A worldwide research trial to find a new drug for a hard-to-treat lung disease is underway at Royal Papworth Hospital NHS Foundation Trust.
Sarcoidosis is a lung disease of unknown cause that affects about 7,000 ‘typically younger’ people in the UK and causes long-term ill health.
It is thought to be due to an abnormality in the immune system. It causes lumps of tissue, called granulomas, to build up in the lungs but these can affect any part of the body including the eyes, skin, brain and heart.
Some patients need steroids long term which can improve their lungs but can have long term side effects such as increased risk of diabetes and bone disease.
This trial, the first one in the world for sarcoidosis at an advanced phase, aims to see if a new medicine can help to reduce the steroid dose, in-turn reducing the risk of side effects.
Approximately 200 patients are involved in the large study across Europe, North America and Asia.
Dr Muhunthan Thillai
“Sarcoidosis is very tricky to treat,” said Dr Muhunthan Thillai, Principal Investigator for the study and Consultant Physician at Royal Papworth Hospital, one of the largest centres in the UK for this disease with more than 300 patients.
“Steroids can be effective but also create other problems. This makes it a very complicated disease to manage as it can cause frailty and a big impact on people’s quality of life, health risks and ability to work.
“Many people affected were previously healthy individuals who, compared to other typical lung diseases, tend to be younger people in their 30s and 40s, which itself places a huge burden on the NHS.”
Simon has lived with sarcoidosis for 20 years
Former plasterer Simon Osborne, 48 from Cambridge, is Royal Papworth’s first patient to have signed up to the trial, having lived with sarcoidosis for the past 20 years.
An active teenager who played football, cycled and once considered himself physically fit, Simon also smoked and first noticed symptoms when regularly climbing stairs to visit a friend.
He explained: “I had a friend who lived in a block of flats and after a while I would get to the top of the stairs, be out of breath and think ‘something’s not right’.
“One day in 2004 I was at home with family about to have Sunday dinner, when I couldn’t get up and could barely walk.
“I was taken to Addenbrooke’s Hospital where I stayed for a month before being diagnosed with pulmonary sarcoidosis and transferred to Royal Papworth Hospital for my ongoing, long-term care.”
Simon stepped away from plastering and moved into factory assembly work, but struggled to keep up with the pace and eventually stopped work completely in 2011.
“The impact on my life since the diagnosis and subsequent long-term treatment on steroids has been huge,” he continued.
“I get tired very quickly and even general housework tires me out now.”
“The side-effects of steroids for 20 years has seen me put weight on and my bones feel weak. When I got a phone call to invite me to this trial, I said yes straightaway.
“If there’s a new drug that helps me feel better, provides me with a better quality of life and also could help others like me in the future, then I’m happy to help.”
Jenny, Clara and Ana from our Clinical Research Facility with Dr Muhunthan Thillai, Principal Investigator for the study
Watch this BBC Look East report about the research trial: