U of M researchers discover potential new pathway to prevent age-related macular degeneration

MINNEAPOLIS/ST. PAUL (08/20/2024) — Published in JCI Insight, a research team led by the University of Minnesota Medical School has identified small molecules that can reduce the production of proteins linked to age-related macular degeneration, or AMD, — the leading cause of irreversible vision loss among seniors in industrialized nations. Worldwide, AMD affects over 200 million people.  

"To our knowledge, this is the first demonstration of a small molecule preventing AMD-like pathology in our preclinical model. In the big picture, these results provide hope that AMD may indeed be preventable, or at least modifiable by administration of a single drug," said John Hulleman, PhD, an associate professor and Larson Endowed Chair for Macular Degeneration Research at the U of M Medical School and corresponding author.

By using unbiased drug screening, these inhibitors — specifically targeting a cellular pathway known as GSK3 — were found to prevent AMD features in a preclinical model. The results show that blocking GSK3 at low levels changes certain proteins linked to AMD, effectively preventing the disease in living organisms. 

Future research will focus on determining whether blocking GSK3 can reverse existing AMD damage, potentially leading to new treatment options. 

Funding was provided by the Fichtenbaum Charitable Trust, Roger and Dorothy Hirl Endowed Research Fund, Edward N. and Della L. Thome Memorial Foundation Award in Age-Related Macular Degeneration Research and National Institutes of Health grants [R01-EY027785, P30-EY030413, S10OD026758-01]. Additional support came from the Promega Advanced Academic Access Program.

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