FDA-Approved Drug May Help Revive Vision in People With Progressive Blinding Disorders

Researchers led by Richard Kramer at the University of California, Berkeley, have discovered that Antabuse (disulfiram), which was a drug that use to be used to wean alcoholics off of drinking is also able to help improve sight in mice with retinal degeneration. This drug could potentially revive vision in humans with retinitis pigmentosa (RP) and other vision disorders. Kramer had discovered that retinoic acid is created when light sensing cells in the retina die off. This causes hyperactivity in the retina’s cells, which interferes with the transfer of information needed for vision. The drug Antabuse stops enzymes that make retinoic acid. Nearly-blind mice treated with Antabuse, were able to detect images. These researchers want to partner with ophthalmologists to conduct a clinical trial using Antabuse on patients with RP. There could be a window of opportunity where using a drug like Antabuse that stops the production of retinoic acid could improve low vision. This is not a permanent fix but it could temporarily improve vision. This could also be proof of the role of retinoic acid in vision loss and could lead to researchers looking into this pathway for drug and therapy development.

What this means for Usher syndrome: Vision loss in Usher syndrome is a type of RP. Antabuse could be a treatment that improves vision of individuals with RP. This is still in its early stages and has not been tested in humans yet for this particular condition, but it could be a temporary vision improvement and could also open the pathway to new research that could help vision loss. 

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