title: Cabozantinib for the treatment of advanced medullary thyroid cancer.
abstract: BACKGROUND
  Patients with advanced medullary thyroid cancer (MTC) have poor prognoses and limited treatment options. Improved knowledge about molecular aberrations associated with MTC and the availability of novel targeted tyrosine kinase inhibitors (TKIs) have led to new potential treatment modalities. Cabozantinib is an oral multitargeted TKI with activity against multiple receptors including RET, vascular endothelial growth factor receptor type 2 (VEGFR2), and MET that has been evaluated in MTC in the preclinical and clinical arenas.
  
  
  METHODS
  This article reviews unmet clinical needs in advanced MTC. The authors consider novel agents that have been studied in MTC, with a focus on the investigational agent cabozantinib. Up-to-date clinical data of cabozantinib in MTC are discussed.
  
  
  RESULTS
  Recent clinical evaluation suggests that cabozantinib is the first agent to prolong progression-free survival in patients with progressive MTC. These findings indicate that cabozantinib may be an effective therapy in advanced MTC. No improvement in overall survival has been demonstrated but data are not mature.
  
  
  CONCLUSIONS
  Cabozantinib may be an effective treatment option for patients with advanced MTC and is worthy of further evaluation.
authors.affiliation: Section of Endocrinology, Diabetes and Metabolism, Department of Medicine, University of Chicago, 5841 S. Maryland Ave., Chicago, IL 60645, USA.
authors.forename: Madhavi
authors.initials: M
authors.lastname: Nagilla
authors.forename: Rebecca L
authors.initials: RL
authors.lastname: Brown
authors.forename: Ezra E W
authors.initials: EE
authors.lastname: Cohen
mesh_terms: D000284:Administration, Oral; D000328:Adult; D000368:Aged; D000813:Anilides / Q000008:administration & dosage* / Q000009:adverse effects; D001707:Biopsy, Needle; D018278:Carcinoma, Neuroendocrine; D017321:Clinical Trials, Phase I as Topic; D018572:Disease-Free Survival; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D007150:Immunohistochemistry; D008297:Male; D020714:Maximum Tolerated Dose; D008875:Middle Aged; D009361:Neoplasm Invasiveness / Q000473:pathology; D009367:Neoplasm Staging; D018579:Patient Selection; D011379:Prognosis; D011505:Protein-Tyrosine Kinases / Q000037:antagonists & inhibitors*; D011725:Pyridines / Q000008:administration & dosage* / Q000009:adverse effects; D016019:Survival Analysis; D013964:Thyroid Neoplasms / Q000188:drug therapy* / Q000401:mortality* / Q000473:pathology; D016896:Treatment Outcome
chemical_list: D000813:Anilides; D011725:Pyridines; C558660:cabozantinib; D011505:Protein-Tyrosine Kinases