Melatonin: A hypothesis regarding its use to treat Wilson disease

Wilson disease is associated with excessive copper accumulation in cells, primarily in the liver and brain. The subcellular lesions caused by an excess of this essential metal accounts for many of the signs of Wilson disease. The drugs used to treat this disease are not always effective, and depending on dose, they may have collateral toxicity. Melatonin is an endogenously-produced molecule that functions as a copper chelator, a potent antioxidant, and as a suppressor of endoplasmic reticulum stress and the unfolded protein response in both the liver and the brain, while also reducing fibrosis/cirrhosis in the liver. Melatonin is inexpensive, non-toxic and can be administered via any route. Melatonin should be tested for its potential utility in experimental models of Wilson disease with extension to the human if melatonin proves to be effective in the animal studies.