Drug for Alcohol Abuse May Restore Vision for People with Retinal Diseases

Foundation-funded researchers from the University of California, Berkeley, used Antabuse, a drug approved by the US Food & Drug Administration for alcohol abuse, to restore vision in mice with retinitis pigmentosa (RP). The research team believes the drug may restore vision in people with RP and perhaps other retinal conditions such as age-related macular degeneration. The investigators are planning a small clinical trial to evaluate the drug’s efficacy for retinal disease. Results from the mouse study were published in the journal Science Advances.

The researchers observed in mice with RP that the hyperactivity of retinal ganglion cells (RGCs), which increases visual noise, interfered with the transfer of visual information from photoreceptors to the brain. Antabuse reduced the hyperactivity and sensory noise, thereby restoring vision in the affected mice.

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.

The UC Berkeley team is also exploring other 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.

Perhaps the biggest downside to Antabuse is that it causes significant side effects – headaches, nausea, and muscle cramps – when alcohol is consumed.