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Experimental models of systemic sclerosis – a tool to understand and treat an incurable disease?


Authors: H. Štorkánová;  M. Tomčík
Authors place of work: Revmatologický ústav a Revmatologická klinika 1. LF UK
Published in the journal: Čes. Revmatol., 23, 2015, No. 2, p. 59-68.
Category: Reviews

Summary

Systemic sclerosis (SSc) is a rare, chronic connective tissue disease affecting the skin, vessels, musculoskeletal system and internal organs. Despite advances in pharmacotherapy of organ manifestation and new knowledge about the pathogenesis of SSc, there is still no effective treatment of this serious disease. The aim of this review is to introduce traditional, most commonly used experimental animal models of SSc, clarify their basic pathomechanism, describe the advantages and limitations and outline their use in preclinical tests of potential therapeutic agents with subsequent clinical trials in patients with SSc.

KEY WORDS:
Systemic sclerosis, experimental models, biological therapy, small molecules


Zdroje

1. Gabrielli A, Avvedimento EV, Krieg T. Mechanisms of Disease: Scleroderma. New Engl J Med 2009 May 7; 360(19): 1989–2003.

2. Varga J, Abraham D. Systemic sclerosis: a prototypic multisystem fibrotic disorder. The Journal of clinical investigation 2007 Mar; 117(3): 557–567.

3. Denton CP, Black CM. Scleroderma--clinical and pathological advances. Best Pract Res Clin Rheumatol 2004 Jun; 18(3): 271–290.

4. Beyer C, Schett G, Distler O, Distler JH. Animal models of systemic sclerosis: prospects and limitations. Arthritis and rheumatism Oct; 62(10): 2831–2844.

5. Umezawa H, Ishizuka M, Maeda K, Takeuchi T. Studies on bleomycin. Cancer 1967 May; 20(5): 891–895.

6. Yamamoto T, Takagawa S, Katayama I, Yamazaki K, Hamazaki Y, Shinkai H, et al. Animal model of sclerotic skin. I: Local injections of bleomycin induce sclerotic skin mimicking scleroderma. J Investigative Dermatol 1999 Apr; 112(4): 456–462.

7. Yoshizaki A, Iwata Y, Komura K, Ogawa F, Hara T, Muroi E, et al. CD19 regulates skin and lung fibrosis via Toll-like receptor signaling in a model of bleomycin-induced scleroderma. Am J Pathol 2008 Jun; 172(6): 1650–1663.

8. Yamamoto T, Nishioka K. Cellular and molecular mechanisms of bleomycin-induced murine scleroderma: current update and future perspective. Exp Dermatol. 2005 Feb; 14(2): 81–95.

9. Vogelsang GB, Lee L, Bensen-Kennedy DM. Pathogenesis and treatment of graft-versus-host disease after bone marrow transplant. Annu Rev Med 2003; 54: 29–52.

10. Ruzek MC, Jha S, Ledbetter S, Richards SM, Garman RD. A modified model of graft-versus-host-induced systemic sclerosis (scleroderma) exhibits all major aspects of the human disease. Arthritis Rheum 2004 Apr; 50(4): 1319–1331.

11. Jaffee BD, Claman HN. Chronic graft-versus-host disease (GVHD) as a model for scleroderma. I. Description of model systems. Cell Immunol 1983 Apr 1; 77(1): 1–12.

12. Claman HN, Jaffee BD, Huff JC, Clark RA. Chronic graft-versus-host disease as a model for scleroderma. II. Mast cell depletion with deposition of immunoglobulins in the skin and fibrosis. Cell Immunol 1985 Aug; 94(1): 73–84.

13. Howell CD, Yoder T, Claman HN, Vierling JM. Hepatic homing of mononuclear inflammatory cells isolated during murine chronic graft-vs-host disease. J Immunol 1989 Jul 15; 143(2): 476–483.

14. Levy S, Nagler A, Okon S, Marmary Y. Parotid salivary gland dysfunction in chronic graft-versus-host disease (cGVHD): a longitudinal study in a mouse model. Bone Marrow Transplant 2000 May; 25(10): 1073–1078.

15. Li J, Helm K, Howell CD. Contributions of donor CD4 and CD8 cells to liver injury during murine graft-versus-host disease. Transplantation 1996 Dec 15; 62(11): 1621–1628.

16. Siracusa LD, McGrath R, Ma Q, Moskow JJ, Manne J, Christner PJ, et al. A tandem duplication within the fibrillin 1 gene is associated with the mouse tight skin mutation. Genome Res 1996 Apr; 6(4): 300–313.

17. Green MC, Sweet HO, Bunker LE. Tight-skin, a new mutation of the mouse causing excessive growth of connective tissue and skeleton. Amer J Pathol 1976 Mar; 82(3): 493–512.

18. Menton DN, Hess RA, Lichtenstein JR, Eisen A. The structure and tensile properties of the skin of tight-skin (Tsk) mutant mice. J Invest Dermatol 1978 Jan; 70(1): 4–10.

19. Menton DN, Hess RA. The ultrastructure of collagen in the dermis of tight-skin (Tsk) mutant mice. J Invest Dermatol 1980 Mar; 74(3): 139–147.

20. Baxter RM, Crowell TP, McCrann ME, Frew EM, Gardner H. Analysis of the tight skin (Tsk1/+) mouse as a model for testing antifibrotic agents. Lab Invest 2005 Oct; 85(10): 1199–1209.

21. Saito E, Fujimoto M, Hasegawa M, Komura K, Hamaguchi Y, Kaburagi Y, et al. CD19-dependent B lymphocyte signaling thresholds influence skin fibrosis and autoimmunity in the tight-skin mouse. J Clin Invest 2002 Jun; 109(11): 1453–1462.

22. Gershwin ME, Abplanalp H, Castles JJ, Ikeda RM, van der Water J, Eklund J, et al. Characterization of a spontaneous disease of white leghorn chickens resembling progressive systemic sclerosis (scleroderma). J Exp Med 1981 Jun 1; 153(6): 1640–1659.

23. Wick G, Andersson L, Hala K, Gershwin ME, Selmi C, Erf GF, et al. Avian models with spontaneous autoimmune diseases. Adv Immunol 2006; 92:71–117.

24. Sgonc R, Gruschwitz MS, Dietrich H, Recheis H, Gershwin ME, Wick G. Endothelial cell apoptosis is a primary pathogenetic event underlying skin lesions in avian and human scleroderma. J Clin Invest 1996 Aug 1; 98(3): 785–792.

25. Sgonc R, Wick G. Pro- and anti-fibrotic effects of TGF-beta in scleroderma. Rheumatology (Oxford) 2008 Oct; 47 Suppl 5: v5–7.

26. Wagner EF, Eferl R. Fos/AP-1 proteins in bone and the immune system. Immunol Rev 2005 Dec; 208: 126–140.

27. Reich N, Maurer B, Akhmetshina A, Venalis P, Dees C, Zerr P, et al. The transcription factor Fra-2 regulates the production of extracellular matrix in systemic sclerosis. Arthritis Rheum Jan; 62(1): 280–290.

28. Maurer B, Busch N, Jungel A, Pileckyte M, Gay RE, Michel BA, et al. Transcription factor fos-related antigen-2 induces progressive peripheral vasculopathy in mice closely resembling human systemic sclerosis. Circulation 2009 Dec 8; 120(23): 2367–2376.

29. Eferl R, Hasselblatt P, Rath M, Popper H, Zenz R, Komnenovic V, et al. Development of pulmonary fibrosis through a pathway involving the transcription factor Fra-2/AP-1. Proc Natl Acad Sci U S A 2008 Jul 29; 105(30): 10525–10530.

30. Bou-Gharios G, Garrett LA, Rossert J, Niederreither K, Eberspaecher H, Smith C, et al. A potent far-upstream enhancer in the mouse pro alpha 2(I) collagen gene regulates expression of reporter genes in transgenic mice. J Cell Biol 1996 Sep; 134(5): 1333–1344.

31. Sonnylal S, Denton CP, Zheng B, Keene DR, He R, Adams HP, et al. Postnatal induction of transforming growth factor beta signaling in fibroblasts of mice recapitulates clinical, histologic, and biochemical features of scleroderma. Arth Rheum 2007 Jan; 56(1): 334–344.

32. Denton CP, Zheng B, Shiwen X, Zhang Z, Bou-Gharios G, Eberspaecher H, et al. Activation of a fibroblast-specific enhancer of the proalpha2(I) collagen gene in tight-skin mice. Arth Rheum 2001 Mar; 44(3): 712–722.

33. Moore BB, Hogaboam CM. Murine models of pulmonary fibrosis. American journal of physiology Lung Cell Mol Physiol 2008 Feb; 294(2): L152–160.

34. Degryse AL, Lawson WE. Progress toward improving animal models for idiopathic pulmonary fibrosis. Ameri J Med Scien 2011 Jun; 341(6): 444–449.

35. Chung MP, Monick MM, Hamzeh NY, Butler NS, Powers LS, Hunninghake GW. Role of repeated lung injury and genetic background in bleomycin-induced fibrosis. Amer J Respir cell Mol Biol 2003 Sep; 29(3 Pt 1): 375–380.

36. B BM, Lawson WE, Oury TD, Sisson TH, Raghavendran K, Hogaboam CM. Animal models of fibrotic lung disease. American journal of respiratory cell and molecular biology. 2013 Aug; 49(2): 167–179.

37. Gomez-Arroyo J, Saleem SJ, Mizuno S, Syed AA, Bogaard HJ, Abbate A, et al. A brief overview of mouse models of pulmonary arterial hypertension: problems and prospects. Amer J Physiol Lung Cell Mol Physiol 2012 May 15; 302(10): L977–991.

38. Nicolls MR, Mizuno S, Taraseviciene-Stewart L, Farkas L, Drake JI, Al Husseini A, et al. New models of pulmonary hypertension based on VEGF receptor blockade-induced endothelial cell apoptosis. Pulm Circul 2012 Oct; 2(4): 434–442.

39. Sakao S, Tatsumi K. The effects of antiangiogenic compound SU5416 in a rat model of pulmonary arterial hypertension. Respiration; international review of thoracic diseases 2011; 81(3): 253–261.

40. Stenmark KR, Meyrick B, Galie N, Mooi WJ, McMurtry IF. Animal models of pulmonary arterial hypertension: the hope for etiological discovery and pharmacological cure. American journal of physiology Lung Cell Molec Physiol 2009 Dec; 297(6): L1013–1032.

41. Firth AL, Choi IW, Park WS. Animal models of pulmonary hypertension: Rho kinase inhibition. Prog Biophysics Mol Biol 2012 Aug; 109(3): 67–75.

42. Koca SS, Isik A, Ozercan IH, Ustundag B, Evren B, Metin K. Effectiveness of etanercept in bleomycin-induced experimental scleroderma. Rheumatology 2008 Feb; 47(2): 172–175.

43. Mauviel A, Daireaux M, Redini F, Galera P, Loyau G, Pujol JP. Tumor necrosis factor inhibits collagen and fibronectin synthesis in human dermal fibroblasts. FEBS letters 1988 Aug 15; 236(1): 47–52.

44. Allanore Y, Devos-Francois G, Caramella C, Boumier P, Jounieaux V, Kahan A. Fatal exacerbation of fibrosing alveolitis associated with systemic sclerosis in a patient treated with adalimumab. Ann Rheum Dis 2006 Jun; 65(6): 834–835.

45. Ramos-Casals M, Perez-Alvarez R, Perez-de-Lis M, Xaubet A, Bosch X, Group BS. Pulmonary disorders induced by monoclonal antibodies in patients with rheumatologic autoimmune diseases. Amer J Med 2011 May; 124(5): 386–394.

46. Denton CP, Engelhart M, Tvede N, Wilson H, Khan K, Shiwen X, et al. An open-label pilot study of infliximab therapy in diffuse cutaneous systemic sclerosis. Ann Rheum Dis 2009 Sep; 68(9): 1433–1439.

47. Distler JH, Jordan S, Airo P, Alegre-Sancho JJ, Allanore Y, Balbir Gurman A, et al. Is there a role for TNFalpha antagonists in the treatment of SSc? EUSTAR expert consensus development using the Delphi technique. Clinical and experimental rheumatology. 2011 Mar-Apr; 29(2 Suppl 65): S40–45.

48. Lam GK, Hummers LK, Woods A, Wigley FM. Efficacy and safety of etanercept in the treatment of scleroderma-associated joint disease. J Rheumatol 2007 Jul; 34(7): 1636–1637.

49. Hasegawa M, Hamaguchi Y, Yanaba K, Bouaziz JD, Uchida J, Fujimoto M, et al. B-lymphocyte depletion reduces skin fibrosis and autoimmunity in the tight-skin mouse model for systemic sclerosis. Amer J Pathol 2006 Sep; 169(3): 954–966.

50. Komura K, Yanaba K, Horikawa M, Ogawa F, Fujimoto M, Tedder TF, et al. CD19 regulates the development of bleomycin-induced pulmonary fibrosis in a mouse model. Arth Rheumat 2008 Nov; 58(11): 3574–3584.

51. Bosello S, De Santis M, Lama G, Spano C, Angelucci C, Tolusso B, et al. B cell depletion in diffuse progressive systemic sclerosis: safety, skin score modification and IL-6 modulation in an up to thirty-six months follow-up open-label trial. Arth Res & Therapy 2010; 12(2): R54.

52. Daoussis D, Liossis SN, Tsamandas AC, Kalogeropoulou C, Kazantzi A, Sirinian C, et al. Experience with rituximab in scleroderma: results from a 1-year, proof-of-principle study. Rheumatology 2010 Feb; 49(2): 271–280.

53. Daoussis D, Liossis SN, Tsamandas AC, Kalogeropoulou C, Paliogianni F, Sirinian C, et al. Effect of long-term treatment with rituximab on pulmonary function and skin fibrosis in patients with diffuse systemic sclerosis. Clinic Experim Rheumat 2012 Mar-Apr; 30(2 Suppl 71): S17–22.

54. Lafyatis R, Kissin E, York M, Farina G, Viger K, Fritzler MJ, et al. B cell depletion with rituximab in patients with diffuse cutaneous systemic sclerosis. Arth Rheumat 2009 Feb; 60(2):578-583.

55. Smith V, Piette Y, van Praet JT, Decuman S, Deschepper E, Elewaut D, et al. Two-year results of an open pilot study of a 2-treatment course with rituximab in patients with early systemic sclerosis with diffuse skin involvement. J Rheumatol 2013 Jan; 40(1): 52–57.

56. Smith V, Van Praet JT, Vandooren B, Van der Cruyssen B, Naeyaert JM, Decuman S, et al. Rituximab in diffuse cutaneous systemic sclerosis: an open-label clinical and histopathological study. Ann Rheum Dis 2010 Jan; 69(1): 193–197.

57. Allanore Y, Saad M, Dieude P, Avouac J, Distler JH, Amouyel P, et al. Genome-wide scan identifies TNIP1, PSORS1C1, and RHOB as novel risk loci for systemic sclerosis. PLoS genetics. 2011 Jul; 7(7):e1002091.

58. Kadono T, Kikuchi K, Ihn H, Takehara K, Tamaki K. Increased production of interleukin 6 and interleukin 8 in scleroderma fibroblasts. J Rheumatol 1998 Feb; 25(2): 296–301.

59. Kawaguchi Y, Hara M, Wright TM. Endogenous IL-1alpha from systemic sclerosis fibroblasts induces IL-6 and PDGF-A. J Clinic Invest 1999 May; 103(9): 1253–1260.

60. Desallais L, Avouac J, Frechet M, Elhai M, Ratsimandresy R, Montes M, et al. Targeting IL-6 by both passive or active immunization strategies prevents bleomycin-induced skin fibrosis. Arth Res & Therapy 2014; 16(4): R157.

61. Kitaba S, Murota H, Terao M, Azukizawa H, Terabe F, Shima Y, et al. Blockade of interleukin-6 receptor alleviates disease in mouse model of scleroderma. Amer J Pathol 2012 Jan; 180(1): 165–176.

62. Le Huu D, Matsushita T, Jin G, Hamaguchi Y, Hasegawa M, Takehara K, et al. IL-6 blockade attenuates the development of murine sclerodermatous chronic graft-versus-host disease. J Invest Dermatol 2012 Dec; 132(12): 2752–2761.

63. Distler JH, Distler O. Imatinib as a novel therapeutic approach for fibrotic disorders. Rheumatology 2009 Jan; 48(1): 2–4.

64. Distler JH, Jungel A, Huber LC, Schulze-Horsel U, Zwerina J, Gay RE, et al. Imatinib mesylate reduces production of extracellular matrix and prevents development of experimental dermal fibrosis. Arth Rheumat 2007 Jan; 56(1): 311–322.

65. Akhmetshina A, Venalis P, Dees C, Busch N, Zwerina J, Schett G, et al. Treatment with imatinib prevents fibrosis in different preclinical models of systemic sclerosis and induces regression of established fibrosis. Arth Rheumat 2009 Jan; 60(1): 219–224.

66. Akhmetshina A, Dees C, Pileckyte M, Maurer B, Axmann R, Jungel A, et al. Dual inhibition of c-abl and PDGF receptor signaling by dasatinib and nilotinib for the treatment of dermal fibrosis. FASEB J. 2008 Jul; 22(7): 2214–2222.

67. Pope J, McBain D, Petrlich L, Watson S, Vanderhoek L, de Leon F, et al. Imatinib in active diffuse cutaneous systemic sclerosis: Results of a six-month, randomized, double-blind, placebo-controlled, proof-of-concept pilot study at a single center. Arth Rheumat 2011 Nov; 63(11): 3547–3551.

68. Prey S, Ezzedine K, Doussau A, Grandoulier AS, Barcat D, Chatelus E, et al. Imatinib mesylate in scleroderma-associated diffuse skin fibrosis: a phase II multicentre randomized double-blinded controlled trial. The British journal of dermatology. 2012 Nov; 167(5): 1138–1144.

69. Spiera RF, Gordon JK, Mersten JN, Magro CM, Mehta M, Wildman HF, et al. Imatinib mesylate (Gleevec) in the treatment of diffuse cutaneous systemic sclerosis: results of a 1-year, phase IIa, single-arm, open-label clinical trial. Ann Rheum Diseas 2011 Jun; 70(6): 1003–1009.

70. Shanmugam E, Jena A, George M. Riociguat: Something new in pulmonary hypertension therapeutics? J Pharmac & Pharmacotherapeutics 2015 Jan-Mar; 6(1): 3–6.

71. Beyer C, Reich N, Schindler SC, Akhmetshina A, Dees C, Tomcik M, et al. Stimulation of soluble guanylate cyclase reduces experimental dermal fibrosis. Ann Rheum Dis 2012 Jun; 71(6): 1019–1026.

72. Beyer C, Zenzmaier C, Palumbo-Zerr K, Mancuso R, Distler A, Dees C, et al. Stimulation of the soluble guanylate cyclase (sGC) inhibits fibrosis by blocking non-canonical TGFbeta signalling. Ann Rheum Dis 2014 Feb 23.

73. Dees C, Beyer C, Distler A, Soare A, Zhang Y, Palumbo-Zerr K, et al. Stimulators of soluble guanylate cyclase (sGC) inhibit experimental skin fibrosis of different aetiologies. Ann Rheum Dis 2015 Mar 27.

Štítky
Dermatology & STDs Paediatric rheumatology Rheumatology
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