Two University of Kentucky scientists are the leaders of a group that reported in the journal Science on Thursday that HIV/AIDS drugs could be repurposed to treat age-related macular degeneration and other inflammatory disorders.
Age-related macular degeneration is a progressive eye condition that is untreatable in as many as 90 percent of patients and is a leading cause of blindness in the elderly. It has two forms, wet and dry, which are classified based on the presence or absence of blood vessels that have invaded the retina.
There are FDA-approved treatments for wet macular degeneration, but so far, there have been no approved treatments for dry macular degeneration.
The Science report says that multiple FDA-approved nucleoside reverse transcriptase inhibitors prevented retinal degeneration in a mouse model of dry macular degeneration. The drugs blocked an immune pathway called the "inflammasome."
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In the report, the researchers — which included UK's Jayakrishna Ambati and Benjamin Fowler — said the drugs were also effective in other disease models, including the wet form of macular degeneration and graft-versus-host disease.
Fowler, the lead author of the article and a postdoctoral fellow in Ambati's lab, said repurposing the HIV/AIDS drugs "could be advantageous, for one, because they are very inexpensive. Moreover, through decades of clinical experience, we know that some of the drugs we tested are incredibly safe."
In addition, he said, they are already approved by the FDA, so they could be made available quickly.
Researchers are excited at the possibility of testing the drugs on patients, Ambati said.
The drugs were originally designed to treat cancer in the 1960s, and they re-emerged in the late '80s to become the first drugs the FDA approved to treat HIV/AIDS.