Age-related macular degeneration (AMD), a significant cause of irreversible vision impairment in the US, continues to baffle scientists as its underlying reasons and effective treatments remain undefined. However, a recent study featured in Developmental Cell offers a promising glimpse into the cellular workings of AMD, opening potential pathways for advanced treatments.
Ruchira Singh, PhD, affiliated with the University of Rochester Flaum Eye Institute and Center for Visual Sciences and the primary author of the study, acknowledges that existing AMD treatments have restricted effectiveness and often carry substantial side effects. Therefore, the objective of their research is to pinpoint new therapeutic targets that can possibly stop the disease from progressing.
The research leveraged human stem cells to simulate AMD, transcending the constraints of prior research that relied on animal models. By scrutinizing genes associated with both AMD and the less common inherited blindness forms known as macular dystrophies, the team identified a crucial protein in the disease’s early stages.
The retinal pigment epithelium (RPE), a layer of cells at the back of the eye, is integral to AMD development. Over time, these cells gather lipid and protein deposits, known as drusen, which are often the first signs of AMD.
The study revealed that a protein, tissue inhibitor of metalloproteinases 3 (TIMP3), is excessively produced in AMD. This protein inhibits the action of enzymes termed matrix metalloproteinases (MMPs), which are vital for eye health. Hindered MMP activity results in an increase in another enzyme that induces inflammation and drusen formation.
The research team managed to lessen drusen formation in their model by employing a small molecule inhibitor to obstruct the action of the inflammation-associated enzyme. This suggests that this pathway could be an encouraging target for AMD prevention.
Dr. Singh emphasized that the cellular pathways involved in drusen formation are critical to AMD progression. If the accumulation of drusen can be halted, the disease may be prevented from reaching a stage where vision loss takes place. This study offers hope for new treatments that could significantly enhance the lives of millions affected by AMD.
This research involved collaboration with numerous other authors and institutions, including the University of Rochester, the National Eye Institute, the University of Melbourne, and the Cleveland Clinic. It was funded by the National Eye Institute, ForeBatten Foundation, and Research to Prevent Blindness.
Comments are closed for this post.