A drug once used to treat alcoholism may cure retinal degeneration

The drug tested on mice was found to revive sight in humans with the inherited disease retinitis pigmentosa (RP), and perhaps in other disorders, including age-related macular degeneration.

Get more updates on this story and more with The Blueprint, our daily newsletter: Sign up here for free.

Antabuse to the rescue

The researchers found that the drug disulfiram (also called Antabuse) disrupts not only enzymes involved in the body’s ability to degrade alcohol, but also enzymes that make retinoic acid. Mice treated with disulfiram saw a decrease in the production of retinoic acid even if they were nearly blind and became much better at detecting images displayed on a computer screen.

“There may be a long window of opportunity in which suppressing retinoic acid with drugs like disulfiram could substantially improve low vision and make a real difference in people’s quality of life,” said Richard Kramer, the CH and Annie Li Chair in Molecular Biology of Diseases at UC Berkeley and a member of the campus’s Helen Wills Neuroscience Institute.

“Because the drug is already FDA-approved, the regulatory hurdles are low. It wouldn’t be a permanent cure, but right now there are no available treatments that even temporarily improve vision.”

For the next steps, the researchers will partner with ophthalmologists to conduct a clinical trial of disulfiram on a small set of people with advanced, but not yet complete, retinal degeneration.

A drawback

The drug does however come with one major drawback: when combined with alcohol consumption it has severe side effects, including headache, nausea, muscle cramps, and flushing.

Still, its results cannot be ignored.

“Treated mice really see better than mice without the drugs. These particular mice could barely detect images at all at this late stage of degeneration. I think that that’s quite dramatic,” Kramer said. Now, the scientists hope this latest development can drive new drug development and a whole new strategy for helping to improve vision.