AAV2-mediated in vivo immune gene therapy of solid tumours

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• 作者：Sara A Collins (1) (2)
  Alexandra Buhles (1)
  Martina F Scallan (2)
  Patrick T Harrison (3)
  Deirdre M O'Hanlon (4)
  Gerald C O'Sullivan (1)
  Mark Tangney (1)
  
• 刊名：Genetic Vaccines and Therapy
• 出版年：2010
• 出版时间：December 2010
• 年：2010
• 卷：8
• 期：1
• 全文大小：1573KB
• 参考文献：1. Cochran AJ, Morton DL, Stern S, Lana AM, Essner R, Wen DR: Sentinel lymph nodes show profound downregulation of antigen-presenting cells of the paracortex: implications for tumor biology and treatment. / Mod Pathol 2001, 14:604-08. modpathol.3880358">CrossRef
  2. Kurnick JT, Ramirez-Montagut T, Boyle LA, Andrews DM, Pandolfi F, Durda PJ, Butera D, Dunn IS, Benson EM, Gobin SJ, van den Elsen PJ: A novel autocrine pathway of tumor escape from immune recognition: melanoma cell lines produce a soluble protein that diminishes expression of the gene encoding the melanocyte lineage melan-A/MART-1 antigen through down-modulation of its promoter. / J Immunol 2001, 167:1204-211.
  3. Uyttenhove C, Maryanski J, Boon T: Escape of mouse mastocytoma P815 after nearly complete rejection is due to antigen-loss variants rather than immunosuppression. / J Exp Med 1983, 157:1040-052. m.157.3.1040">CrossRef
  4. Cabrera CM, Jimenez P, Cabrera T, Esparza C, Ruiz-Cabello F, Garrido F: Total loss of MHC class I in colorectal tumors can be explained by two molecular pathways: beta2-microglobulin inactivation in MSI-positive tumors and LMP7/TAP2 downregulation in MSI-negative tumors. / Tissue Antigens 2003, 61:211-19. CrossRef
  5. Hui K, Grosveld F, Festenstein H: Rejection of transplantable AKR leukaemia cells following MHC DNA-mediated cell transformation. / Nature 1984, 311:750-52. CrossRef
  6. Galea-Lauri J, Farzaneh F, Gaken J: Novel costimulators in the immune gene therapy of cancer. / Cancer Gene Ther 1996, 3:202-14.
  7. Pardoll DM: Cancer vaccines. / Nat Med 1998, 4:525-31. m0598supp-525">CrossRef
  8. Collins SA, Guinn BA, Harrison PT, Scallan MF, O'Sullivan GC, Tangney M: Viral vectors in cancer immunotherapy: which vector for which strategy? / Curr Gene Ther 2008, 8:66-8. CrossRef
  9. Tangney M, Casey G, Larkin JO, Collins CG, Soden D, Cashman J, Whelan MC, O'Sullivan GC: Non-viral in vivo immune gene therapy of cancer: combined strategies for treatment of systemic disease. / Cancer Immunol Immunother 2006, 55:1443-450. CrossRef
  10. Zaiss AK, Muruve DA: Immunity to adeno-associated virus vectors in animals and humans: a continued challenge. / Gene Ther 2008, 15:808-16. CrossRef
  11. Herzog RW, Yang EY, Couto LB, Hagstrom JN, Elwell D, Fields PA, Burton M, Bellinger DA, Read MS, Brinkhous KM, / et al.: Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. / Nat Med 1999, 5:56-3. CrossRef
  12. Wang L, Takabe K, Bidlingmaier SM, Ill CR, Verma IM: Sustained correction of bleeding disorder in hemophilia B mice by gene therapy. / Proc Natl Acad Sci USA 1999, 96:3906-910. CrossRef
  13. Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A, Glader B, Chew AJ, Tai SJ, Herzog RW, / et al.: Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. / Nat Genet 2000, 24:257-61. CrossRef
  14. Aitken ML, Moss RB, Waltz DA, Dovey ME, Tonelli MR, McNamara SC, Gibson RL, Ramsey BW, Carter BJ, Reynolds TC: A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease. / Hum Gene Ther 2001, 12:1907-916. CrossRef
  15. Li C, Bowles DE, van Dyke T, Samulski RJ: Adeno-associated virus vectors: potential applications for cancer gene therapy. / Cancer Gene Ther 2005, 12:913-25. CrossRef
  16. Mohr A, Henderson G, Dudus L, Herr I, Kuerschner T, Debatin KM, Weiher H, Fisher KJ, Zwacka RM: AAV-encoded expression of TRAIL in experimental human colorectal cancer leads to tumor regression. / Gene Ther 2004, 11:534-43. CrossRef
  17. Shih CS, Laurie N, Holzmacher J, Spence Y, Nathwani AC, Davidoff AM, Dyer MA: AAV-mediated local delivery of interferon-beta for the treatment of retinoblastoma in preclinical models. / Neuromolecular Med 2009, 11:43-2. CrossRef
  18. Watanabe M, Boyer JL, Crystal RG: AAVrh.10-mediated genetic delivery of bevacizumab to the pleura to provide local anti-VEGF to suppress growth of metastatic lung tumors. / Gene Ther 2010, 17:1042-051. CrossRef
  19. Ramarathinam L, Castle M, Wu Y, Liu Y: T cell costimulation by B7/BB1 induces CD8 T cell-dependent tumor rejection: an important role of B7/BB1 in the induction, recruitment, and effector function of antitumor T cells. / J Exp Med 1994, 179:1205-214. m.179.4.1205">CrossRef
  20. Borrello I, Pardoll D: GM-CSF-based cellular vaccines: a review of the clinical experience. / Cytokine Growth Factor Rev 2002, 13:185-93. CrossRef
  21. Linsley PS, Clark EA, Ledbetter JA: T-cell antigen CD28 mediates adhesion with B cells by interacting with activation antigen B7/BB-1. / Proc Natl Acad Sci USA 1990, 87:5031-035. CrossRef
  22. Linsley PS, Bradshaw J, Urnes M, Grosmaire L, Ledbetter JA: CD28 engagement by B7/BB-1 induces transient down-regulation of CD28 synthesis and prolonged unresponsiveness to CD28 signaling. / J Immunol 1993, 150:3161-169.
  23. Chen L, Ashe S, Brady WA, Hellstrom I, Hellstrom KE, Ledbetter JA, McGowan P, Linsley PS: Costimulation of antitumor immunity by the B7 counterreceptor for the T lymphocyte molecules CD28 and CTLA-4. / Cell 1992, 71:1093-102. CrossRef
  24. Chen L, McGowan P, Ashe S, Johnston J, Li Y, Hellstrom I, Hellstrom KE: Tumor immunogenicity determines the effect of B7 costimulation on T cell-mediated tumor immunity. / J Exp Med 1994, 179:523-32. m.179.2.523">CrossRef
  25. Townsend SE, Su FW, Atherton JM, Allison JP: Specificity and longevity of antitumor immune responses induced by B7-transfected tumors. / Cancer Res 1994, 54:6477-483.
  26. Isaka Y, Nakamura H, Mizui M, Takabatake Y, Horio M, Kawachi H, Shimizu F, Imai E, Hori M: DNAzyme for TGF-beta suppressed extracellular matrix accumulation in experimental glomerulonephritis. / Kidney Int 2004, 66:586-90. CrossRef
  27. Stripecke R, Cardoso AA, Pepper KA, Skelton DC, Yu XJ, Mascarenhas L, Weinberg KI, Nadler LM, Kohn DB: Lentiviral vectors for efficient delivery of CD80 and granulocyte-macrophage- colony-stimulating factor in human acute lymphoblastic leukemia and acute myeloid leukemia cells to induce antileukemic immune responses. / Blood 2000, 96:1317-326.
  28. Collins CG, Tangney M, Larkin JO, Casey G, Whelan MC, Cashman J, Murphy J, Soden D, Vejda S, McKenna S, / et al.: Local gene therapy of solid tumors with GM-CSF and B7- eradicates both treated and distal tumors. / Cancer Gene Ther 2006, 13:1061-071. CrossRef
  29. Malecki M, Kolsut P, Proczka R: Angiogenic and antiangiogenic gene therapy. / Gene Ther 2005,12(Suppl 1):S159-69. CrossRef
  30. Holleb AI, Folkman J: Tumor angiogenesis. / CA Cancer J Clin 1972, 22:226-29. CrossRef
  31. Ganss R, Arnold B, Hammerling GJ: Mini-review: overcoming tumor-intrinsic resistance to immune effector function. / Eur J Immunol 2004, 34:2635-641. CrossRef
  32. Vande Woude GF, Jeffers M, Cortner J, Alvord G, Tsarfaty I, Resau J: Met-HGF/SF: tumorigenesis, invasion and metastasis. / Ciba Found Symp 1997, 212:119-30. discussion 130-12, 148-54
  33. Jiang W, Hiscox S, Matsumoto K, Nakamura T: Hepatocyte growth factor/scatter factor, its molecular, cellular and clinical implications in cancer. / Crit Rev Oncol Hematol 1999, 29:209-48. CrossRef
  34. Matsumoto K, Nakamura T: Hepatocyte growth factor (HGF) as a tissue organizer for organogenesis and regeneration. / Biochem Biophys Res Commun 1997, 239:639-44. CrossRef
  35. Birchmeier C, Gherardi E: Developmental roles of HGF/SF and its receptor, the c-Met tyrosine kinase. / Trends Cell Biol 1998, 8:404-10. CrossRef
  36. Matsumoto K, Nakamura T: NK4 (HGF-antagonist/angiogenesis inhibitor) in cancer biology and therapeutics. / Cancer Sci 2003, 94:321-27. CrossRef
  37. Date K, Matsumoto K, Shimura H, Tanaka M, Nakamura T: HGF/NK4 is a specific antagonist for pleiotrophic actions of hepatocyte growth factor. / FEBS Lett 1997, 420:1-. CrossRef
  38. Cioce V, Csaky KG, Chan AM, Bottaro DP, Taylor WG, Jensen R, Aaronson SA, Rubin JS: Hepatocyte growth factor (HGF)/NK1 is a naturally occurring HGF/scatter factor variant with partial agonist/antagonist activity. / J Biol Chem 1996, 271:13110-3115. CrossRef
  39. Schwall RH, Chang LY, Godowski PJ, Kahn DW, Hillan KJ, Bauer KD, Zioncheck TF: Heparin induces dimerization and confers proliferative activity onto the hepatocyte growth factor antagonists NK1 and NK2. / J Cell Biol 1996, 133:709-18. CrossRef
  40. Silvagno F, Follenzi A, Arese M, Prat M, Giraudo E, Gaudino G, Camussi G, Comoglio PM, Bussolino F: In vivo activation of met tyrosine kinase by heterodimeric hepatocyte growth factor molecule promotes angiogenesis. / Arterioscler Thromb Vasc Biol 1995, 15:1857-865.
  41. Montesano R, Soriano JV, Malinda KM, Ponce ML, Bafico A, Kleinman HK, Bottaro DP, Aaronson SA: Differential effects of hepatocyte growth factor isoforms on epithelial and endothelial tubulogenesis. / Cell Growth Differ 1998, 9:355-65.
  42. Matsumoto K, Nakamura T: Mechanisms and significance of bifunctional NK4 in cancer treatment. / Biochem Biophys Res Commun 2005, 333:316-27. CrossRef
  43. Kuba K, Matsumoto K, Ohnishi K, Shiratsuchi T, Tanaka M, Nakamura T: Kringle 1- of hepatocyte growth factor inhibits proliferation and migration of human microvascular endothelial cells. / Biochem Biophys Res Commun 2000, 279:846-52. CrossRef
  44. Buhles A, Collins SA, van Pijkeren JP, Rajendran S, Miles M, O'Sullivan GC, O'Hanlon DM, Tangney M: Anti-metastatic effects of viral and non-viral mediated Nk4 delivery to tumours. / Genet Vaccines Ther 2009, 7:5. CrossRef
  45. Winn HJ: Immune mechanisms in homotransplantation. II. Quantitative assay of the immunologic activity of lymphoid cells stimulated by tumor homografts. / J Immunol 1961, 86:228-39.
  46. Tirapu I, Huarte E, Guiducci C, Arina A, Zaratiegui M, Murillo O, Gonzalez A, Berasain C, Berraondo P, Fortes P, / et al.: Low surface expression of B7- (CD80) is an immunoescape mechanism of colon carcinoma. / Cancer Res 2006, 66:2442-450. CrossRef
  47. Lo WD, Qu G, Sferra TJ, Clark R, Chen R, Johnson PR: Adeno-associated virus-mediated gene transfer to the brain: duration and modulation of expression. / Hum Gene Ther 1999, 10:201-13. CrossRef
  48. Rolling F, Shen WY, Barnett NL, Tabarias H, Kanagasingam Y, Constable I, Rakoczy PE: Long-term real-time monitoring of adeno-associated virus-mediated gene expression in the rat retina. / Clin Experiment Ophthalmol 2000, 28:382-86. CrossRef
  49. Vassalli G, Bueler H, Dudler J, von Segesser LK, Kappenberger L: Adeno-associated virus (AAV) vectors achieve prolonged transgene expression in mouse myocardium and arteries in vivo: a comparative study with adenovirus vectors. / Int J Cardiol 2003, 90:229-38. CrossRef
  50. Seymour LW, Fisher KD, Green NK, Hale SJ, Lyons M, Mautner V, Nicum S, Onion D, Oupicky D, Stevenson M, Ulbrich K: Adenovirus retargeting and systemic delivery. / Ernst Schering Res Found Workshop 2003, 107-14.
  51. Tangney M, Ahmad S, Collins SA, O'Sullivan GC: Gene therapy for prostate cancer. / Postgrad Med 2010, 122:166-80. m.2010.05.2154">CrossRef
  52. Cashman JP, Larkin JO, Casey G, Whelan MC, Collins C, Aarons S, Tangney M, O'Sullivan GC: Immune gene therapy as a neoadjuvant to surgical excision to control metastatic cancers. / Cancer Lett 2008, 262:94-02. CrossRef
  53. Casey G, Cashman JP, Morrissey D, Whelan MC, Larkin JO, Soden DM, Tangney M, O'Sullivan GC: Sonoporation mediated immunogene therapy of solid tumors. / Ultrasound Med Biol 2010, 36:430-40. medbio.2009.11.005">CrossRef
  54. Ponnazhagan S, Mahendra G, Kumar S, Thompson JA, Castillas M Jr: Conjugate-based targeting of recombinant adeno-associated virus type 2 vectors by using avidin-linked ligands. / J Virol 2002, 76:12900-2907. CrossRef
  55. Bartlett JS, Kleinschmidt J, Boucher RC, Samulski RJ: Targeted adeno-associated virus vector transduction of nonpermissive cells mediated by a bispecific F(ab'gamma)2 antibody. / Nat Biotechnol 1999, 17:181-86. CrossRef
  56. O'Brien MG, Collins CG, Collins JK, Shanahan F, O'Sullivan GC: Oral immune tolerance to tumor specific antigens may confer growth advantage to esophageal and gastric cancers. / Dis Esophagus 2003, 16:218-23. CrossRef
  57. Peppoloni S, Herberman RB, Gorelik E: Induction of highly immunogenic variants of Lewis lung carcinoma tumor by ultraviolet irradiation. / Cancer Res 1985, 45:2560-566.
  58. Shoham J, Hirsch R, Zakay-Rones Z, Osband ME, Brennert HJ: Augmentation of tumor cell immunogenicity by viruses--an approach to specific immunotherapy of cancer. / Nat Immun Cell Growth Regul 1990, 9:165-72.
  59. Cai KX, Tse LY, Leung C, Tam PK, Xu R, Sham MH: Suppression of lung tumor growth and metastasis in mice by adeno-associated virus-mediated expression of vasostatin. / Clin Cancer Res 2008, 14:939-49. CrossRef
  60. Whelan MC, Casey G, MacConmara M, Lederer JA, Soden D, Collins JK, Tangney M, O'Sullivan GC: Effective immunotherapy of weakly immunogenic solid tumours using a combined immunogene therapy and regulatory T-cell inactivation. / Cancer Gene Ther 2010, 17:501-11. CrossRef
  61. Davis HL, Millan CL, Watkins SC: Immune-mediated destruction of transfected muscle fibers after direct gene transfer with antigen-expressing plasmid DNA. / Gene Ther 1997, 4:181-88. CrossRef
  62. Feldman AL, Libutti SK: Progress in antiangiogenic gene therapy of cancer. / Cancer 2000, 89:1181-194. CrossRef
  63. Hamzah J, Jugold M, Kiessling F, Rigby P, Manzur M, Marti HH, Rabie T, Kaden S, Grone HJ, Hammerling GJ, / et al.: Vascular normalization in Rgs5-deficient tumours promotes immune destruction. / Nature 2008, 453:410-14. CrossRef
  64. Ryschich E, Schmidt J, Hammerling GJ, Klar E, Ganss R: Transformation of the microvascular system during multistage tumorigenesis. / Int J Cancer 2002, 97:719-25. CrossRef
  65. Buckanovich RJ, Facciabene A, Kim S, Benencia F, Sasaroli D, Balint K, Katsaros D, O'Brien-Jenkins A, Gimotty PA, Coukos G: Endothelin B receptor mediates the endothelial barrier to T cell homing to tumors and disables immune therapy. / Nat Med 2008, 14:28-6. m1699">CrossRef
  
• 作者单位：Sara A Collins (1) (2)
  Alexandra Buhles (1)
  Martina F Scallan (2)
  Patrick T Harrison (3)
  Deirdre M O'Hanlon (4)
  Gerald C O'Sullivan (1)
  Mark Tangney (1)
  
  1. Cork Cancer Research Centre, Mercy University Hospital and Leslie C. Quick Jnr. Laboratory, University College Cork, Cork, Ireland
  2. Department of Microbiology, University College Cork, Cork, Ireland
  3. Department of Physiology and Biosciences Institute, University College Cork, Cork, Ireland
  4. Department of Surgery, South Infirmary Victoria University Hospital, Cork, Ireland
  
• ISSN：1479-0556

文摘

Background Many strategies have been adopted to unleash the potential of gene therapy for cancer, involving a wide range of therapeutic genes delivered by various methods. Immune therapy has become one of the major strategies adopted for cancer gene therapy and seeks to stimulate the immune system to target tumour antigens. In this study, the feasibility of AAV2 mediated immunotherapy of growing tumours was examined, in isolation and combined with anti-angiogenic therapy. Methods Immune-competent Balb/C or C57 mice bearing subcutaneous JBS fibrosarcoma or Lewis Lung Carcinoma (LLC) tumour xenografts respectively were treated by intra-tumoural administration of AAV2 vector encoding the immune up-regulating cytokine granulocyte macrophage-colony stimulating factor (GM-CSF) and the co-stimulatory molecule B7-1 to subcutaneous tumours, either alone or in combination with intra-muscular (IM) delivery of AAV2 vector encoding Nk4 14 days prior to tumour induction. Tumour growth and survival was monitored for all animals. Cured animals were re-challenged with tumourigenic doses of the original tumour type. In vivo cytotoxicity assays were used to investigate establishment of cell-mediated responses in treated animals. Results AAV2-mediated GM-CSF, B7-1 treatment resulted in a significant reduction in tumour growth and an increase in survival in both tumour models. Cured animals were resistant to re-challenge, and induction of T cell mediated anti-tumour responses were demonstrated. Adoptive transfer of splenocytes to na?ve animals prevented tumour establishment. Systemic production of Nk4 induced by intra-muscular (IM) delivery of Nk4 significantly reduced subcutaneous tumour growth. However, combination of Nk4 treatment with GM-CSF, B7-1 therapy reduced the efficacy of the immune therapy. Conclusions Overall, this study demonstrates the potential for in vivo AAV2 mediated immune gene therapy, and provides data on the inter-relationship between tumour vasculature and immune cell recruitment.