Tumor-Specific T Cell Dysfunction Is a Dynamic Antigen-Driven Differentiation Program Initiated Early during Tumorigenesis.

CD8(+) T cells recognizing tumor-specific antigens are detected in cancer patients but are dysfunctional. Here we developed a tamoxifen-inducible liver cancer mouse model with a defined oncogenic driver antigen (SV40 large T-antigen) to follow the activation and differentiation of naive tumor-specific CD8(+) T (TST) cells after tumor initiation. Early during the pre-malignant phase of tumorigenesis, TST cells became dysfunctional, exhibiting phenotypic, functional, and transcriptional features similar to dysfunctional T cells isolated from late-stage human tumors. Thus, T cell dysfunction seen in advanced human cancers may already be established early during tumorigenesis. Although the TST cell dysfunctional state was initially therapeutically reversible, it ultimately evolved into a fixed state. Persistent antigen exposure rather than factors associated with the tumor microenvironment drove dysfunction. Moreover, the TST cell differentiation and dysfunction program exhibited features distinct from T cell exhaustion in chronic infections. Strategies to overcome this antigen-driven, cell-intrinsic dysfunction may be required to improve cancer immunotherapy.

[1]  M. Serrano,et al.  A p16INK4a-insensitive CDK4 mutant targeted by cytolytic T lymphocytes in a human melanoma , 1995, Science.

[2]  Fabian Kiessling,et al.  Vascular normalization in Rgs5-deficient tumours promotes immune destruction , 2008, Nature.

[3]  Susan M. Kaech,et al.  Transcriptional control of effector and memory CD8+ T cell differentiation , 2012, Nature Reviews Immunology.

[4]  Michael R Stratton,et al.  High-throughput epitope discovery reveals frequent recognition of neo-antigens by CD4+ T cells in human melanoma , 2014, Nature Medicine.

[5]  A. Regev,et al.  The transcription factor BATF operates as an essential differentiation checkpoint in early effector CD8+ T cells , 2014, Nature Immunology.

[6]  K. Hellström,et al.  Cellular and Humoral immunity to Different Types of Human Neoplasms , 1968, Nature.

[7]  D. Speiser,et al.  Molecular profiling of CD8 T cells in autochthonous melanoma identifies Maf as driver of exhaustion , 2015, The EMBO journal.

[8]  Hans Schreiber,et al.  Specificity in cancer immunotherapy. , 2008, Seminars in immunology.

[9]  H. Schreiber,et al.  Ribosomal versus non‐ribosomal cellular antigens: factors determining efficiency of indirect presentation to CD4+ T cells , 2010, Immunology.

[10]  P. Kloetzel,et al.  The only proposed T-cell epitope derived from the TEL-AML1 translocation is not naturally processed. , 2011, Blood.

[11]  J. Wolchok,et al.  Cancer: Antitumour immunity gets a boost , 2014, Nature.

[12]  C. Drake,et al.  Safety and activity of anti-PD-L1 antibody in patients with advanced cancer. , 2012, The New England journal of medicine.

[13]  Zoltan Szallasi,et al.  In silico prediction of tumor antigens derived from functional missense mutations of the cancer gene census , 2012, Oncoimmunology.

[14]  A. Wells,et al.  Signals from CD28 Induce Stable Epigenetic Modification of the IL-2 Promoter1 , 2005, The Journal of Immunology.

[15]  R. Hammer,et al.  Comparative analysis of SV40 17kT and LT function in vivo demonstrates that LT's C-terminus re-programs hepatic gene expression and is necessary for tumorigenesis in the liver , 2012, Oncogenesis.

[16]  Burton E. Barnett,et al.  Progenitor and Terminal Subsets of CD8+ T Cells Cooperate to Contain Chronic Viral Infection , 2012, Science.

[17]  D. Douek,et al.  PD-1 identifies the patient-specific CD8⁺ tumor-reactive repertoire infiltrating human tumors. , 2014, The Journal of clinical investigation.

[18]  M. Giedlin,et al.  Listeria-based cancer vaccines that segregate immunogenicity from toxicity. , 2004, Proceedings of the National Academy of Sciences of the United States of America.

[19]  M. Stratton,et al.  Tumor exome analysis reveals neoantigen-specific T-cell reactivity in an ipilimumab-responsive melanoma. , 2013, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[20]  I. Melero,et al.  Orchestrating immune check-point blockade for cancer immunotherapy in combinations. , 2014, Current opinion in immunology.

[21]  Pedro Romero,et al.  Exhaustion of tumor-specific CD8⁺ T cells in metastases from melanoma patients. , 2011, The Journal of clinical investigation.

[22]  M. Pfreundschuh,et al.  Naturally occurring T-cell response against mutated p21 ras oncoprotein in pancreatic cancer. , 2006, Clinical cancer research : an official journal of the American Association for Cancer Research.

[23]  Martin L. Miller,et al.  Mutational landscape determines sensitivity to PD-1 blockade in non–small cell lung cancer , 2015, Science.

[24]  Björn Nilsson,et al.  Integrative genomic analysis of HIV-specific CD8+ T cells reveals that PD-1 inhibits T cell function by upregulating BATF , 2010, Nature Medicine.

[25]  R. Ahmed,et al.  Chronic virus infection enforces demethylation of the locus that encodes PD-1 in antigen-specific CD8(+) T cells. , 2011, Immunity.

[26]  E. Wherry,et al.  Molecular signature of CD8+ T cell exhaustion during chronic viral infection. , 2007, Immunity.

[27]  S. Kaech,et al.  Generation of effector CD8+ T cells and their conversion to memory T cells , 2010, Immunological reviews.

[28]  A. Secord,et al.  Priming and Activation of Human Ovarian and Breast Cancer-specific CD8+ T Cells by Polyvalent Listeria monocytogenes-based Vaccines , 2009, Journal of immunotherapy.

[29]  D. Douek,et al.  Mutated PPP1R3B Is Recognized by T Cells Used To Treat a Melanoma Patient Who Experienced a Durable Complete Tumor Regression , 2013, The Journal of Immunology.

[30]  A. Mackensen,et al.  Effector function of human tumor-specific CD8 T cells in melanoma lesions: a state of local functional tolerance. , 2004, Cancer research.

[31]  M. J. Tevethia,et al.  In Vivo Ligation of CD40 Enhances Priming Against the Endogenous Tumor Antigen and Promotes CD8+ T Cell Effector Function in SV40 T Antigen Transgenic Mice1 , 2003, The Journal of Immunology.

[32]  T. Welling,et al.  T cell anergy, exhaustion, senescence, and stemness in the tumor microenvironment. , 2013, Current opinion in immunology.

[33]  Jimmy Lin,et al.  Mining Exomic Sequencing Data to Identify Mutated Antigens Recognized by Adoptively Transferred Tumor-reactive T cells , 2013, Nature Medicine.

[34]  Maxim N. Artyomov,et al.  Checkpoint Blockade Cancer Immunotherapy Targets Tumour-Specific Mutant Antigens , 2014, Nature.

[35]  E. Appella,et al.  A mutated beta-catenin gene encodes a melanoma-specific antigen recognized by tumor infiltrating lymphocytes , 1996, The Journal of experimental medicine.

[36]  S. Kaufmann,et al.  Role of CD28 for the Generation and Expansion of Antigen-Specific CD8+ T Lymphocytes During Infection with Listeria monocytogenes1 , 2001, The Journal of Immunology.

[37]  David T. W. Jones,et al.  Signatures of mutational processes in human cancer , 2013, Nature.

[38]  C. Huber,et al.  The response of autologous T cells to a human melanoma is dominated by mutated neoantigens. , 2005, Proceedings of the National Academy of Sciences of the United States of America.

[39]  P. Greenberg,et al.  Tolerance and exhaustion: defining mechanisms of T cell dysfunction. , 2014, Trends in immunology.

[40]  D. Gabrilovich,et al.  Coordinated regulation of myeloid cells by tumours , 2012, Nature Reviews Immunology.

[41]  D. Fearon,et al.  T cell exclusion, immune privilege, and the tumor microenvironment , 2015, Science.

[42]  Antonio Polley,et al.  Coregulation of CD8+ T cell exhaustion by multiple inhibitory receptors during chronic viral infection , 2009, Nature Immunology.

[43]  J. Wolchok,et al.  Genetic Basis for Clinical Response to CTLA-4 Blockade in Melanoma. , 2015, The New England journal of medicine.

[44]  J. Delrow,et al.  Rescued Tolerant CD8 T Cells Are Preprogrammed to Reestablish the Tolerant State , 2012, Science.

[45]  Sven Malchow,et al.  Basic principles of tumor-associated regulatory T cell biology. , 2013, Trends in immunology.

[46]  G. Hämmerling,et al.  Tumor agonist peptides break tolerance and elicit effective CTL responses in an inducible mouse model of hepatocellular carcinoma. , 2009, Immunology letters.

[47]  E. Wherry,et al.  A role for the transcriptional repressor Blimp-1 in CD8(+) T cell exhaustion during chronic viral infection. , 2009, Immunity.

[48]  G. Freeman,et al.  Restoring function in exhausted CD8 T cells during chronic viral infection , 2006, Nature.

[49]  David C. Smith,et al.  Safety, activity, and immune correlates of anti-PD-1 antibody in cancer. , 2012, The New England journal of medicine.

[50]  S. Rosenberg,et al.  Cancer Immunotherapy Based on Mutation-Specific CD4+ T Cells in a Patient with Epithelial Cancer , 2014, Science.

[51]  K. Isobe,et al.  Cutting Edge: CD8+CD122+ Regulatory T Cells Produce IL-10 to Suppress IFN-γ Production and Proliferation of CD8+ T Cells1 , 2005, The Journal of Immunology.

[52]  R. Maronpot,et al.  Histologic Characterization of Hepatic Carcinogenesis in Transgenic Mice Expressing SV40 T-antigens , 1993, Veterinary pathology.

[53]  G. Willimsky,et al.  Sporadic immunogenic tumours avoid destruction by inducing T-cell tolerance , 2005, Nature.

[54]  Kristin A. Hogquist,et al.  T cell receptor antagonist peptides induce positive selection , 1994, Cell.

[55]  Jeffrey W Pollard,et al.  Tumor-associated macrophages: from mechanisms to therapy. , 2014, Immunity.