Cystic Fibrosis (CF) is a genetic condition for which there is no cure. It affects the lungs and digestive system. The lungs of people with CF are chronically infected with bacteria that cause chronic infection and acute periods of worsening infection known as exacerbations. Antibiotics are used to supress chronic infection and treat acute exacerbations, but antibiotic drug resistance is problematic.

Cysteamine is a licensed drug used in the treatment of cystinosis for more than 20 years. Laboratory based work suggests that cysteamine may be a beneficial addition to conventional antibiotic treatment in CF. The pharmacokinetic characteristics of cysteamine are well established in people with cystinosis; this study aims to characterise the pharmacokinetic profile of cysteamine in people with CF, to ascertain whether cysteamine is absorbed, enters the bronchial secretions and is tolerated.

This is a single centre, single group open label study of oral cysteamine (Cystagon) in patients with Cystic Fibrosis at the dose licensed for use in cystinosis. Oral cysteamine will be increased from 450mg once daily to 450mg four times a day over three weeks, they will remain on 450mg four times a day for two weeks. People with CF who are looked after by the adult CF clinic at Aberdeen are eligible to take part. We aim to recruit 12 patients aged ≥18years with CF associated lung disease who are clinically stable. Outcomes include blood and sputum Cysteamine levels, health status, lung function and adverse reactions. Recruitment and follow-up is complete.

The study showed that oral Cysteamine was absorbed and entered the bronchial secretions in these patients. Although side effects typical of oral Cysteamine were common, the majority of patients continued to take the drug. The results of the study have been published in Clinical Drug Investigation 2016, volume 36, pages 605-612 (