Potential drugs for AMD treatment identified using stem-cell based research

pharmafile | December 16, 2021 | News story | Business Services  

Researchers at the National Institutes of Health (NIH) have identified two drug candidates to slow dry age-related macular degeneration (AMD), using a stem-cell-derived model. This is among the first studies demonstrating the potential of dish-based models to replicate the phenotype (characteristics) of a complex disease, as opposed to diseases caused by a single mutation.

The researchers used the model to screen drugs, in order to examine their potential in slowing or halting disease progression. The researchers screened over 1,200 drugs from a library of pharmacological agents tested for a range of other conditions.

Two drugs prevented the model from developing key phenotypes. These phenotypes were the accumulation of drusen, which are deposits in the retina; and the atrophy of retinal pigment epithelium (RPE) cells. In AMD, RPE cells shrink and die, and loss of RPE leads to the death of photoreceptors in the retina. This in turn leads to the loss of vision. The findings of this research also shed light into how genetic variants affect AMD development.

AMD is a leading cause of blindness, for which there is currently no treatment. The causes of AMD involve a combination of genetic factors, aging, and behaviour-related risk factors such as smoking and diet, not yet fully understood. Scientists from the National Eye Institute, part of the NIH, published their findings on 15 December in Nature Communications.

“This stem-cell-derived model of dry AMD is a game-changer. Scientists have struggled to unravel this incredibly complex disease, and this model could prove to be invaluable for understanding the causes of AMD and discovering new therapies,” shared Michael F. Chiang, MD, and director of the NEI.

Ana Ovey

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