Neuroendocrine tumors. Molecular targeted therapy for carcinoid and islet-cell carcinoma

James C Yao1

  • 1Department of Gastrointestinal Medical Oncology, The University of Texas M. D. Anderson Cancer Center, Houston, TX 77030, USA. jyao@mail.mdanderson.org

Insights

Low-grade neuroendocrine tumors, including carcinoid and islet-cell carcinomas, show promise with molecularly targeted therapies. VEGF and mTOR inhibitors demonstrate encouraging results, with ongoing clinical trials for advanced treatments.

Area of Science:

  • Oncology
  • Molecular Biology
  • Endocrinology

Background:

  • Carcinoid and islet-cell carcinomas are low-grade neuroendocrine tumors.
  • These tumors are often slow-growing and can be resistant to conventional therapies.
  • Current treatments for carcinoid tumor growth are limited, and second-line options for islet-cell carcinoma are scarce.

Purpose of the Study:

  • To review the molecular biology of neuroendocrine tumors.
  • To discuss the potential of molecularly targeted therapies for these diseases.

Main Methods:

  • Review of molecular biology, including genes like MENIN, TSC2, NF-1, vHL, p53, bcl-2, bax, VEGF, IGF, PDGF, EGFR, and mTOR.
  • Analysis of Phase-II study data for targeted agents: imatinib, bevacizumab, sunitinib, gefitinib, temsirolimus, and everolimus (RAD001).

Main Results:

  • Encouraging results observed with vascular endothelial growth factor (VEGF) and mammalian target of rapamycin (mTOR) inhibitors.
  • Phase-II studies with targeted agents have completed accrual, providing preliminary efficacy data.

Conclusions:

  • Molecularly targeted therapies, particularly VEGF and mTOR inhibitors, represent a promising avenue for treating neuroendocrine tumors.
  • Ongoing Phase-II and -III clinical trials, including those for bevacizumab and everolimus, are crucial for establishing new treatment standards.

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