Progressive MS at ECTRIMS: New Directions and Challenges

Greetings from the second full day of the ECTRIMS2016 conference in London. Today was packed with research presentations and poster sessions, all about MS. Anyone who wants to take a deeper dive can freely browse the abstracts (summaries of conference presentations) here.
There are many different topics being covered at this meeting, and I’d like to focus this blog on one I’m particularly passionate about, progressive MS. A press conference yesterday hosted by the International Progressive MS Alliance, which I help lead, announced new investments of over $14 million US dollars to support three Collaborative Network Awards. These international teams were selected to accelerate the pace of research in key areas. 
These new 4-year projects address knowledge gaps to speed new therapies for progressive MS. Many of the ECTRIMS presentations have also focused on some of these gaps, including studies looking for ways to speed up the testing of new treatment strategies. This means developing new outcome measures for clinical trials, providing better ways to detect – with imaging or a blood test or other “biomarkers” – whether or not a new treatment has potential benefit.
In an interesting talk I attended, Dr. Jeremy Chataway from University College London addressed the need for new designs for clinical trials that will make them more efficient at determining whether a treatment works (Abstract 218).
Typical clinical trials run side-by-side groups, where one group gets the experimental treatment and the other serves as a control group that gets either an established treatment or an inactive placebo. This traditional design can take years to complete. To decrease the time needed to get results, Dr. Chataway is leading a trial in the UK called the MS-SMART trial. It’s testing three new therapies in parallel groups, all compared against one control group. Part of the design includes the potential to gradually eliminate treatments that are not working, and switch more people to the one showing the most benefit. So in about the same time it takes to do a traditional trial of one treatment, they will have tested three. Time is critical for people living with progressive MS and this is a trial moving forward with a sense of urgency. And, this is just one example of several new trial designs that I hope will be fruitful.
I talked before about researchers working to find a way to determine with a simple test, or biomarker, how to predict a person’s disease course and progression, and provide a quick and clear signal that a treatment is working. In the world of cancer treatment, there’s been some success with developing this kind of test to help determine the best treatments for an individual.
In this regard I was intrigued by progress reported by the National Institute of Health team led by Dr. Bibiana Bielekova, which used a very high-tech approach to examine proteins in the spinal fluid of people with neurological diseases, including all types of MS (Abstract 219). The team identified a “signature” of markers that distinguished MS from other diseases, and also differentiated relapsing MS from progressive MS. These are early results that need further refinement and development, but it illustrates that this kind of approach is on its way to clinical trials and clinical practice.
Even while scientists are still working to develop methods to quickly determine whether or not a treatment is working for people with progressive MS, new therapies are in the pipeline. For example, there were several reports related to ongoing research on the potential of specific types of stem cells, called mesenchymal stem cells, taken from bone marrow cells. (Read more about mesenchymal stem cell research here.) My colleague Bruce Bebo wrote a blog about the HSCT bone marrow stem cell approach – read it here.
A team from Israel led by Dr. Dimitrios Karussis (who I met last year at a conference on cell therapies) from Hadassah University Hospital reported on an ongoing phase 2 clinical trial they have been conducting to figure out the optimal way to administer mesenchymal stem cells in active, progressive MS (Abstract P1290). Earlier work, especially in mice, suggest that these cells may create conditions that protect the nervous system from damage and the hope is that they may also enhance repair. They plan to enroll 48 people with progressive MS with moderate to more severe disability (EDSS 3.5-6.5). They are administering the cells into the vein or the spinal fluid and then monitoring patients with various measures to try to detect a benefit and to find the best route for administering these cells. So far they have enrolled 39 people, and have not reported any serious adverse events, with the most common side effect being headache. This trial is ongoing and it will be interesting to see whether this approach can show benefit.
Tune in tomorrow and Monday for more blogs from my colleagues about specific results related to treatment of secondary progressive MS, the role of gut bacteria in driving or reducing MS activity, and new advances toward addressing symptoms that interfere with daily living.
It’s clear that to make inroads toward treatments that will stop and even reverse progressive disability, collaboration amongst investigators and patient participation are essential components to success.
Tags Progressive MS, Research      2 Appreciate this

Douglas Landsman, PhD

Dr. Douglas Landsman is Vice President, Researchh at the National MS Society. He leads the biomedical research and fellowship/faculty award programs, and plays a key role in the International Progressive MS Alliance. He has a long-standing interest in nerve-muscle interaction and developing strategies for promoting nervous system repair after disease or injury.