Promising treatments of tomorrow for multiple sclerosis

Annals of Indian Academy of Neurology

Volumn 12, Issue 4, 2009, Pages 283-290

  Harrison, Daniel M ^a   Calabresi, Peter A ^a  

^a JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Drugs; Investigational; Multiple sclerosis; Therapeutics

Indexed keywords

ABATACEPT; ALEMTUZUMAB; ALPHA CRYSTALLIN; ATACICEPT; BETA1A INTERFERON; BG 00012; CARMUSTINE; CHIMERIC ANTIBODY; CLADRIBINE; CYCLOPHOSPHAMIDE; CYTARABINE; DACLIZUMAB; ETOPOSIDE; FINGOLIMOD; FUMARIC ACID DIMETHYL ESTER; GRANULOCYTE COLONY STIMULATING FACTOR; HIRUDIN; LAQUINIMOD; LEUCINE RICH REPEATS AND IG DOMAIN CONTAINING NEURITE OUTGROWTH INHIBITOR RECEPTOR INTERACTING PROTEIN 1; MELPHALAN; MYELIN ASSOCIATED GLYCOPROTEIN; NATALIZUMAB; OCRELIZUMAB; OSTEOPONTIN; PLACEBO; PLASMINOGEN ACTIVATOR INHIBITOR 3; POTASSIUM CHANNEL KV1.3; RITUXIMAB; ROQUINIMEX; TERIFLUNOMIDE; THROMBOPLASTIN; UNCLASSIFIED DRUG;

ADD ON THERAPY; AUTOIMMUNE DISEASE; BONE MARROW SUPPRESSION; BRAIN HEMORRHAGE; CARDIOTOXICITY; CENTRAL NERVOUS SYSTEM INFECTION; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; DRUG CLEARANCE; DRUG DEVELOPMENT; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; ENTEROCOLITIS; FLUSHING; FORCED EXPIRATORY VOLUME; GASTROINTESTINAL SYMPTOM; HEART INFARCTION; HEART RATE; HEMATOPOIETIC STEM CELL; HERPES VIRUS INFECTION; HERPES ZOSTER; HUMAN; IDIOPATHIC THROMBOCYTOPENIC PURPURA; IMMUNOMODULATION; INFLAMMATION; KIDNEY ALLOGRAFT REJECTION; KIDNEY FAILURE; LYMPHOMA; MULTIPLE SCLEROSIS; NEUROPROTECTION; NONHUMAN; PATIENT COMPLIANCE; POSTERIOR REVERSIBLE ENCEPHALOPATHY SYNDROME; PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY; PROTEOMICS; RANDOMIZED CONTROLLED TRIAL; RELAPSE; REVIEW; RHEUMATIC DISEASE; RHEUMATOID ARTHRITIS; SIDE EFFECT; THYROID DISEASE; TREATMENT OUTCOME; UNSPECIFIED SIDE EFFECT;

EID: 72949100481     PISSN: 09722327     EISSN: 19983549     Source Type: Journal    
DOI: 10.4103/0972-2327.58279     Document Type: Review

References (71)

1. Sibley WA; the IFN-β Multiple Sclerosis Study Group. Interferon beta-1b is effective in relapsing-remitting multiple sclerosis: I: Clinical results of a multicenter, randomized, double-blind, placebo-controlled trial. Neurology 1993;43:655-661
2. Johnson KP, Brooks BR, Cohen JA, Ford CC, Goldstein J, Lisak RP, et al. Copolymer 1 reduces relapse rate and improves disability in relapsing-remitting multiple sclerosis: Results of a phase III, multicenter, double-blind, placebo-controlled trial. Neurology 1995;45:1268-1276
3. Jacobs LD, Cookfair DL, Rudick RA, Herndon RM, Richert JR, Salazar AM, et al. Intramuscular interferon beta-1a for disease progression in relapsing multiple sclerosis: The Mutliple Sclerosis Collaborative Research Group (MSCRG). Ann Neurol 1996;39:285-294
4. Comi G, Filippi M, Barkhof F, Durelli L, Edan G, Fernandez O, et al. Effect of early interferon treatment on conversion to definite multiple sclerosis: A randomized study. Lancet 2001;357:1576-1582
5. Barkhof F, Polman CH, Radue EW, Kappos L, Freedman MS, Edan G, et al. Magnetic resonance imaging effects of interferon beta-1b in the BENEFIT study: Integrated 2-year results. Arch Neurol 2007;641292-641298
6. Polman CH, O.Connor PW, Havrdova E, Hutchinson M, Kappos L, Miller DH, et al. A randomized, placebo-controlled trial of natalizumab for relapsing multiple sclerosis. N Engl J Med 2006;354:899-910.
7. Yousry TA, Major EO, Ryschkewitsch C, Fahle G, Fischer S, Hou J, et al. Evaluation of patients treated with natalizumab for progressive multifocal leukoencephalopathy. N Engl J Med 2006;354:924-933
8. Reff ME, Carner K, Chambers KS, et al. Depletion of B cells in vivo by a chimeric mouse human monoclonal antibody to CD20. Blood 1994;83:435-445
9. Anderson DR, Grillo-Lopez A, Varns C, et al. Targeted anticancer therapy using rituximab, a chimaeric anti-CD20 antibody (IDECC2B8) in the treatment of non-Hodgkin's B-cell lymphoma. Biochem Soc Trans 1997;25:705-708
10. Bar-Or A, Calabresi PA, Arnold D, Markowitz C, Shafer S, Kasper LH, et al. Rituximab in relapsing-remitting multiple sclerosis: A 72 week, open label, phase I trial. Ann Neurol 2008;63:395-400.
11. Hauser SL, Waubant E, Arnold DL, Vollmer T, Antel J, Fox RJ, et al. B-cell depletion with rituximab in relapsing-remitting multiple sclerosis. N Engl J Med 2008; 358:676-688
12. Study of the efficacy and safety of ocrelizumab in patients with relapsing-remitting multiple sclerosis. Phase II Clinical Trial: Identifier # NCT00676715. Food and Drug Administration, 2008. Available from: http://www.clinicaltrial.gov/ct2/show/NCT00676 715?intr= %22ocrelizumab%22andrank=6. [accessed on 2009 Feb 23].
13. Kranick SM, Mowry EM, Rosenfeld MR. Progressive multifocal leukoencephalopathy after ritxumab in a case of non-hodgkin lymphoma. Neurology 2007;69:704-706
14. Harris HE. Progressive multifocal leukoencephalopathy in a patient with systemic lupus erythematosus treated with rituximab. Rheumatology 2008;47:224-225
15. Coles AJ, Cox A, Le Page E, Jones J, Trip SA, Deans J, et al. The window of therapeutic opportunity in multiple sclerosis: Evidence from monoclonal antibody therapy. J Neurol 2006;253:98-108.
16. Moreau T, Thorpe J, Miller D, Moseley I, Hale G, Waldmann H, et al. Preliminary evidence from magnetic resonance imaging for reduction in disease activity after lymphocyte depletion in multiple sclerosis. Lancet 1994;344:298-301. [Erratum, Lancet 1994;344:486.]
17. CAMMS223 Trial Investigators, Coles AJ, Compston DA, Selmaj KW, Lake SL, Moran S, et al. Alemtuzumab vs interferon beta-1a in early multiple sclerosis. N Engl J Med 2008;359:1786-1801
18. Clatworthy MR, Wallin EF, Jayne DR. Anti-glomerular basement membrane disease after alemtuzumab. N Engl J Med 2008;359:768-769
19. Cox AL, Thompson SA, Jones JL, Robertson VH, Hale G, Waldmann H, et al. Lymphocyte homeostasis following therapeutic lymphocyte depletion in multiple sclerosis. Eur J Immunol 2005;35:3332-3342
20. Clatworthy MR, Jayne DR. Acquired hemophilia in association with ANCA-associated vasculitis: Response to rituximab. Am J Kidney Dis 2006;47:680-682
21. Morris JC, Waldmann T. Advances in interleukin 2 receptor targeted treatment. Ann Rheum Dis 2000;59:i109-14.
22. Vincenti F, Kirkman R, Light S, Bumgardner G, Pescovitz M, Halloran P, et al. Interleukin-2-receptor blockade with daclizumab to prevent acute rejection in renal transplantation. N Engl J Med 1998;338:161-165 (Pubitemid 28041863)
23. Rose JW, Burns JB, Bjorklund J, Klein J, Watt HE, Carlson NG. Daclizumab phase II trial in relapsing and remitting multiple sclerosis. Neurology 2007;69:785-789
24. Ali EN, Healy BC, Stazzone LA, Brown BA, Weiner Hl, Khoury SJ. Daclizumab in treatment of multiple sclerosis patients. Multiple Sclerosis 2009;15:272-274
25. Viglietta V, Bourcier K, Buckle GJ, Healy B, Weiner HL, Hafl er A, et al. CTLA4Ig treatment in patients with multiple sclerosis: An open label, phase 1 clinical trial. Neurology 2008;71:917-924
26. Tak PP, Thurlings RM, Rossier C, Nestorov I, Dimic A, et al. Atacicept in patients with rheumatoid arthritis: Results of a multicenter, phaseIb, double-blind, placebo-controlled, doseescalating, single- and repeated-dose study. Arthritis Rheum 2008;58:61-72.
27. Dall.Era M, Chakravarty E, Wallace D, Genovese M, Weisman M, Kavanaugh A, et al. Reduced B lymphocyte and immunoglobulin levels after atacicept treatment in patients with systemic lupus erythematosus: Results of a multicenter, phase Ib, doubleblind, placebo-controlled, dose-escalating trial. Arthritis Rheum 2007;56:4142-4150
28. Atacicept in Multiple Sclerosis, Phase II. Phase II clinical trial: Identifier # NCT00642902. Food and drug administration, 2008. Available from: http://www.clinicaltrials.gov/ct2/show/NCT00642 902?term= atacicept+multiple+sclerosisandrank=1. [accessed on 2009 Feb 26].
29. Brinkmann V, Davis MD, Heise CE, Albert R, Cottens S, Hof R, et al. The immune modulator FTY720 targets sphingosine 1-phosphate receptors. J Biol Chem 2002;277:21453-21457
30. Matloubian M, Lo CG, Cinamon G, Lesneski MJ, Xu Y, Brinkmann V, et al. Lymphocyte egress from thymus and peripheral lymphoid organs is dependent on S1P receptor 1. Nature 2004;427:355-360
31. Mehling M, Brinkmann V, Antel J, Bar-Or A, Goebels N, Vedrine C, et al. FTY720 therapy exerts differential effects on t cell subsets in multiple sclerosis. Neurology 2008;71:1261-1267
32. Kappos L, Antel J, Comi G, Montalban X, O.Connor P, Polman CH, et al. Oral fingolimod (FTY720) for relapsing multiple sclerosis. N Engl J Med 2006;355:1124-1140
33. Stott VL, Hurrell MA, Anderson TJ. Reversible posterior leukoencephalopathy syndrome: A misnomer reviewed. Intern Med J 2005;35:83-90.
34. O'Connor P, Comi G, Montalban X, Antel J, Radue EW, de Vera A. Oral Fingolimod (FTY720) in multiple sclerosis: Two-year results of a phase II extension study. Neurology 2009;72:73-79
35. Press release: Oral FTY720 (Fingolimod) reduced relapse rate more effectively Than Avonex in initial results of one-year study in relapsing-remitting MS. Available from: http://www. nationalmssociety.org/ research/research-news/news-detail/index. aspx?nid=557 [accessed on 2009 Mar 5].
36. Efficacy and Safety of Fingolimod (FTY720) in Patients With Relapsing-Remitting Multiple Sclerosis (FREEDOMS II). Phase III clinical trial: Identifier # NCT00355134. Food and drug administration, 2006. Available from: http://www.clinicaltrials.gov/ ct2/show/NCT00355134?term=Fingolimodandrank=1. [accessed on 2009 Feb 23].
37. FTY720 in Patients With Primary Progressive Multiple Sclerosis (INFORMS). Phase III Clinical Trial: Identifier # NCT00731692. Food and drug administration, 2008. Available from: http://www. clinicaltrials.gov/ct2/show/ NCT00731692?term=Fingolimodandra nk=9. [accessed on 2009 Feb 23].
38. Beutler E. Cladribine (2-chlorodeoxyadenosine). Lancet 1992;340:952-956
39. Carson DA, Wasson DB, Taetle R, Yu A. Specific toxicity of 2-chlorodeoxyadenosine toward resting and proliferating human lymphocytes. Blood 1983;62:737-743
40. Beutler E, Koziol JA, McMillan R, Sipe JC, Romine JS, Carrera CJ. Marrow suppression produced by repeated doses of cladribine. Acta Haematol 1994;91:10-15
41. Oral Cladribine in Early Multiple Sclerosis (MS) (ORACLE MS). Phase III Clinical Trial: Identifier # NCT00725985. Food and Drug Administration, 2008. Available from: http://www.clinicaltrials.gov/ ct2/show/NCT00725985?term= cladribine+multiple+sclerosisand rank=1. [accessed on 2009 Feb 23].
42. Sipe JC, Romine JS, Koziol JA, McMillan R, Zyroff J, Beutler E. Cladribine in treatment of chronic progressive multiple sclerosis. Lancet 1994;344:9-13.
43. Rice GP, Filippi M, Comi G. Cladribine and progressive MS. Neurology 2000;54:1145-1155
44. Romine JS, Sipe JC, Koziol JA, Zyroff J, Beutler E. A doubleblind, placebo-controlled, randomized trial of cladribine in relapsing-remitting multiple sclerosis. Proc Assoc Am Physicians 1999;111:35-44.
45. Press release: Oral cladribine reduces MS Relapses in phase 3 clinical trial. Available from: http://www.nationalmssociety. org/research/research-news/ news-detail/index.aspx?nid=650. [accessed on 2009 Mar 5].
46. Tan IL, Lycklama à Nijeholt GJ, Polman CH, Adèr HJ, Barkhof F. Linomide in the treatment of multiple sclerosis: MRI results from prematurely terminated phase-III trials. Mult Scler 2000;6:99-104.
47. Yang JS, Ling-Yun X, Bao-Guo X, Hedlund G, Link H. Laquinimod (ABR-215062) supresses the development of experimental autoimmune encephalomyelitis, modulates the Th1/Th2 balance and induces the Th3 cytokine TGF-Beta in lewis rats. J Neuroimmunol 2004;156:3-9.
48. Zou LP, Abbas N, Volkmann I, Nennesmo I, Levi M, Wahren B, et al. Suppression of experimental autoimmune neuritis by ABR-215062 is associated with altered Th1/Th2 balance and inhibited migration of Inflammatory cells into the peripheral nerve tissue. Neuropharmacology 2002;42:731-739
49. Polman C, Barkhof F, Sandberg-Wollheim M, Linde A, Nordle O, Nederman T, et al. Treatment with laquinimod reduces development of active MRI lesions in relapsing MS. Neurology 2005;64:987-991
50. Comi G, Pulizzi A, Rovaris M, Abrams O, Arbizu T, Boiko A, et al. Effect of laquinimod on mri-monitored disease activity in patients with relapsing-remitting multiple sclerosis: A multicentre, randomised, double-blind, placebo-controlled phase IIb study. Lancet 2008;371:2085-2092
51. BRAVO Study: Laquinimod double blind placebo controlled study in RRMS patients with a rater blinded reference arm of interferon β-1a (Avonex® ). Phase III clinical trial: Identifier # NCT00605215. Available from: http://www.clinicaltrials.gov/ct2/show/NCT006052 15?term=laquinimodandrank=5. [accessed on 2009 Feb 24].
52. Cherwinski HM, Cohn RG, Cheung P, Webster DJ, Xu YZ, Caulfield JP, et al. The immunosuppressant leflunomide inhibits lymphocyte proliferation by inhibiting pyrimidine biosynthesis. J Pharmacol Exp Ther 1995;275:1043-1049
53. Korn T, Toyka K, Hartung HP, Jung S. Suppression of experimental autoimmune neuritis by leflunomide. Brain 2001;124:1791-1802
54. O.Connor PW, Li D, Freedman MS, Bar-Or A, Rice GP, Confavreux C. A phase II study of the safety and efficacy of teriflunomide in multiple sclerosis with relapses. Neurology 2006;66:894-900.
55. Phase III study with teriflunomide versus placebo in patients with First clinical symptom of multiple sclerosis. Phase III clinical trial: Identifier # NCT00622700. Available from: http://www.clinicaltrials. gov/ct2/show/ NCT00622700?term=teriflunomideandrank=1. [accessed on 2009 Feb 24].
56. Kappos L, Gold R, Miller DH, MacManus DG, Havrdova E, Limmroth V. Efficacy and safety of oral fumarate in patients with relapsing-remitting multiple sclerosis: A multicentre, randomised, double-blind, placebo-controlled phase IIb study. Lancet 2008;372:1463-1472
57. Efficacy and safety study of oral BG00012 with active reference in relapsing-remitting multiple sclerosis (CONFIRM). Phase III clinical trial: Identifier # NCT00451451. Available from: http:// www.clinicaltrials.gov/ct2/ show/NCT00451451?term=bg00012a ndrank=3 [accessed on 2009 Feb 24].
58. Mancardi GL, Saccardi R, Filippi M, Gualandi F, Murialdo A, Inglese M, et al. Autologous hematopoietic stem cell transplantation suppresses Gd-enhanced MRI activity in MS. Neurology 2001;57:62-68 (Pubitemid 32634851)
59. Fassas A, Passweg JR, Anagnostopoulos A, Kazis A, Kozac T, Havrdova E, et al. Hematopoietic stem cell transplantation for multiple sclerosis: A retrospective multicenter study. J Neurol 2002;249:1088-1097
60. Krishnan C, Kaplin AI, Brodsky RA, Drachman DB, Jones RJ, Pham DL, et al. Reduction of disease activity with high-dose cyclophosphamide in patients with aggressive multiple sclerosis. Arch Neurol 2008;65:1044-1051
61. Gladstone DE, Zamkoff KW, Krupp L, Peyster R, Sibony P, Christodoulou C. High-dose cyclophosphamide for moderate to severe refractory multiple sclerosis. Arch Neurol 2006;63:1388-1393
62. Steinman L. New targets for treatment of multiple sclerosis: 13th annual charcot foundation lecture. J Neurol Sci 2008;274:1-4.
63. Hur E, Youssef S, Haws M, Zhang S, Sobel R, Steinman L. Osteopontin induced relapse and progression of autoimmune brain disease via enhanced survival of activated T cells. Nat Immunol 2007;8:77-86.
64. Ransohoff RM. Inflammatory disease: assault on the guardian. Nature 2007;448:421-422
65. Han MH, Hwang S, Roy DB, Lundgren DH, Price JV, Ousman S, et al. Proteomic analysis of active multiple sclerosis lesions reveals therapeutic targets. Nature 2008;451:1076-1081
66. Wulff H, Beeton C, Chandy KG. Potassium channels as therapeutic targets for autoimmune disorders. Curr Opin Drug Discov Devel 2003;6:640-647
67. Hu L, Pennington M, Jiang Q, Whartenby KA, Calabresi PA. Characterization of the functional properties of the voltage-gated potassium channel Kv1.3 in human CD4+ T lymphocytes. J Immunol 2007;179:4563-4570
68. Nguyen T, Mehta NR, Conant K, Kim KJ, Jones M, Calabresi PA. Axonal protective effects of the myelin-associated glycoprotein. J Neurosci 2009;29:630-637
69. Mi S, Miller RH, Lee X, Scott ML, Shulag-Morskaya S, Shao Z, et al. LINGO-1 negatively regulates myelination by oligodendrocytes. Nat Neurosci 2005;8:745-751
70. Kuhlmann T, Miron V, Cuo Q, Wegner C, Antel J, Brück W. Differentiation block of oligodendroglial progenitor cells as a cause for remyelination failure in chronic multiple sclerosis. Brain 2008;131:1749-1758
71. Rudick RA, Mi S, Sandrock AW Jr. LINGO-1 antagonists as therapy for multiple sclerosis: in vitro and in vivo evidence. Expert Opin Biol Ther 2008;8:1561-1570