HSCT …a Cure?

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Can multiple sclerosis be cured? A case of highly active relapsing multiple sclerosis treated with autologous hematopoietic stem-cell transplantation 13 years ago

Jan Lycke, Markus AxelssonDOI: https://doi.org/10.1016/j.msard.2020.102253

A 26-year-old man, with five years of highly active deteriorating relapsing multiple sclerosis (MS), unresponsive to conventional therapy, was treated with autologous hematopoietic stem-cell transplantation (AHSCT) 13 years ago. Since then the patient had no clinical or neuroradiological disease activity and disability progression was halted. Repeated analysis of CSF revealed reduced levels of inflammatory biomarkers and the neurofilament light protein level was normalized indicating no further axonal degeneration. The patient is socio-economic independent, is working full time, and has become a father. Measures of quality of life and cognition did not indicate further deterioration. Long-term follow-up has not shown any signs of active disease suggesting that AHSCT may be a cure for MS.

Let’s hope so. I am sure people will make the case that this has happened with alemtuzumab maybe in ten years people will make the claim for cladribine also. The question is how common are the good news stories verses the bad news stories

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MouseDoctor

28 comments

  • Mouse,

    Thanks for this story – makes a change from Covid 19 stories. I think most of the participants in the BMT trial in Canada went into long term remission. Like you, I expect that some Alemtuzumab patients go into long term remission. Like most things in life there will be many factors at play in determining who does really well – how long they have had the disease, their individual response to the therapy..

  • For people with MS presentations like mine (early onset, creeping PPMS), HSCT or Alemtuzumab etc. are very unlikely to be a “cure”.

  • Hi MD, my day has been a bit rubbish (pardon the pun – you’ll get this soon) as I’ve just come back from the tip with my garden waste (lots of it) as there’s a 2 hour wait time! My son is 21 on the 29th & I’ve just arranged a Zoom party (oh yeah, I’m feeling in tune with lock-down!) & my cat has a nasty cut on his back after a fight with the neighbourhood cat gang! So to read this story, has brightened my day & I completely agree with the comments said too! One thing that all HCPs should keep talking about is the cases that are working or doing okay. To give hope, & especially in this case to become a father and maintain a full-time job – is such an amazing achievement, that everyone else takes so much for granted!

    Modern living isn’t so great when no-one appreciates their health and the availability of modern medicine to essentially re-boot this man’s immune system. I would like to hear the good and the not so good, along with a summary as to why sometimes the treatment hasn’t worked or potentially caused other problems. The overseas offers that pwMS receives are extremely tempting to go through this treatment – but we need to know more about its availability, safeness, trials and costs in the UK more, so that we aren’t tempting to go overseas and seek treatment.

    Keep the stories coming – as when there’s hope, we can all have a lift in spirits and keep going.

    Many thanks
    Jane

  • I know many people who have had very successful HSCT for MS. I also know a few who got worse from HSCT. Unfortunately, I was one who got much worse. The evidence is clearly in favor of HSCT being successful, but do not believe the people on the forums who say it’s impossible for HSCT to make one worse. And yes, I get worse despite consistently working my ass off to recover. My HSCT transplant doctor confirmed that it is possible that HSCT can make one worse, especially for those who have had PPMS for many years without active lesions at time of transplant. Also, the vast majority of the HSCT forums on Facebook are highly controlled by one individual who is clearly biased by the fact that he profits indirectly by patients who have HSCT at Clinica Ruiz in Mexico. He is a paid “consultant” with the company that manages the patient housing, food, etc. His views are held as gospel by his followers and he will quickly berate and belittle those who question whether HSCT can make one worse, and he gets very defensive regarding whether he profits from patients. Make no mistake, he absolutely has a financial interest in promoting the positives of HSCT and dismissing the potential risks. But for those with RRMS, SPMS, and those who have had PPMS for less than ten years, the odds are in your favor. You will find many with PPMS longer than 10 years at time of transplant who THINK they had successful HSCT, but if you follow-up with them more than two years post you will be hard pressed to find a single person who had PPMS for more than 10 years who actually had successful HSCT. I only know of one exception, and he was a rare bird who had PPMS for more than 10 years and also had an active lesion at time of transplant. The other PPMSers who had successful HSCT had PPMS for less than 10 years at time of transplant.

  • 20 years

    Sandi Abram Selvi

    Was one of the first patients doing hsct for ms

    31 of march 2020 she celebrate 20 years after tanspant she have writen a book a did a ted talk

    “A Stem Cell Transplant MS Recovery Story: Beating Multiple Sclerosis with Humor, Hope & Science”

    https://www.youtube.com/watch?v=CB52rXR2x_M&feature=emb_title

    These what she have to say

    Well world, can not believe it myself, but tomorrow is my 20th anniversary from having my HSCT, Hematopoietic Stem Cell Transplant for MS, (Multiple Sclerosis). And my first reason I believe politics and science do not make good bed fellows.
    If you don’t know my story, I was diagnosed with MS in 1995. I went from being a totally healthy young woman to a very sick woman quickly, with two spirited young sons, who were at the time, 6 and 8 years old, I had to do something different than the medication that wasn’t working. I quickly went from remitting relapsing, to secondary progressive. My symptoms of: drop foot (left side), voice tremors, body tremors, hot and cold sensitivity, no bladder control, no equilibrium, no energy, double vision, sleep issues and deep depression, were getting worse by the day.
    On March 31, 2000, I had my Stem Cell Transplant using my own stem cells. I was a total experiment based on medical results of past people who had both cancer and MS. When those cancer patients went through bone marrow transplants or stem cell transplants, not only did their cancer go away, but their MS started to abate. It made total sense to me. MS is my immune system attacking itself, so kill the old immune system and build a new one. Like rebooting your computer. I was desperate, and declining fast, so I decided to go ahead, fingers crossed, to be the second person in California, with no cancer, to do a complete Stem Cell Transplant, to see if my MS would go away. Again, fingers crossed. Thank God, the science worked.
    At the time our president, Bush Jr. decided that stem cell transplants were, as he put it, “messin with God.” So, on August 9, 2001, he introduced a ban on federal funding for research on newly created human embryonic stem cell lines, which pretty much shut down all Stem Cell research. Thank the same a God, I had already had my transplant, and was already increasingly getting better and, by the way, have continued to get better every year since. So many people that could have been helped, had to wait, until President Obama realized the mistake that Bush made, and reversed it. Which aloud Stem Cell research to begin again in the US.
    If you knew me back before my transplant, I was walking with a cane, couldn’t talk, and wanted to just stay in bed. You would be surprised to see me now! I’m performing comedy, playing golf and living a life I never thought would be possible, 20 years ago.
    Conclusion… science and politics do not mix. Presidents should not be making decisions on medical issues, EVER! Please leave the major medical decisions to the doctors and people who know what they are talking about, and can act without religious prejudice or fear of looking bad. Religion and Egos should be left completely out of the medical equation.
    I’m so grateful to my husband for being here and having the ability to help me get it done.

  • Prologue
    In September 2003 a young woman fell ill. It began with a facial palsy, soon
    followed by weakness in a leg, pronounced fatigue and loss of vision. A
    diagnosis of multiple sclerosis was made. Although these symptoms got
    better, others more ominous took their place. She became paralyzed from the
    waist down and her bladder stopped working. She got treatment and again
    she got better for a while. However, this was just a short respite and by
    spring she was completely paralyzed.
    She was transferred to the University Hospital. At the darkest hour, she
    was offered a novel treatment. Hematopoietic stem cell transplantation. With
    little to lose and everything to gain, she accepted. None could have guessed
    at the outcome.
    A few days into procedure, she was able move her toes again, for the first
    time in two months. Rapid improvement followed. Some weeks after
    discharge, she could walk with a stroller. After three months she could walk
    unaided. After one year she was working part-time.
    Ten years later, she is living a normal life. She works full-time. She is the
    mother of two healthy children. She has no treatment. She has not had any
    relapses.
    Is she cured from multiple sclerosis?

    Curing Multiple Sclerosis
    How to do it and how to prove it

    Joachim burman

    docent, consultant at Department of Neuroscience, Landtblom: Neurology

    uppsala university

    https://www.diva-portal.org/smash/record.jsf?pid=diva2%3A714003&dswid=-8087

  • This is a lovely heart warming good news story to cheer us all up. Yes, would have tied in with Father’s Day (🤔 when is that btw?) beautifully 😊

    Of course tells us little except treat MS early and aggressively. More’s the pity you have to jump through hoops to get anything like this good… and that was before COVID-19 put a spanner in the works.

  • And the people for whom it did not work? CURE pahhh surely a cure will work for everyone? HSCT I wouldn’t feed it to my dog.

  • An interesting question would be did they become EBV negative. I have heard of EBV being cleared by a transplant from someone else. If it did not remove the EBV completely, then that insinuates it is not the virus on its own that causes the disease but something specific it does to the immune system, a specific mutation. This boldly assumes of course that EBV is the trigger of MS.

    • The way we do HSCT patients lose all immunological memory and patients have to be re-immunized to everything 18 months post transplant. We have never tested EBV reactivity per se post-transplant, but since everything else disappears, it is probable that evidence of immunity has also been lost. This does not mean that the inciting factor causing the disease at the beginning was not linked to EBV.

      • Results: All patients showed a complete and sustained engraftment: median (range) time to PMN≥0.5×109/L and platelets≥20×109/L were 12(9-15) and 11(7-14) days, respectively. Patients showed also a complete return to a normal lymphocyte subset counts within two years from transplant. No case of measles, rubella, or chickenpox occurred after transplant, with a median follow-up of 40 (17-124) months. Table 1 reports the number of patients with positive or negative antibodies titres at baseline, 6 months and 2 years after aHSCT, respectively. A loss of serum protective immunity was observed in a variable rate of patients with all the tested agents: 14% in measles, 16% in rubella, 20% in hepatitis B, and 7% in Varicella Zoster.
        Conclusions: Re-vaccination in patients who underwent an autologous HSCT for a severe autoimmune disease is a common practice in many centers and is also recommended in the EBMT guidelines. However, some concerns about this practice were raised for the putative role of vaccinations as an autoimmunity trigger. In the literature there is no study on the loss of protective antibody titer in either natural or vaccination-driven immunity with regards to patients transplanted for autoimmune diseases. In our series a high variability in antibodies titers at baseline was shown. The larger subset analysed confirms that most patients showing a protective titre against the tested agents, show a trend to maintaining it. A possible approach might be to revaccinate the patients with unprotective titre at 2 years after HSCT, taking into account the individual risk of being exposed to the infection; a larger series of data is necessary to provide conclusive evidences on this topic.

        RECONSTITUTION OF NATURAL OR VACCINATION-DRIVEN IMMUNITY AFTER AHSCT IN MULTIPLE SCLEROSIS
        Chiara Innocenti, Elio Ingenito, Maria Pia Amato, Alessandro Barilaro, Gaetano Bianchini, Teresa Capobianco, Maria Antonia Calia, Romina Ceschini, Arianna Fani, Marta Giannini, Antonella Gozzini, Stefano Guidi, Alice Mariottini, Luca Massacesi, Francesca Materozzi, Chiara Nozzoli, Anna Maria Repice, Gian Maria Rossolini, Serena Urbani, Riccardo Saccardi Careggi University Hospital, Florence, Italy

        Ebmt 2020

        • Perhaps this is the difference between a fully (Bu-Cy) and partially (BEAM) myeloablation? Immunological memory carries memory of the disease. Wiping out that memory requires wiping out all memory.

      • I was not thinking about immunity I was thinking about the EBV virus. As the virus itself resides in the immune system I wondered if you became clear of the virus, not if you lost immunity and had to rebuild it.

        • The virus, particularly Herpes viruses, are part of us as you say. So are many viruses. The concern with HSCT is that under normal circumstances, such viruses are held in check by an active immune system. When you remove the immune system – things can happen. PML is an example of a virus (the JC virus) that can cause significant damage in a compromised host, but not in anyone with a fully functioning immune system. Herpes Zoster – from Chickenpox, causes Shingles in compromised hosts too. We did NOT see emergence of PML or any EBV-related infections post HSCT, but Zoster was predictable, which is why patients were maintained on anti-viral therapy for at least a year.

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