Stiripentol is a clinically useful anticonvulsant used to treat Dravet syndrome. The precise mechanism of action is unknown but it is thought to act via increasing GABAergic activity1, inhibiting LDHA2 and thereby activating ATP-sensitive potassium channels, and blocking Na+ and Ca2+ channels3. It also inhibits the cytochrome P450 enzymes 1A2, 2C9, 2C19, 2D6, and 3A4.4 Stiripentol and the copper ionophore elesclomol (Cat10-4238) synergistically suppressed esophageal squamous cell carcinoma (ESCC) tumor growth.2 Lactate dehydrogenase A inhibition by stiripentol reduced ESCC cuproptosis resistance by inhibiting lactylation of NUDT21.
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