The Development of Herceptin®: Paving the Way for Individualized Cancer Therapy

Modern Biopharmaceuticals: Design, Development and Optimization

Volumn 1, Issue , 2008, Pages 127-150

  Gutjahr, Thorsten S ^a   Reinhardt, Carsten ^a  

^a F HOFFMANN LA ROCHE LTD   (Switzerland)

Author keywords

Docetaxel; Herceptin; Hybridisation; Paclitaxel; Pivotal trial

Indexed keywords

EID: 84889960726     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1002/9783527620982.ch5     Document Type: Chapter

References (104)

1. Owens MA, Horten BC, Da Silva MM, et al. HER2 amplification ratios by fluorescence in situ hybridization and correlation with immunohistochemistry in a cohort of 6556 breast cancer tissues. Clin Breast Cancer 2004, 5, 63-69.
2. Ross JS, Fletcher JA, Bloom KJ, et al. Targeted therapy in breast cancer: the HER-2/neu gene and protein. Mol Cell Proteomics 2004, 3, 379-398.
3. van de Vijver MJ, Mooi WJ, Peterse JL, et al. Amplification and over-expression of the neu oncogene in human breast carcinomas. Eur J Surg Oncol 1988, 14, 111-114.
4. Barnes DM, Bartkova J, Camplejohn RS, et al. Overexpression of the c-erbB-2 oncogene: why does this occur more frequently in ductal carcinoma in situ than in invasive mammary carcinoma and is this of prognostic significance? Eur J Cancer 1992, 28, 644-648.
5. Chong D, Cooke TG, Reeves JR, et al. Quantitation of EGFR and c-erbB-2 expression in preinvasive compared to invasive breast cancer. Eur J Cancer 1999, 35(suppl 4), S203 (abstr 792A).
6. Hynes NE, Gerber HA, Saurer S, et al. Overexpression of the c-erbB-2 protein in human breast tumor cell lines. J Cell Biochem 1989, 39, 167-173.
7. Hynes NE. Amplification and overexpression of the erbB-2 gene in human tumors: its involvement in tumor development, significance as a prognostic factor, and potential as a target for cancer therapy. Semin Cancer Biol 1993, 4, 19-26.
8. Lewis GD, Figari I, Fendly B, et al. Differential responses of human tumor cell lines to anti-p185HER2 monoclonal antibodies. Cancer Immunol Immunother 1993, 37, 255-263.
9. Yarden Y, Sliwkowski MX. Untangling the ErbB signaling network. Nat Rev Mol Cell Biol 2001, 2, 127-137.
10. Alroy I, Yarden Y. The ErbB signaling network in embryogenesis and oncogenesis: signal diversification through combinatorial ligandreceptor interactions. FEBS Lett 1997, 410, 83-86.
11. Yarden Y. The EGFR family and its ligands in human cancer: signaling mechanisms and therapeutic opportunities. Eur J Cancer 2001, 37, S3-8.
12. Baulida J, Kraus MH, Alimandi M, et al. All ErbB receptors other than the epidermal growth factor receptor are endocytosis impaired. J Biol Chem 1996, 271, 5251-5257.
13. Hendriks BS, Opresko LK, Wiley HS, et al. Coregulation of epidermal growth factor receptor/ human epidermal growth factor receptor 2 (HER2) levels and locations: quantitative analysis of HER2 overexpression effects. Cancer Res 2003, 63, 1130-1137.
14. Levkowitz G, Waterman H, Zamir E, et al. c-Cbl/Sli-1 regulates endocytic sorting and ubiquitination of the epidermal growth factor receptor. Genes Dev 1998, 12, 3663-3674.
15. Weiner DB, Liu J, Greene MI. A point mutation in the neu oncogene mimics ligand induction of receptor aggregation. Nature 1989, 339, 230-231.
16. Sliwkowski MX, Lofgren JA, Lewis GD, et al. Nonclinical studies addressing the mechanism of action of trastuzumab (Herceptin). Semin Oncol 1999, 26 (suppl 12), 60-70.
17. Slamon DJ, Godolphin W, Jones LA, et al. Studies of the HER2/neu proto-oncogene in human breast and ovarian cancer. Science 1989, 244, 707-712.
18. Ross JS, Fletcher JA. The HER-2/neu oncogene in breast cancer: prognostic factor, predictive factor, and target for therapy. Stem Cells 1998, 16, 413-428.
19. Gusterson BA, Gelber RD, Goldhirsch KN, et al. Prognostic importance of c-erbB-2 expression in breast cancer. J Clin Oncol 1992, 10, 1049-1056.
20. Hynes NE, Stern DF. The biology of erbB-2/ neu/HER-2 and its role in cancer. Biochim Biophys Acta 1994, 1198, 165-184.
21. King CR, Kraus MH, Aaronsen SA. Amplification of a novel c-erbB-related gene in a human mammary carcinoma. Science 1985, 229, 974-976.
22. Ménard S, Fortis S, Castiglioni F, et al. HER2 as a prognostic factor in breast cancer. Oncology 2001, 61(suppl 2), 67-72.
23. Press MF, Bernstein L, Thomas PA, et al. HER-2/neu gene amplification characterized by fluorescence in situ hybridization: poor prognosis in node negative breast carcinomas. J Clin Oncol 1997, 15, 2894-2904.
24. Slamon DJ, Clark GM, Wong SG, et al. Human breast cancer: correlation of relapse and survival with amplification of the HER-2/neu oncogene. Science 1987, 235, 177-182.
25. Hanna W, Gelmon KA. Review of the literature on HER2/neu testing and the role of HER2/neu as a prognostic and predictive factor in breast cancer - update December 2000 to July 2001. Curr Oncol 2002, 9, S2-17.
26. Lohrisch C, Piccart M. HER2/neu as a predictive factor in breast cancer. Clin Breast Cancer 2001, 2, 129-135.
27. Piccart M, Lohrisch C, Di Leo A, Larsimont D. The predictive value of HER2 in breast cancer. Oncology 2001, 61 (suppl 2), 73-82.
28. Piccart MJ, Di Leo A, Hamilton A. HER2, a predictive factor ready to use in the daily management of breast cancer patients? Eur J Cancer 2000, 36, 1755-1761.
29. Baselga J, Norton L, Albanell J, et al. Recombinant humanized anti-HER2 antibody (HerceptinTM) enhances the antitumor activity of paclitaxel and doxorubicin against HER2/neu overexpressing human breast cancer xenografts. Cancer Res 1998, 58, 2825-2831.
30. Pegram M, Hsu S, Lewis G, et al. Inhibitory effects of combinations of HER-2/neu antibody and chemotherapeutic agents used for treatment of human breast cancers. Oncogene 1999, 18, 2241-2251.
31. Pegram MD, Lipton A, Hayes DF, et al. Phase II study of receptor-enhanced chemosensitivity using recombinant humanized antip185HER2/ neu monoclonal antibody plus cisplatin in patients with HER2/neu-overexpressing metastatic breast cancer refractory to chemotherapy treatment. J Clin Oncol 1998, 16, 2659-2671.
32. De Santes K, Slamon D, Andersen SK, et al. Radiolabeled antibody targeting of the HER-2/ neu oncoprotein. Cancer Res 1992, 52, 1916-1923.
33. Drebin JA, Link VC, Stern DF, et al. Downmodulation of an oncogene protein product and reversion of the transformed phenotype by monoclonal antibodies. Cell 1985, 41, 697-706.
34. Hudziak RM, Lewis GD, Winget M, et al. p185HER2 monoclonal antibody has antiproliferative effects in vitro and sensitizes human breast tumor cells to tumor necrosis factor. Mol Cell Biol 1989, 9, 1165-1172.
35. Klapper LN, Waterman H, Sela M, et al. Tumor- inhibitory antibodies to HER-2/ErbB-2 may act by recruiting c-Cbl and enhancing ubiquitination of HER-2. Cancer Res 2000, 60, 3384-3388.
36. Sarup JC, Johnson RM, King KL, et al. Characterization of an anti-p185HER2 monoclonal antibody that stimulates receptor function and inhibits tumor cell growth. Growth Reg 1991, 1, 72-82.
37. Carter P, Presta L, Gorman CM, et al. Humanization of an anti-p185HER2 antibody for human cancer therapy. Proc Natl Acad Sci USA 1992, 89, 4285-4289.
38. Clynes RA, Towers TL, Presta LG, et al. Inhibitory Fc receptors modulate in vivo cytotoxicity against tumor targets. Nat Med 2000, 6, 443-446.
39. Gennari R, Menard S, Fagnoni F, et al. Pilot study of the mechanism of action of preoperative trastuzumab in patients with primary operable breast tumors overexpressing HER2. Clin Cancer Res 2004, 10, 5650-5655.
40. Hancock MC, Langton BC, Chan T, et al. A monoclonal antibody against the c-erbB-2 protein enhances the cytotoxicity of cis-diaminedichloroplatinum against human breast and ovarian tumor cell lines. Cancer Res 1991, 51, 4575-4580.
41. Burgess AW, Cho HS, Eigenbrot C, et al. An open-and-shut case? Recent insights into the activation of EGF/ErbB receptors. Mol Cell 2003, 12, 541-552.
42. Molina MA, Codony-Servat J, Albanell J, et al. Trastuzumab (Herceptin), a humanized anti-HER2 receptor monoclonal antibody, inhibits basal and activated HER2 ectodomain cleavage in breast cancer cells. Cancer Res 2001, 61, 4744-4749.
43. Holbro T, Beerli RR, Maurer F, et al. The ErbB2/ErbB3 heterodimer functions as an oncogenic unit: ErbB2 requires ErbB3 to drive breast tumor cell proliferation. Proc Natl Acad Sci USA 2003, 100, 8933-8938.
44. McKenzie SJ, Marks PJ, Lam T, et al. Generation and characterization of monoclonal antibodies specific for the human neu oncogene product, p185. Oncogene 1989, 4, 543-548.
45. Stancovski I, Hurwitz E, Leitner O, et al. Mechanistic aspects of the opposing effects of monoclonal antibodies to the ERBB2 receptor on tumor growth. Proc Natl Acad Sci USA 1991, 88, 8691-8695.
46. Baselga J, Tripathy D, Mendelsohn J, et al. Phase II study of weekly intravenous recombinant humanized anti-p185HER2 monoclonal antibody in patients with HER2/neu-overexpressing metastatic breast cancer. J Clin Oncol 1996, 14, 737-44.
47. Baselga J, Mendelsohn J. The epidermal growth factor receptor as a target for therapy in breast carcinoma. Breast Cancer Res Treat 1994, 1, 127-38.
48. Tokuda Y, Ohnishi Y, Shimamura K, et al. In vitro and in vivo anti-tumor effects of a humanised monoclonal antibody against c-erbB-2 product. Br J Cancer 1996, 73, 1362-1365.
49. Pegram MD, Konecny GE, O'Callaghan C, et al. Rational combinations of trastuzumab with chemotherapeutic drugs used in the treatment of breast cancer. J Natl Cancer Inst 2004, 96, 739-749.
50. Pietras RJ, Pegram MD, Finn RS, et al. Remission of human breast cancer xenografts on therapy with humanized monoclonal antibody to HER-2 receptor and DNA-reactive drugs. Oncogene 1998, 17, 2235-2249.
51. Cobleigh MA, Vogel CL, Tripathy D, et al. Multinational study of the efficacy and safety of humanized anti-HER2 monoclonal antibody in women who have HER2-overexpressing metastatic breast cancer that has progressed after chemotherapy for metastatic disease. J Clin Oncol 1999, 17, 2639-2648.
52. Slamon DJ, Leyland-Jones B, Shak S, et al. Use of chemotherapy plus a monoclonal antibody against HER2 for metastatic breast cancer that overexpresses HER2. N Engl J Med 2001, 344, 783-792.
53. Baselga J. Herceptin alone or in combination with chemotherapy in the treatment of HER2- positive metastatic breast cancer: pivotal trials. Oncology 2001, 61, 14-21.
54. Naber SP, Tsutsumi Y, Yin S, et al. Strategies for the analysis of oncogene overexpression. Studies of the neu oncogene in breast carcinoma. Am J Clin Pathol 1990, 94, 125-136.
55. Kallioniemi OP, Kallioniemi A, Kurisu W, et al. ERBB2 amplification in breast cancer analyzed by fluorescence in situ hybridization. Proc Natl Acad Sci USA. 1992, 89, 5321-5335.
56. Pauletti G, Godolphin W, Press MF, Slamon DJ. Detection and quantitation of HER-2/neu gene amplification in human breast cancer archival material using fluorescence in situ hybridization. Oncogene 1996, 13, 63-72.
57. Bilous M, Dowsett M, Hanna W, et al. Current perspectives on HER2 testing: a review of national testing guidelines. Mod Pathol 2003, 16, 173-182.
58. Hanna W and Kwok K. Can CISH be an alternative to FISH in the HER2/neu testing algorithm? The Breast 2005, 14 (suppl 1), S17 (abstr P10).
59. Mass RD, Press M, Anderson S, et al. Improved survival benefit from Herceptin (trastuzumab) in patients selected by fluorescence in situ hybridization (FISH). Proc Am Soc Clin Oncol 2001, 20, 22a (abstr 85).
60. Smith IE. Efficacy and safety of Herceptin in women with metastatic breast cancer: results from pivotal clinical studies. Anticancer Drugs 2001, 12 (suppl 4), S3-10.
61. Vogel CL, Cobleigh M, Tripathy D, et al. Superior outcomes with Herceptin (trastuzumab) (H) in fluorescence in situ hybridization (FISH)-selected patients. Proc Am Soc Clin Oncol 2001, 20, 22a (abstr 86; poster update).
62. Mass RD, Sanders C, Charlene K, et al. The concordance between the clinical trials assay (CTA) and fluorescence in situ hybridization (FISH) in the Herceptin pivotal trials. Proc Am Soc Clin Oncol 2000, 19, 75a (abstr 291).
63. Bast RC Jr, Ravdin P, Hayes DF, et al. 2000 update of recommendations for the use of tumor markers in breast and colorectal cancer: clinical practice guidelines of the American Society of Clinical Oncology. J Clin Oncol 2001, 19, 1865-1878.
64. Vogel CL, Cobleigh MA, Tripathy D, et al. Efficacy and safety of trastuzumab as a single agent in first-line treatment of HER2-overexpressing metastatic breast cancer. J Clin Oncol 2002, 20, 719-726.
65. Di Leo A, Piccart MJ. Paclitaxel activity, dose, and schedule: data from phase III trials in metastatic breast cancer. Semin Oncol 1999, 26 (suppl 8), 27-32.
66. Marty M, Cognetti F, Maraninchi D, et al. Efficacy and safety of trastuzumab combined with docetaxel in patients with HER2-positive metastatic breast cancer administered as firstline treatment: results of a randomized phase II trial by the M77001 study group. J Clin Oncol 2005, 23, published ahead of print.
67. Norris B, Pritchard K, James J, et al. Phase III comparative study of vinorelbine combined with doxorubicin versus doxorubicin alone in disseminated metastatic/recurrent breast cancer: National Cancer Institute of Canada Clinical Trials Group Study MA8. J Clin Oncol 2000, 18, 2385-2394.
68. O'Shaughnessy JA. The evolving role of capecitabine in breast cancer. Clin Breast Cancer 2003, 4 (suppl 1), S20-25.
69. Parideans R, Biganzoli L, Bruning P, et al. Paclitaxel versus doxorubicin as first-line singleagent chemotherapy for metastatic breast cancer: a European organization for research and treatment of cancer randomized study with cross-over. J Clin Oncol 2000, 18, 724-733.
70. Sledge G, Neuberg D, Ingle J, et al. Phase III trial of doxorubicin, paclitaxel, and the combination of doxorubicin and paclitaxel as frontline chemotherapy for metastatic breast cancer: an Intergroup Trial (E1193). J Clin Oncol 2003, 21, 588-592.
71. Cook-Bruns N. Retrospective analysis of the safety of Herceptin® immunotherapy in metastatic breast cancer. Oncology 2001, 61 (suppl 2), 58-66.
72. Tan-Chiu E, Piccart M. Moving forward with Herceptin® in the adjuvant setting. Oncology 2002, 63 (suppl 1), 57-63.
73. Buzdar AU, Ibrahim NK, Francis D, et al. Significantly higher pathologic complete remission rate after neoadjuvant therapy with trastuzumab, paclitaxel, and epirubicin chemotherapy: Results of a randomized trial in human epidermal growth factor receptor 2- positive operable breast cancer. J Clin Oncol 2005, 23 (published ahead of print).
74. Anderson S, Reddy JC, Rai S, et al. Concordance between central and local lab IHC and FISH HER2 testing in a community-based trial of first-line trastuzumab plus a taxane in HER2+ metastatic breast cancer (MBC). ASCO annual meeting proceedings. J Clin Oncol 2004, 22 (abstract 9580) 14S.
75. Yaziji H, Barry TS, Goldstein LC, et al. Discrepancies between immunohistochemistry (IHC) and fluorescence in situ hybridisation (FISH) for HER-2 testing in breast cancer: the role of quality assurance program on a cohort of 3564 patients. ASCO annual meeting proceedings. J Clin Oncol 2004, 22 (abstract 679) 14S.
76. Yaziji H, Goldstein LC, Barry TS, et al. HER-2 testing in breast cancer using parallel tissuebased methods. JAMA 2004, 291, 1972-1977.
77. Dowsett M, Bartlett J, Ellis IO, et al. Correlation between immunohistochemistry (Hercep-Test) and fluorescence in situ hybridization (FISH) for HER-2 in 426 breast carcinomas from 37 centres. J Pathol 2003, 199, 418-423.
78. Hofmann M, Gaiser T, Gross C et al. Predictive value of Herceptest IHC and PathVysion FISH data: analysis from a Herceptin phase II monotherapy study. Proc Am Soc Clin Oncol 2003, 22 (abstract 740) 185.
79. Vincent-Salomon A, MacGrogan G, Couturier J, et al. Calibration of immunohistochemistry for assessment of HER2 in breast cancer: results of the French multicentre GEFPICS study. Histopathology 2003, 42, 337-347.
80. Cianciulli AM, Botti C, Coletta AM, et al. Contribution of fluorescence in situ hybridization to immunohistochemistry for the evaluation of HER-2 in breast cancer. Cancer Genet Cytogenet 2002, 133, 66-71.
81. McCormick SR, Lillemore TJ, Beneke J, et al. HER2 assessment by immunohistochemical analysis and fluorescence in situ hybridization: comparison of HercepTest and PathVysion commercial assays. Am J Clin Pathol 2002, 117, 935-943.
82. Paik S, Bryant J, Tan-Chiu E, et al. Real-world performance of HER2 testing - national surgical adjuvant breast and bowel project experience. J Natl Cancer Inst 2002, 94, 852-854.
83. Roche PC, Suman VJ, Jenkins RB, et al. Concordance between local and central laboratory HER2 testing in the breast intergroup trial N9831. J Natl Cancer Inst 2002, 94, 855-857.
84. Birner P, Oberhuber G, Stani J, and the Austrian Breast and Colorectal Cancer Study Group. Evaluation of the United States Food and Drug Administration-approved scoring and test system of HER-2 protein expression in breast cancer. Clin Cancer Res 2001, 7, 1669-1675.
85. Lebeau A, Deimling D, Kaltz C, et al. HER-2/ neu analysis in archival tissue samples of human breast cancer: comparison of immunohistochemistry and fluorescence in situ hybridization. J Clin Oncol 2001, 19, 354-363.
86. Tsuda H, Akiyama F, Terasaki H, et al. Detection of HER-2/neu (c-erb B-2) DNA amplification in primary breast carcinoma. Interobserver reproducibility and correlation with immunohistochemical HER-2 overexpression. Cancer 2001, 92, 2965-2974.
87. Tubbs RR, Pettay JD, Roche PC, et al. Discrepancies in clinical laboratory testing of eligibility for trastuzumab therapy: apparent immunohistochemical false-positives do not get the message. J Clin Oncol 2001, 19, 2714-2721.
88. Hoang MP, Sahin AA, Ordonez NG, Sneige N. HER-2/neu gene amplification compared with HER-2/neu protein overexpression and interobserver reproducibility in invasive breast carcinoma. Am J Clin Pathol 2000, 113, 852-859.
89. Kakar S, Puangsuvan N, Stevens JM, et al. Her-2/neu assessment in breast cancer by immunohistochemistry and fluorescence in situ hybridization: comparison of results and correlation with survival. Mol Diagn 2000, 5, 199-207.
90. Ridolfi RL, Jamehdor MR, Arber JM. HER-2/ neu testing in breast carcinoma: a combined immunohistochemical and fluorescence in situ hybridization approach. Mod Pathol 2000, 13, 866-873.
91. Tanner M, Gancberg D, Di Leo A, et al. Chromogenic in situ hybridization: a practical alternative for fluorescence in situ hybridization to detect HER-2/neu oncogene amplification in archival breast cancer samples. Am J Pathol 2000, 157, 1467-1472.
92. Bilous M, Morey A, Armes J et al. The interlaboratory reproducibility of CISH testing for HER2 and correlation with IHC and FISH results. Eur J Cancer 2004, 2 (Suppl. 3, abstract 160): 100.
93. Hofmann M, Gaiser T, Kneitz H et al. Comparison of chromogenic in situ hybridisation (CISH) with FISH and IHC for assessment of HER2 status and prediction of response to therapy: analysis from a study of trastuzumab monotherapy. Ann Oncol 2004, 15 (Suppl 3).
94. Peiro G, Mayr D, Hillemanns P, et al. Analysis of HER-2/neu amplification in endometrial carcinoma by chromogenic in situ hybridization. Correlation with fluorescence in situ hybridization, HER-2/neu, p53 and Ki-67 protein expression, and outcome. Mod Pathol 2004, 17, 1-11.
95. Arnould L, Denoux Y, MacGrogan G, et al. Agreement between chromogenic in situ hybridisation (CISH) and FISH in the determination of HER2 status in breast cancer. Br J Cancer 2003, 88, 1587-1591.
96. Kounelis S, Kapranos N, Malamos et al. Evaluation of HER-2/neu gene status in breast cancer by chromogenic in situ hybridisation: comparison with immunohistochemistry. USCAP 2003 (abstract 154).
97. Muller V, Thomssen C, Karakas C, et al. Quantitative assessment of HER-2/neu protein concentration in breast cancer by enzyme- linked immunosorbent assay. Int J Biol Markers 2003, 18, 13-20.
98. Sapino A, Coccorullo Z, Cassoni P, et al. Which breast carcinomas need HER-2/neugene study after immunohistochemical analysis? Results of combined use of antibodies against different c-erbB2 protein domains. Histopathology 2003, 43, 354-362.
99. Van de Vijver MJ, Rueschoff J, Penault-Llorca F, et al. Chromogenic in situ hybridisation (CISH) compared with FISH and IHC for detection of HER2 gene amplification: an international validation ring study. Breast Cancer Res Treat 2003 (abstract).
100. Wixom CR, Albers EA, Weidner N, et al. HER2 amplification: correlation of chromogenic in situ hybridisation (CISH) with immunohistochemistry (IHC) and fluorescence in situ hybridisation (FISH). USCAP 2003 (abstract 219).
101. Dandachi N, Dietze O, Hauser-Kronberger C. Chromogenic in situ hybridization: a novel approach to a practical and sensitive method for the detection of HER2 oncogene in archival human breast carcinoma. Lab Invest 2002, 82, 1007-1014.
102. Zhao J, Wu R, Au A, et al. Determination of HER2 gene amplification by chromogenic in situ hybridization (CISH) in archival breast carcinoma. Mod Pathol 2002, 15, 657-665.
103. Tanner M, Jarvinen P, Isola J. Amplification of HER-2/neu and topoisomerase II alpha in primary and metastatic breast cancer. Cancer Res 2001, 61, 5345-5348.
104. Park K, Kim J, Lim S, et al. Comparing fluorescence in situ hybridization and chromogenic in situ hybridization methods to determine the HER2/neu status in primary breast carcinoma using tissue microarray. Mod Pathol 2003, 16, 937-943.