Gene-modified T lymphocytes in the setting of hematopoietic cell transplantation: potential benefits and possible risks

Bibliography

• Storb R. Reduced-intensity conditioning transplantation in myeloid malignancies. Curr Opin Oncol 2009;21(Suppl 1):S3-5
   PubMedGoogle Scholar
• Appelbaum FR. Hematopoietic cell transplantation as a form of immunotherapy. Int J Hematol 2002;75:222-7
   PubMed Web of Science ®Google Scholar
• Aglietta M, Barkholt L, Schianca FC, Reduced-intensity allogeneic hematopoietic stem cell transplantation in metastatic colorectal cancer as a novel adoptive cell therapy approach. The European group for blood and marrow transplantation experience. Biol Blood Marrow Transplant 2009;15:326-35
   PubMed Web of Science ®Google Scholar
• Carnevale-Schianca F, Cignetti A, Capaldi A, Allogeneic nonmyeloablative hematopoietic cell transplantation in metastatic colon cancer: tumor-specific T cells directed to a tumor-associated antigen are generated in vivo during GVHD. Blood 2006;107:3795-803
   PubMed Web of Science ®Google Scholar
• Childs R, Chernoff A, Contentin N, Regression of metastatic renal-cell carcinoma after nonmyeloablative allogeneic peripheral-blood stem-cell transplantation. N Engl J Med 2000;343:750-8
   PubMed Web of Science ®Google Scholar
• Bethge WA, Hegenbart U, Stuart MJ, Adoptive immunotherapy with donor lymphocyte infusions after allogeneic hematopoietic cell transplantation following nonmyeloablative conditioning. Blood 2004;103:790-5
   PubMed Web of Science ®Google Scholar
• Kolb HJ, Schmid C, Barrett AJ, Schendel DJ. Graft-versus-leukemia reactions in allogeneic chimeras. Blood 2004;103:767-76
   PubMed Web of Science ®Google Scholar
• Boeckh M, Nichols WG, Papanicolaou G, Cytomegalovirus in hematopoietic stem cell transplant recipients: current status, known challenges, and future strategies. Biol Blood Marrow Transplant 2003;9:543-58
   PubMed Web of Science ®Google Scholar
• Li CR, Greenberg PD, Gilbert MJ, Recovery of HLA-restricted cytomegalovirus (CMV)-specific T-cell responses after allogeneic bone marrow transplant: correlation with CMV disease and effect of ganciclovir prophylaxis. Blood 1994;83:1971-9
   PubMed Web of Science ®Google Scholar
• Riddell SR, Watanabe KS, Goodrich JM, Restoration of viral immunity in immunodeficient humans by the adoptive transfer of T cell clones. Science 1992;257:238-41
   PubMed Web of Science ®Google Scholar
• Caldas C, Ambinder R. Epstein-Barr virus and bone marrow transplantation. Curr Opin Oncol 1995;7:102-6
   PubMedGoogle Scholar
• O'Reilly RJ, Small TN, Papadopoulos E, Adoptive immunotherapy for Epstein-Barr virus-associated lymphoproliferative disorders complicating marrow allografts. Springer Semin Immunopathol 1998;20:455-91
   PubMed Web of Science ®Google Scholar
• van de Donk NW, Kroger N, Hegenbart U, Prognostic factors for donor lymphocyte infusions following non-myeloablative allogeneic stem cell transplantation in multiple myeloma. Bone Marrow Transplant 2006;37:1135-41
   PubMed Web of Science ®Google Scholar
• Bonini C, Ciceri F, Marktel S, Bordignon C. Suicide-gene-transduced T-cells for the regulation of the graft-versus-leukemia effect. Vox Sang 1998;74(Suppl 2):341-3
   PubMedGoogle Scholar
• Bonini C, Ferrari G, Verzeletti S, HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia. Science 1997;276:1719-24
   PubMed Web of Science ®Google Scholar
• Bordignon C, Bonini C, Verzeletti S, Transfer of the HSV-tk gene into donor peripheral blood lymphocytes for in vivo modulation of donor anti-tumor immunity after allogeneic bone marrow transplantation. Hum Gene Ther 1995;6:813-19
   PubMed Web of Science ®Google Scholar
• Ciceri F, Bonini C, Gallo-Stampino C, Bordignon C. Modulation of GvHD by suicide-gene transduced donor T lymphocytes: clinical applications in mismatched transplantation. Cytotherapy 2005;7:144-9
   PubMed Web of Science ®Google Scholar
• Ciceri F, Bonini C, Marktel S, Antitumor effects of HSV-TK-engineered donor lymphocytes after allogeneic stem-cell transplantation. Blood 2007;109:4698-707
   PubMed Web of Science ®Google Scholar
• Ciceri F, Bonini C, Stanghellini MT, Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): a non-randomised phase I - II study. Lancet Oncol 2009;10:489-500
   PubMed Web of Science ®Google Scholar
• Champlin R, Bensinger W, Henslee-Downey J, Phase I/II study of thymidine kinase (tk)-transduced donor lymphocyte infusions (DLI) in patients with hematologic malignancies. Blood 1999;94(Suppl 1): abstract 1448
   PubMedGoogle Scholar
• Tiberghien P, Ferrand C, Lioure B, Administration of herpes simplex-thymidine kinase-expressing donor T cells with a T-cell-depleted allogeneic marrow graft. Blood 2001;97:63-72
   PubMed Web of Science ®Google Scholar
• Burt RK, Drobyski WR, Seregina T, Herpes simplex thymidine kinase gene-transduced donor lymphocyte infusions. Exp Hematol 2003;31:903-10
   PubMed Web of Science ®Google Scholar
• Marktel S, Magnani Z, Ciceri F, Immunologic potential of donor lymphocytes expressing a suicide gene for early immune reconstitution after hematopoietic T-cell-depleted stem cell transplantation. Blood 2003;101:1290-8
   PubMed Web of Science ®Google Scholar
• Horowitz MM, Gale RP, Sondel PM, Graft-versus-leukemia reactions after bone marrow transplantation. Blood 1990;75:555-62
   PubMed Web of Science ®Google Scholar
• Cavalieri S, Cazzaniga S, Geuna M, Human T lymphocytes transduced by lentiviral vectors in the absence of TCR activation maintain an intact immune competence. Blood 2003;102:497-505
   PubMed Web of Science ®Google Scholar
• Kaneko S, Mastaglio S, Bondanza A, IL-7 and IL-15 allow the generation of suicide gene-modified alloreactive self-renewing central memory human T lymphocytes. Blood 2009;113:1006-15
   PubMed Web of Science ®Google Scholar
• Sauce D, Tonnelier N, Duperrier A, Influence of ex vivo expansion and retrovirus-mediated gene transfer on primary T lymphocyte phenotype and functions. J Hematother Stem Cell Res 2002;11:929-40
   PubMedGoogle Scholar
• Bondanza A, Valtolina V, Magnani Z, Suicide gene therapy of graft-versus-host disease induced by central memory human T lymphocytes. Blood 2006;107:1828-36
   PubMed Web of Science ®Google Scholar
• Robinet E, Fehse B, Ebeling S, Improving the ex vivo retroviral-mediated suicide-gene transfer process in T lymphocytes to preserve immune function. Cytotherapy 2005;7:150-7
   PubMed Web of Science ®Google Scholar
• Naldini L. Inserting optimism into gene therapy. Nat Med 2006;12:386-8
   PubMed Web of Science ®Google Scholar
• Verhoeyen E, Dardalhon V, Ducrey-Rundquist O, IL-7 surface-engineered lentiviral vectors promote survival and efficient gene transfer in resting primary T lymphocytes. Blood 2003;101:2167-74
   PubMed Web of Science ®Google Scholar
• Qasim W, Mackey T, Sinclair J, Lentiviral vectors for T-cell suicide gene therapy: preservation of T-cell effector function after cytokine-mediated transduction. Mol Ther 2007;15:355-60
   PubMed Web of Science ®Google Scholar
• Sauce D, Mercier P, Battini JL, Preferential retroviral-mediated transduction of EBV- and CMV-specific T cells after polyclonal T-cell activation. Gene Ther 2004;11:1019-22
   PubMed Web of Science ®Google Scholar
• Sauce D, Bodinier M, Garin M, Retrovirus-mediated gene transfer in primary T lymphocytes impairs their anti-Epstein-Barr virus potential through both culture-dependent and selection process-dependent mechanisms. Blood 2002;99:1165-73
   PubMed Web of Science ®Google Scholar
• Bonini C, Bondanza A, Perna SK, The suicide gene therapy challenge: how to improve a successful gene therapy approach. Mol Ther 2007;15:1248-52
   PubMed Web of Science ®Google Scholar
• Mavilio F, Ferrari G, Rossini S, Peripheral blood lymphocytes as target cells of retroviral vector-mediated gene transfer. Blood 1994;83:1988-97
   PubMed Web of Science ®Google Scholar
• Lupo-Stanghellini MT, Provasi E, Bondanza A, Clinical impact of suicide gene therapy in allogeneic hematopoietic stem cell transplantation. Hum Gene Ther 2010;21:241-50
   PubMed Web of Science ®Google Scholar
• Zhan H, Thrasher A, Farzaneh F, Preparation for a Phase I/II clinical trial of T cell suicide gene therapy following haematopoietic stem cell transplantation in children. Hum Gene Ther 2010;21:509
   Web of Science ®Google Scholar
• Tey SK, Dotti G, Rooney CM, Inducible caspase 9 suicide gene to improve the safety of allodepleted T cells after haploidentical stem cell transplantation. Biol Blood Marrow Transplant 2007;13:913-24
   PubMed Web of Science ®Google Scholar
• Di Stasi A, Tey SK, Fujita Y, CASPALLO: phase I clinical trial of allodepleted T cells transduced with inducible caspase 9 suicide gene after haploidentical stem cell transplantation. Mol Ther 2010;18(Suppl 1): abstract 288
   Google Scholar
• Griffioen M, van Egmond EH, Kester MG, Retroviral transfer of human CD20 as a suicide gene for adoptive T-cell therapy. Haematologica 2009;94:1316-20
   PubMed Web of Science ®Google Scholar
• Introna M, Barbui AM, Bambacioni F, Genetic modification of human T cells with CD20: a strategy to purify and lyse transduced cells with anti-CD20 antibodies. Hum Gene Ther 2000;11:611-20
   PubMed Web of Science ®Google Scholar
• Serafini M, Manganini M, Borleri G, Characterization of CD20-transduced T lymphocytes as an alternative suicide gene therapy approach for the treatment of graft-versus-host disease. Hum Gene Ther 2004;15(1):63-76
   PubMed Web of Science ®Google Scholar
• Sangiolo D, Lesnikova M, Nash RA, Lentiviral vector conferring resistance to mycophenolate mofetil and sensitivity to ganciclovir for in vivo T-cell selection. Gene Ther 2007;14:1549-54
   PubMed Web of Science ®Google Scholar
• Fehse B, Kustikova OS, Li Z, A novel ‘sort-suicide’ fusion gene vector for T cell manipulation. Gene Ther 2002;9:1633-8
   PubMed Web of Science ®Google Scholar
• Maloney DG. Immunotherapy for non-Hodgkin's lymphoma: monoclonal antibodies and vaccines. J Clin Oncol 2005;23:6421-8
   PubMed Web of Science ®Google Scholar
• van Meerten T, Claessen MJ, Hagenbeek A, Ebeling SB. The CD20/alphaCD20 ‘suicide’ system: novel vectors with improved safety and expression profiles and efficient elimination of CD20-transgenic T cells. Gene Ther 2006;13:789-97
   PubMed Web of Science ®Google Scholar
• Black ME, Kokoris MS, Sabo P. Herpes simplex virus-1 thymidine kinase mutants created by semi-random sequence mutagenesis improve prodrug-mediated tumor cell killing. Cancer Res 2001;61:3022-6
   PubMed Web of Science ®Google Scholar
• Preuss E, Treschow A, Newrzela S, TK.007: a novel, codon-optimized HSVtk(A168H) mutant for suicide gene therapy. Hum Gene Ther 2010;21:929-41
   PubMed Web of Science ®Google Scholar
• Onodera M. Gene and cell therapy for relapsed leukemia after allo-stem cell transplantation. Front Biosci 2008;13:3408-14
   PubMed Web of Science ®Google Scholar
• Fehse B, Ayuk FA, Kroger N, Evidence for increased risk of secondary graft failure after in vivo depletion of suicide gene-modified T lymphocytes transplanted in conjunction with CD34+-enriched blood stem cells. Blood 2004;104:3408-9
   PubMed Web of Science ®Google Scholar
• Urbano-Ispizua A, Rozman C, Pimentel P, The number of donor CD3+ cells is the most important factor for graft failure after allogeneic transplantation of CD34+ selected cells from peripheral blood from HLA-identical siblings. Blood 2001;97:383-7
   PubMed Web of Science ®Google Scholar
• Dembic Z, Haas W, Weiss S, Transfer of specificity by murine alpha and beta T-cell receptor genes. Nature 1986;320:232-8
   PubMed Web of Science ®Google Scholar
• Kessels HW, Wolkers MC, van den Boom MD, Immunotherapy through TCR gene transfer. Nat Immunol 2001;2:957-61
   PubMed Web of Science ®Google Scholar
• Morgan RA, Dudley ME, Wunderlich JR, Cancer regression in patients after transfer of genetically engineered lymphocytes. Science 2006;314:126-9
   PubMed Web of Science ®Google Scholar
• Heemskerk MH, Hoogeboom M, de Paus RA, Redirection of antileukemic reactivity of peripheral T lymphocytes using gene transfer of minor histocompatibility antigen HA-2-specific T-cell receptor complexes expressing a conserved alpha joining region. Blood 2003;102:3530-40
   PubMed Web of Science ®Google Scholar
• Mutis T, Blokland E, Kester M, Generation of minor histocompatibility antigen HA-1-specific cytotoxic T cells restricted by nonself HLA molecules: a potential strategy to treat relapsed leukemia after HLA-mismatched stem cell transplantation. Blood 2002;100:547-52
   PubMed Web of Science ®Google Scholar
• Brenner MK, Okur FV. Overview of gene therapy clinical progress including cancer treatment with gene-modified T cells. Hematology Am Soc Hematol Educ Program 2009;1:675-81
   Google Scholar
• Xue SA, Gao L, Hart D, Elimination of human leukemia cells in NOD/SCID mice by WT1-TCR gene-transduced human T cells. Blood 2005;106:3062-7
   PubMed Web of Science ®Google Scholar
• Stanislawski T, Voss RH, Lotz C, Circumventing tolerance to a human MDM2-derived tumor antigen by TCR gene transfer. Nat Immunol 2001;2:962-70
   PubMed Web of Science ®Google Scholar
• Gross G, Waks T, Eshhar Z. Expression of immunoglobulin-T-cell receptor chimeric molecules as functional receptors with antibody-type specificity. Proc Natl Acad Sci USA 1989;86:10024-8
   PubMed Web of Science ®Google Scholar
• Kershaw MH, Darcy PK, Trapani JA, Smyth MJ. The use of chimeric human Fcϵ receptor I to redirect cytotoxic T lymphocytes to tumors. J Leukoc Biol 1996;60:721-8
   PubMed Web of Science ®Google Scholar
• Finney HM, Akbar AN, Lawson AD. Activation of resting human primary T cells with chimeric receptors: costimulation from CD28, inducible costimulator, CD134, and CD137 in series with signals from the TCR zeta chain. J Immunol 2004;172:104-13
   PubMed Web of Science ®Google Scholar
• Maher J, Brentjens RJ, Gunset G, Human T-lymphocyte cytotoxicity and proliferation directed by a single chimeric TCRζ /CD28 receptor. Nat Biotechnol 2002;20:70-5
   PubMed Web of Science ®Google Scholar
• Kershaw MH, Westwood JA, Parker LL, A Phase I study on adoptive immunotherapy using gene-modified T cells for ovarian cancer. Clin Cancer Res 2006;12:6106-15
   PubMed Web of Science ®Google Scholar
• Till BG, Jensen MC, Wang J, Adoptive immunotherapy for indolent non-Hodgkin lymphoma and mantle cell lymphoma using genetically modified autologous CD20-specific T cells. Blood 2008;112:2261-71
   PubMed Web of Science ®Google Scholar
• Morgan RA, Yang JC, Kitano M, Case report of a serious adverse event following the administration of T cells transduced with a chimeric antigen receptor recognizing ERBB2. Mol Ther 2010;21:929-41
   Google Scholar
• Brentjens R, Yeh R, Bernal Y, Treatment of chronic lymphocytic leukemia with genetically targeted autologous T cells: case report of an unforeseen adverse event in a Phase I clinical trial. Mol Ther 2010;18:666-8
   PubMed Web of Science ®Google Scholar
• Cobbold M, Khan N, Pourgheysari B, Adoptive transfer of cytomegalovirus-specific CTL to stem cell transplant patients after selection by HLA-peptide tetramers. J Exp Med 2005;202:379-86
   PubMed Web of Science ®Google Scholar
• Heslop HE, Ng CY, Li C, Long-term restoration of immunity against Epstein-Barr virus infection by adoptive transfer of gene-modified virus-specific T lymphocytes. Nat Med 1996;2:551-5
   PubMed Web of Science ®Google Scholar
• Ciceri F, Labopin M, Aversa F, A survey of fully haploidentical hematopoietic stem cell transplantation in adults with high-risk acute leukemia: a risk factor analysis of outcomes for patients in remission at transplantation. Blood 2008;112:3574-81
   PubMed Web of Science ®Google Scholar
• Dey BR, Spitzer TR. Current status of haploidentical stem cell transplantation. Br J Haematol 2006;135:423-37
   PubMed Web of Science ®Google Scholar
• Small TN, Papadopoulos EB, Boulad F, Comparison of immune reconstitution after unrelated and related T-cell-depleted bone marrow transplantation: effect of patient age and donor leukocyte infusions. Blood 1999;93:467-80
   PubMed Web of Science ®Google Scholar
• Schub A, Schuster IG, Hammerschmidt W, Moosmann A. CMV-specific TCR-transgenic T cells for immunotherapy. J Immunol 2009;183:6819-30
   PubMed Web of Science ®Google Scholar
• Jurgens LA, Khanna R, Weber J, Orentas RJ. Transduction of primary lymphocytes with Epstein-Barr virus (EBV) latent membrane protein-specific T-cell receptor induces lysis of virus-infected cells: a novel strategy for the treatment of Hodgkin's disease and nasopharyngeal carcinoma. J Clin Immunol 2006;26:22-32
   PubMed Web of Science ®Google Scholar
• Haque T, Wilkie GM, Taylor C, Treatment of Epstein-Barr-virus-positive post-transplantation lymphoproliferative disease with partly HLA-matched allogeneic cytotoxic T cells. Lancet 2002;360:436-42
   PubMed Web of Science ®Google Scholar
• Melenhorst JJ, Solomon SR, Shenoy A, Robust expansion of viral antigen-specific CD4+ and CD8+ T cells for adoptive T cell therapy using gene-modified activated T cells as antigen presenting cells. J Immunother 2006;29:436-3; discussion 365-6
   PubMed Web of Science ®Google Scholar
• Muul LM, Candotti F. Immune responses to gene-modified T cells. Curr Gene Ther 2007;7:361-8
   PubMed Web of Science ®Google Scholar
• Muul LM, Tuschong LM, Soenen SL, Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial. Blood 2003;101:2563-9
   PubMed Web of Science ®Google Scholar
• Riddell SR, Elliott M, Lewinsohn DA, T-cell mediated rejection of gene-modified HIV-specific cytotoxic T lymphocytes in HIV-infected patients. Nat Med 1996;2:216-23
   PubMed Web of Science ®Google Scholar
• Riddell SR, Bleakley M, Nishida T, Adoptive transfer of allogeneic antigen-specific T cells. Biol Blood Marrow Transplant 2006;12(1 Suppl 1):9-12
   PubMed Web of Science ®Google Scholar
• Traversari C, Marktel S, Magnani Z, The potential immunogenicity of the TK suicide gene does not prevent full clinical benefit associated with the use of TK-transduced donor lymphocytes in HSCT for hematologic malignancies. Blood 2007;109:4708-15
   PubMed Web of Science ®Google Scholar
• Berger C, Flowers ME, Warren EH, Riddell SR. Analysis of transgene-specific immune responses that limit the in vivo persistence of adoptively transferred HSV-TK-modified donor T cells after allogeneic hematopoietic cell transplantation. Blood 2006;107:2294-302
   PubMed Web of Science ®Google Scholar
• Mercier-Letondal P, Deschamps M, Sauce D, Early immune response against retrovirally transduced herpes simplex virus thymidine kinase-expressing gene-modified T cells coinfused with a T cell-depleted marrow graft: an altered immune response? Hum Gene Ther 2008;19:937-50
   PubMed Web of Science ®Google Scholar
• Garin MI, Garrett E, Tiberghien P, Molecular mechanism for ganciclovir resistance in human T lymphocytes transduced with retroviral vectors carrying the herpes simplex virus thymidine kinase gene. Blood 2001;97:122-9
   PubMed Web of Science ®Google Scholar
• Mastaglio S, Stanghellini MT, Bordignon C, Progress and prospects: graft-versus-host disease. Gene Ther 2010;17:1309-17
   PubMed Web of Science ®Google Scholar
• Chalmers D, Ferrand C, Apperley JF, Elimination of the truncated message from the herpes simplex virus thymidine kinase suicide gene. Mol Ther 2001;4:146-8
   PubMed Web of Science ®Google Scholar
• Hacein-Bey-Abina S, Von Kalle C, Schmidt M, LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003;302:415-19
   PubMed Web of Science ®Google Scholar
• Stein S, Ott MG, Schultze-Strasser S, Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med 2010;16:198-204
   PubMed Web of Science ®Google Scholar
• Cavazzana-Calvo M, Payen E, Negre O, Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 2010;467:318-22
   PubMed Web of Science ®Google Scholar
• Recchia A, Bonini C, Magnani Z, Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T cells. Proc Natl Acad Sci USA 2006;103:1457-62
   PubMed Web of Science ®Google Scholar
• Newrzela S, Cornils K, Li Z, Resistance of mature T cells to oncogene transformation. Blood 2008;112:2278-86
   PubMed Web of Science ®Google Scholar
• Brewin J, Mancao C, Straathof K, Generation of EBV-specific cytotoxic T cells that are resistant to calcineurin inhibitors for the treatment of posttransplantation lymphoproliferative disease. Blood 2009;114:4792-803
   PubMed Web of Science ®Google Scholar
• Levine BL, Humeau LM, Boyer J, Gene transfer in humans using a conditionally replicating lentiviral vector. Proc Natl Acad Sci USA 2006;103:17372-7
   PubMed Web of Science ®Google Scholar
• Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009;326:818-23
   PubMed Web of Science ®Google Scholar
• Xue S, Gao L, Gillmore R, WT1-targeted immunotherapy of leukaemia. Blood Cells Mol Dis 2004;33:288-90
   PubMed Web of Science ®Google Scholar
• Sadovnikova E, Jopling LA, Soo KS, Stauss HJ. Generation of human tumor-reactive cytotoxic T cells against peptides presented by non-self HLA class I molecules. Eur J Immunol 1998;28:193-200
   PubMed Web of Science ®Google Scholar
• Bendle GM, Linnemann C, Hooijkaas AI, Lethal graft-versus-host disease in mouse models of T cell receptor gene therapy. Nat Med 2010;16:565-70
   PubMed Web of Science ®Google Scholar
• Schaft N, Lankiewicz B, Drexhage J, T cell re-targeting to EBV antigens following TCR gene transfer: CD28-containing receptors mediate enhanced antigen-specific IFNgamma production. Int Immunol 2006;18:591-601
   PubMed Web of Science ®Google Scholar
• Griffioen M, van Egmond HM, Barnby-Porritt H, Genetic engineering of virus-specific T cells with T-cell receptors recognizing minor histocompatibility antigens for clinical application. Haematologica 2008;93:1535-43
   PubMed Web of Science ®Google Scholar