Everolimus is an active agent in medullary thyroid cancer: a clinical and in vitro study

A Faggiano1, V Ramundo, A Dicitore

  • 1IRCCS Fondazione SDN, Naples, Italy.

Insights

Everolimus combined with octreotide shows promise for treating medullary thyroid cancer (MTC). This combination therapy demonstrated anti-tumour effects and reduced calcitonin levels in patients with progressive metastatic MTC.

Area of Science:

  • Oncology
  • Pharmacology
  • Endocrinology

Background:

  • Medullary thyroid cancer (MTC) is a rare neuroendocrine tumour.
  • Everolimus, an mTOR inhibitor, has shown anti-tumour effects in other neuroendocrine tumours.
  • The efficacy of everolimus in MTC has not been previously evaluated.

Observation:

  • Two patients with progressive metastatic MTC received everolimus and octreotide.
  • In vitro studies assessed everolimus effects on MTC cell lines (TT and MZ-CRC-1).

Findings:

  • Both patients exhibited a tumour response, with significant calcitonin level reductions (86% and 42%).
  • Everolimus demonstrated dose-dependent inhibition of MTC cell proliferation in vitro.
  • Cell cycle arrest at G(0)/G(1) and cellular senescence were observed in MTC cell lines.

Implications:

  • Everolimus combined with octreotide may represent a viable anti-tumour strategy for progressive metastatic MTC.
  • Further investigation in larger prospective studies is warranted to confirm these findings.

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