New Findings on Stem Cell Transplantation, including HSCT, in MS

Transplantation of stem cells is a promising area of research that could lead to strategies that stop or even reverse damage caused by MS. Advances in this approach were presented during the ECTRIMS meeting in Barcelona this week. Stem cells can be found in many parts of the body and they have the remarkable potential to develop into many different cell types that have the potential to regenerate damaged tissue. This ability offers a new approach for treating diseases, including MS. In this update, I will be talking about two promising approaches: hematopoietic stem cell transplantation, and mesenchymal stem cell transplantation.

Hematopoietic Stem Cell Transplantation (HSCT)

HSCT is a procedure that involves transplanting of a person’s own stem cells derived from bone marrow after their immune system has been depleted, in an attempt to regenerate and “reboot” the immune system to stop MS attacks. This week an international consortium – that included 28 scientists from 10 different countries – presented their observations of outcomes of HSCT transplants involving 281 people who were followed anywhere from 2 months to 16 years after undergoing the procedure.

Most (78%) of the people they evaluated had progressive forms of MS, and their disability status measured by the EDSS scale ranged from very mild to very severe (1.5 to 9). The key outcomes the researchers focused on were progression-free survival and overall survival 5 years after the transplant procedure.

They found that the chances that participants were free of progression after 5 years were about 50-50. They also found that the probability of survival after 5 years was 93%, which are much better odds! So who did better, and who did worse? When they looked at the group who continued to experience progression after the procedure, they tended to be over age 37, had progressive MS, and had tried more than 2 disease-modifying therapies before getting the transplant. The researchers also looked at improvements that occurred during the year following the transplant, which were notable in 52% of those with relapsing MS and 31% of those with progressive MS.

These findings underscore for me the dilemma about HSCT, and why it needs more evaluation through additional clinical trials that are now underway. Those who did better were younger people with active, relapsing MS who haven’t tried as many approved therapies. These are the individuals for whom there are other less invasive therapies that might work well. Stem cell therapy, even in the controlled setting of a clinical trial, carries the possibility for substantial risks, and many doctors are uncomfortable recommending this procedure to people who may have other treatment options.  We need to fully understand the risks and benefits of this before a conclusion can be reached about this approach.  The ongoing clinical trials of HSCT will hopefully provide this for us in the near future. (Abstract 193)

MSC’s can be found in most tissues in the body, but the bone marrow, umbilical cord and adipose (fat) tissue are good sources for these cells. MSC’s have tremendous promise to help us understand and treat a range of diseases, including MS, but very few MSC-based treatments have been proven safe and effective so far - despite claims to the contrary.

One roadblock to establishing the clinical usefulness of MSC’s is that different teams use different treatment protocols, so it’s difficult to compare the outcomes of results. That’s why I was also encouraged to learn about a unique collaborative trial that’s getting underway in 8 countries that involves infusing MSC’s without depleting the immune system. The idea is to improve the body’s ability to repair damage to nervous system tissues. This “MESEMS” trial involves a network of independent trials, all following the same protocol and sending their data and MRI scans to centralized locations for combined analysis. This way they can increase the number of participants overall and get more powerful results.

They are planning to enroll about 160 people across Europe and Canada, and they will be recruiting participants with relapsing-remitting, secondary progressive or primary progressive MS as long as they show signs of active disease or inflammation. The researchers will be looking at safety, and also impacts on MRI and disease activity. If this strategy proves to be safe and effective, it will probably lead to a larger phase III trial. This novel approach is exciting, and an important step towards determining the full potential of these cells for the treatment of MS. (Abstract P1087)

Click here for more information about research in stem cells and MS

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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.

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    27 Comments

  • Lizz   Oct 10, 2015 9:03 PM
    Hi , my name Lizz ..and have suffered from MS for 20t now the relapse remit kind..I'd like to be considered for the next stem cell research program
    it's been very difficult having this disease ..I'm a mom & a wife ... and I want more for my family ..I'd like to know the ole me or at least get off these horrible Meds .. thanks for considering me ..Lizz Hahn
  • Cheryl Blanchfield   Oct 10, 2015 11:26 PM
    How old are the articles you are quoting? Radiation treatment is no longer used for MS or other Autoimmune diseases. HSCT is no longer experimental, it has been used to treat MS and other Autoimmune diseases for over 15 years. You are quoting old data. It is about time you pushed for this treatment as an option for ALL MS and other Autoimmune diseases. If I had known about this and been able to access it 5 years ago when I was diagnosed, I would not be as disabled and out of work as I am now.
  • Ally Grant   Oct 11, 2015 1:55 AM
    I would also like to know exactly how old this data is ? I am about to undergo HSCT later this month. Unfortunately it is still not a recognised treatment for MS in Australia, so I must travel overseas. I have only recently ( January 2015 ) been diagnosed with RRMS and I am so glad that I stumbled upon this so early on in this ugly disease. Say what you will but I am a believer of the benefits of this treatment.
  • Anonymous Anon   Oct 11, 2015 2:36 PM
    Unfortunately, many national campaigns seem to seek to minimize the disease. It's very easy to do by focusing on the newly diagnosed or those whose disease course is moving along slowly.
  • Sanford Siegel   Oct 11, 2015 3:21 PM
    So far despite extensive research Stem Cells have been a failure in reproducing Myelin in the brain/spine. YOUR BEST BET, IN FACT YOUR ONLY BET, is to find a doctor you trust and do what he/she says.
  • Eileen Adams   Oct 11, 2015 3:23 PM
    Please if you need anyone for the trials please let me know i am in pain 80% of day & night the treatment i use helps for a short while but i am at my wits end please help me if you can a very tired & painfull ms sufferer thank you.
  • Sanford Siegel   Oct 11, 2015 3:24 PM
    It doesn't matter what you think or believe. The only thing that matters is what works or what partially works. So far, Stem Cells have been a dismal failure in the treatment of MS. These are the facts and the facts can not be disputed.
  • Eleni   Oct 11, 2015 6:49 PM
    The key factor of hsct is the chemo, not the stem cells. The hematopoeitic stem cells help rebuild a new immune system minus it's autoimmune dysfunction. The statics from Dr. Burts trial (northwestern University Hospital) show that over 85% remain relapse free after 5 years. Please don't confuse hsct with msc
  • Ms. Carlotta Cosme   Oct 11, 2015 7:24 PM
    Greetings:
    I have Secondary Progessive MS, and very interested in HSCT Reseach, How may I be apart of the research process.
    Thank you.
  • David M.   Oct 12, 2015 3:11 PM
    Sanford, I'm not quite as convinced as you seem to be regarding MSC treatment. There is significant information that shows promise for MSC/NSC therapy for MS coming from research in Asia.
  • aminfla  Oct 15, 2015 6:30 AM
    Dr Burts HSCT trials at Northwest University is ongoing for RRMS. The "FACTS" stated in the article seem to go back to the days of radiation being used. NOT DONE ANY LONGER. As Michael Barrow is just one of the many treated under the HSCT CURRENT protocol and his success is one of many. But do remember the point of HSCT is stopping progression. Recovery is based on the individual. clinical trial dot gov will tell you about the trial. It is also on facebook by looking up Dr Burt.
  • Vanessa   Oct 15, 2015 7:03 AM
    Where can I find a list of neurologist equipped to prescribe this procedure/treatment?
  • James Mangos   Oct 15, 2015 10:51 AM
    I have primary prgressive MS. I would like to be included in any trials that stem cells are used. I have heard great things about this therapy. Thank you for including me in your trials.
  • Debbie   Oct 15, 2015 3:11 PM
    Dr. Burt in Chicago at Northwestern University is conducting HSCT. You can view some of his videos on YouTube. You can also find him on facebook on a private page if you search for him. My son is the process of HSCT treatment with Dr. Burt for MS. I am so thankful for this man and his research. #STOPMS
  • Vanessa   Oct 22, 2015 5:42 PM
    I've had MS for over 25 yrs & think this may be the future. I have seen the evolution of this disease. From snake venom, scorpion & bee venom to drug therapies, to spinal taps, CT & MRI.. We've come a long way. Neurologist have a full arsenal of drugs to treat r/r MS when I was diagnosed there were two & the first was given based on a lottery. I'm now secondary progressive & would like to be considered for cell transplantation .
  • Andy Newton   Oct 27, 2015 2:23 PM
    This is a very interesting article. My wife has progressive MS. We live in Trinidad and are looking for treatment options for her. We are wondering if she would qualify to participate in one of the clinical trials. We look forward to hearing from you on this.
  • paula tournay   Oct 27, 2015 11:40 PM
    l am always interested in doing trial studies if it can help someone else l have had MS over 40yrs. and only the right sided weakness so if you ever need someone for questionares or trial medicine I live in the Pittsburgh,PA area would be willing to help.
  • Richard G Green   Oct 28, 2015 9:14 AM
    Why is the USA lagging behind so many other countries when it comes to the availability of Stem Cell therapy? Why can't my Neurologist put stem cells on my Myelin Sheath? I know it's political, FDA red tape, and other BS! People with progressive, pain filled, no relief MS in the USA have no hope of seeing anything positive in our lifetime!
  • Richard   Oct 28, 2015 1:01 PM
    I have been looking into HSCT abroad. The Donald says make America great again but this procedure is common practice in other countries. How can we be great when pharmaceutical companies hold us back?
  • Richard   Oct 28, 2015 1:11 PM
    My Tysabri shot is $19,606 every 28 days. HSCT in Mexico is $55,000 add another $10,000 for airfare, hotel, etc... My insurance company is getting ripped off. MS patients are suffering while pharmaceutical compaies are making a fortune. FDA? It will never be as long as it will decline income to the pharmaceutical companies. Ask yourself, Why do they back up only the things that make us live longer with this disease? Could it be, the longer we live the More they get to sell?
  • jacquelynle  Nov 11, 2015 10:52 PM
    I am not soliciting - offering, or attempting to purchase, goods or services so I am hoping this is okay....

    Provide HSCT for Multiple Sclerosis as a recognized treatment option.

    I am hoping to get support for this. I am a Canadian so the only way I can make pressure to get HSCT recognized and allowed as an optional treatment is to post a change.org petition request.

    If I have any success at all with our newly elected government - then I believe it would put pressure on other countries to follow suit, either out of common sense or national pride, or even a sense of competition - hopefully just out of compassion and realization what an ugly disease this is.

    Canadians believing HSCT should be a patient's option for treatment please sign, and even if you are not Canadian, please sign because if this is successful and our country offers this procedure - other countries will follow.

    https://www.change.org/p/info-cahspr-ca-sab-ccs-hc-sc-gc-ca-phecg-secretariat-hc-sc-gc-ca-public-health-agency-of-canada-s-public-health-ethics-consultative-group-phecg-col-maureen-haberstock-jane-philpott-parl-gc-provide-hsct-for-multiple-sclerosis-as-a-recognized-treatment?recruiter=28470825&utm_source=share_petition&utm_medium=email&utm_campaign=share_email_responsive&rp_sharecordion_checklist=control
  • j. Sherst   Nov 28, 2015 11:57 AM
    Two interesting fairly recent success stories - one from a person who had MS for ten years and in a wheelchair before treatment and a grandmother with NMO syndrome (not NMO but lesions damage tends to be spinal).
    http://www.kgw.com/story/news/2014/08/04/13333484/
    https://kassbeatsnmo.wordpress.com/2015/11/25/progress/

    The sad thing is clinical trials are forced to take small segments of people, so they have to have very narrow requirements. All lot of the progressives, and people that have had the disease for a long time, and older people were only being looked at for a short time at the beginning, before more and more refinements were made to the treatment itself. I believe this segment was given up on far too soon.
    It is sad, but if you look online to find people saving up for a trip to places that will do the treatment for these segments, or are fighting in the US to get their insurance company to cover it, you will find that the people that get this treatment are getting their MS halted.
    Yes it looks more splashy for the newly acquired MS'ers because it looks like it reverses lesions, however these are are not completed lesions, they are inflammations that may or may not stay - they may not have had the axon destroyed but are in the twilight time of it becoming destroyed. When the immune system is replaced, these not completely destroyed areas heal, thus it looks like it reverses the damage. It doesn't, it was just that the damage was at a stage where it was a not completed process.

    For those who are older, or who have had MS for longer, may have more axon damage. That is why it does not show up as repair only as a halt to the disease.
    There is also a segment of people - I don't believe it is just because of having MS for a long time, will have MS return after HSCT. I believe it is because whatever triggered MS for them, is still either in their DNA, or environment - they unfortunate came across the same virus, or whatever that their particular autoimmune system can't process properly.

    People with cancer can get chemo/bone marrow transplant - same procedure. They are allowed to go through this procedure as many times as they want, even if it is life threatening, should their cancer return. For many of them it does. People with cancer can get the chemo/bone marrow transplant up to age 65. This is the same procedure as HSCT.

    Most people do not last with MS for more than 20 years before they become incapacitated. A person that has MS for 30 years is fairly rare (and lucky or unlucky depending how miserable their existence was with this debilitating disease) Most people can't walk by their 20th year. 80% of people with MS no longer can hold jobs (from MS Society canada stats). Most of them die from comorbid complications that arise because of MS. People make to much light of the degenerating nature and quality of life for people with MS.

    It should be a choice, for people. Yes it is grueling, yes there chances of negative outcomes but cancer patients are allowed this choice, their outcomes can be even grimmer.

    If you stay on any immune suppressant drug, they also have really bad outcomes - heart issues for some, PML gets to be more and more of a risk with every year you take an immune suppressing drug.

    Also if an MS patient is taking immune suppressant drugs for a long time, and their immune system becomes less robust and they later have an opportunity for HSCT it may raise the chance of possibly developing PML from an HSCT procedure. The HSCT does not cause PML, the prior length of time taking the immune suppressive drug does. In fact most of us have to change MS drugs every several years because the side effects get to the point where it is not to our advantage to take them any longer. No drug stops MS. HSCT can.

    We are not lepers we should be able to be treated with as much respect as the cancer patient that can select to have HSCT. When will anyone take us seriously? The symptoms of MS can be horrible. Loss is horrible. It we want to take that change to stop it, why can't we be allowed that? Not everyone can withstand, nor wants to withstand the challenges of HSCT but for any of us that are healthy enough to try this treatment, we should be given this option.

    People are now going by the masses to other countries to pay between $45,000 and $200,000 us to get this treatment done. This is not fair. It also is hardest on people with families who really can't afford this but really can't afford not to.

    Northwestern University is on a 3rd tier stem cell trial where they are opening up the requirements wide - however it costs $150,000 for those who don't have private insurance to cover it.

    Private insurance companies, however, are on board for most part - lets see $60,000 for ms drug X per year versus $150,000 one time (hopefully) treatment.
    To an insurance company they start saving money by the 3rd year, good business.
    No MS drugs 1st year & 2nd year $60,000 + $60,000. year three save $60,000
    180,000 - 150,000 saves them paying out $30,000 in year three.

    But that again leaves out the people who do not have insurance that recognizes this treatment. Again, like the poor, like lepers - no choice, unseen, and others beyond our power are dictating our options, Not a pretty picture.
  • Nichole   Jan 3, 2016 10:00 PM
    I need this treatment!.. officially I've had ms for a yr, but tested positive back in 2008, but the doc said I was fine.when I found out they lied to me I was mortified. .I've been relapsing for a yr straight, it never stops. I've already tried 2 dmds but they both almost killed me..dmds don't help, they seem to make it worse! Please consider me for this trial
  • Sabina Diestl-MCCauley   Jan 7, 2016 1:50 PM
    MS is NOT a single disease, and cannot be treated as such. It is as diverse and multidimensional/multifaceted as is possible in Nature. Some treatments work for some people, others work for others and in some cases, nothing at all works. Be careful what you try.
  • TIFFANI RATLIFF   Jan 17, 2016 9:16 AM
    I've researched HSCT in depth for many years. Personally, I feel HSCT to be my ONLY option now. How does one afford the cost of treatment when one barely survives with benefits paid through SSDI? Sadly, most insirances
  • TIFFANI RATLIFF   Jan 17, 2016 9:22 AM
    I've researched HSCT in depth for many years. Personally, I feel HSCT is my only option with Progressive-relapsing MS. How does one afford treatment when they're barely surviving through benefits paid by SSDI? Sadly, many insurances do not cover costs of HSCT. Whigh is the equivalency of dangling meat in front of a hungry alligator. A sad form of torture imo.

    Thank you for your time!
  • Ruby Andujar   Jan 16, 2019 6:35 PM
    Hi my name is Ruby andujar and I have a 22 year old daughter who was diagnosed with multiple sclerosis when she was 17 years old she has progressive relapsing remitting multiple sclerosis she has used every medication given except lemtrada for her multiple sclerosis right now we
    have beenn reading all types of researches, journals and documentation of people with Ms who have partaken in the stem cell therapy and I want to know how can we be part of the research we want to try anything to help our daughter and she has been through a lot of changes and we're trying to see how can we get stem cell for her. Please if you can reach out to me , I would surely appreciate it. Thank you for your time thank you for your new findings on stem cell transplantation including hsct in Ms I found your article to be very interesting and I'm hoping that you would contact me . Thank you for your time