Jun 5, 2024

Antabuse Moves into Clinical Trial for People with RP

Eye On the Cure Research News

Researchers believe the FDA-approved drug can reduce the hyperactivity of ganglion cells to improve vision.

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Disulfiram (aka, Antabuse), a drug approved by the US Food & Drug Administration (FDA) for deterring alcohol consumption, has moved into a clinical trial for potentially improving vision in people with retinitis pigmentosa (RP). The 30-participant, placebo-controlled, Phase 1 clinical trial is enrolling patients at the University of Washington in Seattle.

second clinical trial, at the University of Rochester Medical Center, will test the ability of disulfiram for improving vision in patients with macular degeneration and other forms of photoreceptor dystrophy, who are treated with disulfiram for alcohol abuse disorder.

Previously, researchers from the University of California, Berkeley, used disulfiram to restore some vision in mice with advanced RP. Results from the mouse study, led by Richard Kramer, PhD, and Michael Telias, PhD, were published in the journal Science Advances.

In the mouse study, investigators observed that the hyperactivity of retinal ganglion cells (RGCs) increased retinal noise, and as a result, interfered with the transfer of visual information from photoreceptors to the brain. RGCs are downstream from photoreceptors and relay visual information to the brain via the optic nerve. The RGC hyperactivity is caused by the increased production of retinoic acid in the degenerating retina. Antabuse not only inhibits enzymes that breakdown alcohol, it inhibits the enzymes that produce retinoic acid. In the mouse study, disulfiram reduced the hyperactivity and sensory noise in RGCs, thereby restoring vision in the affected mice.

Investigators are also exploring alternate approaches, such as gene therapy, to inhibit retinoic acid production, but Antabuse is attractive because it is available now, FDA-approved, and widely used in humans.

The investigators note that while this approach will restore vision, it isn’t a cure and won’t inhibit retinal degeneration. Rather, it helps patients make the most of their remaining photoreceptors. They believe the drug might also improve vision in people with age-related macular degeneration and other retinal conditions.

People on disulfiram can’t drink alcohol. Doing so causes headaches, nausea, and muscle cramps.