Cancer gene therapy: Connecting basic research with clinical inquiry

Journal of Clinical Oncology

Volumn 16, Issue 7, 1998, Pages 2548-2556

  Dranoff, Glenn ^a,b  

^a HARVARD MEDICAL SCHOOL   (United States)

^b DANA FARBER CANCER INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CYTOTOXIC AGENT; FLUCYTOSINE; GANCICLOVIR; THYMIDINE KINASE; TUMOR CELL VACCINE; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ADOPTIVE IMMUNOTHERAPY; ARTICLE; CANCER; CANCER CHEMOTHERAPY; CANCER IMMUNOTHERAPY; CANCER THERAPY; CLINICAL TRIAL; DRUG TARGETING; GENE EXPRESSION SYSTEM; GENE TARGETING; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; HUMAN; NONHUMAN; PRIORITY JOURNAL; RETROVIRUS;

EID: 0031833036     PISSN: 0732183X     EISSN: None     Source Type: Journal    
DOI: 10.1200/JCO.1998.16.7.2548     Document Type: Article

References (85)

1. Varmus H: Retroviruses. Science 240:1427-1435, 1988
2. Bishop JM: Cellular oncogenes and retroviruses. Ann Rev Biochem 52:301-354, 1983
3. Mulligan RC: The basic science of gene therapy. Science 260:926-932, 1993
4. Miller AD: Human gene therapy comes of age. Nature 357:455-460, 1992
5. Danos O, Mulligan RC: Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges. Proc Natl Acad Sci U S A 85:6460-6464, 1988
6. Donahue RE, Kessler SW, Bodine D, et al: Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer. J Exp Med 176:1125-1135, 1992
7. Naldini L, Blomer U, Gallay P, et al: In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272:263-266, 1996
8. Burns JC, Friedman T, Driever W, et al: Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: Concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells. Proc Natl Acad Sci U S A 90:8033-8037, 1993
9. Ory DS, Neugeboren BA, Mulligan RC: A stable human-derived packaging cell line for production of high titer retrovirus/vesicular stomatitis virus G pseudotypes. Proc Natl Acad Sci U S A 93:11400-11406, 1996
10. Fass D, Davey RA, Hamson CA, et al: Structure of a murine leukemia virus receptor-binding glycoprotein at 2.0 A resolution. Science 277:1662-1666, 1997
11. Shenk T: Adenoviridae: The viruses and their replication, in Fields BN, Knipe DM, Howley PM (eds): Fields Virology (ed 3). Philadelphia, PA, Lippincott-Raven, 1996, pp 2111-2148
12. Berkner KL: Development of adenovirus vectors for the expression of heterologous genes. Biotechniques 6:616-629, 1988
13. Bett A, Haddara W, Prevec L, et al: An efficient and flexible system for construction of adenovirus vectors with insertions or deletions in early regions 1 and 3. Proc Natl Acad Sci U S A 91:8802-8806, 1994
14. Jones N, Shenk T: Isolation of adenovirus type 5 host range deletion mutants defective for transformation of rat embryo cells. Cell 17:683-689, 1979
15. Yang Y, Li Q, Ertle H, et al: Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J Virol 69:2004-2015, 1995
16. Kochanek S, Clemens PR, Mitani K, et al: A new adenoviral vector-replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase. Proc Natl Acad Sci U S A 93:5731-5736, 1996
17. Hardy S, Kitamura M, Harris-Stansil T, et al: Construction of adenovirus vectors through cre-lox recombination. J Virol 71:1842-1849, 1997
18. Parks RJ, Chen L, Anton M, et al: A helper-dependent adenovirus vector system: Removal of helper virus by Cre-mediated excision of the viral packaging signal. Proc Natl Acad Sci U S A 93:13565-13570, 1996
19. Schiedner G, Morral N, Parks R, et al: Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nature Genet 18:180-183, 1998
20. Berns KI: Parvoviridae: The viruses and their replication, in Fields BN, Knipe DM, Howley PM (eds): Fields Virology. Philadelphia, PA, Lippincott-Raven, 1996, pp 2173-2197
21. Linden RM, Ward P, Giraud C, et al: Site-specific integration by adeno-associated virus. Proc Natl Acad Sci U S A 93:11288-11294, 1996
22. Marconi P, Krisky D, Oligin T, et al: Replication-defective herpes simplex virus vectors for gene transfer in vivo. Proc Natl Acad Sci U S A 93:11319-11320, 1996
23. Roizman B: The function of herpes simplex virus genes: A primer for genetic engineering of novel vectors. Proc Natl Acad Sci U S A 93:11307-11312, 1996
24. Martuza RL, Malick A, Markert JM, et al: Experimental therapy of human glioma by means of a genetically engineered virus mutant. Science 252:854-856, 1991
25. Frenkel N, Singer O, Kwong AD: Minireview: The herpes simplex virus amplicon - A versatile defective virus vector. Gene Ther 1:S40-S60, 1994
26. Moss B: Genetically engineered poxviruses for recombinant gene expression, vaccination, and safety. Proc Natl Acad Sci USA 93:11341-11348, 1996
27. Andino R, Silvera D, Suggett S, et al: Engineering poliovirus as a vaccine vector for the expression of diverse antigens. Science 265:1448-1451, 1994
28. Frolov I, Hoffman TA, Pragai BM, et al: Alphavirus-based expression vectors: Strategies and applications. Proc Natl Acad Sci U S A 93:11371-11377, 1996
29. Boyce F, Bucher N: Baculovirus-mediated gene transfer into mammalian cells. Proc Natl Acad Sci U S A 93:2348-2352, 1996
30. Wolff JA, Malone RW, Williams P, et al: Direct gene transfer into mouse muscle in vivo. Science 247:1465-1468, 1990
31. Doe B, Selby M, Barnett S, et al: Induction of cytotoxic T lymphocytes by intramuscular immunization with plasmid DNA is facilitated by bone marrow-derived cells. Proc Natl Acad Sci U S A 93:8578-8583, 1996
32. Ulmer J, Donnelly J, Parker S, et al: Heterologous protection against influenza by injection of DNA encoding a viral protein. Science 259:1745-1749, 1993
33. Yang NS, Sun WH, McCabe D: Developing particle-mediated gene-transfer technology for research into gene therapy of cancer. Mol Med Today 2:476-481, 1996
34. Connor J, Yatvin MB, Huang L: pH-sensitive liposomes: Acid-induced liposome fusion. Proc Natl Acad Sci U S A 81:1715-1718, 1984
35. Wu GY, Wilson JM, Shalaby F, et al: Receptor-mediated gene delivery in vivo. Partial correction of genetic analbuminemia in Nagase rats. J Biol Chem 266:14338-14342, 1991
36. Wagner E, Zatloukal K, Cotten M, et al: Coupling of adenovirus to transferrin-polylysine/DNA complexes greatly enhances receptor-mediated gene delivery and expression of transfected genes. Proc Natl Acad Sci U S A 89:6099-6103, 1992
37. Perales J, Ferkol T, Beegen H, et al: Gene transfer in vivo: Sustained expression and regulation of genes introduced into the liver by receptor-targeted uptake. Proc Natl Acad Sci U S A 91:4086-4090, 1994
38. Elion G, Furman P, Fyfe J, et al: Selectivity of action of an antiherpetic agent, 9-(2-dyroxyethoxymethyl) guanine. Proc Natl Acad Sci U S A 74:5716-5720, 1977
39. Ezzedine Z, Martuza R, Platika O, et al: Selective killing of glioma cells in culture and in vivo by retrovirus transfer of the herpes simplex virus thymidine kinase gene. New Biol 3:608-614, 1991
40. Culver KW, Ram Z, Wallbridge S, et al: In vivo gene transfer with retroviral vector producer cells for treatment of experimental brain tumors. Science 256:1550-1552, 1992
41. Freeman S, Abboud C, Whartenby K, et al: The "bystander effect": Tumor regression when a fraction of the tumor mass is genetically modified. Cancer Res 53:5274-5283, 1993
42. Caruso M, Panis Y, Gagandeep S, et al: Regression of established macroscopic liver metastases after in situ transduction of a suicide gene. Proc Natl Acad Sci U S A 90:7024-7028, 1993
43. Mesnil M, Piccoli C, Tiraby G, et al: Bystander killing of cancer cells by herpes simplex virus thymidine kinase gene is mediated by connexins. Proc Natl Acad Sci U S A 93:1831-1835, 1996
44. Chen S-H, Shine HD, Goodman JC, et al: Gene therapy for brain tumors: Regression of experimental gliomas by adenovirus-mediated gene transfer in vivo. Proc Natl Acad Sci U S A 91:3054-3057, 1994
45. Parr MJ, Manome Y, Tanaka T, et al: Tumor-specific transgene expression in vivo mediated by an E2F-responsive adenoviral vector. Nature Med 3:1145-1149, 1997
46. Trinh Q, Austin E, Murray D, et al: Enzyme/prodrug gene therapy: Comparison of cytosine deaminase/5-fluorocytosine versus thymidine kinase/ganciclovir enzyme/prodrug systems in a human colorectal carcinoma cell line. Cancer Res 55:4808-4812, 1995
47. Wei M, Tamiya T, Chase M, et al: Experimental tumor therapy in mice using the cyclophosphamide-activating cytochrome P450 2B1 gene. Hum Gene Ther 5:969-978, 1994
48. Lowe SW, Ruley HE, Jacks T, et al: p53-dependent apoptosis modulates the cytotoxicity of anticancer agents. Cell 74:957-967, 1993
49. Roth JA, Nguyen D, Lawrence DD, et al: Retrovirus-mediated wild-type p53 gene transfer to tumors of patients with lung cancer. Nature Med 2:985-991, 1996
50. Kashani-Sabet M, Funato T, Florenes V, et al: Suppression of a neoplastic phenotype in vivo by an anti-ras ribozyme. Cancer Res 54:900-902, 1994
51. Deshane J, Cabrera G, Grim J, et al: Targeted eradication of ovarian cancer mediated by intracellular expression of anti-erbB-2 single-chain antibody. Gyn Oncol 59:8-14, 1995
52. Chen SY, Marasco WA: Novel genetic immunotoxins and intracellular antibodies for cancer therapy. Semin Oncol 23:148-153, 1996
53. s adenovirus selectively induces apoptosis in cancer cells but not in normal bone marrow cells. Proc Natl Acad Sci U S A 92:11024-11028, 1995
54. Chen L, Pulsipher M, Chen D, et al: Selective transgene expression for detection and elimination of contaminating carcinoma cells in hematopoietic stem cell sources. J Clin Invest 98:2539-2548, 1996
55. Bischoff JR, Kirn DH, Williams A, et al: An adenovirus mutant that replicates selectively in p53-deficient human tumor cells. Science 274:373-376, 1996
56. Podda S, Ward M, Himelstein A, et al: Transfer and expression of the human multiple drug resistance gene into live mice. Proc Natl Acad Sci U S A 89:9676-9680, 1992
57. Sorrentino BP, Brandt SJ, Bodine D, et al: Selection of drug-resistant bone marrow cells in vivo after retroviral transfer of human MDR1. Science 257:99-103, 1992
58. Brenner MK, Rill DR, Moen RC, et al: Gene-marking to trace origin of relapse after autologous bone-marrow transplantation. Lancet 341:85-86, 1993
59. Deisseroth AB, Zu Z, Claxton D, et al: Genetic marking shows that Ph+ cells present in autologous transplants of chronic myelogenous leukemia (CML) contribute to relapse after autologous bone marrow transplantation in CML. Blood 83:3068-3076, 1994
60. Rill DR, Santana VM, Roberts WM, et al: Direct demonstration that autologous bone marrow transplantation for solid tumors can return a multiplicity of tumorigenic cells. Blood 84:380-383, 1994
61. Townsend A, Bodmer H: Antigen recognition by class I-restricted T lymphocytes. Ann Rev Immunol 7:601-624, 1989
62. Boon T, van der Bruggen P: Human tumor antigens recognized by T lymphocytes. J Exp Med 183:725-729, 1996
63. Guinan E, Gribben J, Boussiotis V, et al: Pivotal role of the B7:CD28 pathway in transplantation tolerance and tumor immunity. Blood 84:3261-3282, 1994
64. Forni G, Fujiwara H, Martino F, et al: Helper strategy in tumor immunology: Expansion of helper lymphocytes and utilization of helper lymphokines for experimental and clinical immunotherapy. Cancer Metast Rev 7:289-309, 1988
65. Dranoff G, Mulligan RC: Gene transfer as cancer therapy. Adv Immunol 58:417-454, 1995
66. Fearon ER, Pardoll DM, Itaya T, et al: Interleukin-2 production by tumor cells bypasses T helper function in the generation of an antitumor response. Cell 60:397-403, 1990
67. Gansbacher B, Zier K, Daniels B, et al: Interleukin-2 gene transfer into tumor cells abrogates tumorigenicity and induces protective immunity. J Exp Med 172:1217-1224, 1990
68. Tahara H, Zeh HJ, Storkus WJ, et al: Fibroblasts genetically engineered to secrete interleukin-12 can suppress tumor growth and induce antitumor immunity to a murine melanoma in vivo. Cancer Res 54:182-189, 1994
69. Putzer BM, Hitt M, Mueller WJ, et al: Interleukin-12 and B7-1 costimulatory molecule expressed by an adenoviral vector act synergistically to facilitate tumor regression. Proc Natl Acad Sci U S A 94:10889-10894, 1997
70. Dranoff G, Jaffee E, Lazenby A, et al: Vaccination with irradiated tumor cells engineered to secrete murine granulocyte-macrophage colony-stimulating factor stimulates potent, specific, and long-lasting anti-tumor immunity. Proc Natl Acad Sci U S A 90:3539-3543, 1993
71. Belli F, Arienti F, Sule-Suso J, et al: Active immunization of metastatic melanoma patients with interleukin-2 - transduced allogeneic melanoma cells: Evaluation of efficacy and tolerability. Cancer Immunol Immunother 44:197-203, 1997
72. Abdel-Wahab Z, Weltz C, Hester D, et al: A phase I clinical trial of immunotherapy with interferon-γ gene-modified autologous melanoma cells. Cancer 80:401-412, 1997
73. Ellem KAO, O'Rourke MGE, Johnson GR, et al: A case report: Immune responses and clinical course of the first human use of granulocyte-macrophage colony-stimulating-factor - transduced autologous melanoma cells for immunotherapy. Cancer Immunol Immunother 44:10-20, 1997
74. Simons JW, Jaffee EM, Weber CE, et al: Bioactivity of autologous irradiated renal cell carcinoma vaccines generated by ex vivo granulocyte-macrophage colony-stimulating factor gene transfer. Cancer Res 57:1537-1546, 1997
75. Hui KM, Ang PT, Huang L, et al: Phase I study of immunotherapy of cutaneous metastases of human carcinoma using allogeneic and xenogeneic MHC DNA-liposome complexes. Gene Ther 4:783-790, 1997
76. Nabel G, Gordon D, Bishop D, et al: Immune response in human melanoma after transfer of an allogeneic class I major histocompatibility complex gene with DNA-liposome complexes. Proc Natl Acad Sci U S A 93:15388-15393, 1996
77. Caux C, Dezutter-Dambuyant C, Schmitt D, et al: GM-CSF and TNF-α cooperate in the generation of dendritic Langerhans cells. Nature 360:258-261, 1992
78. Reeves ME, Royalo RE, Lam JS, et al: Retroviral transduction of human dendritic cells with a tumor-associated antigen gene. Cancer Res 56:5672-5677, 1996
79. Boczkowski D, Nair SK, Snyder D, et al: Dendritic cells pulsed with RNA are potent antigen-presenting cells in vitro and in vivo. J Exp Med 184:465-472, 1996
80. Wan YH, Bramson J, Carter R, et al: Dendritic cells transduced with an adenoviral vector encoding a model tumor-associated antigen for tumor vaccination. Hum Gene Ther 8:1355-1363, 1997
81. Tsang K, Zarema S, Nieroda C, et al: Generation of human carcinoembryonic antigen epitopes from patients immunized with recombinant vaccinia-CEA vaccine. J Natl Cancer Instit 87:982-990, 1995
82. Rosenberg SA, Aebersold P, Cornetta K, et al: Gene transfer into humans - Immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 323:570-578, 1990
83. Rooney C, Smith C, Ng C, et al: Use of gene-modified virus-specific T lymphocytes to control Epstein-Barr-virus - related lymphoproliferation. Lancet 345:9-13, 1995
84. Hege KM, Cooke KS, Finer MH, et al: Systemic T cell-independent tumor immunity after transplantation of universal receptor-modified bone marrow into SCID mice. J Exp Med 184:2261-2269, 1996
85. Bonini C, Ferrari G, Verzeletti S, et al: HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia. Science 276:1719-1724, 1997