Site-specific chemical modification of antibody fragments using traceless cleavable linkers

Nature Protocols

Volumn 8, Issue 11, 2013, Pages 2079-2089

  Bernardes, Gonçalo J L ^a,b,c   Steiner, Martina ^a   Hartmann, Isabelle ^a   Neri, Dario ^a   Casi, Giulio ^d  

^a ETH ZURICH   (Switzerland)

^b UNIVERSITY OF CAMBRIDGE   (United Kingdom)

^c INSTITUTO DE MEDICINA MOLECULAR   (Portugal)

^d PHILOCHEM AG   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

ALDEHYDE DERIVATIVE; ANTIBODY CONJUGATE; ANTIBODY DRUG CONJUGATE; CEMADOTIN; CYSTEINE; DISULFIDE; IMMUNOGLOBULIN; IMMUNOGLOBULIN G ANTIBODY; SMALL IMMUNOPROTEIN; THIAZOLIDINE DERIVATIVE; THIOL GROUP; UNCLASSIFIED DRUG;

AMINO TERMINAL SEQUENCE; ARTICLE; BIOENGINEERING; CARBOXY TERMINAL SEQUENCE; CHEMICAL MODIFICATION; CLINICAL PROTOCOL; DISULFIDE BOND; DRUG CYTOTOXICITY; DRUG DELIVERY SYSTEM; DRUG POTENCY; DRUG SYNTHESIS; PRIORITY JOURNAL;

CYSTEINE; CYTOTOXINS; DRUG DELIVERY SYSTEMS; IMMUNOGLOBULIN FRAGMENTS; IMMUNOPROTEINS; OLIGOPEPTIDES; PROTEIN ENGINEERING; THIAZOLIDINES;

EID: 84886998274     PISSN: 17542189     EISSN: 17502799     Source Type: Journal    
DOI: 10.1038/nprot.2013.121     Document Type: Article

References (39)

1. Aggarwal, S. What?s fueling the biotech engine 2009-2010. Nat. Biotechnol. 28, 1165-1171 (2010).
2. Adamo, R. et al. Synthetically defined glycoprotein vaccines: Current status and future directions. Chem. Sci. 4, 2995-3008 (2013).
3. Webb, S. Pharma interest surges in antibody drug conjugates. Nat. Biotechnol. 29, 297-298 (2011).
4. Webb, S. Back on target. Nat. Biotechnol. 31, 191-193 (2013).
5. Younes, A. et al. Brentuximab vedotin (SGN-35) for relapsed CE30-positive lymphomas. N. Engl. J. Med. 363, 1812-1821 (2010).
6. Senter, P.D. & Sievers, E.L. The discovery and development of brentuximab vedotin for use in relapsed Hodgkin lymphoma and systemic anaplastic large cell lymphoma. Nat. Biotechnol. 30, 631-637 (2012).
7. Burris, H.A. et al. Phase II study of the antibody drug conjugate trastuzumab-dm1 for the treatment of human epidermal growth factor receptor 2 (HER2)-positive breast cancer after prior HER2-directed therapy. J. Clin. Oncol. 29, 398-405 (2011).
8. Senter, P.D. Potent antibody drug conjugates for cancer therapy. Curr. Opin. Chem. Biol. 13, 235-244 (2009).
9. Chari, R.V.J. Targeted cancer therapy: Conferring specificity to cytotoxic drugs. Acc. Chem. Res. 41, 98-107 (2008).
10. Casi, G. & Neri, D. Antibody-drug conjugates: Basic concepts, examples and future perspectives. J. Control. Release 161, 422-428 (2012).
11. Chalker, J.M., Bernardes, G.J.L. & Davis, B.G. A, tag-and-modify, approach to site-selective protein modification. Acc. Chem. Res. 44, 730-741 (2011).
12. Stephanopoulos, N. & Francis, M.B. Choosing an effective protein bioconjugation strategy. Nat. Chem. Biol. 7, 876-884 (2011).
13. Sletten, E.M. & Bertozzi, C.R. Bioorthogonal chemistry: Fishing for selectivity in a sea of functionality. Angew. Chem. Int. Ed. 48, 6974-6998 (2009).
14. Junutula, J.R. et al. Site-specific conjugation of a cytotoxic drug to an antibody improves the therapeutic index. Nat. Biotechnol. 26, 925-932 (2008).
15. Shen, B.-Q. et al. Conjugation site modulates the in vivo stability and therapeutic activity of antibody-drug conjugates. Nat. Biotechnol. 30, 184-189 (2012).
16. Axup, J.Y. et al. Synthesis of site-specific antibody-drug conjugates using unnatural amino acids. Proc. Natl. Acad. Sci. USA 109, 16101-16106 (2012).
17. Rabuka, D., Rush, J.S., deHart, G.W., Wu, P. & Bertozzi, C.R. Site-specific chemical protein conjugation using genetically encoded aldehyde tags. Nat. Protoc. 7, 1052-1067 (2012).
18. Carrico, I.S., Carlson, B.L. & Bertozzi, C.R. Introducing genetically encoded aldehydes into proteins. Nat. Chem. Biol. 3, 321-322 (2007). (Pubitemid 46789266)
19. Katz, J., Janik, J.E. & Younes, A. Brentuximab vedotin (SGN-35). Clin. Cancer Res. 17, 6428-6436 (2011).
20. Voynov, V. et al. Design and application of antibody cysteine variants. Bioconjug. Chem. 21, 385-392 (2010).
21. Wang, L., Amphlett, G., Blättler, W.A., Lambert, J.M. & Zhang, W. Structural characterization of the maytansinoid-monoclonal antibody immunoconjugate, huN901-DM1, by mass spectrometry. Protein Sci. 14, 2436-2446 (2005). (Pubitemid 41252812)
22. Bernardes, G.J.L. et al. A traceless vascular-targeting antibody-drug conjugate for cancer therapy. Angew. Chem. Int. Ed. 51, 941-944 (2012).
23. Casi, G., Huguenin-Dezot, N., Zuberbühler, K., Scheuermann, Jr. & Neri, D. Site-specific traceless coupling of potent cytotoxic drugs to recombinant antibodies for pharmacodelivery. J. Am. Chem. Soc. 134, 5887-5892 (2012).
24. Steiner, M. et al. Spacer length shapes drug release and therapeutic efficacy of traceless disulfide-linked ADCs targeting the tumor neovasculature. Chem. Sci. 4, 297-302 (2013).
25. Zuberbühler, K., Casi, G., Bernardes, G.J.L. & Neri, D. Fucose-specific conjugation of hydrazide derivatives to a vascular-targeting monoclonal antibody in igg format. Chem. Commun. 48, 7100-7102 (2012).
26. Gerber, H.-P., Senter, P.D. & Grewal, I.S. Antibody drug-conjugates targeting the tumor vasculature: Current and future developments. MAbs 1, 247-253 (2009).
27. Sharkey, R.M., Karacay, H., Govindan, S.V. & Goldenberg, D.M. Combination radioimmunotherapy and chemoimmunotherapy involving different or the same targets improves therapy of human pancreatic carcinoma xenograft models. Mol. Cancer Ther. 10, 1072-1081 (2011).
28. Neri, D. & Bicknell, R. Tumour vascular targeting. Nat. Rev. Cancer 5, 436-446 (2005). (Pubitemid 40791486)
29. Villa, A. et al. A high-affinity human monoclonal antibody specific to the alternatively spliced EDA domain of fibronectin efficiently targets tumor neo-vasculature in vivo. Int. J. Cancer 122, 2405-2413 (2008). (Pubitemid 351590471)
30. Tarli, L. et al. A high-affinity human antibody that targets tumoral blood vessels. Blood 94, 192-198 (1999). (Pubitemid 29300149)
31. Holliger, P. & Hudson, P.J. Engineered antibody fragments and the rise of single domains. Nat. Biotechnol. 23, 1126-1136 (2005). (Pubitemid 41486394)
32. Borsi, L. et al. Selective targeting of tumoral vasculature: Comparison of different formats of an antibody (L19) to the ED-B domain of fibronectin. Int. J. Cancer 102, 75-85 (2002).
33. Kim, K.M. et al. Anti-CD30 diabody-drug conjugates with potent antitumor activity. Mol. Cancer Ther. 7, 2486-2497 (2008).
34. Rybak, J.-N., Roesli, C., Kaspar, M., Villa, A. & Neri, D. The extra-domain a of fibronectin is a vascular marker of solid tumors and metastases. Cancer Res. 67, 10948-10957 (2007). (Pubitemid 350145924)
35. Pettit, G.R. et al. The isolation and structure of a remarkable marine animal antineoplastic constituent: Dolastatin. J. Am. Chem. Soc. 109, 6883-6885 (1987). (Pubitemid 18036607)
36. Pettit, G.R. et al. Isolation and structure of the cytostatic linear depsipeptide dolastatin J. Org. Chem. 54, 6005-6006 (1989). (Pubitemid 20063834)
37. Simmons, T.L., Andrianasolo, E., McPhail, K., Flatt, P. & Gerwick, W.H. Marine natural products as anticancer drugs. Mol. Cancer Ther. 4, 333-342 (2005). (Pubitemid 40340212)
38. Sun, X. et al. Design of antibodymaytansinoid conjugates allows for efficient detoxification via liver metabolism. Bioconjug. Chem. 22, 728-735 (2011).
39. Ellman, G.L. Tissue sulfhydryl groups. Arch. Biochem. Biophys. 82, 70-77 (1959).