Understanding the role of white blood cells in juvenile dermatomyositis

Disease - Muscle weakness, myositis

Lead applicant - Professor Lucy Wedderburn

Organisation - University College London

Type of grant - PhD Scholarship

Status of grant - Active

Amount of the original award - £156,209.56

Start date - 25 September 2017

Reference - 21552

Public Summary

What are the aims of this research?

Problems with certain white blood cells, a type of immune cell, are known to be associated with causing juvenile dermatomyositis, but why these problems occur and how they cause disease to develop is still not known. The aim of this research is to understand how certain white blood cells cause juvenile dermatomyositis.

Why is this research important?

Juvenile dermatomyositis is a rare childhood-onset disease that causes severe muscle and skin problems. There is currently no cure for this condition and available treatments have multiple side effects and are not effective for many patients. Therefore, a greater understanding of what causes the condition is important to identify potential new treatments.

This will be the first study of its kind to extensively study whether a type of white blood cell, called T-cells, are different in patients with juvenile dermatomyositis compared to healthy individuals. Early data from this research group has found that the specific proteins inside T-cells, that can control their normal function, may be abnormal in patients with this condition. The researchers will study how these specific proteins go wrong in disease and aim to investigate, for the first time, whether targeting these proteins with drugs can normalise T-cell function in patients with juvenile dermatomyositis.

How will the finding benefit patients?

A further understanding of what causes juvenile dermatomyositis will help to identify new treatments. Previously, drugs that target the specific proteins in T-cells being investigated in this study have already been used in the clinic to treat other conditions. Therefore, if this study reveals that there are problems with these proteins in the T-cells of patients, there may be an opportunity to repurpose drugs that are already in use, as new treatment strategies for patients with juvenile dermatomyositis.