Uncovering a potential means for restoring vision lies within a medication designed to combat alcohol addiction

In a groundbreaking discovery, researchers have found a potential way to revive vision loss caused by age-related macular degeneration and retinitis pigmentosa. The key to this breakthrough is a drug called disulfiram, which is currently marketed under the brand name Antabuse and is primarily used to treat alcoholism.

The sensory noise that arises from the progression of outer retinal degeneration, such as age-related macular degeneration or retinitis pigmentosa, disrupts the signals from surviving photoreceptors in the outer retina to the brain. Disulfiram targets this sensory noise, allowing the surviving photoreceptors to complete the signal to the brain and restore some vision.

The researchers, led by Michael Telias, Ph.D., assistant professor of Ophthalmology, Neuroscience, and Center for Visual Science at the University of Rochester Medical Center, have found that disulfiram helps restore vision by suppressing the sensory noise in the inner retina caused by dying photoreceptors in the outer retina.

The research, which was published in Science Advances, used gene therapy to knock down the receptor, and it also dramatically improved vision in mice with retinitis pigmentosa (RP). The researchers are planning to partner with ophthalmologists to conduct a clinical trial of disulfiram on patients with RP at the University of Tübingen.

If a vision-impaired human were given disulfiram and their vision got better, even a little bit, it would strongly implicate the retinoic acid pathway in vision loss and could drive new drug development and a whole new strategy for helping to improve vision.

However, it's important to note that disulfiram has some severe side-effects if alcohol is consumed while taking it, including headache, nausea, muscle cramps, and flushing. The clinical trial will be carried out on a small set of people with advanced, but not yet complete, retinal degeneration.

The work was funded by the National Institutes of Health and the National Science Foundation. Other authors of the research include Kevin Sit, Ph.D., Daniel Frozenfar, Benjamin Smith, and Arjit Misra. Richard Kramer, Ph.D., professor at the University of California, Berkeley, and Michael Goard, Ph.D., Assistant Professor at University of California, Santa Barbara, were also involved in the research that found disulfiram's effects.

The researchers found that treated mice could see better than mice without the drugs, even at a late stage of degeneration. This discovery could pave the way for more targeted therapies that don't interfere with alcohol breakdown or other metabolic functions. If successful, this could be a significant leap forward in the treatment of vision loss caused by age-related macular degeneration and retinitis pigmentosa.

Uncovering a potential means for restoring vision lies within a medication designed to combat alcohol addiction