Development of a panel of highly sensitive, equivalent assays for detection of antibody responses to velaglucerase alfa or imiglucerase enzyme replacement therapy in patients with Gaucher disease

Journal of Immunological Methods

Volumn 373, Issue 1-2, 2011, Pages 45-53

  Séllos Moura, Márcia ^a   Barzegar, Samad ^a   Pan, Luying ^a   Shi, Philip ^a   Oommen, Susan ^a   Durant, Judith ^a   Ruiz, Juan A ^a  

^a Shire Human Genetic Therapies ^*  (United States)

Author keywords

Antibody; Assay; Gaucher disease; Imiglucerase; Velaglucerase alfa

Indexed keywords

ENZYME; ENZYME ANTIBODY; GLUCOSYLCERAMIDASE; IMIGLUCERASE ENZYME; IMMUNOGLOBULIN A; IMMUNOGLOBULIN E; IMMUNOGLOBULIN G ANTIBODY; IMMUNOGLOBULIN M; NEUTRALIZING ANTIBODY; UNCLASSIFIED DRUG; VELAGLUCERASE ALFA;

ANTIBODY AFFINITY; ANTIBODY RESPONSE; ARTICLE; BINDING KINETICS; CALIBRATOR; CHEMOLUMINESCENCE; CLINICAL ARTICLE; CONTROLLED STUDY; ENZYME ACTIVITY; ENZYME REPLACEMENT; GAUCHER DISEASE; HUMAN; HYDROLYSIS; PRIORITY JOURNAL; RADIOIMMUNOPRECIPITATION;

ANIMALS; ANTIBODIES; ELECTROCHEMICAL TECHNIQUES; ENZYME REPLACEMENT THERAPY; GAUCHER DISEASE; GLUCOSYLCERAMIDASE; HUMANS; IMMUNOASSAY; IMMUNOGLOBULIN A; IMMUNOGLOBULIN E; IMMUNOGLOBULIN G; IMMUNOGLOBULIN M; LUMINESCENT MEASUREMENTS; MICE; RADIOIMMUNOPRECIPITATION ASSAY; REPRODUCIBILITY OF RESULTS;

OVIS ARIES;

EID: 80054834248     PISSN: 00221759     EISSN: 18727905     Source Type: Journal    
DOI: 10.1016/j.jim.2011.07.020     Document Type: Article

References (20)

1. Barton N.W., Brady R.O., Dambrosia J.M., Di Bisceglie A.M., Doppelt S.H., Hill S.C., Mankin H.J., Murray G.J., Parker R.I., Argoff C.E., Grewal R.P., Yu K.-T. Replacement therapy for inherited enzyme deficiency - macrophage-targeted glucocerebrosidase for Gaucher's disease. N. Engl. J. Med. 1991, 324:1464.
2. 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 1997, 100:E11.
3. Brumshtein B., Salinas P., Peterson B., Chan V., Silman I., Sussman J.L., Savickas P.J., Robinson G.S., Futerman A.H. Characterization of gene-activated human acid-beta-glucosidase: crystal structure, glycan composition, and internalization into macrophages. Glycobiology 2010, 20:24.
4. EMEA Committee for Medicinal Products for Human Use (CHMP); Draft Guideline on Validation of Bioanalytical Methods. Ref: EMA/CHMP/EWP/192217/2009. Web Hyperlink: Guideline on Validation of Bioanalytical Methods (Draft) Available at:, Accessed January 11, 2011. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/12/WC500018062.pdf.
5. FDA Center for Drug Evaluation and Research. Guidance for Industry: Bioanalytical Methods Validation. Web Hyperlink: Guidance for Industry: Bioanalytical Method Validation Available at:, Accessed January 13, 2011. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM070107.pdf.
6. Goker-Alpan O. Optimal therapy in Gaucher disease. Ther. Clin. Risk Manag. 2010, 6:315.
7. Grabowski G.A., Barton N.W., Pastores G., Dambrosia J.M., Banerjee T.K., McKee M.A., Parker C., Schiffmann R., Hill S.C., Brady R.O. Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources. Ann. Intern. Med. 1995, 122:33.
8. Gu M.L., Feng S.L., Glenn J.K. Development of an animal-human antibody complex for use as a control in ELISA. J. Pharm. Biomed. Anal. 2003, 32:523.
9. ICH International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use. ICH Harmonised Tripartite Guideline: Validation of Analytical Procedures: Text and Methodology: Q2(R1); November 2005. Web Hyperlink: Q2(R1): Validation of Analytical Procedures: Text and Methodology Available at:, Accessed January 13, 2011. http://private.ich.org/LOB/media/MEDIA417.pdf.
10. Liang M., Klakamp S.L., Funelas C., Lu H., Lam B., Herl C., Umble A., Drake A.W., Pak M., Ageyeva N., Pasumarthi R., Roskos L.K. Detection of high- and low-affinity antibodies against a human monoclonal antibody using various technology platforms. Assay Drug Dev. Technol. 2007, 5:655.
11. Meso Scale Discovery Available at:, Accessed December 22, 2010. http://www.meso-scale.com/CatalogSystemWeb/WebRoot/products/plates.aspx;.
12. Miller K.J., Bowsher R.R., Celniker A., Gibbons J., Gupta S., Lee J.W., Swanson S.J., Smith W.C., Weiner R.S. Workshop on bioanalytical methods validation for macromolecules: summary report. Pharm. Res. 2001, 18:1373.
13. Mire-Sluis A.R., Barrett Y.C., Devanarayan V., Koren E., Liu H., Maia M., Parish T., Scott G., Shankar G., Shores E., Swanson S.J., Taniguchi G., Wierda D., Zuckerman L.A. Recommendations for the design and optimization of immunoassays used in the detection of host antibodies against biotechnology products. J. Immunol. Methods 2004, 289:1.
14. Patten P.A., Schellekens H. The immunogenicity of biopharmaceuticals. Lessons learned and consequences for protein drug development. Dev. Biol. (Basel) 2003, 112:81.
15. 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.
16. Richards S.M., Olson T.A., McPherson J.M. Antibody response in patients with Gaucher disease after repeated infusion with macrophage-targeted glucocerebrosidase. Blood 1993, 82:1402.
17. Rosenberg M., Kingma W., Fitzpatrick M.A., Richards S.M. Immunosurveillance of alglucerase enzyme therapy for Gaucher patients: induction of humoral tolerance in seroconverted patients after repeat administration. Blood 1999, 93:2081.
18. Schellekens H. When biotech proteins go off-patent. Trends Biotechnol. 2004, 22:406.
19. Starzyk K., Richards S., Yee J., Smith S.E., Kingma W. The long-term international safety experience of imiglucerase therapy for Gaucher disease. Mol. Genet. Metab. 2007, 90:157.
20. Zhao H., Bailey L.A., Grabowski G.A. Enzyme therapy of Gaucher disease: clinical and biochemical changes during production of and tolerization for neutralizing antibodies. Blood Cells Mol. Dis. 2003, 30:90.