A major new £60,000 clinical trial at the University of Hull is aiming to halt sarcoidosis – a potentially fatal disease that affects more than 7,000 people across the UK.
Charity SarcoidosisUK, which is funding the programme through donations to its 20th Anniversary Campaign, works to combat the disease which can strike anybody at any time and hopes the trial will prevent hundreds from facing chronic pain, organ damage or even death in the future.
Researchers from the University of Hull, working with the Hull University Teaching Hospitals NHS Trust, have high hopes for the new trial which aims to use an existing drug to stop sarcoidosis in its tracks and prevent anyone else from having their life ruined by the disease. The trial is launched during Sarcoidosis Month in April and will run throughout the rest of 2019.
Sarcoidosis is an auto-immune disease characterised by the formation of inflammatory cells (called granulomas) in affected organs. Each patient’s sarcoidosis is unique. It can affect any organ and, while some sarcoidosis patients are able to live reasonably normal lives, many others endure chronic pain, organ damage and a very poor quality of life. Sarcoidosis is fatal in around 5% of cases, usually when the heart or brain is affected.
Treatment options are limited and rely on suppressing the immune system. However this technique often has adverse side effects or is ineffective. New treatment options are therefore urgently required.
Treatments are limited due to the lack of understanding about the causes of sarcoidosis. One question that continues to evade the scientific community surrounds the formation of granulomas – the sites of inflammation that cause organ damage.
In 2017, there was a potential breakthrough. Research from the University of Vienna suggested granuloma are formed by the activation of a protein called mTOR. This finding suggests that suppressing the mTOR pathway in sarcoidosis patients could provide the elusive therapy that patients and clinicians have been waiting for.
Dr Simon Hart is an immunologist at the University of Hull and the Hull University Teaching Hospitals NHS Trust who leads the Hull Sarcoidosis Service. He identified that the antibiotic Azithromycin is known to reduce mTOR activity and is safe to use with sarcoidosis patients. Moreover the drug is already widely available and is used to treat other conditions. SarcoidosisUK have therefore partnered up with Dr Hart and his team to fund a clinical trial to test the theory that Azithromycin could be used to treat sarcoidosis.
Dr Hart’s team will measure mTOR activation, blood samples, lung function and cough in around 30 sarcoidosis patients from the Hull Sarcoidosis Clinic who have been treated with Azithromycin.
Dr Hart says: “We are delighted that SarcoidosisUK will be funding our trial to examine whether the macrolide drug azithromycin, which has combined antibiotic and immunomodulatory properties, has any beneficial effects in sarcoidosis. To look for ‘proof of principle’ we will study changes in clinical outcomes and anti-inflammatory activity in blood samples using novel cell-based assays that we have developed in our research laboratory.”
Henry Shelford, SarcoidosisUK Chair, thanked everyone who donated and made the trial possible and says: “We are very excited because we have the opportunity to re-purpose an existing drug which is relatively cheap and approved for use in the UK. This means that we can bypass the length and costly drug development stages.
“If Dr Hart’s trial is successful we will have made huge strides into finding a cure for sarcoidosis.”
The research project will be conducted during 2019 by a multidisciplinary immunology team based at Hull Sarcoidosis Service. The results are hoped to be published in 2020 and, in anticipation of positive results, further funding sought for a larger scale trial.
The mTOR trial follows on from previous sarcoidosis research conducted by Dr Hart’s team and funded by SarcoidosisUK. In 2015 the SarcoidosisUK-BLF Sarcoidosis Research Grant awarded £116,000 to investigating protein molecules. The results from that study will be published later this year.