141 An open label investigation of the tolerability and pharmacokinetics of oral cysteamine in adults with cystic fibrosis

Devereux, Graham; Steele, Sandra; Griffiths, Kairen; Cotton, Seonaidh; Fraser-Pitt, D.; Devlin, E.; Chrystyn, Henry; Norrie, John; O’Neil, Deborah A.

doi:10.1016/s1569-1993(15)30318-0

Journal of Cystic Fibrosis

2015

DOI: 10.1016/s1569-1993(15)30318-0

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141 An open label investigation of the tolerability and pharmacokinetics of oral cysteamine in adults with cystic fibrosis

Graham Devereux

Sandra Steele

Kairen Griffiths

et al.

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Rapid therapeutic advances in CFTR modulator science

Clancy

2018

Pediatric Pulmonology

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Cystic fibrosis (CF) is an autosomal recessive genetic disease caused by variants in the gene encoding the cystic fibrosis transmembrane conduction regulator (CFTR) protein. Loss of CFTR function disrupts chloride, bicarbonate and regulation of sodium transport, producing a cascade of mucus obstruction, inflammation, pulmonary infection, and ultimately damage in numerous organs. Established CF therapies treat the downstream consequences of CFTR dysfunction and have led to steady improvements in patient survival. A class of drugs termed CFTR modulators has recently entered the CF therapeutic landscape. These drugs differ fundamentally from prior therapies in that they aim to improve the function of disease‐causing CFTR variants. This review summarizes the science behind CFTR modulators, including their targets, mechanism of action, clinical benefit, and future directions in the field. CFTR modulators have dramatically changed how CF is treated, validated CFTR as a therapeutic target, and opened the door to truly personalized therapies and treatment regimens.

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Rapid therapeutic advances in CFTR modulator science

Clancy

2018

Pediatric Pulmonology

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An update on new and emerging therapies for cystic fibrosis

Hudock

Clancy

2017

Expert Opinion on Emerging Drugs

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Cystic fibrosis (CF) is a genetic disorder that results in a multi-organ disease with progressive respiratory decline that ultimately leads to premature death. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which codes for the CFTR anion channel. Established CF treatments target downstream manifestations of the primary genetic defect, including pulmonary and nutritional interventions. Areas covered: CFTR modulators are novel therapies that improve the function of CFTR, and have been approved in the past five years to mitigate the effects of several CF-disease causing mutations. This review summarizes currently approved CFTR modulators and discusses emerging modulator therapies in phase II and III clinical trials described on clinical trials.gov as of April, 2017. Results of relevant trials reported in peer-reviewed journals in Pubmed, scientific conference abstracts and sponsor press releases available as of November, 2017 are included. Expert opinion: The current scope of CF therapeutic development is robust and CFTR modulators have demonstrated significant benefit to patients with specific CFTR mutations. We anticipate that in the future healthcare providers will be faced with a different treatment paradigm, initiating CFTR-directed therapies well before the onset of progressive lung disease.

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141 An open label investigation of the tolerability and pharmacokinetics of oral cysteamine in adults with cystic fibrosis

Cited by 2 publications

References 0 publications

Rapid therapeutic advances in CFTR modulator science

Rapid therapeutic advances in CFTR modulator science

An update on new and emerging therapies for cystic fibrosis

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