Considerations for the Characterization and Interpretation of Results Related to Alternative Complement Activation in Monkeys Associated with Oligonucleotide-Based Therapeutics

Nucleic Acid Therapeutics

Volumn 26, Issue 4, 2016, Pages 210-215

  Henry, Scott P ^a   Seguin, Rosanne ^b   Cavagnaro, Joy ^c   Berman, Cindy ^d   Tepper, Jeff ^e   Kornbrust, Douglas ^f  

^a ISIS PHARMACEUTICALS   (United States)

^b MCGILL UNIVERSITY   (Canada)

^c Access BIO   (United States)

^d Berman Consulting   (United States)

^e Tepper Nonclinical Consulting   (United States)

^f Preclinsight   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DOUBLE STRANDED RNA; OLIGONUCLEOTIDE PHOSPHOROTHIOATE;

CHEMICAL MODIFICATION; COMPLEMENT ACTIVATION; COMPLEMENT ALTERNATIVE PATHWAY; MACACA FASCICULARIS; NONHUMAN; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; REVIEW; RHESUS MONKEY; SPECIES DIFFERENCE; STRUCTURE ACTIVITY RELATION; ANIMAL; DRUG EFFECTS; HAPLORHINI; HUMAN;

ANIMALS; COMPLEMENT PATHWAY, ALTERNATIVE; HAPLORHINI; HUMANS; PHOSPHOROTHIOATE OLIGONUCLEOTIDES; SPECIES SPECIFICITY;

EID: 84981311948     PISSN: 21593337     EISSN: 21593345     Source Type: Journal    
DOI: 10.1089/nat.2015.0593     Document Type: Review

References (20)

1. Galbraith WM, WC Hobson, PC Giclas, PJ Schechter and S Agrawal. (1994). Complement activation and hemodynamic changes following intravenous administration of phosphorothioate oligonucleotides in the monkey. Antisense Res Dev 4:201-206.
2. Henry SP, J Leeds, PC Giclas, NA Gillett, JP Pribble, DJ Kornbrust and AA Levin. (1996). The toxicity of ISIS 3521, a phosphorothioate oligonucleotide, following intravenous (iv) and subcutaneous (sc) administration in cynomolgus monkeys. In: Society of Toxicology Annual Meeting, Anaheim, CA, p 112.
3. Henry SP, T-W Kim, K Kramer-Stickland, TA Zanardi, RA Fey and AA Levin. (2007). Toxicologic properties of 2′-Omethoxyethyl chimeric antisense inhibitors in animals and man. In: Antisense Drug Technology, Principles, Strategies and Applications. ST Crooke, ed. Taylor & Francis Group, Boca Raton, FL, pp 327-364.
4. Henry SP, PC Giclas, J Leeds, M Pangburn, C Auletta, AA Levin and DJ Kornbrust. (1997). Activation of the alternative pathway of complement by a phosphorothioate oligonucleotide: potential mechanism of action. J Pharmacol Exp Ther 281:810-816.
5. Henry SP, D Monteith, DJ Kornbrust and AA Levin. (1997). Effects of Intravenous infusion of phosphorothioate oligonucleotides on coagulation, complement activation and hemodynamics. Nucleosides Nucleotides 16:1673-1676.
6. Henry SP, D Monteith, CF Bennett and AA Levin. (1997). Toxicological and pharmacokinetic properties of chemically modified antisense oligonucleotide inhibitors of PKCalpha and C-raf kinase. Anticancer Drug Des 12:409-420.
7. Levin AA, SP Henry, D Monteith and M Templin. (2001). Toxicity of antisense oligonucleotides. In: Antisense Drug Technology. ST Crooke, ed. Marcel Dekker, Inc., New York, pp 201-267.
8. Black LE, JG Farrelly, JA Cavagnaro, C-H Ahn, JJ De- George, AS Taylor, AF DeFelice and A Jordan. (1994). Regulatory considerations of oligonucleotide drugs: updated recommendations for pharmacology and toxicology studies. Antisense Res Dev 4:299-301.
9. Sazani P, KP Van Ness, DL Weller, DW Poage, K Palyada and SB Shrewsbury. (2011). Repeat-dose toxicology evaluation in cynomolgus monkeys of AVI-4658, a phosphorodiamidate morpholino oligomer (PMO) drug for the treatment of duchenne muscular dystrophy. Int J Toxicol 30:313-321.
10. Cunningham CC, JT Holmlund, RS Geary, JT Kwoh, A Dorr, JF Johnston, B Monia and J Nemunaitis. (2001). A phase I trial of H-ras antisense oligonucleotide ISIS 2503 administered as a continuous intravenous infusion in patients with advanced carcinoma. Cancer 92:1265-1271.
11. Cunningham CC, JT Holmlund, JH Schiller, RS Geary, TJ Kwoh, A Dorr and JA Nemunaitis. (2000). 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 6:1626-1631.
12. Glover JM, JM Leeds, TG Mant, D Amin, DL Kisner, JE Zuckerman, RS Geary, AA Levin and WR Shanahan, Jr. (1997). Phase I safety and pharmacokinetic profile of an intercellular adhesion molecule-1 antisense oligodeoxynucleotide (ISIS 2302). J Pharmacol Exp Ther 282:1173-1180.
13. Kastelein JJ, MK Wedel, BF Baker, J Su, JD Bradley, RZ Yu, E Chuang, MJ Graham and RM Crooke. (2006). Potent reduction of apolipoprotein B and low-density lipoprotein cholesterol by short-term administration of an antisense inhibitor of apolipoprotein B. Circulation 114:1729-1735.
14. Sewell LK, RS Geary, BF Baker, JM Glover, TGK Mant, RZ Yu, JA Tami and AF Dorr. (2002). Phase I trial of ISIS 104838, a 2′-methoxyethyl modified antisense oligonucleotide targeting tumor necrosis factor-alpha. J Pharmacol Exp Ther 303:1334-1343.
15. Chi KN, E Eisenhauer, L Fazli, EC Jones, SL Goldenberg, J Powers, D Tu and ME Gleave. (2005). A phase I pharmacokinetic and pharmacodynamic study of OGX-011, a 2′-methoxyethyl antisense oligonucleotide to clusterin, in patients with localized prostate cancer. J Natl Cancer Inst 97:1287-1296.
16. McHutchison JG, K Patel, P Pockros, L Nyberg, S Pianko, RZ Yu, FA Dorr and TJ Kwoh. (2006). A phase I trial of an antisense inhibitor of hepatitis C virus (ISIS 14803), administered to chronic hepatitis C patients. J Hepatol 44:88-96.
17. Nemunaitis J, JT Holmlund, M Kraynak, D Richards, J Bruce, N Ognoskie, TJ Kwoh, R Geary, A Dorr, D Von Hoff and SG Eckhardt. (1999). Phase I evaluation of ISIS 3521, an antisense oligodeoxynucleotide to protein kinase C-alpha, in patients with advanced cancer. J Clin Oncol 17:3586-3595.
18. Yuen AR, R Advani, G Fisher, J Halsey, B Lum, RS Geary, TJ Kwoh, A Dorr and BI Sikic. (2000). A phase I/II trial of ISIS 3521, an antisense inhibitor of protein kinase C alpha, combined with carboplatin and paclitaxel in patients with non-small cell lung cancer. In: American Society of Clinical Oncology 36th Annual Meeting, New Orleans, LA, p Abs. 1802.
19. Homeister JW, P Satoh and BR Lucchesi. (1992). Effects of complement activation in the isolated heart. Role of the terminal complement components. Circ Res 71:303-319.
20. Henry SP, G Beattie, G Yeh, A Chappel, PC Giclas, A Mortari, MA Jagels, DJ Kornbrust and AA Levin. (2002). Complement activation is responsible for acute toxicities in rhesus monkeys treated with a phosphorothioate oligodeoxynucleotide. Int J Immunopharmacol 2:1657-1666.