Novel therapeutic uses of alpha-1 antitrypsin: A window to the future

COPD: Journal of Chronic Obstructive Pulmonary Disease

Volumn 9, Issue 6, 2012, Pages 583-588

  Wanner, Adam ^a   Arce, Adriana De ^a   Pardee, Erin ^a  

^a Alpha 1 Foundation   (United States)

Author keywords

COPD; Crohn's disease; Cystic fibrosis; Inflammation; Keywords: alpha 1 antitrypsin; Organ rejection; Type I diabetes; Viral infection

Indexed keywords

ALPHA 1 ANTITRYPSIN; ELASTASE; GLUCOSE; INTERLEUKIN 8; SERINE PROTEINASE INHIBITOR;

ACUTE GRAFT REJECTION; ACUTE HEART INFARCTION; AEROSOL; ALPHA ANTITRYPSIN DEFICIENCY; BRONCHIECTASIS; CHRONIC OBSTRUCTIVE LUNG DISEASE; CROHN DISEASE; CYSTIC FIBROSIS; DIABETES MELLITUS; DRUG FORMULATION; ENZYME ACTIVITY; GLUCOSE BLOOD LEVEL; GRAFT REJECTION; GRAFT VERSUS HOST REACTION; HEART FAILURE; HEART VENTRICLE FAILURE; HUMAN; INFLUENZA A (H1N1); INSULIN DEPENDENT DIABETES MELLITUS; LUNG DISEASE; LUNG LAVAGE; LUNG PARENCHYMA; NEUTROPHIL COUNT; NONHUMAN; ORGAN TRANSPLANTATION; PANCREATITIS; PARENCHYMA; PHASE 2 CLINICAL TRIAL (TOPIC); PLASMA HALF LIFE; PRIORITY JOURNAL; REVIEW; SURVIVAL RATE; VIRUS INFECTION;

ALPHA 1-ANTITRYPSIN; ANIMALS; ANTI-INFLAMMATORY AGENTS, NON-STEROIDAL; DIABETES MELLITUS, TYPE 1; DRUG APPROVAL; HUMANS; IMMUNOLOGIC FACTORS; LUNG DISEASES; PROTEIN ENGINEERING; RECOMBINANT PROTEINS; SERINE PROTEINASE INHIBITORS; UNITED STATES; VIRUS DISEASES;

EID: 84871510833     PISSN: 15412555     EISSN: 15412563     Source Type: Journal    
DOI: 10.3109/15412555.2012.717125     Document Type: Review

References (41)

1. Grimstein, C. Combination of alpha-1 antitrypsin and doxycycline suppresses collagen-induced arthritis. J Gene Med 2001;12:35-44.
2. Grimstein, C. Alpha-1 antitrypsin protein and gene therapies decrease autoimmunity and delay arthritis development in mouse model. J Transl Med 2011; 9:21.
3. Hashemi M, Naderi M, Rashidi H, Ghavami S. (2007) Impaired activity of serum alpha-1-antitrypsin in diabetes mellitus. Diabetes Res Clin Pract 75:246-248.
4. Hill AT, Campbell EJ, Bayley DL, Hill SL, Stockley RA. Evidence for excessive bronchial infl ammation during an acute exacerbation of chronic obstructive pulmonary disease in patients with alpha(1)-antitrypsin defi ciency (PiZ). Am J Respir Crit Care Med 1999; 160:1968-1975.
5. Huang H, Campbell SC, Nelius T, et al. Alpha1-antitrypsin inhibits angiogenesis and tumor growth. Int J Cancer 2004; 112:1042- 1048.
6. Koulmanda M, Bhasin M, Hoff man L, et al. Curative and beta cell regenerative Effects of alpha1-antitrypsin treatment in autoimmune diabetic NOD mice. Proc Natl Acad Sci USA 1008; 105:16242-16247.
7. Lewis EC, Mizrahi M, Toledano M, et al. alpha1-Antitrypsin monotherapy induces immune tolerance during islet allograft transplantation in mice. Proc Natl Acad Sci USA 2008; 105:16236-16241.
8. Lewis EC, Shapiro L, Bowers OJ, Dinarello CA. Alpha1-antitrypsin monotherapy prolongs islet allograft survival in mice. Proc Natl Acad Sci USA 2005; 102:12153-12158.
9. Lu Y. Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adenoassociated virus (rAAV1) vector. J Gene Med 2006; 8: 730-735.
10. Lu Y. Alpha1-antitrypsin gene therapy modulates cellular immunity and effi ciently prevents type 1 diabetes in nonobese diabetic mice. Hum Gene Ther 2006; 17:625-634.
11. Lu Y. Human alpha 1-antitrypsin therapy induces fatal anaphylaxis in non-obese diabetic mice. Clin Exp Immunol 2008; 154: 15-21.
12. Ma H. Intradermal alpha1-antitrypsin therapy avoids fatal anaphylaxis, prevents type 1 diabetes and reverses hyperglycaemia in the NOD mouse model of the disease. Diabetologia 2010; 53:2198-2204.
13. Marcondes AM, Xiang L, Tabellini L, Bartenstein M, Kabacka J, Sale GE, Dinarello CA, Deeg HJ. Inhibition of IL-32 activation by a-1 anti-trypsin reduces alloreactivity and increases survival in an allogeneic murine marrow transplantation model. Blood 2011; 118:5031-5039.
14. Matsumoto S, Okabe Y, Setoyama H, Takayama K, Ohtsuka J, Funahashi H, Imaoka A, Okada Y, Umesaki Y. Infl amatory bowel disease-like enteritis and caecitis in a senescence accelerated mouse P1/Yit strain. Gut 1998; 43:71-78.
15. Petrache I, Fijalkowska I, Zhen L, Medler TR, Brown E, Cruz P, Choe KH, Taraseviciene-Stewart L, Scerbavicius R, Shapiro L, Zhang B, Song S, Hicklin D, Voelkel NF, Flotte T, Tuder RM. A novel antiapoptotic role for alpha1-antitrypsin in the prevention of pulmonary emphysema. Am J Respir Crit Care Med 2006; 173:1222-1228.
16. Rivera-Nieves J, Bamias G, Vidrich A, Marini M, Pizarro TT, McDuffi e MJ, Moskaluk CA, Cohn SM, Cominelli F. Emergence of perianal fi stulizing disease in the SAMP1/YitFc mouse, a spontaneous model of chronic ileitis. Gastroenterology 2003; 124:972-982.
17. Sandler M, Gemperli BM, Hanekom C, Kuhn SH. Serum alpha 1-protease inhibitor in diabetes mellitus: reduced concentration and impaired activity. Diabetes Res Clin Pract. 1988; 5:249-255.
18. Shahaf G, Moser H, Ozeri E, Mizrahi M, Abecassis A, Lewis EC. alpha-1-antitrypsin gene delivery reduces infl ammation, increases T-regulatory cell population size and prevents islet allograft rejection. Mol Med 2011; 17:1000-1011.
19. Song S. Recombinant adeno-associated virus-mediated alpha-1 antitrypsin gene therapy prevents type I diabetes in NOD mice. Gene Ther 2004; 11:181-186.
20. Toldo S, Seropian IM, Mezzaroma E, Van Tassell BW, Salloum FN, Lewis EC, Voelkel N, Dinarello CA, Abbate A. Alpha-1 antitrypsin inhibits caspase-1 and protects from acute myocardial ischemia-reperfusion injury. J Mol Cell Cardiol 2011; 51:244-251.
21. Zhang SQ, Zhong XY, Chen GH, Lu WL, Zhang Q. The antidiabetic Effects and pharmacokinetic profi les of bis(maltolato) oxovanadium in non-di abetic and diabetic rats. J Pharm Pharmacol 2008; 60:99-105.
22. Bryan CL, Beard KS, Pott GB, Rahkola J, ardner EM, Janoff EN, Shapiro L. HIV infection is associated with reduced serum alpha-1-antitrypsin concentrations. Clin Invest Med 2010; 33:384-389.
23. Dhami R, Zay K, Gilks B, Porter S, Wright JL, Churg A. Pulmonary epithelial expression of human alpha1-antitrypsin in transgenic mice results in delivery of alpha1-antitrypsin protein to the interstitium. J Mol Med 1999; 77:377-385.
24. Forssmann WG, The YH, Stoll M, Adermann K, Albrecht U, Barlos K, Busmann A, Canales- Mayordomo A, Gimenez-Gallego G, Hirsch J, Jimenez-Barbero J, Meyer-Olson D, Munch J, Perez- Castells J, Standker L, Kirchhoff F, Schmidt RE. Shortterm monotherapy in HIV-infected patients with a virus entry inhibitor against the gp41 fusion peptide. Sci Transl Med 2010; 2:63.
25. Munch J, Standker L, Adermann K, Schulz A, Schindler M, Chinnadurai R, Pohlmann S, Chaipan C, Biet T, Peters T, Meyer B, Wilhelm D, Lu H, Jing W, Jiang S, Forssmann WG, Kirchhoff F. Discovery and optimization of a natural HIV-1 entry inhibitor targeting the gp41 fusion peptide. Cell 2007; 129:263-275.
26. Shapiro L, Pott GB, Ralston AH. Alpha-1-antitrypsin inhibits human immunodefi ciency virus type-1. FASEB J 2001; 15:115-122.
27. Brand P, Schulte M, Wencker M, Herpich CH, Klein G, Hanna K, Meyer T. Lung deposition of inhaled alpha1-proteinase inhibitor in cystic fi brosis and alpha1-antitrypsin defi ciency. Eur Respir J 2009; 34:354-360.
28. Carp H, Miller F, Hoidal JR, Janoff A. Potential mechanism of emphysema: alpha 1-proteinase inhibitor recovered from lungs of cigarette smokers contains oxidized methionine and has decreased elastase inhibitory capacity. Proc Natl Acad Sci 1982; 79:2041-2045.
29. Griese M, Latzin P, Kappler M, Weckerle K, Heinzlmaier T, Bernhardt T, Hartl D. Alpha1-antitrypsin inhalation reduces airway infl ammation in cystic fi brosis patients. Eur Respir J 2007; 29:240-250.
30. Hansen G, Schuster A, Zubrod C, Wahn V. Alpha 1-proteinase inhibitor abrogates proteolytic and secretagogue activity of cystic fi brosis sputum. Respiration 1995; 62:117-124.
31. Hubbard RC, Brantly ML, Sellers SE, Mitchell ME, Crystal RG. Antineutrophil- elastase defenses of the lower respiratory tract in alpha 1-antitrypsin defi ciency directly augmented with an aerosol of alpha 1-antitrypsin. Ann Intern Med.1989; 111:206-212.
32. Janciauskiene SM, Bals R, Koczulla R, Vogelmeier C, Kohnlein T, Welte T. The discovery of α1-antitrypsin and its role in health and disease. Respir Med 2011; 105:1129-1139.
33. Kerem E, Bauer S, Strauss P, Jaff e N, Armoni S, Pugatsch T, Shoseyov D, Tov N. Safety/Efficacy of inhaled human alpha-1 antitrypsin (AAT) in CF: a phase II clinical study. J Cyst Fibr 2009; 8:S25.
34. Martin SL, Downey D, Bilton D, Keogan MT, Edgar J, Elborn JS. Safety and Efficacy of recombinant alpha1-antitrypsin therapy in cystic fi brosis. Ped Pulmonol 2006; 41:177-183.
35. McElvaney NG, Hubbard RC, Birrer P, Chernick MS, Caplan DB, Frank MM, Crystal RG. Aerosol alpha 1-antitrypsin treatment for cystic fi brosis. Lancet 1991; 337:392-394.
36. Mogayzel PJ Jr, Flume PA. Update in cystic fi brosis. Am J Respir Crit Care Med 2010; 181:539-544.
37. Taggart C, Cervantes-Laurean D, Kim G, McElvaney NG, Wehr N, Moss J, Levine RL. Oxidation of either methionine 351 or methionine 358 in alpha 1-antitrypsin causes loss of antineutrophil elastase activity. J Biol Chem 2000; 275:258-265.
38. Taggart C, Coakley RJ, Greally P, Canny G, O'Neill SJ, McElvaney NG. Increased elastase release by CF neutrophils is mediated by tumor necrosis factor-alpha and interleukin-8. Am J Physiol Lung Cell Mol Physiol 2000; 278:33-41.
39. Tawara I, Sun Y, Lewis EC, Toubai T, Evers R, Nieves E, Azam T, Dinarello CA, Reddy P. Alpha-1 antitrypsin monotherapy reduces graft-vesrsus-host disease after experimental allogeneic bone marrow transplantation. Proc Natl Acad Sci USA 2012; 109:564-569.
40. Travis J. Structural evidence for methionine at the reactive site of human alpha-1- proteinase inhibitor. J Biol Chem 1978;253:7142-7144.
41. Walsh DE, Greene CM, Carroll TP, Taggart CC, Gallagher PM, O'Neill SJ, McElvaney NG. Interleukin-8 up-regulation by neutrophil elastase is mediated by MyD88/IRAK/TRAF-6 in human bronchial epithelium. J Biol Chem 2001; 276:35494-35499.