Immune response to enzyme replacement therapy in lysosomal storage disorder patients and animal models

Molecular Genetics and Metabolism

Volumn 68, Issue 2, 1999, Pages 268-275

  Brooks, Doug A ^a  

^a WOMEN S AND CHILDREN S HOSPITAL   (Australia)

Author keywords

Antibody; Enzyme replacement therapy; Epitope reactivity; Immune reaction; Intracellular targeting; Lysosomal storage disease; Treatment outcome

Indexed keywords

CYCLOPHOSPHAMIDE; GLUCOSYLCERAMIDASE; IMMUNOGLOBULIN G; SULFATASE;

ANAPHYLAXIS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIBODY PRODUCTION; CONTROLLED STUDY; ENZYME DEFICIENCY; ENZYME INACTIVATION; ENZYME METABOLISM; ENZYME REPLACEMENT; GAUCHER DISEASE; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; HURLER SYNDROME; HYPERSENSITIVITY; IMMUNE RESPONSE; IMMUNOSUPPRESSIVE TREATMENT; LYSOSOME STORAGE DISEASE; MUCOPOLYSACCHARIDOSIS; NONHUMAN; PLASMAPHERESIS; PRIORITY JOURNAL; SHORT SURVEY;

ANIMALIA;

EID: 0032737067     PISSN: 10967192     EISSN: None     Source Type: Journal    
DOI: 10.1006/mgme.1999.2894     Document Type: Article

References (40)

1. Neufeld E F, Meunzer J. The Mucopolysaccharidoses. Scriver C R, Beaudet A L, Sly W S, Valle D. The Metabolic Basis of Inherited Disease. 1995;2465-2494 McGraw-Hill, New York.
2. Meikle P J, Hopwood J J, Clague A E, Carey W F. Prevalence of lysosomal storage disorders. J Am Med Assoc. 281:1999;249-254.
3. Pastores G M, Sibille A R, Grabowski G A. Enzyme therapy in Gaucher disease type I: Dosage efficacy and adverse effects in 33 patients treated for 6 to 24 months. Blood. 82:1993;408-416.
4. Berkhan O. Zwei falle von skaphokephalie. Arch Anthropol. 34:1907;8.
5. Hers H G. Alpha-glucosidase deficiency in generalised glycogen-storage disease (Pompe's Disease). Biochem J. 86:1963;11.
6. Hers H G. Inborn lysosomal diseases. Gastroenterology. 48:1965;625-633.
7. Hers H G, van Hoof F. The genetic pathology of lysosomes. Prog Liver Dis. 3:1970;185-205.
8. De Duve C, Wattiaux R. Functions of lysosomes. Annu Rev Physiol. 28:1966;435-492.
9. De Duve C. Lysosomes revisited. Eur J Biochem. 137:1983;391-397.
10. Hopwood J J, Brooks D A. An introduction to the basic science and biology of the lysosome and storage disease. Applegarth D A, Dimmick J E, Hall J G. Organelle Diseases. 1997;7-35 Chapman & Hall, London.
11. De Duve C. From cytases to lysosomes. Fed Proc. 23:1964;1045-1054.
12. Neufeld E F. Replacement of genotype specific proteins in mucopolysaccharidosis enzyme therapy in genetic disease. Desnick R J, Bernlohr R W, Krivit W. Birth Defects, March of Dimes Original Series. 1973;27-30.
13. Sands M S, Vogler C, Kyle J W, Grubb J H, Levy B, Sly W S, Birkenmeier E H. Enzyme replacement therapy for murine mucopolysaccharidosis type VII. J Clin Invest. 93:1994;2324-2331.
14. Sands M S, Vogler C, Torrey A, Levy B, Gwynn B, Grubb J, Sly W S, Birkenmeier E H. Murine mucopolysaccharidosis type VII: Long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation. J Clin Invest. 99:1997;1596-1605.
15. Shull R M, Kakkis E D, McEntee M F, Kania S A, Jonas A J, Neufeld E F. Enzyme replacement in a canine model of Hurler syndrome. Proc Natl Acad Sci USA. 91:1994;12937-12941.
16. Clarke L A, Russell C S, Pownall S, Warrington C L, Borowski A, Dimmick J E, Toone J, Jirik F R. Murine mucopolysaccharidosis type I: Targeted disruption of the murine α-L-iduronidase gene. Hum Mol Genet. 6:1997;503-511.
17. Clarke, L, A, Russell, C, Brooks, D, A, Matlock, T, Gibson, S, Pourbahrami, T, Jevon, G, Hendson, G, Hopwood, J, J, Enzyme replacement in a murine model of MPS I. Abstract from the 5th International Symposium on the Mucopolysaccharidoses, Vienna, Austria, 1999.
18. Crawley A C, Brooks D A, Muller V J, Petersen B A, Isaac E L, Bielicki J, King B M, Boulter C D, Moore A J, Fazzalari N L, Anson D S, Byers S, Hopwood J J. Enzyme replacement therapy in a feline model of Maroteaux-Lamy syndrome. J Clin Invest. 97:1996;1864-1873.
19. Crawley A C, Neidzielski K H, Isaac E L, Davey R C, Byers S, Hopwood J J. Enzyme replacement therapy from birth in a feline model of mucopolysaccharidosis type VI. J Clin Invest. 99:1997;651-662.
20. Barton N W, Brady R O, Dambrosia J M, Di-Bisceglie A M, Dopplet S H, Hill S C, Mankin H J, Murray G J, Parker R I, Argoff C E, Grewal R P, Yu K. Replacement therapy for inherited enzyme deficiency: Macrophage-targeted glucocerebrosidase for Gaucher's disease. N Engl J Med. 324:1991;1464-1470.
21. Barton N W, Brady R O, Dambrosia J M, Doppelt S H, Hill S C, Holder C A, Mankin H J, Murray G J, Zirzow G C, Parker R I. Dose-dependent response to macrophage-targeted glucocerebrosidase in a child with Gaucher disease. J Pediatr. 120:1992;277-280.
22. Pastores G M, Sibille A R, Grabowski G A. Enzyme therapy in Gaucher Disease Type I: Dosage efficacy and adverse effects in 33 patients treated for 6 to 24 months. Blood. 82:1993;408-416.
23. Rosenthal D I, Doppelt S H, Mankin H J, Dambrosia J M, Xavier R J, McKusick K A, Rosen B R, Baker J, Niklason L T, Hill S C, Miller S PF, Brady R O, Barton N W. Enzyme replacement therapy for Gaucher disease: skeletal response to macrophage-targeted glucocerebrosidase. Pediatrics. 96:1995;629-637.
24. Brady R O. Gaucher's disease: Past, present and future. Bailliere's Clin Haematol. 10:1997;621-634.
25. Beutler E. Enzyme replacement therapy for Gaucher's disease. Bailliere's Clin Haematol. 10:1997;751-763.
26. Kakkis, E, Muenzer, J, Tiller, G, Waber, L, Belmont, J, Passage, M, Izykowski, B, Phillips, J, Walot, I, Doroshow, R, Hoft, R, Yu, K, T, Okazaki, S, Lewis, D, Lachman, R, Neufeld, E, F, Recombinant alpha-L-iduronidase replacement therapy in mucopolysaccharidosis I: Results of a human clinical trial. Published abstract No. 128, 48th annual meeting, American Society for Human Genetics, Denver, 1998.
27. Brooks D A. Review: Immunochemical analysis of lysosomal enzymes in mucopolysaccharidosis type I and type VI patients. J Inherit Metab Dis. 16:1993;3-15.
28. Brooks D A, King B M, Crawley A C, Byers S, Hopwood J J. Enzyme replacement therapy in Mucopolysaccharidosis VI: Evidence for immune responses and altered efficacy of treatment in animal models. Biochem Biophys Acta. 1361:1997;203-216.
29. Brooks D A, Hopwood J J, King B M. Immune response to enzyme replacement therapy: Clinical signs of hypersensitivity reactions and altered enzyme distribution in a high titre rat model. Biochem Biophys Acta. 1407:1998;163-172.
30. Peters, C, Enzyme substitution therapy of a mucopolysaccharidosis VI mouse model. Abstract, 7th International Congress of Inborn Errors of Metabolism, Vienna, Austria, 1997.
31. Kakkis E D, McEntee M F, Schmidtchen A, Neufeld E F, Ward D A, Gompf R E, Kania S, Bedolla C, Chien S L, Shull R M. Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I. Biochem Mol Med. 58:1996;156-167.
32. Grabowski G A. Current issues in enzyme replacement therapy for Gaucher disease. Drugs. 52:1996;159-167.
33. Brady R O, Murray G J, Oliver K L, Leitman S F, Sneller M C, Fleisher T A, Barton N W. Management of neutralizing antibody to ceredase in a patient with type 3 Gaucher disease. Pediatrics. 100:1997;111-114.
34. Ponce E, Moskovitz J, Grabowski G. Enzyme therapy in Gaucher disease type I: Effect of neutralizing antibodies to acid β-glucosidase. Blood. 90:1997;43-48.
35. Rosenberg M, Kingma W, Fitzpatrick M A, Richards S M. Immunosurveillance of aglucerase enzyme therapy for Gaucher patients: Induction of humoral tolerance in seroconverted patients after repeated administration. Blood. 93:1999;2081-2088.
36. Turner C T, Hopwood J J, Bond C S, Brooks D A. Immune response to enzyme replacement therapy: 4-Sulphatase epitope reactivity of plasma antibodies from MPS VI cats. Mol Genet Metab. 67:1999;194-205.
37. Wilson C, Kay M A. Immunomodulation to enhance gene therapy. Nature Med. 1:1995;887-889.
38. Potter M A, Hymus S, Stockley T, Chang P L. Suppression of immunological response against a transgene product delivered from microencapsulated cells. Hum Gene Ther. 9:1998;1275-1282.
39. Amigorena S, Drake J R, Webster P, Mellman I. Transient accumulation of new class II MHC molecules in a novel endocytic compartment in B lymphocytes. Nature. 369:1994;113-120.
40. Drake J R, Webster P, Cambier J C, Mellman I. Delivery of B cell receptor-internalised antigen to endosomes and class II vesicles. J Exp Med. 186:1997;1299-1306.