Inhibition of pre-protein convertase serine kexin-9 (PCSK-9) as a treatment for hyperlipidaemia

Bibliography

• Steinberg D. Thematic review series: the pathogenesis of atherosclerosis. An interpretive history of the cholesterol controversy: part I. J Lipid Res 2004;45:1583-93
   PubMed Web of Science ®Google Scholar
• Steinberg D. Thematic review series: the pathogenesis of atherosclerosis. An interpretive history of the cholesterol controversy: part II: the early evidence linking hypercholesterolemia to coronary disease in humans. J Lipid Res 2005;46:179-90
   PubMed Web of Science ®Google Scholar
• Di Angelantonio E., Sarwar N, Perry P, Major lipids, apolipoproteins, and risk of vascular disease. JAMA 2009;302:1993-2000
   PubMed Web of Science ®Google Scholar
• Turner RC, Millns H, Neil HA, Risk factors for coronary artery disease in non-insulin dependent diabetes mellitus: united Kingdom Prospective Diabetes Study (UKPDS: 23). BMJ 1998;316:823-8
   PubMed Web of Science ®Google Scholar
• Goldstein JL, Brown MS. The LDL receptor. Arterioscler Thromb Vasc Biol 2009;29:431-8
   PubMed Web of Science ®Google Scholar
• Kearney PM, Baigent C. Statins: are any questions unanswered? Curr Opin Lipidol 2006;17:418-25
   PubMed Web of Science ®Google Scholar
• Fellstrom BC, Jardine AG, Schmieder RE, Rosuvastatin and cardiovascular events in patients undergoing hemodialysis. N Engl J Med 2009;360:1395-407
   PubMed Web of Science ®Google Scholar
• Kjekshus J, Apetrei E, Barrios V, Rosuvastatin in older patients with systolic heart failure. N Engl J Med 2007;357:2248-61
   PubMed Web of Science ®Google Scholar
• Baigent C, Blackwell L, Emberson J, Efficacy and safety of more intensive lowering of LDL cholesterol: a meta-analysis of data from 170,000 participants in 26 randomised trials. Lancet 2010;376:1670-81
   PubMed Web of Science ®Google Scholar
• Stein EA, Lane M, Laskarzewski P. Comparison of statins in hypertriglyceridemia. Am J Cardiol 1998;81:66B-9B
   PubMed Web of Science ®Google Scholar
• Davidson MH, Stein EA, Dujovne CA, The efficacy and six-week tolerability of simvastatin 80 and 160 mg/day. Am J Cardiol 1997;79:38-42
   PubMed Web of Science ®Google Scholar
• Wierzbicki AS. Searching for jupiter: starry eyed optimism is not warranted. Int J Clin Pract 2009;63:685-8
   PubMed Web of Science ®Google Scholar
• Joy TR, Hegele RA. Narrative review: statin-related myopathy. Ann Intern Med 2009;150:858-68
   PubMed Web of Science ®Google Scholar
• Meek C, Wierzbicki AS, Jewkes C, Daily and intermittent rosuvastatin 5 mg therapy in statin intolerant patients: an observational study. Curr Med Res Opin 2012;28:371-8
   PubMed Web of Science ®Google Scholar
• Ginsberg HN, Elam MB, Lovato LC, Effects of combination lipid therapy in type 2 diabetes mellitus. N Engl J Med 2010;362:1563-74
   PubMed Web of Science ®Google Scholar
• Boden WE, Probstfield JL, Anderson T, Niacin in patients with low HDL cholesterol levels receiving intensive statin therapy. N Engl J Med 2011;365:2255-67
   PubMed Web of Science ®Google Scholar
• Wierzbicki AS. Fibrates: no accord on their use in the treatment of dyslipidaemia. Curr Opin Lipidol 2010;21:352-8
   PubMed Web of Science ®Google Scholar
• Wierzbicki AS. HDL: who needs it? Int J Clin Pract 2011;65:1111-13
   PubMed Web of Science ®Google Scholar
• Wierzbicki AS. Muddy waters: more stormy SEAS for ezetimibe. Int J Clin Pract 2008;62:1470-3
   PubMed Web of Science ®Google Scholar
• Baigent C, Landray MJ, Reith C, The effects of lowering LDL cholesterol with simvastatin plus ezetimibe in patients with chronic kidney disease (Study of Heart and Renal Protection): a randomised placebo-controlled trial. Lancet 2011;377:2181-92
   PubMed Web of Science ®Google Scholar
• Wierzbicki AS, Hardman TC, Viljoen A. New lipid-lowering drugs: an update. Int J Clin Pract 2012;66:270-80
   PubMed Web of Science ®Google Scholar
• Wierzbicki AS, Hardman TC, Viljoen A. Novel lipid-lowering therapies: an update. Int J Clin Pract 2012;66:270-80
   PubMed Web of Science ®Google Scholar
• Tibolla G, Norata GD, Artali R, Proprotein convertase subtilisin/kexin type 9 (PCSK9): from structure-function relation to therapeutic inhibition. Nutr Metab Cardiovasc Dis 2011;21:835-43
   PubMed Web of Science ®Google Scholar
• Lambert G, Charlton F, Rye KA, Piper DE. Molecular basis of PCSK9 function. Atherosclerosis 2009;203:1-7
   PubMed Web of Science ®Google Scholar
• Nassoury N, Blasiole DA, Tebon OA, The cellular trafficking of the secretory proprotein convertase PCSK9 and its dependence on the LDLR. Traffic 2007;8:718-32
   PubMed Web of Science ®Google Scholar
• Seidah NG, Benjannet S, Wickham L, The secretory proprotein convertase neural apoptosis-regulated convertase 1 (NARC-1): liver regeneration and neuronal differentiation. Proc Natl Acad Sci USA 2003;100:928-33
   PubMed Web of Science ®Google Scholar
• Ferri N, Tibolla G, Pirillo A, Proprotein convertase subtilisin kexin type 9 (PCSK9) secreted by cultured smooth muscle cells reduces macrophages LDLR levels. Atherosclerosis 2012;220:381-6
   PubMed Web of Science ®Google Scholar
• Poirier S, Mayer G, Benjannet S, The proprotein convertase PCSK9 induces the degradation of low density lipoprotein receptor (LDLR) and its closest family members VLDLR and ApoER2. J Biol Chem 2008;283:2363-72
   PubMed Web of Science ®Google Scholar
• Zhang DW, Garuti R, Tang WJ, Structural requirements for PCSK9-mediated degradation of the low-density lipoprotein receptor. Proc Natl Acad Sci USA 2008;105:13045-50
   PubMed Web of Science ®Google Scholar
• Li J, Tumanut C, Gavigan JA, Secreted PCSK9 promotes LDL receptor degradation independently of proteolytic activity. Biochem J 2007;406:203-7
   PubMed Web of Science ®Google Scholar
• Abifadel M, Varret M, Rabes JP, Mutations in PCSK9 cause autosomal dominant hypercholesterolemia. Nat Genet 2003;34:154-6
   PubMed Web of Science ®Google Scholar
• Cohen JC, Boerwinkle E, Mosley TH Jr, Hobbs HH. Sequence variations in PCSK9, low LDL, and protection against coronary heart disease. N Engl J Med 2006;354:1264-72
   PubMed Web of Science ®Google Scholar
• Cariou B, Ouguerram K, Zair Y, PCSK9 dominant negative mutant results in increased LDL catabolic rate and familial hypobetalipoproteinemia. Arterioscler Thromb Vasc Biol 2009;29:2191-7
   PubMed Web of Science ®Google Scholar
• Huijgen R, Fouchier SW, Denoun M, Plasma levels of proprotein convertase subtilisin Kexin type 9 (PCSK9) and phenotypic variability in familial hypercholesterolemia. J Lipid Res 2012; published online; doi:jlr.P023994 [pii];10.1194/jlr.P023994 [doi]
   PubMed Web of Science ®Google Scholar
• Mbikay M, Sirois F, Mayne J, PCSK9-deficient mice exhibit impaired glucose tolerance and pancreatic islet abnormalities. FEBS Lett 2010;584:701-6
   PubMed Web of Science ®Google Scholar
• Hooper AJ, Marais AD, Tanyanyiwa DM, Burnett JR. The C679X mutation in PCSK9 is present and lowers blood cholesterol in a Southern African population. Atherosclerosis 2007;193:445-8
   PubMed Web of Science ®Google Scholar
• Baass A, Dubuc G, Tremblay M, Plasma PCSK9 is associated with age, sex, and multiple metabolic markers in a population-based sample of children and adolescents. Clin Chem 2009;55:1637-45
   PubMed Web of Science ®Google Scholar
• Costet P, Cariou B, Lambert G, Hepatic PCSK9 expression is regulated by nutritional status via insulin and sterol regulatory element-binding protein 1c. J Biol Chem 2006;281:6211-18
   PubMed Web of Science ®Google Scholar
• Le MC, Kourimate S, Langhi C, Proprotein convertase subtilisin kexin type 9 null mice are protected from postprandial triglyceridemia. Arterioscler Thromb Vasc Biol 2009;29:684-90
   PubMed Web of Science ®Google Scholar
• Leblond F, Seidah NG, Precourt LP, Regulation of the proprotein convertase subtilisin/kexin type 9 in intestinal epithelial cells. Am J Physiol Gastrointest Liver Physiol 2009;296:G805-15
   PubMed Web of Science ®Google Scholar
• Dong B, Wu M, Cao A, Suppression of Idol expression is an additional mechanism underlying statin-induced up-regulation of hepatic LDL receptor expression. Int J Mol Med 2011;27:103-10
   PubMed Web of Science ®Google Scholar
• Poirier S, Mayer G, Poupon V, Dissection of the endogenous cellular pathways of PCSK9-induced low density lipoprotein receptor degradation: evidence for an intracellular route. J Biol Chem 2009;284:28856-64
   PubMed Web of Science ®Google Scholar
• Denis M, Marcinkiewicz J, Zaid A, Gene inactivation of proprotein convertase subtilisin/kexin type 9 reduces atherosclerosis in mice. Circulation 2012;125:894-901
   PubMed Web of Science ®Google Scholar
• Dubuc G, Tremblay M, Pare G, A new method for measurement of total plasma PCSK9: clinical applications. J Lipid Res 2010;51:140-9
   PubMed Web of Science ®Google Scholar
• Persson L, Henriksson P, Westerlund E, Endogenous estrogens lower plasma PCSK9 and LDL cholesterol but not Lp(a) or bile acid synthesis in women. Arterioscler Thromb Vasc Biol 2012;32:810-4
   PubMed Web of Science ®Google Scholar
• Persson L, Cao G, Stahle L, Circulating proprotein convertase subtilisin kexin type 9 has a diurnal rhythm synchronous with cholesterol synthesis and is reduced by fasting in humans. Arterioscler Thromb Vasc Biol 2010;30:2666-72
   PubMed Web of Science ®Google Scholar
• Labonte P, Begley S, Guevin C, PCSK9 impedes hepatitis C virus infection in vitro and modulates liver CD81 expression. Hepatology 2009;50:17-24
   PubMed Web of Science ®Google Scholar
• Nilsson LM, Abrahamsson A, Sahlin S, Bile acids and lipoprotein metabolism: effects of cholestyramine and chenodeoxycholic acid on human hepatic mRNA expression. Biochem Biophys Res Commun 2007;357:707-11
   PubMed Web of Science ®Google Scholar
• Careskey HE, Davis RA, Alborn WE, Atorvastatin increases human serum levels of proprotein convertase subtilisin/kexin type 9. J Lipid Res 2008;49:394-8
   PubMed Web of Science ®Google Scholar
• Awan Z, Seidah NG, Macfadyen JG, Rosuvastatin, proprotein convertase subtilisin/kexin type 9 concentrations, and LDL cholesterol response: the jupiter trial. Clin Chem 2012;58:183-9
   PubMed Web of Science ®Google Scholar
• Welder G, Zineh I, Pacanowski MA, High-dose atorvastatin causes a rapid sustained increase in human serum PCSK9 and disrupts its correlation with LDL cholesterol. J Lipid Res 2010;51:2714-21
   PubMed Web of Science ®Google Scholar
• Tremblay AJ, Lamarche B, Lemelin V, Atorvastatin increases intestinal expression of NPC1L1 in hyperlipidemic men. J Lipid Res 2011;52:558-65
   PubMed Web of Science ®Google Scholar
• Georges A, Bonneau J, Bonnefont-Rousselot D, Molecular analysis and intestinal expression of SAR1 genes and proteins in Anderson's disease (Chylomicron retention disease). Orphanet J Rare Dis 2011;6:1
   PubMed Web of Science ®Google Scholar
• Lambert G, Ancellin N, Charlton F, Plasma PCSK9 concentrations correlate with LDL and total cholesterol in diabetic patients and are decreased by fenofibrate treatment. Clin Chem 2008;54:1038-45
   PubMed Web of Science ®Google Scholar
• Li H, Dong B, Park SW, Hepatocyte nuclear factor 1alpha plays a critical role in PCSK9 gene transcription and regulation by the natural hypocholesterolemic compound berberine. J Biol Chem 2009;284:28885-95
   PubMed Web of Science ®Google Scholar
• Kong W, Wei J, Abidi P, Berberine is a novel cholesterol-lowering drug working through a unique mechanism distinct from statins. Nat Med 2004;10:1344-51
   PubMed Web of Science ®Google Scholar
• Kourimate S, Le MC, Langhi C, Dual mechanisms for the fibrate-mediated repression of proprotein convertase subtilisin/kexin type 9. J Biol Chem 2008;283:9666-73
   PubMed Web of Science ®Google Scholar
• Costet P, Hoffmann MM, Cariou B, Plasma PCSK9 is increased by fenofibrate and atorvastatin in a non-additive fashion in diabetic patients. Atherosclerosis 2010;212:246-51
   PubMed Web of Science ®Google Scholar
• Noguchi T, Kobayashi J, Yagi K, Comparison of effects of bezafibrate and fenofibrate on circulating proprotein convertase subtilisin/kexin type 9 and adipocytokine levels in dyslipidemic subjects with impaired glucose tolerance or type 2 diabetes mellitus: results from a crossover study. Atherosclerosis 2011;217:165-70
   PubMed Web of Science ®Google Scholar
• Wierzbicki AS, Mikhailidis DP, Wray R, Statin-fibrate combination: therapy for hyperlipidemia: a review. Curr Med Res Opin 2003;19:155-68
   PubMed Web of Science ®Google Scholar
• Davignon J, Dubuc G. Statins and ezetimibe modulate plasma proprotein convertase subtilisin kexin-9 (PCSK9) levels. Trans Am Clin Climatol Assoc 2009;120:163-73
   PubMedGoogle Scholar
• Ason B, Tep S, Davis HR Jr, Improved efficacy for ezetimibe and rosuvastatin by attenuating the induction of PCSK9. J Lipid Res 2011;52:679-87
   PubMed Web of Science ®Google Scholar
• Abifadel M, Pakradouni J, Collin M, Strategies for proprotein convertase subtilisin kexin 9 modulation: a perspective on recent patents. Expert Opin Ther Patents 2010;20:1547-71
   PubMed Web of Science ®Google Scholar
• Bottomley MJ, Cirillo A, Orsatti L, Structural and biochemical characterization of the wild type PCSK9-EGF(AB) complex and natural familial hypercholesterolemia mutants. J Biol Chem 2009;284:1313-23
   PubMed Web of Science ®Google Scholar
• Ni YG, Di MS, Condra JH, A PCSK9-binding antibody that structurally mimics the EGF(A) domain of LDL-receptor reduces LDL cholesterol in vivo. J Lipid Res 2011;52:78-86
   PubMed Web of Science ®Google Scholar
• Crunkhorn S. Trial watch: PCSK9 antibody reduces LDL cholesterol. Nat Rev Drug Discov 2011;11:11
   Web of Science ®Google Scholar
• Duff CJ, Scott MJ, Kirby IT, Antibody-mediated disruption of the interaction between PCSK9 and the low-density lipoprotein receptor. Biochem J 2009;419:577-84
   PubMed Web of Science ®Google Scholar
• Verch T, Chilewski S, Bouaraphan S, Pharmacokinetic immunoassay methods in the presence of soluble target. J Immunol Methods 2010;361:75-81
   PubMedGoogle Scholar
• Chilewski SD, Follmer T, Ansbro F, Validation of preclinical pharmacokinetic and immunogenicity assays for an anti-PCSK9 antibody. J Immunoassay Immunochem 2011;32:296-317
   PubMed Web of Science ®Google Scholar
• Liang H, Chaparro-Riggers J, Strop P, Proprotein convertase substilisin/kexin type 9 antagonism reduces low-density lipoprotein cholesterol in statin-treated hypercholesterolemic nonhuman primates. J Pharmacol Exp Ther 2012;340:228-36
   PubMed Web of Science ®Google Scholar
• Swergold G, Biedermann S, Renard R, Safety, Lipid, and Lipoprotein Effects of REGN727/SAR236553, a Fully-Human Proprotein Convertase Subtilisin Kexin 9 (PCSK9) Monoclonal Antibody Administered Intravenously to Healthy Volunteers. Circulation 2010; 122 supplement 21: A23251 (abstract).
   Google Scholar
• Amgen. TIMI-57 - A Double-blind, Randomized, Placebo-controlled, Multicenter, Dose-ranging Study to Evaluate Tolerability and Efficacy of AMG 145 on Low-Density Lipoprotein Cholesterol (LDL-C) in Combination With HMG-CoA Reductase Inhibitors in Hypercholesterolemic Subjects. ClinicalTrials Gov,2011. Available from: http://clinicaltrials.gov/ct2/show/NCT01380730 [Last accessed 10 October 2011]
   Google Scholar
• Frank-Kamenetsky M, Grefhorst A, Anderson NN, Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates. Proc Natl Acad Sci USA 2008;105:11915-20
   PubMed Web of Science ®Google Scholar
• Stein EA, Mellis S, Yancopoulos GD, Effect of a monoclonal antibody to PCSK9 on LDL cholesterol. N Engl J Med. 2012. 366:1108-18
   Google Scholar
• Dias C, Shaywitz A, Smith B, A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Ascending Single Dose Study to Evaluate the Safety, Tolerability and Pharmacodynamics of AMG145. Circulation 2011; 124 Supplement 21 : A10701 (abstract)
   Google Scholar
• Gouni-Berthold I, Berthold HK. Antisense oligonucleotides for the treatment of dyslipidemia. Curr Pharm Des 2011;17:950-60
   PubMed Web of Science ®Google Scholar
• Frank-Kamenetsky M, Grefhorst A, Anderson NN, Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates. Proc Natl Acad Sci USA 2008;105:11915-20
   PubMed Web of Science ®Google Scholar
• Graham MJ, Lemonidis KM, Whipple CP, Antisense inhibition of proprotein convertase subtilisin/kexin type 9 reduces serum LDL in hyperlipidemic mice. J Lipid Res 2007;48:763-7
   PubMed Web of Science ®Google Scholar
• Lalanne F, Lambert G, Amar MJ, Wild-type PCSK9 inhibits LDL clearance but does not affect apoB-containing lipoprotein production in mouse and cultured cells. J Lipid Res 2005;46:1312-19
   PubMed Web of Science ®Google Scholar
• Lindholm MW, Elmen J, Fisker N, PCSK9 LNA antisense oligonucleotides induce sustained reduction of LDL cholesterol in nonhuman primates. Mol Ther 2011; published online; doi:mt2011260 [pii];10.1038/mt.2011.260 [doi]
   PubMed Web of Science ®Google Scholar
• Wierzbicki AS. The ezetimibe Jonah: the trials and tribulations of an unlucky drug. Int J Clin Pract 2011;65:1207-8
   PubMed Web of Science ®Google Scholar
• Fruchart JC, Sacks F, Hermans MP, The residual risk reduction initiative: a call to action to reduce residual vascular risk in patients with dyslipidemia. Am J Cardiol 2008;102:1K-34K
   PubMed Web of Science ®Google Scholar
• Chapman MJ, Ginsberg HN, Amarenco P, Triglyceride-rich lipoproteins and high-density lipoprotein cholesterol in patients at high risk of cardiovascular disease: evidence and guidance for management. Eur Heart J 2011;32:1345-61
   PubMed Web of Science ®Google Scholar
• Nicholls SJ, Ballantyne CM, Barter PJ, Effect of two intensive statin regimens on progression of coronary disease. N Engl J Med 2011;365:2078-87
   PubMed Web of Science ®Google Scholar
• Wiviott SD, Cannon CP. The safety and efficacy of achieving very low LDL-cholesterol concentrations with high dose statin therapy. Curr Opin Lipidol 2006;17:626-30
   PubMed Web of Science ®Google Scholar
• Wierzbicki AS. Surrogate markers, atherosclerosis and cardiovascular disease prevention. Int J Clin Pract 2008;62:981-7
   PubMed Web of Science ®Google Scholar
• Expert Panel on Detection Evaluation and Treatment of High Blood Cholesterol In Adults (Adult Treatment Panel III). Executive Summary of The Third Report of The National Cholesterol Education Program (NCEP). JAMA 2001 285:2486-97
   PubMed Web of Science ®Google Scholar
• Graham I, Atar D, Borch-Johnsen K, European guidelines on cardiovascular disease prevention in clinical practice: executive summary. Atherosclerosis 2007;194:1-45
   PubMed Web of Science ®Google Scholar
• British Cardiac Society, British Hypertension Society, Diabetes UK, et al. JBS 2: the joint British Societies' guidelines for prevention of cardiovascular disease in clinical practice. Heart 2005 91(Suppl V):v1-v52
   Google Scholar
• National Institute for Health and Clinical Excellence. Lipid modification. London, UK: National Institute for Health and Clinical Excellence; 2008 Jun 17. Report No.: CG67
   Google Scholar
• Wierzbicki AS, Humphries SE, Minhas R. Familial hypercholesterolaemia: summary of NICE guidance. BMJ 2008;337:a1095
   PubMed Web of Science ®Google Scholar
• Raal FJ, Santos RD, Blom DJ, Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial. Lancet 2010;375:998-1006
   PubMed Web of Science ®Google Scholar
• Akdim F, Visser ME, Tribble DL, Effect of mipomersen, an apolipoprotein B synthesis inhibitor, on low-density lipoprotein cholesterol in patients with familial hypercholesterolemia. Am J Cardiol 2010;105:1413-19
   PubMed Web of Science ®Google Scholar
• Kling J. Safety signal dampens reception for mipomersen antisense. Nat Biotechnol 2010;28:295-7
   PubMed Web of Science ®Google Scholar
• Cuchel M, Bloedon LT, Szapary PO, Inhibition of microsomal triglyceride transfer protein in familial hypercholesterolemia. N Engl J Med 2007;356:148-56
   PubMed Web of Science ®Google Scholar
• Wierzbicki AS, Hardman T, Prince WT. Future challenges for microsomal transport protein inhibitors. Curr Vasc Pharmacol 2009;7:277-86
   PubMed Web of Science ®Google Scholar