Antisense oligonucleotides in cancer

Current Opinion in Oncology

Volumn 26, Issue 6, 2014, Pages 584-589

  Castanotto, Daniela ^a   Stein, Cy A ^a  

^a CITY OF HOPE NATIONAL MEDICAL CENTER   (United States)

Author keywords

Antisense; Endocytosis; Oligonucleotide; Phase III; Phosphorothioate

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; PHOSPHOROTHIOIC ACID;

DRUG DELIVERY SYSTEM; DRUG DESIGN; DRUG EFFICACY; DRUG METABOLISM; HUMAN; HYPOXIA; NEOPLASM; PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; QUANTUM MECHANICS; REVIEW; TREATMENT FAILURE; ANIMAL; GENE THERAPY; NEOPLASMS; PROCEDURES;

ANIMALS; GENETIC THERAPY; HUMANS; NEOPLASMS; OLIGONUCLEOTIDES, ANTISENSE;

EID: 84927622814     PISSN: 10408746     EISSN: 1531703X     Source Type: Journal    
DOI: 10.1097/CCO.0000000000000127     Document Type: Review

References (36)

1. Paterson B, Roberts B, Kuff E. Structural gene identification and mapping by DNA-mRNA hybrid arrested cell free translation. Proc Natl Acad Sci U S A 1977; 74:4370-4374.
2. Zamecnik P, Stephenson ML. Inhibiton of Rous sarcoma virus replication and transformation by aspecifric oligodeoxynucleotide. Proc Natl Acad Sci U S A 1978; 75:280-284.
3. Piascik P. Fomiversen sodium approved to treat CMV retinitis. J Am Pharm Assoc (Wash) 1999; 39:84-85.
4. Hovingh K, Besseling J, Kastelein J. Efficacy and safety of mipomersen sodium (Kynamro). Expert Opin Drug Saf 2013; 12:568-579.
5. Crooke S, Geary R. Clinical pharmacological properties of mipomersen (Kynamro), a second generation antisense inhibitor of apolipoprotein B. Br J Clin Pharmacol 2013; 76:269-276.
6. Gebhard C, Huard G, Kritikou E, Tardif J. Apolipoprotein B antisense inhibition: update on mipomersen. Curr Pharm Des 2013; 19:3132-3142.
7. Saad F, Hotte S, North S, et al. Randomized phase II trial of Custirsen (OGX-011) in combination with docetaxel or mitoxantrone as second-line therapy in patients with metastatic castrate-resistant prostate cancer progressing after first-line docetaxel: CUOG trial P-06c. Clin Cancer Res 2011; 17:5765-5773.
8. Blumenstein B, Saad F, Hotte S, et al. Reduction in serum clusterin is a potential therapeutic biomarker in patients with castration-resistant prostate cancer treated with custirsen. Cancer Med 2013; 2:468-477.
9. Talbot DC, Ranson M, Davies J, et al. Tumor surviving is downregulated by the antisense oligonucleotide LY2181308: a proof-of-concept, first-in-human dose study. Clin Cancer Res 2010; 16:6150-6158.
10. Erba HP, Sayar H, Juckett M, et al. Safety and pharmacokinetics of the antisense oligonucleotide (ASO) LY2181308 as a single-agent or in combination with idarubicin and cytarabine in patients with refractory or relapsed acute myeloid leukemia (AML). Invest New Drugs 2013; 4:1023-1034.
11. Wiechno P, Somer BG, Mellado B, et al. A randomised phase 2 study combining LY2181308 sodium (survivin antisense oligonucleotide) with first-line docetaxel/prednisone in patients with castration-resistant prostate cancer. Eur Urol 2014; 65:516-520.
12. Agarwala S, Keilholz U, Gilles E, et al. LDH correlation with survival in advanced melanoma from two large, randomized trials (Oblimersen GM301 and EORTC 18951). Eur J Cancer 2009; 45:1807-1814.
13. Bedikian A, Millward M, Pehamberger H, et al. Bcl-2 antisense (oblimersen sodium) plus dacarbazine in patients with advanced melanoma: the Oblimersen Melanoma Study Group. J Clin Oncol 2006; 24:4738-4745.
14. Cheson B. Oblimersen for the treatment of patients with chronic lymphocytic leukemia. Ther Clin Risk Manag 2007; 3:855-870.
15. Bianchini D, Omlin A, Pezaro C, et al. First-in-human phase I study of EZN-4176, a locked nucleic acid antisense oligonucleotide to exon 4 of the androgen receptor (AR) mRNA in patients with castration-resistant prostate cancer. Br J Cancer 2013; 109:2579-2586.
16. Zorzi F, Angelucci E, Sedda S, et al. Smad7 antisense oligonucleotide-based therapy for inflammatory bowel diseases. Dig Liver Dis 2013; 45:552-555.
17. Monteleone C, Fantini M, Onali S, et al. Phase I clinical trial of Smad7 knockdown using antisense oligonucleotide in patients with active Crohn's disease. Mol Ther 2012; 20:870-876.
18. Cursiefen C, Viaud E, Bock F, et al. Aganirsen antisense aligonucleotide eye drops inhibit keratitis-induced corneal neovascularization and reduce need for transplantation: the I-CAN study. Ophthalmology 2014; 121:1683-1692.
19. Stein C, Hansen B, Lai J, et al. Efficient gene silencing by delivery of locked nucleic acid antisense oligonucleotides, unassisted by transfection reagents. Nucleic Acids Res 2010; 38:e3.
20. Borges O, Cordeiro-da-Silva A, Tavares J, et al. Immune response by nasal delivery of hepatitis B surface antigen and codelivery of a CpG ODN in alginate coated chitosan nanoparticles. Eur J Pharm Biopharm 2008; 69:405-416.
21. Trembley J, Unger G, Tobolt D, et al. Systemic administration of antisense oligonucleotides simultaneously targeting CK2alpha and alpha' subunits reduces orthotopic xenograft prostate tumors in mice. Mol Cell Biochem 2011; 356:21-35.
22. Trembley J, Wang G, Unger G, et al. Proteins kinase CK2 in health and disease: CK2: a key player in cancer biology. Cell Mol Life Sci 2009; 66:1858-1867.
23. Stein C, Wu S, Voskresenskiy A, et al. G3139, an anti-Bcl-2 antisense oligomer that binds heparin-binding growth factors and collagen I, alters in-vitro endothelial cell growth and tubular morphogenesis. Clin Cancer Res 2009; 15:2797-2807.
24. Khaled Z, Benimetskaya L, Khan T, et al. Multiple mechanisms may contribute to the cellular antiadhesive effects of phosphorothioate oligodeoxynucleotides. Nucleic Acids Res 1996; 24:737-745.
25. Benimetskaya L, Loike J, Khaled Z, et al. Mac-1 (CD11b/CD18) is a cell surface oligodeoyxnucleotide binding protein. Nat Med 1997; 3:414-420.
26. Lai J, Tan W, Benimetskaya L, et al. A pharmacologic target of G3139 in melanoma cells may be the mitochondrial VDAC. Proc Natl Acad Sci U S A 2006; 103:7494-7499.
27. Sirois C, Jin T, Miller A, et al. RAGE is a nucleic acid receptor that promotes inflammatory responses to DNA. J Exp Med 2013; 210:2447-2463.
28. Juliano R, Ming X, Nakagawa O. Cellular uptake and intracellular trafficking of antisense and siRNA oligonucleotides. Bioconjug Chem 2012; 23:147-157.
29. Ming X, Carver K, Fisher M, et al. The small molecule Retro-1 enhances the pharmacological actions of antisense and splice switching oligonucleotides. Nucleic Acids Res 2013; 41:3673-3687.
30. Hagedorn P, Yakimov V, Ottosen S, et al. Hepatotoxic potential of therapeutic oligonucleotides can be predicted from their sequence and modification pattern. Nucleic Acid Ther 2013; 23:302-310.
31. Seth P, Siwkowski A, Allerson C, et al. Short antisense oligonucleotides with novel 20-40 conformationally restricted nucleoside analogues show improved potency without increased toxicity in animals. J Med Chem 2009; 52:10-13.
32. Koch T, Shim I, Lindow M, et al. Quantum mechanical studies of DNA and LNA. Nucleic Acid Ther 2014; 24:139-148.
33. Juliano R, Alam M, Dixit V, Kang H. Mechanisms and strategies for effective delivery of antisense and siRNA oligonucleotides. Nucleic Acids Res 2008; 36:4158-4171.
34. Leal-Pinto E, Teixeira A, Tran B, et al. Presence of the nucleic acid channel in renal brush-border membranes: allosteric modulation by extracellular calcium. Am J Physiol Renal Physiol 2005; 289:F97-106.
35. Shi F, Gounko N, Wang X, et al. In situ entry of oligonucleotides into brain cells can occur through a nucleic acid channel. Oligonucleotides 2007; 17:122-133.
36. Jain R. Normalization of tumor vasculature: an emerging concept in antiangiogenic therapy. Science 2005; 307:58-62.