Elevating the Horizon: Emerging Molecular and Genomic Targets in the Treatment of Advanced Urothelial Carcinoma

Clinical Genitourinary Cancer

Volumn 13, Issue 5, 2015, Pages 410-420

  Kurtoglu, Metin ^a   Davarpanah, Nicole N ^a   Qin, Rui ^b   Powles, Thomas ^c   Rosenberg, Jonathan E ^d   Apolo, Andrea B ^a  

^a NATIONAL CANCER INSTITUTE   (United States)

^b MAYO CLINIC   (United States)

^c QUEEN MARY UNIVERSITY OF LONDON   (United Kingdom)

^d MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

Author keywords

Bladder cancer; Clinical trials; Immune checkpoints; Novel agents; Targeted therapy

Indexed keywords

4 AMINO N [1 (4 CHLOROPHENYL) 3 HYDROXYPROPYL] 1 (7H PYRROLO[2,3 D]PYRIMIDIN 4 YL) 4 PIPERIDINECARBOXAMIDE; ALPELISIB; ALT 801; ANGIOGENESIS INHIBITOR; AZD 8835; BAY 1163877; BEVACIZUMAB; BINIMETINIB; BUPARLISIB; CABOZANTINIB; CHECKPOINT KINASE INHIBITOR; CISPLATIN; DOVITINIB; EPIDERMAL GROWTH FACTOR RECEPTOR ANTIBODY; EVEROLIMUS; FPA 144; GSK 3052230; INFIGRATINIB; LAPATINIB; MLN 1117; MM 141; MOCETINOSTAT; NILOTINIB; NINTEDANIB; PACLITAXEL; PAZOPANIB; RAPAMYCIN; RIBOCICLIB; SORAFENIB; TRASTUZUMAB; UNCLASSIFIED DRUG; ANILIDE; ANTINEOPLASTIC AGENT; PDCD1 PROTEIN, HUMAN; PROGRAMMED DEATH 1 RECEPTOR; PYRIDINE DERIVATIVE;

ADVANCED CANCER; CANCER ADJUVANT THERAPY; CANCER COMBINATION CHEMOTHERAPY; CANCER RESISTANCE; CANCER SURVIVAL; CARCINOGENESIS; CHEMOSENSITIVITY; CLINICAL TRIAL (TOPIC); DOSE DENSIFICATION; DRUG EFFICACY; DRUG TOLERABILITY; HUMAN; MOLECULARLY TARGETED THERAPY; NEOPLASM; REVIEW; TRANSITIONAL CELL CARCINOMA; ANTAGONISTS AND INHIBITORS; CARCINOMA, TRANSITIONAL CELL; DRUG EFFECTS; GENETICS; PATHOLOGY; PERSONALIZED MEDICINE; PROCEDURES; UROLOGIC NEOPLASMS; UROTHELIUM;

ANILIDES; ANTINEOPLASTIC AGENTS; ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; BEVACIZUMAB; CARCINOMA, TRANSITIONAL CELL; CLINICAL TRIALS AS TOPIC; HUMANS; MOLECULAR TARGETED THERAPY; PRECISION MEDICINE; PROGRAMMED CELL DEATH 1 RECEPTOR; PYRIDINES; TRASTUZUMAB; UROLOGIC NEOPLASMS; UROTHELIUM;

EID: 84941599458     PISSN: 15587673     EISSN: 19380682     Source Type: Journal    
DOI: 10.1016/j.clgc.2015.02.009     Document Type: Review

References (81)

1. H. von der Maase, L. Sengelov, J.T. Roberts, and et al. Long-term survival results of a randomized trial comparing gemcitabine plus cisplatin, with methotrexate, vinblastine, doxorubicin, plus cisplatin in patients with bladder cancer J Clin Oncol 23 2005 4602 4608
2. H. von der Maase, S.W. Hansen, J.T. Roberts, and et al. Gemcitabine and cisplatin versus methotrexate, vinblastine, doxorubicin, and cisplatin in advanced or metastatic bladder cancer: results of a large, randomized, multinational, multicenter, phase III study J Clin Oncol 18 2000 3068 3077
3. R. Dreicer Second-line chemotherapy for advanced urothelial cancer: because we should or because we can? J Clin Oncol 27 2009 4444 4445
4. W.M. Stadler, S.P. Lerner, S. Groshen, and et al. Phase III study of molecularly targeted adjuvant therapy in locally advanced urothelial cancer of the bladder based on p53 status J Clin Oncol 29 2011 3443 3449
5. F. Cognetti, E.M. Ruggeri, A. Felici, and et al. Adjuvant chemotherapy with cisplatin and gemcitabine versus chemotherapy at relapse in patients with muscle-invasive bladder cancer submitted to radical cystectomy: an Italian, multicenter, randomized phase III trial Ann Oncol 23 2012 695 700
6. C.N. Sternberg, I. Skoneczna, and J. Kerst Final results of EORTC intergroup randomized phase III trial comparing immediate versus deferred chemotherapy after radical cystectomy in patients with pT3T4 and/or N+ M0 transitional cell carcinoma (TCC) of the bladder J Clin Oncol 5s 2014 32 abstract 4500
7. A. Bamias, U. Dafni, A. Karadimou, and et al. Prospective, open-label, randomized, phase III study of two dose-dense regimens MVAC versus gemcitabine/cisplatin in patients with inoperable, metastatic or relapsed urothelial cancer: a Hellenic Cooperative Oncology Group study (HE 16/03) Ann Oncol 24 2013 1011 1017
8. M.D. Galsky, N.M. Hahn, J. Rosenberg, and et al. Treatment of patients with metastatic urothelial cancer "unfit" for cisplatin-based chemotherapy J Clin Oncol 29 2011 2432 2438
9. A. Dash, M.D. Galsky, A.J. Vickers, and et al. Impact of renal impairment on eligibility for adjuvant cisplatin-based chemotherapy in patients with urothelial carcinoma of the bladder Cancer 107 2006 506 513
10. National Cancer Institute Surveillance, Epidemiology, and End Results Program. SEER Stat Fact Sheets: Bladder Cancer. [February 2015]. Available at: http://seer.cancer.gov/statfacts/html/urinb.html. Accessed: December 17, 2014.
11. National Cancer Institute Surveillance, Epidemiology, and End Results Program. SEER Stat Fact Sheets: Lung and Bronchus Cancer. [February 2015]. Available at: http://seer.cancer.gov/statfacts/html/lungb.html. Accessed: December 17, 2014.
12. M.D. Galsky, R. Hendricks, R. Svatek, and et al. Critical analysis of contemporary clinical research in muscle-invasive and metastatic urothelial cancer: a report from the Bladder Cancer Advocacy Network Clinical Trials Working Group Cancer 119 2013 1994 1998
13. Cancer Genome Atlas Research Network Comprehensive molecular characterization of urothelial bladder carcinoma Nature 507 2014 315 322
14. M. Ghosh, S.J. Brancato, P.K. Agarwal, and et al. Targeted therapies in urothelial carcinoma Curr Opin Oncol 26 2014 305 320
15. J. Bellmunt, B.T. Teh, G. Tortora, and et al. Molecular targets on the horizon for kidney and urothelial cancer Nat Rev Clin Oncol 10 2013 557 570
16. R.M. Bambury, and J.E. Rosenberg Actionable mutations in muscle-invasive bladder cancer Curr Opin Urol 23 2013 472 478
17. C.A. Rabik, and M.E. Dolan Molecular mechanisms of resistance and toxicity associated with platinating agents Cancer Treat Rev 33 2007 9 23
18. Z.H. Siddik Cisplatin: mode of cytotoxic action and molecular basis of resistance Oncogene 22 2003 7265 7279
19. L. Galluzzi, L. Senovilla, I. Vitale, and et al. Molecular mechanisms of cisplatin resistance Oncogene 31 2012 1869 1883
20. E.M. Van Allen, K.W. Mouw, P. Kim, and et al. Somatic ERCC2 mutations correlate with cisplatin sensitivity in muscle-invasive urothelial carcinoma Cancer Discov 4 2014 1140 1153
21. Groenendijk FH, de Jong J, Fransen van de Putte EE, et al. ERBB2 mutations characterize a subgroup of muscle-invasive bladder cancers with excellent response to neoadjuvant chemotherapy. Eur Urol Epub 2015 Jan 27.
22. L. Norton, R. Simon, H.D. Brereton, and et al. Predicting the course of gompertzian growth Nature 264 1976 542 545
23. C.N. Sternberg, P. de Mulder, J.H. Schornagel, and et al. Seven year update of an EORTC phase III trial of high-dose intensity M-VAC chemotherapy and G-CSF versus classic M-VAC in advanced urothelial tract tumours Eur J Cancer 42 2006 50 54
24. H.B. Grossman, R.B. Natale, C.M. Tangen, and et al. Neoadjuvant chemotherapy plus cystectomy compared with cystectomy alone for locally advanced bladder cancer N Engl J Med 349 2003 859 866
25. International Collaboration of Trialists Neoadjuvant cisplatin, methotrexate, and vinblastine chemotherapy for muscle-invasive bladder cancer: a randomised controlled trial Lancet 354 1999 533 540
26. G. Griffiths, R. Hall, R. Sylvester, and et al. International phase III trial assessing neoadjuvant cisplatin, methotrexate, and vinblastine chemotherapy for muscle-invasive bladder cancer: long-term results of the BA06 30894 trial J Clin Oncol 29 2011 2171 2177
27. T.K. Choueiri, S. Jacobus, J. Bellmunt, and et al. Neoadjuvant dose-dense methotrexate, vinblastine, doxorubicin, and cisplatin with pegfilgrastim support in muscle-invasive urothelial cancer: pathologic, radiologic, and biomarker correlates J Clin Oncol 32 2014 1889 1894
28. E.R. Plimack, J.H. Hoffman-Censits, R. Viterbo, and et al. Accelerated methotrexate, vinblastine, doxorubicin, and cisplatin is safe, effective, and efficient neoadjuvant treatment for muscle-invasive bladder cancer: results of a multicenter phase II study with molecular correlates of response and toxicity J Clin Oncol 32 2014 1895 1901
29. E.R. Plimack, J. Hoffman-Censits, A. Kutikov, and et al. Neoadjuvant dose-dense gemcitabine and cisplatin (DDGC) in patients (pts) with muscle-invasive bladder cancer (MIBC): final results of a multicenter phase II study J Clin Oncol 32 5S 2014 abstract 4513
30. A.O. Siefker-Radtke, C.P. Dinney, Y. Shen, and et al. A phase 2 clinical trial of sequential neoadjuvant chemotherapy with ifosfamide, doxorubicin, and gemcitabine followed by cisplatin, gemcitabine, and ifosfamide in locally advanced urothelial cancer: final results Cancer 119 2013 540 547
31. D.C. Smith, N.J. Mackler, R.L. Dunn, and et al. Phase II trial of paclitaxel, carboplatin and gemcitabine in patients with locally advanced carcinoma of the bladder J Urol 180 2008 2384 2388
32. C.A. Ortmann, and D. Mazhar Second-line systemic therapy for metastatic urothelial carcinoma of the bladder Future Oncol 9 2013 1637 1651
33. Y.J. Ko, C.M. Canil, S.D. Mukherjee, and et al. Nanoparticle albumin-bound paclitaxel for second-line treatment of metastatic urothelial carcinoma: a single group, multicentre, phase 2 study Lancet Oncol 14 2013 769 776
34. J. Bellmunt, C. Theodore, T. Demkov, and et al. Phase III trial of vinflunine plus best supportive care compared with best supportive care alone after a platinum-containing regimen in patients with advanced transitional cell carcinoma of the urothelial tract J Clin Oncol 27 2009 4454 4461
35. N.M. Hahn, W.M. Stadler, R.T. Zon, and et al. Phase II trial of cisplatin, gemcitabine, and bevacizumab as first-line therapy for metastatic urothelial carcinoma: Hoosier Oncology Group GU 04-75 J Clin Oncol 29 2011 1525 1530
36. A.V. Balar, A.B. Apolo, I. Ostrovnaya, and et al. Phase II study of gemcitabine, carboplatin, and bevacizumab in patients with advanced unresectable or metastatic urothelial cancer J Clin Oncol 31 2013 724 730
37. U. Chaudhary, A. Golshayan, A. Brisendine, and et al. Phase II trial of neoadjuvant cisplatin, gemcitabine, and bevacizumab followed by radical cystectomy (RC) in patients with muscle-invasive transitional cell carcinoma (TCC) of the bladder J Clin Oncol 29 suppl 7 2011 abstract 276
38. A. Siefker-Radtke, A. Kamat, P. Corn, and et al. Neoadjuvant chemotherapy with DD-MVAC and bevacizumab in high-risk urothelial cancer: results from a phase II trial at the University of Texas M. D. Anderson Cancer Center J Clin Oncol 30 2012 abstract 4523
39. D.J. Gallagher, M.I. Milowsky, S.R. Gerst, and et al. Phase II study of sunitinib in patients with metastatic urothelial cancer J Clin Oncol 28 2010 1373 1379
40. J. Bellmunt, J.L. Gonzalez-Larriba, C. Prior, and et al. Phase II study of sunitinib as first-line treatment of urothelial cancer patients ineligible to receive cisplatin-based chemotherapy: baseline interleukin-8 and tumor contrast enhancement as potential predictive factors of activity Ann Oncol 22 2011 2646 2653
41. M.D. Galsky, N.M. Hahn, T. Powles, and et al. Gemcitabine, cisplatin, and sunitinib for metastatic urothelial carcinoma and as preoperative therapy for muscle-invasive bladder cancer Clin Genitourin Cancer 11 2013 175 181
42. A.V. Balar, G. Iyer, A.B. Apolo, and et al. Phase II trial of neoadjuvant gemcitabine (G) and cisplatin (C) with sunitinib in patients (pts) with muscle-invasive bladder cancer (MIBC) J Clin Oncol 2012 30S abstract 4581
43. P.D. Grivas, S. Daignault, S.T. Tagawa, and et al. Double-blind, randomized, phase 2 trial of maintenance sunitinib versus placebo after response to chemotherapy in patients with advanced urothelial carcinoma Cancer 120 2014 692 701
44. S.S. Sridhar, E. Winquist, A. Eisen, and et al. A phase II trial of sorafenib in first-line metastatic urothelial cancer: a study of the PMH Phase II Consortium Invest New Drugs 29 2011 1045 1049
45. R. Dreicer, H. Li, M. Stein, and et al. Phase 2 trial of sorafenib in patients with advanced urothelial cancer: a trial of the Eastern Cooperative Oncology Group Cancer 115 2009 4090 4095
46. R. Pili, R. Qin, P.J. Flynn, and et al. A phase II safety and efficacy study of the vascular endothelial growth factor receptor tyrosine kinase inhibitor pazopanib in patients with metastatic urothelial cancer Clin Genitourin Cancer 11 2013 477 483
47. A. Necchi, L. Mariani, N. Zaffaroni, and et al. Pazopanib in advanced and platinum-resistant urothelial cancer: an open-label, single group, phase 2 trial Lancet Oncol 13 2012 810 816
48. H. Gerullis, C. Eimer, T.H. Ecke, and et al. Combined treatment with pazopanib and vinflunine in patients with advanced urothelial carcinoma refractory after first-line therapy Anticancer Drugs 24 2013 422 425
49. R.J. Lee, and M.R. Smith Cabozantinib and prostate cancer: inhibiting seed and disrupting soil? Clin Cancer Res 20 2014 525 527
50. J. Bellmunt, M. Hussain, and C.P. Dinney Novel approaches with targeted therapies in bladder cancer: therapy of bladder cancer by blockade of the epidermal growth factor receptor family Crit Rev Oncol Hematol 46 suppl 2003 S85 104
51. J.E. Nutt, H.P. Lazarowicz, J.K. Mellon, and et al. Gefitinib ("Iressa," ZD1839) inhibits the growth response of bladder tumour cell lines to epidermal growth factor and induces TIMP2 Br J Cancer 90 2004 1679 1685
52. P. Perrotte, T. Matsumoto, K. Inoue, and et al. Anti-epidermal growth factor receptor antibody C225 inhibits angiogenesis in human transitional cell carcinoma growing orthotopically in nude mice Clin Cancer Res 5 1999 257 265
53. R.S. Pruthi, M. Nielsen, S. Heathcote, and et al. A phase II trial of neoadjuvant erlotinib in patients with muscle-invasive bladder cancer undergoing radical cystectomy: clinical and pathological results BJU Int 106 2010 349 354
54. D.P. Petrylak, C.M. Tangen, P.J. Van Veldhuizen Jr., and et al. Results of the Southwest Oncology Group phase II evaluation (study S0031) of ZD1839 for advanced transitional cell carcinoma of the urothelium BJU Int 105 2010 317 321
55. G.K. Philips, S. Halabi, B.L. Sanford, and et al. A phase II trial of cisplatin (C), gemcitabine (G) and gefitinib for advanced urothelial tract carcinoma: results of Cancer and Leukemia Group B (CALGB) 90102 Ann Oncol 20 2009 1074 1079
56. G.K. Philips, S. Halabi, B.L. Sanford, and et al. A phase II trial of cisplatin, fixed dose-rate gemcitabine and gefitinib for advanced urothelial tract carcinoma: results of the Cancer and Leukaemia Group B 90102 BJU Int 101 2008 20 25
57. M. Hussain, S. Daignault, N. Agarwal, and et al. A randomized phase 2 trial of gemcitabine/cisplatin with or without cetuximab in patients with advanced urothelial carcinoma Cancer 120 2014 2684 2693
58. Y.N. Wong, S. Litwin, D. Vaughn, and et al. Phase II trial of cetuximab with or without paclitaxel in patients with advanced urothelial tract carcinoma J Clin Oncol 30 2012 3545 3551
59. K. Inoue, J.W. Slaton, P. Perrotte, and et al. Paclitaxel enhances the effects of the anti-epidermal growth factor receptor monoclonal antibody ImClone C225 in mice with metastatic human bladder transitional cell carcinoma Clin Cancer Res 6 2000 4874 4884
60. T.K. Choueiri, R.W. Ross, S. Jacobus, and et al. Double-blind, randomized trial of docetaxel plus vandetanib versus docetaxel plus placebo in platinum-pretreated metastatic urothelial cancer J Clin Oncol 30 2012 507 512
61. M.H. Hussain, G.R. MacVicar, D.P. Petrylak, and et al. Trastuzumab, paclitaxel, carboplatin, and gemcitabine in advanced human epidermal growth factor receptor-2/neu-positive urothelial carcinoma: results of a multicenter phase II National Cancer Institute trial J Clin Oncol 25 2007 2218 2224
62. Oudard S, Culine S, Viellefond A, et al. Multicenter randomized phase 2 trial of gemcitabine-platinum with or without trastuzumab (T) in advanced/metastatic urothelial carcinoma (a/mUC) with HER2 overexpression. ESMO Congress 2012; abstract 786O.
63. C. Wulfing, J.P. Machiels, D.J. Richel, and et al. A single-arm, multicenter, open-label phase 2 study of lapatinib as the second-line treatment of patients with locally advanced or metastatic transitional cell carcinoma Cancer 115 2009 2881 2890
64. L.C. Kompier, I. Lurkin, M.N. van der Aa, and et al. FGFR3, HRAS, KRAS, NRAS and PIK3CA mutations in bladder cancer and their potential as biomarkers for surveillance and therapy PLoS One 5 2010 e13821
65. E. Seront, S. Rottey, B. Sautois, and et al. Phase II study of everolimus in patients with locally advanced or metastatic transitional cell carcinoma of the urothelial tract: clinical activity, molecular response, and biomarkers Ann Oncol 23 2012 2663 2670
66. M.I. Milowsky, G. Iyer, A.M. Regazzi, and et al. Phase II study of everolimus in metastatic urothelial cancer BJU Int 112 2013 462 470
67. G. Iyer, A.J. Hanrahan, M.I. Milowsky, and et al. Genome sequencing identifies a basis for everolimus sensitivity Science 338 2012 221
68. J.C. Bendell, J. Rodon, H.A. Burris, and et al. Phase I, dose-escalation study of BKM120, an oral pan-class I PI3K inhibitor, in patients with advanced solid tumors J Clin Oncol 30 2012 282 290
69. P. Munster, J. Specht, T. Werner, and et al. PI3K kinase inhibitor GSK2126458 (GSK458): clinical activity in select patient (PT) populations defined by predictive markers (study P3K112826) Ann Oncol 23 suppl 10 2013 abstract 442O
70. F.S. Hodi, S.J. O'Day, D.F. McDermott, and et al. Improved survival with ipilimumab in patients with metastatic melanoma N Engl J Med 363 2010 711 723
71. S.L. Topalian, F.S. Hodi, J.R. Brahmer, and et al. Safety, activity, and immune correlates of anti-PD-1 antibody in cancer N Engl J Med 366 2012 2443 2454
72. J.D. Wolchok, H. Kluger, M.K. Callahan, and et al. Nivolumab plus ipilimumab in advanced melanoma N Engl J Med 369 2013 122 133
73. T. Powles, N.J. Vogelzang, and G. Fine Inhibition of PD-L1 by MPDL3280A and clinical activity in pts with metastatic urothelial bladder cancer (UBC) J Clin Oncol 32 5S 2014 abstract 5011
74. T. Powles, J.P. Eder, G.D. Fine, and et al. MPDL3280A (anti-PD-L1) treatment leads to clinical activity in metastatic bladder cancer Nature 515 2014 558 562
75. E.R. Plimack, S. Gupta, J. Bellmunt, and et al. A phase 1b study of pembrolizumab (Pembro; MK-3475) in patients (Pts) with advanced urothelial tract cancer Ann Oncol 25 2014 1 41
76. A.B. Apolo, Y. Tomita, M. Lee, and et al. Effect of cabozantinib on immunosuppressive subsets in metastatic urothelial carcinoma J Clin Oncol 2014 32s abstract 4501
77. T. Friede, N. Parsons, and N. Stallard A conditional error function approach for subgroup selection in adaptive clinical trials Stat Med 31 2012 4309 4320
78. H. Miyamoto, Y. Zheng, and K. Izumi Nuclear hormone receptor signals as new therapeutic targets for urothelial carcinoma Curr Cancer Drug Targets 12 2012 14 22
79. J.K. Lee, D.M. Havaleshko, H. Cho, and et al. A strategy for predicting the chemosensitivity of human cancers and its application to drug discovery Proc Natl Acad Sci U S A 104 2007 13086 13091
80. P.D. Williams, S. Cheon, D.M. Havaleshko, and et al. Concordant gene expression signatures predict clinical outcomes of cancer patients undergoing systemic therapy Cancer Res 69 2009 8302 8309
81. S.C. Smith, A.S. Baras, J.K. Lee, and et al. The COXEN principle: translating signatures of in vitro chemosensitivity into tools for clinical outcome prediction and drug discovery in cancer Cancer Res 70 2010 1753 1758