UNDERSTAND, PREVENT AND REVERSE PROGRESSION
Accelerating new, life-changing treatments for people with progressive MS requires new insights and knowledge into the causes of progression. By determining what triggers MS progression, researchers will be better prepared to focus their efforts on developing breakthroughs to slow, prevent, and ultimately, reverse disability.
To meet the challenge of not only understanding progression but identifying treatments to prevent and reverse progression, the Alliance has established a series of global research initiatives that work together to achieve necessary results.
- Drug Discovery International Collaborative Research Networks – two networks are unlocking the mystery of progression to identify breakthrough treatments. These are first-of-their-kind large-scale networks bringing together leading scientists from MS research institutions from multiple countries in order to drive innovation and expedite results. These networks represent an investment of over €8 million in research funding.
- One network is developing a drug discovery platform to identify compounds that can protect and repair the brain.
- Another network is focused on preventing brain damage in MS through new understanding of how the innate immune process affects progression and identifying drug candidates.
- Research Challenge awards – are short-term, €75,000 research grants that focus on identifying new understandings into the cause of progression and help identify new paths for treatment development. Innovation and multi-national collaboration are key elements of these grants.
- 22 awards totaling €1.6 million in funding in 2014 led to the development of new frontiers in progressive MS research, findings helped lead to the development of two of the Collaborative Research Networks, other international research programs and the publication of 16 scientific journal publications.
- In 2021, up to 20 new awards will be granted through €1.5 million in funding. These projects will target mechanisms underlying progressive MS. Emphasis will be placed on high-risk proposals that have the potential to result in a paradigm shift in progressive MS and lead to new focus areas for treatment development.
SPEED UP AND IMPROVE CLINICAL TRIALS
Clinical trials for a new progressive MS therapy can take up to five years and require millions of Euros. But people living with progressive MS simply can’t wait that long to see if a new treatment is viable. That is why it’s critically important to be able to test multiple approaches in a shorter time span with a lower level of investment required.
Additionally, there are challenges that must be addressed to improve clinical trials. There is a vital need for biomarkers in progressive MS that can measure progression and indicate if a potential treatment is effective. Clinical trials also need to be designed that can provide deeper insights into the biology of progression which will lead to targeting areas where a drug intervention could be successful in preventing disease progression or stimulating nervous system repair. Finally, clinical trials need to include a broader spectrum of people with progressive MS and people affected by MS need to be involved in the design of trials.
The Alliance is working in multiple ways to speed up and improve clinical trials:
- MRI Imaging Biomarker International Collaborative Research Network – Artificial Intelligence can lead to new tools that can predict, via MRI’s, the changes in the brain as MS progresses. Such tools could be used to quickly evaluate whether a potential treatment is effective and ultimately be used in a clinical setting for customizing an individual’s treatment. The Alliance has invested €4 million in a research network that is using machine learning to create tools that would be a major advancement in progressive MS clinical trials.
- Serum Neurofilament light chain (sNfL) – a fluid biomarker – If sNfL is further confirmed as a biomarker for progressive MS, a blood test could help predict the future course of the disease and indicate whether a treatment is working. The Alliance has formed an expert panel that has published a set of recommendations and is working with the FDA, EMA and the MS scientific community to further this effort.
- Expert panel recommendations – the Alliance has convened a panel of experts to give recommendations on ways to improve progressive MS clinical trials. This panel has produced a paper that provides a number of steps for the design of trials in progressive MS that will not only effectively test new treatments but also lead to new understandings of the biology of progression. The panel also describes the importance of including different cohorts of people with progressive MS in trials and that people affected by MS should be involved in trial design. Next steps in this work include Alliance funding initiatives that would support the testing of new clinical trial approaches.
IMPROVE WELL-BEING THROUGH PROVEN TREATMENTS
Ensuring people with progressive MS can live fulfilling lives requires effectively addressing the multitude of symptoms that disrupt daily life. The Alliance is working to establish a global research strategy that can lead to proven methods for rehabilitation and symptom management to improve the lives of people living with progressive MS.
- A 2018 International Congress integrated multiple fields and the most promising areas of research, the outcome set a course forward including the development of a paper to provide global recommendations on research priorities in well-being, including fatigue, mobility, cognition and pain.