Perspectives from B cell immunology: Fact and fancy

International Journal of Clinical Pharmacology and Therapeutics

Volumn 47, Issue SUPPL. 1, 2009, Pages

  Hunt, S V ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

Author keywords

Antibodies; Biomarkers; Efficacy; Enzyme replacement therapy; Lysosomal storage diseases

Indexed keywords

ANTIBODY; BIOLOGICAL MARKER;

ANIMAL; ANTIGEN ANTIBODY REACTION; B LYMPHOCYTE; CHEMICAL STRUCTURE; ENZYME REPLACEMENT; GENE THERAPY; GENETICS; HUMAN; IMMEDIATE TYPE HYPERSENSITIVITY; IMMUNE SYSTEM; IMMUNOLOGICAL TOLERANCE; IMMUNOLOGY; LYSOSOME STORAGE DISEASE; METABOLISM; METHODOLOGY; REVIEW;

ANIMALS; ANTIBODIES; ANTIGEN-ANTIBODY REACTIONS; B-LYMPHOCYTES; BIOLOGICAL MARKERS; ENZYME REPLACEMENT THERAPY; GENE THERAPY; HUMANS; HYPERSENSITIVITY, IMMEDIATE; IMMUNE SYSTEM; IMMUNE TOLERANCE; LYSOSOMAL STORAGE DISEASES; MODELS, MOLECULAR;

EID: 71849097532     PISSN: 09461965     EISSN: None     Source Type: Journal    
DOI: 10.5414/cpp47086     Document Type: Conference Paper

References (89)

1. Aerts JM, Groener JE, Kuiper S, Donker-Koopman WE, Strijland A, Ottenhoff R et al. Elevated globotriaosylsphingosine is a hallmark of Fabry disease. Proc Natl Acad Sci USA. 2008a; 105: 2812-2817. (Pubitemid 351723626)
2. Aerts JM, van Breemen MJ, Bussink AP, Ghauharali K, Sprenger R, Boot RG et al. Biomarkers for lysosomal storage disorders: identification and application as exemplified by chitotriosidase in Gaucher disease. Acta Paediatr. 2008b; 97 (Suppl 457): 7-14. (Pubitemid 351421284)
3. Ainsworth MA, Bendtzen K, Brynskov J. Tumor necrosis factor-α binding capacity and anti-infliximab antibodies measured by fluid-phase radioimmunoassays as predictors of clinical efficacy of infliximab in Crohn's disease. Am J Gastroenterol. 2008; 103: 944-948. (Pubitemid 351501078)
4. Andersson HC, Charrow J, Kaplan P, Mistry P, Pastores GM, Prakash-Cheng A et al. Individualization of long-term enzyme replacement therapy for Gaucher disease. Genet Med. 2005; 7: 105-110. (Pubitemid 40261835)
5. Arnon R, Shapira E. Antibodies to papain. A selective fractionation according to inhibitory capacity. Biochemistry. 1967; 6: 3942-3950.
6. Astermark J, Lacroix-Desmazes S, Reding MT. Inhibitor development. Haemophilia. 2008; 14 (Suppl 3): 36-42. (Pubitemid 351722381)
7. Baert F, Noman M, Vermeire S, Van Assche G, D'Haens G, Carbonez A et al. Influence of immunogenicity on the long-term efficacy of infliximab in Crohn's disease. N Engl J Med. 2003; 348: 601-608. (Pubitemid 36204954)
8. Barbey F, Lidove O, Schwarting A. Fabry nephropathy: 5 years of enzyme replacement therapy - a short review. Nephrol Dial Transplant Plus. 2008; 1: 11-19. (Pubitemid 351308810)
9. Baxter AG, Hodgkin PD. Activation rules: the two-signal theories of immune activation. Nat Rev Immunol. 2002; 2: 439-446. (Pubitemid 37328751)
10. Bénichou B, Goyal S, Sung C, Norfleet AM, O'Brien F. A retrospective analysis of the potential impact of IgG antibodies to agalsidase beta on efficacy during enzyme replacement therapy for Fabry disease. Mol Genet Metab. 2009; 96: 4-12.
11. Bennett CL, Cournoyer D, Carson KR, Rossert J, Luminari S, Evens AM et al. Long-term outcome of individuals with pure red cell aplasia and antierythropoietin antibodies in patients treated with recombinant epoetin: a follow-up report from the Research on Adverse Drug Events and Reports (RADAR) Project. Blood. 2005; 106: 3343-3347. (Pubitemid 41609162)
12. Bodensteiner D, Scott CR, Sims KB, Shepherd GM, Cintron RD, Germain DP. Successful reinstitution of agalsidase beta therapy in Fabry disease patients with previous IgE-antibody or skin-test reactivity to the recombinant enzyme. Genet Med. 2008; 10: 353-358. (Pubitemid 351770406)
13. Brooks DA, Kakavanos R, Hopwood JJ. Significance of immune response to enzyme-replacement therapy for patients with a lysosomal storage disorder.Trends Mol Med. 2003; 9: 450-453. (Pubitemid 37309647)
14. Burrow TA, Hopkin RJ, Leslie ND, Tinkle BT, Grabowski GA. Enzyme reconstitution/replacement therapy for lysosomal storage diseases. Curr Opin Pediatr. 2007; 19: 628-635. (Pubitemid 350145229)
15. +T cells in tolerance to the transgene product following hepatic in vivo gene transfer. Blood. 2007; 110: 1132-1140.
16. Castaneda JA, Lim MJ, Cooper JD, Pearce DA. Immune system irregularities in lysosomal storage disorders. Acta Neuropathol. 2008; 115: 159-174.
17. Chen CS, Patterson MC, Wheatley CL, O'Brien JF, Pagano RE. Broad screening test for sphingolipidstorage diseases. Lancet. 1999; 354: 901-905. (Pubitemid 29424020)
18. Cox TM. Biomarkers in lysosomal storage diseases: a review. Acta Paediatr. 2005; 94 (Suppl 447): 39-42.
19. De Groot AS, Scott DW. Immunogenicity of protein therapeutics. Trends Immunol. 2007; 28: 482-490. (Pubitemid 350087812)
20. Deegan PB, Moran MT, McFarlane I, Schofield JP, Boot RG, Aerts JM et al. Clinical evaluation of chemokine and enzymatic biomarkers of Gaucher disease. Blood Cells Mol Dis. 2005; 35: 259-267. (Pubitemid 41267058)
21. DiMichele D. Inhibitors: resolving diagnostic and therapeutic dilemmas. Haemophilia. 2002; 8: 280-287.
22. DiMichele D. The North American Immune Tolerance Registry: contributions to the thirty-year experience with immune tolerance therapy. Haemophilia. 2009; 15: 320-328.
23. DiMichele D, Hoots WK, Pipe SW, Rivard GE, Santagostino E. International workshop on immune tolerance induction: consensus recommendations. Haemophilia. 2007; 13 (Suppl 1): 1-22. (Pubitemid 47009242)
24. Dresser DW. Specific inhibition of antibody production. II. Paralysis induced in adult mice by small quantities of protein antigen. Immunology. 1962; 5: 378-388.
25. Eng CM, Guffon N, Wilcox WR, Germain DP, Lee P, Waldek S et al. Safety and efficacy of recombinant human α-galactosidase A: replacement therapy in Fabry's disease. N Engl J Med. 2001; 345: 9-16. (Pubitemid 32634266)
26. + T-cell anergy. Nat Rev Immunol. 2007; 7: 599- 609.
27. Finkelman FD. Anaphylaxis: lessons from mouse models. J Allergy Clin Immunol. 2007; 120: 506-515. (Pubitemid 47334841)
28. Gell PGH, Coombs RRA. Clinical aspects of immunology. Oxford: Blackwells Scientific Publications; 1963.
29. Grabowski GA. Phenotype, diagnosis, and treatment of Gaucher's disease. Lancet. 2008; 372: 1263- 1271.
30. Grabowski GA, Kacena K, Cole JA, Hollak CE, Zhang L, Yee J et al. Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease Type 1. Genet Med. 2009; 11: 92-100.
31. Graca L, Chen TC, Le Moine A, Cobbold SP, Howie D, Waldmann H. Dominant tolerance: activation thresholds for peripheral generation of regulatory T cells. Trends Immunol. 2005; 26: 130-135. (Pubitemid 40312519)
32. Harmatz P, Giugliani R, Schwartz I, Guffon N, Leão Teles E, Miranda MC et al. Enzyme replacement therapy for mucopolysaccharidosis VI: a Phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. J Pediatr. 2006; 148: 533-539.
33. Hay CR, Brown S, Collins PW, Keeling DM, Liesner R. The diagnosis and management of Factor VIII and IX inhibitors: a guideline from the United Kingdom Haemophilia Centre Doctors Organisation. Br J Haematol. 2006; 133: 591-605. (Pubitemid 43742773)
34. Hermeling S, Crommelin DJ, Schellekens H, Jiskoot W. Structure- immunogenicity relationships of therapeutic proteins. Pharm Res. 2004; 21: 897-903. (Pubitemid 38870136)
35. Hogquist KA, Baldwin TA, Jameson SC. Central tolerance: learning self-control in the thymus. Nat Rev Immunol. 2005; 5: 772-782. (Pubitemid 41400851)
36. Hollak CE, Linthorst GE. Immune response to enzyme replacement therapy in Fabry disease: impact on clinical outcome? Mol Genet Metab. 2009; 96: 1-3.
37. Howard JG, Mitchison NA. Immunological tolerance. Prog Allergy. 1975; 18: 43-96.
38. Hughes DA. Early therapeutic intervention in females with Fabry disease? Acta Paediatr. 2008; 97 (Suppl 457): 41-47. (Pubitemid 351421292)
39. Hughes DA, Elliott PM, Shah J, Zuckerman J, Coghlan G, Brookes J et al. Effects of enzyme replacement therapy on the cardiomyopathy of Anderson-Fabry disease: a randomised, double-blind, placebo-controlled clinical trial of agalsidase alfa. Heart. 2008; 94: 153-158. (Pubitemid 351211719)
40. Humphrey JH, White RG. Immunology for students of medicine. Oxford & Edinburgh: Blackwells Scientific Publications; 1970.
41. Kakavanos R, Lehn P, Callebaut I, Meikle PJ, Parkinson-Lawrence EJ, Hopwood JJ et al. Common antigenicity for two glycosidases. FEBS Lett. 2006; 580: 87-92. (Pubitemid 43005318)
42. (2+) homeostasis in lysosomal storage diseases. Cell Calcium. 2008; 44: 103-111.
43. Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL et al. Recombinant human acid α-glucosidase: major clinical benefits in infantile-onset Pompe disease. Neurology. 2007; 68: 99-109. (Pubitemid 46071892)
44. Kruse-Jarres R, Barnett B, Leissinger C. Immune tolerance induction for the eradication of inhibitors in patients with hemophilia A. Expert Opin Biol Ther. 2008; 8: 1885-1896.
45. Lachmann RH, te Vruchte D, Lloyd-Evans E, Reinkensmeier G, Sillence DJ, Fernandez-Guillen L et al. Treatment with miglustat reverses the lipid-trafficking defect in Niemann-Pick disease Type C. Neurobiol Dis. 2004; 16: 654-658. (Pubitemid 38942998)
46. Lavigne-Lissalde G, Schved JF, Granier C, Villard S. Anti-Factor VIII antibodies: a 2005 update. Thromb Haemost. 2005; 94: 760-769. (Pubitemid 41489234)
47. Lidove O, Joly D, Barbey F, Bekri S, Alexandra JF, Peigne V et al. Clinical results of enzyme replacement therapy in Fabry disease: a comprehensive review of literature. Int J Clin Pract. 2007; 61: 293-302.
48. Lieberman P, Kemp SF, Oppenheimer J, Lang DM, Bernstein DI, Nicklas RA et al. The diagnosis andmanagement of anaphylaxis: an updated practice parameter. J Allergy Clin Immunol. 2005; 115: S483-S523. (Pubitemid 40327251)
49. Linthorst GE, Hollak CE, Donker-Koopman WE, Strijland A, Aerts JM. Enzyme therapy for Fabry disease: neutralizing antibodies toward agalsidase alfa and beta. Kidney Int. 2004; 66: 1589-1595. (Pubitemid 39298394)
50. Liou B, Kazimierczuk A, Zhang M, Scott CR, Hegde RS, Grabowski GA. Analyses of variant acid beta-glucosidases: effects of Gaucher disease mutations. J Biol Chem. 2006; 281: 4242-4253. (Pubitemid 43847853)
51. Matzinger P. The danger model: a renewed sense of self. Science. 2002; 296: 301-305. (Pubitemid 34303674)
52. Matzner U, Matthes F, Weigelt C, Andersson C, Eistrup C, Fogh J et al. Non-inhibitory antibodies impede lysosomal storage reduction during enzyme replacement therapy of a lysosomal storage disease. J Mol Med. 2008; 86: 433-442.
53. Mehta A, Beck M, Kampmann C, Frustaci A, Germain DP, Pastores GM et al. Enzyme replacement therapy in Fabry disease: comparison of agalsidase alfa and agalsidase beta. Mol Genet Metab. 2008; 95: 114-115.
54. Mendelsohn NJ, Messinger YH, Rosenberg AS, Kishnani PS. Elimination of antibodies to recombinant enzyme in Pompe's disease. N Engl J Med. 2009; 360: 194- 195.
55. Mitchison NA. Induction of immunological paralysis in two zones of dosage. Proc R Soc Lond B Biol Sci. 1964; 161: 275-292.
56. Mu TW, Fowler DM, Kelly JW. Partial restoration of mutant enzyme homeostasis in three distinct lysosomal storage disease cell lines by altering calcium homeostasis. PLoS Biol. 2008; 6: e26.
57. Muenzer J, Wraith JE, Beck M, Giugliani R, Harmatz P, Eng CM et al. A Phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet Med. 2006; 8: 465-473.
58. Nimmerjahn F, Ravetch JV. Fcγ receptors as regulators of immuneresponses. Nat Rev Immunol.2008; 8: 34-47.
59. Ohashi T, Sakuma M, Kitagawa T, Suzuki K, Ishige N, Eto Y. Influence of antibody formation on reduction of globotriaosylceramide (GL-3) in urine from Fabry patients during agalsidase beta therapy. Mol Genet Metab. 2007; 92: 271-273. (Pubitemid 47513559)
60. Pauling L. A theory of the structure and process of formation of antibodies. J Am Chem Soc. 1940; 62: 2643-2657.
61. Ponce E, Moskovitz J, Grabowski G. Enzyme therapy in Gaucher disease Type 1: effect of neutralizing antibodies to acid beta-glucosidase. Blood. 1997; 90: 43-48. (Pubitemid 27276545)
62. Ponder KP. Immune response hinders therapy for lysosomal storage diseases. J Clin Invest. 2008; 118: 2686-2689.
63. Rajewsky K. Clonal selection and learning in the antibody system. Nature. 1996; 381: 751-758. (Pubitemid 26243107)
64. Reipert BM, van Helden PM, Schwarz HP, Hausl C. Mechanisms of action of immune tolerance induction against Factor VIII in patients with congenital haemophilia A and Factor VIII inhibitors. Br J Haematol. 2007; 136: 12-25. (Pubitemid 44912584)
65. Richards SM. Immunologic considerations for enzyme replacement therapy in the treatment of lysosomal storage disorders. Clinical and Applied Immunology Reviews. 2002; 2: 241-253. (Pubitemid 35439108)
66. Richards SM, Olson TA, McPherson JM. Antibody response in patients with Gaucher disease after repeated infusion with macrophage-targeted glucocerebrosidase. Blood. 1993; 82: 1402-1409. (Pubitemid 23263954)
67. Ries M, Clarke JT, Whybra C, Mehta A, Loveday KS, Brady RO et al. Enzyme replacement in Fabry disease: pharmacokinetics and pharmacodynamics of agalsidase alfa in children and adolescents. J Clin Pharmacol. 2007; 47: 1222-1230. (Pubitemid 47459666)
68. Rohrbach M, Clarke JT. Treatment of lysosomal storage disorders: progress with enzyme replacement therapy. Drugs. 2007; 67: 2697-2716. (Pubitemid 350224380)
69. Rosenberg M, Kingma W, Fitzpatrick MA, Richards SM. Immunosurveillance of alglucerase enzyme therapy for Gaucher patients: induction of humoral tolerance in seroconverted patients after repeat administration. Blood. 1999; 93: 2081-2088. (Pubitemid 29128512)
70. Sandin S, Ofverstedt LG, Wikstrom AC, Wrange O, Skoglund U. Structure and flexibility of individual immunoglobulin G molecules in solution. Structure. 2004; 12: 409-415. (Pubitemid 38353062)
71. Settembre C, Fraldi A, Jahreiss L, Spampanato C, Venturi C, Medina D et al. A block of autophagy in lysosomal storage disorders. Hum Mol Genet. 2008; 17: 119-129.
72. Singh NJ, Schwartz RH. Primer: mechanisms of immunologic tolerance. Nat Clin Pract Rheumatol. 2006; 2: 44-52. (Pubitemid 43153648)
73. Starzyk K, Richards S, Yee J, Smith SE, Kingma W. The long-term international safety experience of imiglucerase therapy for Gaucher disease. Mol Genet Metab. 2007; 90: 157-163. (Pubitemid 46108612)
74. Sun B, Bird A, Young SP, Kishnani PS, Chen YT, Koeberl DD. Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance. Am J Hum Genet. 2007; 81: 1042-1049. (Pubitemid 47580255)
75. + regulatory T cell: a jack of all trades, master of regulation. Nat Immunol. 2008; 9: 239-244.
76. Urban DJ, Zheng W, Goker-Alpan O, Jadhav A, Lamarca ME, Inglese J et al. Optimization and validation of two miniaturized glucocerebrosidase enzyme assays for high throughput screening. Comb Chem High Throughput Screen. 2008; 11: 817-824.
77. Van den Hout JM, Kamphoven JH, Winkel LP, Arts WF, De Klerk JB, Loonen MC et al. Long-term intravenous treatment of Pompe disease with recombinant human α-glucosidase from milk. Pediatrics. 2004; 113: e448- e457.
78. van der Ploeg AT, Reuser AJ. Pompe's disease. Lancet. 2008; 372: 1342-1353.
79. Vedder AC, Breunig F, Donker-Koopman WE, Mills K, Young E, Winchester B et al. Treatment of Fabry disease with different dosing regimens of agalsidase: effects on antibody formation and GL-3. Mol Genet Metab. 2008; 94: 319-325.
80. Waldmann H, Adams E, Cobbold S. Reprogramming the immune system: co-receptor blockade as a paradigm for harnessing tolerance mechanisms. Immunol Rev. 2008; 223: 361-370. (Pubitemid 351986177)
81. Wang J, Lozier J, Johnson G, Kirshner S, Verthelyi D, Pariser A et al. Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatment. Nat Biotechnol. 2008; 26: 901-908.
82. Watanabe M, Uchida K, Nakagaki K, Kanazawa H, Trapnell BC, Hoshino Y et al. Anti-cytokine autoantibodies are ubiquitous in healthy individuals. FEBS Lett. 2007; 581: 2017-2021. (Pubitemid 46670127)
83. Wilcox WR, Banikazemi M, Guffon N, Waldek S, Lee P, Linthorst GE et al. Long-term safety and efficacy of enzyme replacement therapy for Fabry disease. Am J Hum Genet. 2004; 75: 65-74.
84. Wraith JE, Beck M, Lane R, van der Ploeg A, Shapiro E, Xue Y et al. Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human α-L-iduronidase (laronidase). Pediatrics. 2007; 120: e37-e46. (Pubitemid 47036214)
85. Wraith JE, Scarpa M, Beck M, Bodamer OA, De Meirleir L, Guffon N et al. Mucopolysaccharidosis Type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. Eur J Pediatr. 2008a; 167: 267-277.
86. Wraith JE, Tylki-Szymanska A, Guffon N, Lien YH, Tsimaratos M, Vellodi A et al. Safety and efficacy of enzyme replacement therapy with agalsidase beta: an international, open-label study in pediatric patients with Fabry disease. J Pediatr. 2008b; 152: 563-570.
87. Zarate YA, Hopkin RJ. Fabry's disease. Lancet. 2008; 372: 1427-1435.
88. Zhao H, Keddache M, Bailey L, Arnold G, Grabowski G. Gaucher's disease: identification of novel mutant alleles and genotype-phenotype relationships. Clin Genet. 2003; 64: 57-64. (Pubitemid 36949950)
89. Ziegler RJ, Cherry M, Barbon CM, Li C, Bercury SD, Armentano D et al. Correction of the biochemical and functional deficits in Fabry mice following AAV8- mediated hepatic expression of α-galactosidase A. Mol Ther. 2007; 15: 492-500.