Debio-025 is more effective than prednisone in reducing muscular pathology in mdx mice

Neuromuscular Disorders

Volumn 20, Issue 11, 2010, Pages 753-760

  Wissing, Erin R ^a,b   Millay, Douglas P ^a   Vuagniaux, Grégoire ^c   Molkentin, Jeffery D ^a  

^a UNIVERSITY OF CINCINNATI   (United States)

^b UNIVERSITY OF CINCINNATI COLLEGE OF MEDICINE   (United States)

^c DEBIOPHARM SA   (Switzerland)

Author keywords

Mouse models; Muscular dystrophy; Necrosis

Indexed keywords

CREATINE KINASE; CYCLOSPORIN A [3 N METHYL DEXTRO ALANINE 4 N ETHYLVALINE]; PREDNISONE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL INFILTRATION; CONTROLLED STUDY; CREATINE KINASE BLOOD LEVEL; DRUG EFFECT; DRUG EFFICACY; GRIP STRENGTH; MACROPHAGE ACTIVATION; MALE; MOUSE; MUSCLE DISEASE; MUSCULAR DYSTROPHY; MYOFIBROSIS; NONHUMAN; PRIORITY JOURNAL; TREATMENT DURATION; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE;

ANALYSIS OF VARIANCE; ANIMALS; CYCLOSPORINE; HAND STRENGTH; IMMUNOHISTOCHEMISTRY; MALE; MICE; MICE, INBRED MDX; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, ANIMAL; PREDNISONE; TREATMENT OUTCOME;

EID: 77957752614     PISSN: 09608966     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.nmd.2010.06.016     Document Type: Article

References (32)

1. Durbeej M., Campbell K.P. Muscular dystrophies involving the dystrophin-glycoprotein complex: an overview of current mouse models. Curr Opin Genet Dev 2002, 12:349-361.
2. Petrof B.J., Shrager J.B., Stedman H.H., Kelly A.M., Sweeney H.L. Dystrophin protects the sarcolemma from stresses developed during muscle contraction. Proc Natl Acad Sci U S A 1993, 90:3710-3714.
3. Biggar W.D., Klamut H.J., Demacio P.C., Stevens D.J., Ray P.N. Duchenne muscular dystrophy: current knowledge, treatment, and future prospects. Clin Orthop Relat Res 2002, 401:88-106.
4. Lapidos K.A., Kakkar R., Mcnally E.M. The dystrophin-glycoprotein complex: signaling strength and integrity for the sarcolemma. Circ Res 2004, 94:1023-1031.
5. Hopf F.W., Turner P.R., Steinhardt R.A. Calcium misregulation and the pathogenesis of muscular dystrophy. Subcell Biochem 2007, 45:429-464.
6. Whitehead N.P., Yeung E.W., Allen D.G. Muscle damage in mdx (dystrophic) mice. Role of calcium and reactive oxygen species. Clin Exp Pharmacol Physiol 2006, 33:657-662.
7. Turner P.R., Westwood T., Regen C.M., Steinhardt R.A. Increased protein degradation results from elevated free calcium levels found in muscle from mdx mice. Nature 1988, 335:735-738.
8. + channels. Proc Natl Acad Sci U S A 2000, 97:4950-4955.
9. 2+ transients in isolated muscle fibres from normal and dystrophic mice. J Physiol 1993, 469:11-19.
10. 2+ determination in resting muscle fibres from normal and dystrophic (mdx) mice. Cell Calcium 1993, 14:473-483.
11. Bernardi P. Mitochondrial transport of cations: channels, exchangers, and permeability transition. Physiol Rev 1999, 79:1127-1155.
12. Zamzami N., Kroemer G. The mitochondrion in apoptosis: how Pandora's box opens. Nat Rev Mol Cell Biol 2001, 2:67-71.
13. Halestrap A.P., Davidson A.M. Inhibition of Ca2(+)-induced large-amplitude swelling of liver and heart mitochondria by cyclosporin is probably caused by the inhibitor binding to mitochondrial-matrix peptidyl-prolyl cis-trans isomerase and preventing it interacting with the adenine nucleotide translocase. Biochem J 1990, 268:153-160.
14. Mcguinness O., Yafei N., Costi A., Crompton M. The presence of two classes of high-affinity cyclosporin A binding sites in mitochondria. Evidence that the minor component is involved in the opening of an inner-membrane Ca(2+)-dependent pore. Eur J Biochem 1990, 194:671-679.
15. Hansson M.J., Mattiasson G., Månsson R., et al. The nonimmunosuppressive cyclosporin analogs NIM811 and UNIL025 display nanomolar potencies on permeability transition in brain-derived mitochondria. J Bioenerg Biomembr 2004, 36:407-413.
16. Millay D.P., Sargent M.A., Osinska H., et al. Genetic and pharmacologic inhibition of mitochondrial-dependent necrosis attenuates muscular dystrophy. Nat Med 2008, 14:442-447.
17. Reutenauer J., Dorchies O.M., Patthey-Vuadens O., Vuagniaux G., Ruegg U.T. Investigation of Debio 025, a cyclophilin inhibitor, in the dystrophic mdx mouse, a model for Duchenne muscular dystrophy. Br J Pharmacol 2008, 155:574-584.
18. Tiepolo T., Angelin A., Palma E., et al. The cyclophilin inhibitor Debio 025 normalizes mitochondrial function, muscle apoptosis and ultrastructural defects in Col6a1(-/-) myopathic mice. Br J Pharmacol. 2009, 157:1045-1052.
19. De Luca A., Nico B., Liantonio A., et al. A multidisciplinary evaluation of the effectiveness of cyclosporine a in dystrophic mdx mice. Am J Pathol 2005, 166:477-489.
20. Merlini L., Angelin A., Tiepolo T., et al. Cyclosporin A corrects mitochondrial dysfunction and muscle apoptosis in patients with collagen VI myopathies. Proc Natl Acad Sci U S A. 2008, 105:5225-5229.
21. Hicks D., Lampe A.K., Laval S.H., et al. Cyclosporine A treatment for Ullrich congenital muscular dystrophy: a cellular study of mitochondrial dysfunction and its rescue. Brain 2009, 132:147-155.
22. Sharma K.R., Mynhier M.A., Miller R.G. Cyclosporine increases muscular force generation in Duchenne muscular dystrophy. Neurology 1993, 43:527-532.
23. Stupka N., Gregorevic P., Plant D.R., Lynch G.S. The calcineurin signal transduction pathway is essential for successful muscle regeneration in mdx dystrophic mice. Acta Neuropathol (Berl) 2004, 107:299-310.
24. Friday B.B., Horsley V., Pavlath G.K. Calcineurin activity is required for the initiation of skeletal muscle differentiation. J Cell Biol 2000, 149:657-666.
25. Muntoni F., Fisher I., Morgan J.E., Abraham D. Steroids in Duchenne muscular dystrophy: from clinical trials to genomic research. Neuromuscul Disord 2002, 12(Suppl 1):S162-S165.
26. Angelini C. The role of corticosteroids in muscular dystrophy: a critical appraisal. Muscle Nerve 2007, 36:424-435.
27. Balaban B., Matthews D.J., Clayton G.H., Carry T. Corticosteroid treatment and functional improvement in Duchenne muscular dystrophy: long-term effect. Am J Phys Med Rehabil 2005, 84:843-850.
28. Bianchi M.L., Mazzanti A., Galbiati E., et al. Bone mineral density and bone metabolism in Duchenne muscular dystrophy. Osteoporos Int 2003, 14:761-767.
29. Parsons S.A., Millay D.P., Sargent M.A., Mcnally E.M., Molkentin J.D. Age-dependent effect of myostatin blockade on disease severity in a murine model of limb-girdle muscular dystrophy. Am J Pathol 2006, 168:1975-1985.
30. Jacobs S.C., Bootsma A.L., Willems P.W., Bär P.R., Wokke J.H. Prednisone can protect against exercise-induced muscle damage. J Neurol 1996, 243:410-416.
31. Anderson J.E., Weber M., Vargas C. Deflazacort increases laminin expression and myogenic repair, and induces early persistent functional gain in mdx mouse muscular dystrophy. Cell Transplant 2000, 9:551-564.
32. St-Pierre S.J., Chakkalakal J.V., Kolodziejczyk S.M., Knudson J.C., Jasmin B.J., Megeney L.A. Glucocorticoid treatment alleviates dystrophic myofiber pathology by activation of the calcineurin/NF-AT pathway. FASEB J 2004, 18:1937-1939.