July 12, 2024

Promising Results in Neuroendocrine Tumor Trial with Dual-Action Drug

A recent study led by researchers from Dana-Farber Cancer Institute has shown that a dual-action drug has produced encouraging results in a clinical trial involving patients with advanced neuroendocrine tumors. The findings of the CABINET trial, led by Dr. Jennifer Chan and senior author Dr. Jeffrey Meyerhardt, will be presented at the annual European Society for Medical Oncology (ESMO) Congress in October 2023.

Neuroendocrine tumors affect over 12,000 people in the United Statess each year. These tumors originate in the neuroendocrine cells, which have characteristics of both nerve and hormone-producing cells. They most commonly occur in the gastrointestinal tract, lungs, and pancreas. Current treatments for neuroendocrine tumors include surgery, targeted therapy, chemotherapy, and other local treatments, depending on the location and stage of the cancer. However, for patients whose tumors continue to grow and spread despite these treatments, more effective options are needed.

The drug being studied, cabozantinib, has already been approved by the U.S. Food and Drug Administration for the treatment of renal cell carcinoma, hepatocellular carcinoma, and thyroid cancer. It works by simultaneously targeting the growth circuits of cancer cells and their pipeline to the bloodstream. Cabozantinib blocks receptors for VEGF, a protein used to tap into the body’s blood supply, as well as other receptors that are crucial for tumor cell survival and metastasis, including c-MET, AXL, and RET.

The CABINET trial enrolled 290 patients with advanced neuroendocrine tumors, divided into two groups: those with tumors outside of the pancreas, and those with pancreatic tumors. Two-thirds of the participants were randomly assigned to take a daily 60 mg pill of cabozantinib, while the others received a placebo. The main measurement used in the study was progression-free survival (PFS), which measures how long patients lived before their disease worsened.

The results of the trial showed that patients with extra-pancreatic tumors who took cabozantinib had a median PFS of 8.3 months, compared to 3.2 months for those who took the placebo. For patients with pancreatic tumors, the median PFS was 11.4 months for those on cabozantinib, compared to 3.0 months for those on the placebo. These results indicate that cabozantinib can significantly improve outcomes for patients with neuroendocrine tumors that have not responded to previous treatments.

The side effects of cabozantinib were found to be similar to those observed in other studies of the drug. These included hypertension, fatigue, diarrhea, and skin rash. Despite these side effects, the results of the CABINET trial have been described as very encouraging by Dr. Chan. Cabozantinib shows promise as a new treatment option for patients with previously treated neuroendocrine tumors.

The CABINET trial was sponsored by the National Cancer Institute (NCI) and conducted by the NCI-funded Alliance for Clinical Trials in Oncology. It is part of Exelixis’ collaboration with the NCI’s Cancer Therapy Evaluation Program. The results of the trial provide hope for patients with neuroendocrine tumors and may lead to the approval of cabozantinib as a treatment option for this patient population.


  1. Source: Coherent Market Insights, Public sources, Desk research
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