Emerging drug treatments for cystic fibrosis

Bibliography

• ZEITLIN PL: Emerging drug treatments for cystic fibrosis. Expert. Opin. Emerg. Drugs (2003) 8:523-535.
   PubMedGoogle Scholar
• LIOU TG, WOO MS, CAHILL BC: Lung transplantation for cystic fibrosis. Curr. Opin. Pulm. Med. (2006) 12:459-463.
   PubMed Web of Science ®Google Scholar
• KONSTAN MW, BUTLER SM, SCHIDLOW DV, MORGAN WJ, JULIUS JR, JOHNSON CA: Patterns of medical practice in cystic fibrosis:part II. Use of therapies. Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis. Pediatr. Pulmonol. (1999) 28:248-254.
   PubMed Web of Science ®Google Scholar
• ZUERCHER AW, HORN MP, QUE JU et al.: Antibody responses induced by long-term vaccination with an octovalent conjugate Pseudomonas aeruginosa vaccine in children with cystic fibrosis. FEMS Immunol. Med. Microbiol. (2006) 47:302-308.
   PubMed Web of Science ®Google Scholar
• BERKOWITZ BA, BEVINS CL, ZASLOFF MA: Magainins: a new family of membrane-active host defense peptides. Biochem. Pharmacol. (1990) 39:625-629.
   PubMed Web of Science ®Google Scholar
• DIAMOND G, RUSSELL JP, BEVINS CL: Inducible expression of an antibiotic peptide gene in lipopolysaccharide-challenged tracheal epithelial cells.. Proc. Natl. Acad. Sci. USA (1996) 93:5156-5160.
   PubMed Web of Science ®Google Scholar
• GANZ T, WEISS J: Antimicrobial peptides of phagocytes and epithelia [In Process citation]. Semin. Hematol. (1997) 34:343-354.
   PubMed Web of Science ®Google Scholar
• PHADKE SM, DESLOUCHES B, HILEMAN SE, MONTELARO RC, WIESENFELD HC, MIETZNER TA: Antimicrobial peptides in mucosal secretions: the importance of local secretions in mitigating infection. J. Nutr. (2005) 135:1289-1293.
   PubMed Web of Science ®Google Scholar
• ZASLOFF M: Magainins, a class of antimicrobial peptides from Xenopus skin: isolation, characterization of two active forms, and partial cDNA sequence of a precursor. Proc. Natl. Acad. Sci.USA (1987) 84:5449-5453.
   PubMed Web of Science ®Google Scholar
• ZASLOFF M, MARTIN B, CHEN HC: Antimicrobial activity of synthetic magainin peptides and several analogues. Proc. Natl. Acad. Sci. USA (1988) 85:910-913.
   PubMed Web of Science ®Google Scholar
• KEREM E, CONWAY S, ELBORN S, HEIJERMAN H: Standards of care for patients with cystic fibrosis: a European consensus. J. Cyst. Fibros. (2005) 4:7-26.
   PubMedGoogle Scholar
• DORING G, HOIBY N: Early intervention and prevention of lung disease in cystic fibrosis: a European consensus. J. Cyst. Fibros. (2004) 3:67-91.
   PubMedGoogle Scholar
• KONSTAN MW, BUTLER SM, SCHIDLOW DV, MORGAN WJ, JULIUS JR, JOHNSON CA: Patterns of medical practice in cystic fibrosis: part I. Evaluation and monitoring of health status of patients. Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis. Pediatr. Pulmonol. (1999) 28:242-247.
   PubMed Web of Science ®Google Scholar
• KONSTAN MW, DAVIS PB, WAGENER JS et al.: Compacted DNA nanoparticles administered to the nasal mucosa of cystic fibrosis subjects are safe and demonstrate partial to complete cystic fibrosis transmembrane regulator reconstitution. Hum. Gene Ther. (2004) 15:1255-1269.
   PubMed Web of Science ®Google Scholar
• HAMED SA: Drug evaluation: PTC-124 – a potential treatment of cystic fibrosis and Duchenne muscular dystrophy. IDrugs (2006) 9:783-789.
   PubMed Web of Science ®Google Scholar
• RUBENSTEIN RC, EGAN ME, ZEITLIN PL: In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta F508-CFTR. J. Clin. Invest. (1997) 100:2457-2465.
   PubMed Web of Science ®Google Scholar
• ZEITLIN PL, DIENER-WEST M, RUBENSTEIN RC, BOYLE MP, LEE CK, BRASS-ERNST L: Evidence of CFTR function in cystic fibrosis after systemic administration of 4-phenylbutyrate. Mol. Ther. (2002) 6:19-126.
   PubMed Web of Science ®Google Scholar
• EGAN ME, GLOCKNER-PAGEL J, AMBROSE CA et al.: Calcium-pump inhibitors induce functional surface expression of delta F508-CFTR protein in cystic fibrosis epithelial cells. Nat. Med. (2002) 8:485-492.
   PubMed Web of Science ®Google Scholar
• EGAN ME, PEARSON M, WEINER SA et al.: Curcumin, a major constituent of turmeric, corrects cystic fibrosis defects. Science (2004) 304:600-602.
   PubMed Web of Science ®Google Scholar
• DRAGOMIR A, BJORSTAD J, HJELTE L, ROOMANS GM: Curcumin does not stimulate cAMP-mediated chloride transport in cystic fibrosis airway epithelial cells. Biochem. Biophys. Res. Commun. (2004) 322:447-451.
   PubMed Web of Science ®Google Scholar
• SONG Y, SONAWANE ND, SALINAS D et al.: Evidence against rescue of defective deltaF508-CFTR cellular processing by curcumin in cell culture and mouse models. J. Biol. Chem. (2004) 279(39):40629-40635.
   PubMed Web of Science ®Google Scholar
• MA T, VETRIVEL L, YANG H et al.:High-affinity activators of cystic fibrosis transmembrane conductance regulator (CFTR) chloride conductance identified by high-throughput screening. J. Biol. Chem. (2002) 277:37235-37241.
   PubMed Web of Science ®Google Scholar
• MA T, THIAGARAJAH JR, YANG H et al.: Thiazolidinone CFTR inhibitor identified by high-throughput screening blocks cholera toxin-induced intestinal fluid secretion. J. Clin. Invest. (2002) 110:1651-1658.
   PubMed Web of Science ®Google Scholar
• VAN GOOR F, STRALEY KS, CAO D et al.: Rescue of deltaF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules. Am. J. Physiol. Lung Cell Mol. Physiol. (2006) 290:L1117-L1130.
   PubMed Web of Science ®Google Scholar
• DETERDING R, RETSCH-BOGART G, MILGRAM L et al.: Safety and tolerability of denufosol tetrasodium inhalation solution, a novel P2Y2 receptor agonist: results of a Phase 1/Phase 2 multicenter study in mild to moderate cystic fibrosis. Pediatr. Pulmonol. (2005) 39:339-348.
   PubMed Web of Science ®Google Scholar
• ZEITLIN PL, BOYLE MP, GUGGINO WB, MOLINA L: A Phase I trial of intranasal Moli1901 for cystic fibrosis. Chest (2004) 125:143-149.
   PubMed Web of Science ®Google Scholar
• ROGERS DF: Pulmonary mucus: pediatric perspective. Pediatr. Pulmonol. (2003) 36:178-188.
   PubMed Web of Science ®Google Scholar
• ROSSI L, CASTRO M, D'ORIO F et al.: Low doses of dexamethasone constantly delivered by autologous erythrocytes slow the progression of lung disease in cystic fibrosis patients. Blood Cells Mol. Dis. (2004) 33:57-63.
   PubMed Web of Science ®Google Scholar
• LUCIDI V, TOZZI AE, BELLA S, TURCHETTA A: A pilot trial on safety and efficacy of erythrocyte-mediated steroid treatment in CF patients. BMC Pediatr. (2006) 6:17.
   PubMedGoogle Scholar
• ELKINS MR, ROBINSON M, ROSE BR et al.: A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis. N. Engl. J. Med. (2006) 354:229-240.
   PubMed Web of Science ®Google Scholar
• BOUCHER RC: Evidence for airway surface dehydration as the initiating event in CF airway disease. J. Intern. Med. (2007) 26:15-16.
   Web of Science ®Google Scholar
• WILLS P, GREENSTONE M: Inhaled hyperosmolar agents for bronchiectasis. Cochrane Database Syst. Rev. (2006):CD002996.
   Google Scholar
• DAVISKAS E, ANDERSON SD: Hyperosmolar agents and clearance of mucus in the diseased airway. J. Aerosol Med. (2006) 19:100-109.
   PubMedGoogle Scholar
• APP EM, BARAN D, DAB I et al.: Dose-finding and 24-h monitoring for efficacy and safety of aerosolized Nacystelyn in cystic fibrosis. Eur. Respir. J. (2002) 19:294-302.
   PubMed Web of Science ®Google Scholar
• APP EM, ZAYAS JG, KING M: Rheology of mucus and transepithelial potential difference: small airways versus trachea. Eur. Respir. J. (1993) 6:67-75.
   PubMed Web of Science ®Google Scholar
• APP EM, KING M: Tracheal mucus rheology and epithelial potential difference in two day old puppies. Biorheology (1990) 27:515-526.
   PubMed Web of Science ®Google Scholar
• SERISIER DJ, SHUTE JK, HOCKEY PM, HIGGINS B, CONWAY J, CARROLL MP: Inhaled heparin in cystic fibrosis. Eur. Respir. J. (2006) 27:354-358.
   PubMed Web of Science ®Google Scholar
• APP EM, KING M, HELFESRIEDER R, KOHLER D, MATTHYS H: Acute and long-term amiloride inhalation in cystic fibrosis lung disease. A rational approach to cystic fibrosis therapy. Am. Rev. Respir. Dis. (1990) 141:605-612.
   PubMed Web of Science ®Google Scholar
• KNOWLES MR, CHURCH NL, WALTNER WE et al.: A pilot study of aerosolized amiloride for the treatment of lung disease in cystic fibrosis [see comments]. N. Engl. J. Med. (1990) 322:1189-1194.
   PubMed Web of Science ®Google Scholar
• KNOWLES MR, STUTTS MJ, SPOCK A, FISCHER N, GATZY JT, BOUCHER RC: Abnormal ion permeation through cystic fibrosis respiratory epithelium. Science (1983) 221:1067-1070.
   PubMed Web of Science ®Google Scholar
• KOHLER D, APP E, SCHMITZ-SCHUMANN M, WURTEMBERGER G, MATTHYS H: Inhalation of amiloride improves the mucociliary and the cough clearance in patients with cystic fibroses. Eur. J. Respir. Dis. Suppl. (1986) 146:319-326.
   PubMedGoogle Scholar
• PONS G, MARCHAND MC, D'ATHIS P et al.: French multicenter randomized double-blind placebo-controlled trial on nebulized amiloride in cystic fibrosis patients. The Amiloride-AFLM Collaborative Study Group. Pediatr. Pulmonol. (2000) 30:25-31.
   PubMed Web of Science ®Google Scholar
• GRAHAM A, HASANI A, ALTON EW et al.: No added benefit from nebulized amiloride in patients with cystic fibrosis. Eur. Respir. J. (1993) 6:1243-1248.
   PubMed Web of Science ®Google Scholar
• BOWLER IM, KELMAN B, WORTHINGTON D et al.: Nebulised amiloride in respiratory exacerbations of cystic fibrosis: a randomised controlled trial. Arch. Dis. Child. (1995) 73:427-430.
   PubMed Web of Science ®Google Scholar
• HIRSH AJ, MOLINO BF, ZHANG J et al.: Design, synthesis, and structure–activity relationships of novel 2-substituted pyrazinoylguanidine epithelial sodium channel blockers: drugs for cystic fibrosis and chronic bronchitis. J. Med. Chem. (2006) 49:4098-4115.
   PubMed Web of Science ®Google Scholar
• HIRSH AJ, SABATER JR, ZAMURS A et al.: Evaluation of second generation amiloride analogs as therapy for cystic fibrosis lung disease. J. Pharmacol. Exp. Ther. (2004) 311:929-938.
   PubMed Web of Science ®Google Scholar
• GIBSON RL, RETSCH-BOGART GZ, OERMANN C et al.: Microbiology, safety, and pharmacokinetics of aztreonam lysinate for inhalation in patients with cystic fibrosis. Pediatr. Pulmonol. (2006) 41:656-665.
   PubMed Web of Science ®Google Scholar
• CHILDERS M, ECKEL G, HIMMEL A, CALDWELL J: A new model of cystic fibrosis pathology: lack of transport of glutathione and its thiocyanate conjugates. Med. Hypotheses (2007) 68:101-112.
   PubMed Web of Science ®Google Scholar
• KOGAN I, RAMJEESINGH M, LI C et al.: CFTR directly mediates nucleotide-regulated glutathione flux. EMBO J. (2003) 22:1981-1989.
   PubMed Web of Science ®Google Scholar
• 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. USA (2006) 103:4628-4633.
   PubMed Web of Science ®Google Scholar
• ZAMAN K, CARRARO S, DOHERTY J et al.: S-nitrosylating agents: a novel class of compounds that increase cystic fibrosis transmembrane conductance regulator expression and maturation in epithelial cells. Mol. Pharmacol. (2006) 70:1435-1442.
   PubMed Web of Science ®Google Scholar
• SERVETNYK Z, KRJUKOVA J, GASTON B et al.: Activation of chloride transport in CF airway epithelial cell lines and primary CF nasal epithelial cells by S-nitrosoglutathione. Respir. Res. (2006) 7:124.
   PubMed Web of Science ®Google Scholar
• THOMAS SR, KHARITONOV SA, SCOTT SF, HODSON ME, B ARNES PJ: Nasal and exhaled nitric oxide is reduced in adult patients with cystic fibrosis and does not correlate with cystic fibrosis genotype. Chest (2000) 117:1085-1089.
   PubMed Web of Science ®Google Scholar
• GRASEMANN H, SCHWIERTZ R, MATTHIESEN S, RACKE K, RATJEN F: Increased arginase activity in cystic fibrosis airways. Am. J. Respir. Crit. Care Med. (2005) 172:1523-1528.
   PubMed Web of Science ®Google Scholar
• GRASEMANN H, GRASEMANN C, KURTZ F, TIETZE-SCHILLINGS G, VESTER U, RATJEN F: Oral l-arginine supplementation in cystic fibrosis patients: a placebo-controlled study. Eur. Respir. J. (2005) 25:62-68.
   PubMed Web of Science ®Google Scholar
• GRASEMANN H, KURTZ F, RATJEN F: Inhaled l-arginine improves exhaled nitric oxide and pulmonary function in patients with cystic fibrosis. Am. J. Respir. Crit. Care Med. (2006) 174:208-212.
   PubMed Web of Science ®Google Scholar
• MOODY M, PENNINGTON C, SCHULTZ C et al.: Inositol polyphosphate derivative inhibits Na+ transport and improves fluid dynamics in cystic fibrosis airway epithelia. Am. J. Physiol. Cell Physiol. (2005) 289:C512-C520.
   PubMed Web of Science ®Google Scholar
• BOROWITZ D, GOSS CH, STEVENS C et al.: Safety and preliminary clinical activity of a novel pancreatic enzyme preparation in pancreatic insufficient cystic fibrosis patients. Pancreas (2006) 32:258-263.
   PubMed Web of Science ®Google Scholar
• BOROWITZ D, GOSS CH, LIMAURO S et al.: Study of a novel pancreatic enzyme replacement therapy in pancreatic insufficient subjects with cystic fibrosis. J. Pediatr. (2006) 149:658-662.
   PubMed Web of Science ®Google Scholar
• GOSS CH, MAYER-HAMBLETT N, KRONMAL RA, RAMSEY BW: The Cystic Fibrosis Therapeutics Development Network (CF TDN): a paradigm of a clinical trials network for genetic and orphan diseases. Adv. Drug Deliv. Rev. (2002) 54:1505-1528.
   PubMed Web of Science ®Google Scholar