Thirty years ago, one of Gregory Hageman's family members started to go blind. The diagnosis: age-related macular degeneration (AMD), the most common cause of blindness among people over 65. The symptoms: blurred vision in the center of the visual field, eventually leading to complete central vision loss. There was no cure for AMD. There were no effective therapies. The disease was considered an inevitability of aging.
Hageman has little patience for inevitabilities.
The then-marine biologist pivoted his entire field of study to focus on AMD, working to understand the disease from the ground up. Now, Hageman is a distinguished professor of ophthalmology and visual sciences at University of Utah-and he's on the brink of a therapy that could stop AMD in its tracks. "The ultimate goal is to slow or halt AMD with a single injection into the eye," Hageman says.
Seeing a brighter future
The key lies in the genetics of the disease. Hageman discovered that a mutation in a gene called CFH dramatically increases the risk of developing AMD. He also found that a slightly different version of CFH strongly protects against the disease.
Based on this knowledge, Hageman helped develop a gene therapy that delivers extra copies of the protective gene directly to the part of the eye that's most affected by AMD. It's a precision therapy designed to treat the disease at its source, made possible by decades of research into the underlying biology of the disease. Now, phase 2 clinical trials are underway at the University of Utah's John A. Moran Eye Center and beyond to test if the gene therapy can stop the progression of AMD.
Change in sight
Hageman leads the Sharon Eccles Steele Center for Translational Medicine (SCTM), which focuses on building research discoveries into sight-saving therapies. Having helped AMD research progress from fundamental discovery to clinical trials, Hageman is hopeful that the new therapy could finally provide a solution to slow or stop vision loss.
"When I decided to work on AMD, we knew practically nothing about its biology or clinical course, and many individuals told me I was wasting my time. Now I look back and think, 'Wow, we're actually going to give treatment a go,' he says. "I have great confidence in the science, the unique genetic targeting behind our approach, and the SCTM's extremely talented staff. I really believe this is going to work."
Because that's how research at U of U Health gets done: by people seeking to change the status quo and stop what's thought to be unstoppable, building from a foundation of biological discovery to create treatments for real people.
"There is no one who understands AMD at the level of Dr. Hageman and his team," adds Randall Olson, MD, CEO of Moran Eye Center. "Personalized medicine is the future, and Dr. Hageman is leading us into that future."
