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.
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.
Below are some of the projects we have awarded funding to in 2023.
Optimism – Rapamycin for Inclusion Body Myositis
Dr Stefen Brady (John Radcliffe Hospital – Oxford)
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.
Disease pathways for IBM stratification and therapy
Professor Pedro Machado (University College London – UCL)
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.
Improving clinician utilisation and data quality on the MYOACT patient register
Dr Saadia Ali (King’s College Hospital – KCH)
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.
MyGRATE – Supporting the migration of JDCBS to a Trusted Research Environment with dataset Quality Control
Dr Sokratis Varakliotis (Great Ormond Street Hospital – GOSH)
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.
Myositis UK has also been part of a number of other research projects, studies and data collaborations which have resulted in publications.
You can view a list of the latest published work below in which we have had some involvement (by funding salaries, research consumables, disseminating information etc).