Researchers in Leeds are hoping for a breakthrough in the treatment of the devastating immune system disease scleroderma, thanks to the support of a medical research charity.
Scleroderma is an inflammatory disease which leads to the scarring and thickening of the skin and internal organs.
Though rare, it can have a devastating effect on patients’ lives. Some suffer highly debilitating symptoms – their hands and fingers become permanently stiff and clenched, their muscles and internal organs are damaged, they can suffer shortness of breath, kidney failure and pulmonary hypertension. Once patients get to this stage the condition is not reversible and long-term prognosis is very poor.
The new research programme has two main aims – to find tell-tale markers which will allow doctors to identify patients most at risk of developing the disease and to find the processes at work in those patients which have the potential to be blocked by drugs.
The work has been supported by a grant of £470,000 from The Kennedy Trust for Rheumatology Research and further support from the University of Leeds. Established in 1965 by Mathilda Kennedy – the daughter of M&S founder Michael Marks – the Kennedy Trust provides financial support for research into rheumatic and related musculoskeletal, immunological and inflammatory diseases.
“We are really grateful for this support,” says Dr Francesco Del Galdo, who sees patients from across the region in his clinic at Chapel Allerton Hospital, part of Leeds Teaching Hospitals NHS Trust. “Right now we don’t know what triggers the scarring process. If we can get a clearer understanding of this, we can then search for the molecules that might actually prevent the scarring.
“And if we can develop a test to identify patients at risk, we can concentrate on giving early treatments to those most urgently in need.”
During the three-year trial Dr Del Galdo will work with colleagues from London and Manchester to establish a cohort of 400 patients identified as being at risk of developing scleroderma. Studying these patients will help the researchers to determine which methods of diagnosis – both scanning and blood tests – provide the most accurate picture of the patient’s future. At the same time, the researchers will seek out the possible future targets for drugs.
The clinical, biological and lifestyle data gathered from these patients – alongside information about which of them went on to develop scleroderma – will also become a rich resource for future studies into the disease, both in the UK and overseas.
“At present, patients with the most aggressive forms of scleroderma have a very poor prognosis,” says Dr Del Galdo. “The support of the Kennedy Trust will hopefully enable us to identify patients before they suffer irreversible organ damage, and make early interventions which will stop this disease in its tracks.”
Pierre Espinasse, General Manager of the Kennedy Trust says: “We are very pleased to support Dr Del Galdo’s research into what is an important area of rheumatology and which could significantly advance treatment of early scleroderma.”
Based at Chapel Allerton, the Leeds Institute of Rheumatic and Musculoskeletal Medicine and the NIHR Leeds Biomedical Research Centre are world-leading research groups dedicated to improving diagnosis, therapy, intervention and outcomes across the spectrum of rheumatic and musculoskeletal medicine.
Its work exemplifies ‘bench to bedside’ research, which is made possible by a close working relationship between the Leeds Teaching Hospitals NHS Trust and the University of Leeds. Through this partnership, researchers involved in the search for new treatments also work with patients visiting our clinics. This personal contact brings renewed motivation to academics who are working to transform their research advances into new treatments to improve the lives of patients.
The research continues a long-standing relationship between the University of Leeds and the Marks family. Marks & Spencer was founded in the city in 1884; the company’s archive is now housed in the Michael Marks Building on the University campus.