Gene Therapy for Lung Neoplasms

Clinics in Chest Medicine

Volumn 32, Issue 4, 2011, Pages 865-885

  Vachani, Anil ^a   Moon, Edmund ^a   Wakeam, Elliot ^b   Haas, Andrew R ^a   Sterman, Daniel H ^a   Albelda, Steven M ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF TORONTO   (Canada)

Author keywords

Gene therapy; Immunotherapy; Lung cancer; Mesothelioma

Indexed keywords

ALPHA 1,3 GALACTOSYLTRANSFERASE; APRINOCARSEN; CARBOPLATIN; CGP 69846A; CISPLATIN; CYTOTOXIC T LYMPHOCYTE ANTIGEN 4; ETOPOSIDE; FLUOROURACIL; FUS1 PROTEIN; GEMCITABINE; GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR; HLA A1 ANTIGEN; HLA A2 ANTIGEN; INTERLEUKIN 12; IPILIMUMAB; L 5235; LIPOSOME; MESSENGER RNA; MUCIN 1; NAVELBINE; OBLIMERSEN; OLIGONUCLEOTIDE; PROTEIN P53; SMALL INTERFERING RNA; TG 4010; TUMOR CELL VACCINE; TUMOR SUPPRESSOR PROTEIN; UNCLASSIFIED DRUG; UNINDEXED DRUG; VACCINIA VACCINE; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ADENOVIRUS; ANTISENSE THERAPY; CANCER RADIOTHERAPY; CANCER SURGERY; DISEASE COURSE; DNA MODIFICATION; DRUG DELIVERY SYSTEM; FOWLPOX VIRUS; GENE TARGETING; GENE THERAPY; GENE TRANSFER; GENETIC TRANSFECTION; HUMAN; IMMUNE RESPONSE; IMMUNOTHERAPY; LENTIVIRINAE; LUNG CANCER; LUNG LAVAGE; LUNG NON SMALL CELL CANCER; MESOTHELIOMA; PRIORITY JOURNAL; PROGNOSIS; RETROVIRUS; REVIEW; TUMOR GROWTH; TUMOR SUPPRESSOR GENE; VACCINIA VIRUS;

GENE EXPRESSION REGULATION, NEOPLASTIC; GENE THERAPY; GENETIC VECTORS; HUMANS; IMMUNOTHERAPY; LUNG NEOPLASMS; MESOTHELIOMA;

EID: 80455176694     PISSN: 02725231     EISSN: 15578216     Source Type: Journal    
DOI: 10.1016/j.ccm.2011.08.006     Document Type: Review

References (86)

1. Maguire A.M., Simonelli F., Pierce E.A., et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 2008, 358(21):2240-2248.
2. Fischer A., Cavazzana-Calvo M. Gene therapy of inherited diseases. Lancet 2008, 371(9629):2044-2047.
3. Young L.S., Searle P.F., Onion D., et al. Viral gene therapy strategies: from basic science to clinical application. J Pathol 2006, 208(2):299-318.
4. Bachtarzi H., Stevenson M., Fisher K. Cancer gene therapy with targeted adenoviruses. Expert Opin Drug Deliv 2008, 5(11):1231-1240.
5. Douglas J.T. Adenoviral vectors for gene therapy. Mol Biotechnol 2007, 36(1):71-80.
6. Breckpot K., Emeagi P.U., Thielemans K. Lentiviral vectors for anti-tumor immunotherapy. Curr Gene Ther 2008, 8(6):438-448.
7. Schambach A., Baum C. Clinical application of lentiviral vectors-concepts and practice. Curr Gene Ther 2008, 8(6):474-482.
8. Daya S., Berns K.I. Gene therapy using adeno-associated virus vectors. Clin Microbiol Rev 2008, 21(4):583-593.
9. Schultz B.R., Chamberlain J.S. Recombinant adeno-associated virus transduction and integration. Mol Ther 2008, 16(7):1189-1199.
10. Garber D.A., O'Mara L.A., Zhao J., et al. Expanding the repertoire of Modified Vaccinia Ankara-based vaccine vectors via genetic complementation strategies. PLoS One 2009, 4(5):e5445.
11. Kirn D.H., Thorne S.H. Targeted and armed oncolytic poxviruses: a novel multi-mechanistic therapeutic class for cancer. Nat Rev Cancer 2009, 9(1):64-71.
12. Chesnoy S., Huang L. Structure and function of lipid-DNA complexes for gene delivery. Annu Rev Biophys Biomol Struct 2000, 29:27-47.
13. Niidome T., Huang L. Gene therapy progress and prospects: nonviral vectors. Gene Ther 2002, 9(24):1647-1652.
14. Roth J.A., Nguyen D., Lawrence D.D., et al. Retrovirus-mediated wild-type p53 gene transfer to tumors of patients with lung cancer. Nat Med 1996, 2(9):985-991.
15. Schuler M., Rochlitz C., Horowitz J.A., et al. A phase I study of adenovirus-mediated wild-type p53 gene transfer in patients with advanced non-small cell lung cancer. Hum Gene Ther 1998, 9(14):2075-2082.
16. Swisher S.G., Roth J.A., Nemunaitis J., et al. Adenovirus-mediated p53 gene transfer in advanced non-small-cell lung cancer. J Natl Cancer Inst 1999, 91(9):763-771.
17. Fujiwara T., Tanaka N., Kanazawa S., et al. Multicenter phase I study of repeated intratumoral delivery of adenoviral p53 in patients with advanced non-small-cell lung cancer. J Clin Oncol 2006, 24(11):1689-1699.
18. Nemunaitis J., Swisher S.G., Timmons T., et al. Adenovirus-mediated p53 gene transfer in sequence with cisplatin to tumors of patients with non-small-cell lung cancer. J Clin Oncol 2000, 18(3):609-622.
19. Swisher S.G., Roth J.A., Komaki R., et al. Induction of p53-regulated genes and tumor regression in lung cancer patients after intratumoral delivery of adenoviral p53 (INGN 201) and radiation therapy. Clin Cancer Res 2003, 9(1):93-101.
20. Schuler M., Herrmann R., De Greve J.L., et al. Adenovirus-mediated wild-type p53 gene transfer in patients receiving chemotherapy for advanced non-small-cell lung cancer: results of a multicenter phase II study. J Clin Oncol 2001, 19(6):1750-1758.
21. Keedy V., Wang W., Schiller J., et al. Phase I study of adenovirus p53 administered by bronchoalveolar lavage in patients with bronchioloalveolar cell lung carcinoma: ECOG 6597. J Clin Oncol 2008, 26(25):4166-4171.
22. Guan Y.S., Liu Y., Zou Q., et al. Adenovirus-mediated wild-type p53 gene transfer in combination with bronchial arterial infusion for treatment of advanced non-small-cell lung cancer, one year follow-up. J Zhejiang Univ Sci B 2009, 10(5):331-340.
23. Peng Z. Current status of gendicine in China: recombinant human ad-p53 agent for treatment of cancers. Hum Gene Ther 2005, 16(9):1016-1027.
24. Ji L., Roth J.A. Tumor suppressor FUS1 signaling pathway. J Thorac Oncol 2008, 3(4):327-330.
25. Dunn G.P., Bruce A.T., Ikeda H., et al. Cancer immunoediting: from immunosurveillance to tumor escape. Nat Immunol 2002, 3(11):991-998.
26. Brower V. Approval of Provenge seen as first step for cancer treatment vaccines. J Natl Cancer Inst 2010, 102(15):1108-1110.
27. Morgan R.A., Dudley M.E., Wunderlich J.R., et al. Cancer regression in patients after transfer of genetically engineered lymphocytes. Science 2006, 314(5796):126-129.
28. Leen A.M., Myers G.D., Sili U., et al. Monoculture-derived T lymphocytes specific for multiple viruses expand and produce clinically relevant effects in immunocompromised individuals. Nat Med 2006, 12(10):1160-1166.
29. Heslop H.E., Slobod K.S., Pule M.A., et al. Long-term outcome of EBV-specific T-cell infusions to prevent or treat EBV-related lymphoproliferative disease in transplant recipients. Blood 2010, 115(5):925-935.
30. Brenner C.D., King S., Przewoznik M., et al. Requirements for control of B-cell lymphoma by NK cells. Eur J Immunol 2010, 40(2):494-504.
31. Hodi F.S., O'Day S.J., McDermott D.F., et al. Improved survival with ipilimumab in patients with metastatic melanoma. N Engl J Med 2010, 363(8):711-723.
32. Nemunaitis J., Dillman R.O., Schwarzenberger P.O., et al. Phase II study of belagenpumatucel-L, atransforming growth factor beta-2 antisense gene-modified allogeneic tumor cell vaccine in non-small-cell lung cancer. J Clin Oncol 2006, 24(29):4721-4730.
33. Nemunaitis J., Nemunaitis M., Senzer N., et al. Phase II trial of belagenpumatucel-L, a TGF-beta2 antisense gene modified allogeneic tumor vaccine in advanced non small cell lung cancer (NSCLC) patients. Cancer Gene Ther 2009, 16(8):620-624.
34. Salgia R., Lynch T., Skarin A., et al. Vaccination with irradiated autologous tumor cells engineered to secrete granulocyte-macrophage colony-stimulating factor augments antitumor immunity in some patients with metastatic non-small-cell lung carcinoma. J Clin Oncol 2003, 21(4):624-630.
35. Nemunaitis J., Sterman D., Jablons D., et al. Granulocyte-macrophage colony-stimulating factor gene-modified autologous tumor vaccines in non-small-cell lung cancer. J Natl Cancer Inst 2004, 96(4):326-331.
36. Nemunaitis J., Jahan T., Ross H., et al. Phase 1/2 trial of autologous tumor mixed with an allogeneic GVAX vaccine in advanced-stage non-small-cell lung cancer. Cancer Gene Ther 2006, 13(6):555-562.
37. Raez L.E., Cassileth P.A., Schlesselman J.J., et al. Allogeneic vaccination with a B7.1 HLA-A gene-modified adenocarcinoma cell line in patients with advanced non-small-cell lung cancer. J Clin Oncol 2004, 22(14):2800-2807.
38. Morris J.C., Vahanian N., Janik J.E., et al. Phase I study of an antitumor vaccination using alpha(1,3)galactosyltransferase expressing allogeneic tumor cells in patients with refractory or recurrent non-small cell lung cancer (NSCLC). J Clin Oncol 2005, 23(16S):2586.
39. Antonia S.J., Mirza N., Fricke I., et al. Combination of p53 cancer vaccine with chemotherapy in patients with extensive stage small cell lung cancer. Clin Cancer Res 2006, 12(3 Pt 1):878-887.
40. Baratelli F., Takedatsu H., Hazra S., et al. Pre-clinical characterization of GMP grade CCL21-gene modified dendritic cells for application in a phase I trial in non-small cell lung cancer. J Transl Med 2008, 6:38.
41. Ramlau R., Quoix E., Rolski J., et al. A phase II study of Tg4010 (mva-Muc1-Il2) in association with chemotherapy in patients with stage III/IV non-small cell lung cancer. J Thorac Oncol 2008, 3(7):735-744.
42. Nemunaitis J., Meyers T., Senzer N., et al. Phase I trial of sequential administration of recombinant DNA and adenovirus expressing L523S protein in early stage non-small-cell lung cancer. Mol Ther 2006, 13(6):1185-1191.
43. Tursz T., Cesne A.L., Baldeyrou P., et al. Phase I study of a recombinant adenovirus-mediated gene transfer in lung cancer patients. J Natl Cancer Inst 1996, 88(24):1857-1863.
44. Molinier-Frenkel V., Le Boulaire C., Le Gal F.A., et al. Longitudinal follow-up of cellular and humoral immunity induced by recombinant adenovirus-mediated gene therapy in cancer patients. Hum Gene Ther 2000, 11(13):1911-1920.
45. Griscelli F., Opolon P., Saulnier P., et al. Recombinant adenovirus shedding after intratumoral gene transfer in lung cancer patients. Gene Ther 2003, 10(5):386-395.
46. Villalona-Calero M.A., Ritch P., Figueroa J.A., et al. A phase I/II study of LY900003, an antisense inhibitor of protein kinase C-alpha, in combination with cisplatin and gemcitabine in patients with advanced non-small cell lung cancer. Clin Cancer Res 2004, 10(18 Pt 1):6086-6093.
47. Vansteenkiste J., Canon J.L., Riska H., et al. Randomized phase II evaluation of aprinocarsen in combination with gemcitabine and cisplatin for patients with advanced/metastatic non-small cell lung cancer. Invest New Drugs 2005, 23(3):263-269.
48. Ritch P., Rudin C.M., Bitran J.D., et al. Phase II study of PKC-alpha antisense oligonucleotide aprinocarsen in combination with gemcitabine and carboplatin in patients with advanced non-small cell lung cancer. Lung Cancer 2006, 52(2):173-180.
49. Paz-Ares L., Douillard J.Y., Koralewski P., et al. Phase III study of gemcitabine and cisplatin with or without aprinocarsen, a protein kinase C-alpha antisense oligonucleotide, in patients with advanced-stage non-small-cell lung cancer. J Clin Oncol 2006, 24(9):1428-1434.
50. Cunningham C.C., Holmlund J.T., Schiller J.H., et al. A phase I trial of c-Raf kinase antisense oligonucleotide ISIS 5132 administered as a continuous intravenous infusion in patients with advanced cancer. Clin Cancer Res 2000, 6(5):1626-1631.
51. Rudin C.M., Holmlund J., Fleming G.F., et al. Phase I trial of ISIS 5132, an antisense oligonucleotide inhibitor of c-Raf-1, administered by 24-hour weekly infusion to patients with advanced cancer. Clin Cancer Res 2001, 7(5):1214-1220.
52. Stevenson J.P., Yao K.S., Gallagher M., et al. Phase I clinical/pharmacokinetic and pharmacodynamic trial of the c-Raf-1 antisense oligonucleotide ISIS 5132 (CGP 69846A). J Clin Oncol 1999, 17(7):2227-2236.
53. Coudert B., Anthoney A., Fiedler W., et al. Phase II trial with ISIS 5132 in patients with small-cell (SCLC) and non-small cell (NSCLC) lung cancer. A European Organization for Research and Treatment of Cancer (EORTC) early clinical studies group report. Eur J Cancer 2001, 37(17):2194-2198.
54. Rudin C.M., Otterson G.A., Mauer A.M., et al. A pilot trial of G3139, a bcl-2 antisense oligonucleotide, and paclitaxel in patients with chemorefractory small-cell lung cancer. Ann Oncol 2002, 13(4):539-545.
55. Rudin C.M., Kozloff M., Hoffman P.C., et al. Phase I study of G3139, a bcl-2 antisense oligonucleotide, combined with carboplatin and etoposide in patients with small-cell lung cancer. J Clin Oncol 2004, 22(6):1110-1117.
56. Rudin C.M., Salgia R., Wang X., et al. Randomized phase II study of carboplatin and etoposide with or without the bcl-2 antisense oligonucleotide oblimersen for extensive-stage small-cell lung cancer: CALGB 30103. J Clin Oncol 2008, 26(6):870-876.
57. Nemunaitis J., Cunningham C., Buchanan A., et al. Intravenous infusion of a replication-selective adenovirus (ONYX-015) in cancer patients: safety, feasibility and biological activity. Gene Ther 2001, 8(10):746-759.
58. Nemunaitis J., Khuri F., Ganly I., et al. Phase II trial of intratumoral administration of ONYX-015, a replication-selective adenovirus, in patients with refractory head and neck cancer. J Clin Oncol 2001, 19(2):289-298.
59. Nemunaitis J., Senzer N., Sarmiento S., et al. A phase I trial of intravenous infusion of ONYX-015 and enbrel in solid tumor patients. Cancer Gene Ther 2007, 14(11):885-893.
60. Horig H., Lee D.S., Conkright W., et al. Phase I clinical trial of a recombinant canarypoxvirus (ALVAC) vaccine expressing human carcinoembryonic antigen and the B7.1 co-stimulatory molecule. Cancer Immunol Immunother 2000, 49(9):504-514.
61. Marshall J.L., Gulley J.L., Arlen P.M., et al. Phase I study of sequential vaccinations with fowlpox-CEA(6D)-TRICOM alone and sequentially with vaccinia-CEA(6D)-TRICOM, with and without granulocyte-macrophage colony-stimulating factor, in patients with carcinoembryonic antigen-expressing carcinomas. J Clin Oncol 2005, 23(4):720-731.
62. Morse M.A., Clay T.M., Hobeika A.C., et al. Phase I study of immunization with dendritic cells modified with fowlpox encoding carcinoembryonic antigen and costimulatory molecules. Clin Cancer Res 2005, 11(8):3017-3024.
63. Senzer N., Mani S., Rosemurgy A., et al. TNFerade biologic, an adenovector with a radiation-inducible promoter, carrying the human tumor necrosis factor alpha gene: a phase I study in patients with solid tumors. J Clin Oncol 2004, 22(4):592-601.
64. McLoughlin J.M., McCarty T.M., Cunningham C., et al. TNFerade, an adenovector carrying the transgene for human tumor necrosis factor alpha, for patients with advanced solid tumors: surgical experience and long-term follow-up. Ann Surg Oncol 2005, 12(10):825-830.
65. Tiberghien P. Use of suicide genes in gene therapy. J Leukoc Biol 1994, 56(2):203-209.
66. Matthews T., Boehme R. Antiviral activity and mechanism of action of ganciclovir. Rev Infect Dis 1988, 10(Suppl 3):S490-S494.
67. Sterman D.H., Treat J., Litzky L.A., et al. Adenovirus-mediated herpes simplex virus thymidine kinase/ganciclovir gene therapy in patients with localized malignancy: results of a phase I clinical trial in malignant mesothelioma. Hum Gene Ther 1998, 9(7):1083-1092.
68. Sterman D.H., Molnar-Kimber K., Iyengar T., et al. A pilot study of systemic corticosteroid administration in conjunction with intrapleural adenoviral vector administration in patients with malignant pleural mesothelioma. Cancer Gene Ther 2000, 7(12):1511-1518.
69. Sterman D.H., Recio A., Vachani A., et al. Long-term follow-up of patients with malignant pleural mesothelioma receiving high-dose adenovirus herpes simplex thymidine kinase/ganciclovir suicide gene therapy. Clin Cancer Res 2005, 11(20):7444-7453.
70. Harrison L.H., Schwarzenberger P.O., Byrne P.S., et al. Gene-modified PA1-STK cells home to tumor sites in patients with malignant pleural mesothelioma. Ann Thorac Surg 2000, 70(2):407-411.
71. Mukherjee S., Haenel T., Himbeck R., et al. Replication-restricted vaccinia as a cytokine gene therapy vector in cancer: persistent transgene expression despite antibody generation. Cancer Gene Ther 2000, 7(5):663-670.
72. Pitako J., Squiban P., Acres B. A randomized phase II single center study of gene transfer-based non-specific immunotherapy of malignant mesothelioma (MM) by intratumoral injections of an interleukin-2 producing vero cells. Proc Am Soc Clin Oncol 2003, 22. [abstract: 920].
73. Tan Y., Xu M., Wang W., et al. IL-2 gene therapy of advanced lung cancer patients. Anticancer Res 1996, 16(4A):1993-1998.
74. Odaka M., Sterman D.H., Wiewrodt R., et al. Eradication of intraperitoneal and distant tumor by adenovirus-mediated interferon-beta gene therapy is attributable to induction of systemic immunity. Cancer Res 2001, 61(16):6201-6212.
75. Odaka M., Wiewrodt R., DeLong P., et al. Analysis of the immunologic response generated by ad.IFN-beta during successful intraperitoneal tumor gene therapy. Mol Ther 2002, 6(2):210-218.
76. Sterman D.H., Gillespie C.T., Carroll R.G., et al. Interferon beta adenoviral gene therapy in a patient with ovarian cancer. Nat Clin Pract Oncol 2006, 3(11):633-639.
77. Sterman D.H., Recio A., Carroll R.G., et al. A phase I clinical trial of single-dose intrapleural IFN-beta gene transfer for malignant pleural mesothelioma and metastatic pleural effusions: high rate of antitumor immune responses. Clin Cancer Res 2007, 13(15 Pt 1):4456-4466.
78. Sterman D.H., Recio A., Haas A.R., et al. A phase I trial of repeated intrapleural adenoviral-mediated interferon-beta gene transfer for mesothelioma and metastatic pleural effusions. Mol Ther 2010, 18(4):852-860.
79. Lambright E.S., Force S.D., Lanuti M.E., et al. Efficacy of repeated adenoviral suicide gene therapy in a localized murine tumor model. Ann Thorac Surg 2000, 70(6):1865-1870. [discussion: 1870-1].
80. Sterman D.H., Haas A.R., Moon E., et al. A phase I trial of intrapleural adenoviral-mediated interferon-alpha2b gene transfer for malignant pleural mesothelioma. Am J Respir Crit Care Med 2011, 10.1164/rccm.201103-0554CR.
81. Kruklitis R.J., Singhal S., Delong P., et al. Immuno-gene therapy with interferon-beta before surgical debulking delays recurrence and improves survival in a murine model of malignant mesothelioma. J Thorac Cardiovasc Surg 2004, 127(1):123-130.
82. Dong M., Li X., Hong L.J., et al. Advanced malignant pleural or peritoneal effusion in patients treated with recombinant adenovirus p53 injection plus cisplatin. J Int Med Res 2008, 36(6):1273-1278.
83. Zhao W.Z., Wang J.K., Li W., et al. Ai Zheng 2009, 28(12):1324-1327. [in Chinese].
84. Kim R., Emi M., Tanabe K., et al. Tumor-driven evolution of immunosuppressive networks during malignant progression. Cancer Res 2006, 66(11):5527-5536.
85. June C.H. Adoptive T cell therapy for cancer in the clinic. J Clin Invest 2007, 117(6):1466-1476.
86. Carpenito C., Milone M.C., Hassan R., et al. Control of large, established tumor xenografts with genetically retargeted human T cells containing CD28 and CD137 domains. Proc Natl Acad Sci U S A 2009, 106(9):3360-3365.