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In May 2011, the FDA approved everolimus as a treatment for patients with advanced progressive pancreatic neuroendocrine tumors (pNET) based on findings from the phase III RADIANT-3 trial. In the pivotal trial, progression-free survival was 11.0 months with everolimus compared with 4.6 months with placebo.
Rodney F. Pommier, MD, first observed the activity of everolimus in patients with pNET who were taking the drug as an immunosuppressant during organ transplants. In these observations the mTOR inhibitor everolimus suppressed the growth of the tumors. These findings led to further studies of everolimus in patients with pNETs, including the RADIANT-1 study that assessed everolimus as monotherapy and in combination with octreotide IM for patients whose disease had progressed on chemotherapy.
In the study, if a patient was already receiving treatment with a somatostatin analog for any purpose when they came to the trial, they remained on it. If they were not taking a somatostatin analog at the time of enrollment, they were given everolimus as monotherapy. Although this was not a randomized study, the results were promising. Pommier notes that every patient who was formerly progressing on chemotherapy had tumor stabilization with everolimus. The results also showed that the time to progression and survival was much longer with patients who were taking the combination. Time to progression in the combination arm had not yet been reached, Pommier suggests. This trial led to further study of everolimus in pNET.