Antabuse moves into clinical trial for people with RP
Disulfiram (Antabuse), FDA-approved for deterring alcohol, is in a phase 1 trial at the University of Washington to improve vision in retinitis pigmentosa.
A drug called Disulfiram (also known as Antabuse) which is approved by the US Food & Drug Administration (FDA) for deterring alcohol consumption, has moved into a phase 1 clinical trial for potentially improving vision in people with retinitis pigmentosa (RP). The placebo-controlled trial will enrol 30 participants who are patients at the University of Washington in Seattle.
Previously, researchers from the University of California used disulfiram to restore some vision in mice with advanced RP.
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, but it also 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.
The study researchers 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.
We will keep you informed as more updates are released.