Everolimus (RAD001) induces tumor response in patients with medullary thyroid cancer: an in vivo and in vitro study

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Introduction: Medullary thyroid cancer (MTC) is a neuroendocrine tumor (NET) of the thyroid C cells which is known to express somatostatin receptors. Octreotide, a somatostatin analogue exerting both antisecretory and antitumor activity in different types of NETs, has shown low efficacy in MTC. Everolimus (RAD001), an inhibitor of mTOR, has shown antitumor effects in patients with NETs and synergistic effects when combined with octreotide. Until recently, RAD001 has never been evaluated in MTC.

Aim(s): To evaluate the effect of RAD001 in combination with octreotide in patients with MTC.

Materials and methods: Two patients (patient 1, a 60-year-old male; patient 2, a 74-year-old male) with progressive metastatic MTC under treatment with octreotide LAR 30 mg/month received RAD001 per os 10 mg/day in combination with the somatostatin analogue. At the time of the study, patient 1 had diffuse bone metastases with spinal nerve compression and paraplegia, patient 2 had bone and lymph node metastases. Both patients had elevated calcitonin levels which were increased by >30% over the last six months before the study. Treatment efficacy was evaluated every month for six months in both patients. An in vitro study was also performed to assess RAD001 effects on TT cells, a MTC cell line.

Conference: 7th Annual ENETSConcerence (2010)

Presenting Author:

Authors: Faggiano A, Ferolla P, Dicitore A, Castiglioni S, Borghi M,

Keywords: medullary thyroid cancer, neuroendocrine tumor, RAD001, Everolimus, octreotide, antitumor activity,