Immunological monitoring to rationally guide AAV gene therapy

Frontiers in Immunology

Volumn 4, Issue SEP, 2013, Pages

  Britten, Cedrik Michael ^a,b   Walter, Steffen ^c   Janetzki, Sylvia ^d  

^a JOHANNES GUTENBERG UNIVERSITY   (Germany)

^b Association for Cancer Immunotherapy (CIMT)   (Germany)

^c IMMATICS BIOTECHNOLOGIES GMBH   (Germany)

^d ZellNet Consulting Inc   (United States)

Author keywords

Adeno associated virus; Assay harmonization; Biomarkers; Gene therapy; Immunological monitoring

Indexed keywords

T LYMPHOCYTE RECEPTOR; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; CELLULAR IMMUNITY; CLINICAL TRIAL (TOPIC); CRYOPRESERVATION; ENZYME LINKED IMMUNOSPOT ASSAY; GENE THERAPY; HUMAN; HUMORAL IMMUNITY; IMMUNOASSAY; IMMUNOLOGICAL MONITORING; MULTICENTER STUDY (TOPIC); PROTOCOL COMPLIANCE; QUALITY CONTROL; REVIEW; STANDARDIZATION; T LYMPHOCYTE; TRANSGENE;

EID: 84886049165     PISSN: None     EISSN: 16643224     Source Type: Journal    
DOI: 10.3389/fimmu.2013.00273     Document Type: Review

References (40)

1. Wirth T, Parker N, Yla-Herttuala S. History of gene therapy. Gene (2013) 525(2):162-9. doi:10.1016/j.gene.2013.03.137.
2. Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med (2011) 365:2357-65. doi:10.1056/NEJMoa1108046.
3. Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr., Mingozzi F, Bennicelli J, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med (2008) 358:2240-8. doi:10.1056/NEJMoa0802315.
4. Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science (2009) 326:818-23. doi:10.1126/science.1171242.
5. Kastelein JJ, Ross CJ, Hayden MR. From mutation identification to therapy: discovery and origins of the first approved gene therapy in the Western world. Hum Gene Ther (2013) 24:472-8. doi:10.1089/hum.2013.063.
6. Mingozzi F, High KA. Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat Rev Genet (2011) 12:341-55. doi:10.1038/nrg2988.
7. Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, et al. Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response. Nat Med (2006) 12:342-7. doi:10.1038/nm1358.
8. Mingozzi F, High KA. Immune responses to AAV in clinical trials. Curr Gene Ther (2011) 11:321-30. doi:10.2174/156652311796150354.
9. Mingozzi F, High KA. Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood (2013) 122:23-36. doi:10.1182/blood-2013-01-306647.
10. Mendell JR, Campbell K, Rodino-Klapac L, Sahenk Z, Shilling C, Lewis S, et al. Dystrophin immunity in Duchenne's muscular dystrophy. N Engl J Med (2010) 363:1429-37. doi:10.1056/NEJMoa1000228.
11. Hoffman BE, Ertl HC, Terhorst C, High KA, Herzog RW. Gene therapy research at the frontiers of viral immunology. Front Microbiol (2012) 3:182. doi:10.3389/fmicb.2012.00182.
12. Bennett J, Ashtari M, Wellman J, Marshall KA, Cyckowski LL, Chung DC, et al. Maguire, AAV2 gene therapy readministration in three adults with congenital blindness. Sci Transl Med (2012) 4:120ra15. doi:10.1126/scitranslmed.3002865.
13. Office of Biotechnology Activities, NIH Recombinant DNA Advisory Committee Meeting. Report on Immune Responses to Adeno-Associated Virus (AAV) Vectors. (2007). Available from: http://oba.od.nih.gov/oba/rac/minutes/RAC_minutes_06-07.pdf.
14. van der Burg SH, Kalos M, Gouttefangeas C, Janetzki S, Ottensmeier C, Welters MJ, et al. Harmonization of immune biomarker assays for clinical studies. Sci Transl Med (2011) 3:108s44. doi:10.1126/scitranslmed.3002785.
15. Maecker HT, McCoy JP Jr, Amos M, Elliott J, Gaigalas A, Wang L, et al. A model for harmonizing flow cytometry in clinical trials. Nat Immunol (2010) 11:975-8. doi:10.1038/ni1110-975.
16. Kalos M. An integrative paradigm to impart quality to correlative science. J Transl Med (2010) 8:26. doi:10.1186/1479-5876-8-26.
17. Janetzki S, Romero P, Roederer M, Bolton DLM, Jandus C. Immunemonitoring design within the developmental pipeline for an immunotherapeutic or preventive vaccine. First ed. In: Morrow WJW, Sheikh NA, Schmidt CS, Huw Davies D editors. Vaccinology: Principles and Practice. Oxford: Blackwell Publishing Ltd (2012). p. 417-40.
18. Bull M, Lee D, Stucky J, Chiu YL, Rubin A, Horton H, et al. Defining blood processing parameters for optimal detection of cryopreserved antigen-specific responses for HIV vaccine trials. J Immunol Methods (2007) 322:57-69. doi:10.1016/j.jim.2007.02.003.
19. Kierstead LS, Dubey S, Meyer B, Tobery TW, Mogg R, Fernandez VR, et al. Enhanced rates and magnitude of immune responses detected against an HIV vaccine: effect of using an optimized process for isolating PBMC. AIDS Res Hum Retroviruses (2007) 23:86-92. doi:10.1089/aid.2006.0129.
20. Mallone R, Mannering SI, Brooks-Worrell BM, Durinovic-Bello I, Cilio CM, Wong FS, et al. Isolation and preservation of peripheral blood mononuclear cells for analysis of islet antigen-reactive T cell responses: position statement of the T-cell workshop committee of the immunology of diabetes society. Clin Exp Immunol (2011) 163:33-49. doi:10.1111/j.1365-2249.2010.04272.x.
21. Scheibenbogen C, Romero P, Rivoltini L, Herr W, Schmittel A, Cerottini JC, et al. Quantitation of antigen-reactive T cells in peripheral blood by IFNgamma-ELISPOT assay and chromium-release assay: a four-centre comparative trial. J Immunol Methods (2000) 244:81-9. doi:10.1016/S0022-1759(00)00257-X.
22. Cox JH, Ferrari G, Kalams SA, Lopaczynski W, Oden N, D'souza MP. Results of an ELISPOT proficiency panel conducted in 11 laboratories participating in international human immunodeficiency virus type 1 vaccine trials. AIDS Res Hum Retroviruses (2005) 21:68-81. doi:10.1089/aid.2005.21.68.
23. Britten CM, Gouttefangeas C, Welters MJ, Pawelec G, Koch S, Ottensmeier C, et al. The CIMT-monitoring panel: a two-step approach to harmonize the enumeration of antigen-specific CD8+ T lymphocytes by structural and functional assays. Cancer Immunol Immunother (2008) 57:289-302. doi:10.1007/s00262-007-0378-0.
24. Janetzki S, Panageas KS, Ben-Porat L, Boyer J, Britten CM, Clay TM, et al. Results and harmonization guidelines from two large-scale international Elispot proficiency panels conducted by the cancer vaccine consortium (CVC/SVI). Cancer Immunol Immunother (2008) 57:303-15. doi:10.1007/s00262-007-0380-6.
25. Britten CM, Janetzki S, Ben-Porat L, Clay TM, Kalos M, Maecker H, et al. Harmonization guidelines for HLA-peptide multimer assays derived from results of a large scale international proficiency panel of the cancer vaccine consortium. Cancer Immunol Immunother (2009) 58:1701-13. doi:10.1007/s00262-009-0681-z.
26. Attig S, Price L, Janetzki S, Kalos M, Pride M, McNeil L, et al. A critical assessment for the value of markers to gate-out undesired events in HLA-peptide multimer staining protocols. J Transl Med (2011) 9:108. doi:10.1186/1479-5876-9-108.
27. Janetzki S, Britten CM. The impact of harmonization on ELISPOT assay performance. Methods Mol Biol (2012) 792:25-36. doi:10.1007/978-1-61779-325-7_2.
28. Filbert H, Attig S, Bidmon N, Renard BY, Janetzki S, Sahin U, et al. Serum-free freezing media support high cell quality and excellent ELISPOT assay performance across a wide variety of different assay protocols. Cancer Immunol Immunother (2013) 62:615-27. doi:10.1007/s00262-012-1359-5.
29. Janetzki S, Price L, Britten CM, van der Burg SH, Caterini J, Currier JR, et al. Performance of serum-supplemented and serum-free media in IFNgamma Elispot assays for human T cells. Cancer Immunol Immunother (2010) 59:609-18. doi:10.1007/s00262-009-0788-2.
30. Welters MJ, Gouttefangeas C, Ramwadhdoebe TH, Letsch A, Ottensmeier CH, Britten CM, et al. Harmonization of the intracellular cytokine staining assay. Cancer Immunol Immunother (2012) 61:967-78. doi:10.1007/s00262-012-1282-9.
31. McNeil LK, Price L, Britten CM, Jaimes M, Maecker H, Odunsi K, et al. A harmonized approach to intracellular cytokine staining gating: results from an international multiconsortia proficiency panel conducted by the cancer immunotherapy consortium (CIC/CRI). Cytometry A (2013) 83:728-38.
32. Janetzki S, Britten CM, Kalos M, Levitsky HI, Maecker HT, Melief CJ, et al. "MIATA"-minimal information about T cell assays. Immunity (2009) 31:527-8. doi:10.1016/j.immuni.2009.09.007.
33. Britten CM, Janetzki S, Butterfield LH, Ferrari G, Gouttefangeas C, Huber C, et al. T cell assays and MIATA: the essential minimum for maximum impact. Immunity (2012) 37:1-2. doi:10.1016/j.immuni.2012.07.010.
34. Smith JG, Joseph HR, Green T, Field JA, Wooters M, Kaufhold RM, et al. Establishing acceptance criteria for cell-mediated-immunity assays using frozen peripheral blood mononuclear cells stored under optimal and suboptimal conditions. Clin Vaccine Immunol (2007) 14:527-37. doi:10.1128/CVI.00435-06.
35. McKenna KC, Beatty KM, Vicetti MR, Bilonick RA. Delayed processing of blood increases the frequency of activated CD11b+ CD15+ granulocytes which inhibit T cell function. J Immunol Methods (2009) 341:68-75. doi:10.1016/j.jim.2008.10.019.
36. Ruitenberg JJ, Mulder CB, Maino VC, Landay AL, Ghanekar SA. VACUTAINER CPT and Ficoll density gradient separation perform equivalently in maintaining the quality and function of PBMC from HIV seropositive blood samples. BMC Immunol (2006) 7:11. doi:10.1186/1471-2172-7-11.
37. Butterfield LH, Palucka AK, Britten CM, Dhodapkar MV, Hakansson L, Janetzki S, et al. Recommendations from the iSBTc-SITC/FDA/NCI workshop on immunotherapy biomarkers. Clin Cancer Res (2011) 17:3064-76. doi:10.1158/1078-0432.CCR-10-2234.
38. Maecker HT, McCoy JP, Nussenblatt R. Standardizing immunophenotyping for the human immunology project. Nat Rev Immunol (2012) 12:191-200. doi:10.1038/nri3158.
39. Walter S, Weinschenk T, Stenzl A, Zdrojowy R, Pluzanska A, Szczylik C, et al. Multipeptide immune response to cancer vaccine IMA901 after single-dose cyclophosphamide associates with longer patient survival. Nat Med (2012) 18:1254-61. doi:10.1038/nm.2883.
40. Singh SK, Tummers B, Schumacher TN, Gomez R, Franken KL, Verdegaal EM, et al. The development of standard samples with a defined number of antigen-specific T cells to harmonize T cell assays: a proof-of-principle study. Cancer Immunol Immunother (2013) 62:489-501. doi:10.1007/s00262-012-1351-0.