Can immune suppressant drugs help to prevent worsening of systemic sclerosis?

Disease - Scleroderma

Lead applicant - Professor Christopher Denton

Organisation - University College London

Type of grant - Priorities in Clinical Research

Status of grant - Active

Amount of the original award - £496,380.43

Start date - 1 August 2019

Reference - 22398

Public Summary

What are the aims of this research?

This research will investigate whether the immune suppressant drug mycophenolate mofetil (MMF) can help to treat a subgroup of people with scleroderma. The drug is often used in more severe cases when the condition causes widespread thickening of the skin or lungs complications, however this study will test the benefit for other people less severe scleroderma. This will be a pilot study, hoping to support move towards a full clinical trial in the future.

Why is this research important?

Scleroderma, also called systemic sclerosis (SSc), is a severe autoimmune disease which has symptoms that may affect different parts of the body. These include circulation problems causing Raynaud's phenomenon and finger ulcers, changes to the skin, and more serious complications such as lung fibrosis.

There are two main types of scleroderma; diffuse scleroderma which causes widespread skin thickening and limited scleroderma which has less severe skin thickening. Patients with diffuse scleroderma are often treated with immunosuppressive drugs (e.g. MMF) which can help to prevent major organ complications. However, people with limited scleroderma are not often treated with these drugs but can still develop serious internal organ complications. This research hopes to understand if immunosuppressant drugs can help prevent worsening of the condition in people with limited scleroderma.

How will the findings benefit patients?

If the pilot study shows the benefit of treatment with MMF for people with limited scleroderma, this could support the move to a full clinical study in the future. This could provide a new treatment option for this group of people, helping to prevent their condition from worsening and therefore avoiding serious complications.