Preliminary report of the use of everolimus in a patient with progressive medullary thyroid carcinoma

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Introduction: Medullary thyroid carcinoma (MTC) accounts for about 5-10% of all thyroid cancers, and while disease localized to the thyroid can be cured by surgery, there is no effective treatment for disseminated MTC. Constitutive activation of the RET proto-oncogene is implicated in familial and a proportion of sporadic cases, and up-regulates the AKT/PI3-kinase/mTOR signal transduction pathway. Recent studies have shown that the inhibitor of mTOR, everolimus (RAD001, Novartis, Basel), can inhibit the proliferation of the TT cell line and human MTC tumor cells in vitro. Clinically, everolimus has been shown to attenuate the progression of some gastrointestinal neuroendocrine tumors, but its clinical use in MTC has not yet been evaluated.

Aim(s): An early progress report on the use of RAD001 in the clinical treatment of a patient with progressive MTC.

Materials and methods: A patient with metastatic MTC received RAD001 (with institutional approval for compassionate use) and the effects evaluated.

Conference: 7th Annual ENETSConcerence (2010)

Presenting Author:

Authors: Druce M, Maher K, Grozinsky-Glasberg S, Gross D, Chambers J,

Keywords: medullary thyroid cancer, calcitonin, everolimus, RAD001,