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Chemotherapeutic treatment approaches may be beneficial in some cases of neuroendocrine tumors (NETs). As an example, Pamela L. Kunz, MD, notes that patients with bulky, hepatic or rapidly progressive disease may benefit from treatment with cytotoxic agents. These agents are most beneficial in patients with symptoms caused by hormone secretion who will benefit from tumor shrinkage beyond prolonged stabilization of disease achieved by targeted therapies.
Overall, the only approved chemotherapy in this space is streptozocin, which was approved at a time when clinical trial standards weren't as rigorous, Kunz states. To date, many of the results reported in the study that led to the approval of streptozocin have not been replicated. At this time, Kunz notes, the agent temozolomide represents the most exciting cytotoxic in this space.
Kunz describes that a small study of 30 patients with pancreatic NETs demonstrated a 70% response rate to the cytotoxic agent temozolomide, which is an alkylating agent like streptozocin. To cull this data out further, a phase II study will examine temozolomide with or without capecitabine as a treatment for patients with pancreatic NETs. However, despite promising early data in pancreatic NETs, patients with carcinoid tumors remained unresponsive to chemotherapeutic approaches.