What are the challenges in multiple sclerosis research that the National Multiple Sclerosis Society can help address?
Significant progress has been made in the development of immune-modulating therapies to treat the relapsing forms of multiple sclerosis (MS). But these treatments do not stop the disease, do not reverse injury to the nervous system, and are not effective for people with progressive forms of MS. This disease is complex. We do not know its cause, nor do we understand its precise pathogenesis. There are insufficient disease models for preclinical testing, and we have no biomarkers that can be used for early detection, to predict disease course, or to finely track nervous system injury and health.
Given these complexities, our approach is to identify and fill gaps in our understanding, to push innovation and collaboration, to support both academic and commercial research, to support shared resources such as DNA and tissue banks, and to convene and empower the research community toward breakthroughs for people with MS.
Here are two examples. Through the MS Outcome Assessments Consortium, the Society is gathering data from industry and academic clinical trials to develop clinical outcome measure that is qualified by the U.S. Food and Drug Administration and European Medicines Agency to speed trials of new treatments, especially for progressive forms of the disease. On another front, research in nervous system repair and protection has been gaining momentum since the Society’s 2005 global initiative funded four collaborative teams who engaged more than 100 investigators, produced over 180 research papers, identified new therapeutic targets, and launched clinical trials to test neuroprotective strategies. There are myelin repair strategies being tested in people with MS right now, thanks in large part to our putting that stake in the ground in 2005.
While we’ve had many successes, there is still much to do.
How does the Society balance focusing on its three established priority areas with seizing promising new opportunities?
The Society implements a comprehensive approach to stopping MS, restoring function that’s been lost, and in ending MS forever, which allows us to tap expertise around the world in the pursuit of any opportunity that will speed our goal of creating a world free of MS. We establish research priorities based on input from scientific advisors who identify gap areas and opportunities to drive progress. We also engage the MS community to understand what topics matter to them and to ensure we are listening and finding solutions so that people can live their best lives today.
When we establish research priorities, we don’t predetermine how much of our $52 million annual research budget we will commit to those priority topics. It is less about saying we’re going to spend this much money in those areas, and more about saying that we are going to convene experts around these topics, collaborate with other partners around these priorities, promote awareness of priority areas, and make strategic investments. That leaves us with a measure of fluidity to explore and invest in the best and most impactful research that may arise in other breakthrough fields.
Science is moving at an incredible pace. This necessitates a degree of openness and an awareness that the game is changing and we don’t want to be locked in to a particular approach.
How does the subsidiary Fast Forward help drive commercial progress?
Getting better treatments to people with MS requires creative leadership, tenacity, and investment at every stage of the research process. Often the biggest hurdle in moving potential treatments forward is securing the necessary early investments for commercial development, when the potential return of those investments is largely unknown. The Society helps break down these barriers through mechanisms such as Fast Forward, which closes the gap between promising discoveries and the commercial development necessary to get new treatments to people with MS as fast as possible.
Through Fast Forward, we have been able to “de-risk” promising new approaches, generally by supporting preclinical research needed to generate data that enable candidate agents to be submitted for clinical evaluation under an investigational new drug application. Our careful and thorough review process assesses not only scientific merit, but also the business and intellectual property surrounding the approach, and we employ rigorous business practices including milestone-driven project management. For these reasons, funding from Fast Forward adds to the credibility of the company and the project, which in turn leverages investment from other sources. If the project continues to show promise, often a company can attract capital needed to progress through clinical trials. An example is Canbex, which has been successful in developing an anti-spasticity therapy and now has follow-on funding to conduct a phase 2 trial to completion.
We’ve funded 34 projects through Fast Forward to date -- 21 through regular philanthropic funds, and 13 through a collaborative partnership with EMD Serono. Early successes from our seed investments have enabled $111 million in follow-on financing and a potential $855 million in licensing or acquisition for four companies in our portfolio.
What do you consider the National MS Society’s greatest accomplishments?
When the National MS Society was founded in 1946, relatively little was known about the disease. It was often misdiagnosed (some doctors mistakenly believed MS was contagious), and there was no private or government agency charged with conducting research into multiple sclerosis. Through its grassroots efforts, the Society is a global leader in funding MS research and offering a comprehensive suite of programs and services for people with MS. By funding innovative research without geographic restrictions, and supporting the training of over 900 postdoctoral fellows and junior faculty, the Society has been instrumental in forging the global MS research community. We have been a catalyst for most major advancements in MS, laying the groundwork for 12 approved disease-modifying therapies, speeding and improving diagnosis, discovering the benefits of exercise and rehabilitation, and driving research in nerve and myelin repair.
The Society also leveraged interest and investment in MS – in the United States by advocating for a National Institutes of Health institute focusing on neurological diseases (the National Institute of Neurological Disorders and Stroke), and later globally by organizing the Multiple Sclerosis International Federation. More recently, the Society played a key founding and leadership role in the international Progressive MS Alliance, a global alliance focusing on developing solutions for the 1 million people living with progressive forms of MS.
Another significant accomplishment is our focus on ensuring that each person with MS can live his or her best life with more connections to information, resources, and others with shared experiences. While our work proceeds on multi-fronts and through multi-channels, the Society also makes sure that the voices of people affected by MS are heard and help drive change wherever it is needed. For example, more than 75,000 activists across the country have united to secure an additional $25 million in government research funding over the last six years.
What are your top research goals over the next year, and what will it take to reach those goals?
Key priorities for this year include driving research in progressive MS; in nervous system repair, recovery, and wellness; and in genetics and environmental risk factors for MS.
Progressive MS: We continue to focus on finding solutions for people experiencing worsening of disability that characterizes progressive MS, for whom there are few treatment options. The Society led an international effort culminating in the creation of the International Progressive MS Alliance, a growing global initiative to end progressive MS that has already funded 22 research grants across 9 countries, and is evaluating proposals for collaborative research networks – all part of an ambitious program to allocate at least $30 million over the next six years to find solutions for progressive MS. The Society is also investing in strategies to protect the nervous system from injury, and in advanced imaging techniques and other biomarkers to track progression and the success of therapies.
Nervous system repair, recovery, and wellness: Repairing myelin may represent the best strategy for protecting nerve fibers from injury and improving function for people with MS. Thanks in part to our pioneering funding, potential cell therapies and myelin repair strategies are now approaching or are already in clinical trials. Recovery can take other forms, and we are supporting more than 30 studies of novel exercise, rehabilitation, and other non-pharmaceutical programs to address symptoms and restore function. We recently launched a Wellness Initiative and are formulating priorities to drive research and programs aimed at helping people with MS live their best lives.
Genes/environmental risk factors: Researchers are starting to drill down into how genes and the environment interact to cause MS and possibly to influence the course of the disease. This area is ripe for additional efforts and could lead directly to new therapeutic targets for treating the disease and also eventually for preventing MS.