Table 1.

Selected drugs targeting pathways involved in medullary thyroid cancer

Drug	Alternative Name	Targets	Selected trials
Vandetanib	ZD6474	VEGFR2, VEGFR3, EGFR, RET, PDGFR25,35	MTC Early Phase37,38 Phase-III: 331 MTC patients10
Cabozantinib	XL184	MET, VEGFR2, RET, KIT, AXL, FLT341	MTC Phase-I40 Phase-III: 330 MTC patients11
Sorafenib	BAY 43–9006	RAF, RET, VEGFR1-3, PDGFR, KIT, FLT327,39	Phase-I: 1 MTC patient27 13 MTC patients46 Phase-II: Thyroid cancer; 1 MTC patient;141 21 MTC patients45
Sunitinib	SU11248	VEGFR1,2, PDGFR, RET, KIT, FLT3, CSF1R39,50	Phase-II: Thyroid cancer including 7 MTC patients50
Axitinib	AG-013736	VEGFR1-3, much lower for KIT, PDGFR57	Phase-II: Thyroid cancer including 11 MTC cases57
Motesanib	AMG 706	VEGFR1-3, PDGFR, KIT58	Phase-I: 1 MTC patient142 Phase-II:91 MTC patients58
Ponatinib	AP24534	BCR-ABL, SRC, FLT3, KIT, FGFR1, PDGFR, VEGFR2, RET59	MTC cell culture, animal model59 MTC cell culture, comparison with other MTC agents53 Phase-II: CML – High rate of arterial thrombotic events60
Imatinib	STI571	BCR-ABL, PDGFR, CSF1R, KIT, lower activity against RET54,55	Phase-II: MTC54,55
Gefitinib	ZD1839	EGFR56	Trial including 4 MTC patients56

Abbreviations: MTC: Medullary thyroid cancer; AXL: AXL receptor tyrosine kinase; BCR-ABL: Breakpoint cluster region-Abelson tyrosine-protein kinase; CML: Chronic myeloid leukemia; CSF1R: Colony stimulating factor-1 receptor (c-Fms); EGFR: Epidermal growth factor receptor; FGFR: Fibroblast growth factor receptor; FLT3: fms-related tyrosine kinase 3; KIT: c-Kit/Stem cell factor receptor; MET: Mesenchymal epithelial transition factor/hepatocyte growth factor receptor; PDGFR: Platelet-derived growth factor recptor; RAF: Rapidly accelerated fibrosarcoma kinase; RET: Rearranged during transfection; SRC: Avian sarcoma viral oncogene homolog; VEGFR: Vascular endothelial growth factor receptor