Discovery of small molecule inhibitors for prevention of complement-mediated immune hemolysis

Drug Design Reviews Online

Volumn 2, Issue 2, 2005, Pages 137-143

  Yazdanbakhsh, Karina ^a  

^a NEW YORK BLOOD CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

1,2,3,4,4A,5,6,7,8,8A DECAHYDRO 6,7 DIHYDROXY 2,5,5,8A TETRAMETHYLNAPHTHALENE 1 SPIRO 2' (7' CARBOXY 6' FORMYL 2',3' DIHYDRO 4' HYDROXYBENZOFURAN); ALLOANTIBODY; AUTOANTIBODY; BCX 1470; C1S INHIBITOR 248; COMPLEMENT; COMPLEMENT COMPONENT C1; COMPLEMENT COMPONENT C1S INHIBITOR; COMPLEMENT FIXING ANTIBODY; COMPLEMENT INHIBITOR; COMPLEMENT RECEPTOR; ERYTHROCYTE ANTIGEN; HEPARIN; NAFAMSTAT MESILATE; SERINE PROTEINASE INHIBITOR; SURAMIN; UNCLASSIFIED DRUG;

ALLOIMMUNIZATION; AUTOIMMUNE DISEASE; BLOOD TRANSFUSION REACTION; COMPETITIVE INHIBITION; COMPLEMENT ACTIVATION; COMPLEMENT CLASSICAL PATHWAY; COMPLEMENT DEPENDENT CYTOTOXICITY; COMPLEMENT INHIBITION; COMPLEMENT MEDIATED IMMUNE HEMOLYSIS; COMPLEMENT SYSTEM; COST EFFECTIVENESS ANALYSIS; DRUG APPROVAL; DRUG DESIGN; DRUG EFFECT; DRUG EFFICACY; DRUG IDENTIFICATION; DRUG MECHANISM; DRUG PENETRATION; DRUG POTENCY; DRUG SAFETY; DRUG SELECTIVITY; ENZYME INHIBITION; ERYTHROCYTE; EXPERIMENTAL ANIMAL; EXPERIMENTAL MODEL; GUINEA PIG; HEALTH HAZARD; HEMOLYSIS; HOST RESISTANCE; HUMAN; IMMUNOGENICITY; IN VITRO STUDY; IN VIVO STUDY; LONG TERM CARE; NONHUMAN; PRIORITY JOURNAL; PROPHYLAXIS; PROTEIN PROTEIN INTERACTION; REVIEW; RISK ASSESSMENT; SENSITIZATION;

EID: 23944508087     PISSN: 15672697     EISSN: None     Source Type: Journal    
DOI: 10.2174/1567269053202642     Document Type: Review

References (67)

1. Volanakis J.E.; Frank, M.M. The Human Complement System in Health and Disease; Marcel Dekker, Inc: New York, 1998.
2. Hugli, T. E.; Muller-Eberhard, H. J. Anaphylatoxins: C3a and C5a. Adv. Immunol., 1978, 26, 1.
3. Liszewski, M. K.; Farries, T. C.; Lublin, D. M.; Rooney, I. A.; Atkinson, J. P. Control of the Complement System. Adv. Immunol., 1996, 61, 201.
4. Snyder, E. L. Transfusion Reactions: State-of-the-Art 1994. [Review]. Vox Sang., 1994, 67 Suppl 3, 143.
5. Baldwin, W. M. III.; Larsen, C. P.; Fairchild, R. L. Innate Immune Responses to Transplants: a Significant Variable With Cadaver Donors. Immunity, 2001, 14, 369.
6. Aukrust, P.; Gullestad, L.; Lappegard, K. T.; Ueland, T.; Aass, H.; Wikeby, L.; Simonsen, S.; Froland, S. S.; Mollnes, T. E. Complement Activation in Patients With Congestive Heart Failure: Effect of High-Dose Intravenous Immunoglobulin Treatment. Circulation, 2001, 104, 1494.
7. Hourcade, D.; Liszewski, M. K.; Krych-Goldberg, M.; Atkinson, J. P. Functional Domains, Structural Variations and Pathogen Interactions of MCP, DAF and CR1. Immunopharmacology, 2000,49, 103.
8. Schapira, M.; de Agostini, A.; Schifferli, J. A.; Colman, R. W. Biochemistry and Pathophysiology of Human C1 Inhibitor: Current Issues. Complement, 1985, 2, 111.
9. Rollins, S. A.; Zhao, J.; Ninomiya, H.; Sims, P. J. Inhibition of Homologous Complement by CD59 Is Mediated by a Species-Selective Recognition Conferred Through Binding to C8 Within C5b-8 or C9 Within C5b-9. J. Immunol., 1991, 146, 2345.
10. Podack, E. R.; Kolb, W. P.; Muller-Eberhard, H. J. The SC5b-7 Complex: Formation, Isolation, Properties, and Subunit Composition. J. Immunol., 1977, 119, 2024.
11. Choi, N. H.; Mazda, T.; Tomita, M. A Serum Protein SP40, 40 Modulates the Formation of Membrane Attack Complex of Complement on Erythrocytes. Mol. Immunol., 1989, 26, 835.
12. Jenne, D. E.; Tschopp, J. Molecular Structure and Functional Characterization of a Human Complement Cytolysis Inhibitor Found in Blood and Seminal Plasma: Identity to Sulfated Glycoprotein 2, a Constituent of Rat Testis Fluid. Proc. Natl. Acad. Sci. USA, 1989, 86, 7123.
13. Murphy, B. F.; Saunders, J. R.; O'Bryan, M. K.; Kirszbaum, L.; Walker, I. D.; d'Apice, A. J. SP-40, 40 Is an Inhibitor of C5b-6-Initiated Haemolysis. Int. Immunol., 1989, 1, 551.
14. Davis, A. E. III.; C1 Inhibitor and Hereditary Angioneurotic Edema. Ann. Rev. Immunol., 1988, 6, 595.
15. Chung, L. P.; Bentley, D. R.; Reid, K. B. Molecular Cloning and Characterization of the cDNA Coding for C4b-Binding Protein, a Regulatory Protein of the Classical Pathway of the Human Complement System. Biochem. J., 1985, 230, 133.
16. Gigli, I.; Fujita, T.; Nussenzweig, V. Modulation of the Classical Pathway C3 Convertase by Plasma Proteins C4 Binding Protein and C3b Inactivator. Proc. Natl. Acad. Sci. USA, 1979, 76, 6596.
17. Weiler, J. M.; Daha, M. R.; Austen, K. F.; Fearon, D. T. Control of the Amplification Convertase of Complement by the Plasma Protein Beta1H. Proc. Natl. Acad. Sci. USA, 1976, 73, 3268.
18. Whaley, K.; Ruddy, S. Modulation of C3b Hemolytic Activity by a Plasma Protein Distinct From C3b Inactivator. Science, 1976, 193, 1011.
19. Ahearn, J. M.; Fearon, D. T. Structure and Function of the Complement Receptors, CR1 (CD35) and CR2 (CD21). Adv. Immunol., 1989, 46, 183.
20. Sim, R. B.; Day, A. J.; Moffatt, B. E.; Fontaine, M. Complement Factor I and Cofactors in Control of Complement System Convertase Enzymes. Methods Enzymol., 1993, 223, 13.
21. Makrides, S. C. Therapeutic Inhibition of the Complement System. Pharmacol. Rev., 1998, 50, 59-87.
22. Kirschfink, M. Targeting Complement in Therapy. Immunol. Rev., 2001, 180, 177.
23. Hack, C. E.; Ogilvie, A. C.; Eisele, B.; Jansen, P. M.; Wagstaff, J.; Thijs, L. G. Initial Studies on the Administration of C1-Esterase Inhibitor to Patients With Septic Shock or With a Vascular Leak Syndrome Induced by Interleukin-2 Therapy. Prog. Clin. Biol. Res., 1994, 388, 335.
24. Quigg, R. J. Use of Complement Inhibitors in Tissue Injury. Trends Mol. Med., 2002, 8, 430.
25. Sahu, A.; Kay, B. K.; Lambris, J. D. Inhibition of Human Complement by a C3-Binding Peptide Isolated From a Phage-Displayed Random Peptide Library. J. Immunol., 1996, 157, 884.
26. Morikis, D.; Lambris, J. D. Structural Aspects and Design of Low-Molecular-Mass Complement Inhibitors. Biochem. Soc. Trans., 2002, 30, 1026.
27. Hillmen, P.; Hall, C.; Marsh, J. C.; Elebute, M.; Bombara, M. P.; Petro, B. E.; Cullen, M. J.; Richards, S. J.; Rollins, S. A.; Mojcik, C. F.; Rother, R. P. Effect of Eculizumab on Hemolysis and Transfusion Requirements in Patients With Paroxysmal Nocturnal Hemoglobinuria. N. Engl. J. Med., 2004, 350, 552.
28. Johnson, B. J. Complement: a Host Defense Mechanism Ready for Pharmacological Manipulation? J. Pharm. Sci., 1977, 66, 1367.
29. Asghar, S. S. Pharmacological Manipulation of Complement System. Pharmacol. Rev., 1984, 36, 223-244.
30. Reynard, A. M. The Regulation of Complement Activity by Pharmacologic Agents. J. Immunopharmacol., 1980, 2, 1.
31. Weiler, J. M.; Yurt, R. W.; Fearon, D. T.; Austen, K. F. Modulation of the Formation of the Amplification Convertase of Complement, C3b, Bb, by Native and Commercial Heparin. J. Exp. Med., 1978,147, 409.
32. Wuillemin, W. A.; te, V. H.; Lubbers, Y. T.; de Ruig, C. P.; Eldering, E.; Hack, C. E. Potentiation of C1 Inhibitor by Glycosaminoglycans: Dextran Sulfate Species Are Effective Inhibitors of in Vitro Complement Activation in Plasma. J. Immunol., 1997, 159, 1953.
33. Kirschfink, M.; Blase, L.; Engelmann, S.; Schwartz-Albiez, R. Secreted Chondroitin Sulfate Proteoglycan of Human B Cell Lines Binds to the Complement Protein C1q and Inhibits Complex Formation of C1. J. Immunol., 1997, 158, 1324.
34. Gralinski, M. R.; Park, J. L.; Ozeck, M. A.; Wiater, B. C.; Lucchesi, B. R. LU 51198, a Highly Sulfated, Low-Molecular-Weight Heparin Derivative, Prevents Complement-Mediated Myocardial Injury in the Perfused Rabbit Heart. J. Pharmacol. Exp. Ther., 1997, 282, 554.
35. te Velthuis, H.; Jansen, P. G.; Hack, C. E.; Eijsman, L.; Wildevuur, C. R. Specific Complement Inhibition With Heparin-Coated Extracorporeal Circuits. Ann. Thorac. Surg., 1996, 61, 1153.
36. Carobbi, A.; Di Stefano, R.; Danesi, R.; Carmellini, M.; Ferrari, M.; Galatioto, C.; Ermini, M.; Mosca, F. Suramin As an Anticomplementary Agent in Xenotransplantation. Transplant. Proc., 1992, 24, 700.
37. Asghar, S. S.; Dingemans, K. P.; Kammeijer, A.; Faber, W. R.; Abdel Mawla, M. Y. Suppression of Complement-Mediated Vascular Injury at Arthus Reaction Sites by Complement Inhibitors. Complement, 1986, 3, 40.
38. Brackertz, D.; Kueppers, F. A One Year's Follow Up of Treatment of Hereditary Angioneurotic Edema (HAE) With Suramin. Allergol. Immunopathol. (Madr.), 1974, 2, 163.
39. Hays, S. J.; Caprathe, B. W.; Gilmore, J. L.; Amin, N.; Emmerling, M. R.; Michael, W.; Nadimpalli, R.; Nath, R.; Raser, K. J.; Stafford, D.; Watson, D.; Wang, K.; Jaen, J. C. 2-Amino-4H-3, 1-Benzoxazin-4-Ones As Inhibitors of C1r Serine Protease. J. Med. Chem., 1998, 41, 1060.
40. Buerke, M.; Schwertz, H.; Seitz, W.; Meyer, J.; Darius, H. Novel Small Molecule Inhibitor of C1s Exerts Cardioprotective Effects in Ischemia-Reperfusion Injury in Rabbits. J. Immunol., 2001, 167,5375.
41. Miyata, T.; Fujita, Y.; Inagi, R.; Inoue, I.; Sugiyama, S.; Maeda, K. Effectiveness of Nafamostat Mesilate on Glomerulonephritis in Immune-Complex Diseases. Lancet, 1993, 341, 1353.
42. Fujii, S.; Hitomi, Y. New Synthetic Inhibitors of C1r, C1 Esterase, Thrombin, Plasmin, Kallikrein and Trypsin. Biochim. Biophys. Acta, 1981, 661, 342.
43. Hitomi, Y.; Fujii, S. Inhibition of Various Immunological Reactions in Vivo by a New Synthetic Complement Inhibitor. Int. Arch. Allergy Appl. Immunol., 1982, 69, 262.
44. Inagi, R.; Miyata, T.; Maeda, K.; Sugiyama, S.; Miyama, A.; Nakashima, I. FUT-175 As a Potent Inhibitor of C5/C3 Convertase Activity for Production of C5a and C3a. Immunol. Lett., 1991, 27,49.
45. Fujita, Y.; Inoue, I.; Inagi, R.; Miyata, T.; Shinzato, T.; Sugiyama, S.; Miyama, A.; Maeda, K. Inhibitory Effect of FUT-175 on Complement Activation and Its Application for Glomerulonephritis With Hypocomplementemia. Nippon Jinzo Gakkai Shi, 1993, 35, 393.
46. Ueda, N.; Midorikawa, A.; Ino, Y.; Oda, M.; Nakamura, K.; Suzuki, S.; Kurumi, M. Inhibitory Effects of Newly Synthesized Active Center-Directed Trypsin-Like Serine Protease Inhibitors on the Complement System. Inflamm. Res., 2000, 49, 42.
47. Sahu, A.; Lambris, J. D. Complement Inhibitors: a Resurgent Concept in Anti-Inflammatory Therapeutics. Immunopharmacology, 2000, 49, 133.
48. Hong, K.; Kinoshita, T.; Kitajima, H.; Inoue, K. Inhibitory Effect of K-76 Monocarboxylic Acid, an Anticomplementary Agent, on the C3b Inactivator System. J. Immunol., 1981, 127, 104.
49. Konno, S.; Tsurufuji, S. Induction of Zymosan-Air-Pouch Inflammation in Rats and Its Characterization With Reference to the Effects of Anticomplementary and Anti-Inflammatory Agents. Br. J. Pharmacol., 1983, 80, 269.
50. Yamada, H.; Kudoh, I.; Nishizawa, H.; Kaneko, K.; Miyazaki, H.; Ohara, M.; Okumura, F. Complement Partially Mediates Acid Aspiration-Induced Remote Organ Injury in the Rat. Acta Anaesthesiol. Scand., 1997, 41, 713.
51. Miyagawa, S.; Shirakura, R.; Matsumiya, G.; Fukushima, N.; Nakata, S.; Matsuda, H.; Matsumoto, M.; Kitamura, H.; Seya, T. Prolonging Discordant Xenograft Survival With Anticomplement Reagents K76COOH and FUT175. Transplantation, 1993, 55, 709.
52. Petz, L. D.; Garratty, G. Acquired Immune Hemolytic Anemias; Churchill Livingstone: New York, 1980.
53. Sazama, K. Reports of 355 Transfusion-Associated Deaths: 1976 Through 1985. Transfusion, 1990, 30, 583.
54. Fischer, J. T.; Petz, L. D.; Garratty, G.; Cooper, N. R. Correlations Between Quantitive Assay of Red Cell-Bound C3, Serologic Reactions, and Hemolytic Anemia. Blood, 1974, 44, 359.
55. Test, S. T.; Woolworth, V. S. Defective Regulation of Complement by the Sickle Erythrocyte: Evidence for a Defect in Control of Membrane Attack Complex Formation. Blood, 1994, 83, 842.
56. Wang, R. H.; Phillips, G. Jr.; Medof, M. E.; Mold, C. Activation of the Alternative Complement Pathway by Exposure of Phosphatidylethanolamine and Phosphatidylserine on Erythrocytes From Sickle Cell Disease Patients. J. Clin. Invest., 1993, 92, 1326.
57. Wenisch, C.; Spitzauer, S.; Florris-Linau, K.; Rumpold, H.; Vannaphan, S.; Parschalk, B.; Graninger, W.; Looareesuwan, S. Complement Activation in Severe Plasmodium Falciparum Malaria. Clin. Immunol. Immunopathol., 1997, 85, 166.
58. Goka, B. Q.; Kwarko, H.; Kurtzhals, J. A.; Gyan, B.; Ofori-Adjei, E.; Ohene, S. A.; Hviid, L.; Akanmori, B. D.; Neequaye, J. Complement Binding to Erythrocytes Is Associated With Macrophage Activation and Reduced Haemoglobin in Plasmodium Falciparum Malaria. Trans. R. Soc. Trop. Med. Hyg., 2001, 95,545.
59. Ham, T. H. Studies on Destruction of Red Cell Blood Cells. I. Chronic Hemolytic Anemia With Paroxysmal Hemoglobinuria: An Investigation of the Mechanism of Hemolysis, With Observations on Five Cases. Archives of Internal Medicine, 1939, 64, 1271.
60. Ham, T. H.; Dingle, J. H. Studies on Destruction of Red Blood Cells. II. Chronic Hemolytic Anemia With Paroxysmal Nocturnal Hemoglobinuria: Certain Immunological Aspects of the Hemolytic Mechanism With Special Reference to Serum Complement. J. Clin. Invest., 1939, 18, 657.
61. Yazdanbakhsh, K.; Kang, S.; Tamasauskas, D.; Sung, D.; Scaradavou, A. Complement Receptor 1 Inhibitors for Prevention of Immune-Mediated Red Cell Destruction: Potential Use in Transfusion Therapy. Blood, 2003, 101, 5046-5052.
62. Yamamoto, S.; Kubotsu, K.; Kida, M.; Kondo, K.; Matsuura, S.; Uchiyama, S.; Yonekawa, O.; Kanno, T. Automated Homogeneous Liposome-Based Assay System for Total Complement Activity. Clin. Chem., 1995, 41, 586.
63. Maadmi, A.; Zheng, X.; Song, J.; Abramowitz, S.; Giclas, P.; Yazdanbakhsh, K. Prevention of complement-mediated immune hemolysis by a small molecule compound. Biochem. Biophys. Res. Commun., 2004, 325, 1465.
64. Issitt, P. D.; Anstee, D. J. Applied Blood Group Serology; Montgomery Scientific Publications: Durham, N.C., 1998.
65. Saez, C.; Thielens, N. M.; Bjes, E. S.; Esser, A. F. Association of Terminal Complement Proteins in Solution and Modulation by Suramin. Biochemistry, 1999, 38, 6807.
66. Singer, S. T.; Wu, V.; Mignacca, R.; Kuypers, F. A.; Morel, P.; Vichinsky, E. P. Alloimmunization and Erythrocyte Autoimmunization in Transfusion-Dependent Thalassemia Patients of Predominantly Asian Descent. Blood, 2000, 96, 3369.
67. Telen, M. J. Principles and problems of transfusion in sickle cell disease. Semin. Hematol., 2001, 38, 315.