Current research
Find out more about the current research projects receiving funding from Myositis UK.
About our research
Myositis UK funds vital research into all the different types of myositis. We also participate in medical meetings and seminars.
Although a small charity, our generous members and supporters have helped us raise over £700,000 for research both in the UK and overseas. Together we are dedicated to funding research to improve the lives of those affected by myositis.
The types of the condition we focus on include dermatomyositis (DM), juvenile dermatomyositis (JDM), polymyositis (PM), inclusion body myositis (IBM) and myositis antisynthetase syndrome.
We offer a range of funding opportunities, including seedcorn grants, funding for research consumables, top-up funding, PhD studentships and overheads. All the projects we put funding towards seek to improve the diagnosis, treatment, understanding and prognosis of myositis.
The funding process
Funding opportunities are now advertised on our website and researchers working in the field of myositis are invited to apply. All applications are sent for independent review before a consensus decision is reached by our ‘Scientific Advisory Committee’ and ‘Medical Advisory Board’. Once the outcome has been agreed by our ‘Board of Trustees’, the successful candidates are notified, and the award is made. The aim of this process is to ensure that funding is awarded to high quality research projects that deliver patient benefits, in an open, fair, and transparent manner.
The funding process was introduced in March 2023, since when we have received 17 funding applications and awarded 11 grants. Of those awarded, three were for large grants (£100,000 plus for both) and seven were for small grants (£10,000 – £25,000 for each) with around £485,000 awarded in total. Below is a summary of the research funded under this new process.
Below is a summary of the research funded under this process.
Project 1
Optimism – Rapamycin for Inclusion Body Myositis
Dr Stefen Brady (John Radcliffe Hospital – Oxford)
Awarded: May 2023, £111,247.75
Inclusion body myositis (IBM) causes progressive disability and a seven-fold increased risk of dying early. Unlike other forms of myositis, no treatment has been shown to affect the progression of IBM.
In 2017, a small pilot study of an anti-transplant rejection medication, Rapamycin, was the first to show benefit. We have been invited to join Optimism, an international collaborative study whose goal is to ascertain the benefit of Rapamycin.
We expect Rapamycin treatment to produce similar benefits as previously observed in the pilot study including slowing disease progression and weakness.
Rapamycin is cheap, widely used in the UK, and licensed for treatment of autoimmune disease. In the event of a positive outcome, it can easily be made available to people living with IBM, improving their quality of life through slowing or stabilising the progression of their disease.
Project 2
IBM – Path: Identifying disease pathways for inclusion body myositis (IBM) patient stratification and therapy.
Professor Pedro Machado (University College London – UCL)
Awarded: May 2023, £129,933
Inclusion Body Myositis (IBM) is a disease that causes progressive muscle wasting and disability. There is currently no known treatment, and the cause of IBM is unknown. It is believed that genetic and environmental factors may trigger inflammatory and degenerative pathways in the body, particularly as someone ages.
We are part of the IBM genetics collaboration and have completed a genetic study that found new genetic associations. Moving forward, we plan to expand our study by collecting genetic data from more IBM patients and comparing it with data from people with polymyositis or dermatomyositis.
We will investigate specific genetic regions in IBM using advanced techniques such as exome and genome sequencing to identify exact genetic variations associated with the disease. We will investigate altered and/or reduced protein production by sequencing IBM muscle samples.
Pathological studies and tissue culture investigation of muscle tissue and patient cells will be used to gain insight into disease mechanisms and potential drug targets.
Project 3
Improving clinician utilisation and data quality on the MYOACT patient register
Dr Saadia Ali (King’s College Hospital – KCH)
Award: £9,998.37, May 2023
The idiopathic inflammatory myopathies (IIMs) are rare group of autoimmune conditions affecting 10,000 patients in the UK. There are two advanced treatments available for this condition in the UK, Rituximab and Abatacept. However, large clinical trials have failed to confirm a benefit from these drugs in the IIMs.
Further analysis of these studies suggested that some types of IIMs may benefit from these drugs. This represents a significant unmet clinical need and there is an urgent demand for further data to confirm these findings and elucidate which patients benefit from these drugs.
Real-life patient registries can be complementary to clinical trial data as they pool together patient information from across the country, increasing the amount of information available to analyse, making it an asset in rare diseases like IIMs. The MYOACT patient audit was set up with the aim of monitoring the safety of these drugs in the myositis population and confirming which patients are likely to benefit. As more drugs become available the registry will help us better select right drug for the right patient.
However, despite the audit being a requirement for NHS funding for these drugs, the audit has not been widely used by clinicians. A survey and focus group of myositis experts established that many clinicians found the MYOACT website too complicated and there was too much data to be completed in our busy clinical environments as the main reasons for underuse.
The aim of my research is to re-engage clinicians with the MYOACT audit by making it more user friendly and designing a smart phone application to input data quickly into the register ultimately improving data quantity and quality. This would make it easier to monitor the safety and clinical use of these important treatments in myositis ensuring their appropriate use in patients.
Project 4
MyGRATE – Supporting the migration of JDCBS to a Trusted Research Environment with dataset Quality Control
Dr Sokratis Varakliotis (Great Ormond Street Hospital – GOSH)
Awarded: May 2023, £9,680
The Juvenile Dermatomyositis Cohort Biomarker Study and Repository (JDCBS) is the largest cohort study with linked patient biological samples for JDM and related inflammatory myositis conditions that begin in childhood. Since 2000, 16 UK centres have been contributing data and samples, supporting multiple national and international studies on genes, immune cells, and muscle in childhood myositis. The study has recently undergone two vital changes:
- migrated to using the internationally agreed set of data collected, called the JDM Consensus Dataset, designed for clinical use, to improve collaborative research and allow easier sharing of data between different researchers, and
- migrated its data collection away from a standalone system to a secure research environment, called the GOSH Digital Research Environment or DRE, which will be maintained long into the future.
This proposal focuses on the crucial process of data migration from the old standalone database to the DRE, while providing continuous operation of the study, to make sure this happens smoothly. The aim is to apply quality control, eg, data cleaning, checking and recovery methods, using specialised software techniques.
This will ensure more accurate migration and will maintain 20 years of high-quality data continuity between the historic and the modernised version of the dataset, to the benefit of patients through all the current and future research projects which use this unique dataset.
Project 5
Biomarker identification for treatment response to the oral JAK 1/2 inhibitor, baricitinib, in adult idiopathic inflammatory myopathy
Dr Janine Lamb (University of Manchester).
Awarded: December 2023, £20,000
Inflammation in the muscles and other symptoms has a significant impact on quality of life of people living with myositis. While immune suppression drugs can help, there are often side-effects and not all individuals respond to these drugs. The applicants are currently completing a clinical trial of a new drug, baricitinib, in adult-onset myositis, a drug which blocks signalling of inflammatory chemicals.
In the proposed study, we aim to build on the clinical data and biological samples collected during this clinical trial to identify molecular predictors and markers of response to treatment with baricitinib.
We have collected biological samples (including blood, serum, DNA) from all patients in the trial at baseline (pre-treatment), and at 3 timepoints throughout the trial. Using these samples, we will apply a range of complementary experimental approaches to look at the levels of inflammatory markers and myositis autoantibodies (immune proteins), the relative frequency of different types of immune cells in the blood, and genetic differences between patients. We will compare the samples collected pre-treatment to post-treatment and at the different timepoints throughout the trial to look at changes over time and in response to treatment. We will correlate these molecular measurements to levels of inflammatory muscle enzymes, and clinical measures of response to baricitinib treatment and changes in disease activity.
We anticipate this research may identify molecular predictors and markers of response to treatment with baricitinib and disease activity in individuals with adult-onset myositis. This knowledge may help to develop more individually targeted treatment approaches and to improve future disease management in myositis. Characterisation of these molecular markers will also give insight into the biological mechanisms and causes of myositis.
Project 6
A mitochondrial gene signature to stratify juvenile dermatomyositis patients for targeted treatments.
Lead Researcher Dr Meredyth Wilkinson (University College London).
Awarded: June 2024, £19,973.70
Juvenile dermatomyositis (JDM) is a rare autoimmune condition in which the body’s defence system (designed to stop infections) attacks itself leading to muscle weakness and skin rashes. Some patients get damage to lungs, gut and even brain. Treatments used in JDM suppress the body’s defence system, but do not work for all patients and can cause unwanted side effects. If we understood exactly which part of the body’s defence system was going wrong it would help us target this with more effective medication. Our recent publication showed that genes (the human code) that code for mitochondria (‘powerhouse’ energy producers of the cell) were less active in JDM patients (even those already on strong treatment) than in healthy children of the same age. We believe that these discoveries allow us to develop tests to measure mitochondrial problems and identify better treatments for patients. Importantly, fatigue is one of the most common symptoms described by JDM patients and needs to be addressed. We believe that mitochondrial problems may relate to the levels of fatigue in children with myositis.
In two groups of JDM patients measuring expression of thousands of genes in blood cells, we have identified a set of 37 genes that represent the mitochondrial problems seen in immune cells. Using a technology called nCounter made by NanoString (measure expression of a fixed set of genes), the mitochondrial gene signature (MGS) will be measured as a set to measure mitochondrial problems.
Aim 1: Investigate if the MGS can characterise groups of patients, and predict disease type, severity or outcome.
Aim 2: Establish if the MGS measured in blood relates to levels of fatigue experienced by JDM patients.
Project 7
MIRAGE: Myositis Inflammation Revealed through Advanced Magnetic Resonance ImaGing Evaluation A Prospective Study Amount requested.
Professor Pedro Machado (University College London)
Awarded: June 2024, £19,908.65
Our study focuses on understanding and better assessing a group of autoimmune muscle disorders called idiopathic inflammatory myopathies (IIMs), which can affect various body parts beyond muscles. To evaluate these conditions, we can use advanced imaging techniques, particularly magnetic resonance imaging (MRI), which helps us visualise muscle inflammation and damage. We aim to develop a new MRI method called T2-water quantifiable MRI (Q-MRI), which can accurately measure muscle inflammation in adults with IIMs. We hypothesise that this method will provide more precise and objective assessments of muscle inflammation compared to current MRI techniques. Our study involves two visits over a period of 3-6 months for 16 adult patients with IIMs. During these visits, we will conduct various assessments, including MRI scans of the pelvis and thighs, evaluations of disease activity and damage, tests of physical function and mobility, and assessments of fatigue and organ involvement. We will also evaluate how patients respond to treatment using established criteria. By the end of our study, we hope to validate the new MRI method as a reliable tool for assessing muscle inflammation in IIMs. This could lead to better monitoring of disease activity, improved treatment decisions, and ultimately, better outcomes for patients with these challenging conditions. This study has the potential to advance our understanding and management of IIMs, ultimately improving the lives of individuals affected by these conditions.
Project 8
The UKMYONET study: Increasing accuracy of autoantibody testing in myositis using new approaches and exploring genotype-autoantibody associations
Professor Hector Chinoy (University of Manchester).
Awarded: December 2024, £97,724
This will help answer questions that matter to patients such as ‘will what happen to me?’ and ‘why me?’
Autoantibodies are a blood test used by doctors to diagnose myositis and to predict problems like lung disease or cancer. Testing happens routinely in the NHS, but hospital tests don’t work well and often give the wrong result. Different autoantibodies are linked to different genetic markers. This in part explains why different people develop different types of myositis. Until now myositis genetic studies have focussed on White people.
UKMyoNet is a large study of nearly 2000 UK myositis patients. Samples have been collected and stored. About 600 samples do not have reliable autoantibody and genetic data. We will accurately test these samples for autoantibodies. We will compare our findings to data from hospital testing and show where improvements are needed. We will also test the genetics of these samples to confirm links between autoantibodies and genetics and whether the same links are seen in non-White people. Importantly, we will show the value of a new version of what is currently the best autoantibody test. Going forwards, using the new test will increase access to accurate autoantibody testing and lead to new research prospects.
Project 9
Sleep disturbances and fatigue in children with Juvenile Dermatomyositis (JDM); do they relate to disease activity and pathology?
Dr Charalampia Papadopoulou (University College London).
Awarded: December 2024, £20,000
Desirable long-term outcomes in chronic childhood diseases include normal growth and development, participation in normal social, educational and sporting activities and a good quality of life. Sleep disturbance is often seen in rheumatological diseases and may contribute to the fatigue seen in many of these conditions, including juvenile dermatomyositis (JDM). In JDM the frequency of sleep disorders and fatigue, as well as the contributing factors to their occurrence remain poorly understood. Sleep alterations in JDM have been associated with steroid treatment and disease markers in the blood.
The aim of this study is to assess how common sleep disturbances and fatigue are in children with JDM at the start of disease (at diagnosis) and over time, using developed patient-reported outcome measures (PROMIS) which are efficient, precise, and easy to use in routine clinical practice. We will then test how these relate to clinical measures of the disease and disease markers in the blood. We will measure interferon (an inflammatory protein) and mitochondria (powerhouse of the cell) markers in the blood to identify what is going wrong in the body and how these contribute to fatigue, sleep problems and also cause JDM disease specific symptoms.
Project 10
Developing a dietary intervention to address muscle loss in inclusion body myositis (IBM).
Dr Paul Morgan (Manchester Metropolitan University)
Awarded: December 2024, £20,000
Sporadic inclusion-body myositis (IBM) is a common type of muscle disease in people over 50 years of age. Sufferers experience muscle weakness, particularly in the arms and legs, which gets worse over time and results in disability. Most people lose their ability to walk independently within seven years. IBM does not respond well to medication, and the costs of this for our healthcare system are large. While physical therapy is recommended, little is known about how diet, particularly how much protein these people eat, affects the progression of IBM. Protein intake is important for maintaining muscle mass and strength, but there are no specific guidelines for people with IBM. In healthy individuals, eating protein helps build muscle, but in people with low muscle, this process doesn’t work as well. It is unclear if this happens in people with IBM, and finding out information about this will help us to understand whether providing more protein to IBM sufferers will help them. If so, we can also provide dietary advice for them about how they might achieve this increase in protein intake. We will study the dietary habits, nutritional status, and measure chemical processes that occur in the muscles of IBM patients, which has not happened before. We will also assess how well people with IBM function, how much fat and muscle they have and investigate how their muscles respond to protein intake, compared with healthy people of a similar age. We will do this by: 1. assessing dietary intake and physical activity habits, challenges to good nutrition, energy use, muscle and strength, swallowing difficulties and markers of quality of life in people with IBM. 2. studying how IBM patients’ muscles respond to protein consumption. 3. identify dietary factors that influence muscle health, strength, and quality of life in people with IBM.
Project 11
Innovative strategies for topical and intradermal delivery of citric acid for regression of myositis-related calcinosis
Dr Mohamed Elsawy (University of Manchester)
Awarded June 2025, £24, 931
Calcinosis (the formation of calcium deposits under the skin), is one of the most unpleasant symptoms associated with juvenile dermatomyositis (as well as in adults with systemic sclerosis). Calcinosis causes severe skin ulceration and frequent infections, and is painful, disabling and disfiguring. In this collaborative project, we are developing innovative healthcare technologies to tackle calcinosis. The goal is a new localised treatment approach involving the use of citric acid, a non-toxic agent that helps to dissolve calcium deposits in the skin, which will be easier and more comfortable for patients to use, improving quality of life and reducing costs for calcinosis treatment. The technology is based on the development of both topical water-based gel formulations and skin ‘microneedle patches’. These patches have very tiny needles containing the citric acid gel treatment, for painless injection into the top layers of the skin. Applying the gel treatment to the skin can reach calcinosis lesions at the surface of the skin and acts to soften ulcers but is not always effective. These new microneedle patches will enable the treatment to get deeper into the skin, and last longer, helping to treat lesions that are further beneath the skin surface. The microneedle patch will help to make the treatment more effective and reduce the number of times a patient has to apply the treatment. It will also avoid the pain of injected treatments. Patients will be able to apply the patches themselves, or be helped to do so by a carer making treatment easier and cheaper. The short-term goal of this project is development and pre-clinical testing of a skin patch prototype for the treatment of calcinosis, paving the way towards clinical testing in patients.
Next funding call
The next funding call deadline is Friday 26 September 2025 (5pm) where two small grants of up to £25,000 are available to UK researchers working within the field of myositis.
