New study could help find a treatment for scleroderma-related fibrosis

Background

Fibrosis is usually a normal bodily response to injury, in which specialised cells known as fibroblasts deposit collagen to help repair damage. People with systemic sclerosis experience excessive fibrosis. This may lead to thickening of the skin and ‘scarring’ of internal organs in the body which contributes to a range of debilitating effects observed in systemic sclerosis.

The mechanisms which cause fibrosis are becoming better understood through research. Professor Richard Stratton, of the Royal Free Hospital London, received two SRUK research grants in this area. These projects confirmed that a certain type of immune cell known as the ‘macrophage’ can cause fibrosis in systemic sclerosis by sending pro-fibrotic ‘signals’ to fibroblasts; and that a molecule developed by collaborators in the United States can prevent these processes, meaning it has potential as a new treatment for fibrosis in scleroderma.  

What is happening now?

Prof Stratton is now working with a pharmaceutical company who are interested in progressing this molecule to clinical trial. Clinical trials within rare diseases like scleroderma are tricky due to the small number of patients affected and the molecular differences in the disease between individual patients.   

Before progressing to a clinical trial Dr Stratton’s team must carry out further research to identify the exact target patient group(s) to be included in any future trial, refine the clinical trial methodology and the precise data to be collected. To do this the team need blood and skin samples from individuals with diffuse systemic sclerosis at different stages of the disease to carry out their in-depth molecular studies.    

Yes, I’m interested – what’s involved?  

Patients with diffuse systemic sclerosis will be invited to attend the Royal Free Hospital on two consecutive days. Travel expenses and accommodation costs will be paid to enable participation from across the UK.  

On day one, a small (4mm or approx. 1/8 inch) suction blister will be induced on the forearm skin of participants. On day two, participants will return and the fluid from this blister will be extracted for laboratory analysis and provide blood samples. A small number of participants may be asked to provide a small skin biopsy (4mm diameter). Participants will not receive any treatment above usual standard of care, but any individuals judged as being potentially suitable candidates for this line of treatment may be invited to participate in any eventual clinical trial.

When and how can I take part? 

Prof Stratton and team are expecting to start this research in December 2022.

To find out more and register your interest for this study please contact r.stratton@ucl.ac.uk and sandra.garces.19@ucl.ac.uk using email subject line SRUK Macrophages in Scleroderma, or by telephone to discuss further 020 8016 8332.