Halofuginone to treat fibrosis in chronic graft-versus-host disease and scleroderma

Biology of Blood and Marrow Transplantation

Volumn 9, Issue 7, 2003, Pages 417-425

  Pines, Mark ^a   Snyder, David ^b   Yarkoni, Shai ^b   Nagler, Arnon ^c  

^a VOLCANI CENTER   (Israel)

^b Medica L P   (Israel)

^c SHEBA MEDICAL CENTER   (Israel)

Author keywords

Collagen; Smad; Tsk

Indexed keywords

CLOFAZIMINE; COLLAGEN TYPE 1; CORTICOSTEROID; CYCLOFENIL; CYCLOSPORIN; ETRETINATE; HALOFUGINONE; HYDROXYCHLOROQUINE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; NEDOCROMIL SODIUM; PENICILLAMINE; RAPAMYCIN; SMAD3 PROTEIN; TACROLIMUS; THALIDOMIDE; TRANSFORMING GROWTH FACTOR BETA; URSODEOXYCHOLIC ACID; PIPERIDINE DERIVATIVE; PROTEIN SYNTHESIS INHIBITOR; QUINAZOLINE DERIVATIVE; QUINAZOLINONE DERIVATIVE;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTICLE; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; BONE MARROW TRANSPLANTATION; COLLAGEN SYNTHESIS; CONNECTIVE TISSUE; CORRELATION ANALYSIS; DISEASE COURSE; DISEASE MODEL; DISEASE SEVERITY; DRUG BLOOD LEVEL; DRUG EXPOSURE; DRUG MECHANISM; DRUG TOLERABILITY; EXTRACELLULAR MATRIX; GASTROINTESTINAL DISEASE; GRAFT VERSUS HOST REACTION; HEART MUSCLE FIBROSIS; HUMAN; KIDNEY FIBROSIS; LIVER FIBROSIS; LUNG FIBROSIS; LYMPH NODE IRRADIATION; NAUSEA AND VOMITING; NONHUMAN; PERIPHERAL BLOOD STEM CELL TRANSPLANTATION; PHOTOTHERAPY; PILOT STUDY; PREDICTION; PROMYELOCYTIC LEUKEMIA; PROTEIN PHOSPHORYLATION; PUVA; SCORING SYSTEM; SKIN FIBROSIS; SYSTEMIC SCLEROSIS; TISSUE INJURY; TISSUE STRUCTURE; TREATMENT OUTCOME; ANIMAL; DRUG EFFECT; FIBROSIS; INTRADERMAL DRUG ADMINISTRATION; METABOLISM; MOUSE; PATHOLOGY; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; REVIEW;

ADMINISTRATION, CUTANEOUS; ANIMALS; CLINICAL TRIALS, PHASE I; CLINICAL TRIALS, PHASE II; COLLAGEN TYPE I; FIBROSIS; GRAFT VS HOST DISEASE; HUMANS; MICE; PIPERIDINES; PROTEIN SYNTHESIS INHIBITORS; QUINAZOLINES; QUINAZOLINONES; SCLERODERMA, SYSTEMIC;

EID: 1442271244     PISSN: 10838791     EISSN: None     Source Type: Journal    
DOI: 10.1016/S1083-8791(03)00151-4     Document Type: Article

References (77)

1. Pines M. Vlodavsky I. Nagler A. Halofuginone: a novel anti-fibrotic therapy Drug of the Future 21 1996 569-599
2. Pines M. Vlodavsky I. Nagler A. Halofuginone: a novel anti-fibrotic therapy Gen Pharmacol. 30 1997 445-450
3. Pines M. Vlodavsky I. Nagler A. Halofuginone: from veterinary use to human therapy Drug Develop Res. 50 2000 371-378
4. Benyon R.C. Arthur M.J. Extracellular matrix degradation and the role of hepatic stellate cells Semin Liver Dis. 21 2001 373-384
5. Ravanti L. Kahari V.M. Matrix metalloproteinases in wound repair Int J Mol Med. 6 2000 391-407
6. Rodemann H.P. Bamberg M. Cellular basis of radiation-induced fibrosis Radiother Oncol. 35 1995 83-90
7. Rodemann H.P. Binder A. Burger A. Guven N. Loffler H. Bamberg M. The underlying cellular mechanism of fibrosis Kidney Int Suppl. 54 1996 S32-S36
8. Friedman S.L. The cellular basis of hepatic fibrosis. Mechanisms and treatment strategies N Engl J Med. 328 1993 1828-1835
9. Haber P.S. Keogh G.W. Apte M.V. et al. Activation of pancreatic stellate cells in human and experimental pancreatic fibrosis Am J Pathol. 155 1999 1087-1095
10. Kissin E . Korn J.H. Apoptosis and myofibroblasts in the pathogenesis of systemic sclerosis Curr Rheumatol Rep. 4 2002 129-135
11. Safadi R. Friedman S.L. Hepatic fibrosis: role of hepatic stellate cell activation Med Gen Med. 4 2002 27
12. Schmitz N. Beksac M. Hasenclever D. et al. Transplantation of mobilized peripheral blood cells to HLA-identical siblings with standard-risk leukemia Blood 100 2002 761-767
13. Beatty P.G. Hansen J.A. Longton G.M. et al. Marrow transplantation from HLA-matched unrelated donors for treatment of hematologic malignancies Transplantation 2 1991 443-447
14. Storek J. Gooley T. Siadak M. et al. Allogeneic peripheral blood stem cell transplantation may be associated with a high risk of chronic graft-versus-host-disease Blood 12 1997 4705-4709
15. Rocha V. Wagner Jr. JE. Sobocinski K.A. et al. Graft-versus-host disease in children who have received a cord-blood or bone marrow transplant from an HLA-identical sibling: Eurocord and International Bone Marrow Transplant Registry Working Committee on Alternative Donor and Stem Cell Sources N Engl J Med. 342 2000 1846-1854
16. Aversa F. Tabilio A. Velardi A. et al. Treatment of high-risk acute leukemia with T cell depleted stem cells from related donors with one fully mismatched HLA haplotype N Engl J Med. 339 1998 1186-1193
17. Sullivan K.M. Shulman H.M. Storb R. Chronic graft versus host disease in 52 patients: adverse natural course and successful treatment with combination immunosuppression Blood 57 1981 267-276
18. Shimoni A. Nagler A. Non-myeloablative stem cell transplantation (NST): chimerism testing as guidance for immune-therapeutic manipulation Leukemia 15 2001 1967-1975
19. Shulman H.M. Sale G.E. Lerner K.G. et al. Chronic cutaneous graft-versus-host disease in man Am J Pathol. 91 1978 545-570
20. Janin-Mercier A. Devergie A. Van Cauwenberge D. et al. Immunohistologic and ultrastructural study of the sclerotic skin in chronic graft versus host disease in man Am J Pathol. 115 1984 296-306
21. Nagler A. Pines M. Abadi U. et al. Oral tolerization ameliorated liver disorders associated with chronic graft versus host disease in mice Hepatology. 13 2000 641-648
22. Ilan Y. Gotsman I. Pines M. et al. Induction of oral tolerance in splenocytes recipients towards pre-transplant splenocyte antigens ameliorates chronic graft versus host disease in murine model Blood 95 2000 3613-3619
23. Gaziev D. Galimberti M. Lucarelli G. Polchi P. Chronic graft versus host disease: is there an alternative to the conventional treatment? Bone Marrow Transplant. 25 2000 689-696
24. Sapadin A.N. Fleischmajer R. Treatment of scleroderma Arch Dermatol. 138 2002 99-105
25. Haustein U.F. Systemic sclerosis-scleroderma Dermatol Online J. 8 2002 3-73
26. Trojanowska M. Molecular aspects of scleroderma Front Biosci. 7 2002 d608-d618
27. Kahari V.M. Activation of dermal connective tissue in scleroderma Ann Intern Med. 25 1994 511-518
28. Tyndall A. Haematopoietic stem cell transplantation (HSCT) for severe autoimmune disease: an update of results and planned trials Arthritis Rheum. 43 2000 1999
29. Clements P.J. Furst D.E. Choosing appropriate patients with systemic sclerosis for treatment by autologous stem cell transplantation J Rheumatol. 48 1997 85-88
30. Denton C.P. Black C.M. Novel therapeutic strategies in scleroderma Cur Rheumatol Rep. 1 1999 22-27
31. Kikuchi H. Tasaka H. Hirai S. et al. Potent antimalarial febrifugine analogues against the plasmodium malaria parasite J Med Chem. 45 2002 2563-2570
32. McDougal L.R. Control of coccidiosis chemotherapy, in Long P.L. (Ed.), Coccidiosis of man and domestic animals 1990 307-320 CRC PressBoca Raton, FL
33. Granot I. Hurwitz S. Halevy O. Pines M. Halofuginone: an inhibitor of collagen type I synthesis Biochim Biophys Acta. 1156 1993 107-112
34. Choi E.T. Callow A.D. Sehgal N.L. Brown D.M. Ryan U.S. Halofuginone, a specific collagen type I inhibitor, reduces anastomotic intima hyperplasia Arch Surg. 130 1995 257-261
35. Nagler A. Miao H.Q. Eingorn H. et al. Inhibition of collagen synthesis, smooth muscle cell proliferation and injury-induced intimal hyperplasia by halofuginone Arterio Thromb Vascular Biol. 17 1997 194-202
36. Bruck R. Genina O. Aeed H. Alexiev R. Nagler A. Pines M. Halofuginone to prevent and treat thioacetamide-induced liver fibrosis in rats Hepatology. 33 2001 379-386
37. Nagler A. Rivkind A.I. Raphael J. et al. Halofuginone: an inhibitor of collagen type I synthesis: prevents post-operation abdominal adhesions formation Ann Surg. 227 1998 575-582
38. Nagler A. Genina O. Lavelin I. Ohana M. Pines M. Halofuginone: an inhibitor of collagen type I synthesis: prevents formation of post-operative adhesions formation in the rat uterine horn model Am J Obstet Gyn. 180 1999 558-563
39. Nagler A. Gofrit O. Ohana M. Pode D. Genina O. Pines M. The effect of halofuginone, an inhibitor of collagen type I synthesis, on urethral stricture formation in vivo and in vitro study in a rat model J Urol. 164 2000 1776-1780
40. Pines M. Knopov V. Genina O. Lavelin I. Nagler A. Halofuginone, a specific inhibitor of collagen type I synthesis, prevents dimethylnitrosamine-induced liver cirrhosis J Hepatol. 27 1997 391-398
41. Nagler A. Firman N. Feferman R. Cotev S. Pines M. Shoshan S. Reduction in pulmonary fibrosis in vivo by halofuginone Am J Resp Crit Care Med. 154 1996 1082-1086
42. Levi-Schaffer F. Nagler A. Slavin S. Knopov V. Pines M. Inhibition of collagen synthesis and changes in skin morphology in murine graft-versus-host disease and tight skin mice: effect of halofuginone J Invest Dermatol. 106 1996 84-88
43. Pines M. Domb A. Ohana M. et al. Reduction in dermal fibrosis in the tight-skin (Tsk) mouse after local application of halofuginone Biochem Pharmacol. 62 2001 1221-1227
44. McGaha T.L. Phelps R.G. Spiera H. Bona C. Halofuginone, an inhibitor of type-I collagen synthesis and skin sclerosis, blocks transforming-growth-factor-beta-mediated Smad3 activation in fibroblasts J Invest Dermatol. 118 2002 461-470
45. Halevy O. Nagler A. Levi-Schaffer F. Genina O. Pines M. Inhibition of collagen type I synthesis by skin fibroblasts of graft versus host disease and scleroderma patients: effect of halofuginone Biochem Pharmacol. 52 1996 1057-1063
46. Zhang Y. Gilliam A.C. Animal models for scleroderma: an update Cur Rheumatol Rep. 4 2002 150-162
47. Bocchieri M.H. Jimenez S.A. Animal models of fibrosis Rheum Dis Clin North Am. 16 1990 153-167
48. Jaffee B.D. Claman H.N. Chronic graft-versus-host disease (GVHD) as a model for scleroderma: I. Description of model systems Cell Immunol. 77 1983 1-12
49. Siracusa L.D. McGarth R. Ma Q. et al. A tandem duplication within the fibrillin 1 gene is associated with the mouse tight skin mutation Genome Res. 6 1996 300-313
50. Green M.C. Sweet H.O. Bunker L.E. Tight skin, a new mutation of the mouse causing excessive growth of the connective tissue and skeleton Am J Pathol. 82 1975 493-512
51. Osborn T.G. Bashey R.I. Moore T. Fischer V.W. Collagenous abnormalities in the heart of tight-skin mouse J Mol Cell Cardiol. 19 1987 581-587
52. Jimenez S.A. Williams C.J. Myers J.C. Bashey R.I. Increased collagen biosynthesis and increased expression of type I and type III procollagen genes in tight skin (Tsk) mouse fibroblasts J Biol Chem. 261 1986 657-662
53. Pablos J.L. Everett E.T. Harley R. LeRoy E.C. Norris J.S. Transforming growth factor-β and collagen gene expression during postnatal skin development and fibrosis in tight-skin mouse Lab Invest. 72 1995 670-678
54. Siracusa L.D. Christner P. McGrath R. Mowers S.D. Nelson K.K. Jimenez S.A. The tight skin (Tsk) mutation in the mouse, a model for human fibrotic diseases, is tightly linked to the beta 2-microglobulin (B2m) gene on chromosome 2 Genomics. 17 1993 748-751
55. McGaha T. Kodera T. Phelps R. Spiera H. Pines M. Bona C. Effect of halofuginone on the development of tight skin (TSK) syndrome Autoimmunity. 35 2002 277-282
56. Chen W. Wahl S.M. Manipulation of TGF-beta to control autoimmune and chronic inflammatory diseases Microbes Infect. 1 1999 1367-1380
57. Scharffetter K. Lankat-Buttgereit B. Krieg T. Localization of collagen mRNA in normal and scleroderma skin by in situ hybridization Eur J Clin Invest. 19 1988 9-17
58. Jelaska A. Arakawa M. Broketa G. Korn J.H. Heterogeneity of collagen synthesis in normal and systemic sclerosis skin fibroblasts: Increase proportion of high collagen-producing cells in systemic sclerosis fibroblasts Arthritis Rheum. 39 1996 1338-1346
59. Needelman B.W. Ordonez J.V. Taramelli D. Alms W. Gayer K. Choi J. In vitro identification of a subpopulation of fibroblasts that produces high levels of collagen in scleroderma patients Arthritis Rheum. 33 1990 842-852
60. Verrecchia F. Mauviel A. Transforming growth factor-beta signaling through the Smad pathway: role in extracellular matrix gene expression and regulation J Invest Dermatol. 118 2002 211-215
61. Ghosh A.K. Factors involved in the regulation of type I collagen gene expression: implication in fibrosis Exp Biol Med (Maywood) 227 2002 301-314
62. Kulozik M. Hogg A. Lankat-Buttgereit B. et al. Co-localization of transforming growth factor beta 2 with alpha 1(I) procollagen mRNA in tissue sections of patients with systemic sclerosis J Clin Invest. 86 1990 917-922
63. Sfikakis P.P. McCune B.K. Tsokos M. et al. Immunohistological demonstration of transforming growth factor-beta isoforms in the skin of patients with systemic sclerosis Clin Immunol Immunopathol. 69 1993 199-204
64. Zhang Y. McCormick L.L. Desai S.R. et al. Murine sclerodermatous graft-versus-host disease, a model for human scleroderma: cutaneous cytokines, chemokines, and immune cell activation J Immunol. 168 2002 3088-3098
65. Holweg C.T. Baan C.C. Balk A.H. et al. The transforming growth factor-beta1 codon 10 gene polymorphism and accelerated graft vascular disease after clinical heart transplantation Transplantation 71 2001 1463-1467
66. Leffell M.S. Vogelsang G.B. Lucas D.P. et al. Association between TGF-beta expression and severe GvHD in allogeneic bone marrow transplantation Transplant Proc. 33 2001 485-486
67. Verrecchia F. Chu M.L. Mauviel A. Identification of novel TGF-beta/Smad gene targets in dermal fibroblasts using a combined cDNA microarray/promoter transactivation approach J Biol Chem. 276 2001 17058-17062
68. Chen K. Wei Y. Sharp G.C. Braley-Mullen H. Inhibition of TGF beta 1 by anti-TGF beta 1 antibody or lisinopril reduces thyroid fibrosis in granulomatous experimental autoimmune thyroiditis J Immunol. 169 2002 6530-6538
69. Zhang W. Ou J. Inagaki Y. Greenwel P. Ramirez F. Synergistic cooperation between Sp1 and Smad3/Smad4 mediates transforming growth factor beta1 stimulation of alpha 2(I)-collagen (COL1A2) transcription J Biol Chem. 275 2000 39237-39245
70. Varga J. Scleroderma and Smads: dysfunctional Smad family dynamics culminating in fibrosis Arthritis Rheum. 46 2002 1703-1713
71. Prud'homme G.J. Piccirillo C.A. The inhibitory effects of transforming growth factor-beta-1 (TGF-beta1) in autoimmune diseases J Autoimmun. 14 2000 23-42
72. Mallat Z. Gojova A. Marchiol-Fournigault C. et al. Inhibition of transforming growth factor-beta signaling accelerates atherosclerosis and induces an unstable plaque phenotype in mice Circ Res. 89 2001 930-934
73. Chen W. Wahl S.M. Manipulation of TGF-beta to control autoimmune and chronic inflammatory diseases Microbes Infect. 1 1999 1367-1380
74. Kawakami T. Ihn H. Xu W. Smith E. LeRoy C. Trojanowska M. Increased expression of TGF-beta receptors by scleroderma fibroblasts: evidence for contribution of autocrine TGF-beta signaling to scleroderma phenotype J Invest Dermatol. 110 1998 47-51
75. Spira G. Mawasi N. Paizi M. et al. Halofuginone, a collagen type I inhibitor improves liver regeneration in cirrhotic rats J Hepatol. 37 2002 331-339
76. Nagler A. Pines M. Topical treatment of cutaneous chronic graft versus host disease (cGvHD) with halofuginone: a novel inhibitor of collagen type I synthesis Transplantation 68 1999 1806-1809
77. Silman A. Harrison M. Brennan P. Is it possible to reduce observer variability in skin score assessment of scleroderma? The ad hoc International Group on the Assessment of Disease Outcome in Scleroderma J Rheumatol. 22 1995 1277-1280