This article summarizes the clinical features and molecular pathogenesis of medullary thyroid cancer (MTC) and focuses on the current use of molecular, biochemical, and imaging disease markers as a basis for selection of appropriate therapy. Clinicians treating patients who have MTC face the following challenges: (1) distinguishing MTC as early as possible from benign nodular disease and differentiated thyroid cancer to choose the appropriate initial surgery, (2) managing low-level residual cancer in otherwise asymptomatic individuals, and (3) treating progressive metastatic disease. Early clinical trials using small molecules targeting Ret or vascular endothelial growth factor receptors suggest that such approaches could be effective and well tolerated. This article highlights early progress in targeted therapy of MTC and significant challenges in disease monitoring to appropriately select and evaluate patients being treated with these therapies.
