New Molecular Targets for Treatment of Cushing's Disease.

Despite the best outcomes from trans-sphenoidal surgery, approximately one-third of patients with Cushing's disease will need medical therapy. Current treatments have drawbacks and there is a clear clinical need for new therapies. Recent understanding of molecular pathways leading to excess ACTH secretion has identified key components that may be targeted with the aim to provide novel effective treatment for this devastating disease. These include testicular orphan nuclear receptor 4, heat shock protein 90, and epidermal growth factor receptor. Based on data from preclinical studies, clinical trials are seeking to assess whether targeting these novel pathways can translate into patient benefit.