Everolimus is a selective mTOR- inhibitor which was approved for the new indication “for the treatment of advanced neuroendocrine tumours of pancreatic origin (pNET) with progressive disease” in August 2011. The approval was mainly based on the results of a phase III trial that showed a prolongation of PFS by 6.4 months for everolimus compared to placebo. A tumour response, all of which partial responses, was observed in 4.8% of patients treated with everolimus compared to 2.0% in the placebo group. Thus the effect of everolimus primarily lies in disease stabilisation. No advantage in overall survival has yet been shown, but available data is not well-suited to draw final conclusions. Adverse events were largely reversible.
Treatment options for advanced pNETs are limited but may comprise cytotoxic chemotherapy and targeted therapy with sunitinib. To date, comparative data from head-to-head studies to inform about the most beneficial therapy is lacking. The sequence in which the available therapies are best administered also remains an open question.