Addressing safety liabilities of TfR bispecific antibodies that cross the blood-brain barrier

Science Translational Medicine

Volumn 5, Issue 183, 2013, Pages

  Couch, Jessica A ^a   Yu, Y Joy ^a   Zhang, Yin ^a   Tarrant, Jacqueline M ^a   Fuji, Reina N ^a   Meilandt, William J ^a   Solanoy, Hilda ^a   Tong, Raymond K ^a   Hoyte, Kwame ^a   Luk, Wilman ^a   Lu, Yanmei ^a   Gadkar, Kapil ^a   Prabhu, Saileta ^a   Ordonia, Benjamin A ^a   Nguyen, Quyen ^a   Lin, Yuwen ^a   Lin, Zhonghua ^a   Balazs, Mercedesz ^a   Scearce Levie, Kimberly ^a   Ernst, James A ^a   Dennis, Mark S ^a   Watts, Ryan J ^a   more..

^a GENENTECH INC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA SECRETASE 1; BISPECIFIC ANTIBODY; COMPLEMENT; IMMUNOGLOBULIN FC FRAGMENT; TRANSFERRIN RECEPTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BINDING AFFINITY; BLOOD BRAIN BARRIER; CONTROLLED STUDY; DOSE RESPONSE; DRUG BLOOD LEVEL; DRUG SAFETY; DRUG UPTAKE; FEMALE; HUMAN; HUMAN CELL; LOW DRUG DOSE; MOUSE; NONHUMAN; PRIORITY JOURNAL; RETICULOCYTE;

ACUTE DISEASE; AMYLOID BETA-PEPTIDES; AMYLOID PRECURSOR PROTEIN SECRETASES; ANIMALS; ANTIBODIES, BISPECIFIC; ANTIBODY AFFINITY; ANTIBODY SPECIFICITY; ASPARTIC ACID ENDOPEPTIDASES; BLOOD-BRAIN BARRIER; COMPLEMENT SYSTEM PROTEINS; DOSE-RESPONSE RELATIONSHIP, IMMUNOLOGIC; HAPLORHINI; HUMANS; MICE; RECEPTORS, TRANSFERRIN; RETICULOCYTE COUNT;

EID: 84878276906     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.3005338     Document Type: Article

References (31)

1. W. A. Banks, Developing drugs that can cross the blood-brain barrier: Applications to Alzheimer's disease. BMC Neurosci. 9 (Suppl. 3), S2 (2008).
2. J. Lichota, T. Skjørringe, L. B. Thomsen, T. Moos, Macromolecular drug transport into the brain using targeted therapy. J. Neurochem. 113, 1-13 (2010).
3. A. R. Jones, E. V. Shusta, Blood-brain barrier transport of therapeutics via receptor-mediation. Pharm. Res. 24, 1759-1771 (2007).
4. W. A. Jefferies, M. R. Brandon, S. V. Hunt, A. F. Williams, K. C. Gatter, D. Y. Mason, Transferrin receptor on endothelium of brain capillaries. Nature 312, 162-163 (1984).
5. P. M. Friden, L. R. Walus, G. F. Musso, M. A. Taylor, B. Malfroy, R. M. Starzyk, Anti-transferrin receptor antibody and antibody-drug conjugates cross the blood-brain barrier. Proc. Natl. Acad. Sci. U.S.A. 88, 4771-4775 (1991).
6. J. B. Fishman, J. B. Rubin, J. V. Handrahan, J. R. Connor, R. E. Fine, Receptor-mediated transcytosis of transferrin across the blood-brain barrier. J. Neurosci. Res. 18, 299-304 (1987).
7. W. M. Pardridge, J. L. Buciak, P. M. Friden, Selective transport of an anti-transferrin receptor antibody through the blood-brain barrier in vivo. J. Pharmacol. Exp. Ther. 259, 66-70 (1991).
8. R. L. Roberts, R. E. Fine, A. Sandra, Receptor-mediated endocytosis of transferrin at the blood-brain barrier. J. Cell Sci. 104 (Pt. 2), 521-532 (1993).
9. H. J. Lee, B. Engelhardt, J. Lesley, U. Bickel, W. M. Pardridge, Targeting rat anti-mouse transferrin receptor monoclonal antibodies through blood-brain barrier in mouse. J. Pharmacol. Exp. Ther. 292, 1048-1052 (2000).
10. S. Gosk, C. Vermehren, G. Storm, T. Moos, Targeting anti-transferrin receptor antibody (OX26) and OX26-conjugated liposomes to brain capillary endothelial cells using in situ perfusion. J. Cereb. Blood Flow Metab. 24, 1193-1204 (2004).
11. T. Moos, E. H. Morgan, Restricted transport of anti-transferrin receptor antibody (OX26) through the blood-brain barrier in the rat. J. Neurochem. 79, 119-129 (2001).
12. S. Paris-Robidas, V. Emond, C. Tremblay, D. Soulet, F. Calon, In vivo labeling of brain capillary endothelial cells after intravenous injection of monoclonal antibodies targeting the transferrin receptor. Mol. Pharmacol. 80, 32-39 (2011).
13. Y. J. Yu, Y. Zhang, M. Kenrick, K. Hoyte, W. Luk, Y. Lu, J. Atwal, J. M. Elliott, S. Prabhu, R. J. Watts, M. S. Dennis, Boosting brain uptake of a therapeutic antibody by reducing its affinity for a transcytosis target. Sci. Transl. Med. 3, 84ra44 (2011).
14. J. K. Atwal, Y. Chen, C. Chiu, D. L. Mortensen, W. J. Meilandt, Y. Liu, C. E. Heise, K. Hoyte, W. Luk, Y. Lu, K. Peng, P. Wu, L. Rouge, Y. Zhang, R. A. Lazarus, K. Scearce-Levie,W. Wang, Y. Wu, M. Tessier-Lavigne, R. J. Watts, A therapeutic antibody targeting BACE1 inhibits amyloid-β production in vivo. Sci. Transl. Med. 3, 84ra43 (2011).
15. B. J. Iacopetta, E. H. Morgan, G. C. Yeoh, Transferrin receptors and iron uptake during erythroid cell development. Biochim. Biophys. Acta 687, 204-210 (1982).
16. P. Ponka, C. N. Lok, The transferrin receptor: Role in health and disease. Int. J. Biochem. Cell Biol. 31, 1111-1137 (1999).
17. R. S. Riley, J. M. Ben-Ezra, R. Goel, A. Tidwell, Reticulocytes and reticulocyte enumeration. J. Clin. Lab. Anal. 15, 267-294 (2001).
18. T. Takai, M. Li, D. Sylvestre, R. Clynes, J. V. Ravetch, FcR γ chain deletion results in pleiotrophic effector cell defects. Cell 76, 519-529 (1994).
19. A. Circolo, G. Garnier, W. Fukuda, X. Wang, T. Hidvegi, A. J. Szalai, D. E. Briles, J. E. Volanakis, R. A. Wetsel, H. R. Colten, Genetic disruption of the murine complement C3 promoter region generates deficient mice with extrahepatic expression of C3 mRNA. Immunopharmacology 42, 135-149 (1999).
20. G. Garratty, The James Blundell Award Lecture 2007: Do we really understand immune red cell destruction? Transfus. Med. 18, 321-334 (2008).
21. K. Kissel, S. Hamm, M. Schulz, A. Vecchi, C. Garlanda, B. Engelhardt, Immunohistochemical localization of the murine transferrin receptor (TfR) on blood-tissue barriers using a novel anti-TfR monoclonal antibody. Histochem. Cell Biol. 110, 63-72 (1998).
22. T. H. Oh, G. J. Markelonis, G. M. Royal, B. S. Bregman, Immunocytochemical distribution of transferrin and its receptor in the developing chicken nervous system. Brain Res. 395, 207-220 (1986).
23. R. N. Kalaria, S. M. Sromek, I. Grahovac, S. I. Harik, Transferrin receptors of rat and human brain and cerebral microvessels and their status in Alzheimer's disease. Brain Res. 585, 87-93 (1992).
24. H. Tabata, S. Izumisawa, M. Kubo, T. Matsuzawa, Reticulocyte counts in laboratory animals using a manual and a flow cytometric procedure (Sysmex R-3000). Compar. Haematol. Int. 7, 100-103 (1997).
25. L. R. Walus, W. M. Pardridge, R. M. Starzyk, P. M. Friden, Enhanced uptake of rsCD4 across the rodent and primate blood-brain barrier after conjugation to anti-transferrin receptor antibodies. J. Pharmacol. Exp. Ther. 277, 1067-1075 (1996).
26. R. J. Boado, Y. Zhang, Y. Zhang, W. M. Pardridge, Humanization of anti-human insulin receptor antibody for drug targeting across the human blood-brain barrier. Biotechnol. Bioeng. 96, 381-391 (2007).
27. R. J. Boado, Y. Zhang, Y. Zhang, C. F. Xia, W. M. Pardridge, Fusion antibody for Alzheimer's disease with bidirectional transport across the blood-brain barrier and Aβ fibril disaggregation. Bioconjug. Chem. 18, 447-455 (2007).
28. R. Vassar, B. D. Bennett, S. Babu-Khan, S. Kahn, E. A. Mendiaz, P. Denis, D. B. Teplow, S. Ross, P. Amarante, R. Loeloff, Y. Luo, S. Fisher, J. Fuller, S. Edenson, J. Lile, M. A. Jarosinski, A. L. Biere, E. Curran, T. Burgess, J. C. Louis, F. Collins, J. Treanor, G. Rogers, M. Citron, β-Secretase cleavage of Alzheimer's amyloid precursor protein by the transmembrane aspartic protease BACE. Science 286, 735-741 (1999).
29. Q. H. Zhou, R. J. Boado, E. K. Hui, J. Z. Lu, W. M. Pardridge, Chronic dosing of mice with a transferrin receptor monoclonal antibody-glial-derived neurotrophic factor fusion protein. Drug Metab. Dispos. 39, 1149-1154 (2011).
30. S. M. Paul, Therapeutic antibodies for brain disorders. Sci. Transl. Med. 3, 84ps20 (2011).
31. J. B. Ridgway, L. G. Presta, P. Carter, 'Knobs-into-holes' engineering of antibody CH3 domains for heavy chain heterodimerization. Protein Eng. 9, 617-621 (1996).