Tivantinib (ARQ 197), a selective inhibitor of MET, in patients with microphthalmia transcription factor-associated tumors: Results of a multicenter phase 2 trial

Cancer

Volumn 118, Issue 23, 2012, Pages 5894-5902

  Wagner, Andrew J ^a   Goldberg, John M ^b   Dubois, Steven G ^c   Choy, Edwin ^d   Lee, Rosen ^e   Pappo, Alberto ^f   Geller, James ^g   Judson, Ian ^h   Hogg, David ⁱ   Senzer, Neil ^j   Davis, Ian J ^k   Feng, Chai ^l   Waghorne, Carol ^l   Schwartz, Brian ^l   Demetri, George D ^a  

^a DANA FARBER CANCER INSTITUTE   (United States)

^b UNIVERSITY OF MIAMI MILLER SCHOOL OF MEDICINE   (United States)

^c UNIVERSITY OF CALIFORNIA   (United States)

^d MASSACHUSETTS GENERAL HOSPITAL   (United States)

^e UNIVERSITY OF CALIFORNIA   (United States)

^f ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^g CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^h INSTITUTE OF CANCER RESEARCH   (United Kingdom)

ⁱ UNIVERSITY OF TORONTO   (Canada)

^j Mary Crowley Cancer Research Centers   (United States)

^k UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

^l ARQULE INC   (United States)

more..

Author keywords

alveolar soft part sarcoma; antagonists and inhibitors; ARQ 197; clear cell sarcoma; microphthalmia associated transcription factor; pediatric; protein kinase inhibitors pharmacokinetics; proto oncogene proteins c met ; renal cell carcinoma; tivantinib

Indexed keywords

SCATTER FACTOR RECEPTOR; TIVANTINIB;

ADOLESCENT; ADULT; AGE DISTRIBUTION; AGED; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ANOREXIA; ANTINEOPLASTIC ACTIVITY; AREA UNDER THE CURVE; ARTICLE; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; CANCER GRADING; CANCER SURVIVAL; CHEMOTHERAPY INDUCED ANEMIA; CHEMOTHERAPY INDUCED EMESIS; CHILD; CLINICAL ARTICLE; CONTROLLED STUDY; COUGHING; DEEP VEIN THROMBOSIS; DIARRHEA; DRUG BLOOD LEVEL; DRUG DOSE ESCALATION; DRUG DOSE INCREASE; DRUG EFFICACY; DRUG ERUPTION; DRUG FEVER; DRUG INDUCED HEADACHE; DRUG RESPONSE; DRUG SAFETY; DRUG TARGETING; DRUG TOLERABILITY; DYSPNEA; FATIGUE; FEBRILE NEUTROPENIA; FEMALE; GENE EXPRESSION; HUMAN; INSOMNIA; LYMPHOCYTOPENIA; MALE; MALIGNANT NEOPLASTIC DISEASE; MAXIMUM PLASMA CONCENTRATION; MICROPHTHALMIA TRANSCRIPTION FACTOR ASSOCIATED TUMOR; MULTICENTER STUDY; MULTIPLE CYCLE TREATMENT; NEUTROPENIA; ONCOGENE; ONCOGENE MET; OVERALL SURVIVAL; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; PROGRESSION FREE SURVIVAL; RETCHING; SCHOOL CHILD; SIDE EFFECT; SINGLE BLIND PROCEDURE; SINUS BRADYCARDIA; THROMBOCYTOPENIA;

ADOLESCENT; ADULT; AGED; CARCINOMA, RENAL CELL; CHILD; FEMALE; HUMANS; IMMUNOHISTOCHEMISTRY; KIDNEY NEOPLASMS; MALE; MICROPHTHALMIA-ASSOCIATED TRANSCRIPTION FACTOR; MIDDLE AGED; NEOPLASMS; PROTO-ONCOGENE PROTEINS C-MET; PYRROLIDINONES; QUINOLINES; SARCOMA, ALVEOLAR SOFT PART; SARCOMA, CLEAR CELL;

EID: 84869493002     PISSN: 0008543X     EISSN: 10970142     Source Type: Journal    
DOI: 10.1002/cncr.27582     Document Type: Article

References (47)

1. Davis IJ, Fisher DE,. MiT transcription factor associated malignancies in man. Cell Cycle. 2007; 6: 1724-1729. (Pubitemid 47327925)
2. Steingrimsson E, Copeland NG, Jenkins NA,. Melanocytes and the microphthalmia transcription factor network. Annu Rev Genet. 2004; 38: 365-411. (Pubitemid 40013733)
3. Beckmann H, Kadesch T,. The leucine zipper of TFE3 dictates helix-loop-helix dimerization specificity. Genes Dev. 1991; 5: 1057-1066. (Pubitemid 21906018)
4. Fisher DE, Carr CS, Parent LA, Sharp PA,. TFEB has DNA-binding and oligomerization properties of a unique helix-loop-helix/leucine-zipper family. Genes Dev. 1991; 5: 2342-2352. (Pubitemid 21905922)
5. Hemesath TJ, Steingrímsson E, McGill G, et al. microphthalmia, a critical factor in melanocyte development, defines a discrete transcription factor family. Genes Dev. 1994; 8: 2770-2780. (Pubitemid 24375425)
6. Carreira S, Goodall J, Aksan I, et al. Mitf cooperates with Rb1 and activates p21Cip1 expression to regulate cell cycle progression. Nature. 2005; 433: 764-769. (Pubitemid 40292987)
7. McGill GG, Haq R, Nishimura EK, Fisher DE,. c-Met expression is regulated by Mitf in the melanocyte lineage. J Biol Chem. 2006; 281: 10365-10373. (Pubitemid 43864575)
8. McGill GG, Horstmann M, Widlund HR, et al. Bcl2 regulation by the melanocyte master regulator Mitf modulates lineage survival and melanoma cell viability. Cell. 2002; 109: 707-718. (Pubitemid 34722267)
9. Davis IJ, Kim JJ, Ozsolak F, et al. Oncogenic MITF dysregulation in clear cell sarcoma: Defining the MiT family of human cancers. Cancer Cell. 2006; 9: 473-484. (Pubitemid 43832189)
10. Garraway LA, Widlund HR, Rubin MA, et al. Integrative genomic analyses identify MITF as a lineage survival oncogene amplified in malignant melanoma. Nature. 2005; 436: 117-122. (Pubitemid 40966196)
11. Argani P, Antonescu CR, Illei PB, et al. Primary renal neoplasms with the ASPL-TFE3 gene fusion of alveolar soft part sarcoma: a distinctive tumor entity previously included among renal cell carcinomas of children and adolescents. Am J Pathol. 2001; 159: 179-192. (Pubitemid 33704203)
12. Argani P, Lui MY, Couturier J, Bouvier R, Fournet JC, Ladanyi M,. A novel CLTC-TFE3 gene fusion in pediatric renal adenocarcinoma with t(X;17)(p11.2;q23). Oncogene. 2003; 22: 5374-5378. (Pubitemid 37056101)
13. Clark J, Lu YJ, Sidhar SK, et al. Fusion of splicing factor genes PSF and NonO (p54nrb) to the TFE3 gene in papillary renal cell carcinoma. Oncogene. 1997; 15: 2233-2239. (Pubitemid 27493913)
14. Heimann P, El Housni H, Ogur G, Weterman MA, Petty EM, Vassart G,. Fusion of a novel gene, RCC17, to the TFE3 gene in t(X;17)(p11.2;q25.3)-bearing papillary renal cell carcinomas. Cancer Res. 2001; 61: 4130-4135. (Pubitemid 32720981)
15. Ladanyi M, Lui MY, Antonescu CR, et al. The der(17)t(X;17)(p11;q25) of human alveolar soft part sarcoma fuses the TFE3 transcription factor gene to ASPL, a novel gene at 17q25. Oncogene. 2001; 20: 48-57. (Pubitemid 32142337)
16. Lee M, Cho KJ, Yu C, et al. Perivascular epithelioid cell tumor of the sigmoid colon with transcription factor E3 expression. Ann Diagn Pathol. 2011; doi:10.1016/j.anndiagpath.2011.01.007
17. Davis IJ, McFadden AW, Zhang Y, et al. Identification of the receptor tyrosine kinase c-Met and its ligand, hepatocyte growth factor, as therapeutic targets in clear cell sarcoma. Cancer Res. 2010; 70: 639-645.
18. Beviglia L, Matsumoto K, Lin CS, Ziober BL, Kramer RH,. Expression of the c-Met/HGF receptor in human breast carcinoma: correlation with tumor progression. Int J Cancer. 1997; 74: 301-309. (Pubitemid 27321800)
19. Birchmeier C, Birchmeier W, Gherardi E, Vande Woude GF,. Met, metastasis, motility and more. Nat Rev Mol Cell Biol. 2003; 4: 915-925. (Pubitemid 37493640)
20. Furge KA, Kiewlich D, Le P, et al. Suppression of Ras-mediated tumorigenicity and metastasis through inhibition of the Met receptor tyrosine kinase. Proc Natl Acad Sci U S A. 2001; 98: 10722-10727. (Pubitemid 32878688)
21. Ma PC, Tretiakova MS, MacKinnon AC, et al. Expression and mutational analysis of MET in human solid cancers. Genes Chromosomes Cancer. 2008; 47: 1025-1037.
22. Qian CN, Guo X, Cao B, et al. Met protein expression level correlates with survival in patients with late-stage nasopharyngeal carcinoma. Cancer Res. 2002; 62: 589-596. (Pubitemid 34106436)
23. Qiao H, Hung W, Tremblay E, et al. Constitutive activation of met kinase in non-small-cell lung carcinomas correlates with anchorage-independent cell survival. J Cell Biochem. 2002; 86: 665-677. (Pubitemid 34839968)
24. Takeuchi H, Bilchik A, Saha S, et al. c-MET expression level in primary colon cancer: a predictor of tumor invasion and lymph node metastases. Clin Cancer Res. 2003; 9: 1480-1488. (Pubitemid 36418405)
25. Takayama H, LaRochelle WJ, Sharp R, et al. Diverse tumorigenesis associated with aberrant development in mice overexpressing hepatocyte growth factor/scatter factor. Proc Natl Acad Sci U S A. 1997; 94: 701-706.
26. Tsuda M, Davis IJ, Argani P, et al. TFE3 fusions activate MET signaling by transcriptional up-regulation, defining another class of tumors as candidates for therapeutic MET inhibition. Cancer Res. 2007; 67: 919-929.
27. Munshi N, Jeay S, Li Y, et al. ARQ 197, a novel and selective inhibitor of the human c-Met receptor tyrosine kinase with antitumor activity. Mol Cancer Ther. 2010; 9: 1544-1553.
28. Yap TA, Olmos D, Brunetto AT, et al. Phase I trial of a selective c-MET inhibitor ARQ 197 incorporating proof of mechanism pharmacodynamic studies. J Clin Oncol. 2011; 29: 1271-1279.
29. Therasse P, Arbuck SG, Eisenhauer EA, et al. New guidelines to evaluate the response to treatment in solid tumors. European Organization for Research and Treatment of Cancer, National Cancer Institute of the United States, National Cancer Institute of Canada. J Natl Cancer Inst. 2000; 92: 205-216.
30. Simon R,. Optimal two-stage designs for phase II clinical trials. Control Clin Trials. 1989; 10: 1-10. (Pubitemid 19099520)
31. van Ruth S, van Coevorden F, Peterse JL, Kroon BB,. Alveolar soft part sarcoma. a report of 15 cases. Eur J Cancer. 2002; 38: 1324-1328. (Pubitemid 34686841)
32. Skubitz KM, Adamo DR,. Sarcoma. Mayo Clin Proc. 2007; 82: 1409-1432. (Pubitemid 350060138)
33. Jones RL, Constantinidou A, Thway K, et al. Chemotherapy in clear cell sarcoma. Med Oncol. 2010; 28: 859-863.
34. Schaefer KL, Brachwitz K, Wai DH, et al. Expression profiling of t(12;22) positive clear cell sarcoma of soft tissue cell lines reveals characteristic up-regulation of potential new marker genes including ERBB3. Cancer Res. 2004; 64: 3395-3405. (Pubitemid 38657911)
35. Segal NH, Pavlidis P, Antonescu CR, et al. Classification and subtype prediction of adult soft tissue sarcoma by functional genomics. Am J Pathol. 2003; 163: 691-700. (Pubitemid 36909430)
36. Segal NH, Pavlidis P, Noble WS, et al. Classification of clear-cell sarcoma as a subtype of melanoma by genomic profiling. J Clin Oncol. 2003; 21: 1775-1781. (Pubitemid 46638591)
37. Ghose A, Tariq Z, Veltri S,. Treatment of multidrug resistant advanced alveolar soft part sarcoma with sunitinib. Am J Ther. 2012; 19: e56-e58.
38. Stacchiotti S, Grosso F, Negri T, et al. Tumor response to sunitinib malate observed in clear-cell sarcoma. Ann Oncol. 2010; 21: 1130-1131.
39. Stacchiotti S, Negri T, Zaffaroni N, et al. Sunitinib in advanced alveolar soft part sarcoma: evidence of a direct antitumor effect. Ann Oncol. 2011; 22: 1682-1690.
40. Stacchiotti S, Tamborini E, Marrari A, et al. Response to sunitinib malate in advanced alveolar soft part sarcoma. Clin Cancer Res. 2009; 15: 1096-1104.
41. Gardner K, Judson I, Leahy M, et al. Activity of cediranib, a highly potent and selective VEGF signaling inhibitor, in alveolar soft part sarcoma [abstract]. J Clin Oncol. 2009; 27 (15 suppl): 541s. Abstract 10523.
42. Azizi AA, Haberler C, Czech T, et al. Vascular-endothelial-growth-factor (VEGF) expression and possible response to angiogenesis inhibitor bevacizumab in metastatic alveolar soft part sarcoma. Lancet Oncol. 2006; 7: 521-523. (Pubitemid 43779166)
43. Choueiri TK, Mosquera JM, Hirsch MS,. A case of adult metastatic Xp11 translocation renal cell carcinoma treated successfully with sunitinib. Clin Genitourin Cancer. 2009; 7: E93-E94.
44. Malouf GG, Camparo P, Oudard S, et al. Targeted agents in metastatic Xp11 translocation/TFE3 gene fusion renal cell carcinoma (RCC): a report from the Juvenile RCC Network. Ann Oncol. 2010; 21: 1834-1838.
45. Schiller JH, Akerley WL, Brugger W, et al. Results from ARQ 197-209: a global randomized placebo-controlled phase 2 clinical trial of erlotinib plus ARQ 197 versus erlotinib plus placebo in previously treated EGFR inhibitor-naïve patients with locally advanced or metastatic non-small cell lung cancer [abstract]. J Clin Oncol. 2010; 28 (15 suppl): 954s. Abstract LBA7502.
46. Sequist LV, Akerley WL, Brugger W, et al. Final results from ARQ 197-209: a global randomized placebo-controlled phase 2 clinical trial of erlotinib plus ARQ 197 versus erlotinib plus placebo in previously treated EGFR-inhibitor naïve patients with advanced non-small cell lung cancer [abstract]. Presented at: 35th ESMO Congress; October 8-12, 2010; Milan, Italy. Abstract 363O.
47. Eng C, Bendell J, Bessudo A, et al. Phase I results of the randomized, placebo controlled, phase I/II study of the novel oral c-Met inhibitor, ARQ 197, irinotecan (CPT-11), and cetuximab (C) in patients (pts) with wild-type (WT) KRAS metastatic colorectal cancer (mCRC) who have received front-line systemic therapy [abstract]. J Clin Oncol. 2011; 29:Abstract 527.