Small Molecule Target for Inhibition of Cortisol in Cushing's Syndrome

? DESCRIPTION (provided by applicant): Endogenous Cushing's syndrome is a classic dysfunction of the endocrine system caused by prolonged exposure to inappropriately high levels of cortisol. The condition is tumor-driven and leads to obesity, diabetes,hypertension and psychiatric dysfunction, symptoms which improve with a reduction in cortisol. Cushing's syndrome disproportionately affects females, who make up 75% of the 3,000 new cases diagnosed each year in the United States. Two drugs recently approved for Cushing's, mifepristone and pasireotide, underscore the importance of developing new therapies. Pasireotide, indicated for patients with an adrenocorticotropic hormone (ACTH)-secreting pituitary tumor (Cushing's Disease), is effective in just 25% of cases and can cause hyperglycemia and diabetes. Mifepristone blocks glucocorticoid and progesterone receptors, leading to elevated cortisol and endometrial bleeding in women. Significantly, medical therapy of hypercortisolism in Cushing's is still predom