Common HIV drugs may prevent macular degeneration, a major cause of vision loss

CHARLOTTESVILLE, Va. — Sometimes solutions come from the most unexpected of sources. Researchers at the University of Virginia report a common drug usually used to treat HIV may be able to help stop one of the leading causes of blindness around the globe. Scientists uncovered the connection thanks to a major discovery which calls into question modern science’s fundamental beliefs regarding DNA.

Medical professionals call these drugs Nucleoside Reverse Transcriptase Inhibitors (NRTIs) and researchers believe they can help treat dry macular degeneration, a very common cause of blindness. These results are especially puzzling considering the fact that dry macular degeneration isn’t caused by a virus like HIV.

Researchers analyzed four separate health insurance databases. That process led them to the discovery that individuals taking NRTIs are at a significantly lower risk of developing dry macular degeneration.

“We are extremely excited that the reduced risk was reproduced in all the databases, each with millions of patients,” says Jayakrishna Ambati, MD, a top macular degeneration researcher, in a university release. “This finding provides real hope in developing the first treatment for this blinding disease.”

DNA’s connection to vision loss

Ambati macular degeneration
Jayakrishna Ambati and his collaborators have identified a group of drugs that may help stop a leading cause of blindness. (Credit: UVA Health)

This all started when researchers discovered that DNA can indeed be created in the cytoplasm of human cells. The cytoplasm is located outside of the nucleus, which had always been considered the “home” of our genetic material and the only place where DNA is produced.

Following this major revelation, researchers realized that if a certain type of DNA (Alu) builds up in the cytoplasm it can contribute to macular degeneration. As far as study authors can tell, too much Alu can destroy an important cell layer responsible for sustaining the retina’s visual cells.

With these revelations in mind, the research team opted to investigate drugs which can block the production of DNA. Their theory being that such medications may be able to slow vision loss caused by macular degeneration. After analyzing more than 100 million patients over two decades, they found that people taking NRTIs are nearly 40 percent less likely to suffer from dry macular degeneration.

Now that these initial findings have been established, the next step is a clinical trial.

“A clinical trial of these inflammasome-inhibiting drugs is now warranted,” Ambati, the founding director of UVA’s Center for Advanced Vision Science, concludes. “It’s also fascinating how uncovering the intricate biology of genetics and combining it with big data archeology can propel insights into new medicines.”

The study is published in PNAS.

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John Anderer

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