Validation of a flow cytometry-based assay to assess C5aR receptor occupancy on neutrophils and monocytes for use in drug development

Cytometry Part B - Clinical Cytometry

Volumn 90, Issue 2, 2016, Pages 177-190

  Quadrini, Karen J ^a   Hegelund, Anne Charlotte ^b   Cortes, Kasia E ^a   Xue, Chengsen ^a   Kennelly, Susan M ^a   Ji, Hong ^b   Högerkorp, Carl Magnus ^b   Mc Closkey, Thomas W ^a  

^a ICON PLC   (United Kingdom)

^b NOVO NORDISK A S   (Denmark)

Author keywords

biomarker; clinical trial; drug development; flow cytometry; receptor occupancy; validation

Indexed keywords

COMPLEMENT COMPONENT C5A RECEPTOR; FLUOROCHROME; MONOCLONAL ANTIBODY; NN 0384; UNCLASSIFIED DRUG; C5AR1 PROTEIN, HUMAN; NNC0215-0384;

ANALYTICAL PARAMETERS; ARTICLE; CLINICAL LABORATORY; CONTROLLED STUDY; DRUG BINDING; DRUG DEVELOPMENT; FLOW CYTOMETRY; FLUORESCENCE; HUMAN; HUMAN CELL; INTERMETHOD COMPARISON; MEASUREMENT ACCURACY; MEDIAN FLUORESCENCE INTENSITY; MOLECULES OF EQUIVALENT SOLUBLE FLUOROCHROME; MONOCYTE; NEUTROPHIL; PRIORITY JOURNAL; RECEPTOR OCCUPANCY; REPRODUCIBILITY; RHEUMATOID ARTHRITIS; VALIDATION STUDY; ARTHRITIS, RHEUMATOID; DRUG EFFECTS; IMMUNOLOGY; ISOLATION AND PURIFICATION; PATHOLOGY;

ANTIBODIES, MONOCLONAL; ARTHRITIS, RHEUMATOID; FLOW CYTOMETRY; HUMANS; MONOCYTES; NEUTROPHILS; RECEPTOR, ANAPHYLATOXIN C5A;

EID: 84941794928     PISSN: 15524949     EISSN: 15524957     Source Type: Journal    
DOI: 10.1002/cyto.b.21260     Document Type: Article

References (39)

1. Robas N., Use of PD biomarkers to drive dose selection and early clinical decision making. Bioanalysis 2012; 4: 2485-2497.
2. Jackson RC., Pharmacodynamic modelling of biomarker data in oncology. ISRN Pharmacol 2012; 2012: 590626
3. Walport M., Complement. In:, Roitt I, Brostoff J, Male D, editors. Immunology, 3rd ed. London: Mosby-Year Book Europe Limited; 1993. p 12.1-12.17.
4. Giclas PC., Hereditary and Acquired Complement Deficiencies. In:, Detrick B, Hamilton RG, Folds JD, editors. Manual of Molecular and Clinical Laboratory Immunology, 7th ed. Washington DC: ASM Press; 2006. p 914-923.
5. Monk PN, Scola AM, Madala P, Fairlie DP., Function, structure and therapeutic potential of complement C5a receptors. Br J Pharmacol 2007; 152: 429-448.
6. Lee H, Whitfeld PL, MacKay CR., Receptors for complement C5a. The importance of C5aR and the enigmatic role of C5L2. Immunol Cell Biol 2008; 86: 153-160.
7. Ricklin D, Lambris JD., Complement-targeted therapeutics. Nat Biotechnol 2007; 25: 1265-1275.
8. Woodruff TM, Nandakumar KS, Tedesco F., Inhibiting the C5-C5a receptor axis. Mol Immunol 2011; 48: 1631-1642.
9. Sternebring O, Alifrangis L, Christensen TF, Hegelund AC, Ji H, Högerkorp CM., A weighted method for estimation of receptor occupancy for pharmacodynamic response measurements in drug development. Cytometry B Clin Cytom (in press).
10. Prevoo ML, van 't Hof MA, Kuper HH, van Leeuwen MA, van de Putte LB, van Riel PL., Modified disease activity scores that include twenty-eight-joint counts. Development and validation in a prospective longitudinal study of patients with rheumatoid arthritis. Arthritis Rheum 1995; 38: 44-48.
11. Wells G, Becker J-C, Teng J, Dougados M, Schiff M, Smolen J, Aletaha D, van Riel PLCM., Validation of the 28-joint Disease Activity Score (DAS28) and European League Against Rheumatism response criteria based on C-reactive protein against disease progression in patients with rheumatoid arthritis, and comparison with the DAS28 based on erythrocyte sedimentation rate. Ann Rheum Dis 2009; 68: 954-960.
12. Schwartz A, Marti GE, Poon R, Gratama JW, Fernandez-Repollet E., Standardizing flow cytometry: A classification system of fluorescence standards used for flow cytometry. Cytometry 1998; 33: 106-114.
13. Schwartz A, Gaigalas AK, Wang L, Marti GE, Vogt RF, Fernandez-Repollet E., Formalization of the MESF unit of fluorescence intensity. Cytometry B Clin Cytom 2004; 57B: 1-6.
14. Tanqri S, Vall H, Kaplan D, Hoffman B, Purvis N, Porwit A, Hunsberger B, Shankey TV., Validation of cell-based fluorescence assays: practice guidelines from the ICSH and ICCS- part III-analytical issues. Cytometry B Clin Cytom 2013; 84B: 291-308.
15. Cunliffe J, Derbyshire N, Keeler S, Coldwell R., An approach to the validation of flow cytometry methods. Pharm Res 2009; 26: 2551-2557.
16. O'Hara D, Xu Y, Liang Z, Reddy M, Wue D, Litwin V., Recommendations for the validation of flow cytometric testing during drug development: II assays. J Immunol Methods 2011; 363: 120-134.
17. FDA Guidance for industry: Bioanalytical method validation. Available at: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM368107.pdf. 2013. Accessed October 14, 2014.
18. Wood B, Jevremovic D, Bene MC, Yan M, Jacobs P, Litwin V., Validation of cell-based fluorescence assays: Practice guidelines from the ICSH and ICCS-part V- assay performance criteria. Cytometry B Clin Cytom 2013; 84B: 315-323.
19. Brahmer JR, Drake CG, Wollner I, Powderly JD, Picus J, Sharfman WH, Stankevich E, Pons A, Salay TM, McMiller TL, et al., Phase I study of single-agent anti-programmed death-1 (MDX-1106) in refractory solid tumors: Safety, clinical activity, pharmacodynamics, and immunologic correlates. J Clin Oncol 2010; 28: 3167-3175.
20. Scheerens H, Su Z, Irving B, Townsend MJ, Zheng Y, Stefanich E, Chindalore V, Bingham CO III, Davis JC Jr,. MTRX1011A, a humanized anti-CD4 monoclonal antibody, in the treatment of patients with rheumatoid arthritis: A phase I randomized, double-blind, placebo-controlled study incorporating pharmacodynamic biomarker assessments. Arthritis Res Therapy 2011; 13: R177
21. Green CL, Brown L, Stewart JJ, Xu Y, Litwin V, Mc Closkey TW., Recommendations for the validation of flow cytometric testing during drug development: I instrumentation. J Immunol Methods 2011; 363: 104-119.
22. Davis BH, Dasgupta A, Kussick S, Han J-Y, Estrellado A., Validation of cell-based fluorescence assays: Practice guidelines from the ICSH and ICCS-part II- preanalytical issues. Cytometry B Clin Cytom 2013; 84B: 286-290.
23. Wright SD, Ramos RA, Tobias PS, Ulevitch RJ, Mathison JC., CD14, a receptor for complexes of lipopolysaccharide (LPS) and LPS binding protein. Science 1990; 249: 1431-1433.
24. Kielian TL, Blecha F., CD14 and other recognition molecules for lipopolysaccharide: A review. Immunopharmacology 1995; 29: 187-205.
25. Loken MR., Immunofluorescence Techniques. In:, Melamed MR, Lindmo T, Mendelsohn ML, editors. Flow cytometry and sorting, 2nd ed. New York: Wiley; 1990. p 346, 348-349.
26. Cartwright GE, Athens JW, Wintrobe MM., Analytical review: the kinetics of granulopoiesis in normal man. Blood 1964; 24: 780-803.
27. Scheel-Toellner D, Wang K, Craddock R, Webb PR, McGettrick HM, Assi LK, Parkes N, Clough LE, Gulbins E, Salmon M, Lord JM., Reactive oxygen species limit neutrophil life span by activating death receptor signaling. Blood 2004; 104: 2557-2564.
28. Pliyev BK., Chemotactically active proteins of neutrophils. Biochemistry (Moscow) 2008; 73: 970-984.
29. Wright HL, Moots RJ, Bucknall RC, Edwards SW., Neutrophil function in inflammation and inflammatory diseases. Rheumatology (Oxford) 2010; 49: 1618-1631.
30. Summers C, Rankin SM, Condliffe AM, Singh N, Peters AM, Chilvers ER., Neutrophil kinetics in health and disease. Trends Immunol 2010; 31: 318-324.
31. van Eeden SF, Klut ME, Walker BAM, Hogg JC., The use of flow cytometry to measure neutrophil function. J Immunol Methods 1999; 232: 23-43.
32. Davis C, Wu X, Lia W, Fan H, Reddy M., Stability of immunophenotypic markers in fixed peripheral blood for extended analysis using flow cytometry. J Immunol Methods 2011; 363: 158-165.
33. Canonico B, Zamai L, Burattini S, Granger V, Mannello F, Gobbi P, Felici C, Falcieri E, Reilly JT, Barnett D, et al., Evaluation of leukocyte stabilisation in TransFix-treated blood samples by flow cytometry and transmission electron microscopy. J Immunol Methods 2004; 295: 67-78.
34. Bergeron M, Shafaie A, Ding T, Phaneuf S, Soucy N, Mandy F, Bradley J, Fahey J., Evaluation of stabilized blood cell products as candidate preparations for quality assessment programs for CD4 T-cell counting. Cytometry 2002; 50: 86-91.
35. Canonico B, Betti M, Luchetti F, Battistelli M, Falcieri E, Ferri P, Zamai L, Barnett D, Papa S., Flow cytomteric profiles, biomolecular and morphological aspects of transfixed leukocytes and red cells. Cytometry B Clin Cytom 2010; 78: 267-278.
36. Ng AA, Lee BT, Teo TS, Poidinger M, Connolly JE., Optimal cellular preservation for high dimensional flow cytometric analysis of multicentre trials. J Immunol Methods 2012; 385: 79-89.
37. Kendal AP, Snyder R, Garrison PJ., Validation of cold chain procedures suitable for distribution of vaccines by public health programs in the USA. Vaccine 1997; 15: 1459-1465.
38. Elliott MA, Halbert GW., Maintaining the cold chain shipping environment for Phase I clinical trial distribution. Int J Pharm 2005; 299: 49-54.
39. Olson WC, Smolkin ME, Farris EM, Fink RJ, Czarkowski AR, Fink JH, Chianese-Bullock KA, Slingluff CL Jr., Shipping blood to a central laboratory in multicenter clinical trials: Effect of ambient temperature on specimen temperature, and effects of temperature on mononuclear cell yield, viability and immunologic function. J Transl Med 2011; 9: 26.