Targeted therapy for endocrine cancer: The medullary thyroid carcinoma paradigm

Endocrine Practice

Volumn 15, Issue 6, 2009, Pages 597-604

  Ye, Lei ^a   Santarpia, Libero ^a   Gagel, Robert F ^a  

^a UNIVERSITY OF TEXAS   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CALCITONIN; CARCINOEMBRYONIC ANTIGEN; E 7080; GEFITINIB; IMATINIB; MAMMALIAN TARGET OF RAPAMYCIN INHIBITOR; MOTESANIB; PROTEIN RET; PROTEIN TYROSINE KINASE INHIBITOR; SORAFENIB; SUNITINIB; TIPIFARNIB; TYROSINE KINASE RECEPTOR; UNCLASSIFIED DRUG; VANDETANIB; XL 184; ANTINEOPLASTIC AGENT; PROTEIN KINASE INHIBITOR; PROTEIN TYROSINE KINASE;

ANTINEOPLASTIC ACTIVITY; BLEEDING; CANCER SURVIVAL; CANCER THERAPY; CARCINOID; CARDIOMYOPATHY; CLINICAL TRIAL; CONGESTIVE HEART FAILURE; DIARRHEA; DIFFERENTIATED THYROID CARCINOMA; DRUG DOSE REDUCTION; ENDOCRINE TUMOR; FATIGUE; FOOD AND DRUG ADMINISTRATION; GASTROINTESTINAL STROMAL TUMOR; GENE MUTATION; GENETIC DISORDER; HAND FOOT SYNDROME; HEART FAILURE; HUMAN; HYPERTENSION; KIDNEY DYSFUNCTION; LUNG CANCER; LUNG METASTASIS; MEDICAL INFORMATION; MEDICAL LITERATURE; METASTASIS; MULTIPLE CYCLE TREATMENT; NAUSEA; NAUSEA AND VOMITING; NONHUMAN; OVERALL SURVIVAL; PANCREAS ISLET CELL CARCINOMA; PHEOCHROMOCYTOMA; PREVALENCE; QT PROLONGATION; REVIEW; SIDE EFFECT; SIGNAL TRANSDUCTION; SKIN TOXICITY; THYROID MEDULLARY CARCINOMA; THYROID PAPILLARY CARCINOMA; TREATMENT DURATION; TREATMENT RESPONSE; TUMOR GROWTH; TUMOR VOLUME; UNITED STATES; WEIGHT REDUCTION; DRUG ANTAGONISM; GENETICS; MEDULLARY CARCINOMA; METABOLISM; THYROID TUMOR;

ANTINEOPLASTIC AGENTS; CARCINOMA, MEDULLARY; CLINICAL TRIALS AS TOPIC; ENDOCRINE GLAND NEOPLASMS; HUMANS; PROTEIN KINASE INHIBITORS; RECEPTOR PROTEIN-TYROSINE KINASES; THYROID NEOPLASMS;

EID: 75549089866     PISSN: 1530891X     EISSN: None     Source Type: Journal    
DOI: 10.4158/EP09148.RAR     Document Type: Review

References (41)

1. Smallridge RC, Marlow LA, Copland JA. Anaplastic thyroid cancer: molecular pathogenesis and emerging therapies. Endocr Relat Cancer. 2009;16:17-44.
2. Rodgers SE, Evans DB, Lee JE, Perrier ND. Adrenocortical carcinoma. Surg Oncol Clin N Am. 2006;15: 535-553.
3. Nocera M, Baudin E, Pellegriti G, Cailleux AF, Mechelany-Corone C, Schlumberger M (Groupe d'Etude des Tumeurs à Calcitonine [GETC]). Treatment of advanced medullary thyroid cancer with an alternating combination of doxorubicin-streptozocin and 5 FU-dacarbazine. Br J Cancer. 2000;83:715-718.
4. Juweid ME, Hajjar G, Swayne LC, et al. Phase I/II trial of (131)I-MN-14F(ab)2 anti-carcinoembryonic antigen monoclonal antibody in the treatment of patients with metastatic medullary thyroid carcinoma. Cancer. 1999;85:1828-1842.
5. Jiménez C, Hu MI, Gagel RF. Management of medullary thyroid carcinoma. Endocrinol Metab Clin North Am. 2008;37:481-496, x-xi.
6. Kaltsas GA, Besser GM, Grossman AB. The diagnosis and medical management of advanced neuroendocrine tumors. Endocr Rev. 2004;25:458-511.
7. Mulligan LM, Kwok JB, Healey CS, et al. Germ-line mutations of the RET proto-oncogene in multiple endocrine neoplasia type 2A. Nature. 1993;363:458-460.
8. Donis-Keller H, Dou S, Chi D, et al. Mutations in the RET proto-oncogene are associated with MEN 2a and FMTC. Hum Mol Genet. 1993;2:851-856.
9. Cote GJ, Gagel RF. Lessons learned from the management of a rare genetic cancer. N Engl J Med. 2003;349:1566-1568.
10. Drosten M, Pützer BM. Mechanisms of disease: cancer targeting and the impact of oncogenic RET for medullary thyroid carcinoma therapy. Nat Clin Pract Oncol. 2006;3:564-574.
11. Lugo TG, Pendergast AM, Muller AJ, Witte ON. Tyrosine kinase activity and transformation potency of bcr-abl oncogene products. Science. 1990;247:1079-1082.
12. Kantarjian H, Sawyers C, Hochhaus A, et al (International STI571 CML Study Group). Hematologic and cytogenetic responses to imatinib mesylate in chronic myelogenous leukemia [published correction appears in N Engl J Med. 2002;346:1923]. N Engl J Med. 2002;346: 645-652.
13. Joensuu H. Treatment of inoperable gastrointestinal stromal tumor (GIST) with imatinib (Glivec, Gleevec). Med Klin (Munich). 2002;97(suppl 1):28-30.
14. Druker BJ, Tamura S, Buchdunger E, et al. Effects of a selective inhibitor of the Abl tyrosine kinase on the growth of Bcr-Abl positive cells. Nat Med. 1996;2:561-566.
15. Tuveson DA, Willis NA, Jacks T, et al. STI571 inactivation of the gastrointestinal stromal tumor c-KIT oncoprotein: biological and clinical implications. Oncogene. 2001;20:5054-5058.
16. Lynch TJ, Bell DW, Sordella R, et al. Activating mutations in the epidermal growth factor receptor underlying responsiveness of non-small-cell lung cancer to gefitinib. N Engl J Med. 2004;350:2129-2139.
17. Therasse P, Arbuck SG, Eisenhauer EA, et al (European Organization for Research and Treatment of Cancer, National Cancer Institute of the United States, National Cancer Institute of Canada). New guidelines to evaluate the response to treatment in solid tumors. J Natl Cancer Inst. 2000;92:205-216.
18. Schlumberger MJ, Elisei R, Bastholt L, et al. Phase II study of safety and efficacy of motesanib in patients with progressive or symptomatic, advanced or metastatic medullary thyroid cancer. J Clin Oncol. 2009;27:3794-3801.
19. Akeno-Stuart N, Croyle M, Knauf JA, et al. The RET kinase inhibitor NVP-AST487 blocks growth and calcitonin gene expression through distinct mechanisms in medullary thyroid cancer cells. Cancer Res. 2007;67:6956-6964.
20. Carlomagno F, Vitagliano D, Guida T, et al. ZD6474, an orally available inhibitor of KDR tyrosine kinase activity, efficiently blocks oncogenic RET kinases. Cancer Res. 2002;62:7284-7290.
21. Wedge SR, Ogilvie DJ, Dukes M, et al. ZD6474 inhibits vascular endothelial growth factor signaling, angiogenesis, and tumor growth following oral administration. Cancer Res. 2002;62:4645-4655.
22. Wells S, You YN, Lakhani V, et al. A phase II trial of ZD6474 in patients with hereditary metastatic medullary thyroid cancer [abstract]. J Clin Oncol. 2006;24(18)(suppl):5533.
23. Wells SA Jr, Gosnell JE, Gagel RF, et al. Vandetanib in metastatic hereditary medullary thyroid cancer: followup results of an open-label phase II trial [abstract]. J Clin Oncol. 2007;25(18)(suppl):6018.
24. Salgia R, Sherman S, Hong DS, et al. A phase I study of XL184, a RET, VEGFR2, and MET kinase inhibitor, in patients (pts) with advanced malignancies, including pts with medullary thyroid cancer (MTC) [abstract]. J Clin Oncol. 2008;26(15)(suppl):3522.
25. Carlomagno F, Anaganti S, Guida T, et al. BAY 43-9006 inhibition of oncogenic RET mutants. J Natl Cancer Inst. 2006;98:326-334.
26. Hong D, Ye L, Gagel R, et al. Medullary thyroid cancer: targeting the RET kinase pathway with sorafenib/tipifarnib. Mol Cancer Ther. 2008;7:1001-1006.
27. Lam ET, Ringel MD, Kloos RT, et al. A phase II study of sorafenib in patients with metastatic medullary thyroid carcinoma. In: Proceedings of the 100th Annual Meeting of the American Association for Cancer Research; April 18-22, 2009; Denver, CO. Abstract 4513.
28. Kelleher FC, McDermott R. Response to sunitinib in medullary thyroid cancer. Ann Intern Med. 2008;148:567.
29. Carr L, Goulart B, Martins R, et al. Phase II trial of continuous dosing of sunitinib in advanced, FDG-PET avid, medullary thyroid carcinoma (MTC) and well-differentiated thyroid cancer (WDTC) [abstract]. J Clin Oncol. 2009:27(15)(suppl):6056.
30. Chu TF, Rupnick MA, Kerkela R, et al. Cardiotoxicity associated with tyrosine kinase inhibitor sunitinib. Lancet. 2007;370:2011-2019.
31. Sherman SI, Wirth LJ, Droz JP, et al (Motesanib Thyroid Cancer Study Group). Motesanib diphosphate in progressive differentiated thyroid cancer. N Engl J Med. 2008;359:31-42.
32. Kobayashi S, Boggon TJ, Dayaram T, et al. EGFR mutation and resistance of non-small-cell lung cancer to gefitinib. N Engl J Med. 2005;352:786-792.
33. Desai J, Shankar S, Heinrich MC, et al. Clonal evolution of resistance to imatinib in patients with metastatic gastrointestinal stromal tumors. Clin Cancer Res. 2007;13(18, pt 1):5398-5405.
34. Miselli FC, Casieri P, Negri T, et al. C-Kit/PDGFRA gene status alterations possibly related to primary imatinib resistance in gastrointestinal stromal tumors. Clin Cancer Res. 2007;13:2369-2377.
35. Castellone MD, Carlomagno F, Salvatore G, Santoro M. Receptor tyrosine kinase inhibitors in thyroid cancer. Best Pract Res Clin Endocrinol Metab. 2008;22:1023-1038.
36. Jimenez C, Cabanillas ME, Santarpia L, et al. Use of the tyrosine kinase inhibitor sunitinib in a patient with von Hippel-Lindau disease: targeting angiogenic factors in pheochromocytoma and other von Hippel-Lindau disease-related tumors. J Clin Endocrinol Metab. 2009;94:386-391.
37. Joshua AM, Ezzat S, Asa SL, et al. Rationale and evidence for sunitinib in the treatment of malignant paraganglioma/pheochromocytoma. J Clin Endocrinol Metab. 2009;94:5-9.
38. Druce MR, Kaltsas GA, Fraenkel M, Gross DJ, Grossman AB. Novel and evolving therapies in the treatment of malignant phaeochromocytoma: experience with the mTOR inhibitor everolimus (RAD001) [published online ahead of print May 7, 2009]. Horm Metab Res. doi:10.1055/s-0029-1220687.
39. Yao JC, Phan AT, Chang DZ, et al. Efficacy of RAD001 (everolimus) and octreotide LAR in advanced low- to intermediate-grade neuroendocrine tumors: results of a phase II study [published correction appears in J Clin Oncol. 2008;26:5660]. J Clin Oncol. 2008;26:4311-4318.
40. Zitzmann K, De Toni EN, Brand S, et al. The novel mTOR inhibitor RAD001 (everolimus) induces antiproliferative effects in human pancreatic neuroendocrine tumor cells. Neuroendocrinology. 2007;85:54-60.
41. Vetter ML, Kaul S, Iqbal N. Tyrosine kinase inhibitors and the thyroid as both an unintended and an intended target. Endocr Pract. 2008;14:618-624.