Inhalation delivery of protein therapeutics

Inflammation and Allergy - Drug Targets

Volumn 12, Issue 2, 2013, Pages 81-87

  Kane, Colleen ^a   O'Neil, Karyn ^a   Conk, Michelle ^a   Picha, Kristen ^a  

^a JANSSEN RESEARCH AND DEVELOPMENT   (Belgium)

Author keywords

Alternative scaffolds; Inhalation; Protein therapeutics; Pulmonary delivery

Indexed keywords

BERACTANT; CALFACTANT; CETUXIMAB; DEOXYRIBONUCLEASE; DEPELESTAT; ERYTHROPOIETIN; IMMUNOGLOBULIN E ANTIBODY; INSULIN; INTERLEUKIN 13; PALIVIZUMAB; PORACTANT; PROTEIN; RECOMBINANT HUMAN INSULIN; RESPIRATORY SYNCYTIAL VIRUS ANTIBODY; RESPIRATORY SYNCYTIAL VIRUS VACCINE; SMALL INTERFERING RNA;

ADULT RESPIRATORY DISTRESS SYNDROME; ANEMIA; ASTHMA; CYSTIC FIBROSIS; DRUG DELIVERY SYSTEM; DRUG DEVELOPMENT; DRUG EFFICACY; GENE THERAPY; HUMAN; IMMUNE RESPONSE; LUNG DISEASE; MULTICENTER STUDY (TOPIC); NEBULIZATION; NONHUMAN; PNEUMONIA; RANDOMIZED CONTROLLED TRIAL (TOPIC); RESPIRATORY SYNCYTIAL PNEUMOVIRUS; RESPIRATORY SYNCYTIAL VIRUS INFECTION; REVIEW; SYSTEMIC DISEASE;

ADMINISTRATION, INHALATION; ANIMALS; BIOLOGICAL PRODUCTS; HUMANS; LUNG DISEASES; PROTEINS;

EID: 84876718607     PISSN: 18715281     EISSN: None     Source Type: Journal    
DOI: 10.2174/1871528111312020002     Document Type: Review

References (73)

1. National Institutes of Health NH, Lung, and Blood Institute. Fact Book, Fiscal Year 2011 2012. Available from: http://www.nhlbi.nih.gov/about/factbook/chapter4.htm.
2. Breathing in America: Diseases, Progress, and Hope 2010. Available from: http://www.thoracic.org/education/breathing-in-america/index.php.
3. Grainger, D.W. Controlled-release and local delivery of therapeutic antibodies. Expert. Opin. Biol. Ther., 2004, 4(7), 1029-1044.
4. Leach, C.L. Inhalation aspects of therapeutic aerosols. Toxicol. Pathol., 2007, 35(1), 23-26.
5. Beck A.; Wurch T.; Bailly C.; Corvaia N. Strategies and challenges for the next generation of therapeutic antibodies. Nat. Rev. Immunol., 2010, 10(5), 345-352.
6. Reichert, J.M.; Dhimolea, E. The future of antibodies as cancer drugs. Drug Discov. Today, 2012, 17(17-18), 954-963.
7. Reichert, J.; Pavlou A. Monoclonal antibodies market. 2004 2004/05//print; 3(5), 383-384.
8. Scolnik, P.A. mAbs-a business perspective. mAbs, 2009, 1, 179-184.
9. Fuchs, H.; Borowitz D.S.; Christiansen DH et al. Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. N. Engl. J. Med., 1994, 331(10), 637-642.
10. Suri, R. The use of human deoxyribonuclease (rhDNase) in the management of cystic fibrosis. Biodrugs, 2005, 19(3),135-144.
11. Singh, N.; Hawley, K. L.; Viswanathan, K. Efficacy of porcine versus bovine surfactants for preterm newborns with respiratory distress syndrome: systematic review and meta-analysis. Pediatrics, 2011, 128(6), e1588-e1595.
12. Gansslen, M. Uber inhalation von insulin. Klin Wochenschr., 1925, 4, 71.
13. Patton, J.S.; Byron, P.R. Inhaling medicines: delivering drugs to the body through the lungs. Nat. Rev. Drug Discov., 2007, 6(1), 67-74.
14. Skyler, J.S.; Cefalu, W.T.; Kourides, I.A.; Landschulz, W.H Balagtas, C.C.; Cheng, S.L.; Gelfand, R.A. Efficacy of inhaled human insulin in type 1 diabetes mellitus: a randomised proof-of-concept study. Lancet, 2001, 357(9253), 331-315.
15. Cappelleri, J.C.; Cefalu, W.T.; Rosenstock J.; Kourides IA.; Gerber RA. Treatment satisfaction in type 2 diabetes: a comparison between an inhaled insulin regimen and a subcutaneous insulin regimen. Clin. Ther., 2002, 24(4), 552-564.
16. Hollander, P.A.; Blonde, L.; Rowe R.; Mehta AE.; Milburn JL Hershon KS. Efficacy and safety of inhaled insulin (Exubera) compared with subcutaneous insulin therapy in patients with type 2 diabetes. Diabetes Care, 2004, 27, 2356-2362.
17. Quattrin, T.; Bélanger, A.; Bohannon, N J, Schwartz S L. Efficacy and Safety of Inhaled Insulin (Exubera) Compared with Subcutaneous Insulin Therapy in Patients with Type 1 Diabetes. Diabetes Care, 2004, 27(11), 2622-2627.
18. Siekmeier R.; Scheuch G. Inhaled Insulin -Does It Become Reality? J. Physiol. Pharmacol., 2008, 59(Suppl 6), 81-113.
19. Piazza, F.M.; Johnson, S.A.; Ottolini, M.G.; Schmidt, H.J.; Darnell, M.E.; Hemming, V.G.; Prince, G.A. Immunotherapy of Respiratory Syncytial Virus Infection in Cotton Rats (Sigmodon Fulviventer) Using IgG in a Small-Particle Aerosol. J. Inf. Dis., 1992, 166, 1422-1424.
20. Rimensberger PC.; Burek-Kozlowska A.; Morell A.; Germann D Eigenmann AK.; Steiner F.; Burger R.; Kuenzli M.; Schaad UB. Aerosolized immunoglobulin treatment of respiratory syncytial virus infection in infants. Pediatr. Infect. Dis. J., 1996, 15(3), 209-216.
21. Groothuis, J.R.; Simoes, E.A.; Levin, M.J.; Hall, C.B.; Long, C.E Rodriguez, W.J.; Arrobio, J.; Meissner, H.C.; Fulton, D.R.; Welliver, R.C. Prophylactic administration of respiratory syncytial virus immune globulin to high-risk infants and young children. N. Engl. J. Med., 1993, 329, 1524-1530.
22. Group TPS. Reduction of respiratory syncytial virus hospitalization among premature infants and infants with bronchopulmonary dysplasia using respiratory syncytial virus immune globulin prophylaxis. Pediatrics, 1997, 99, 93-99.
23. Group, T.I.R.S. Reduction of respiratory syncytial virus hospitalization among premature infants and infants with bronchopulmonary dysplasia using respiratory syncytial virus monoclonal antibody prophylaxis. Pediatrics, 1998, 102, 531-537.
24. Fahy, J.V.; Cockcroft, D.W.; Boulet, L.P.; Wong, H.H Deschesnes, F.; Davis, E.E.; Ruppel, J.; Su, J.Q.; Adelman, D.C. Effect of aerosolized anti-IgE (E25) on airway responses to inhaled allergen in asthmatic subjects. Am. J. Respir. Crit. Care Med., 1999, 160, 1023-1027.
25. Patton, J.S. Mechanisms of macromolecule absorption by the lungs. Adv. Drug Delivery Rev., 1996, 19, 3-36.
26. Maillet A.; Congy-Jolivet, N.; Le Guellec, S.; Vecellio, L Hamard, S.; Courty, Y.; Courtois, A.; Gauthier, F.; Diot, P.; Thibault, G.; Lemarie, E.; Heuze-Vourc'h, N. Aerodynamical, immunological and pharmacological properties of the anticancer antibody cetuximab following nebulization. Pharm. Res., 2008, 25(6), 1318-1326.
27. Dumont, J.A.; Bitonti, A.J.; Clark, D.; Evans, S.; Pickford, M Newman, S.P. Delivery of an erythropoietin-Fc fusion protein by inhalation in humans through an immunoglobulin transport pathway. J. Aerosol Med., 2005, 18(3), 294-303.
28. Israel, E.J.; Patel, V.K.; Taylor, S.F.; Marshak-Rothstein, A Simister, N.E. Requirement for a beta 2-microglobulin-associated Fc receptor for acquisition of maternal IgG by fetal and neonatal mice. J. Immunol., 1995, 154, 6246-6251.
29. Israel E.J.; Taylor, S.; Wu, Z.; Mizoguchi, E.; Blumberg, R.S Bhan, A.; Simister, N.E. Expression of the neonatal Fc, receptor, FcRn, on human intestinal epithelial cells. Immunology, 1997, 92, 69.
30. Spiekermann, G.M.; Finn, P. W.; Ward, E. S.; Dumont, J Dickinson, B. L.; Blumberg, R. S.; Lencer, W. I. Receptor-mediated Immunoglobulin G Transport Across Mucosal Barriers in Adult Life: Functional expression of FcRn in the mammalian lung. J. Exp. Med., 2002, 196(3), 303-310.
31. Dickinson, B.L.; Badizadegan, K.; Wu, Z.; Ahouse, J.C.; Zhu, X Simister, N.E.; Blumberg, R.S.; Lencer, W.I. Bidirectional FcRn-dependent IgG transport in a polarized human intestinal epithelial cell line. J. Clin, Invest., 1997, 1999(104), 7.
32. Stattel, J.M.; Lu, Y.; Tan, C.A.; Song, J.J.; Garcia, A.M.; Simister, N.E.; Spiekermann, G.M.; Lencer, W.I.; Blumberg, R.S. Pulmonary delivery of an erythropoietin Fc fusion protein in non-human primates through an immunoglobulin transport pathway. Proc. Natl. Acad. Sci. USA, 2004, 101(26), 9763-9768.
33. Low SC.; Nunes, S. L.; Bitonti, A. J.; Dumont, J. A. Oral and pulmonary delivery of FSH-Fc fusion proteins via neonatal Fc receptor-mediated transcytosis. Hum. Reprod., 2005, 20(7), 1805-1813.
34. Roberts, B.L.; Markland, W.; Ley, A.C.; Kent, R.B.; White, D.W Guterman, S.K.; Ladner, R.C. Directed evolution of a protein: Selection of potent neutrophil elastase inhibitors displayed on M13 fusion phage. Proc. Natl. Acad. Sci. USA, 1992, 89, 2429-2433.
35. Delacourt C.; Herigault S.; Delclaux C.; Poncin A.; Levame M Harf A.; Saudubray F.; Lafuma C. Protection Against Acute Lung Injury by Intravenous or Intratracheal Pretreatment with EPI-HNE-4, a New Potent Neutrophil Elastase Inhibitor. Am. J. Respir. Cell Mol. Biol., 2002, 26, 290-297.
36. Corp D. Dyax 2000 Annual Report. Cambridge, MA, 2000.
37. www.clinicaltrials.gov. Available from: http://www.clinicaltrials.gov/ct2/show/NCT00455767?term=DX-890.
38. Hacha J TK.; Maertens L.; Paulissen G.; Rocks N.; Foidart JM Noel A.; Palframan R.; Gueders M.; Cataldo Hacha, J.; Tomlinson, K.; Maertens, L.; Paulissen, G.; Rocks, N.; Foidart, J.M.; Noel, A.; Palframan, R.; Gueders, M.; Cataldo, D.D. Nebulized Anti-IL-13 Monoclonal Antibody Fab' Fragment Reduces Allergen-Induced Asthma. Am. J. Respir. Cell Mol. Biol., 2012, 47(5), 709-717.
39. Schellekens, H. Factors influencing the immunogenicity of therapeutic proteins. Nephrol. Dial. Transplant., 2005, 20(Suppl 6), vi3-9.
40. Patton, J.S.; Brain, J.D.; Davies, L.A.; Fiegel, J.; Gumbleton, M Kim, K.J.; Sakagami, M.; Vanbever, R.; Ehrhardt, C. The particle has landed-characterizing the fate of inhaled pharmaceuticals. J. Aerosol Med. Pulm. Drug Deliv., 2010, 23(Suppl 2), S71-S87.
41. Patton JS.; Fishburn CS.; Weers JG. The lungs as a portal of entry for systemic drug delivery. Proc. Am. Thorac. Soc., 2004, 1(4), 3383-3344.
42. Carvalho, T.C.; Peters, J.I.; Williams, R.O. Influence of particle size on regional lung deposition-what evidence is there? Int. J. Pham., 2011, 406, 1-10.
43. Lombry C.; Edwards, D. A.; Preat, V.; Vanbever, R. Alveolar macrophages are a primary barrier to pulmonary absorption of macromolecules. Am. J. Physiol. Lung Cell. Mol. Physiol., 2004, 286(5), L1002-1008.
44. Holt, P.G.; Strickland, D. H.; Wikstrom, M. E.; Jahnsen, F. L. Regulation of immunological homeostasis in the respiratory tract. Nat. Rev. Immunol., 2008, 8(2), 142-152.
45. Geiser, M. Update on macrophage clearance of inhaled micro-and nanoparticles. J. Aerosol Med. Pulm. Drug Deliv., 2010, 23(4), 207-217.
46. Kawai, T.; Akira, S. The role of pattern-recognition receptors in innate immunity: update on Toll-like receptors. Nat. Immunol., 2010, 11(5), 373-384.
47. Demedts IK.; Brusselle, G. G.; Vermaelen, K. Y.; Pauwels, R. A. Identification and characterization of human pulmonary dendritic cells. Am. J. Respir. Cell Mol. Biol., 2005, 32(3), 177-184.
48. Masten, B.J.; Olson, G.K.; Tartleton, C.A.; Rund, C.; Schuyler, M Mahran, R.; Archibeque, T.; Lipscomb, M.F. Characterization of myeloid and plasmacytoid dendritic cells in human lung. J. Immunol., 2006, 177, 7784.
49. Tibaldi, J.M. Evolution of insulin development: focus on key parameters. Adv.Ther., 2012, 29(7), 590-619.
50. Lambrecht, B.N. Alveolar macrophage in the driver's seat. Immunity, 2006, 24(4), 366-368.
51. Jakubzick, C.; Tackle, F.; Llodra, J.; van Rooijen, J.; Randolph, G.J. Modulation of dendritic cell trafficking to and from the airways. J. Immunol., 2006, 176, 3578-3584.
52. Casadevall, N.; Nataf, J.; Viron, B.; Kolta, A.; Kiladjian, J.J Martin-Dupont, P.; Michaud, P.; Papo, T.; Ugo, V.; Teyssandier, I.; Varet, B.; Mayeux, P. Pure red-cell aplasia and antierythropoietin antibodies in patients treated with recombinant erythropoietin. N. Engl. J. Med., 2002, 346, 469-475.
53. Gebauer, M.; Skerra, A. Engineered protein scaffolds as next-generation antibody therapeutics. Curr. Opin. Chem. Biol., 2009, 13(3), 245-255.
54. Skerra, A. Lipocalins as a scaffold. Biochim. Biophys. Acta, 2000, 1482(1-2), 337-50.
55. Lipovsek, D. Adnectins: engineered target-binding protein therapeutics. Protein Eng. Des. Sel., 2011, 24(1-2), 3-9.
56. Nixon, A.; Wood, C.R. Engineered protein inhibitors of proteases. Curr. Opin. Drug Discov. Devel., 2006, 9(2), 261-268.
57. Nygren, P.A. Alternative binding proteins: affibody binding proteins developed from a small three-helix bundle scaffold. FEBS J., 2008, 275(11), 2668-2676.
58. Stumpp, M.T.; Binz, H.K.; Amstutz, P. DARPins: a new generation of protein therapeutics. Drug Discov. Today, 2008, 13(15-16), 695-701.
59. Jacobs, S.A.; Diem, M.; Luo, J.; Teplyakov, A.; Obmolova, G Malia, T.; Gilliland, G.L.; O'Neil, K.T. Design of novel FN3 domains with high stability by a consensus sequence approach. Protein Eng. Des. Sel., 2012, 25(3), 107-117.
60. Van Bockstaele, F.; Holz, J.B.; Revets, H. The development of nanobodies for therapeutic applications. Curr. Opin. Invest. Drugs, 2009, 10(11), 1212-1224.
61. Tijink, B.; Laeremans, T.; Budde, M.; Stigter-van Walsum, M Dreier, T.; de Haard, H.J.; Leemans, C.R.; van Dongen, G.A.M.S. Improved tumor targeting of anti-epidermal growth factor receptor Nanobodies through albumin binding: taking advantage of modular Nanobody technology. Mol. Cancer Ther., 2008, 7(8), 2288-2297.
62. Zahnd C.; Kawe, M.; Stumpp, M.T.; de Pasquale, C.; Tamaskovic, R.; Nagy-Davidescu, G.; Dreier, B.; Schibli, R.; Binz, H.K.; Waibel, R.; Plückthun, A. Efficient tumor targeting with high-affinity designed ankyrin repeat proteins: effects of affinity and molecular size. Cancer Res., 2010, 70(4), 1595.
63. de Wildt, R.M.; Holmes, S.; Tomlinson, I.M.; Winter, G.P Fitzgerald, M.F.; Fox, J.C.; Sepp, A.; Luckett, J.; Woolven, B.P. Inventor; Domantis Limited, assignee. Agents that bind a target in pulmonary tissue for targeting respiratory diseases. United States 2012.
64. Hanton, G.; Detalle, L.; Priem, S.; Bouche, M.P.; Depla, E Baumeister, J.; Holz, J.B. Eds. ALX-0171 toxicity study in RSV-infected cotton rats: safety and therapeutic potential Congress of the European Society of Toxicologies, 2012, Stockholm, Sweden.
65. Ablynx. Ablynx's first inhaled nanobody successfully completes phase 1 study. PipelineReviewcom. 2012.
66. DeGeorge, J.J.; Ahn, C.H.; Andrews, P.A.; Brower, M.E.; Choi, Y.S.; Chun, M.Y.; Du, T.; Lee-Ham, D.Y.; McGuinn, W.D.; Pei, L.; Sancilio, L.F.; Schmidt, W.; Sheevers, H.V.; Sun, C.J.; Tripathi, S.; Vogel, W.M.; Whitehurst, V.; Williams, S.; Taylor, A.S. Considerations for toxicology studies of respiratory drug products. Regul. Toxicol. Pharmacol., 1997, 25(2), 189-193.
67. Owen, K. Regulatory toxicology considerations for the development of inhaled pharmaceuticals. Drug Chem. Toxicol., 2013, 36(1), 109-118.
68. Bloom, B.; Clark, R.H.; for the Infasurf Survanta Clinical Trial Group. Comparison of infasurf (calfactant) and survanta (beractant) in the prevention and treatment of respiratory distress syndrome. Pediatrics, 2005, 116(2), 392-399.
69. Ramanathan, R. Animal-derived surfactants: where are we? The evidence from randomized, controlled clinical trials animal-derived surfactants. J. Perinatol., 2009, 29, S38-S43.
70. Pringle, I.; Hyde, S.C.; Connolly, M.M.; Lawton, A.E.; Xu, B Nunez-Alonso, G.; Davies, L.A.; Sumner-Jones, S.G.; Gill, D.R. CpG-free plasmid expression cassettes for cystic fibrosis gene therapy. Biomaterials, 2012, 33, 6833-6842.
71. Ripple, M.; You, D.; Honnegowda, S.; Giaimo, J.D.; Sewell, A.B Becnel, D.M.; Cormier, S.A. Immunomodulation with IL-4Ra antisense oligonucleotide prevents respiratory syncytial virus-mediated pulmonary disease. J. Immunol., 2010, 185(8), 4804-4811.
72. Huang, Y.; Wang, C.H. Pulmonary delivery of insulin by liposomal carriers. J. Control. Release, 2006, 113(1), 9-14.
73. Dong, H.; Stanek, O.; Rudilla Salvador, F.; Langer, U.; Morrillon, E.; Ung, C.; Sebo, P.; Leclerc, C.; Majlessi, L. Induction of protective immunity against Mycobacterium tuberculosis by delivery of ESX antigens into airway dendritic cells. Mucosal Immunol., 2012, [E-pub ahead of pint].