Cystic fibrosis (CF) is a common autosomal recessive inherited disorder affecting 1 in 2,500 births and ∼75,000 people in North America, Europe, and Australia. The purpose of this review is to discuss the most recent advances in therapies for CF patients. The CF drug development pipeline provides information regarding the phase of each new therapy and those that are already in use by patients. The ultimate goal of therapy will be to deliver tailored individualized therapies to those who need it the most. This review will focus on new research on CF transmembrane conductance regulator protein modulator agents and the effort of gene therapy.
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References
1.
JenningsM, RiekertK, BoyleM. Update on key emerging challenges in cystic fibrosis. Med Princ Pract, 2014 [Epub ahead of print]; DOI: 10.1159/000357646
Sermet-GaudelusI. Ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation. Eur Respir Rev, 2013; 22:66–71.
4.
RamseyBW, DaviesJ, McElvaneyNG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med, 2011; 365:1663–1672.
5.
DaviesJ, YenK, AhrensR. Ivacaftor in subjects 6 to 11 years of age with cystic fibrosis and the G551D-CFTR mutation. J Cyst Fibros, 2012; 11:Suppl. 1:S13.
6.
McKoneELH, YenK, DaviesJC. Long-term safety and efficacy of ivacaftor in subjects with cystic fibrosis who have the G551D-CFTR mutation. J Cyst Fibros, 2012; 11:Suppl. 1:S13.
7.
ClancyJP, RoweSM, AccursoFJ, 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; 67:12–18.
ArmstrongDK, CunninghamS, DaviesJC, et al. Gene therapy in cystic fibrosis. Arch Dis Child, 2014; 99:465–468.
12.
AltonEW, MiddletonPG, CaplenNJ, et al. Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice. Nat Genet, 1993; 5:312.
BallR, SouthernKW, McCormackP, et al. Adherence to nebulized therapies in adolescents with cystic fibrosis is best on week-days during school term-time. J Cyst Fibros, 2013; 12:440–444.
15.
McNamaraPS, McCormackP, McDonaldAJ, et al. Open adherence monitoring using routine data download from an adaptive aerosol delivery nebulizer in children with cystic fibrosis. J Cyst Fibros, 2009; 8:258–263.
16.
EakinMN, BilderbackA, BoyleMPet al. Longitudinal association between medication adherence and lung health in people with cystic fibrosis. J Cyst Fibros, 2011; 10:258–264.
