The CLUSTER consortium has been awarded £5 million from the UK’s Medical Research Council (MRC) with partnership funding from Arthritis Research UK.
One in 1,000 under 16-year-olds in the UK are affected by childhood arthritis, and researchers aim to improve the lives of children living with these life-changing and complex conditions.
Starting in July 2018, the CLUSTER childhood arthritis study will team scientists including IoO's Director Professor Andrew Dick, Professor Lucy Wedderburn of UCL Great Ormond Street Institute of Child Health (ICH), and scientists other UK institutions*, and help clinicians to target specific treatments for patients.
The initiative is one of four ‘stratified medicine’ projects being funded by the MRC, which is investing £15 million in studies that will establish ground-breaking approaches in treating prostate cancer, kidney disease, alcoholic hepatitis and childhood arthritis.
Stratified medicine, also referred to as personalised medicine or precision medicine, is an emerging approach for disease diagnosis and treatment that considers patients’ genes, environment and lifestyle to create tailored therapies instead of a one-size-fits-all approach.
Childhood arthritis can cause long-term disability and poor quality of life – sometimes well into adulthood. If it isn’t diagnosed and treated early, patients may require hip and knee replacements, are significantly shorter than their peers, and some end up in wheelchairs. For those patients who also have uveitis, a condition where the inside of the eyes become inflamed, there is also a significant risk of vision loss and blindness.
Currently, young people diagnosed with arthritis in the UK are given a single drug therapy, but it only works in about 50% of cases. The remaining half must try other treatments, one after the other, to find a therapy which works for them. Along the way, they may experience painful side effects, time out of school and even a worsening of their symptoms.
Ultimately, the researchers in the trial hope to identify a simple biomarker test that will:
- Pave the way to new treatment options, identifying potential new therapies and eventually the trialling of new drugs which work better with fewer side effect
- Help doctors define the right medication for the right duration for each individual patient on the first try. Identifying the right treatment ensures symptoms are managed and that the condition doesn’t develop into lifelong disability
- Identify which children are at risk of uveitis, a serious eye-inflammation which affects 15% those with childhood arthritis. Currently, patients visit eye specialists every three to six months to screen for this secondary condition
- Project long-term outcomes for these children – scientists will track patient health over decades, so they can better predict what may be ahead for these children
Lead researcher, Professor Wedderburn said: “Using a stratified medicine approach could be a gamechanger in childhood arthritis and its associated uveitis. A biomarker test could lead to methods for accurately predicting the right treatment for the right duration, halting the worsening of symptoms and leading to shorter time to remission. Nothing like that has been done before in this area of research.”
Professor Sir John Savill, Chief Executive Officer at the MRC, said: “Stratified medicine is reshaping the medical landscape by taking a patient-first approach that looks at how – and why – different groups of patients respond differently to therapies. Instead of looking for a ‘one-size-fits-all’ solution, stratified medicine is increasing what we know about diseases and how they affect individuals, and applying these findings to current tools in diagnostics and treatment to improve health outcomes. I am particularly pleased to see the potential benefits of this research approach extended to children with a life-changing condition.”
Notes
*The CLUSTER study includes collaboration with the UCL Institute of Ophthalmology, and scientists from Manchester, Liverpool, Cambridge, Bristol and London. CLUSTER is receiving additional co-funding from GOSH Children’s Charity and Olivia’s Vision.
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The CLUSTER team: Back row (L-R): Professor Kimme Hyrich, Ms Eilean MacDonald, Professor Michael Barnes, Dr Chris Wallace, Prof Andrew Dick, Professor A.V Ramanan. Front row: Professor Wendy Thomson, Ms Nicki Davies, Professor Lucy Wedderburn, Dr Nophar Geifman, Professor Michael Beresford.