Clinical drug development using dynamic biomarkers to enable personalized health care in COPD

Chest

Volumn 148, Issue 1, 2015, Pages 16-23

  Bihlet, Asger R ^a   Karsdal, Morten A ^a   Bay Jensen, Anne Christine ^a   Read, Simon ^b   Kristensen, Jacob Hull ^a   Sand, Jannie Marie Bülow ^a   Leeming, Diana Julie ^a   Andersen, Jeppe Ragnar ^a   Lange, Peter ^c   Vestbo, Jørgen ^d  

^a NORDIC BIOSCIENCE A S   (Denmark)

^b GRÜNENTHAL GMBH   (Germany)

^c UNIVERSITY OF COPENHAGEN   (Denmark)

^d MANCHESTER UNIVERSITY NHS FOUNDATION TRUST   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

BIOLOGICAL MARKER; COLLAGEN TYPE 1; FIBRONECTIN; GELATINASE A; INTERSTITIAL COLLAGENASE; MACROPHAGE ELASTASE; MATRILYSIN; MATRIX METALLOPROTEINASE; PROADRENOMEDULLIN; SCLEROPROTEIN; TRANSFORMING GROWTH FACTOR BETA;

AIRWAY REMODELING; ARTICLE; CHRONIC OBSTRUCTIVE LUNG DISEASE; CLINICAL TRIAL (TOPIC); COMPUTER ASSISTED TOMOGRAPHY; CONNECTIVE TISSUE; DISEASE ACTIVITY; DISEASE EXACERBATION; DISEASE MARKER; DRUG IDENTIFICATION; DRUG RESEARCH; EXTRACELLULAR MATRIX; FORCED EXPIRATORY VOLUME; HUMAN; PHENOTYPE; PRIORITY JOURNAL; PROTEIN EXPRESSION; SEROLOGY; UPREGULATION; HEALTH SERVICES RESEARCH; METABOLISM; PATIENT SELECTION; PERSONALIZED MEDICINE; PULMONARY DISEASE, CHRONIC OBSTRUCTIVE;

BIOLOGICAL MARKERS; HEALTH SERVICES RESEARCH; HUMANS; INDIVIDUALIZED MEDICINE; PATIENT SELECTION; PULMONARY DISEASE, CHRONIC OBSTRUCTIVE;

EID: 84937571276     PISSN: 00123692     EISSN: 19313543     Source Type: Journal    
DOI: 10.1378/chest.15-0296     Document Type: Article

References (58)

1. Karsdal MA, Henriksen K, Leeming DJ, et al. Biochemical markers and the FDA critical path: how biomarkers may contribute to the understanding of pathophysiology and provide unique and necessary tools for drug development. Biomarkers. 2009;14(3):181-202.
2. Lander ES, Linton LM, Birren B, et al International Human Genome Sequencing Consortium. Initial sequencing and analysis of the human genome [published correction appears in Nature. 2001;411(6838):720]. Nature. 2001;409(6822):860-921.
3. Cook D, Brown D, Alexander R, et al. Lessons learned from the fate of AstraZeneca's drug pipeline: a five-dimensional framework. Nat Rev Drug Discov. 2014;13(6):419-431.
4. Sacks LV, Shamsuddin HH, Yasinskaya YI, Bouri K, Lanthier ML, Sherman RE. Scientific and regulatory reasons for delay and denial of FDA approval of initial applications for new drugs, 2000-2012. JAMA. 2014;311(4):378-384.
5. Scannell JW, Blanckley A, Boldon H, Warrington B. Diagnosing the decline in pharmaceutical R&D efficiency. Nat Rev Drug Discov. 2012;11(3):191-200.
6. Calverley PM. New treatments for COPD: many miles still to go. Lancet Respir Med. 2014;2(1):6-7.
7. Paving the Way for Personalized Medicine: FDA's Role in a New Era of Medical Product Development. Rockville, MD: US Food and Drug Administration; 2013.
8. Forde PM, Ettinger DS. Targeted therapy for non-small-cell lung cancer: past, present and future. Expert Rev Anticancer Ther. 2013;13(6):745-758.
9. Accurso FJ, Rowe SM, Clancy JP, et al. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med. 2010;363(21):1991-2003.
10. Burney P, Jithoo A, Kato B, et al Burden of Obstructive Lung Disease (BOLD) Study. Chronic obstructive pulmonary disease mortality and prevalence: the associations with smoking and poverty-a BOLD analysis. Thorax. 2014;69(5):465-473.
11. Smolonska J, Wijmenga C, Postma DS, Boezen HM. Meta-analyses on suspected chronic obstructive pulmonary disease genes: a summary of 20 years' research. Am J Respir Crit Care Med. 2009;180(7):618-631.
12. Vestbo J, Hurd SS, Agustí AG, et al. Global strategy for the diagnosis, management, and prevention of chronic obstructive pulmonary disease: GOLD executive summary. Am J Respir Crit Care Med. 2013;187(4):347-365.
13. Segreti A, Stirpe E, Rogliani P, Cazzola M. Defining phenotypes in COPD: an aid to personalized healthcare. Mol Diagn Ther. 2014;18(4):381-388.
14. Hurst JR, Vestbo J, Anzueto A, et al Evaluation of COPD Longitudinally to Identify Predictive Surrogate Endpoints (ECLIPSE) Investigators. Susceptibility to exacerbation in chronic obstructive pulmonary disease. N Engl J Med. 2010;363(12):1128-1138.
15. Langsetmo L, Platt RW, Ernst P, Bourbeau J. Underreporting exacerbation of chronic obstructive pulmonary disease in a longitudinal cohort. Am J Respir Crit Care Med. 2008;177(4):396-401.
16. Madani A, Zanen J, de Maertelaer V, Gevenois PA. Pulmonary emphysema: objective quantification at multi-detector row CT - comparison with macroscopic and microscopic morphometry. Radiology. 2006;238(3):1036-1043.
17. Thomsen M, Ingebrigtsen TS, Marott JL, et al. Inflammatory biomarkers and exacerbations in chronic obstructive pulmonary disease. JAMA. 2013;309(22):2353-2361.
18. Calverley PM, Sanchez-Toril F, McIvor A, Teichmann P, Bredenbroeker D, Fabbri LM. Effect of 1-year treatment with roflumilast in severe chronic obstructive pulmonary disease. Am J Respir Crit Care Med. 2007;176(2):154-161.
19. Wedzicha JA, Rabe KF, Martinez FJ, et al. Efficacy of roflumilast in the COPD frequent exacerbator phenotype. Chest. 2013;143(5):1302-1311.
20. Calverley PM, Rabe KF, Goehring UM, Kristiansen S, Fabbri LM, Martinez FJ; M2-124 and M2-125 Study Groups. Roflumilast in symptomatic chronic obstructive pulmonary disease: two randomised clinical trials. Lancet. 2009;374(9691):685-694.
21. Vestbo J, Agusti A, Wouters EF, et al Evaluation of COPD Longitudinally to Identify Predictive Surrogate Endpoints Study Investigators. Should we view chronic obstructive pulmonary disease differently after ECLIPSE? A clinical perspective from the study team. Am J Respir Crit Care Med. 2014;189(9):1022-1030.
22. Decramer M, Celli B, Kesten S, Lystig T, Mehra S, Tashkin DP; UPLIFT Investigators. Effect of tiotropium on outcomes in patients with moderate chronic obstructive pulmonary disease (UPLIFT): a prespecified subgroup analysis of a randomised controlled trial. Lancet. 2009;374(9696):1171-1178.
23. Calverley PM, Anderson JA, Celli B, et al TORCH Investigators. Salmeterol and fluticasone propionate and survival in chronic obstructive pulmonary disease. N Engl J Med. 2007;356(8):775-789.
24. Celli BR, Thomas NE, Anderson JA, et al. Effect of pharmacotherapy on rate of decline of lung function in chronic obstructive pulmonary disease: results from the TORCH study. Am J Respir Crit Care Med. 2008;178(4):332-338.
25. Tashkin DP, Celli B, Senn S, et al UPLIFT Study Investigators. A 4-year trial of tiotropium in chronic obstructive pulmonary disease. N Engl J Med. 2008;359(15):1543-1554.
26. Vestbo J, Edwards LD, Scanlon PD, et al ECLIPSE Investigators. Changes in forced expiratory volume in 1 second over time in COPD. N Engl J Med. 2011;365(13):1184-1192.
27. Jenkins CR, Jones PW, Calverley PM, et al. Efficacy of salmeterol/fluticasone propionate by GOLD stage of chronic obstructive pulmonary disease: analysis from the randomised, placebo-controlled TORCH study. Respir Res. 2009;10:59.
28. Fletcher C, Peto R, Tinker CM, et al. The Natural History of Chronic Bronchitis and Emphysema. New York, NY: Oxford University Press; 1976.
29. Svanes C, Sunyer J, Plana E, et al. Early life origins of chronic obstructive pulmonary disease. Thorax. 2010;65(1):14-20.
30. Karsdal MA, Christiansen C, Ladel C, Henriksen K, Kraus VB, Bay-Jensen AC. Osteoarthritis - a case for personalized health care? Osteoarthritis Cartilage. 2014;22(1):7-16.
31. Felson D, Niu J, Sack B, Aliabadi P, McCullough C, Nevitt MC. Progression of osteoarthritis as a state of inertia. Ann Rheum Dis. 2013;72(6):924-929.
32. Vestbo J, Anderson W, Coxson HO, et al ECLIPSE Investigators. Evaluation of COPD Longitudinally to Identify Predictive Surrogate End-points (ECLIPSE). Eur Respir J. 2008;31(4):869-873.
33. Vestbo J, Rennard S. Chronic obstructive pulmonary disease biomarker(s) for disease activity needed - urgently. Am J Respir Crit Care Med. 2010;182(7):863-864.
34. Stolz D, Kostikas K, Blasi F, et al. Adrenomedullin refines mortality prediction by the BODE index in COPD: the "BODE-A" index. Eur Respir J. 2014;43(2):397-408.
35. Leeming DJ, Sand JM, Nielsen MJ, et al. Serological investigation of the collagen degradation profile of patients with chronic obstructive pulmonary disease or idiopathic pulmonary fibrosis. Biomark Insights. 2012;7:119-126.
36. Kristensen JH, Karsdal MA, Genovese F, et al. The role of extracellular matrix quality in pulmonary fibrosis. Respiration. 2014;88(6):487-499.
37. Wedzicha JA, Calverley PM, Seemungal TA, Hagan G, Ansari Z, Stockley RA; INSPIRE Investigators. The prevention of chronic obstructive pulmonary disease exacerbations by salmeterol/fluticasone propionate or tiotropium bromide. Am J Respir Crit Care Med. 2008;177(1):19-26.
38. Mushtaq Y. The COPD pipeline. Nat Rev Drug Discov. 2014;13(4):253.
39. Watz H, Barnacle H, Hartley BF, Chan R. Efficacy and safety of the p38 MAPK inhibitor losmapimod for patients with chronic obstructive pulmonary disease: a randomised, double-blind, placebo-controlled trial. Lancet Respir Med. 2014;2(1):63-72.
40. Karsdal MA, Krarup H, Sand JM, et al. Review article: the efficacy of biomarkers in chronic fibroproliferative diseases - early diagnosis and prognosis, with liver fibrosis as an exemplar. Aliment Pharmacol Ther. 2014;40(3):233-249.
41. Salazar LM, Herrera AM. Fibrotic response of tissue remodeling in COPD. Lung. 2011;189(2):101-109.
42. Karsdal MA, Nielsen MJ, Sand JM, et al. Extracellular matrix remodeling: the common denominator in connective tissue diseases. Possibilities for evaluation and current understanding of the matrix as more than a passive architecture, but a key player in tissue failure. Assay Drug Dev Technol. 2013;11(2):70-92.
43. Crosby LM, Waters CM. Epithelial repair mechanisms in the lung. Am J Physiol Lung Cell Mol Physiol. 2010;298(6):L715-L731.
44. Kranenburg AR, Willems-Widyastuti A, Moori WJ, et al. Enhanced bronchial expression of extracellular matrix proteins in chronic obstructive pulmonary disease. Am J Clin Pathol. 2006;126(5):725-735.
45. Finlay GA, O'Driscoll LR, Russell KJ, et al. Matrix metalloproteinase expression and production by alveolar macrophages in emphysema. Am J Respir Crit Care Med. 1997;156(1):240-247.
46. Hallgren O, Nihlberg K, Dahlbäck M, et al. Altered fibroblast proteoglycan production in COPD. Respir Res. 2010;11:55.
47. Sandhaus RA, Turino G. Neutrophil elastase-mediated lung disease. COPD. 2013;10(suppl 1):60-63.
48. Churg A, Zhou S, Wright JL. Series "matrix metalloproteinases in lung health and disease": matrix metalloproteinases in COPD. Eur Respir J. 2012;39(1):197-209.
49. Karsdal MA, Bay-Jensen AC, Leeming DJ, Henriksen K, Christiansen C. Quantification of "end products" of tissue destruction in inflammation may reflect convergence of cytokine and signaling pathways - implications for modern clinical chemistry. Biomarkers. 2013;18(5):375-378.
50. Skjøt-Arkil H, Clausen RE, Nguyen QH, et al. Measurement of MMP-9 and -12 degraded elastin (ELM) provides unique information on lung tissue degradation. BMC Pulm Med. 2012;12(1):34.
51. Leeming Dj, He Y, Veidal S, et al. A novel marker for assessment of liver matrix remodeling: an enzyme-linked immunosorbent assay (ELISA) detecting a MMP generated type I collagen neo-epitope (C1M). Biomarkers. 2011;16(7):616-628.
52. Segovia-Silvestre T, Reichenbach V, Fernández-Varo G, et al. Circulating CO3-610, a degradation product of collagen III, closely reflects liver collagen and portal pressure in rats with fibrosis. Fibrogenesis Tissue Repair. 2011;4:19.
53. Veidal SS, Larsen DV, Chen X, et al. MMP mediated type V collagen degradation (C5M) is elevated in ankylosing spondylitis. Clin Biochem. 2012;45(7-8):541-546.
54. Veidal SS, Karsdal MA, Nawrocki A, et al. Assessment of proteolytic degradation of the basement membrane: a fragment of type IV collagen as a biochemical marker for liver fibrosis. Fibrogenesis Tissue Repair. 2011;4(1):22.
55. Bay-Jensen AC, Liu Q, Byrjalsen I, et al. Enzyme-linked immunosorbent assay (ELISAs) for metalloproteinase derived type II collagen neoepitope, CIIM - increased serum CIIM in subjects with severe radiographic osteoarthritis. Clin Biochem. 2011;44(5-6):423-429.
56. Bay-Jensen AC, Platt A, Byrjalsen I, Vergnoud P, Christiansen C, Karsdal MA. Effect of tocilizumab combined with methotrexate on circulating biomarkers of synovium, cartilage, and bone in the LITHE study. Semin Arthritis Rheum. 2014;43(4):470-478.
57. Veidal SS, Karsdal MA, Vassiliadis E, et al. MMP mediated degradation of type VI collagen is highly associated with liver fibrosis-identification and validation of a novel biochemical marker assay. PLoS One. 2011;6(9):e24753.
58. Genovese F, Barascuk N, Larsen L, et al. Biglycan fragmentation in pathologies associated with extracellular matrix remodeling by matrix metalloproteinases. Fibrogenesis Tissue Repair. 2013;6(1):9.