Medical treatment of neuroendocrine tumours

Purpose of review This review summarizes results of recent clinical trials regarding the treatment of advanced neuroendocrine tumours (NETs) and pancreatic NETs (PNETs). Recent findings Most NETs occur sporadically in the lung and the gastrointestinal tract, and their prevalence has apparently increased over the last decades. Although curative treatment can be accomplished by surgery, for some NETs, most present in advanced stages and alternative, medical therapy is indicated. Recent randomized clinical treatment trials using somatostatin analogues in well differentiated midgut NET and therapies targeting the mammalian target of rapamycin (mTOR) signalling pathway and various tyrosine kinases provided evidence of improved progression-free survival. Treatment of functional PNETs with the mTOR inhibitor everolimus also showed reduction of peptide secretion relevant to the presenting clinical syndrome. Summary Previous work regarding the molecular pathology of NETs identified mTOR and tyrosine kinase signalling pathways as relevant targets in the neuroendocrine tumour biology. Subsequently, recent randomized clinical trials targeting these pathways with inhibitor therapies have provided encouraging results demonstrating prolonged progression-free survival and improvement of secretion-related clinical syndromes.