Therapeutic utility and medicinal chemistry of cathepsin C inhibitors

Current Topics in Medicinal Chemistry

Volumn 10, Issue 7, 2010, Pages 708-716

  Guay, Daniel ^a   Beaulieu, Christian ^a   Percival, M David ^a  

^a MERCK FROSST CENTRE FOR THERAPEUTIC RESEARCH   (Canada)

Author keywords

Cathepsin C; Dipeptidyl peptidase i

Indexed keywords

4 NITRO BENZOYL DERIVATIVE; ACYLOXYMETHYLKETONE DERIVATIVE; AMINONITRILE DERIVATIVE; AZAPEPTIDE DERIVATIVE; CATHEPSIN C INHIBITOR; CYANAMIDE; CYSTEINE PROTEINASE INHIBITOR; DIAZOMETHYLKETONE DERIVATIVE; DIPEPTIDYL PEPTIDASE I; FLUOROMETHYLKETONE DERIVATIVE; HYDROXAMIC ACID DERIVATIVE; N [N (3 CARBOXYOXIRANE 2 CARBONYL)LEUCYL]AGMATINE; O ACYL HYDROXAMIC ACID DERIVATIVE; TRIPEPTIDE DERIVATIVE; UNCLASSIFIED DRUG; VINYLSULFONE DERIVATIVE;

ARTICLE; DRUG EFFECT; DRUG MECHANISM; DRUG STRUCTURE; ENZYME ACTIVITY; ENZYME STRUCTURE; HUMAN; IN VITRO STUDY; IN VIVO STUDY; NONHUMAN;

ANIMALS; CATHEPSIN C; CYSTEINE PROTEINASE INHIBITORS; HUMANS; INHIBITORY CONCENTRATION 50; SUBSTRATE SPECIFICITY;

EID: 77952475301     PISSN: 15680266     EISSN: None     Source Type: Journal    
DOI: 10.2174/156802610791113469     Document Type: Article

References (62)

1. Turk, D.; Janjic, V.; Stern, I.; Podobnik, M.; Lamba, D.; Dahl, S.W.; Lauritzen, C.; Pedersen, J.; Turk, V.; Turk, B. Structure of human dipeptidyl peptidase I (cathepsin C): exclusion domain added to an endopeptidase framework creates the machine for activation of granular serine proteases. EMBO J., 2001, 20, 6570-6582.
2. Tran, T.V.; Ellis, K.A.; Kam, C.M.; Hudig, D.; Powers, J.C. Dipeptidyl peptidase I: importance of progranzyme activation sequences, other dipeptide sequences, and the N-terminal amino group of synthetic substrates for enzyme activity. Arch. Biochem. Biophys., 2002, 403, 160-170.
3. Kuribayashi, M.; Yamada, H.; Ohmori, T.; Yanai, M.; Imoto, T. Endopeptidase activity of cathepsin C, dipeptidyl aminopeptidase I, from bovine spleen. J. Biochem., 1993, 113, 441-449.
4. Wolters, P.J.; Laig-Webster, M.; Caughey, G.H. Dipeptidyl peptidase I cleaves matrix-associated proteins and is expressed mainly by mast cells in normal dog airways. Am. J. Respir. Cell Mol. Biol., 2000, 22, 183-190.
5. Getachew, Y.; Stout-Delgado, H.; Miller, B.C.; Thiele, D.L. Granzyme C supports efficient CTL-mediated killing late in primary alloimmune responses. J. Immunol., 2008, 181, 7810-7817.
6. Pham, C.T.; Ley, T.J. Dipeptidyl peptidase I is required for the processing and activation of granzymes A and B in vivo. Proc. Natl. Acad. Sci. USA, 1999, 96, 8627-8632.
7. Sutton, V.R.; Waterhouse, N.J.; Browne, K.A.; Sedelies, K.; Ciccone, A.; Anthony, D.; Koskinen, A.; Mullbacher, A.; Trapani, J.A. Residual active granzyme B in cathepsin C-null lymphocytes is sufficient for perforin-dependent target cell apoptosis. J. Cell Biol., 2007, 176, 425-433.
8. Adkison, A.M.; Raptis, S.Z.; Kelley, D.G.; Pham, C.T. Dipeptidyl peptidase I activates neutrophil-derived serine proteases and regulates the development of acute experimental arthritis. J. Clin. Invest, 2002, 109, 363-371.
9. Wolters, P.J.; Pham, C.T.; Muilenburg, D.J.; Ley, T.J.; Caughey, G.H. Dipeptidyl peptidase I is essential for activation of mast cell chymases, but not tryptases, in mice. J. Biol. Chem., 2001, 276, 18551-18556.
10. Henningsson, F.; Wolters, P.; Chapman, H.A.; Caughey, G.H.; Pejler, G. Mast cell cathepsins C and S control levels of carboxy- peptidase A and the chymase, mouse mast cell protease 5. Biol. Chem., 2003, 384, 1527-1531.
11. Mallen-St Clair, J.; Shi, G.P.; Sutherland, R.E.; Chapman, H.A.; Caughey, G.H.; Wolters, P.J. Cathepsins L and S are not required for activation of dipeptidyl peptidase I (cathepsin C) in mice. Biol. Chem., 2006, 387, 1143-1146.
12. Pham, C.T.; Armstrong, R.J.; Zimonjic, D.B.; Popescu, N.C.; Payan, D.G.; Ley, T.J. Molecular cloning, chromosomal localization, and expression of murine dipeptidyl peptidase I. J. Biol. Chem., 1997, 272, 10695-10703.
13. Brown, G.R.; McGuire, M.J.; Thiele, D.L. Dipeptidyl peptidase I is enriched in granules of in vitro- and in vivo-activated cytotoxic T lymphocytes. J. Immunol., 1993, 150, 4733-4742.
14. Hart, T.C.; Hart, P.S.; Michalec, M.D.; Zhang, Y.; Firatli, E.; Van Dyke, T.E.; Stabholz, A.; Zlotogorski, A.; Shapira, L.; Soskolne, W.A. Haim-Munk syndrome and Papillon-Lefevre syndrome are allelic mutations in cathepsin C. J. Med. Genet., 2000, 37, 88-94.
15. Pham, C.T.; Ivanovich, J.L.; Raptis, S.Z.; Zehnbauer, B.; Ley, T.J. Papillon-Lefevre syndrome: correlating the molecular, cellular, and clinical consequences of cathepsin C/dipeptidyl peptidase I deficiency in humans. J. Immunol., 2004, 173, 7277-7281.
16. Meade, J.L.; de Wynter, E.A.; Brett, P.; Sharif, S.M.; Woods, C.G.; Markham, A.F.; Cook, G.P. A family with Papillon-Lefevre syndrome reveals a requirement for cathepsin C in granzyme B activation and NK cell cytolytic activity. Blood, 2006, 107, 3665-3668.
17. Hu, Y.; Pham, C.T. Dipeptidyl peptidase I regulates the development of collagen-induced arthritis. Arthritis Rheum., 2005, 52, 2553-2558.
18. Sohar, N.; Hammer, H.; Sohar, I. Lysosomal peptidases and glycosidases in rheumatoid arthritis. Biol. Chem., 2002, 383, 865-869.
19. Sohar, N.; Sohar, I.; Hammer, H. Lysosomal enzyme activities: new potential markers for Sjogren's syndrome. Clin. Biochem., 2005, 38, 1120-1126.
20. Pagano, M.B.; Bartoli, M.A.; Ennis, T.L.; Mao, D.; Simmons, P.M.; Thompson, R.W.; Pham, C.T. Critical role of dipeptidyl peptidase I in neutrophil recruitment during the development of experimental abdominal aortic aneurysms. Proc. Natl. Acad. Sci. USA, 2007, 104, 2855-2860.
21. Akk, A.M.; Simmons, P.M.; Chan, H.W.; Agapov, E.; Holtzman, M.J.; Grayson, M.H.; Pham, C.T. Dipeptidyl peptidase I-dependent neutrophil recruitment modulates the inflammatory response to Sendai virus infection. J. Immunol., 2008, 180, 3535-3542.
22. Mallen-St Clair, J.; Pham, C.T.; Villalta, S.A.; Caughey, G.H.; Wolters, P.J. Mast cell dipeptidyl peptidase I mediates survival from sepsis. J. Clin. Invest., 2004, 113, 628-634.
23. Altorjay, A.; Paal, B.; Sohar, N.; Kiss, J.; Szanto, I.; Sohar, I. Significance and prognostic value of lysosomal enzyme activities measured in surgically operated adenocarcinomas of the gastroesophageal junction and squamous cell carcinomas of the lower third of esophagus. World J. Gastroenterol., 2005, 11, 5751-5756.
24. Gocheva, V.; Joyce, J.A. Cysteine cathepsins and the cutting edge of cancer invasion. Cell Cycle, 2007, 6, 60-64.
25. Cordes, C.; Bartling, B.; Simm, A.; Afar, D.; Lautenschlager, C.; Hansen, G.; Silber, R.E.; Burdach, S.; Hofmann, H.S. Simultaneous expression of Cathepsins B and K in pulmonary adenocarcinomas and squamous cell carcinomas predicts poor recurrence-free and overall survival. Lung Cancer, 2009, 64, 79-85.
26. Pham, C.T. Neutrophil serine proteases fine-tune the inflammatory response. Int. J. Biochem. Cell Biol. 2008, 40, 1317-1333.
27. Willoughby, C.A.; Bull, H.G.; Garcia-Calvo, M.; Jiang, J.; Chapman, K.T.; Thornberry, N.A. Discovery of potent, selective human granzyme B inhibitors that inhibit CTL mediated apoptosis. Bioorg. Med. Chem. Lett., 2002, 12, 2197-2200.
28. Que, X.; Engel, J.C.; Ferguson, D.; Wunderlich, A.; Tomavo, S.; Reed, S.L. Cathepsin Cs are key for the intracellular survival of the protozoan parasite, Toxoplasma gondii. J. Biol. Chem., 2007, 282, 4994-5003.
29. Schneck, J.L.; Villa, J.P.; McDevitt, P.; McQueney, M.S.; Thrall, S.H.; Meek, T.D. Chemical mechanism of a cysteine protease, cathepsin C, as revealed by integration of both steady-state and pre- steady-state solvent kinetic isotope effects. Biochemistry, 2008, 47, 8697-8710.
30. Dahl, S.W.; Halkier, T.; Lauritzen, C.; Dolenc, I.; Pedersen, J.; Turk, V.; Turk, B. Human recombinant pro-dipeptidyl peptidase I (cathepsin C) can be activated by cathepsins L and S but not by autocatalytic processing. Biochemistry, 2001, 40, 1671-1678.
31. Lauritzen, C.; Pedersen, J.; Madsen, M.T.; Justesen, J.; Martensen, P.M.; Dahl, S.W. Active recombinant rat dipeptidyl amino- peptidase I (cathepsin C) produced using the baculovirus expression system. Protein Expr. Purif., 1998, 14, 434-442.
32. McGuire, M.J.; Lipsky, P.E.; Thiele, D.L. Generation of active myeloid and lymphoid granule serine proteases requires processing by the granule thiol protease dipeptidyl peptidase I. J. Biol. Chem., 1993, 268, 2458-2467.
33. Thiele, D.L.; McGuire, M.J.; Lipsky, P.E. A selective inhibitor of dipeptidyl peptidase I impairs generation of CD8+ T cell cytotoxic effector function. J. Immunol., 1997, 158, 5200-5210.
34. Methot, N.; Rubin, J.; Guay, D.; Beaulieu, C.; Ethier, D.; Reddy, T.J.; Riendeau, D.; Percival, M.D. Inhibition of the activation of multiple serine proteases with a cathepsin C inhibitor requires sustained exposure to prevent pro-enzyme processing. J Biol. Chem., 2007, 282, 20836-20846.
35. Korver, G.E.; Kam, C.M.; Powers, J.C.; Hudig, D. Dipeptide vinyl sulfones suitable for intracellular inhibition of dipeptidyl peptidase I. Int. Immunopharmacol., 2001, 1, 21-32.
36. Methot, N.; Guay, D.; Rubin, J.; Ethier, D.; Ortega, K.; Wong, S.; Normandin, D.; Beaulieu, C.; Reddy, T.J.; Riendeau, D.; Percival, M.D. In vivo inhibition of serine protease processing requires a high fractional inhibition of cathepsin C. Mol. Pharmacol., 2008, 73, 1857-1865.
37. Bondebjerg, J.; Fuglsang, H.; Valeur, K.R.; Kaznelson, D.W.; Hansen, J.A.; Pedersen, R.O.; Krogh, B.O.; Jensen, B.S.; Lauritzen, C.; Petersen, G.; Pedersen, J.; Naerum, L. Novel semicarbazide- derived inhibitors of human dipeptidyl peptidase I (hDPPI). Bioorg. Med. Chem., 2005; 13, 4408-4424.
38. Li, J.; Petrassi, H.M.; Tumanut, C.; Masick, B.T.; Trussell, C.; Harris, J.L. Substrate optimization for monitoring cathepsin C activity in live cells. Bioorg. Med. Chem., 2009, 17, 1064-1070.
39. Yuan, F.; Verhelst, S.H.; Blum, G.; Coussens, L.M.; Bogyo, M. A selective activity-based probe for the papain family cysteine protease dipeptidyl peptidase I/cathepsin C. J. Am. Chem. Soc., 2006, 128, 5616-5617.
40. Hewitt, C.; McCormick, D.; Linden, G.; Turk, D.; Stern, I.; Wallace, I.; Southern, L.; Zhang, L.; Howard, R.; Bullon, P.; Wong, M.; Widmer, R.; Gaffar, K.A.; Awawdeh, L.; Briggs, J.; Yaghmai, R.; Jabs, E.W.; Hoeger, P.; Bleck, O.; Rudiger, S.G. Petersilka, G.; Battino, M.; Brett, P.; Hattab, F.; Al-Hamed, M.; Sloan, P.; Toomes, C.; Dixon, M.; James, J.; Read, A. P.; Thakker, N. The role of cathepsin C in Papillon-Lefevre syndrome, prepubertal periodontitis, and aggressive periodontitis. Hum Mutat., 2004, 23, 222-228.
41. Nikawa, T.; Towatari, T.; Katunuma, N. Purification and characterization of cathepsin J from rat liver. Eur. J. Biochem., 1992, 204, 381-393.
42. Nicklin, M.J.; Barrett, A.J. Inhibition of cysteine proteinases and dipeptidyl peptidase I by egg-white cystatin. Biochem J., 1984, 223, 245-253.
43. Dolenc, I.; Turk, B.; Kos, J.; Turk, V. Interaction of human cathepsin C with chicken cystatin. FEBS Lett., 1996, 392, 277-280.
44. Molgaard, A.; Arnau, J.; Lauritzen, C.; Larsen, S.; Petersen, G.; Pedersen, J. The crystal structure of human dipeptidyl peptidase I (cathepsin C) in complex with the inhibitor Gly-Phe-CHN2. Biochem. J., 2007, 401, 645-650.
45. Barrett, A.J.; Kembhavi, A.A.; Brown, M.A.; Kirschke, H.; Knight, C.G.; Tamai, M.; Hanada, K. L-trans-Epoxysuccinyl-leucyl-amido(4-guanidino)butane (E-64) and its analogues as inhibitors of cysteine proteinases including cathepsins B, H and L. Biochem. J., 1982, 201, 189-198.
46. McGuire, M.J.; Lipsky, P.E.; Thiele, D.L. Purification and characterization of dipeptidyl peptidase I from human spleen. Arch. Biochem. Biophys., 1992, 295, 280-288.
47. Kam, C.M.; Gotz, M.G.; Koot, G.; McGuire, M.; Thiele, D.; Hudig, D.; Powers, J.C. Design and evaluation of inhibitors for dipeptidyl peptidase I (Cathepsin C). Arch. Biochem. Biophys., 2004, 427, 123-134.
48. Demuth, H.-U.; Heiser, U.; Niestroj, A. Novel inhibitors of dipeptidyl peptidase I. WO 2003022871A2, 2003.
49. Horn, M.; Pavlik, M.; Doleckova, L.; Baudys, M.; Mares, M. Arginine-based structures are specific inhibitors of cathepsin C. Application of peptide combinatorial libraries. Eur. J. Biochem, 2000, 267, 3330-3336.
50. Mucha, A.; Pawel, M.; Hurek, J.; Kafarski, P. Synthesis and activity of phosphinic tripeptide inhibitors of cathepsin C. Bioorg. Med. Chem. Lett., 2004, 14, 3113-3116.
51. Latajka, R.; Makowski, M.; Jewginski, M.; Pawelczak, M.; Koroniak, H.; Kafarski, P. Peptide p-nitrophenylanilides containing (E)-dehydrophenylalanine-: synthesis, structural studies and evaluation of their activity towards cathepsin C. New J. Chem., 2006, 30, 1009-1018.
52. Latajka, R.; Jewginski, M.; Makowski, M.; Pawelczak, M.; Huber, T.; Sewald, N.; Kafarski, P. Pentapeptides containing two dehydrophenylalanine residues--synthesis, structural studies and evaluation of their activity towards cathepsin C. J. Pept. Sci., 2008, 14, 1084-1095.
53. Robichaud, J.; Black, W.C.; Therien, M.; Paquet, J.; Oballa, R.M.; Bayly, C.I.; McKay, D.J.; Wang, Q.; Isabel, E.; Leger, S.; Mellon, C.; Kimmel, D.B.; Wesolowski, G.; Percival, M.D.; Masse, F.; Desmarais, S.; Falgueyret, J.P.; Crane, S.N. Identification of a nonbasic, nitrile-containing cathepsin K inhibitor (MK-1256) that is efficacious in a monkey model of osteoporosis. J. Med. Chem., 2008, 51, 6410-6420.
54. Gauthier, J.Y.; Black, W.C.; Courchesne, I.; Cromlish, W.; Desmarais, S.; Houle, R.; Lamontagne, S.; Li, C.S.; Masse, F.; McKay, D.J.; Ouellet, M.; Robichaud, J.; Truchon, J.F.; Truong, V.L.; Wang, Q.; Percival, M.D. The identification of potent, selective, and bioavailable cathepsin S inhibitors. Bioorg Med. Chem. Lett., 2007, 17, 4929-4933.
55. Bondebjerg, J.; Fuglsang, H.; Valeur, K.R.; Pedersen, J.; Naerum, L. Dipeptidyl nitriles as human dipeptidyl peptidase I inhibitors. Bioorg. Med. Chem. Lett., 2006, 16, 3614-3617.
56. Bondebjerg, J.; Fuglsang, H.; Naerum, L. Protease inhibitors. WO 2004/110988 A1, 2004.
57. Guay, D.; Beaulieu, C.; Reddy, T.J.; Zamboni, R.; Methot, N.; Rubin, J.; Ethier, D.; Percival, M.D. Design and synthesis of dipeptidyl nitriles as potent, selective and reversible inhibitors of cathepsin C. Bioorg, Med. Chem. Lett., 2009, 19, 5392-5396.
58. Cage, P.A.; Furber, M.; Luckhurst, C.A.; Sanganee, H.J.; Stein, L.A. Peptidyl nitriles and use thereof as dipeptidylpeptidase I inhibitors. WO120091074829, 2009.
59. Fosbenner, D.T.; Liu, R.; Moore, M.L. Novel cathepsin C inhibitors and their use. WO 2006/094003 A1, 2006.
60. Deng, J.; Laine, D.I.; Palovich, M.R.; Xie, H. Novel cathepsin C inhibitors and their use. WO 2009/026197 A1, 2009.
61. Oballa, R.M.; Truchon, J.F.; Bayly, C.I.; Chauret, N.; Day, S.; Crane, S.; Berthelette, C. A generally applicable method for assessing the electrophilicity and reactivity of diverse nitrile- containing compounds. Bioorg. Med. Chem. Lett., 2007, 17, 998-1002.
62. Falgueyret, J.P.; Oballa, R.M.; Okamoto, O.; Wesolowski, G.; Aubin, Y.; Rydzewski, R.M.; Prasit, P.; Riendeau, D.; Rodan, S.B.; Percival, M.D. Novel, nonpeptidic cyanamides as potent and reversible inhibitors of human cathepsins K and L. J. Med. Chem., 2001, 44, 94-104.