Novel therapeutic directions in Cushing's syndrome

Cushing's disease is the most common cause of endogenous hypercortisolemia. It is a rare disease of unknown etiology that is prone to relapse and damaging to quality of life, even in remission. A significant proportion of patients are not cured by primary surgical treatment, and adjuvant radiotherapy has become an increasingly unattractive proposition for clinicians who wish to spare their patients hypopituitarism and other potential complications. The few effective pharmacological options that are available currently tend to have dose-limiting side effects and new alternatives that are safer and more effective than the adrenal cortical enzyme inhibitors and adrenolytic drugs would be most welcome. This article discusses preliminary efficacy data for several potential new groups of drugs.