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Update: Experts Convened to Explore How Studies Tracking People with MS May Be Leveraged to Speed Solutions for Progressive Disease

March 15, 2017

UPDATE

(Originally published February 24, 2016)
SUMMARY
  • Experts were convened by the National Institute of Neurological Disorders and Stroke and the National MS Society to evaluate how MS “cohorts” – studies designed to track people with MS over time – might be leveraged to answer urgent questions about what causes progressive MS and how to stop it.
  • Presenters provided details of current and planned cohort studies around the world, and participants provided advice on key questions, opportunities and challenges related to the use of cohort studies in MS research.
  • Participants noted the critical need for better and more sensitive ways to measure MS progression to speed clinical trials, and identified opportunities for aligning with and leveraging ongoing research efforts including the Multiple Sclerosis Outcomes Assessments Consortium and the International Progressive MS Alliance.
  • UPDATE: An open-access paper has been published (Published early online in Multiple Sclerosis Journal on March 1, 2017) that provides a review of the cohort landscape, a table of major existing and planned MS cohorts, and outlines important research directions. This should help guide funders and MS researchers on the best ways to speed answers for people with progressive MS.
DETAILS
What are the underlying mechanisms that cause MS progression? Will intensely tracking people with MS over time help answer this question? These were among key questions identified by experts who were convened by the National Institute of Neurological Disorders and Stroke and the National MS Society in Washington, DC in early February to survey the landscape of current and planned MS “cohorts” – studies designed to track people with MS over time – and to recommend the best ways to answer urgent questions that will speed knowledge and treatments to stop MS progression.
 
Background:  MS cohorts have been an important research tool for many years, and have contributed much about what we know about MS today. Just one example is a recent study showing that smoking after an MS diagnosis caused faster progression. This important result was from an add-on study to the Swedish MS Registry cohort. As valuable as cohorts can be for these kinds of insights, much of what we know about the expected course of MS (called its “natural history”) came out of older cohorts that were followed before there was widespread availability of disease-modifying MS therapies.
 
There are thousands of people being followed in various types of MS cohorts around the world. Some cohort studies were built to track individuals’ medical histories and exams as a way to better inform office visits and treatment decisions in real-world settings outside of clinical trials. Others are sophisticated collections of nervous system images, DNA, blood cells, clinical outcomes, and information about a person’s symptoms and concerns, and other data over time. Several ambitious cohorts are just starting or are in planning stages. Depending on the frequency and types of data and specimens collected, maintaining MS cohorts can be very expensive and complex, and they are not necessarily open to investigation by other researchers.
 
With the urgent need for specific therapies for progressive forms of MS, better information is required about progression, including when it begins and what are the underlying causes. Modern cohort studies may be able to answer these and many other questions. But many cohorts have not been built to answer specific questions about progressive MS, and without a clear view of the cohort landscape, there are likely to be opportunities for leveraging current efforts and reducing duplication of effort.
 
Meeting Highlights: With these issues in mind, the National MS Society and National Institutes of Neurological Disorders and Stroke convened “Progressive MS: Using Prospective Longitudinal Cohorts to Understand Pathophysiology and Develop Biomarkers/Outcome Measures to Predict Progression.” The aim was to understand the MS cohort landscape and identify opportunities and challenges related to the potential for harmonizing and using cohorts to develop better tools and treatments to stop MS progression.
 
The organizing committee included volunteers Drs. Robert Fox (Mellen Center, Cleveland Clinic), Rick Rudick (Value Based Medicine, Biogen), and Alan J. Thompson (Faculty of Brain Sciences, University College London), as well as Drs. Ursula Utz (National Institutes of Neurological Disorders and Stroke/NIH), Karen Lee (MS Society of Canada), and Bruce Bebo (National MS Society). 
 
Presentations: Invited representatives from a sampling of current MS cohorts, patient registries and new or planned MS cohorts presented in-depth information about the types of data being collected from people with MS who in many cases volunteer to participate. The current cohorts discussed included:  
Presentations on new or planned cohorts included: 
  • Opt-UP and iConquerMS (Accelerated Cure Project); OPTIMISE (United Kingdom);NARCRMS (North America) ; MS Neuroimaging Initiative; Multiple Sclerosis Partners Advancing Technology and Healthcare Solutions (MS PATHS);  SUMMIT (Brigham and Women’s Hospital and University of California, San Francisco); Patient-Centered Outcomes Research Institute (PCORI, U.S.)
Dr. Fred Lublin (Icahn School of Medicine at Mount Sinai, NYC) summarized challenges to conducting clinical trials in progressive MS. He highlighted the need for both basic knowledge of what’s driving progression to identify optimal therapy targets, and better ways to identify progression and quickly detect therapeutic benefit without waiting what could be years for a person to show worsening disability. He pointed out that although many clinical trials have failed to show benefit against progression, the MS community continues to learn from these studies. Dr. Lublin is the recent recipient of a planning grant from the International Progressive MS Alliance to develop a collaborative network that will explore data from clinical trials to determine the most reliable indicators of the earliest onset of secondary-progressive MS, which would speed research and clinical trials.
 
Participants noted the critical need for better and more sensitive ways to measure MS progression. One effort focused on this need is the MS Outcome Assessments Consortium (MSOAC), a global effort aimed at developing a more sensitive tool for detecting the benefit of potential treatments that slow or reverse progressive MS. Dr. Nicholas LaRocca (National MS Society) presented an update on this 4-year, $3 million project supported by the National MS Society, which is collecting data from many MS clinical trials to identify the optimal measures for inclusion in this tool. This will then be submitted to the FDA and EMA for regulatory approval, a step toward validating its acceptance for use as a primary outcome measure in clinical trials. Dr. LaRocca noted that this project, which involves a partnership of academic investigators and pharmaceutical companies that have shared data, can serve as a model not only for collaboration but also for standardizing data that allows comparison from separate cohorts.  
 
Group Work: A panel discussion focused on the place for and value of medical practice-based cohorts and patient registries compared to rigorous research cohorts designed to answer key questions. Participants also commented on the need for smaller, very well-characterized cohorts as well as larger, less detailed cohorts, with both types offering advantages for answering different questions.
 
In breakout groups, meeting participants were charged with developing priority questions that need to be answered to achieve treatment breakthroughs for people with progressive forms of MS, as well ideas about opportunities presented by cohorts and current impediments to optimizing their value. Meeting participants noted the importance of aligning with and leveraging other ongoing efforts, including those of MSOAC, the International Progressive MS Alliance, and the data sharing initiative of the Alliance’s Industry Forum. 
 
Next Steps: UPDATE: An open-access paper has been published (Published early online in Multiple Sclerosis Journal on March 1, 2017) that provides a review of the cohort landscape, a table of major existing and planned MS cohorts, and outlines important research directions. This should help guide funders and MS researchers on the best ways to speed answers for people with progressive MS.
 
Read more about research to find solutions for progressive MS

About Multiple Sclerosis

Multiple sclerosis is an unpredictable, often disabling disease of the central nervous system that disrupts the flow of information within the brain, and between the brain and body. Symptoms range from numbness and tingling to blindness and paralysis. The progress, severity and specific symptoms of MS in any one person cannot yet be predicted, but advances in research and treatment are leading to better understanding and moving us closer to a world free of MS. Most people with MS are diagnosed between the ages of 20 and 50, with at least two to three times more women than men being diagnosed with the disease. MS affects more than 2.3 million people worldwide.

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