WITHDRAWN: A novel bifunctional T regulatory cell engaging (BiTE) TGF-β1/PD-L1 fusion protein with therapeutic potential for autoimmune diseases

Marvin I. De los Santos1,2, Denise Mirano Bacos2, Samuel D. Bernal1,3
1Biomolecular Research Group, Globetek Science Foundation, Makati City, 1203, Philippines
2National Institute of Molecular Biology and Biotechnology, University of the Philippines Diliman, Quezon City, Philippines
3Cedars-Sinai Medical Center, Section of Hematology & Oncology, Los Angeles, California, USA

Tài liệu tham khảo

Li, 2017, Drugs for autoimmune inflammatory diseases: From small molecule compounds to anti-TNF biologics, Frontiers in Pharmacology M. Sheth, C.M. Benedum, L.A. Celi, R.G. Mark, N. Markuzon, The association between autoimmune disease and 30-day mortality among sepsis ICU patients: A cohort study, Critical Care. (2019). https://doi.org/10.1186/s13054-019-2357-1. Migita, 2013, Rates of serious intracellular infections in autoimmune disease patients receiving initial glucocorticoid therapy, PLoS ONE, 10.1371/journal.pone.0078699 Hemminki, 2012, Autoimmune disease and subsequent digestive tract cancer by histology, Annals of Oncology A.L. Franks, J.E. Slansky, Multiple associations between a broad spectrum of autoimmune diseases, chronic inflammatory diseases and cancer, Anticancer Research. (2012). K.-H. Yu, C.-F. Kuo, L.H. Huang, W.-K. Huang, L.-C. See, Cancer Risk in Patients With Inflammatory Systemic Autoimmune Rheumatic Diseases, Medicine. (2016). https://doi.org/10.1097/md.0000000000003540. A. Kornbluth, Infliximab Approved for Use in Crohnʼs Disease: A Report on the FDA GI Advisory Committee Conference, Inflammatory Bowel Diseases. (1998). https://doi.org/10.1097/00054725-199811000-00014. P.E. Lipsky, D.M. van der Heijde, E.W. St Clair, D.E. Furst, F.C. Breedveld, J.R. Kalden, J.S. Smolen, M. Weisman, P. Emery, M. Feldmann, G.R. Harriman, R.N. Maini, G. Anti-Tumor Necrosis Factor Trial in Rheumatoid Arthritis with Concomitant Therapy Study, Infliximab and methotrexate in the treatment of rheumatoid arthritis. Anti-Tumor Necrosis Factor Trial in Rheumatoid Arthritis with Concomitant Therapy Study Group.[see comment], New England Journal of Medicine. (2000). Maini, 1999, Infliximab (chimeric anti-tumour necrosis factor α monoclonal antibody) versus placebo in rheumatoid arthritis patients receiving concomitant methotrexate: A randomised phase III trial, Lancet, 10.1016/S0140-6736(99)05246-0 G. Malviya, S. Salemi, B. Laganà, A.P. Diamanti, R. D’Amelio, A. Signore, Biological therapies for rheumatoid arthritis: Progress to date, BioDrugs. (2013). https://doi.org/10.1007/s40259-013-0021-x. Hahn, 2012, American College of Rheumatology guidelines for screening, treatment, and management of lupus nephritis, Arthritis Care and Research, 10.1002/acr.21664 Massagué, 2014, TGFbeta signalling in context, Nat Rev Mol Cell Biol, 13, 616, 10.1038/nrm3434 Li, 2007, T Cell-Produced Transforming Growth Factor-β1 Controls T Cell Tolerance and Regulates Th1- and Th17-Cell Differentiation, Immunity, 10.1016/j.immuni.2007.03.014 Varga, 2009, Transforming growth factor β as a therapeutic target in systemic sclerosis, Nature Reviews Rheumatology, 10.1038/nrrheum.2009.26 Bottinger, 1997, Biology of TGF-β in knockout and transgenic mouse models, Kidney International, 10.1038/ki.1997.185 X.J. Wang, D.A. Greenhalgh, J.R. Bickenbach, A. Jiang, D.S. Bundman, T. Krieg, R. Derynck, D.R. Roop, Expression of a dominant-negative type II transforming growth factor β (TGF-β) receptor in the epidermis of transgenic mice blocks TGF-β-mediated growth inhibition, Proceedings of the National Academy of Sciences of the United States of America. (1997). https://doi.org/10.1073/pnas.94.6.2386. Böttinger, 1997, Expression of a dominant-negative mutant TGF-β type II receptor in transgenic mice reveals essential roles for TGF-β in regulation of growth and differentiation in the exocrine pancreas, EMBO Journal, 10.1093/emboj/16.10.2621 Tinoco, 2009, Cell-Intrinsic Transforming Growth Factor-β Signaling Mediates Virus-Specific CD8+ T Cell Deletion and Viral Persistence In Vivo, Immunity, 10.1016/j.immuni.2009.06.015 Budhu, 2017, Blockade of surface-bound TGF-ß on regulatory T cells abrogates suppression of effector T cell function in the tumor microenvironment, Science Signaling, 10.1126/scisignal.aak9702 Śledzińska, 2013, TGF-β Signalling Is Required for CD4+ T Cell Homeostasis But Dispensable for Regulatory T Cell Function, PLoS Biology, 10.1371/journal.pbio.1001674 Gros, 2008, Cell Intrinsic TGF-β1 Regulation of B Cells, The Journal of Immunology, 10.4049/jimmunol.180.12.8153 MALYGIN, 1993, Regulation of Natural Killer Cell Activity by Transforming Growth Factor‐β and Prostaglandin E2, Scandinavian Journal of Immunology, 10.1111/j.1365-3083.1993.tb01667.x Kubiczkova, 2012, TGF-β - an excellent servant but a bad master, Journal of Translational Medicine, 10.1186/1479-5876-10-183 Bally, 2016, Genetic and Epigenetic Regulation of PD-1 Expression, The Journal of Immunology, 10.4049/jimmunol.1502643 Sampedro-Núñez, 2018, Analysis of expression of the PD-1/PD-L1 immune checkpoint system and its prognostic impact in gastroenteropancreatic neuroendocrine tumors, Scientific Reports, 8, 1, 10.1038/s41598-018-36129-1 Mariotti, 2019, Innate lymphoid cells: Expression of PD-1 and other checkpoints in normal and pathological conditions, Frontiers in Immunology, 10.3389/fimmu.2019.00910 Zamani, 2016, PD-1/PD-L and autoimmunity: A growing relationship, Cellular Immunology, 310, 27, 10.1016/j.cellimm.2016.09.009 Nishimura, 1999, Development of lupus-like autoimmune diseases by disruption of the PD-1 gene encoding an ITIM motif-carrying immunoreceptor, Immunity, 10.1016/S1074-7613(00)80089-8 Mahoney, 2015, The next immune-checkpoint inhibitors: Pd-1/pd-l1 blockade in melanoma, Clinical Therapeutics, 10.1016/j.clinthera.2015.02.018 Akinleye, 2019, Immune checkpoint inhibitors of PD-L1 as cancer therapeutics, Journal of Hematology and Oncology, 12, 1, 10.1186/s13045-019-0779-5 De Sousa Linhares, 2019, Therapeutic PD-L1 antibodies are more effective than PD-1 antibodies in blocking PD-1/PD-L1 signaling, Scientific Reports, 9, 1, 10.1038/s41598-019-47910-1 Smits, 2016, Bispecific T-cell engagers (BiTES) as treatment of B-cell lymphoma, Journal of Clinical Oncology, 10.1200/JCO.2015.64.9970 Hagness, 2012, Kinetics and Activation Requirements of Contact-Dependent Immune Suppression by Human Regulatory T Cells, The Journal of Immunology, 10.4049/jimmunol.1101367 Arce-Sillas, 2016, Regulatory T Cells: Molecular Actions on Effector Cells in Immune Regulation, Journal of Immunology Research, 10.1155/2016/1720827 Von Boehmer, 2013, Therapeutic opportunities for manipulating TReg cells in autoimmunity and cancer, Nature Reviews Drug Discovery, 10.1038/nrd3683 Sharma, 2018, Emerging functions of regulatory T cells in tissue homeostasis, Frontiers in Immunology, 10.3389/fimmu.2018.00883 Esensten, 2018, Regulatory T-cell therapy for autoimmune and autoinflammatory diseases: The next frontier, Journal of Allergy and Clinical Immunology, 10.1016/j.jaci.2018.10.015 Dhabaria, 2015, A High-Efficiency Cellular Extraction System for Biological Proteomics, Journal of Proteome Research, 10.1021/acs.jproteome.5b00547 De los Santos, 2019, Cellular Retention Assay to Determine the Interaction Frequency of CD19-directed Chimeric Antigen Receptor (CAR) Engineered Cells against CD19+ Leukemic Cells, Bio-Protocol, 9, 1, 10.21769/BioProtoc.3358 J.S. Huston, D. Levinson, M. Mudgett-Hunter, M.S. Tai, J. Novotny, M.N. Margolies, R.J. Ridge, R.E. Bruccoleri, E. Haber, R. Crea, H. Oppermann, Protein engineering of antibody binding sites: Recovery of specific activity in an anti-digoxin single-chain Fv analogue produced in Escherichia coli, Proceedings of the National Academy of Sciences of the United States of America. (1988). https://doi.org/10.1073/pnas.85.16.5879. Chen, 2013, Fusion protein linkers: Property, design and functionality, Advanced Drug Delivery Reviews, 10.1016/j.addr.2012.09.039 Kelley, 2015, The Phyre2 web portal for protein modeling, prediction and analysis, Nature Protocols, 10.1038/nprot.2015.053 Lin, 2006, The structural basis of TGF-β, bone morphogenetic protein, and activin ligand binding, Reproduction, 10.1530/rep.1.01072 Yamashita, 1994, Formation of hetero-oligomeric complexes of type I and type II receptors for transforming growth factor-β, Journal of Biological Chemistry Radaev, 2010, Ternary complex of transforming growth factor-β1 reveals isoform-specific ligand recognition and receptor recruitment in the superfamily, Journal of Biological Chemistry, 10.1074/jbc.M109.079921 Tabe, 2012, TGF-β1 Supports Leukemia Cell Survival Via Negative Regulation of FLI-1 Transcription Factor, ERK Inactivation and MMP-1 Secretion, Blood, 10.1182/blood.V120.21.3543.3543 Sun, 2013, Regulation of nuclear factor-κB in autoimmunity, Trends in Immunology, 10.1016/j.it.2013.01.004 Miraghazadeh, 2018, Nuclear factor-kappaB in autoimmunity: Man and mouse, Frontiers in Immunology, 10.3389/fimmu.2018.00613 Langer, 2018, Neither Lys- and DAP-type peptidoglycans stimulate mouse or human innate immune cells via Toll-like receptor 2, PLoS ONE, 10.1371/journal.pone.0193207 Zuazo, 2017, Molecular mechanisms of programmed cell death-1 dependent T cell suppression: Relevance for immunotherapy, Annals of Translational Medicine, 10.21037/atm.2017.06.11 Meng, 2010, Association of Shp2 with phosphorylated IL-22R1 is required for interleukin-22-induced MAP kinase activation, Journal of Molecular Cell Biology, 10.1093/jmcb/mjq017 Zhu, 2011, Kit-Shp2-Kit signaling acts to maintain a functional hematopoietic stem and progenitor cell pool, Blood Park, 2017, Reciprocal regulation of TLR2-mediated IFN-β production by SHP2 and Gsk3β, Scientific Reports Hl, 1999, Activators and target genes of Rel/NF-kappaB transcription factors, Oncogene, 18, 6853, 10.1038/sj.onc.1203239 B. V. Park, Z.T. Freeman, A. Ghasemzadeh, M.A. Chattergoon, A. Rutebemberwa, J. Steigner, M.E. Winter, T. V. Huynh, S.M. Sebald, S.J. Lee, F. Pan, D.M. Pardoll, A.L. Cox, TGFβ1-mediated SMAD3 enhances PD-1 expression on antigen-specific T cells in cancer, Cancer Discovery. (2016). https://doi.org/10.1158/2159-8290.CD-15-1347. Giroux, 2010, T Cell Activation Leads to Protein Kinase Cθ-Dependent Inhibition of TGF-β Signaling, The Journal of Immunology, 10.4049/jimmunol.1000137 Sun, 2017, A novel bispecific c-MET/PD-1 antibody with therapeutic potential in solid cancer, Oncotarget, 8, 29067, 10.18632/oncotarget.16173 Yang, 2008, PD-L1: PD-1 interaction contributes to the functional suppression of T-cell responses to human uveal melanoma cells in vitro, Investigative Ophthalmology and Visual Science, 10.1167/iovs.07-1606 Zhang, 1998, Regulation of the activity of IFN-γ promoter elements during Th cell differentiation, Journal of Immunology, 10.4049/jimmunol.161.11.6105 Girdlestone, 1996, Autocrine activation by interferon-γ of STAT factors following T cell activation, European Journal of Immunology, 10.1002/eji.1830260329 A.N. Shatrova, E. V. Mityushova, N.A. Aksenov, I.I. Marakhova, CD25 expression on the surface of Jurkat cells, Cell and Tissue Biology. (2015). https://doi.org/10.1134/S1990519X15050119. Castiglioni, 2017, Femtograms of interferon-γ suffice to modulate the behavior of jurkat cells: A new light in immunomodulation, International Journal of Molecular Sciences, 10.3390/ijms18122715 Lin, 2013, The talented interferon-gamma, Advances in Bioscience and Biotechnology, 10.4236/abb.2013.47A3002 Valentine, 1985, Phytohemagglutinin binds to the 20‐kDa molecule of the T3 complex, European Journal of Immunology, 10.1002/eji.1830150821 T. Liu, L. Zhang, D. Joo, S.C. Sun, NF-κB signaling in inflammation, Signal Transduction and Targeted Therapy. (2017). https://doi.org/10.1038/sigtrans.2017.23. Gerondakis, 2010, Roles of the NF-kappaB pathway in lymphocyte development and function, Cold Spring Harbor Perspectives in Biology, 10.1101/cshperspect.a000182 Wu, 2015, Blinatumomab: a bispecific T cell engager (BiTE) antibody against CD19/CD3 for refractory acute lymphoid leukemia, Journal of Hematology and Oncology, 10.1186/s13045-015-0195-4 Ross, 2017, Bispecific T cell engager (BiTE®) antibody constructs can mediate bystander tumor cell killing, PLoS ONE, 10.1371/journal.pone.0183390 Marie, 2005, TGF-β1 maintains suppressor function and Foxp3 expression in CD4 +CD25+ regulatory T cells, Journal of Experimental Medicine, 10.1084/jem.20042276 Chen, 2010, TGF-β and ’adaptive ’Foxp3+ regulatory T cells, Journal of Molecular Cell Biology, 10.1093/jmcb/mjp004 M. Wong, A. La Cava, B.H. Hahn, I.M. Rheumatology, PD-1 Signaling Promotes Suppressive Function of CD4 + Regulatory T Cells in ( New Zealand Black x New Zealand White ) F 1 Lupus-Prone Mice in a Dose-Dependent Manner, (n.d.) 3–4. Han, 2019, Turning the tide against regulatory T cells, Frontiers in Oncology, 10.3389/fonc.2019.00279 Gianchecchi, 2018, Inhibitory receptors and pathways of lymphocytes: The role of PD-1 in Treg development and their involvement in autoimmunity onset and cancer progression, Frontiers in Immunology, 10.3389/fimmu.2018.02374 Flanders, 2003, Medical applications of transforming growth factor-beta, Clinical Medicine & Research, 10.3121/cmr.1.1.13 Marafini, 2013, TGF-Beta Signaling Manipulation as Potential Therapy for IBD, Current Drug Targets, 10.2174/13894501113149990157 H. Ishigame, L.A. Zenewicz, S. Sanjabi, P. Licona-Limóa, M. Nakayama, W.J. Leonard, R.A. Flavell, Excessive Th1 responses due to the absence of TGF-β signaling cause autoimmune diabetes and dysregulated Treg cell homeostasis, Proceedings of the National Academy of Sciences of the United States of America. (2013). https://doi.org/10.1073/pnas.1304498110. Becker-Merok, 2010, Levels of transforming growth factor-β are low in systemic lupus erythematosus patients with active disease, Journal of Rheumatology, 10.3899/jrheum.100180 Herrington, 2016, Modulation of NF-κB Signaling as a Therapeutic Target in Autoimmunity, Journal of Biomolecular Screening, 10.1177/1087057115617456 Ai, 2013, Optimal method to stimulate cytokine production and its use in immunotoxicity assessment, International Journal of Environmental Research and Public Health, 10.3390/ijerph10093834 Kim, 2006, Quantitative Analysis of Phosphotyrosine Signaling Networks Triggered by CD3 and CD28 Costimulation in Jurkat Cells, The Journal of Immunology, 10.4049/jimmunol.176.5.2833 Schmidt, 2012, Molecular mechanisms oftreg-mediatedt cell suppression, Frontiers in Immunology, 10.3389/fimmu.2012.00051 Janikashvili, 2016, Immunotherapeutic Targeting in Autoimmune Diseases, Mediators of Inflammation, 2 Tran, 2012, TGF-β: The sword, the wand, and the shield of FOXP3 + regulatory T cells, Journal of Molecular Cell Biology, 10.1093/jmcb/mjr033 P.H. Jeon, K.I. Oh, IL2 is required for functional maturation of regulatory T cells, Animal Cells and Systems. (2017). https://doi.org/10.1080/19768354.2016.1272489. Herppich, 2019, Dynamic Imprinting of the Treg Cell-Specific Epigenetic Signature in Developing Thymic Regulatory T Cells, Frontiers in Immunology, 10.3389/fimmu.2019.02382 Beriou, 2010, TGF-β Induces IL-9 Production from Human Th17 Cells, The Journal of Immunology, 10.4049/jimmunol.1000356 D.E. Smilek, M.R. Ehlers, G.T. Nepom, Restoring the balance: Immunotherapeutic combinations for autoimmune disease, DMM Disease Models and Mechanisms. (2014). https://doi.org/10.1242/dmm.015099. Löffek, 2018, Transforming of the Tumor Microenvironment: Implications for TGF-β Inhibition in the Context of Immune-Checkpoint Therapy, Journal of Oncology, 2018 Wang, 2008, Protective role of programmed death 1 ligand 1 (PD-L1) in nonobese diabetic mice: The paradox in transgenic models, Diabetes, 10.2337/db07-1260 2019, Nature Biomedical Engineering, 3 McNamee, 2017, Timelines of translational science: From technology initiation to FDA approval, PLoS ONE, 10.1371/journal.pone.0177371 Vincent, 2000, Molecular targets for autoimmune and genetic disorders of neuromuscular transmission, European Journal of Biochemistry, 10.1046/j.1432-1033.2000.01785.x Pérez-De-Lis, 2017, Autoimmune diseases induced by biological agents. A review of 12,731 cases (BIOGEAS Registry), Expert Opinion on Drug Safety, 10.1080/14740338.2017.1372421 Le Rouzic, 2017, Th17 cytokines: Novel potential therapeutic targets for COPD pathogenesis and exacerbations, European Respiratory Journal, 10.1183/13993003.02434-2016