Moving towards a customized approach for drug development: Lessons from clinical trials with immune checkpoint inhibitors in lung cancer

Translational Lung Cancer Research

Volumn 4, Issue 6, 2015, Pages 704-712

  Pilotto, Sara ^a   Carbognin, Luisa ^a   Karachaliou, Niki ^b   Garassino, Marina ^c   Cuppone, Federica ^d   Petraglia, Sandra ^d   Rosell, Rafael ^b,e,f,g,h   Tortora, Giampaolo ^a   Bria, Emilio ^a  

^a UNIVERSITY HOSPITAL OF VERONA   (Italy)

^b INSTITUT UNIVERSITARI DEXEUS   (Spain)

^c NATIONAL CANCER INSTITUTE   (Italy)

^d ITALIAN MEDICINES AGENCY   (Italy)

^e Pangaea Biotech   (Spain)

^f HOSPITAL UNIVERSITARI GERMANS TRIAS I PUJOL   (Spain)

^g Molecular Oncology Research (MORe) Foundation   (Spain)

^h Germans Trias i Pujol Health Sciences Institute and Hospital   (Spain)

Author keywords

Cancer; Design; Immunotherapy; Lung; Trial

Indexed keywords

EID: 84960082975     PISSN: 22186751     EISSN: 22264477     Source Type: Journal    
DOI: 10.3978/j.issn.2218-6751.2015.10.08     Document Type: Review

References (31)

1. Rizvi NA, Hellmann MD, Snyder A, et al. Cancer immunology. Mutational landscape determines sensitivity to PD-1 blockade in non-small cell lung cancer. Science 2015;348:124-8.
2. Hoos A, Eggermont AM, Janetzki S, et al. Improved endpoints for cancer immunotherapy trials. J Natl Cancer Inst 2010;102:1388-97.
3. Garon EB. Issues surrounding clinical trial endpoints in solid malignancies with a focus on metastatic non-small cell lung cancer. Lung Cancer 2012;77:475-81.
4. Rosell R, Carcereny E, Gervais R, et al. Erlotinib versus standard chemotherapy as first-line treatment for European patients with advanced EGFR mutation-positive non-small-cell lung cancer (EURTAC): a multicentre, open-label, randomised phase 3 trial. Lancet Oncol 2012;13:239-46.
5. Rosell R, Moran T, Queralt C, et al. Screening for epidermal growth factor receptor mutations in lung cancer. N Engl J Med 2009;361:958-67.
6. Shaw AT, Kim DW, Nakagawa K, et al. Crizotinib versus chemotherapy in advanced ALK-positive lung cancer. N Engl J Med 2013;368:2385-94.
7. Solomon BJ, Mok T, Kim DW, et al. First-line crizotinib versus chemotherapy in ALK-positive lung cancer. N Engl J Med 2014;371:2167-77.
8. Pilotto S, Bonomi M, Massari F, et al. Anti-angiogenic drugs and biomarkers in non-small-cell lung cancer: a 'hard days night'. Curr Pharm Des 2014;20:3958-72.
9. Garon EB, Ciuleanu TE, Arrieta O, et al. Ramucirumab plus docetaxel versus placebo plus docetaxel for secondline treatment of stage IV non-small-cell lung cancer after disease progression on platinum-based therapy (REVEL):a multicentre, double-blind, randomised phase 3 trial. Lancet 2014;384:665-73.
10. Reck M, Kaiser R, Mellemgaard A, et al. Docetaxel plus nintedanib versus docetaxel plus placebo in patients with previously treated non-small-cell lung cancer (LUME-Lung 1): a phase 3, double-blind, randomised controlled tri. Docetaxel plus nintedanib versus docetaxel plus placebo in patients with previously treated non-small-cell lung cancer (LUME-Lung 1): a phase 3, double-blind, randomised controlled trial. Lancet Oncol 2014;15:143-55.
11. Sandler A, Gray R, Perry MC, et al. Paclitaxel-carboplatin alone or with bevacizumab for non-small-cell lung cancer. N Engl J Med 2006;355:2542-50.
12. Reck M, von Pawel J, Zatloukal P, et al. Overall survival with cisplatin-gemcitabine and bevacizumab or placebo as first-line therapy for nonsquamous non-small-cell lung cancer: results from a randomised phase III trial (AVAiL). Ann Oncol 2010;21:1804-9.
13. Bria E, Massari F, Maines F, et al. Progression-free survival as primary endpoint in randomized clinical trials of targeted agents for advanced renal cell carcinoma. Correlation with overall survival, benchmarking and power analysis. Crit Rev Oncol Hematol 2015;93:50-9.
14. Ciani O, Davis S, Tappenden P, et al. Validation of surrogate endpoints in advanced solid tumors: systematic review of statistical methods, results, and implications for policy makers. Int J Technol Assess Health Care 2014;30:312-24.
15. Scagliotti GV, Parikh P, von Pawel J, et al. Phase III study comparing cisplatin plus gemcitabine with cisplatin plus pemetrexed in chemotherapy-naive patients with advanced-stage non-small-cell lung cancer. J Clin Oncol 2008;26:3543-51.
16. Brahmer J, Reckamp KL, Baas P, et al. Nivolumab versus Docetaxel in Advanced Squamous-Cell Non-Small-Cell Lung Cancer. N Engl J Med 2015;373:123-35.
17. Borghaei H, Paz-Ares L, Horn L, et al. Nivolumab versus Docetaxel in Advanced Nonsquamous Non-Small-Cell Lung Cancer. N Engl J Med 2015;373:1627-39.
18. Spira AI, Park K, Mazieres J, et al. Efficacy, safety and predictive biomarker results from a randomized phase II study comparing MPDL3280A vs docetaxel in 2L/3L NSCLC (POPLAR). J Clin Oncol 2015;33:abstr 8010.
19. Spigel DR, Reckamp KL, Rizvi NA, et al. A phase III study (CheckMate 017) of nivolumab (NIVO; anti-programmed death-1 (PD-1)) vs docetaxel (DOC) in previously treated advanced or metastatic squamous (SQ) cell non-small cell lung cancer (NSCLC). J Clin Oncol 2015;33:abstr 8009.
20. Patel SP, Kurzrock R. PD-L1 Expression as a Predictive Biomarker in Cancer Immunotherapy. Mol Cancer Ther 2015;14:847-56.
21. Carbognin L, Pilotto S, Milella M, 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.
22. Herbst RS, Soria JC, Kowanetz M, et al. Predictive correlates of response to the anti-PD-L1 antibody MPDL3280A in cancer patients. Nature 2014;515:563-7.
23. Tumeh PC, Harview CL, Yearley JH, et al. PD-1 blockade induces responses by inhibiting adaptive immune resistance. Nature 2014;515:568-71.
24. Schalper KA, Brown J, Carvajal-Hausdorf D, et al. Objective measurement and clinical significance of TILs in non-small cell lung cancer. J Natl Cancer Inst 2015;107.
25. Snyder A, Wolchok JD, Chan TA. Genetic basis for clinical response to CTLA-4 blockade. N Engl J Med 2015;372:783.
26. Ellis LM, Bernstein DS, Voest EE, et al. American Society of Clinical Oncology perspective: Raising the bar for clinical trials by defining clinically meaningful outcomes. J Clin Oncol 2014;32:1277-80.
27. Schadendorf D, Hodi FS, Robert C, et al. Pooled Analysis of Long-Term Survival Data From Phase II and Phase III Trials of Ipilimumab in Unresectable or Metastatic Melanoma. J Clin Oncol 2015;33:1889-94.
28. Sobrero AF, Pastorino A, Sargent DJ, et al. Raising the bar for antineoplastic agents: how to choose threshold values for superiority trials in advanced solid tumors. Clin Cancer Res 2015;21:1036-43.
29. Garon EB, Rizvi NA, Hui R, et al. Pembrolizumab for the treatment of non-small-cell lung cancer. N Engl J Med 2015;372:2018-28.
30. Hyman DM, Puzanov I, Subbiah V, et al. Vemurafenib in Multiple Nonmelanoma Cancers with BRAF V600 Mutations. N Engl J Med 2015;373:726-36.
31. Chen TT. Milestone Survival: A Potential Intermediate Endpoint for Immune Checkpoint Inhibitors. J Natl Cancer Inst 2015;107.