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 Appreciate this
| Reply
Bruce

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.

Leave a Comment

Thanks for sharing your thoughts with the MSconnection.org community. Please note comments are moderated.

    13 Comments

  • Kim   Sep 15, 2016 1:52 PM
    Thank you so much for the informative blog posts!
  • Kevin Rhodes   Sep 15, 2016 11:21 PM
    Thanks, Bruce. I appreciate you and your work. I confess that, as someone with primary progressive MS, my stomach churns every time I read that HSCT is not for me. I understand the propensity of researchers to be cautious, but frankly I would gladly take the risk. I suspect many -- maybe even most -- people with MS might feel the same. Kevin
  • Brian Melton   Sep 15, 2016 11:29 PM
    There are many PPMS patients that have halted there disease with HSCT. There are no trials so you won't here of any results.
  • Debby   Sep 17, 2016 6:40 AM
    My God daughter, Birthday 2 days ago, age 35, now in Hosice, dying end stage MS. She has had MS about 15 years, been bed ridden 10 last years. Mom was taking care of her, her 3 young children. Now has stayed at her bedside in hosice 10 days. Drs told her at hosp dx: seticemia, nothing more they could do, big new lesion in brain. Her husband told the girls mother shes not welcome to live there, so shes homeless and losing her daughter. The ms has her breathing shallow, only mouths the words, mostly un understable. Hands tremoring. Hallucing. Looking off in distant. Mostly sleeing. At times, restless, irritable. Has ain um. They are giving her 2 other meds that allow her rest, no agitation, no irritability. Its so hard for her family to watch. She is able to open her eyes to tell her mom and kids she loves them. Thats a beautiful thing. Only God knows the end. Just wanted to share this very fast rogressive ms story.
  • Myke   Sep 17, 2016 3:08 PM
    Are there any studies under way that would satisfy these doctor's needs for more data? Or are we decades away from that still?
  • Sue   Sep 17, 2016 9:19 PM
    Fascinating update.

    I diagnosed w/ MS through combined MRIs over 14 years ago when neurologists were looking for the cause of my headaches. Haven't solved headache dilemma, but basically physically symptom-free from MS for approx. 14 years, Originally tried Avonex & other interferons, even daily Copaxone injections, but found the treatment more disabling then the diagnosed disease.
    Still wonder if I should be considering renewing "preventative" treatment.
  • BrianD88  Sep 21, 2016 10:57 AM
    I was Diagnosed August of 2015 with an extremely aggressive form of MS and was lucky enough to meet the right Dr's to get help before it was to late. On January 21st I received my Stem Cell transplant and in the following months have gone from needing a wheelchair to a walker to nothing at all. I understand how lucky I am to have had this opportunity and I truly hope more research is able to be done to make this procedure more mainstream because it has changed my life, and truly hope it can change many more.
  • Kathy   Oct 3, 2016 12:34 AM
    I have Progressive ms and osteoporosis. I would like to know if Prolia has been researched by the MS researchers?
  • Jacquey mccreedy   Oct 4, 2016 8:00 AM
    I would love my info on hsct in Germany or Russia - are these
    The best places for treating my ms. And cost of treatment,
    And what is my next step
  • Cheltz   Oct 8, 2016 3:48 AM
    Very,very informative, thank you. There's a shocking, sadly Their Secret, their being the medical field, are extremely slow @ sharing any info with us the patients.
    M.S. My Shock - Stage 1
    M.S. My Secret - Stage 2
    M. S. My Sucks - Stage 3
    M.S. My SHUT UP
    M.S. My Simply a chronic illness that could be worse for heaven sake. : - )
  • HARVEY STELMAN   Oct 20, 2016 8:26 AM
    I'VE BEEN SUFFERING WITH SECOND DEGREE CHRONIC-PROGRESSIVE MS, DIAGNOSED IN 1981. I MYSELF TRACE IT BACK TO 1957. AT LEAST I HAVE A FUNCTIONAL RIGHT ARM AND HAND.

    I TRULY APPRECIATE ANY RESEARCH THAT IS BEING DONE ON MS. BUT THERE IS A POSITIVE IN THIS MISERY. I HAVE BECOME AN AUTHOR, NO BOOKS ON MS. BUT FOUR, AND VERY SHORTLY SOON FIVE BOOKS, ARE ON AMAZON. WE SHOULD ALL SEARCH FOR SOMETHING THAT STIMULATES US.
  • carolyn joyner   Dec 7, 2016 1:36 PM
    interesting article.i think hsct is the future treatment for ms
  • RITA   Sep 28, 2017 9:26 PM
    I want to thank prophet jeremiah omoto for helping my wife get pregnant, when we get married, my wife could not give birth and my family people were all saying it has been 11years now we are married no child, that i should get another wife for myself. i was tried and frustrated till i came across jeremiah omoto , who i contacted and behold my wife became pregnant. again all my thanks to prophet jeremiah omoto, the great man of God from Africa. If you out there need help to get pregnant, or for your wife to get pregnant, should contact the great man i contacted also his email prophetmercyland@gmail.com or watch him television marcylandtv com or +2347055176615 .