Researchers at the University of Illinois Chicago have developed a promising small-molecule inhibitor that could revolutionize the treatment of wet age-related macular degeneration (AMD). This innovative compound, administered via eye drops, offers a less invasive alternative to the current standard of care, which involves frequent intravitreal injections.
Wet AMD is marked by abnormal blood vessel growth and leakage in the retina, leading to vision loss. Current treatments rely on anti-VEGF injections, which target vascular endothelial growth factor (VEGF)—a protein that promotes abnormal blood vessel formation. Although effective, these injections must be administered regularly, placing a significant burden on patients. This new compound, however, may reduce or eliminate the need for frequent injections.
The study, led by Yulia Komarova and published in Cell Reports Medicine, underscores the potential of this breakthrough treatment. The compound, called EBIN (End Binding-3 Inhibitor), works by targeting endothelial cells in the retina, specifically inhibiting the End Binding-3 (EB3) protein, which plays a key role in the blood vessel leakage associated with wet AMD.
In preclinical studies using animal models, EBIN not only reduced retinal damage but also stimulated tissue regeneration, promoting healing. Applying the eye drops twice daily for two to three weeks significantly reduced eye damage in these models, suggesting that a similar approach could be effective in humans.
EBIN’s ability to reverse age-related genetic changes and inflammation is another critical finding. The compound was shown to restore normal gene expression in retinal endothelial cells, reducing the impact of hypoxia and inflammation, which are key drivers of wet AMD. By addressing these underlying causes, EBIN could offer a more comprehensive treatment compared to current therapies.
The research team is optimistic about EBIN’s broader potential, as it may also be effective in treating other vascular conditions, such as diabetic retinopathy and acute lung injury. Future studies will explore additional drug delivery methods, including implantable lenses, to further optimize treatment outcomes.
The development of EBIN marks a major advancement in AMD treatment, offering hope for a more convenient and effective therapy that could improve the quality of life for millions of patients.
Reference
Quinn Lee et al, End binding-3 inhibitor activates regenerative program in age-related macular degeneration, Cell Reports Medicine (2023). DOI: 10.1016/j.xcrm.2023.101223