Common HIV drugs can prevent the leading cause of vision loss, study finds

Scientists have identified a group of drugs that can help stop a leading cause of vision loss after making an unexpected discovery that overturns a fundamental belief about DNA.

The drugs, known as nucleoside reverse transcriptase inhibitors or NRTIs, are often used to treat HIV. The new discovery suggests they may also be useful against dry macular degeneration, even though a virus doesn’t cause that vision-blocking condition.

A review of four different health insurance databases suggests that people taking these drugs have significantly reduced the risk of developing dry macular degeneration, a condition that affects millions of Americans.

“We are extremely excited that the reduced risk was reproduced across all databases, each with millions of patients,” said Jayakrishna Ambati, MD, a top macular degeneration researcher at the University of Virginia School of Medicine. “This finding offers real hope in the development of the first treatment for this blind disease.”

Targeting macular degeneration

The new discovery comes from Ambati; Fred H. Gage, Ph.D., of the Salk Institute for Biological Studies; and employees around the world. The work rewrites our understanding of DNA, revealing for the first time that it can be manufactured in the cytoplasm of our cells, outside the nucleus that houses our genetic material.

The build-up of a certain type of DNA in the cytoplasm, Alu, contributes to macular degeneration, the researchers found. This build-up appears to kill an important layer of cells that nourish the visual cells of the retina.

Based on this discovery, the researchers decided to look at drugs that block the production of this DNA to see if they can help prevent vision. They analyzed multiple U.S. health insurance databases – with more than 100 million patients over two decades – and found that people using NRTIs were nearly 40% less likely to develop dry macular degeneration.

The researchers urge further research to determine whether these drugs or safer derivatives, known as Kamuvudines, both of which block a major inflammatory pathway, can help prevent vision loss from dry macular degeneration.

“A clinical trial with these anti-inflammatory drugs is now warranted,” said Ambati, the founder and director of UVA’s Center for Advanced Vision Science. “It’s also fascinating how uncovering the complex biology of genetics and combining it with big data archeology can drive insights into new drugs.”

Ambati, of UVA’s Department of Ophthalmology, has previously found that NRTIs can also help prevent diabetes.

The researchers have published their findings in the scientific journal PNAS.