Cystic fibrosis therapeutics: The road ahead

Chest

Volumn 143, Issue 1, 2013, Pages 207-213

  Hoffman, Lucas R ^a   Ramsey, Bonnie W ^a  

^a UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ACETYLCYSTEINE; AMIKACIN; AMINOGLYCOSIDE ANTIBIOTIC AGENT; ARIKACE; ATALUREN; AZITHROMYCIN; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DOCOSAHEXAENOIC ACID; DORNASE ALFA; FOSFOMYCIN; GALLIUM NITRATE; GS 9411; IBUPROFEN; IVACAFTOR; LEVOFLOXACIN; LIPOSOME; LUMACAFTOR; MANNITOL; PLACEBO; QUINOLINE DERIVED ANTIINFECTIVE AGENT; SILDENAFIL; SODIUM CHANNEL BLOCKING AGENT; TOBRAMYCIN; UNCLASSIFIED DRUG;

AIRWAY OBSTRUCTION; ANTIINFLAMMATORY ACTIVITY; ANTIMICROBIAL ACTIVITY; ARTICLE; BRONCHUS MUCUS; CFTR GENE; CLINICAL FEATURE; CYSTIC FIBROSIS; DRUG APPROVAL; DRUG RESEARCH; DRUG TARGETING; FORCED EXPIRATORY VOLUME; GENE; GENE MUTATION; GENE THERAPY; HUMAN; LIPOSOMAL DELIVERY; NONHUMAN; PATHOPHYSIOLOGY; PRIORITY JOURNAL; PROTEIN DEFECT; PSEUDOMONAS INFECTION; RESPIRATORY TRACT INFLAMMATION; STEM CELL TRANSPLANTATION; TREATMENT OUTCOME;

EID: 84871946692     PISSN: 00123692     EISSN: 19313543     Source Type: Journal    
DOI: 10.1378/chest.12-1639     Document Type: Article

References (52)

1. Ashlock MA, Beall RJ, Hamblett NM, et al. A pipeline of therapies for cystic fibrosis. Semin Respir Crit Care Med. 2009;30(5):611-626.
2. Drug development pipeline. Cystic Fibrosis Foundation website. http://www.cff.org/research/drugdevelopmentpipeline. Accessed June 26, 2012.
3. 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.
4. Ramsey BW, Davies J, McElvaney NG, et al; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18):1663-1672.
5. Updated questions and answers for healthcare professionals and the public: use an approved pancreatic enzyme product (PEP). US Food and Drug Administration website. http://www.Fda.Gov/drugs/drugsafety/ postmarketdrugsafetyinformation forpatientsandproviders/ucm204745.Htm. Accessed June 26, 2012.
6. Ratjen F. What's new in CF airway inflammation: an update. Paediatr Respir Rev. 2006;7(suppl 1):S70-S72. (Pubitemid 43906243)
7. Geller DE, Flume PA, Staab D, Fischer R, Loutit JS, Conrad DJ; Mpex 204 Study Group. Levofloxacin inhalation solution (MP-376) in patients with cystic fibrosis with Pseudomonas aeruginosa. Am J Respir Crit Care Med. 2011;183(11):1510-1516.
8. Okusanya OO, Bhavnani SM, Hammel J, et al. Pharmacokinetic and pharmacodynamic evaluation of liposomal amikacin for inhalation in cystic fibrosis patients with chronic pseudomonal infection. Antimicrob Agents Chemother. 2009;53(9):3847-3854.
9. Study to evaluate Arikace™ in CF patients with chronic Pseudomonas aeruginosa infections. NCT01315678. Clinical trials.gov. Bethesda, MD; National Institutes of Health; 2011. http://clinicaltrials.gov/ct2/show/NCT01315678. Updated March 2012. Accessed June 26, 2012.
10. Arikace® for nontuberculous mycobacteria. NCT01315236. Clinicaltrials.gov. Bethesda, MD; National Institutes of Health; 2011. http://clinicaltrials.gov/ct2/show/NCT01315236. Updated June 2012. Accessed June 26, 2012.
11. Trapnell BC, McColley SA, Kissner DG, et al; Phase 2 FTI Study Group. Fosfomycin/tobramycin for inhalation in patients with cystic fibrosis with pseudomonas airway infection. Am J Respir Crit Care Med. 2012;185(2):171-178.
12. Rogers GB, Carroll MP, Serisier DJ, Hockey PM, Jones G, Bruce KD. characterization of bacterial community diversity in cystic fibrosis lung infections by use of 16s ribosomal DNA terminal restriction fragment length polymorphism profiling. J Clin Microbiol. 2004;42(11):5176-5183. (Pubitemid 39506227)
13. Tunney MM, Klem ER, Fodor AA, et al. Use of culture and molecular analysis to determine the effect of antibiotic treatment on microbial community diversity and abundance dur ing exacerbation in patients with cystic fibrosis. Thorax. 2011;66(7):579-584.
14. Zhao J, Schloss PD, Kalikin LM, et al. Decade-long bacterial community dynamics in cystic fibrosis airways. Proc Natl Acad Sci U S A. 2012;109(15):5809-5814.
15. Rogers GB, Hoffman LR, Döring G. Novel concepts in evaluating antimicrobial therapy for bacterial lung infections in patients with cystic fibrosis. J Cyst Fibros. 2011;10(6):387-400.
16. A pharmacokinetic and safety study of IV gallium nitrate (Ganite) in cystic fibrosis patients. NCT01093521. Clinical Trials.gov. Bethesda, MD: National Institutes of Health; 2010. http://clinicaltrials.gov/ct2/show/ nct01093521. Updated June 14, 2012. Accessed June 26, 2012.
17. Kaneko Y, Thoendel M, Olakanmi O, Britigan BE, Singh PK. The transition metal gallium disrupts Pseudomonas aeruginosa iron metabolism and has antimicrobial and antibiofilm activity. J Clin Invest. 2007;117(4):877-888. (Pubitemid 46556744)
18. Parsek MR, Singh PK. Bacterial biofilms: an emerging link to disease pathogenesis. Annu Rev Microbiol. 2003;57:677-701. (Pubitemid 37392958)
19. Halwani M, Yebio B, Suntres ZE, Alipour M, Azghani AO, Omri A. Co-encapsulation of gallium with gentamicin in liposomes enhances antimicrobial activity of gentamicin against Pseudomonas aeruginosa. J Antimicrob Chemother. 2008;62(6):1291-1297.
20. Konstan MW, Schluchter MD, Xue W, Davis PB. Clinical use of Ibuprofen is associated with slower FEV1 decline in children with cystic fibrosis. Am J Respir Crit Care Med. 2007;176(11):1084-1089. (Pubitemid 350223754)
21. Konstan MW, Byard PJ, Hoppel CL, Davis PB. Effect of high-dose ibuprofen in patients with cystic fibrosis. N Engl J Med. 1995;332(13):848-854.
22. Chmiel JF, Konstan MW. Inflammation and anti-inflammatory therapies for cystic fibrosis. Clin Chest Med. 2007;28(2):331-346. (Pubitemid 46654475)
23. Tirouvanziam R, Conrad CK, Bottiglieri T, Herzenberg LA, Moss RB, Herzenberg LA. High-dose oral N-acetylcysteine, a glutathione prodrug, modulates inflammation in cystic fibrosis. Proc Natl Acad Sci U S A. 2006;103(12):4628- 4633.
24. De Vizia B, Raia V, Spano C, Pavlidis C, Coruzzo A, Alessio M. Effect of an 8-month treatment with omega-3 fatty acids (eicosapentaenoic and docosahexaenoic) in patients with cystic fibrosis. JPEN J Parenter Enteral Nutr. 2003;27(1):52-57. (Pubitemid 36105644)
25. Meyer M, Huaux F, Gavilanes X, et al. Azithromycin reduces exaggerated cytokine production by M1 alveolar macrophages in cystic fibrosis. Am J Respir Cell Mol Biol. 2009;41(5):590-602.
26. Wolter JM, Seeney SL, McCormack JG. Macrolides in cystic fibrosis: is there a role? Am J Respir Med. 2002;1(4):235-241. (Pubitemid 37038942)
27. Jones AM, Helm JM. Emerging treatments in cystic fibrosis. Drugs. 2009;69(14):1903-1910.
28. Prescott WA Jr, Johnson CE. Antiinflammatory therapies for cystic fibrosis: past, present, and future. Pharmacotherapy. 2005;25(4):555-573.
29. Konstan MW, Ratjen F. Effect of dornase alfa on inflammation and lung function: potential role in the early treatment of cystic fibrosis. J Cyst Fibros. 2012;11(2):78-83.
30. Rubin BK. Mucus, phlegm, and sputum in cystic fibrosis. Respir Care. 2009;54(6):726-732.
31. Innes JA. DNase in cystic fibrosis: the challenge of assessing response and maximising benefit. Thorax. 1998;53(12):1003-1004. (Pubitemid 28566995)
32. Fuchs HJ, Borowitz DS, Christiansen DH, et al The Pulmozyme Study Group. 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. (Pubitemid 24276863)
33. Cystic Fibrosis Foundation. Patient registry: annual data report 2010. Cystic Fibrosis Foundation website. http://www.cff.org/UploadedFiles/ LivingWithCF/CareCenterNetwork/PatientRegistry/2010-Patient-Registry-Report.pdf. Accessed June 26, 2012.
34. Elkins MR, Robinson M, Rose BR, et al National Hypertonic Saline in Cystic Fibrosis (NHSCF) Study Group. A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis. N Engl J Med. 2006;354(3):229-240. (Pubitemid 43113028)
35. Aitken ML, Bellon G, De Boeck K, et al; CF302 Investigators. Long-term inhaled dry powder mannitol in cystic fibrosis: an international randomized study. Am J Respir Crit Care Med. 2012;185(6):645-652.
36. Bilton D, Robinson P, Cooper P, et al; CF301 Study Inves tigators. Inhaled dry powder mannitol in cystic fibrosis: an efficacy and safety study. Eur Respir J. 2011;38(5):1071-1080.
37. Donaldson SH, Boucher RC. Sodium channels and cystic fibrosis. Chest. 2007;132(5):1631-1636. (Pubitemid 350156022)
38. Rommens JM, Iannuzzi MC, Kerem B, et al. Identification of the cystic fibrosis gene: chromosome walking and jumping. Science. 1989;245(4922):1059- 1065. (Pubitemid 19231813)
39. Riordan JR, Rommens JM, Kerem B, et al. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science. 1989;245(4922):1066-1073. (Pubitemid 19231814)
40. Kerem B, Rommens JM, Buchanan JA, et al. Identification of the cystic fibrosis gene: genetic analysis. Science. 1989;245(4922):1073-1080. (Pubitemid 19231815)
41. Oakland M, Sinn PL, McCray PB Jr. Advances in cell and gene-based therapies for cystic fibrosis lung disease. Mol Ther. 2012;20(6):1108-1115.
42. Rogers CS, Stoltz DA, Meyerholz DK, et al. Disruption of the CFTR gene produces a model of cystic fibrosis in newborn pigs. Science. 2008;321(5897):1837-1841.
43. Sun X, Sui H, Fisher JT, et al. Disease phenotype of a ferret CFTR-knockout model of cystic fibrosis. J Clin Invest. 2010;120(9):3149-3160.
44. Van Goor F, Hadida S, Grootenhuis PD, et al. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770. Proc Natl Acad Sci U S A. 2009;106(44):18825-18830.
45. Van Goor F, Hadida S, Grootenhuis PD, et al. Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809. Proc Natl Acad Sci U S A. 2011;108(46):18843-18848.
46. Clancy JP, Rowe SM, Accurso FJ, et al. Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation. Thorax. 2012;67(1):12-18.
47. Vertex corrects and provides additional data from recent interim analysis of phase 2 combination study of VX-809 and KALYDECO™ (ivacaftor) in people with cystic fibrosis who have two copies of the F508del mutation. Vertex Pharmaceuticals Inc website. http://investors.vrtx.com/release detail.Cfm?Releaseid 5 677520. Published May 29, 2012. Accessed June 26, 2012.
48. Kerem B, Chiba-Falek O, Kerem E. Cystic fibrosis in Jews: frequency and mutation distribution. Genet Test. 1997;1(1):35-39. (Pubitemid 127526935)
49. Top-line data from phase 3 trial of ataluren in patients with nonsense mutation cystic fibrosis show promising results. PTC Therapeutics, Inc website. http://ptct.Client.Shareholder.Com/releasedetail.Cfm?Releaseid 5 681445. Published June 8, 2012. Accessed June 26, 2012.
50. Pillarisetti N, Williamson E, Linnane B, et al Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST CF). Infection, inflammation, and lung function decline in infants with cystic fibrosis. Am J Respir Crit Care Med. 2011;184(1):75-81.
51. Ranganathan SC, Parsons F, Gangell C, Brennan S, Stick SM, Sly PD; Australian Respiratory Early Surveillance Team for Cystic Fibrosis. Evolution of pulmonary inflammation and nutritional status in infants and young children with cystic fibrosis. Thorax. 2011;66(5):408-413.
52. Gangell C, Gard S, Douglas T, et al; AREST CF. Inflammatory responses to individual microorganisms in the lungs of children with cystic fibrosis. Clin Infect Dis. 2011;53(5):425-432.