cures for glaucoma and macular degeneration.

So they’re off!  More to be shared ahead…

BrightFocus–Funded Research Designated 'Hot Topic' at ARVO

A BrightFocus grantee, University of Oklahoma researcher Michael Elliott, PhD, gave a poster presentation that was picked as a "hot topic" at the ARVO meeting. Of literally thousands of presentations given here, only three percent achieve that distinction.

Last year, Elliott received a National Glaucoma Research grant to investigate polymorphisms in the caveolin-1 gene (CAV-1) gene and its relationship to pressure regulation as a possible treatment for open-angle glaucoma.

For glaucoma, “while there are medications and surgeries that are effective, all of them target other aspects of pressure regulation, not conventional pathways,” Elliott said. Yet that’s where 80 percent of outflow takes place.

He’s found that the same gene that’s associated with glaucoma in humans, when manipulated in mice, results in ocular pressure changes that are, at least in part, mediated by aqueous humor outflow through conventional pathways. If his work in mice can be translated to humans, it could represent a novel therapeutic target.

"The 'holy grail’ of glaucoma medications would be to find something that targets outflow,” he said. “The CAV-1 gene is a candidate in that it is somehow involved in the mechanical ability of eye tissue to respond to increased pressure.”

New to Glaucoma Research

Elliott is actually a retinal research specialist who moved over to the glaucoma side to pursue this topic.

“It hit me like a sledgehammer,” he said. “At the time, I was looking at a different gene [related to retinal vasculature] and I got an email from a friend about a publication describing [CAV-1’s] association with glaucoma. Before then, I hadn’t even thought about it.”

In a truly global fashion, he arranged to collaborate with Dan Stamer, PhD, at Duke University and Dr. Ernst Tamm at the University of Regensburg (Germany). Stamer is lending his expertise at measuring outflow facility to corroborate results, and Tamm, an outflow anatomy expert, is imaging and characterizing the response to pressure in the caveola tissue, which hasn’t been well studied in the eye.

Elliott has learned the CAV-1 gene is expressed in several places in the eye (and also outside of the eye) and is linked with the presence of caveolae, which he described as flask-shaped particles, with their neck opening to the plasma membrane.

“In cells where they’re abundant, caveolae act like springs; they can sense pressure in the cell,” he said, whereas in the knock-out mouse model he’s developed, the membrane seems to lose its ability to respond to pressure.

In knockouts, “there’s also evidence that outflow pathway cells look morphologically pathologic,” he said. The endothelium is thickened and there are other changes. “The tissue is not responding normally,” he said, causing him to speculate that CAV-1 is responsible for creating a domain in caveolae that are part of a mechanism for endothelium to respond to pressure.

Winner’s Circle

Last year, Elliott received BrightFocus’ Thomas R. Lee Award recognizing the second-highest rated proposal in the National Glaucoma Research (NGR) program. While it's not a race, BrightFocus knows how to pick them!

For very personal reasons, Elliott is grateful to BrightFocus for giving him the chance to pursue this project alongside his ongoing work in the retinal area.

“My father has had glaucoma since he was in his forties,” he said. “And even though we're lucky in that it's been well controlled with medications, he's always asking me when I'm going to do something for glaucoma.”

Now, with BrightFocus support, he’s onto something big!

Exciting Research Projects Unveiled at BrightFocus Breakfast

Speaking of awards, each year BrightFocus kicks off the busiest day at ARVO—Monday's opening—with a breakfast to honor our top vision grantees who receive named awards in macular degeneration and glaucoma research endowed by generous donors.

Invitations to the event were sent to the entire pool of 2014 vision grantees, as well as the Scientific Review Committee members who generously donated their time to review and rate grant applications. The breakfast also pays tribute to all BrightFocus donors who contributed to this year's pool of $3.5 million in vision research funding granted to 30 scientists.

At this year's event, held at the Orlando Hilton May 5, it was standing room only—and not just to partake of the breakfast buffet. In fact, the more than 80 attendees seemed much more interested in swapping stories than eating. It's always a treat to hear the group's excitement about finally being able to purchase some new lab equipment, solve a methodological quandary, or hire a stellar graduate student onto their project, and this year's awardees seemed especially grateful.

"Each year, we give these awards in recognition of the most promising and forward-thinking ideas in the fields of glaucoma and macular degeneration research," said BrightFocus President and CEO Stacy Haller. "These special grant awards are funded in honor of exceptional people who fought these diseases and wanted to support outstanding researchers."

The five 2014 award winners include:

• Debra Thompson, PhD, University of Michigan, receiving the $120,000 Elizabeth Anderson Award for Macular Degeneration Research honoring the beloved late wife of  BrightFocus Scientific Review Committee member Robert Anderson. Thompson is looking at special proteins that may help control inflammatory responses which, even at low levels, can damage the retina over time.
• Debasish Sinha, PhD, Johns Hopkins University, receiving the $120,000 Carolyn K. McGillvray Award for Macular Degeneration Research, provided in memory of McGillvray’s fighting spirit as she battled macular degeneration. Sinha and his team are working to understand how the disruption of certain processes—known as autophagy and phagocytosis—activates the immune system in retinal pigmented epithelium (RPE) cells, which can lead to some manifestations of macular degeneration.
• Douglas Vollrath, MD, Stanford University, receiving the $120,000 Helen Juanita Reed Award for Macular Degeneration Research, named after a beloved BrightFocus supporter and philanthropist who lived with, and supported research on, macular degeneration. Vollrath examining how the function of RPE cells varies among individuals due to genetic differences. His project may help scientists better understand why macular degeneration affects some people more than others.
• Colleen McDowell, PhD, University of North Texas Health Science Center, receiving the $100,000 Dr. Douglas H. Johnson Award for Glaucoma Research honoring its namesake for years of service as chairman of the Scientific Review Committee for Glaucoma. Presented annually to the top-rated research proposal in glaucoma, the award will support McDowell and her team in their efforts to better understand how the drainage structures work in the front of the eye and investigate a novel drainage pathway that could help prevent glaucoma caused by increased pressure from increased pressure inside the eye when fluid is not drained properly.
• Christopher Passaglia, PhD, University of South Florida, receiving the $100,000 Thomas R. Lee Award for Glaucoma Research. Lee, who bequeathed the annual award, was a philanthropist, farmer, and businessman who battled glaucoma. Passaglia is working on a smart pump that would give glaucoma researchers and clinicians an innovative system for measuring and controlling eye pressure.