Uveal melanoma (UM) is a rare and deadly cancer of the eye, and the mortality rate has remained unimproved for 40 years. Half of the melanomas spread to other organs of the body, causing death in less than a year, so new treatments to preserve vision and prevent death are an urgent need. Now a preclinical study by researchers at the University of Alabama at Birmingham (UAB) and Emory University, Atlanta, offers hope--a small molecule inhibitor has been identified that dampens the potent drivers of this tumor. In mouse models, the inhibitor, KCN1 (3,4-dimethoxy-N-[(2,2-dimethyl-2H-chromen-6-yl) methyl]-N-phenylbenzenesulfonamide), strongly limited primary disease in the eye and metastatic tumor dissemination to the liver, and animals survived longer, without overt side effects. Thus, this class of inhibitory compounds shows promise, though the co-leaders of the research -- Erwin Van Meir, PhD, Professor of Neurosurgery at UAB, and Hans Grossniklaus, MD, MBA, Professor of Ophthalmic Pathology at Emory--say the drug needs further optimization before clinical use.
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