Predictive biomarkers in PD-1/PD-L1 checkpoint blockade immunotherapy

Cancer Treatment Reviews

Volumn 41, Issue 10, 2015, Pages 868-876

  Meng, Xiangjiao ^a   Huang, Zhaoqin ^b   Teng, Feifei ^a   Xing, Ligang ^a   Yu, Jinming ^a  

^a SHANDONG CANCER HOSPITAL AND INSTITUTE   (China)

^b SHANDONG PROVINCIAL HOSPITAL AFFILIATED TO SHANDONG UNIVERSITY   (China)

Author keywords

Biomarkers; Immunotherapy; PD 1; PD L1; Response

Indexed keywords

BIM PROTEIN; DOCETAXEL; GAMMA INTERFERON; JANUS KINASE 3; NIVOLUMAB; PEMBROLIZUMAB; PROGRAMMED DEATH 1 LIGAND 1; PROGRAMMED DEATH 1 RECEPTOR; ANTINEOPLASTIC AGENT; ATEZOLIZUMAB; IMMUNOLOGIC FACTOR; IPILIMUMAB; MONOCLONAL ANTIBODY; TUMOR MARKER;

ARTICLE; CANCER CELL; CANCER IMMUNOTHERAPY; DRUG EFFICACY; HUMAN; IMMUNOCOMPETENT CELL; KIDNEY CARCINOMA; MISMATCH REPAIR; MUTATIONAL ANALYSIS; NON SMALL CELL LUNG CANCER; PROTEIN DETERMINATION; PROTEIN EXPRESSION; PROTEIN PROTEIN INTERACTION; RELIABILITY; SOMATIC MUTATION; STUDY DESIGN; TREATMENT OUTCOME; TUMOR ASSOCIATED LEUKOCYTE; TUMOR MICROENVIRONMENT; ANTAGONISTS AND INHIBITORS; CELL CYCLE CHECKPOINT; IMMUNOTHERAPY; METABOLISM; NEOPLASMS; PROGNOSIS;

ANTIBODIES, MONOCLONAL; ANTIBODIES, MONOCLONAL, HUMANIZED; ANTIGENS, CD274; ANTINEOPLASTIC AGENTS; BIOMARKERS, TUMOR; CELL CYCLE CHECKPOINTS; HUMANS; IMMUNOLOGIC FACTORS; IMMUNOTHERAPY; NEOPLASMS; PROGNOSIS; PROGRAMMED CELL DEATH 1 RECEPTOR;

EID: 84958759492     PISSN: 03057372     EISSN: 15321967     Source Type: Journal    
DOI: 10.1016/j.ctrv.2015.11.001     Document Type: Review

References (69)

1. Homet Moreno B, Ribas A. Anti-programmed cell death protein-1/ligand-1 therapy in different cancers. Br J Cancer 2015;112:1421-7.
2. Sharpe AH, Wherry EJ, Ahmed R, Freeman GJ. The function of programmed cell death 1 and its ligands in regulating autoimmunity and infection. Nat Immunol 2007;8:239-45.
3. Barber DL, Wherry EJ, Masopust D, Zhu B, Allison JP, Sharpe AH, et al. Restoring function in exhausted CD8 T cells during chronic viral infection. Nature 2006;439:682-7.
4. Paterson AM, Brown KE, Keir ME, Vanguri VK, Riella LV, Chandraker A, et al. The programmed death-1 ligand 1:B7-1 pathway restrains diabetogenic effector T cells in vivo. J Immunol 2011;187:1097-105.
5. Blank C, Gajewski T, Mackensen A. Interaction of PD-L1 on tumor cells with PD-1 on tumor-specific T cells as a mechanism of immune evasion: implications for tumor immunotherapy. Cancer Immunol Immunother 2005;54:307-14.
6. Patel SP, Kurzrock R. PD-L1 expression as a predictive biomarker in cancer immunotherapy. Mol Cancer Ther 2015;14:847-56.
7. Wang A, Wang HY, Liu Y, Zhao MC, Zhang HJ, Lu ZY, et al. The prognostic value of PD-L1 expression for non-small cell lung cancer patients: a meta-analysis. Eur J Surg Oncol 2015;41:450-6.
8. Yang CY, Lin MW, Chang YL, Wu CT, Yang PC. Programmed cell death-ligand 1 expression in surgically resected stage I pulmonary adenocarcinoma and its correlation with driver mutations and clinical outcomes. Eur J Cancer 2014;50:1361-9.
9. Hino R, Kabashima K, Kato Y, Yagi H, Nakamura M, Honjo T, et al. Tumor cell expression of programmed cell death-1 ligand1 is a prognostic factor for malignant melanoma. Cancer 2010;116:1757-66.
10. Brahmer JR, Tykodi SS, Chow LQ, Hwu WJ, Topalian SL, Hwu P, et al. Safety and activity of anti-PD-L1 antibody in patients with advanced cancer. N Engl J Med 2012;366:2455-65.
11. Robert C, Long GV, Brady B, Dutriaux C, Maio M, Mortier L, et al. Nivolumab in previously untreated melanoma without BRAF mutation. N Engl J Med 2015;372:320-30.
12. Wolchok JD, Kluger H, Callahan MK, Postow MA, Rizvi NA, Lesokhin AM, et al. Nivolumab plus ipilimumab in advanced melanoma. N Engl J Med 2013;369:122-33.
13. Hamid O, Robert C, Daud A, Hodi FS, Hwu W-J, Kefford R, et al. Safety and tumor responses with lambrolizumab (anti-PD-1) in melanoma. N Engl J Med 2013;369:134-44.
14. Garon EB, Rizvi NA, Hui R, Leighl N, Balmanoukian AS, Eder JP, et alKEYNOTE-001 Investigators. Pembrolizumab for the treatment of non-small-cell lung cancer. N Engl J Med 2015;372:2018-28.
15. Brahmer J, Reckamp KL, Baas P, Crinò L, Eberhardt WE, Poddubskaya E, et al. Nivolumab versus docetaxel in advanced squamous-cell non-small-cell lung cancer. N Engl J Med 2015;373:123-35.
16. Powles T, Eder JP, Fine GD, Braiteh FS, Loriot Y, Cruz C, et al. MPDL3280A (anti-PD-L1) treatment leads to clinical activity in metastatic bladder cancer. Nature 2014;515:558-62.
17. Motzer RJ, Rini BI, McDermott DF, Redman BG, Kuzel TM, Harrison MR, et al. Nivolumab for metastatic renal cell carcinoma: results of a randomized phase II trial. J Clin Oncol 2015;33:1430-7.
18. Herbst RS, Soria JC, Kowanetz M, Fine GD, Hamid O, Gordon MS, et al. Predictive correlates of response to the anti-PD-L1 antibody MPDL3280A in cancer patients. Nature 2014;515:563-7.
19. Ansell SM, Lesokhin AM, Borrello I, Halwani A, Scott EC, Gutierrez M, et al. PD-1 blockade with nivolumab in relapsed or refractory Hodgkin's lymphoma. N Engl J Med 2015;372:311-9.
20. Borghaei H, Paz-Ares L, Horn L, Spigel DR, Steins M, Ready NE, et al. Nivolumab versus docetaxel in advanced nonsquamous non-small-cell lung cancer. N Engl J Med 2015;373:1627-39.
21. Weber JS, D'Angelo SP, Minor D, Hodi FS, Gutzmer R, Neyns B, et al. Nivolumab versus chemotherapy in patients with advanced melanoma who progressed after anti-CTLA-4 treatment (CheckMate 037): a randomised, controlled, openlabel, phase 3 trial. Lancet Oncol 2015;16:375-84.
22. Ribas A, Puzanov I, Dummer R, Schadendorf D, Hamid O, Robert C, et al. Pembrolizumab versus investigator-choice chemotherapy for ipilimumabrefractory melanoma (KEYNOTE-002): a randomised, controlled, phase 2 trial. Lancet Oncol 2015 [S1470-2045(15)00083-2].
23. Motzer RJ, Escudier B, McDermott DF, George S, Hammers HJ, Srinivas S, et al. Nivolumab versus everolimus in advanced renal-cell carcinoma. N Engl J Med 2015 [Epub ahead of print].
24. Robert C, Schachter J, Long GV, Arance A, Grob JJ, Mortier L, et alKEYNOTE-006 investigators. Pembrolizumab versus ipilimumab in advanced melanoma. N Engl J Med 2015;372:2521-32.
25. Taube JM, Klein A, Brahmer JR, Xu H, Pan X, Kim JH, et al. 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 2014;20:5064-74.
26. Topalian SL, Hodi FS, Brahmer JR, Gettinger SN, Smith DC, Mc Dermott DF, et al. Safety, activity, and immune correlates of anti-PD-1 antibody in cancer. N Engl J Med 2012;366:2443-54.
27. Kefford R, Ribas A, Hamid O, Robert C, Daud A, Wolchok JD, et al. Clinical efficacy and correlation with tumor PD-L1 expression in patients (pts) with melanoma (MEL) treated with the anti-PD-1 monoclonal antibody MK-3475. J Clin Oncol 2014;32 [suppl: abstr 3005].
28. Disis ML, Patel M, Pant S, Hamilton EP, Lockhart AC, Kelly K, et al. Avelumab (MSB0010718C), an anti-PD-L1 antibody, in patients with recurrent or refractory ovarian cancer: a phase Ib trial reporting safety and clinical activity. European Cancer Congress 2015 [abstr:2749].
29. Weber JS, Kudchadkar RR, Yu B, Gallenstein D, Horak CE, Inzunza HD, et al. Safety, efficacy, and biomarkers of nivolumab with vaccine in ipilimumabrefractory or -naive melanoma. J Clin Oncol 2013;31:4311-8.
30. Muro K, Bang YY, Shankaran V, Geva R, Catenacci DVT, Gupta S, et al. Relationship between PD-L1 expression and clinical outcomes in patients (Pts) with advanced gastric cancer treated with the anti-PD-1 monoclonal antibody pembrolizumab (Pembro; MK-3475) in KEYNOTE-012. J Clin Oncol 2015;33 [suppl; abstr 3].
31. Carbognin L, Pilotto S, Milella M, Vaccaro V, Brunelli M, Caliò A, et al. Differential activity of nivolumab, pembrolizumab and MPDL3280A according to the tumor expression of programmed death-ligand-1 (PD-L1): sensitivity analysis of trials in melanoma, lung and genitourinary cancers. PLoS One 2015;10:e0130142.
32. Llosa NJ, Cruise M, Tam A, Wick EC, Hechenbleikner EM, Taube JM, et al. The vigorous immune microenvironment of microsatellite instable colon cancer is balanced by multiple counter-inhibitory checkpoints. Cancer Discov 2015;5:43-51.
33. Petrylak DP, Powles T, Bellmunt J, Braiteh FS, Loriot Y, Zambrano CC, et al. A phase Ia study of MPDL3280A (anti-PDL1): updated response and survival data in urothelial bladder cancer (UBC). J Clin Oncol 2015;33 [suppl; abstr 4501].
34. Spira AI, Park K, Mazières J, Vansteenkiste JF, Rittmeyer A, Ballinger M, et al. Efficacy, safety and predictive biomarker results from a randomized phase II study comparing atezolizumab (MPDL3280 A) vs docetaxel in 2L/3L NSCLC. J Clin Oncol 2015;33 [suppl: abstr 8010].
35. Taube JM, Anders RA, Young GD, Xu H, Sharma R, Mcmiller TL, et al. Colocalization of Inflammatory response with B7-h1 expression in human melanocytic lesions supports an adaptive resistance mechanism of immune escape. Sci Transl Med 2012;4:127ra137.
36. Gatalica Z, Snyder C, Maney T, Ghazalpour A, Holterman DA, Xiao N, et al. Programmed cell death 1 (PD-1) and its ligand (PD-L1) in common cancers and their correlation with molecular cancer type. Cancer Epidemiol Biomarkers Prev 2014;23:2965-70.
37. Gainor JF, Sequist LV, Shaw AT, Azzoli CG, Piotrowska Z, Huynh T, et al. Clinical correlation and frequency of programmed death ligand-1 (PD-L1) expression in EGFR-mutant and ALK-rearranged non-small cell lung cancer (NSCLC). J Clin Oncol 2015;33 [suppl; abstr 8012].
38. Madore J, Vilain RE, Menzies AM, Kakavand H, Wilmott JS, Hyman J, et al. PDL1 expression in melanoma shows marked heterogeneity within and between patients: implications for anti-PD-1/PD-L1 clinical trials. Pigment Cell Melanoma Res 2015;28:245-53.
39. Callea M, Albiges L, Gupta M, Cheng SC, Genega EM, Fay AP, et al. Differential expression of PD-L1 between primary and metastatic sites in clear cell renal cell carcinoma. Cancer Immunol Res 2015;3:1158-64.
40. Mitchell P, Murone C, Asadi K, Harbison C, Knight S, John T, et al. PD-L1 expression in NSCLC: analysis of a large early stage cohort: and concordance of expression in primary, node and metastasis. J Thorac Oncol 2015;10 [suppl; S199 abstr].
41. Sheffield BS, Geller G, Pleasance E, Zachara-Szczakowski S, Miline K, Kalloger SE, et al. Predictive biomarker testing for programmed cell death 1 inhibition in non-small cell lung cancer. J Thorac Oncol 2015;10 [suppl; S199 abstr].
42. Dong H, Zhu G, Tamada K, Chen L. B7-H1, a third member of the B7 family, costimulates T-cell proliferation and interleukin-10 secretion. Nat Med 1999;5:1365-9.
43. Sznol M, Chen L. Antagonist Antibodies to PD-1 and B7-H1 (PD-L1) in the treatment of advanced human cancer. Clin Cancer Res 2013;19:1021-34.
44. Kaptain S, Tan LK, Chen B. Her-2/neu and breast cancer. Diagn Mol Pathol 2001;10:139-52.
45. Bang YJ, Van Cutsem E, Feyereislova A, Chung HC, Shen L, Sawaki A, et alToGA Trial Investigators. Trastuzumab in combination with chemotherapy versus chemotherapy alone for treatment of HER2-positive advanced gastric or gastro-oesophageal junction cancer (ToGA): a phase 3, open-label, randomised controlled trial. Lancet 2010;376:687-97.
46. Tumeh PC, Harview CL, Yearley JH, Shintaku IP, Taylor EJ, Robert L, et al. PD-1 blockade induces responses by inhibiting adaptive immune resistance. Nature 2014;515:568-71.
47. Prendergast GC, Smith C, Thomas S, Mandik-Nayak L, Laury-Kleintop L, Metz R, et al. Indoleamine 2,3-dioxygenase pathways of pathogenic inflammation and immune escape in cancer. Cancer Immunol Immunother 2014;63:721-35.
48. Zhang G, Liu WL, Zhang L, Wang JY, Kuang MH, Liu P, et al. Involvement of indoleamine 2,3-dioxygenase in impairing tumor-infiltrating CD8 T-cell functions in esophageal squamous cell carcinoma. Clin Dev Immunol 2011;2011:384726.
49. Spranger S, Spaapen RM, Zha Y, Williams J, Meng Y, Ha TT, et al. Up-regulation of PD-L1, IDO, and Tregs in the melanoma tumor microenvironment is driven by CD8+ T cells. Sci Transl Med 2013;5:200ra116.
50. Hamid O, Schmidt H, Nissan A, Ridolfi L, Aamdal S, Hansson J, et al. A prospective phase II trial exploring the association between tumor microenvironment biomarkers and clinical activity of ipilimumab in advanced melanoma. J Transl Med 2011;9:204.
51. Dronca RS, Markovic S, Kottschade LA, McWilliams RR, Block MS, Nevala WK, et al. Bim as a predictive T-cell biomarker for response to anti-PD-1 therapy in metastatic melanoma (MM). J Clin Oncol 2015;33 [suppl:abstr 9013].
52. Ribas A, Robert C, Hodi FS, Wolchok JD, Joshua AM, Hwu WJ, et al. Association of response to programmed death receptor 1 (PD-1) blockade with pembrolizumab (MK-3475) with an interferon-inflammatory immune gene signature. J Clin Oncol 2015;33 [suppl; abstr 3001].
53. Van Allen EM, Golay HG, Liu Y, Koyama S, Wong K, Taylor-Weiner A, et al. Long-term benefit of PD-L1 blockade in lung cancer associated with JAK3 activation. Cancer Immunol Res 2015;3:855-63.
54. Koopman M, Kortman GA, Mekenkamp L, Ligtenberg MJ, Hoogerbrugge N, Antonini NF, et al. Deficient mismatch repair system in patients with sporadic advanced colorectal cancer. Br J Cancer 2009;100:266-73.
55. Goldstein J, Tran B, Ensor J, Gibbs P, Wong HL, Wong SF, et al. Multicenter retrospective analysis of metastatic colorectal cancer (CRC) with high-level microsatellite instability (MSI-H). Ann Oncol 2014;25:1032-8.
56. Le DT, Uram JN, Wang H, Bartlett BR, Kemberling H, Eyring AD, et al. PD-1 blockade in tumors with mismatch-repair deficiency. N Engl J Med 2015;372:2509-20.
57. Timmermann B, Kerick M, Roehr C, Fischer A, Isau M, Boerno ST, et al. Somatic mutation profiles of MSI and MSS colorectal cancer identified by whole exome next generation sequencing and bioinformatics analysis. PLoS One 2010;5: e15661.
58. Eshleman JR, Lang EZ, Bowerfind GK, Parsons R, Vogelstein B, Willson JK, et al. Increased mutation rate at the hprt locus accompanies microsatellite instability in colon cancer. Oncogene 1995;10:33-7.
59. Dolcetti R, Viel A, Doglioni C, Russo A, Guidoboni M, Capozzi E, et al. High prevalence of activated intraepithelial cytotoxic T lymphocytes and increased neoplastic cell apoptosis in colorectal carcinomas with microsatellite instability. Am J Pathol 1999;154:1805-13.
60. Smyrk TC, Watson P, Kaul K, Lynch HT. Tumor-infiltrating lymphocytes are a marker for microsatellite instability in colorectal carcinoma. Cancer 2001;91:2417-22.
61. Watson IR, Takahashi K, Futreal PA, Chin L. Emerging patterns of somatic mutations in cancer. Nat Rev Genet 2013;14:703-18.
62. Yadav M, Jhunjhunwala S, Phung QT, Lupardus P, Tanguay J, Bumbaca S, et al. Predicting immunogenic tumour mutations by combining mass spectrometry and exome sequencing. Nature 2014;515:572-6.
63. Snyder A, Makarov V, Merghoub T, Yuan J, Zaretsky JM, Desrichard A, et al. Genetic basis for clinical response to CTLA-4 blockade in melanoma. N Engl J Med 2014;371:2189-99.
64. Boussiotis VA. Somatic mutations and immunotherapy outcome with CTLA-4 blockade in melanoma. N Engl J Med 2014;371:2230-2.
65. Rizvi NA, Hellmann MD, Snyder A, Kvistborg P, Makarov V, Havel JJ, et al. Mutational landscape determines sensitivity to PD-1 blockade in non-small cell lung cancer. Science 2015;348:124-8.
66. Gubin MM, Zhang X, Schuster H, Caron E, Ward JP, Noguchi T, et al. Checkpoint blockade cancer immunotherapy targets tumour-specific mutant antigens. Nature 2014;515:577-81.
67. Lawrence MS, Stojanov P, Polak P, Kryukov GV, Cibulskis K, Sivachenko A, et al. Mutational heterogeneity in cancer and the search for new cancer-associated genes. Nature 2013;499:214-8.
68. Govindan R, Ding L, Griffith M, Subramanian J, Dees ND, Kanchi KL, et al. Genomic landscape of non-small cell lung cancer in smokers and neversmokers. Cell 2012;150:1121-34.
69. Pilotto S, Kinspergher S, Peretti U, Calio A, Carbognin L, Ferrara R, et al. Immune checkpoint inhibitors for non-small-cell lung cancer: does that represent a 'new frontier'? Anticancer Agents Med Chem 2015;15:307-13.