Innovative tumour targeting therapeutics in Cushing's disease

Cushing's disease (CD) is the most frequent form of endogenous hypercortisolism. Management of this devastating condition relies on pituitary surgery, while effective pharmacological treatment mainly focus on periphery targeting pharmaceuticals. Approved tumour-targeting drugs are limited to dopamine agonists and somatostatin analogues with frequently low efficacy and substantial side effects. Discoveries on the genetics and pathophysiology of corticotroph tumorigenesis brought forward new potential pharmacological targets. Compounds such as retinoic acid although promising in preclinical studies, are not as efficient in the clinic. Others, such as, silibinin, gefitinib and roscovitine are effective in preclinical models, but their efficacy and safety still needs to be determined in patients with CD.