mitotane

Mitotane is an adrenolytic small molecule primarily indicated for the treatment of inoperable, functional, or nonfunctional adrenocortical carcinoma (ACC). It acts by selectively targeting the adrenal cortex, where it induces mitochondrial dysfunction and endoplasmic reticulum (ER) stress. Specifically, mitotane inhibits sterol-O-acyltransferase 1 (SOAT1), leading to the accumulation of free cholesterol and subsequent lipotoxicity. It also impairs steroidogenesis by blocking various cytochrome P450-mediated reactions. Due to its highly lipophilic nature, mitotane accumulates in fatty tissues and circulating lipoproteins, which often results in clinically significant dyslipidemia, including elevations in LDL, HDL, and triglycerides. Beyond its use in oncology, it is also utilized in the management of Cushing's syndrome to suppress excessive cortisol production.

Brand names

LysodrenOpeprimOpeblim

Other names

1,1-dichloro-2-(o-chlorophenyl)-2-(p-chlorophenyl)ethane