Therapeutic targeting of the complement system in age-related macular degeneration: A review

Clinical and Experimental Ophthalmology

Volumn 40, Issue 1, 2012, Pages 18-26

  Troutbeck, Robyn ^a   Al Qureshi, Salmaan ^a   Guymer, Robyn H ^a  

^a UNIVERSITY OF MELBOURNE   (Australia)

Author keywords

AMD; Complement factor H; Complement system

Indexed keywords

AFLIBERCEPT; ALTERNATIVE COMPLEMENT PATHWAY C3 C5 CONVERTASE; ANTIFACTOR D; ARC 1905; C REACTIVE PROTEIN; COMPLEMENT COMPONENT C1Q; COMPLEMENT COMPONENT C5A; COMPLEMENT FACTOR D; COMPLEMENT FACTOR H; COMPLEMENT MEMBRANE ATTACK COMPLEX; COMPSTATIN; ECULIZUMAB; FIBROBLAST GROWTH FACTOR; JPE 1375; JSM 7717; MONOCLONAL ANTIBODY; OPHTHOTEC; OT 551; POTENTIA; RANIBIZUMAB; TA 106; TNX 234; TRANSFORMING GROWTH FACTOR BETA2; UNCLASSIFIED DRUG;

ANGIOGENESIS; ANTIBODY RESPONSE; ASTHMA; BINDING AFFINITY; BRUCH MEMBRANE; CLINICAL EFFECTIVENESS; CLINICAL EVALUATION; CLINICAL TRIAL (TOPIC); COMPARATIVE EFFECTIVENESS; COMPLEMENT SYSTEM; DRUG EFFICACY; DRUG HALF LIFE; HEMOGLOBINURIA; HUMAN; IMMUNE RESPONSE; IMMUNOPATHOLOGY; MAINTENANCE DRUG DOSE; MOLECULAR RECOGNITION; NONHUMAN; OFF LABEL DRUG USE; OPSONIZATION; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PREDICTIVE VALUE; PROTEIN BINDING; PROTEIN CLEAVAGE; REACTOR CASCADE; RETINA MACULA AGE RELATED DEGENERATION; REVIEW; RISK FACTOR; TREATMENT RESPONSE;

COMPLEMENT INACTIVATING AGENTS; COMPLEMENT SYSTEM PROTEINS; HUMANS; MACULAR DEGENERATION;

EID: 84856633285     PISSN: 14426404     EISSN: 14429071     Source Type: Journal    
DOI: 10.1111/j.1442-9071.2011.02581.x     Document Type: Review

References (49)

1. Klein R, Wang Q, Klein BE, Moss SE, Meuer SM. The relationship of age-related maculopathy, cataract, and glaucoma to visual acuity. Invest Ophthalmol Vis Sci 1995; 36: 182-91.
2. Hageman GS, Anderson DH, Johnson LV etal. A common haplotype in the complement regulatory gene factor H (HF1/CFH) predisposes individuals to age-related macular degeneration. Proc Natl Acad Sci USA 2005; 102: 7227-32.
3. Klein RJ, Zeiss C, Chew EY etal. Complement factor H polymorphism in age-related macular degeneration. Science 2005; 308: 385-9.
4. Edwards AO, Ritter RR, Abel KJ, Manning A, Panhuysen C, Farrer LA. Complement factor H polymorphism and age-related macular degeneration. Science 2005; 308: 421-4.
5. Haines JL, Hauser MA, Schmidt S etal. Complement factor H variant increases the risk of age-related macular degeneration. Science 2005; 308: 419-21.
6. Markiewski MM, Lambris JD. The role of complement in inflammatory diseases from behind the scenes into the spotlight. Am J Pathol 2007; 171: 715-27.
7. Amara U, Rittirsch D, Flierl M etal. Interaction between the coagulation and complement system. Adv Exp Med Biol 2008; 632: 71-9.
8. Manderson AP, Botto M, Walport MJ. The role of complement in the development of systemic lupus erythematosus. Annu Rev Immunol 2004; 22: 431-56.
9. Bradt BM, Kolb WP, Cooper NR. Complement-dependent proinflammatory properties of the Alzheimer's disease beta-peptide. J Exp Med 1998; 188: 431-8.
10. Anderson DH, Mullins RF, Hageman GS, Johnson LV. A role for local inflammation in the formation of drusen in the aging eye. Am J Ophthalmol 2002; 134: 411-31.
11. van der Schaft TL, Mooy CM, de Bruijn WC, de Jong PT. Early stages of age-related macular degeneration: an immunofluorescence and electron microscopy study. Br J Ophthalmol 1993; 77: 657-61.
12. Mullins RF, Aptsiauri N, Hageman GS. Structure and composition of drusen associated with glomerulonephritis: implications for the role of complement activation in drusen biogenesis. Eye (Lond) 2001; 15: 390-5.
13. Hageman GS, Luthert PJ, Victor Chong NH, Johnson LV, Anderson DH, Mullins RF. An integrated hypothesis that considers drusen as biomarkers of immune-mediated processes at the RPE-Bruch's membrane interface in aging and age-related macular degeneration. Prog Retin Eye Res 2001; 20: 705-32.
14. Yates JR, Sepp T, Matharu BK etal. Complement C3 variant and the risk of age-related macular degeneration. N Engl J Med 2007; 357: 553-61.
15. Gold B, Merriam JE, Zernant J etal. Variation in factor B (BF) and complement component 2 (C2) genes is associated with age-related macular degeneration. Nat Genet 2006; 38: 458-62.
16. Rodríguez de Córdoba S, Esparza-Gordillo J, Goicoechea de Jorge E, Lopez-Trascasa M, Sánchez-Corral P. The human complement factor H: functional roles, genetic variations and disease associations. Mol Immunol 2004; 41: 355-67.
17. Hageman GS, Hancox LS, Taiber AJ etal. Extended haplotypes in the complement factor H (CFH) and CFH-related (CFHR) family of genes protect against age-related macular degeneration: characterization, ethnic distribution and evolutionary implications. Ann Med 2006; 38: 592-604.
18. Shuler RK, Schmidt S, Gallins P etal. Peripheral reticular pigmentary change is associated with complement factor H polymorphism (Y402H) in age-related macular degeneration. Ophthalmology 2008; 115: 520-4.
19. Bora PS, Sohn JH, Cruz JM etal. Role of complement and complement membrane attack complex in laser-induced choroidal neovascularization. J Immunol 2005; 174: 491-7.
20. Bora NS, Kaliappan S, Jha P etal. Complement activation via alternative pathway is critical in the development of laser-induced choroidal neovascularization: role of factor B and factor H. J Immunol 2006; 177: 1872-8.
21. Jha P, Bora PS, Bora NS. The role of complement system in ocular diseases including uveitis and macular degeneration. Mol Immunol 2007; 44: 3901-8.
22. Anderson DH, Radeke MJ, Gallo NB etal. The pivotal role of the complement system in aging and age-related macular degeneration: hypothesis re-visited. Prog Retin Eye Res 2010; 29: 95-112.
23. Johnson PT, Betts KE, Radeke MJ, Hageman GS, Anderson DH, Johnson LV. Individuals homozygous for the age-related macular degeneration risk-conferring variant of complement factor H have elevated levels of CRP in the choroid. Proc Natl Acad Sci USA 2006; 103: 17456-61.
24. Robman L, Baird PN, Dimitrov PN, Richardson AJ, Guymer RH. C-reactive protein levels and complement factor H polymorphism interaction in age-related macular degeneration and its progression. Ophthalmology 2010; 117: 1982-8.
25. Brown DM, Kaiser PK, Michels M etal. Ranibizumab versus verteporfin for neovascular age-related macular degeneration. N Engl J Med 2006; 355: 1432-44.
26. Rosenfeld PJ, Brown DM, Heier JS etal. Ranibizumab for neovascular age-related macular degeneration. N Engl J Med 2006; 355: 1419-31.
27. Nguyen QD, Shah SM, Hafiz G etal. A phase I trial of an IV-administered vascular endothelial growth factor trap for treatment in patients with choroidal neovascularization due to age-related macular degeneration. Ophthalmology 2006; 113: 1522. e1-1522.e14.
28. Stewart MW, Rosenfeld PJ. Predicted biological activity of intravitreal VEGF Trap. Br J Ophthalmol 2008; 92: 667-8.
29. Wong WT, Kam W, Cunningham D etal. Treatment of geographic atrophy by the topical administration of OT-551: results of a phase II clinical trial. Invest Ophthalmol Vis Sci 2010; 51: 6131-9.
30. Qin S, Rodrigues G. Progress and perspectives on the role of RPE cell inflammatory responses in the development of age-related macular degeneration. J Inflamm Res 2008; 1: 49-65.
31. Do DV. Antiangiogenic approaches to age-related macular degeneration in the future. Ophthalmology 2009; 116: S24-6.
32. Tolentino MJ. Current molecular understanding and future treatment strategies for pathologic ocular neovascularization. Curr Mol Med 2009; 9: 973-81.
33. Ni Z, Hui P. Emerging pharmacologic therapies for wet age-related macular degeneration. Ophthalmologica 2009; 223: 401-10.
34. Holland MC, Morikis D, Lambris JD. Synthetic small-molecule complement inhibitors. Curr Opin Investig Drugs 2004; 5: 1164-73.
35. Kaushal S, Grossi F, Francois C, Slakter J, ASaP Study Group. Complement C3 inhibitor POT-4: clinical safety of intravitreal administration. 2009. ARVO abstract - 5010 - A611.
36. Brekke OH, Sandlie I. Therapeutic antibodies for human diseases at the dawn of the twenty-first century. Nat Rev Drug Discov 2003; 2: 52.
37. Taube C, Thurman JM, Takeda K etal. Factor B of the alternative complement pathway regulates development of airway hyperresponsiveness and inflammation. Proc Natl Acad Sci USA 2006; 103: 8084-9.
38. Allegretti M, Moriconi A, Beccari AR etal. Targeting C5a: recent advances in drug discovery. Curr Med Chem 2005; 12: 217-36.
39. Schnatbaum K, Locardi E, Scharn D etal. Peptidomimetic C5a receptor antagonists with hydrophobic substitutions at the C-terminus: increased receptor specificity and in vivo activity. Bioorg Med Chem Lett 2006; 16: 5088-92.
40. Rohrer B, Long Q, Coughlin B etal. A targeted inhibitor of the alternative complement pathway reduces angiogenesis in a mouse model of age-related macular degeneration. Invest Ophthalmol Vis Sci 2009; 50: 3056-64.
41. Wagenaar-Bos IG, Hack CE. Structure and function of C1-inhibitor. Immunol Allergy Clin North Am 2006; 26: 615-32.
42. Mullins RF, Faidley EA, Daggett HT, Jomary C, Lotery AJ, Stone EM. Localization of complement 1 inhibitor (C1INH/SERPING1) in human eyes with age-related macular degeneration. Exp Eye Res 2009; 89: 767-73.
43. Ennis S, Jomary C, Mullins R etal. Association between the SERPING1 gene and age-related macular degeneration: a two-stage case-control study. Lancet 2008; 372: 1828-34.
44. Park KH, Ryu E, Tosakulwong N, Wu Y, Edwards AO. Common variation in the SERPING1 gene is not associated with age-related macular degeneration in two independent groups of subjects. Mol Vis 2009; 15: 200-7.
45. Allikmets R, Dean M, Hageman GS etal. The SERPING1 gene and age-related macular degeneration. Lancet 2009; 374: 875-6. author reply 876-7.
46. Ma K, Cashman S, Sweigard J, Kumar-Singh R. Decay accelerating factor (CD55) mediated attenuation of complement: therapeutic implications for age-related macular degeneration. Invest Ophthalmol Vis Sci 2010; 51: 6776-83.
47. Guymer R, Robman L. Chlamydia pneumoniae and age-related macular degeneration: a role in pathogenesis or merely a chance association? Clin Experiment Ophthalmol 2007; 35: 89-93.
48. Baird PN, Robman LD, Richardson AJ etal. Gene-environment interaction in progression of AMD: the CFH gene, smoking and exposure to chronic infection. Hum Mol Genet 2008; 17: 1299-305.
49. Fujimoto T, Sonoda KH, Hijioka K etal. Choroidal neovascularization enhanced by Chlamydia pneumoniae via Toll-like receptor 2 in the retinal pigment epithelium. Invest Ophthalmol Vis Sci 2010; 51: 4694-702.