title: Emerging drugs for Cushing's disease.
abstract: BACKGROUND
  Considering the effects of uncontrolled hypercortisolism on morbidity and mortality, there is a clear need for effective medical therapy for patients with Cushing's disease (CD). Therefore, the search for new medical effective tools remains active, and already promising results have been obtained.
  
  
  METHODS
  The importance of the design and conduct of trials to validate old drugs or to test new compounds is discussed. The results of the ongoing clinical trials, targeting the specific properties of drugs, such as ketoconazole, LCI699, mifepristone, etomidate and pasireotide, are also reported. The authors also emphasise the advantages and drawbacks of each particular drug, and the potential combined use of agents with complementary mechanisms of action.
  
  
  CONCLUSIONS
  CD is an excellent example of a situation where effective therapy is essential, but where the balance of risk and benefit must be carefully judged. Metyrapone is the drug of choice when rapid control of the hypercortisolaemia is required, ketoconazole represents a good second-line drug, although in the future LCI699 may be a better alternative. Mifepristone can also be used in the rare situation when previous drugs are inappropriate. Etomidate is useful where immediate parenteral action is required. For drugs working directly on the pituitary, cabergoline is occasionally effective and pasireotide can be attempted in patients with mild CD.
authors.affiliation: a 1 Department of Endocrinology, Diabetes and Metabolism of Coimbra Hospital and University Centre , Portugal danielafsguelho85@gmail.com.
authors.forename: Daniela
authors.initials: D
authors.lastname: Guelho
authors.forename: Ashley B
authors.initials: AB
authors.lastname: Grossman
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic / Q000379:methods; D015195:Drug Design; D004359:Drug Therapy, Combination; D006801:Humans; D047748:Pituitary ACTH Hypersecretion / Q000188:drug therapy* / Q000503:physiopathology
keywords: Cushing disease; LCI699; ketoconazole; mifepristone; pasireotide