A drug for pancreatic cancer that is also used to prevent rejection of organ transplants has been shown to delay progression of another form of cancer in patients with limited treatment options.
In a recent international study, patients with advanced lung and gastrointestinal neuroendocrine tumors who took the drug everolimus experienced a 52% drop in the risk their disease would progress. Neuroendocrine tumors, or NETs, arise from cells of the endocrine and nervous systems. Data from population-based registries indicate that more than 51% of NETs appear in the GI tract, 27% in the lungs and 6% in the pancreas.
“Although we knew from previous studies that everolimus could delay the growth of pancreatic NETs, this is the first time we have been able to conclusively show that it is effective in other NET sites,” said James Yao, M.D., chair of Gastrointestinal Medical Oncology. “We hope our results will provide a new treatment option for lung and gastrointestinal NETs.”
Yao and MD Anderson have a long history of development with the drug for neuroendocrine tumors. In 2011, the once-daily regimen was approved as frontline therapy for pancreatic neuroendocrine tumors, with Yao and MD Anderson leading all phases of clinical research — from early studies to the pivotal Phase III trial showing everolimus’ progression-free survival benefits.
In this most recent international trial, which enrolled 302 patients, everolimus was associated with an increase in median progression-free survival of more than seven months. The researchers also found a trend toward improved overall survival; however, survival analysis was an interim analysis, and too early to conclusively determine. Both survival and quality of life data will be analyzed in follow-up analyses.
Everolimus was well-tolerated. Common side effects associated with the therapy include an inflammation or ulceration of the mouth, rash, diarrhea, fatigue and infections.
“We were pretty confident that the drug would be active in this broader range of neuroendocrine tumors, but the magnitude of the treatment benefit is wonderful to see — even stronger than we saw in previous studies,” said Yao.
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