Rationale for Biomarkers and Surrogate End Points in Mechanism-Driven Oncology Drug Development

Recent progress in understanding the molecular basis of cancer has redefined the landscape for cancer drug discovery and development. Among the most exciting and promising benefits are molecular targeted cancer therapies, which can be seen as progress toward personalized medicine for cancer patients

[1]  D. Hanahan,et al.  Heritable formation of pancreatic beta-cell tumours in transgenic mice expressing recombinant insulin/simian virus 40 oncogenes. , 1985, Nature.

[2]  R. Gale,et al.  Chronic myeloid leukemia. , 1992, The American journal of medicine.

[3]  W. McGuire,et al.  Human breast cancer: correlation of relapse and survival with amplification of the HER-2/neu oncogene. , 1987, Science.

[4]  P. Leder,et al.  Single-step induction of mammary adenocarcinoma in transgenic mice bearing the activated c-neu oncogene , 1988, Cell.

[5]  W Godolphin,et al.  Studies of the HER-2/neu proto-oncogene in human breast and ovarian cancer. , 1989, Science.

[6]  O. Witte,et al.  Tyrosine kinase activity and transformation potency of bcr-abl oncogene products. , 1990, Science.

[7]  W. Gullick,et al.  Prevalence of aberrant expression of the epidermal growth factor receptor in human cancers. , 1991, British medical bulletin.

[8]  R. Dickson,et al.  Genes, oncogenes, and hormones : advances in cellular and molecular biology of breast cancer , 1991 .

[9]  P. Carter,et al.  Anti-p185HER2 monoclonal antibodies: biological properties and potential for immunotherapy. , 1992, Cancer treatment and research.

[10]  E. Canaani,et al.  Chronic myelogenous leukemia: biology and therapy. , 1993, Leukemia.

[11]  N. Normanno,et al.  Epidermal growth factor-related peptides and their receptors in human malignancies. , 1995, Critical reviews in oncology/hematology.

[12]  P. Ravdin,et al.  The c-erbB-2 proto-oncogene as a prognostic and predictive marker in breast cancer: a paradigm for the development of other macromolecular markers--a review. , 1995, Gene.

[13]  D Tripathy,et al.  Phase II study of weekly intravenous recombinant humanized anti-p185HER2 monoclonal antibody in patients with HER2/neu-overexpressing metastatic breast cancer. , 1996, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[14]  V. Steele,et al.  Epidermal growth factor receptor tyrosine kinase inhibitors as potential cancer chemopreventives. , 1996, Cancer epidemiology, biomarkers & prevention : a publication of the American Association for Cancer Research, cosponsored by the American Society of Preventive Oncology.

[15]  Thomas Ried,et al.  Hidden chromosome abnormalities in haematological malignancies detected by multicolour spectral karyotyping , 1997, Nature Genetics.

[16]  Takayuki Asahara,et al.  Isolation of Putative Progenitor Endothelial Cells for Angiogenesis , 1997, Science.

[17]  S. Fox,et al.  Expression of the angiogenic factors vascular endothelial cell growth factor, acidic and basic fibroblast growth factor, tumor growth factor beta-1, platelet-derived endothelial cell growth factor, placenta growth factor, and pleiotrophin in human primary breast cancer and its relation to angiogenes , 1997, Cancer research.

[18]  F. Révillion,et al.  ERBB2 oncogene in human breast cancer and its clinical significance. , 1998, European journal of cancer.

[19]  A. Weiss,et al.  Detection and characterization of carcinoma cells in the blood. , 1998, Proceedings of the National Academy of Sciences of the United States of America.

[20]  D A Berry,et al.  erbB-2, p53, and efficacy of adjuvant therapy in lymph node-positive breast cancer. , 1998, Journal of the National Cancer Institute.

[21]  J. Herman,et al.  Detection of aberrant promoter hypermethylation of tumor suppressor genes in serum DNA from non-small cell lung cancer patients. , 1999, Cancer research.

[22]  A. Harris,et al.  Anticancer agents targeting signaling molecules and cancer cell environment: challenges for drug development? , 1999, Journal of the National Cancer Institute.

[23]  R. Hoffman.  Green fluorescent protein to visualize cancer progression and metastasis. , 1999, Methods in enzymology.

[24]  N. Robert,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. , 1999, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[25]  W. Lau,et al.  Detection of aberrant p16 methylation in the plasma and serum of liver cancer patients. , 1999, Cancer research.

[26]  D. Hanahan,et al.  Effects of angiogenesis inhibitors on multistage carcinogenesis in mice. , 1999, Science.

[27]  J. Herman,et al.  Predicting lung cancer by detecting aberrant promoter methylation in sputum. , 2000, Cancer research.

[28]  B. Druker,et al.  Lessons learned from the development of an abl tyrosine kinase inhibitor for chronic myelogenous leukemia. , 2000, The Journal of clinical investigation.

[29]  J. Folkman,et al.  The Hemostatic System as a Regulator of Angiogenesis* , 2000, The Journal of Biological Chemistry.

[30]  M. Fernö,et al.  ErbB2 status and the benefit from two or five years of adjuvant tamoxifen in postmenopausal early stage breast cancer. , 2000, Annals of oncology : official journal of the European Society for Medical Oncology.

[31]  Shigeyoshi Itohara,et al.  Matrix metalloproteinase-9 triggers the angiogenic switch during carcinogenesis , 2000, Nature Cell Biology.

[32]  Gabriele Bergers,et al.  Less is more, regularly: metronomic dosing of cytotoxic drugs can target tumor angiogenesis in mice. , 2000, The Journal of clinical investigation.

[33]  S. Paik,et al.  HER2 and choice of adjuvant chemotherapy for invasive breast cancer: National Surgical Adjuvant Breast and Bowel Project Protocol B-15. , 2000, Journal of the National Cancer Institute.

[34]  R. Kerbel,et al.  'Accidental' anti-angiogenic drugs. anti-oncogene directed signal transduction inhibitors and conventional chemotherapeutic agents as examples. , 2000, European journal of cancer.

[35]  J. Folkman,et al.  Antiangiogenic scheduling of chemotherapy improves efficacy against experimental drug-resistant cancer. , 2000, Cancer research.

[36]  S. Baruchel,et al.  Continuous low-dose therapy with vinblastine and VEGF receptor-2 antibody induces sustained tumor regression without overt toxicity. , 2000, The Journal of clinical investigation.

[37]  Y. Yarden,et al.  Untangling the ErbB signalling network , 2001, Nature Reviews Molecular Cell Biology.

[38]  K. Kinzler,et al.  Secreted and cell surface genes expressed in benign and malignant colorectal tumors. , 2001, Cancer research.

[39]  B. Druker,et al.  Chronic myeloid leukemia: current treatment options. , 2001, Blood.

[40]  C. Sawyers,et al.  Efficacy and safety of a specific inhibitor of the BCR-ABL tyrosine kinase in chronic myeloid leukemia. , 2001, The New England journal of medicine.

[41]  T. Fleming,et al.  Use of chemotherapy plus a monoclonal antibody against HER2 for metastatic breast cancer that overexpresses HER2. , 2001, The New England journal of medicine.

[42]  S. Rafii,et al.  Impaired recruitment of bone-marrow–derived endothelial and hematopoietic precursor cells blocks tumor angiogenesis and growth , 2001, Nature Medicine.

[43]  Christopher B Umbricht,et al.  Detection of breast cancer cells in ductal lavage fluid by methylation-specific PCR , 2001, The Lancet.

[44]  I. Smith,et al.  Efficacy and safety of Herceptin® in women with metastatic breast cancer: results from pivotal clinical studies , 2001, Anti-cancer drugs.

[45]  J. Isner,et al.  Bone Marrow as a Source of Endothelial Cells for Natural and Iatrogenic Vascular Repair , 2001, Annals of the New York Academy of Sciences.

[46]  A. Goldhirsch,et al.  Resting and activated endothelial cells are increased in the peripheral blood of cancer patients. , 2001, Blood.

[47]  R. Perez-soler.  A phase II trial of the epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor OSI-774, following platinum-based chemotherapy, in patients (pts) with advanced-EGFR-expressing, non-small cell lung cancer (NSCLC) , 2001 .

[48]  R. Brekken,et al.  VEGF-VEGF receptor complexes as markers of tumor vascular endothelium. , 2001, Journal of controlled release : official journal of the Controlled Release Society.

[49]  G. Doyle,et al.  Changes in circulating carcinoma cells in patients with metastatic prostate cancer correlate with disease status. , 2001, Urology.

[50]  E. Petricoin,et al.  Use of proteomic patterns in serum to identify ovarian cancer , 2002, The Lancet.

[51]  J. Baselga,et al.  Pharmacodynamic studies of the epidermal growth factor receptor inhibitor ZD1839 in skin from cancer patients: histopathologic and molecular consequences of receptor inhibition. , 2002, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[52]  J. Folkman,et al.  Clinical translation of angiogenesis inhibitors , 2002, Nature Reviews Cancer.

[53]  Sigrid Stroobants,et al.  Update of phase I study of imatinib (STI571) in advanced soft tissue sarcomas and gastrointestinal stromal tumors: a report of the EORTC Soft Tissue and Bone Sarcoma Group. , 2002, European journal of cancer.

[54]  R. Larson,et al.  Imatinib induces hematologic and cytogenetic responses in patients with chronic myelogenous leukemia in myeloid blast crisis: results of a phase II study. , 2002, Blood.

[55]  L. Ellis,et al.  Development of biologic markers of response and assessment of antiangiogenic activity in a clinical trial of human recombinant endostatin. , 2002, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[56]  Peter Bohlen,et al.  Antitumor effects in mice of low-dose (metronomic) cyclophosphamide administered continuously through the drinking water. , 2002, Cancer research.

[57]  A. D. Van den Abbeele,et al.  Efficacy and safety of imatinib mesylate in advanced gastrointestinal stromal tumors. , 2002, The New England journal of medicine.

[58]  W. Carney,et al.  Elevated serum Her-2/neu level predicts decreased response to hormone therapy in metastatic breast cancer. , 2002, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[59]  E. Scarpi,et al.  Vascular endothelial growth factor and prognosis in patients with node‐negative breast cancer , 2002, International journal of cancer.

[60]  M. Mottolese,et al.  Prognostic value of (99m)Tc-sestamibi washout in predicting response of locally advanced breast cancer to neoadjuvant chemotherapy. , 2002, Journal of nuclear medicine : official publication, Society of Nuclear Medicine.

[61]  P. Rosée,et al.  Insights from pre-clinical studies for new combination treatment regimens with the Bcr-Abl kinase inhibitor imatinib mesylate (Gleevec/Glivec) in chronic myelogenous leukemia: a translational perspective , 2002, Leukemia.

[62]  E. Rowinsky,et al.  The ErbB receptor family: a therapeutic target for cancer. , 2002, Trends in molecular medicine.

[63]  M. Baccarani,et al.  Imatinib induces durable hematologic and cytogenetic responses in patients with accelerated phase chronic myeloid leukemia: results of a phase 2 study. , 2002, Blood.

[64]  J. Baselga,et al.  HER-Targeted Tyrosine-Kinase Inhibitors , 2002, Oncology.

[65]  Koichi Hattori,et al.  Vascular and haematopoietic stem cells: novel targets for anti-angiogenesis therapy? , 2002, Nature Reviews Cancer.

[66]  T. Jacks,et al.  Cancer Modeling in the Modern Era Progress and Challenges , 2002, Cell.

[67]  O. Alonso,et al.  Predictive value of 99mTc sestamibi scintigraphy in the evaluation of doxorubicin based chemotherapy response in patients with advanced breast cancer , 2002 .

[68]  M. Baccarani,et al.  Hematologic and cytogenetic responses to imatinib mesylate in chronic myelogenous leukemia. , 2002, The New England journal of medicine.

[69]  M. Jeffers,et al.  An evaluation of the prognostic significance of vascular endothelial growth factor in node positive primary breast carcinoma. , 2002, International journal of oncology.

[70]  A. Harris,et al.  Drug inhibition of angiogenesis. , 2002, Current opinion in pharmacology.

[71]  Lyndsay N Harris,et al.  Efficacy and safety of trastuzumab as a single agent in first-line treatment of HER2-overexpressing metastatic breast cancer. , 2002, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[72]  David H. Johnson.  Gefitinib (Iressa) trials in non-small cell lung cancer. , 2003, Lung cancer.

[73]  R. Kerbel.  Human Tumor Xenografts as Predictive Preclinical Models for Anticancer Drug Activity in Humans: Better than Commonly Perceived—But They Can Be Improved , 2003, Cancer biology & therapy.

[74]  J. Baselga,et al.  Clinical trial design and end points for epidermal growth factor receptor-targeted therapies: implications for drug development and practice. , 2003, Clinical cancer research : an official journal of the American Association for Cancer Research.

[75]  J. Grandis,et al.  Targeting epidermal growth factor receptor: novel therapeutics in the management of cancer , 2003, Expert review of anticancer therapy.

[76]  W. Kaelin,et al.  The von Hippel-Lindau protein, vascular endothelial growth factor, and kidney cancer. , 2003, The New England journal of medicine.

[77]  R. Herbst.  Dose-comparative monotherapy trials of ZD1839 in previously treated non-small cell lung cancer patients. , 2003, Seminars in oncology.

[78]  R. Jain,et al.  A mathematical model of the contribution of endothelial progenitor cells to angiogenesis in tumors: implications for antiangiogenic therapy. , 2003, Blood.

[79]  F. Kabbinavar,et al.  Phase II, randomized trial comparing bevacizumab plus fluorouracil (FU)/leucovorin (LV) with FU/LV alone in patients with metastatic colorectal cancer. , 2003, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[80]  S. Hilsenbeck,et al.  Effect of epidermal growth factor receptor inhibitor on development of estrogen receptor-negative mammary tumors. , 2004, Journal of the National Cancer Institute.

[81]  Francisco Cervantes,et al.  Imatinib compared with interferon and low-dose cytarabine for newly diagnosed chronic-phase chronic myeloid leukemia. , 2003, The New England journal of medicine.

[82]  P. Bonomi.  Clinical studies with non-iressa EGFR tyrosine kinase inhibitors. , 2003, Lung cancer.

[83]  Giovanni Martinelli,et al.  Continuous infusion of endostatin inhibits differentiation, mobilization, and clonogenic potential of endothelial cell progenitors. , 2003, Clinical cancer research : an official journal of the American Association for Cancer Research.

[84]  M. Sliwkowski,et al.  Preclinical studies with Erlotinib (Tarceva). , 2003, Seminars in oncology.

[85]  R. Natale.  O-244 Quantitative gene expression in non-small cell lung cancer from paraffin-embedded tissue specimens: Predicting response to gefitinib, an EGFR kinase inhibitor , 2003 .

[86]  B. Druker.  Imatinib alone and in combination for chronic myeloid leukemia. , 2003, Seminars in hematology.

[87]  J. Baselga,et al.  Pharmacodynamic studies of tumor biopsy specimens from patients with advanced gastric carcinoma undergoing treatment with genfinitib (ZD1839) , 2003 .

[88]  M. Deininger.  Cytogenetic studies in patients on imatinib. , 2003, Seminars in hematology.

[89]  S. Ho,et al.  Usefulness of chest single photon emission computed tomography with technetium-99m methoxyisobutylisonitrile to predict taxol based chemotherapy response in advanced non-small cell lung cancer. , 2003, Cancer letters.

[90]  M. Hidalgo,et al.  Pharmacodynamic evaluation of the epidermal growth factor receptor inhibitor OSI-774 in human epidermis of cancer patients. , 2003, Clinical cancer research : an official journal of the American Association for Cancer Research.

[91]  J. Ford,et al.  Practical management of patients with chronic myeloid leukemia receiving imatinib. , 2003, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[92]  Seth M Steinberg,et al.  A randomized trial of bevacizumab, an anti-vascular endothelial growth factor antibody, for metastatic renal cancer. , 2003, The New England journal of medicine.

[93]  Manuel Hidalgo,et al.  Developing inhibitors of the epidermal growth factor receptor for cancer treatment. , 2003, Journal of the National Cancer Institute.

[94]  S. Goodison,et al.  Prolonged dormancy and site-specific growth potential of cancer cells spontaneously disseminated from nonmetastatic breast tumors as revealed by labeling with green fluorescent protein. , 2003, Clinical cancer research : an official journal of the American Association for Cancer Research.

[95]  Lesley Seymour,et al.  Inhibitors of epidermal-growth-factor receptors: a review of clinical research with a focus on non-small-cell lung cancer. , 2003, The Lancet. Oncology.

[96]  R. Bast,et al.  Use of CA-125 in Clinical Trial Evaluation of New Therapeutic Drugs for Ovarian Cancer , 2004, Clinical Cancer Research.

[97]  P. Harari,et al.  Combining EGFR inhibitors with radiation or chemotherapy: will preclinical studies predict clinical results? , 2004, International journal of radiation oncology, biology, physics.

[98]  Adam P Dicker,et al.  Prostate-Specific Antigen Doubling Time as a Surrogate Marker for Evaluation of Oncologic Drugs to Treat Prostate Cancer , 2004, Clinical Cancer Research.

[99]  F. Penault-Llorca,et al.  Prognostic Value of Residual Node Involvement in Operable Breast Cancer after Induction Chemotherapy , 2002, Breast Cancer Research and Treatment.

[100]  P. Carter,et al.  Differential responses of human tumor cell lines to anti-p185HER2 monoclonal antibodies , 1993, Cancer Immunology, Immunotherapy.