An improved bicistronic CD20/tCD34 vector for efficient purification and in vivo depletion of gene-modified T cells for adoptive immunotherapy

Molecular Therapy

Volumn 18, Issue 7, 2010, Pages 1330-1338

  Vogler, Isabel ^a,b   Newrzela, Sebastian ^a   Hartmann, Sylvia ^b   Schneider, Nadine ^a   Von Laer, Dorothee ^a   Koehl, Ulrike ^b   Grez, Manuel ^a  

^a INSTITUTE FOR TUMOR BIOLOGY AND EXPERIMENTAL THERAPY   (Germany)

^b GOETHE UNIVERSITY   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

CD20 ANTIGEN; CD34 ANTIGEN; RETROVIRUS VECTOR; RITUXIMAB; TRASTUZUMAB;

ADAPTIVE IMMUNITY; ADOPTIVE IMMUNOTHERAPY; AMINO ACID SEQUENCE; ANIMAL EXPERIMENT; ANTIGEN EXPRESSION; ARTICLE; CD34 SELECTION; CD4 CD8 RATIO; CELL EXPANSION; COLITIS; CONTROLLED STUDY; DRUG SENSITIVITY; EXPRESSION VECTOR; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; IN VIVO STUDY; MOUSE; NONHUMAN; PHENOTYPE; PROTEIN BINDING; PROTEIN DEPLETION; PURIFICATION; SIGNAL TRANSDUCTION; SUICIDE GENE; T CELL DEPLETION; T LYMPHOCYTE;

ANIMALIA; MUS;

EID: 77954242723     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2010.83     Document Type: Article

References (50)

1. Aversa, F, Tabilio, A, Velardi, A, Cunningham, I, Terenzi, A, Falzetti, F et al. (1998). Treatment of high-risk acute leukemia with T-cell-depleted stem cells from related donors with one fully mismatched HLA haplotype. N Engl J Med 339: 1186-1193.
2. Ciceri, F, Labopin, M, Aversa, F, Rowe, JM, Bunjes, D, Lewalle, P et al.; Acute Leukemia Working Party (ALWP) of European Blood and Marrow Transplant (EBMT) Group. (2008). 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 112: 3574-3581.
3. Bader, P, Kreyenberg, H, Hoelle, W, Dueckers, G, Handgretinger, R, Lang, P et al. (2004). Increasing mixed chimerism is an important prognostic factor for unfavorable outcome in children with acute lymphoblastic leukemia after allogeneic stem-cell transplantation: possible role for pre-emptive immunotherapy? J Clin Oncol 22: 1696-1705.
4. Kolb, HJ, Schattenberg, A, Goldman, JM, Hertenstein, B, Jacobsen, N, Arcese, W et al.; European Group for Blood and Marrow Transplantation Working Party Chronic Leukemia. (1995). Graft-versus-leukemia effect of donor lymphocyte transfusions in marrow grafted patients. Blood 86: 2041-2050.
5. Dazzi, F and Goldman, JM (1998). Adoptive immunotherapy following allogeneic bone marrow transplantation. Annu Rev Med 49: 329-340.
6. Bonini, C, Ferrari, G, Verzeletti, S, Servida, P, Zappone, E, Ruggieri, L et al. (1997). HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia. Science 276: 1719-1724.
7. Cohen, JL, Boyer, O, Thomas-Vaslin, V and Klatzmann, D (1999). Suicide gene-mediated modulation of graft-versus-host disease. Leuk Lymphoma 34: 473-480.
8. Tiberghien, P, Ferrand, C, Lioure, B, Milpied, N, Angonin, R, Deconinck, E et al. (2001). Administration of herpes simplex-thymidine kinase-expressing donor T cells with a T-cell-depleted allogeneic marrow graft. Blood 97: 63-72.
9. Fehse, B, Ayuk, FA, Kröger, N, Fang, L, Kühlcke, K, Heinzelmann, M et al. (2004). Evidence for increased risk of secondary graft failure after in vivo depletion of suicide gene-modifed T lymphocytes transplanted in conjunction with CD34+-enriched blood stem cells. Blood 104: 3408-3409.
10. Ciceri, F, Bonini, C, Stanghellini, MT, Bondanza, A, Traversari, C, Salomoni, M et al. (2009). 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 10: 489-500.
11. Traversari, C, Marktel, S, Magnani, Z, Mangia, P, Russo, V, Ciceri, F et al. (2007). The potential immunogenicity of the TK suicide gene does not prevent full clinical beneft associated with the use of TK-transduced donor lymphocytes in HSCT for hematologic malignancies. Blood 109: 4708-4715.
12. Berger, C, Flowers, ME, Warren, EH and Riddell, SR (2006). Analysis of transgene-specifc immune responses that limit the in vivo persistence of adoptively transferred HSV-TK-modifed donor T cells after allogeneic hematopoietic cell transplantation. Blood 107: 2294-2302.
13. Tiberghien, P (2001). Use of suicide gene-expressing donor T-cells to control alloreactivity after haematopoietic stem cell transplantation. J Intern Med 249: 369-377.
14. Junker, K, Koehl, U, Zimmerman, S, Stein, S, Schwabe, D, Klingebiel, T et al. (2003). Kinetics of cell death in T lymphocytes genetically modifed with two novel suicide fusion genes. Gene Ther 10: 1189-1197.
15. Sato, T, Neschadim, A, Konrad, M, Fowler, DH, Lavie, A and Medin, JA (2007). Engineered human tmpk/AZT as a novel enzyme/prodrug axis for suicide gene therapy. Mol Ther 15: 962-970.
16. Introna, M, Barbui, AM, Bambacioni, F, Casati, C, Gaipa, G, Borleri, G et al. (2000). Genetic modifcation of human T cells with CD20: a strategy to purify and lyse transduced cells with anti-CD20 antibodies. Hum Gene Ther 11: 611-620.
17. Serafni, M, Manganini, M, Borleri, G, Bonamino, M, Imberti, L, Biondi, A et al. (2004). Characterization of CD20-transduced T lymphocytes as an alternative suicide gene therapy approach for the treatment of graft-versus-host disease. Hum Gene Ther 15: 63-76.
18. van Meerten, T, Claessen, MJ, Hagenbeek, A and Ebeling, SB (2006). The CD20/αCD20 'suicide' system: novel vectors with improved safety and expression profles and effcient elimination of CD20-transgenic T cells. Gene Ther 13: 789-797.
19. Szymczak, AL, Workman, CJ, Wang, Y, Vignali, KM, Dilioglou, S, Vanin, EF et al. (2004). Correction of multi-gene defciency in vivo using a single 'self-cleaving' 2A peptide-based retroviral vector. Nat Biotechnol 22: 589-594.
20. Fehse, B, Kustikova, OS, Li, Z, Wahlers, A, Bohn, W, Beyer, WR et al. (2002). A novel 'sort-suicide' fusion gene vector for T cell manipulation. Gene Ther 9: 1633-1638.
21. Newrzela, S, Cornils, K, Li, Z, Baum, C, Brugman, MH, Hartmann, M et al. (2008). Resistance of mature T cells to oncogene transformation. Blood 112: 2278-2286.
22. van Meerten, T, van Rijn, RS, Hol, S, Hagenbeek, A and Ebeling, SB (2006). Complement-induced cell death by rituximab depends on CD20 expression level and acts complementary to antibody-dependent cellular cytotoxicity. Clin Cancer Res 12: 4027-4035.
23. Moreno-Carranza, B, Gentsch, M, Stein, S, Schambach, A, Santilli, G, Rudolf, E et al. (2009). Transgene optimization signifcantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells. Gene Ther 16: 111-118.
24. Yang, S, Cohen, CJ, Peng, PD, Zhao, Y, Cassard, L, Yu, Z et al. (2008). Development of optimal bicistronic lentiviral vectors facilitates high-level TCR gene expression and robust tumor cell recognition. Gene Ther 15: 1411-1423.
25. Griffoen, M, van Egmond, HM, Barnby-Porritt, H, van der Hoorn, MA, Hagedoorn, RS, Kester, MG et al. (2008). Genetic engineering of virus-specifc T cells with T-cell receptors recognizing minor histocompatibility antigens for clinical application. Haematologica 93: 1535-1543.
26. Leisegang, M, Engels, B, Meyerhuber, P, Kieback, E, Sommermeyer, D, Xue, SA et al. (2008). Enhanced functionality of T cell receptor-redirected T cells is defned by the transgene cassette. J Mol Med 86: 573-583.
27. Tedder, TF and Engel, P (1994). CD20: a regulator of cell-cycle progression of B lymphocytes. Immunol Today 15: 450-454.
28. Li, H, Ayer, LM, Lytton, J and Deans, JP (2003). Store-operated cation entry mediated by CD20 in membrane rafts. J Biol Chem 278: 42427-42434.
29. Cragg, MS, Morgan, SM, Chan, HT, Morgan, BP, Filatov, AV, Johnson, PW et al. (2003). Complement-mediated lysis by anti-CD20 mAb correlates with segregation into lipid rafts. Blood 101: 1045-1052.
30. Deans, J P, Li, H and Polyak, MJ (2002). CD20-mediated apoptosis: signalling through lipid rafts. Immunology 107: 176-182.
31. Deans, J P, Kalt, L, Ledbetter, JA, Schieven, GL, Bolen, JB and Johnson, P (1995). Association of 75/80-kDa phosphoproteins and the tyrosine kinases Lyn, Fyn, and Lck with the B cell molecule CD20. Evidence against involvement of the cytoplasmic regions of CD20. J Biol Chem 270: 22632-22638.
32. Lange, C, Li, Z, Fang, L, Baum, C and Fehse, B (2007). CD34 modulates the traffcking behavior of hematopoietic cells in vivo. Stem Cells Dev 16: 297-304.
33. Bondanza, A, Valtolina, V, Magnani, Z, Ponzoni, M, Fleischhauer, K, Bonyhadi, M et al. (2006). Suicide gene therapy of graft-versus-host disease induced by central memory human T lymphocytes. Blood 107: 1828-1836.
34. Kaneko, S, Mastaglio, S, Bondanza, A, Ponzoni, M, Sanvito, F, Aldrighetti, L et al. (2009). IL-7 and IL-15 allow the generation of suicide gene-modifed alloreactive self-renewing central memory human T lymphocytes. Blood 113: 1006-1015.
35. Frecha, C, Costa, C, Nègre, D, Gauthier, E, Russell, SJ, Cosset, FL et al. (2008). Stable transduction of quiescent T cells without induction of cycle progression by a novel lentiviral vector pseudotyped with measles virus glycoproteins. Blood 112: 4843-4852.
36. Mombaerts, P, Iacomini, J, Johnson, RS, Herrup, K, Tonegawa, S and Papaioannou, VE (1992). RAG-1-defcient mice have no mature B and T lymphocytes. Cell 68: 869-877.
37. Bryson, JS, Zhang, L, Goes, SW, Jennings, CD, Caywood, BE, Carlson, SL et al. (2004). CD4+ T cells mediate murine syngeneic graft-versus-host disease-associated colitis. J Immunol 172: 679-687.
38. Koehl, U, Bochennek, K, Esser, R, Brinkmann, A, Quaritsch, R, Becker, M et al. (2008). ISHAGE-based single-platform fowcytometric analysis for measurement of absolute viable T cells in fresh or cryopreserved products: CD34/CD133 selected or CD3/CD19 depleted stem cells, DLI and purifed CD56+CD3-NK cells. Int J Hematol 87: 98-105.
39. Kimby, E (2005). Tolerability and safety of rituximab (MabThera). Cancer Treat Rev 31: 456-473.
40. Kapur, R, Ebeling, S and Hagenbeek, A (2008). B-cell involvement in chronic graft-versus-host disease. Haematologica 93: 1702-1711.
41. Christopeit, M, Schütte, V, Theurich, S, Weber, T, Grothe, W and Behre, G (2009). Rituximab reduces the incidence of acute graft-versus-host disease. Blood 113: 3130-3131.
42. Glennie, MJ, French, RR, Cragg, MS and Taylor, RP (2007). Mechanisms of killing by anti-CD20 monoclonal antibodies. Mol Immunol 44: 3823-3837.
43. Maloney, DG, Grillo-López, AJ, White, CA, Bodkin, D, Schilder, RJ, Neidhart, JA et al. (1997). IDEC-C2B8 (Rituximab) anti-CD20 monoclonal antibody therapy in patients with relapsed low-grade non-Hodgkin's lymphoma. Blood 90: 2188-2195.
44. Harjunpää, A, Wiklund, T, Collan, J, Janes, R, Rosenberg, J, Lee, D et al. (2001). Complement activation in circulation and central nervous system after rituximab (anti-CD20) treatment of B-cell lymphoma. Leuk Lymphoma 42: 731-738.
45. Teeling, JL, Mackus, WJ, Wiegman, LJ, van den Brakel, JH, Beers, SA, French, RR et al. (2006). The biological activity of human CD20 monoclonal antibodies is linked to unique epitopes on CD20. J Immunol 177: 362-371.
46. Beers, SA, Chan, CH, James, S, French, RR, Attfeld, KE, Brennan, CM et al. (2008). Type II (tositumomab) anti-CD20 monoclonal antibody out performs type I (rituximab-like) reagents in B-cell depletion regardless of complement activation. Blood 112: 4170-4177.
47. Stolz, C and Schuler, M (2009). Molecular mechanisms of resistance to Rituximab and pharmacologic strategies for its circumvention. Leuk Lymphoma 50: 873-885.
48. Milani, C and Castillo, J (2009). Veltuzumab, an anti-CD20 mAb for the treatment of non-Hodgkin's lymphoma, chronic lymphocytic leukemia and immune thrombocytopenic purpura. Curr Opin Mol Ther 11: 200-207.
49. Müller, T, Uherek, C, Maki, G, Chow, KU, Schimpf, A, Klingemann, HG et al. (2008). Expression of a CD20-specifc chimeric antigen receptor enhances cytotoxic activity of NK cells and overcomes NK-resistance of lymphoma and leukemia cells. Cancer Immunol Immunother 57: 411-423.
50. Zimmermann, SY, Esser, R, Rohrbach, E, Klingebiel, T and Koehl, U (2005). A novel four-colour fow cytometric assay to determine natural killer cell or T-cell-mediated cellular cytotoxicity against leukaemic cells in peripheral or bone marrow specimens containing greater than 20% of normal cells. J Immunol Methods 296: 63-76.