title: Vandetanib therapy in medullary thyroid cancer.
abstract: The U.S. Food and Drug Administration (FDA) approved vandetanib in April 2011 for the treatment of unresectable, locally advanced or metastatic medullary thyroid cancer (MTC). In Europe it was approved in March 2012, but only for the treatment of aggressive and symptomatic MTC. This small molecule is a tyrosine kinase inhibitor of several growth factors involved in cellular proliferation and angiogenesis, including the epidermal growth factor receptor (EGFR) and the vascular endothelial growth factor receptors 2 and 3 (VEGFR-2, VEGFR-3). In addition, vandetanib is an inhibitor of the RET (rearranged during transfection) gene, a proto-oncogene often mutated in familial MTC. Since MTC is a rare disease, for which no previous medical therapies are approved, vandetanib is the first drug shown to be effective in a large phase III trial treating patients with metastatic or locally advanced MTC. Common adverse events are diarrhea, nausea, hypertension, headache and QT prolongation that are manageable and are commonly outweighed by the benefits of vandetanib in terms of delaying disease progression and inducing tumor response.
authors.affiliation: Department of Gastroenterology/Endocrinology, Charité-Universitätsmedizin Berlin, CBF, Medical Clinic I, Dept. of Gastroenterology, Infectious Diseases, Rheumatology, Berlin, Germany. patricia.grabowski@charite.de
authors.forename: P
authors.initials: P
authors.lastname: Grabowski
authors.forename: F
authors.initials: F
authors.lastname: Briest
authors.forename: R P
authors.initials: RP
authors.lastname: Baum
authors.forename: J J
authors.initials: JJ
authors.lastname: Zaknun
authors.forename: H R
authors.initials: HR
authors.lastname: Kulkarni
authors.forename: M
authors.initials: M
authors.lastname: Zeitz
authors.forename: D
authors.initials: D
authors.lastname: Hörsch
mesh_terms: D000818:Animals; D000970:Antineoplastic Agents / Q000494:pharmacology / Q000627:therapeutic use*; D018278:Carcinoma, Neuroendocrine; D004347:Drug Interactions; D066246:ErbB Receptors / Q000378:metabolism; D006801:Humans; D010880:Piperidines / Q000494:pharmacology / Q000627:therapeutic use*; D047428:Protein Kinase Inhibitors / Q000494:pharmacology / Q000627:therapeutic use*; D000090063:Proto-Oncogene Mas; D051096:Proto-Oncogene Proteins c-ret / Q000378:metabolism; D011799:Quinazolines / Q000494:pharmacology / Q000627:therapeutic use*; D040262:Receptors, Vascular Endothelial Growth Factor / Q000378:metabolism; D013964:Thyroid Neoplasms / Q000188:drug therapy* / Q000378:metabolism
chemical_list: D000970:Antineoplastic Agents; C000715470:MAS1 protein, human; D010880:Piperidines; D047428:Protein Kinase Inhibitors; D000090063:Proto-Oncogene Mas; D011799:Quinazolines; D066246:ErbB Receptors; D051096:Proto-Oncogene Proteins c-ret; D040262:Receptors, Vascular Endothelial Growth Factor; C452423:vandetanib