Ángel Cabrera, President | Georgia Institute of Technology-Main Campus
Ángel Cabrera, President | Georgia Institute of Technology-Main Campus
Researchers at Georgia Tech have made a potential breakthrough in the treatment of glaucoma, an incurable eye disease affecting four million Americans. The team, led by Raquel Lieberman, a professor in the School of Chemistry and Biochemistry and the Parker H. Petit Institute for Bioengineering and Bioscience, discovered two antibodies that could treat glaucoma by targeting the protein myocilin.
Glaucoma occurs when myocilin malfunctions, leading to increased eye pressure due to blocked drainage of aqueous humor. Lieberman's research indicates that mutated myocilin clumps together, preventing proper fluid circulation. “Your eyeball is kind of like a basketball,” Lieberman explained. “If you want it to work optimally, it has to be pressurized.”
The study identified two antibodies capable of breaking down rogue myocilin. One antibody prevents aggregation while the other does not interfere with clumping but still effectively degrades the protein. Alice Ma, a Ph.D. graduate involved in the research, stated: “These exciting results provide proof of concept that targeted antibodies for mutant myocilin aggregation could be therapeutic.”
This research represents nearly two decades of collaborative efforts with Jennifer Maynard from the University of Texas at Austin. Maynard's group initially discovered the antibodies responsive to mutation; Lieberman's team further analyzed their function.
“This study builds on 10 years of work that explains how myocilin folds to how to break it down,” said Lieberman. She is hopeful these findings will lead to clinical applications for treating early-onset glaucoma patients.
Lieberman is collaborating with Rebecca Neustein from Emory University to explore treatment possibilities for young patients who currently lack curative options. “She doesn't have much hope to give her patients for curing glaucoma,” Lieberman noted about Neustein's current situation.
Beyond glaucoma treatment, this discovery opens avenues for addressing other diseases associated with protein aggregation such as Alzheimer's and Parkinson’s.
Funding for this research was provided by the National Institutes of Health.
Animation by Raul Perez