Perspectives on the integration of Immuno-Oncology Biomarkers and drugs in a Health Care setting

Seminars in Cancer Biology - Tập 52 - Trang 166-177 - 2018
K. Vermaelen1, A. Waeytens2, O. Kholmanskikh3, M. Van den Bulcke4, E. Van Valckenborgh4
1Tumor Immunology Laboratory, Department of Respiratory Medicine, Ghent University Hospital, Ghent, Belgium
2Department of Pharmaceutical Policy, National Institute for Health and Disability Insurance, Brussels, Belgium
3Scientific Institute of Public Health, Brussels, Belgium and Federal Agency for Medicines and Health Products (FAMHP), Brussels, Belgium
4Belgian Cancer Centre, Scientific Institute of Public Health, Brussels, Belgium

Tài liệu tham khảo

Hodi, 2010, Improved survival with ipilimumab in patients with metastatic melanoma, N. Engl. J. Med., 363, 711, 10.1056/NEJMoa1003466 Garon, 2015, Pembrolizumab for the treatment of non-small-cell lung cancer, N. Engl. J. Med., 372, 2018, 10.1056/NEJMoa1501824 Motzer, 2015, Nivolumab for metastatic renal cell carcinoma: results of a randomized phase II trial, J. Clin. Oncol., 33, 1430, 10.1200/JCO.2014.59.0703 Powles, 2014, MPDL3280A (anti-PD-L1) treatment leads to clinical activity in metastatic bladder cancer, Nature, 515, 558, 10.1038/nature13904 Younes, 2016, Nivolumab for classical Hodgkin's lymphoma after failure of both autologous stem-cell transplantation and brentuximab vedotin: a multicentre, multicohort, single-arm phase 2 trial, Lancet Oncol., 17, 1283, 10.1016/S1470-2045(16)30167-X Robert, 2015, Pembrolizumab versus ipilimumab in advanced melanoma, N. Engl. J. Med., 372, 2521, 10.1056/NEJMoa1503093 Kumar, 2017, Current diagnosis and management of immune related adverse events (irAEs) induced by immune checkpoint inhibitor therapy, Front. Pharmacol., 8, 49, 10.3389/fphar.2017.00049 Yuan, 2016, Novel technologies and emerging biomarkers for personalized cancer immunotherapy, J Immunother Cancer, 4, 3, 10.1186/s40425-016-0107-3 Ung, 2016, Challenges & perspectives of immunotherapy biomarkers & the histoOncoImmune methodology, Exp. Rev. Precision Med. Drug Dev., 1, 9, 10.1080/23808993.2016.1140005 Kelderman, 2015, Mismatch repair-deficient cancers are targets for anti-PD-1 therapy, Cancer Cell, 28, 11, 10.1016/j.ccell.2015.06.012 Le, 2015, PD-1 blockade in tumors with mismatch-repair deficiency, N. Engl. J. Med., 372, 2509, 10.1056/NEJMoa1500596 Akhmetov, 2015, Market access advancements and challenges in drug-companion diagnostic test Co-development in europe, J. Pers Med., 5, 213, 10.3390/jpm5020213 Masucci, 2016, Validation of biomarkers to predict response to immunotherapy in cancer: volume I − pre-analytical and analytical validation, J. Immunother. Cancer, 4, 76, 10.1186/s40425-016-0178-1 Lee, 2006, Fit-for-purpose method development and validation for successful biomarker measurement, Pharm. Res., 23, 312, 10.1007/s11095-005-9045-3 Dobbin, 2016, Validation of biomarkers to predict response to immunotherapy in cancer: volume II − clinical validation and regulatory considerations, J. Immunother. Cancer, 4, 77, 10.1186/s40425-016-0179-0 Fridlyand, 2013, Considerations for the successful co-development of targeted cancer therapies and companion diagnostics, Nat. Rev. Drug Discov., 12, 743, 10.1038/nrd4101 Simon, 2009, Use of archived specimens in evaluation of prognostic and predictive biomarkers, J. Natl. Cancer Inst., 101, 1446, 10.1093/jnci/djp335 Antoniou, 2016, Biomarker-Guided adaptive trial designs in phase II and phase III: a methodological review, PLoS One, 11, e0149803, 10.1371/journal.pone.0149803 https://www.cdc.gov/genomics/gtesting/acce/2010. Lyman, 2016, Biomarker tests for molecularly targeted therapies: laying the foundation and fulfilling the dream, J. Clin. Oncol., 34, 2061, 10.1200/JCO.2016.67.3160 Vansteenkiste, 2016, Efficacy of the MAGE-A3 cancer immunotherapeutic as adjuvant therapy in patients with resected MAGE-A3-positive non-small-cell lung cancer (MAGRIT): a randomised, double-blind, placebo-controlled, phase 3 trial, Lancet Oncol., 17, 822, 10.1016/S1470-2045(16)00099-1 Brahmer, 2012, Safety and activity of anti-PD-L1 antibody in patients with advanced cancer, N. Engl. J. Med., 366, 2455, 10.1056/NEJMoa1200694 Gettinger, 2015, Overall survival and long-Term safety of nivolumab (Anti-Programmed death 1 antibody, BMS-936558, ONO-4538) in patients with previously treated advanced non-Small-Cell lung cancer, J. Clin. Oncol., 33, 2004, 10.1200/JCO.2014.58.3708 Brahmer, 2017, Five-year follow-up from the CA209-003 study of nivolumab in previously treated advanced non-small cell lung cancer (NSCLC): Clinical characteristics of long-term survivors, Cancer Res., 77, CT077, 10.1158/1538-7445.AM2017-CT077 Borghaei, 2015, Nivolumab versus docetaxel in advanced nonsquamous non-small-cell lung cancer, N. Engl. J. Med., 373, 1627, 10.1056/NEJMoa1507643 Herbst, 2016, Pembrolizumab versus docetaxel for previously treated, PD-L1-positive, advanced non-small-cell lung cancer (KEYNOTE-010): a randomised controlled trial, Lancet, 387, 1540, 10.1016/S0140-6736(15)01281-7 Brahmer, 2015, Nivolumab versus docetaxel in advanced squamous-cell non-small-cell lung cancer, N. Engl. J. Med., 373, 123, 10.1056/NEJMoa1504627 Horn, 2017, Nivolumab versus docetaxel in previously treated patients with advanced non-small-cell lung cancer: two-year outcomes from two randomized, open-label, phase III trials (CheckMate 017 and CheckMate 057), J. Clin. Oncol., 10.1200/JCO.2017.74.3062 Fehrenbacher, 2016, Atezolizumab versus docetaxel for patients with previously treated non-small-cell lung cancer (POPLAR): a multicentre, open-label, phase 2 randomised controlled trial, Lancet, 387, 1837, 10.1016/S0140-6736(16)00587-0 Rittmeyer, 2017, Atezolizumab versus docetaxel in patients with previously treated non-small-cell lung cancer (OAK): a phase 3, open-label, multicentre randomised controlled trial, Lancet, 389, 255, 10.1016/S0140-6736(16)32517-X Reck, 2016, Pembrolizumab versus chemotherapy for PD-L1-positive non-small-cell lung cancer, N. Engl. J. Med., 375, 1823, 10.1056/NEJMoa1606774 Casadevall, 2017, Heterogeneity of tumor and immune cell PD-L1 expression and lymphocyte counts in surgical NSCLC samples, Clin. Lung Cancer, 18, 682, 10.1016/j.cllc.2017.04.014 McLaughlin, 2016, Quantitative assessment of the heterogeneity of PD-L1 expression in non-small-cell lung cancer, JAMA Oncol., 2, 46, 10.1001/jamaoncol.2015.3638 Ilie, 2016, Comparative study of the PD-L1 status between surgically resected specimens and matched biopsies of NSCLC patients reveal major discordances: a potential issue for anti-PD-L1 therapeutic strategies, Ann. Oncol., 27, 147, 10.1093/annonc/mdv489 Uruga, 2017, Programmed cell death ligand (PD-L1) expression in stage II and III lung adenocarcinomas and nodal metastases, J. Thorac. Oncol., 12, 458, 10.1016/j.jtho.2016.10.015 Noman, 2014, PD-L1 is a novel direct target of HIF-1alpha, and its blockade under hypoxia enhanced MDSC-mediated T cell activation, J. Exp. Med., 211, 781, 10.1084/jem.20131916 Ikeda, 2016, PD-L1 is upregulated by simultaneous amplification of the PD-L1 and JAK2 genes in non-small cell lung cancer, J. Thorac. Oncol., 11, 62, 10.1016/j.jtho.2015.09.010 Green, 2010, Integrative analysis reveals selective 9p24.1 amplification, increased PD-1 ligand expression, and further induction via JAK2 in nodular sclerosing Hodgkin lymphoma and primary mediastinal large B-cell lymphoma, Blood, 116, 3268, 10.1182/blood-2010-05-282780 Schildhaus, 2016, Occurrence of PDL1/2 copy number gains detected by FISH in adeno and squamous cell carcinomas of the lung and association with PDL1 overexpression in adenocarcinomas, JCO, 34, 3031, 10.1200/JCO.2016.34.15_suppl.3031 Chen, 2017, Elements of cancer immunity and the cancer-immune set point, Nature, 541, 321, 10.1038/nature21349 Gabrilovich, 2017, Myeloid-derived suppressor cells, Cancer Immunol. Res., 5, 3, 10.1158/2326-6066.CIR-16-0297 Hirsch, 2017, PD-L1 immunohistochemistry assays for lung cancer: results from phase 1 of the blueprint PD-L1 IHC assay comparison project, J. Thorac. Oncol., 12, 208, 10.1016/j.jtho.2016.11.2228 Ratcliffe, 2017, Agreement between programmed cell death ligand-1 diagnostic assays across multiple protein expression cutoffs in non-small cell lung cancer, Clin. Cancer Res., 23, 3585, 10.1158/1078-0432.CCR-16-2375 Tsao, 2017, Blueprint 2: PD-L1 immunohistochemistry comparability study in real-life, clinical samples Gadgeel, 2017, 1296OClinical efficacy of atezolizumab (Atezo) in PD-L1 subgroups defined by SP142 and 22C3 IHC assays in 2L+ NSCLC: Results from the randomized OAK study, Ann. Oncol., 28 Rimm, 2017, A prospective, multi-institutional, pathologist-based assessment of 4 immunohistochemistry assays for PD-L1 expression in non-small cell lung cancer, JAMA Oncol., 3, 1051, 10.1001/jamaoncol.2017.0013 Cheung, 2016, Evolution of quality assurance for clinical immunohistochemistry in the era of precision medicine: part 4: tissue tools for quality assurance in immunohistochemistry, Appl. Immunohistochem. Mol. Morphol. Cheung, 2017, Evolution of quality assurance for clinical immunohistochemistry in the era of precision medicine: part 1: fit-for-purpose approach to classification of clinical immunohistochemistry biomarkers, Appl. Immunohistochem. Mol. Morphol., 25, 4, 10.1097/PAI.0000000000000451 Torlakovic, 2017, Evolution of quality assurance for clinical immunohistochemistry in the era of precision medicine. part 3: technical validation of immunohistochemistry (IHC) assays in clinical IHC laboratories, Appl. Immunohistochem. Mol. Morphol., 25, 151, 10.1097/PAI.0000000000000470 Torlakovic, 2017, Evolution of quality assurance for clinical immunohistochemistry in the era of precision medicine − part 2: immunohistochemistry test performance characteristics, Appl. Immunohistochem. Mol. Morphol., 25, 79, 10.1097/PAI.0000000000000444 Sheng, 2016, Expression of programmed death ligand-1 on tumor cells varies pre and post chemotherapy in non-small cell lung cancer, Sci. Rep., 6, 20090, 10.1038/srep20090 Galluzzi, 2015, Immunological effects of conventional chemotherapy and targeted anticancer agents, Cancer Cell, 28, 690, 10.1016/j.ccell.2015.10.012 Taube, 2014, Association of PD-1, PD-1 ligands, and other features of the tumor immune microenvironment with response to anti-PD-1 therapy, Clin. Cancer Res., 20, 5064, 10.1158/1078-0432.CCR-13-3271 Midha, 2016, PD-L1 expression in advanced NSCLC: Primary lesions versus metastatic sites and impact of sample age, JCO, 34, 3025, 10.1200/JCO.2016.34.15_suppl.3025 Rebelatto, 2016, Development of a programmed cell death ligand-1 immunohistochemical assay validated for analysis of non-small cell lung cancer and head and neck squamous cell carcinoma, Diagn. Pathol., 11, 95, 10.1186/s13000-016-0545-8 Skov, 2017, Paired comparison of PD-L1 expression on cytologic and histologic specimens from malignancies in the lung assessed with PD-L1 IHC 28-8pharmDx and PD-L1 IHC 22C3pharmDx, Appl. Immunohistochem. Mol. Morphol., 10.1097/PAI.0000000000000540 Lerner, 2017, PD-L1 assessment in FNA (EBUS) dervied samples, JCO, 35, 11615, 10.1200/JCO.2017.35.15_suppl.11615 Herbst, 2014, Predictive correlates of response to the anti-PD-L1 antibody MPDL3280A in cancer patients, Nature, 515, 563, 10.1038/nature14011 Tumeh, 2014, PD-1 blockade induces responses by inhibiting adaptive immune resistance, Nature, 515, 568, 10.1038/nature13954 Loi, 2017, Relationship between tumor infiltrating lymphocyte (TIL) levels and response to pembrolizumab (pembro) in metastatic triple-negative breast cancer (mTNBC): results from KEYNOTE-086, Ann. Oncol., 28, 10.1093/annonc/mdx440.005 Dieci, 2017, Semin. Cancer Biol. Hendry, 2017, Adv. Anat. Pathol., 24, 235, 10.1097/PAP.0000000000000162 Hendry, 2017, Adv. Anat. Pathol., 24, 311, 10.1097/PAP.0000000000000161 Higgs, 2016, Relationship of baseline tumoral IFNγ mRNA and PD-L1 protein expression to overall survival in durvalumab-treated NSCLC patients, JCO, 34, 3036, 10.1200/JCO.2016.34.15_suppl.3036 Wallden, 2016, Development and analytical performance of a molecular diagnostic for anti-PD1 response on the nCounter Dx Analysis System, JCO, 34, 3034, 10.1200/JCO.2016.34.15_suppl.3034 Antonia, 2016, Safety and clinical activity of durvalumab (MEDI4736), an anti-PD-L1 antibody, in treatment-naïve patients with advanced non‒small-cell lung cancer, JCO, 34, 9029, 10.1200/JCO.2016.34.15_suppl.9029 Rizvi, 2015, Cancer immunology. Mutational landscape determines sensitivity to PD-1 blockade in non-small cell lung cancer, Science, 348, 124, 10.1126/science.aaa1348 Peters, 2017, Abstract CT082: impact of tumor mutation burden on the efficacy of first-line nivolumab in stage iv or recurrent non-small cell lung cancer: an exploratory analysis of CheckMate 026, Cancer Res., 77, CT082, 10.1158/1538-7445.AM2017-CT082 Johnson, 2016, Targeted next generation sequencing identifies markers of response to PD-1 blockade, Cancer Immunol Res, 4, 959, 10.1158/2326-6066.CIR-16-0143 Gandara, 2017, Blood-based biomarkers for cancer immunotherapy: tumor mutational burden in blood (bTMB) is associated with improved atezolizumab (atezo) efficacy in 2L+ NSCLC (POPLAR and OAK), Ann. Oncol., 28, v460, 10.1093/annonc/mdx380 Spigel, 2016, Total mutation burden (TMB) in lung cancer (LC) and relationship with response to PD-1/PD-L1 targeted therapies, JCO, 34, 9017, 10.1200/JCO.2016.34.15_suppl.9017 McGranahan, 2016, Clonal neoantigens elicit T cell immunoreactivity and sensitivity to immune checkpoint blockade, Science, 351, 1463, 10.1126/science.aaf1490 Turajlic, 2017, Insertion-and-deletion-derived tumour-specific neoantigens and the immunogenic phenotype: a pan-cancer analysis, Lancet Oncol., 18, 1009, 10.1016/S1470-2045(17)30516-8 Champiat, 2017, Hyperprogressive disease is a new pattern of progression in cancer patients treated by anti-PD-1/PD-L1, Clin. Cancer Res., 23, 1920, 10.1158/1078-0432.CCR-16-1741 Kato, 2017, Hyperprogressors after immunotherapy: analysis of genomic alterations associated with accelerated growth rate, Clin. Cancer Res. Spitzer, 2017, Systemic immunity is required for effective cancer immunotherapy, Cell, 168, 487, 10.1016/j.cell.2016.12.022 Kamphorst, 2017, Proliferation of PD-1+ CD8T cells in peripheral blood after PD-1-targeted therapy in lung cancer patients, Proc. Natl. Acad. Sci. U. S. A., 114, 4993, 10.1073/pnas.1705327114 Oh, 2016, Association between T cell repertoire diversification and both clinical response as well as toxicity following immune checkpoint blockade in metastatic cancer patients, JCO, 34, 3029, 10.1200/JCO.2016.34.15_suppl.3029 Routy, 2017, Gut microbiome influences efficacy of PD-1-based immunotherapy against epithelial tumors, Science Vetizou, 2015, Anticancer immunotherapy by CTLA-4 blockade relies on the gut microbiota, Science, 350, 1079, 10.1126/science.aad1329 Wargo, 2017, Association of the diversity and composition of the gut microbiome with responses and survival (PFS) in metastatic melanoma (MM) patients (pts) on anti-PD-1 therapy, JCO, 35, 3008, 10.1200/JCO.2017.35.15_suppl.3008 Vandenbroeck, 2016 Van Dyck, 2016 Karasaki, 2017, An immunogram for the cancer-immunity cycle: towards personalized immunotherapy of lung cancer, J. Thorac. Oncol., 12, 791, 10.1016/j.jtho.2017.01.005 Salgado, 2017, Societal challenges of precision medicine: bringing order to chaos, Eur. J. Cancer, 84, 325, 10.1016/j.ejca.2017.07.028 Pignatti, 2014, Cancer drug development and the evolving regulatory framework for companion diagnostics in the European union, Clin. Cancer Res., 20, 1458, 10.1158/1078-0432.CCR-13-1571 Pignatti, 2015, Assessment of benefits and risks in development of targeted therapies for cancer–The view of regulatory authorities, Mol. Oncol., 9, 1034, 10.1016/j.molonc.2014.10.003 Garassino, 2016, PL04a.03: durvalumab in 3rd-line locally advanced or metastatic, EGFR/ALK wild-Type NSCLC: results from the phase 2 ATLANTIC study, J. Thorac. Oncol., 12, S10, 10.1016/j.jtho.2016.11.012