Non-viral delivery of ribozymes for cancer gene therapy

Expert Opinion on Biological Therapy

Volumn 4, Issue 11, 2004, Pages 1749-1755

  Kashani Sabet, Mohammed ^a,b  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Metastasis; Molecular therapeutics; RNA catalysis; Targeted therapy

Indexed keywords

ANGIOZYME; BEVACIZUMAB; EPIDERMAL GROWTH FACTOR RECEPTOR 2; HERZYME; RIBOZYME; TRASTUZUMAB; UNCLASSIFIED DRUG; VASCULOTROPIN RECEPTOR 1; VASCULOTROPIN RECEPTOR 2;

ANTINEOPLASTIC ACTIVITY; BREAST METASTASIS; CANCER THERAPY; COLORECTAL CANCER; DRUG ABSORPTION; DRUG BLOOD LEVEL; DRUG ELIMINATION; DRUG HALF LIFE; DRUG MECHANISM; DRUG RECEPTOR BINDING; HUMAN; LUNG METASTASIS; METASTASIS; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; REVIEW; RNA CLEAVAGE; SIDE EFFECT;

ANIMALS; CLINICAL TRIALS, PHASE II; DRUG CARRIERS; EPSTEIN-BARR VIRUS NUCLEAR ANTIGENS; GENE EXPRESSION REGULATION, NEOPLASTIC; GENE THERAPY; GENETIC VECTORS; HUMANS; MELANOMA, EXPERIMENTAL; MICE; MICE, INBRED C57BL; NEOPLASM CIRCULATING CELLS; NEOPLASM METASTASIS; NEOPLASMS; NF-KAPPA B; PLASMIDS; RNA, CATALYTIC; VASCULAR ENDOTHELIAL GROWTH FACTOR RECEPTOR-1; XENOGRAFT MODEL ANTITUMOR ASSAYS;

ARENA;

EID: 8744318238     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.4.11.1749     Document Type: Review

References (51)

1. GUERRIER-TAKADA C, CARDINER K, MARCH T et al.: The RNA moiety of ribonuclease P is the catalytic subunit of the enzyme. Cell (1983) 35:849-857.
2. ZAUG AJ, CECH T: The intervening sequence RNA of Tetrahymena is an enzyme. Science (1986) 231:470-475.
3. BAGHERI S, KASHANI-SABET M: Ribozymes in the age of molecular therapeutics. Curr. Mol. Med. (2004) 4:489-506.
4. KASHANI-SABET M, SCANLON KJ: Application of ribozymes to cancer gene therapy. Cancer Gene Ther. (1995) 2:213-225.
5. PANTEL K, BRAKENHOFF RH: Dissecting the metastatic cascade. Nat. Rev. Cancer (2004) 4:448-456.
6. CHAMBERS AF, GROOM AC, MACDONALD IC: Dissemination and growth of cancer cells in metastatic sites. Nat. Rev. Cancer (2002) 2:563-572.
7. USMAN N, BLATT LM: Nuclease-resistant synthetic ribozymes: developing a new class of therapeutics. J. Clin. Invest. (2000) 106:1197-1202.
8. FERRARA N, HILLAN KJ, GERBER HP, NOVOTNY W: Discovery and development of bevacizumab, an anti-VEGF antibody for treating cancer. Nat. Rev. Drug Discov. (2004) 3:391-400.
9. KIM KJ, LI B, WINER J et al.: Inhibition of vascular endothelial growth fector-induced angiogenesis suppresses tumour growth in vivo. Nature (1993) 362:841-844.
10. MILLAUER B, SHAWVER LK, PLATE KH et al.: Glioblastoma growth inhibited in vivo by a dominant-negative Flk-1 mutant. Nature (1994) 367:576-579.
11. HURWITZ H, FEHRENBACHER L, NOVOTNY W et al.: Bevacizumab plus irinotecan, fluorouracil, and leucovorin for metastatic colorectal cancer. N. Engl. J. Med. (2004) 350:2335-2342.
12. WINCOTT F, DIRENZO A, SHAFFER C et al.: Synthesis, deprotection, analysis and purification of RNA and ribozymes. Nucleic Acids Res. (1995) 23:2677-2684.
13. SANDBERG JA, BOUHANA KS, GALLEGOS AM et al.: Pharmacokinetics of an antiangiogenic ribozyme (ANGIOZYME) in the mouse. Antisense Nucleic Acid Drug Dev. (1999) 9:271-277.
14. SANDBERG JA, SPROUL CD, BLANCHARD KS et al.: Acute toxicology and pharmacokinetic assessment of a ribozyme (ANGIOZYME) targeting vascular endothelial growth factor receptor mRNA in the cynomolgus monkey. Antisense Nucleic Acid Drug Dev. (2000) 10:153-162.
15. PAVCO PA, BOUHANA KS, GALLEGOS AM et al.: Antitumor and antimetastatic activity of ribozymes targeting the messenger RNA of vascular endothelial growth factor receptors. Clin. Cancer Res. (2000) 6:2094-2103. This study demonstrated antitumour activity for Angiozyme in preclinical models of tumour metastasis.
16. SANDBERG JA, PARKER VP, BLANCHARD KS et al.: Pharmacokinetics and tolerability of an antiangiogenic ribozyme (ANGIOZYME) in healthy volunteers. J. Clin. Pharmacol. (2000) 40:1462-1469.
17. VENOOK A, HURWITZ H, CUNNINGHAM C et al.: Relationship of clinical outcome in metastatic colorectal carcinoma to levels of soluble VEGFR-1: Results of a Phase II trial of a ribozyme targeting the pre-mRNA of VEGFR-1 (angiozyme), in combination with chemotherapy. Proceedings of the 39th Annual Meeting of the American Society of Clinical Oncology. Chicago, USA (31 May - 3 June 2003) Abstract 1025. This study evaluated the biological and potential clinical efficacy of Angiozyme in a clinical trial of colorectal carcinoma patients.
18. SLAMON DJ, GODOLPHIN W, JONES LA et al.: Studies of the HER-2/neu proto-oncogene in human breast and ovarian cancer. Science (1989) 244:707-712.
19. MUSS HB, THOR AD, BERRY DA et al.: c-erbB-2 expression and response to adjuvant therapy in women with node-positive early breast cancer. N. Engl. J. Med. (1994) 330:1260-1266.
20. SLAMON DJ, LEYLAND-JONES B, SHAK S et al.: Use of chemotherapy plus a monoclonal antibody against HER2 for metastatic breast cancer that overexpresses HER2. N. Engl. J. Med. (2001) 344:783-792.
21. SUZUKI T, ANDEREGG B, OHKAWA T et al.: Adenovirus-mediated ribozyme targeting of HER-2/neu inhibits in vivo growth of human breast cancer cells. Gene Ther. (2000) 7:241-248.
22. JUHL H, DOWNING SG, WELLSTEIN A, CZUBAYKO F: HER-2/ neu is rate-limiting for ovarian cancer growth. Conditional depletion of HER-2/ neu by ribozyme targeting. J. Biol. Chem. (1997) 272:29482-29486.
23. WIECHEN K, ZIMMER C, DIETEL M: Selection of a high activity c-erbB-2 ribozyme using a fusion gene of c-erbB-2 and the enhanced green fluorescent protein. Cancer Gene Ther. (1998) 5:45-51.
24. GELMON KA, CHIA S, D'ALOISIO et al.: A Phase I study of HERZYME™, an anti-HER2 ribozyme, in patients with HER2 overexpressing cancers. Proceedings of the 94th Annual Meeting of the American Association for Cancer Research. Toronto, Canada (11 -14 Jury 2003) Abstract 6443.
25. AIGNER A, FISCHER D, MERDAN T, BRUS C, KISSEL T, CZUBAYKO F: Delivery of unmodified bioactive ribozymes by an RNA-stabilizing polyethylenimine (LMW-PEI) efficiently down-regulates gene expression. Gene Ther. (2002) 24:1700-1707. This study used a PEI-based approach for ribozyme delivery to tumours.
26. SLEPUSHKIN V, SIMOES S, DE LIMA MC, DUZGUNES N: Sterically stabilized pH-sensitive liposomes. Methods Enzymol. (2004) 387:134-147.
27. SANDRIN V, RUSSELL SJ, COSSET FL: Targeting retroviral and lentiviral vectors. Curr. Top. Microbiol. Immunol. (2003) 281:137-178.
28. IMPERIALE MJ, KOCHANEK S: Adenovirus vectors: biology, design, and production. Curr. Top. Microbiol. Immunol. (2004) 273:335-357.
29. YANG Y, NUNES FA, BERENCSI K, FURTH E, GONCZOL E, WILSON JM: Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc. Natl. Acad. Sci. USA (1994) 91:4407-4411.
30. WILSON JM: Adenoviruses as gene-delivery vehicles. N. Engl. J. Med. (1996) 334:1185-1187.
31. TU G, KIRCHMAIER AL, LIGGITT D et al.: Non-replicating Epstein-Barr virus-based plasmids extend gene expression and can improve gene therapy in vivo. J. Biol. Chem. (2000) 275:30408-30416. This study identified a novel EBV-based delivery approach for systemic gene delivery.
32. NOSRATI M, LI S, BAGERI S et al.: Anti-tumor activity of systemically delivered ribozymes targeting murine telomerase RNA. Clin. Cancer Res. (2004) 10:4983-4990. This study identified a role for telomerase in the metasatic progression of melanoma in a murine model.
33. GILMORE TD: The Rel/NF-kappa B/I kappa B signal transduction pathway and cancer. Cancer Treat. Res. (2003) 115:241-265.
34. SUN SC, XIAO G: Deregulation of NF-kappaB and its upstream kinases in cancer. Cancer Metastasis Rev. (2003) 22:404-422.
35. GRETEN FR, KARIN M: The IKK/NF-kappaB activation pathway-a target for prevention and treatment of cancer. Cancer Lett. (2004) 206:193-199.
36. KASHANI-SABET M, LIU Y, FONG S et al.: Identification of gene function and functional pathways by systemic plasmid-based ribozyme targeting in adult mice. Proc. Natl. Acad. Sci. USA (2002) 99:3878-3883. This study identified a novel role for NF-κB in the metastatic progression of melanoma using a systemic, plasmid-based delivery approach.
37. LIU Y, MOUNKES LC, LIGGITT D et al.: Factors influencing the efficiency of cationic liposome-mediated intravenous gene delivery. Nat. Biotechnol. (1997) 15:67-73.
38. KASHANI-SABET M, SHAIKH L, SAGEBIEL RW et al.: NF-κB in the vascular progression of melanoma. J. Clin. Oncol. (2004) 22:617-623.
39. PIALI L, HAMMEL P, UHEREK C et al.: CD31/PECAM-1 is a ligand for alpha v beta 3 integral involved in adhesion of leukocytes to endothelium. J. Cell Biol. (1995) 130:451-460.
40. RITCHIE CK, GIORDANO A, KHALILI K: Integrin involvement in glioblastoma multiforme: possible regulation by NF-kappa B. J. Cell. Physiol. (2000) 184:214-221.
41. BOTELLA LM, PUIG-KROGER A, ALMENDRO N et al.: Identification of a functional NF-kappa B site in the platelet endothelial cell adhesion molecule-1 promoter. J. Immunol. (2002) 164:1372-1378.
42. AKIYAMA M, HIDESHIMA T, HAYASHI T et al.: Nuclear factor-kappaB p65 mediates tumor necrosis factor alpha-induced nuclear translocation of telomerase reverse transcriptase protein. Cancer Res. (2003) 63:18-21.
43. YIN L, HUBBARD AK, GIARDINA C: NF-kappa B regulates transcription of the mouse telomerase catalytic subunit. J. Biol. Chem. (2000) 275:36671-36675.
44. SUYAMA E, WADHWAS R, KAMALJIT K et al.: Identification of metastasis-related genes in a mouse model using a library of randomized ribozymes. J. Biol. Chem. (2004) 279:38083-38086. This study used a ribozyme library to identify novel genes involved in tumour metastasis.
45. KASHANI-SABET M, FUNATO T, FLORENES VA, FODSTAD O et al.: Suppression of the neoplastic phenotype in vivo by an anti-ras ribozyme. Cancer Res. (1994) 54:900-902.
46. MAELANDSMO GM, HOVIG E, SKREDE M et al.: Reversal of the in vivo metastatic phenotype of human tumor cells by an anti-CAPL (mts1) ribozyme. Cancer Res. (1996) 56:5490-5498.
47. CZUBAYKO F, SCHULTE AM, BERCHEM GJ, WELLSTEIN A: Melanoma angiogenesis and metastasis modulated by ribozyme targeting of the secreted growth factor pleiotrophin. Proc. NatL Acad. Sei. USA (1996) 93:14753-14758. This study used ribozymes to identify a novel role for pleiotrophin in the angiogenic and metastatic phenotype of melanoma.
48. HUA J, MUSCHEL RJ: Inhibition of matrix metalloproteinase 9 expression by a ribozyme blocks metastasis in a rat sarcoma model system. Cancer Res. (1996) 56:5279-5284.
49. YAMAMOTO H, IRE A, FUKUSHIMA Y et al.: Abrogation of lung metastasis of human fibrosarcoma cells by ribozyme-mediated suppression of integral alpha 6 subunit expression. Int. J. Cancer (1996) 65:519-524.
50. KIM SJ, JOHNSON M, KOTERBA K et al.: Reduced c-Met expression by an adenovirus expressing a c-Met ribozyme inhibits tumorigenic growth and lymph node metastases of PC3-LN4 prostate tumor cells in an orthotopic nude mouse model. Clin. Cancer Res. (2003) 9:5161-5170.
51. TOKUNAGA T, ABE Y, TSUCHIDA T et al:. Ribozyme mediated cleavage of cell-associated isoform of vascular endothelial growth factor inhibits liver metastasis of a pancreatic cancer cell line. Int. J. Oncol. (2002) 21:1027-1032.