Exploring the uses of inhaled antibiotics in the treatment of Cystic Fibrosis

Dr Jo Fothergill, part of the Microbiome Innovation Centre management team, has been working in partnership with the Liverpool Heart and Chest Hospital researching potential new treatments for people with Cystic Fibrosis (CF).

The research project, funded by Gilead, looked into the clinical and microbiological outcomes of inhaled treatments for sufferers of CF, comparing the results to that of the usual intravenous antibiotics treatment.


Cystic Fibrosis (CF) is a genetic condition, affecting over 10,000 people in the UK. The gene affected by CF controls the movement of salt and water in and out of cells. People with the condition experience a build-up of thick sticky mucus in the lungs, digestive system and other organs, causing a wide range of challenging symptoms.

The natural course of the condition is one of chronic progression with intermittent episodes of acute worsening of symptoms, which are commonly known as pulmonary exacerbations.

There is no cure for CF, but a range of treatments can help control the symptoms and prevent or reduce complications, making the condition easier to live with. Intravenous (IV) antibiotics are commonly used to help control the pulmonary exacerbations that are common in chronic lung infections.


The trial explored the use of inhaled antibiotics such as Inhaled Aztreonam Lysine (AZLI). These inhaled antibiotics target the end organ (the lung) directly and achieve high sputum concentrations with minimal systemic exposure, making them an attractive potential therapeutic strategy for pulmonary exacerbations.

The study consisted of an open-label randomised crossover trial. Patients usually tend to suffer from pulmonary exacerbations regularly, this gave researchers the chance to give each patient two different types of treatment, allowing the group to compare results of different treatments on the same patient.

Over the course of two exacerbations, participants were randomised to sequentially receive 14 days of inhaled antibiotics (AZLI), or dual IV antibiotics (two doses of IV treatment). As part of the clinical trial, changes in the lung microbiome were studied and compared between treatments.


In adults with CF suffering from pulmonary exacerbations, the treatment of inhaled treatments improved lung function and quality of life compared to standard treatment. These findings support the need for larger definitive trials of inhaled antibiotics in the acute setting. 

Dr Freddy Frost, who led the trial said: “We found that the inhaled treatment was associated with positive clinical outcomes and appeared to disturb the core lung microbiota less than IV antibiotics. This could represent a new therapeutic approach to the management of exacerbations in people with CF and paves the way for larger trials to look into this.”

The Microbiome Innovation Centre has an international reputation for academic research and industry collaboration, undertaking a broad range of cutting-edge projects across a wide range of subjects. Read more about our research expertise in Human microbiome here.

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