ECTRIMS 2016: Bone Marrow Transplantation (HSCT)

It’s nighttime here in London, England after the first full day of ECTRIMS – the European Committee for Treatment and Research in MS. This meeting is the world’s largest gathering of MS researchers in the world, with more than 8,000 clinical and research professionals from across the globe, including many Society-funded researchers and fellows, meeting to share their cutting-edge research findings, to network and collaborate. (All of the short summaries, or abstracts, presented at the meeting can be freely accessed here.)
It was a jam-packed day of science!  For this blog, I want to share my impressions of a staged debate that was focused on the topic of hematopoietic (bone marrow) stem cell transplantation – HSCT for short.
There is  a lot of interest in the potential of HSCT for treating MS, especially aggressive relapsing remitting forms, and this interest has intensified by recent success stories from studies like this one from the University of Ottawa and this one from Northwestern University. Yet there are still lots of unknowns in terms of who might benefit, what’s the best treatment regimen, and how to weigh potential benefits against risks for any particular individual.
For those familiar with this approach, feel free to skip to the next paragraph. For those not familiar, HSCT attempts to “reboot” the immune system and stop the inflammation that damages the brain and spinal cord in MS. Blood-producing stem cells from a person’s own bone marrow or blood are collected and stored, and the rest of a person’s immune cells are depleted by chemotherapy. Then the stored stem cells are reintroduced to the body, and they migrate to the bone marrow and over time produce new white blood cells. Eventually – usually within 3 to 6 months – they repopulate the body with immune cells. This serves to reset the immune system and stop the inflammation that contributes to active relapsing MS. (More information about HSCT is available here.)
For the ECTRIMS debate, arguing for the use of HSCT to treat very active relapsing remitting MS was Dr. Gianluigi Mancardi from the University of Genoa in Italy. Arguing not exactly against, but for more head-to-head evidence as to how HSCT stacks up against the most powerful approved treatments, was Dr. Jeffrey Cohen from the Cleveland Clinic. Both are experienced and thoughtful MS neurologists and researchers.
Dr. Mancardi made the point that HSCT was originally attempted for people with advanced disability in later, progressive stages of MS, for whom there were – and still are – few other treatment options. For these early attempts, the results were unreliable, and there was also a high risk of death from the procedure, so it was reserved as a “last resort.” Over many years the procedure has been refined making HSCT safer (although a risk for serious side effects and even death remains for some approaches). Through trial and error, there’s been a realization that this approach addresses the early inflammatory stages of relapsing MS, and doesn’t appear to be beneficial for less inflammatory, more progressive stages of MS. Dr. Mancardi argued that there is a place for HSCT for treating aggressive MS that is unresponsive to conventional MS therapies, but also noted that definitive trials are needed to determine its place in the MS treatment landscape. (Abstract 149)
Dr. Cohen agreed that improved approaches to immune cell depletion have improved the safety of HSCT when done by highly experienced centers, and its use in aggressive, relapsing MS has led to success stories of long-term absence of further disease activity and in some cases, even recovery of function. He reasoned, though, that HSCT trials have generally been small and uncontrolled (not randomized or blinded), and that HSCT has not been pitted against a powerful disease-modifying therapy in a head-to-head, well controlled clinical trial. He suggested that until such a trial is completed, HSCT should not generally be used to treat relapsing MS. (Abstract 150)
I’m not sure who won the debate, but one thing most agree on is the need for evidence from a larger, well-controlled trial to understand the best regimen, the most responsive patient population, and the comparative effectiveness of this approach against powerful immune-modulating therapies now available.
Want to read more? I recommend our Webpages focusing on HSCT, which provide FAQs and information on clinical trials recruiting participants. You’ll also find other ECTRIMS presentations on HSCT by following these links: 1, 2, 3, 4, 5
Stay tuned for more blogs providing the latest research results from ECTRIMS. 
Tags Relapsing MS, Research, Treatment      2

Bruce Bebo, PhD

Bruce Bebo, PhD, is Executive Vice President, Research at the National MS Society, and was previously a research immunologist focusing on the influence of sex hormones on MS. He is a driven and passionate Society volunteer, successful fundraiser and advocate, fueled in part by the fact that his mother had MS.