Written by Simon Stones, patient insight partner
On Monday 9 April 2018, I attended the Arthritis Research UK cure strategic workshop, held in London. The purpose of this workshop was to bring together a wide range of experts (including people with musculoskeletal (MSK) conditions, basic and clinical researchers, healthcare professionals and other research funders) to identify and discuss current unmet patient and research needs. The ultimate goal was to define the scope of the next ambitious strategic challenge funding call, which researchers will be able to apply to in order to attempt to secure funding for their research proposals. This specific call seeks to find a cure and more effective treatments for MSK conditions through research from the bench to the bedside. This call builds on the November 2016 challenge funding call, called 'Stacking the odds towards a cure'.
Stacking the odds towards a cure
In their 2020 strategy, Arthritis Research UK ambitiously outlined their strategic focus to prevent the onset of MSK conditions, while striving to develop a cure and transforming the lives of those with MSK conditions. In the last decade, Arthritis Research UK has funded over £250 million into research across the spectrum of MSK conditions, which has enabled boundaries to be pushed beyond what was previously thought unimaginable. Since the turn of the century, we've seen the rise of biologics and biosimilars, which have revolutionised the way that MSK conditions are treated. We've seen an investment in personalised medicine, recognising the uniqueness of our bodies, and the concept that our genes influence the way in which we respond to different treatments.
In November 2016, Arthritis Research UK launched the 'Stacking the odds towards a cure' funding call, in which the charity invited research applications that would accelerate the pathway to discovery (laboratory-based research) and early translation (first-in human research), with the goal of developing a cure and more effective treatments for people with MSK conditions in the future. But what does cure mean to you?
What cure means to different people
Cure is a bold statement. In the context of long-term conditions like MSK conditions, cure means ‘A time without recurrence of disease so that the risk of recurrence is small.’ This is usually the definition that springs to mind for most people when we think of cure - curing cancer, curing dementia, curing MSK conditions. In reality, cure isn't that straightforward - and indeed, the pros of a 'cure' need to be compared to the cons of a 'cure'.
While some research won't directly lead to a cure or new treatment in the short-term, it could do in the longer-term - enabling the community to move closer to the goal of fully understanding the disease processes underlying all MSK conditions, as well as the pathways and potential targets for future treatment. The journey to a cure may well be different for each disease area, but nevertheless, an important journey.
During the workshop, we had many discussions about what 'cure' could mean to patients, and often, patients expressed differing perspectives to healthcare professionals. Healthcare professionals and researchers are naturally focused on achieving disease remission and, ultimately, eliminating the disease from affecting people's quality of life. However, for patients, the journey to a cure could be worse than the disease itself, particularly if treatments have a negative effect on people's physical and psychosocial wellbeing. Therefore, a cure for a patient could mean minimal disease activity, no treatment side effects and a good quality of life.
Cure part II
Therefore, to fulfil the aims of this research challenge funding call, some research may be of higher risk, but may also have longer-term outcomes with respect to transforming the lives of people with MSK conditions. This type of research often requires new, collaborative teams of researchers, healthcare professionals, patients and industry, which can only be a positive move. Examples of innovative and bold moves were highlighted in presentations from Professor Iain McInnes, Professor of Experimental Medicine at the University of Glasgow and President-elect of the European League Against Rheumatism (EULAR), and Professor Ali Mobasheri, Professor of Musculoskeletal Physiology at the University of Surrey and a Board Member of the Osteoarthritis Research Society International (OARSI). Both Iain and Ali led discussions based upon national and international policy documents and priority setting exercises, including EULAR's research roadmap, RheumaMap (PDF 982 KB) and Arthritis Research UK's State of Musculoskeletal Health report. In the report, some disturbing figures were described, such as 78% of people with MSK conditions experience pain most days, and 53% of people feel as though they are a nuisance to their family.
We also heard from Dr Natalie Carter, Head of Research Liaison and Evaluation at Arthritis Research UK, who initiated a discussion led by patient insight partners regarding what cure looks like to patients. Subsequently, Jane Taylor, Colin Wilkinson and Lynn Laidlaw spoke about their experience of a recent Arthritis Research UK patient insight day, and subsequent dialogues, which related to the concepts of cure meaning different things for people with arthritis, in terms of experiencing a more favourable quality of life, compared to eradication of disease for eternity.
After lunch, the group were separated into four breakout sessions aligned with Arthritis Research UK's newly established research advisory groups (RAGs), which evolved from their existing clinical studies groups (CSGs). These were:
- Inflammatory arthritis;
- Autoimmune rheumatic disease;
- Musculoskeletal disorders; and
- Paediatric rheumatology.
During these sessions, groups discussed what unmet needs and previously identified research priorities reflect the ambition of providing a cure, as well as what gaps are believed to be missing, and where are the possibilities of discovery, pre-clinical and clinical research in the UK. Within our paediatric rheumatology breakout session, we had some deep and meaningful conversations about the purpose of 'cure' for children, young people and their families. We initiated our discussions based upon the co-created paediatric rheumatology CSG research strategy from 2015, which included a series of research questions across different paediatric rheumatology areas, including patient- and parent- designed research questions. One of the most important priorities for us is about improving the tolerability and delivery of methotrexate. Many of us have experiences of methotrexate, and understand how unpleasant the treatment experience can be for children, young people and families – sometimes even life-changing. I emphasised here that for these individuals, cure could well be a better experience of methotrexate without the side effects, which in turn, could help to reduce disease activity, since the medicine is generally effective at treating certain paediatric-onset MSK conditions, such as juvenile idiopathic arthritis. It is only by having these conversations that we can begin to unpack the complexity around what cure means to different people, but mostly importantly, what needs to be addressed to improve the quality of life of people with MSK conditions. While cure in the broadest sense seems a destination far on the horizon for the MSK community, we must never lose hope of a brighter future for everyone, and the many avenues that could take.