Use of tyrosine kinase inhibitors for treatment of medullary thyroid carcinoma

Recent Results in Cancer Research

Volumn 204, Issue , 2015, Pages 227-249

  Dadu, Ramona ^a   Hu, Mimi N ^a   Grubbs, Elizabeth G ^a   Gagel, Robert F ^a  

^a UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

Author keywords

Cabozantinib; Calcitonin; Medullary thyroid carcinoma; Metastasis; Multiple endocrine neoplasia type 2; RET proto oncogene; Tyrosine kinase inhibitor; Vandetanib

Indexed keywords

ACTIVATING TRANSCRIPTION FACTOR 4; ALANINE AMINOTRANSFERASE; ALKALINE PHOSPHATASE; ASPARTATE AMINOTRANSFERASE; AXITINIB; BILIRUBIN; CABOZANTINIB; EVEROLIMUS; GLUCOSE; IMATINIB; LENVATINIB; MAGNESIUM; MAMMALIAN TARGET OF RAPAMYCIN INHIBITOR; MOTESANIB; PAZOPANIB; PHOSPHORUS; POTASSIUM; PROTEIN TYROSINE KINASE INHIBITOR; SODIUM; SORAFENIB; SUNITINIB; THYROTROPIN; VANDETANIB; ANTINEOPLASTIC AGENT; PROTEIN KINASE INHIBITOR; PROTEIN RET; RET PROTEIN, HUMAN;

ABDOMINAL PAIN; ACNE; APOPTOSIS; CANCER CHEMOTHERAPY; CANCER LOCALIZATION; CONSTIPATION; DECISION MAKING; DECREASED APPETITE; DEPRESSION; DIARRHEA; DRUG DOSE REDUCTION; DRUG EFFICACY; DRUG SAFETY; DRY SKIN; DYSGEUSIA; DYSPEPSIA; DYSPHAGIA; DYSPHONIA; FATIGUE; FOOD AND DRUG ADMINISTRATION; GASTROINTESTINAL STROMAL TUMOR; HAIR COLOR; HAND FOOT SYNDROME; HEADACHE; HUMAN; HYPERTENSION; HYPOCALCEMIA; METASTASIS; NAUSEA; NEUTROPENIA; ONCOGENE RET; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PHOTOSENSITIVITY; PRIORITY JOURNAL; PROGRESSION FREE SURVIVAL; PROTEINURIA; PRURITUS; QT PROLONGATION; RANDOMIZED CONTROLLED TRIAL (TOPIC); RASH; SALVAGE THERAPY; STOMATITIS; THROMBOCYTOPENIA; THYROID MEDULLARY CARCINOMA; TREATMENT RESPONSE; TUMOR GROWTH; UNSPECIFIED SIDE EFFECT; VOMITING; WEIGHT REDUCTION; ANIMAL; ANTAGONISTS AND INHIBITORS; CARCINOMA, NEUROENDOCRINE; DRUG EFFECTS; ENZYMOLOGY; GENETIC PREDISPOSITION; GENETICS; METABOLISM; MOLECULARLY TARGETED THERAPY; MUTATION; PATHOLOGY; PATIENT SELECTION; PHENOTYPE; RISK FACTOR; SIGNAL TRANSDUCTION; THYROID NEOPLASMS; TREATMENT OUTCOME;

ANIMALS; ANTINEOPLASTIC AGENTS; CARCINOMA, NEUROENDOCRINE; GENETIC PREDISPOSITION TO DISEASE; HUMANS; MOLECULAR TARGETED THERAPY; MUTATION; PATIENT SELECTION; PHENOTYPE; PROTEIN KINASE INHIBITORS; PROTO-ONCOGENE PROTEINS C-RET; RISK FACTORS; SIGNAL TRANSDUCTION; THYROID NEOPLASMS; TREATMENT OUTCOME;

EID: 84944888408     PISSN: 00800015     EISSN: 21976767     Source Type: Book Series    
DOI: 10.1007/978-3-319-22542-5_11     Document Type: Chapter

References (41)

1. Agrawal N, Jiao Y, Sausen M, Leary R, Bettegowda C, Roberts NJ, Bhan S, Ho AS, Khan Z, Bishop J, Westra WH, Wood LD, Hruban RH, Tufano RP, Robinson B, Dralle H, Toledo SP, Toledo RA, Morris LG, Ghossein RA, Fagin JA, Chan TA, Velculescu VE, Vogelstein B, Kinzler KW, Papadopoulos N, Nelkin BD, Ball DW (2013) Exomic sequencing of medullary thyroid cancer reveals dominant and mutually exclusive oncogenic mutations in RET and RAS. J Clin Endocrinol Metab 98(2):E364-E369. doi:10.1210/jc.2012-2703
2. Bagheri-Yarmand R, Sinha KM, Gururaj AE, Ahmed Z, Rizvi YQ, Huang SC, Ladbury JE, Bogler O, Williams MD, Cote GJ, Gagel RF (2015) A novel dual kinase function of the RET proto-oncogene negatively regulates activating transcription factor 4-mediated apoptosis. J Biol Chem 290(18):11749-11761. doi:10.1074/jbc.M114.619833
3. Barroso-Sousa R, Lerario AM, Evangelista J, Papadia C, Lourenco DM Jr, Lin CS, Kulcsar MA, Fragoso MC, Hoff AO (2014) Complete resolution of hypercortisolism with sorafenib in a patient with advanced medullary thyroid carcinoma and ectopic ACTH (adrenocorticotropic hormone) syndrome. Thyroid Official J Am Thyroid Assoc 24(6):1062-1066. doi:10.1089/thy.2013.0571
4. Bible KC, Suman VJ, Molina JR, Smallridge RC, Maples WJ, Menefee ME, Rubin J, Karlin N, Sideras K, Morris JC, 3rd, McIver B, Hay I, Fatourechi V, Burton JK, Webster KP, Bieber C, Traynor AM, Flynn PJ, Cher Goh B, Isham CR, Harris P, Erlichman C, Endocrine Malignancies Disease Oriented Group MCCC, the Mayo Phase C (2014) A multicenter phase 2 trial of pazopanib in metastatic and progressive medullary thyroid carcinoma: MC057H. J Clin Endocrinol Metab 99(5):1687-1693. doi:10.1210/jc.2013-3713
5. Boichard A, Croux L, Al Ghuzlan A, Broutin S, Dupuy C, Leboulleux S, Schlumberger M, Bidart JM, Lacroix L (2012) Somatic RAS mutations occur in a large proportion of sporadic RET-negative medullary thyroid carcinomas and extend to a previously unidentified exon. J Clin Endocrinol Metab 97(10):E2031-E2035. doi:10.1210/jc.2012-2092
6. Brassard M, Neraud B, Trabado S, Salenave S, Brailly-Tabard S, Borget I, Baudin E, Leboulleux S, Chanson P, Schlumberger M, Young J (2011) Endocrine effects of the tyrosine kinase inhibitor vandetanib in patients treated for thyroid cancer. J Clin Endocrinol Metab 96 (9):2741-2749. doi:10.1210/jc.2010-2771
7. Carlomagno F, Vitagliano D, Guida T, Ciardiello F, Tortora G, Vecchio G, Ryan AJ, Fontanini G, Fusco A, Santoro M (2002) ZD6474, an orally available inhibitor of KDR tyrosine kinase activity, efficiently blocks oncogenic RET kinases. Cancer Res 62(24):7284-7290
8. Carr LL, Mankoff DA, Goulart BH, Eaton KD, Capell PT, Kell EM, Bauman JE, Martins RG (2010) Phase II study of daily sunitinib in FDG-PET-positive, iodine-refractory differentiated thyroid cancer and metastatic medullary carcinoma of the thyroid with functional imaging correlation. Clin Cancer Res Official J Am Assoc Cancer Res 16(21):5260-5268. doi:10.1158/1078-0432.CCR-10-0994
9. Cho YT, Chan CC (2013) Cabozantinib-induced hand-foot skin reaction with subungual splinter hemorrhages and hypertension: a possible association with inhibition of the vascular endothelial growth factor signaling pathway. Eur J Dermatol EJD 23(2):274-275. doi:10.1684/ejd.2013.1930
10. Dadu R, Devine C, Hernandez M, Waguespack SG, Busaidy NL, Hu MI, Jimenez C, Habra MA, Sellin RV, Ying AK, Cote GJ, Sherman SI, Cabanillas ME (2014) Role of salvage targeted therapy in differentiated thyroid cancer patients who failed first-line sorafenib. J Clin Endocrinol Metab 99(6):2086-2094. doi:10.1210/jc.2013-3588
11. Donis-Keller H, Dou S, Chi D, Carlson KM, Toshima K, Lairmore TC, Howe JR, Moley JF, Goodfellow P, Wells SA Jr (1993) Mutations in the RET proto-oncogene are associated with MEN 2A and FMTC. Hum Mol Genet 2(7):851-856
12. Elisei R, Schlumberger MJ, Muller SP, Schoffski P, Brose MS, Shah MH, Licitra L, Jarzab B, Medvedev V, Kreissl MC, Niederle B, Cohen EE, Wirth LJ, Ali H, Hessel C, Yaron Y, Ball D, Nelkin B, Sherman SI (2013) Cabozantinib in progressive medullary thyroid cancer. J Clin Oncol Official J Am Soc Clin Oncol 31(29):3639-3646. doi:10.1200/JCO.2012.48.4659
13. Faggiano A, Ramundo V, Dicitore A, Castiglioni S, Borghi MO, Severino R, Ferolla P, Crino L, Abbruzzese A, Sperlongano P, Caraglia M, Ferone D, Hofland L, Colao A, Vitale G (2012) Everolimus is an active agent in medullary thyroid cancer: a clinical and in vitro study. J Cell Mol Med 16(7):1563-1572. doi:10.1111/j.1582-4934.2011.01438.x
14. Fox E, Widemann BC, Chuk MK, Marcus L, Aikin A, Whitcomb PO, Merino MJ, Lodish M, Dombi E, Steinberg SM, Wells SA, Balis FM (2013) Vandetanib in children and adolescents with multiple endocrine neoplasia type 2B associated medullary thyroid carcinoma. Clin Cancer Res Official J Am Assoc Cancer Res 19(15):4239-4248. doi:10.1158/1078-0432.CCR-13-0071
15. Gild ML, Landa I, Ryder M, Ghossein RA, Knauf JA, Fagin JA (2013) Targeting mTOR in RET mutant medullary and differentiated thyroid cancer cells. Endocr Relat Cancer 20(5):659-667. doi:10.1530/ERC-13-0085
16. Goutas N, Vlachodimitropoulos D, Bouka M, Lazaris AC, Nasioulas G, Gazouli M (2008) BRAF and K-RAS mutation in a Greek papillary and medullary thyroid carcinoma cohort. Anticancer Res 28(1):305-308
17. Hsu SC, Hung MC (2007) Characterization of a novel tripartite nuclear localization sequence in the EGFR family. J Biol Chem 282(14):10432-10440. doi:10.1074/jbc.M610014200
18. Ibanez CF (2013) Structure and physiology of the RET receptor tyrosine kinase. Cold Spring Harb Perspect Biol 5(2). doi:10.1101/cshperspect.a009134
19. Joensuu H, Roberts PJ, Sarlomo-Rikala M, Andersson LC, Tervahartiala P, Tuveson D, Silberman S, Capdeville R, Dimitrijevic S, Druker B, Demetri GD (2001) Effect of the tyrosine kinase inhibitor STI571 in a patient with a metastatic gastrointestinal stromal tumor. N Eng J Med 344(14):1052-1056. doi:10.1056/NEJM200104053441404
20. Kurzrock R, Sherman SI, Ball DW, Forastiere AA, Cohen RB, Mehra R, Pfister DG, Cohen EE, Janisch L, Nauling F, Hong DS, Ng CS, Ye L, Gagel RF, Frye J, Muller T, Ratain MJ, Salgia R (2011) Activity of XL184 (Cabozantinib), an oral tyrosine kinase inhibitor, in patients with medullary thyroid cancer. J Clin Oncol Official J Am Soc Clin Oncol 29(19):2660-2666. doi:10.1200/JCO.2010.32.4145
21. Laure Giraudet A, Al Ghulzan A, Auperin A, Leboulleux S, Chehboun A, Troalen F, Dromain C, Lumbroso J, Baudin E, Schlumberger M (2008) Progression of medullary thyroid carcinoma: assessment with calcitonin and carcinoembryonic antigen doubling times. Eur J Endocrinol Eur Fed Endocr Soc 158(2):239-246. doi:10.1530/EJE-07-0667
22. Lemmon MA, Schlessinger J, Ferguson KM (2014) The EGFR family: not so prototypical receptor tyrosine kinases. Cold Spring Harb Perspect Biol 6(4):a020768. doi:10.1101/cshperspect.a020768
23. Lyra J, Vinagre J, Batista R, Pinto V, Prazeres H, Rodrigues F, Eloy C, Sobrinho-Simoes M, Soares P (2014) MTOR activation in medullary thyroid carcinoma with RAS mutation. Eur J Endocrinol Eur Fed Endocr Soc 171(5):633-640. doi:10.1530/EJE-14-0389
24. Moura MM, Cavaco BM, Pinto AE, Leite V (2011) High prevalence of RAS mutations in RET-negative sporadic medullary thyroid carcinomas. J Clin Endocrinol Metab 96(5):E863-E868. doi:10.1210/jc.2010-1921
25. Mulligan LM, Kwok JB, Healey CS, Elsdon MJ, Eng C, Gardner E, Love DR, Mole SE, Moore JK, Papi L et al (1993) Germ-line mutations of the RET proto-oncogene in multiple endocrine neoplasia type 2A. Nature 363(6428):458-460. doi:10.1038/363458a0
26. Nella AA, Lodish MB, Fox E, Balis FM, Quezado MM, Whitcomb PO, Derdak J, Kebebew E, Widemann BC, Stratakis CA (2014) Vandetanib successfully controls medullary thyroid cancer-related Cushing syndrome in an adolescent patient. J Clin Endocrinol Metab 99 (9):3055-3059. doi:10.1210/jc.2013-4340
27. Pagliarini R, Shao W, Sellers WR (2015) Oncogene addiction: pathways of therapeutic response, resistance, and road maps toward a cure. EMBO Rep 16(3):280-296. doi:10.15252/embr.201439949
28. Robinson BG, Paz-Ares L, Krebs A, Vasselli J, Haddad R (2010) Vandetanib (100 mg) in patients with locally advanced or metastatic hereditary medullary thyroid cancer. J Clin Endocrinol Metab 95(6):2664-2671. doi:10.1210/jc.2009-2461
29. Santoro M, Carlomagno F (2013) Central role of RET in thyroid cancer. Cold Spring Harb Perspect Biol 5(12):a009233. doi:10.1101/cshperspect.a009233
30. Schlumberger MJ, Elisei R, Bastholt L, Wirth LJ, Martins RG, Locati LD, Jarzab B, Pacini F, Daumerie C, Droz JP, Eschenberg MJ, Sun YN, Juan T, Stepan DE, Sherman SI (2009) Phase II study of safety and efficacy of motesanib in patients with progressive or symptomatic, advanced or metastatic medullary thyroid cancer. J Clin Oncol Official J Am Soc Clin Oncol 27(23):3794-3801. doi:10.1200/JCO.2008.18.7815
31. Schlumberger M, Jarzab B, Cabanillas ME (2012) A Phase II trial of the multitargeted kinase inhibitor lenvatinib (E7080) in advanced medullary thyroid cancer (MTC). J Clin Oncol Official J Am Soc Clin Oncol 30; Suppl; Abstr 5591
32. Schlumberger M, Elisei R, Muller SP, Schoffski P, Brose MS, Shah MH, Licitra L, Jarzab B, Medvedev V, Kreissl MC, Cohen EE, Wirth LJ, Ali H, Hessel C, Yaron Y, Ball D, Nelkin B, Sherman SI (2015) Final overall survival analysis of EXAM, an international, doubleblind, randomized, placebocontrolled phase III trial of cabozantinib (Cabo) in medullarythyroid carcinoma (MTC) patients with documented RECIST progression at baseline. J Clin Oncol 33 suppl; abstr 6012
33. Shah RR, Morganroth J, Shah DR (2013) Cardiovascular safety of tyrosine kinase inhibitors: with a special focus on cardiac repolarisation (QT interval). Drug Saf 36(5):295-316. doi:10.1007/s40264-013-0047-5
34. Sherman SI, Wirth LJ, Droz JP, Hofmann M, Bastholt L, Martins RG, Licitra L, Eschenberg MJ, Sun YN, Juan T, Stepan DE, Schlumberger MJ, Motesanib Thyroid Cancer Study G (2008) Motesanib diphosphate in progressive differentiated thyroid cancer. N Eng J Med 359(1):31-42. doi:10.1056/NEJMoa075853
35. Sipple JH (1961) The association of pheochromocytoma with carccinoma of the thyroid gland. Am J Med 31:163-166
36. Stratton MR, Campbell PJ, Futreal PA (2009) The cancer genome. Nature 458(7239):719-724. doi:10.1038/nature07943
37. Tsao AS, Liu S, Lee JJ, Alden CM, Blumenschein GR Jr, Herbst R, Davis SE, Kim E, Lippman S, Heymach J, Tran H, Tang X, Wistuba I, Hong WK (2013) Clinical and biomarker outcomes of the phase II vandetanib study from the BATTLE trial. J Thorac Oncol Official Pub Int Assoc Study Lung Cancer 8(5):658-661. doi:10.1097/JTO.0b013e31828d08ae
38. Wells SA Jr, Gosnell JE, Gagel RF, Moley J, Pfister D, Sosa JA, Skinner M, Krebs A, Vasselli J, Schlumberger M (2010) Vandetanib for the treatment of patients with locally advanced or metastatic hereditary medullary thyroid cancer. J Clin Oncol Official J Am Soc Clin Oncol 28 (5):767-772. doi:10.1200/JCO.2009.23.6604
39. Wells SA Jr, Robinson BG, Gagel RF, Dralle H, Fagin JA, Santoro M, Baudin E, Elisei R, Jarzab B, Vasselli JR, Read J, Langmuir P, Ryan AJ, Schlumberger MJ (2012) Vandetanib in patients with locally advanced or metastatic medullary thyroid cancer: a randomized, double-blind phase III trial. J Clin Oncol Official J Am Soc Clin Oncol 30(2):134-141. doi:10.1200/JCO.2011.35.5040
40. Wells SA Jr, Asa SL, Dralle H, Elisei R, Evans DB, Gagel RF, Lee N, Machens A, Moley JF, Pacini F, Raue F, Frank-Raue K, Robinson B, Rosenthal MS, Santoro M, Schlumberger M, Shah M, Waguespack SG (2015) Revised american thyroid association guidelines for the management of medullary thyroid carcinoma. Thyroid Official J Am Thyroid Assoc 25 (6):567-610. doi:10.1089/thy.2014.0335
41. Wohllk N, Cote GJ, Bugalho MM, Ordonez N, Evans DB, Goepfert H, Khorana S, Schultz P, Richards CS, Gagel RF (1996) Relevance of RET proto-oncogene mutations in sporadic medullary thyroid carcinoma. J Clin Endocrinol Metab 81(10):3740-3745. doi:10.1210/jcem.81.10.8855832