Abstract
Introduction:
Several types of mutations in the cystic fibrosis transmembrane regulator (CFTR) gene lead to abnormal CFTR protein and alterations of chloride and sodium transmembrane transportation in cystic fibrosis (CF). Some investigational compounds such as VX-770 can improve CFTR protein function.
Areas covered:
This paper discusses the results of a Phase II study investigating the safety and efficacy of VX-770 in patients with CF.
Expert opinion:
VX-770 is able to improve chloride and sodium transportation and has a good safety profile. Although such compounds have limited therapeutic targeting potential, preliminary results show great promise in the context of CF therapy.
Publication types
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Clinical Trial, Phase II
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Controlled Clinical Trial
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Multicenter Study
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Comment
MeSH terms
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Adolescent
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Adult
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Aged
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Aged, 80 and over
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Aminophenols / adverse effects
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Aminophenols / therapeutic use*
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Cell Membrane / metabolism
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Cystic Fibrosis / drug therapy*
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Cystic Fibrosis / genetics
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Cystic Fibrosis / metabolism
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Cystic Fibrosis Transmembrane Conductance Regulator / genetics
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Cystic Fibrosis Transmembrane Conductance Regulator / metabolism*
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Double-Blind Method
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Humans
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Ion Transport / drug effects
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Middle Aged
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Mutation
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Quinolones / adverse effects
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Quinolones / therapeutic use*
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Young Adult
Substances
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Aminophenols
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Quinolones
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Cystic Fibrosis Transmembrane Conductance Regulator
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ivacaftor