title: Medullary thyroid cancer: targeting the RET kinase pathway with sorafenib/tipifarnib.
abstract: Medullary thyroid carcinoma (MTC) is an uncommon malignancy of hereditary and sporadic presentation. Mutations in the RET proto-oncogene are involved in the pathogenesis of familial MTC and >50% of the sporadic cases. Currently, there is no effective treatment for recurrent or metastatic MTC. We report here a rapid response to a sorafenib (RET and RAF kinase and vascular endothelial growth factor receptor inhibitor)--based regimen in a patient with sporadic MTC who had advanced, progressive disease and a novel RET kinase aberration at exon 11 shown in tumor tissue.
authors.affiliation: Department of Investigational Therapeutics (Phase I Program), Unit 455, Division of Cancer Medicine, M. D. Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA. dshong@mdanderson.org
authors.forename: David
authors.initials: D
authors.lastname: Hong
authors.forename: Lei
authors.initials: L
authors.lastname: Ye
authors.forename: Robert
authors.initials: R
authors.lastname: Gagel
authors.forename: Lakshmi
authors.initials: L
authors.lastname: Chintala
authors.forename: Adel K
authors.initials: AK
authors.lastname: El Naggar
authors.forename: John
authors.initials: J
authors.lastname: Wright
authors.forename: Razelle
authors.initials: R
authors.lastname: Kurzrock
mesh_terms: D000328:Adult; D000595:Amino Acid Sequence; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001557:Benzenesulfonates / Q000627:therapeutic use*; D018276:Carcinoma, Medullary / Q000188:drug therapy* / Q000201:enzymology; D045744:Cell Line, Tumor; D005091:Exons; D006801:Humans; D008297:Male; D008969:Molecular Sequence Data; D009536:Niacinamide / Q000031:analogs & derivatives; D010671:Phenylurea Compounds; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use*; D000090063:Proto-Oncogene Mas; D051096:Proto-Oncogene Proteins c-ret / Q000037:antagonists & inhibitors* / Q000235:genetics; D011725:Pyridines / Q000627:therapeutic use*; D015363:Quinolones / Q000627:therapeutic use*; D000077157:Sorafenib; D013964:Thyroid Neoplasms / Q000188:drug therapy* / Q000201:enzymology
chemical_list: D000970:Antineoplastic Agents; D001557:Benzenesulfonates; C000715470:MAS1 protein, human; D010671:Phenylurea Compounds; D047428:Protein Kinase Inhibitors; D000090063:Proto-Oncogene Mas; D011725:Pyridines; D015363:Quinolones; D009536:Niacinamide; D000077157:Sorafenib; D051096:Proto-Oncogene Proteins c-ret; C402769:tipifarnib