A new federally funded research effort in Rochester is aiming to tackle one of the biggest gaps in treating age-related macular degeneration — stopping vision loss before it starts.
Researchers at University of Rochester Medicine have secured a grant from the National Institutes of Health to study early drivers of the disease and develop a more precise treatment that could slow or prevent damage to the retina.
Age-related macular degeneration, or AMD, is a leading cause of vision loss among older adults, affecting the macula — the part of the eye responsible for sharp, central vision used for reading, driving and recognizing faces. Existing treatments primarily target advanced stages of the disease and often come with limitations, including side effects and incomplete effectiveness.
The new project, led by Dr. Ruchira Singh of the Flaum Eye Institute, focuses on a protein known as sPLA2-IIA, which researchers believe plays a key role in the development and progression of AMD. Using patient-derived stem cell models, Singh’s team found that elevated levels of the protein are linked to early retinal damage, including the formation of drusen — a hallmark of the disease.
By identifying a potential root cause, the research opens the door to a different type of treatment approach. The team is working to develop a small-molecule therapy designed to eliminate the protein, targeting the disease earlier and more precisely than current options.
Researchers say that specificity could be key. Traditional treatments often affect broader biological processes, increasing the risk of side effects. A more targeted therapy could allow for lower doses and improved outcomes, particularly in earlier stages when intervention may have the greatest impact.
The project is part of the NIH Blueprint Neurotherapeutics Network, which is designed to help move promising treatments from early discovery toward clinical testing. The program provides access to specialized expertise and resources to support development, including drug formulation and early-stage clinical planning.
If successful, the work could lead to new treatment options not only for AMD but also for related macular diseases with similar underlying mechanisms.
Researchers say the goal is straightforward but ambitious: preserve vision — and potentially restore it — by intervening before irreversible damage occurs.
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