University clinical trial for pulmonary sarcoidosis treatment shows promise

A team at the University of Hull and Hull University Teaching Hospitals NHS Trust has conducted new research during a recent clinical trial.

Funded by the charity SarcoidosisUK in 2018, and led by Dr Simon Hart, Respiratory Consultant and ILD Research Specialist, the research project aimed to test the antibiotic azithromycin as a treatment for pulmonary sarcoidosis.

The first results of the clinical trial showed that after three months of azithromycin treatment, there was a significant reduction in the cough count of patients and meaningful improvements in their quality of life.

Sarcoidosis is characterised by the formation of inflammatory cells (called granulomas) in affected organs (usually the lungs and skin). For some patients, they are able to live reasonably normal lives with the condition, whilst others endure chronic pain, organ damage and a very poor quality of life.

Dr Hart said: “Chronic cough can have a distressing impact on quality of life for those suffering with pulmonary sarcoidosis. This trial’s aim was to re-purpose existing drugs as treatment options for sarcoidosis, and we found that azithromycin reduced coughing significantly without any serious side effects.”

The inflammatory cells, or granulomas, that cause sarcoidosis are formed by the activation of a protein called ‘mTOR’. Dr Hart and his team identified the fact that the antibiotic azithromycin is known to reduce mTOR activity within cells and is also safe to use within sarcoidosis patients.

As a result, the 21 participants in the trial suffering from pulmonary sarcoidosis and reporting a chronic cough, took 250mg of azithromycin once daily for 3 months.

More than half of the patients reported clinically meaningful improvements in their cough-related quality of life, while more than three quarters had clinically meaningful improvements in their sarcoidosis-related quality of life.

Dr Hart noted: “We believe that the improvements were sufficiently large to merit further investigation of azithromycin as a treatment for sarcoidosis cough.”

Mr Henry Shelford, SarcoidosisUK Chairperson, said: “This is a really exciting discovery by Dr Hart and his research partners at the University of Hull.

There is currently no known cure for the condition, and 3,000 to 4,000 people are diagnosed every year in the UK. Sarcoidosis is fatal in around 5% of cases, although this is usually limited to when the heart or the brain is affected by the disease.

Dr Hart and his researchers, backed by SarcoidosisUK, believe this is a huge step in the right direction for effective treatment of the illness, avoiding the side effects often associated with previous treatment options aimed at suppressing the immune system.

Mr Shelford added: “We want to thank everybody who has donated to or supported the charity – without you this research wouldn’t have been possible. We are delighted with the work of Dr Hart and his team, and wholeheartedly support his conclusion in relation to further trials for this promising treatment.”

SarcoidosisUK is the UK’s national sarcoidosis charity, providing information and support to anyone affected by sarcoidosis. They also raise awareness and fund research into a cure for the condition. They are committed to funding at least one major research project each year until a cure has been found and they are one of the largest sarcoidosis research-funders in the world.