Three Years After Stem Cell Transplant Therapy Most MS Patient Subjects Still In Remission

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A new study published online before print in the journal JAMA Neurology reports that three years on, most members of a small subject group of patients with active relapsing-remitting (RR) multiple sclerosis (MS) who had received an experimental high-dose immunosuppressive therapy (HDIT) followed by a transplant of their own hematopoietic stem cells, remain in sustained remission with lasting neurological function improvement.

The Open Source research update, entitled “High-Dose Immunosuppressive Therapy and Autologous Hematopoietic Cell Transplantation for Relapsing-Remitting Multiple Sclerosis (HALT-MS) – A 3-Year Interim Report,” (JAMA Neurol. Published online December 29, 2014; DOI: 10.1001/jamaneurol.2014.378) is coauthored by Richard A. Nash, MD, George J. Hutton, MD; Michael K. Racke, MD; Uday Popat, MD; Steven M. Devine, MD; Linda M. Griffith, MD, PhD; Paolo A. Muraro, MD, PhD; Harry Openshaw, MD; Peter H. Sayre, MD, PhD9,10; Olaf Stüve, MD, PhD11,12,13; Douglas L. Arnold, MD; Meagan E. Spychala, DrPH; Kaitlyn C. McConville, MS Kristina M. Harris, PhD; Deborah Phippard, PhD; George E. Georges, MD, Annette Wundes, MD; George H. Kraft, MD, MS, and James D. Bowen, MD, representing 22 different medical research institutions in the U.S..Canada, and the U.K.

In an article study, lead author Richard A. Nash, M.D. of the Colorado Blood Cancer Institute at Presbyterian/St. Luke’s Medical Center, Denver, and coauthors report on the safety, efficacy and sustainability of MS disease stabilization though three years after the procedures.

The coauthors observe that most patients with relapsing-remitting multiple sclerosis who are treated with approved disease-modifying therapies will experience breakthrough disease and accumulate neurologic disability. However RRMS patient participants in a clinical trial who received high-dose immunosuppressive therapy (HDIT) with autologous hematopoietic cell transplant (HCT) may, by contrast, experience sustained remissions in cases of MS treated early.

The update published this week in JAMA Neurology evaluates the safety, efficacy, and durability of MS disease stabilization achieved in the three years after the HDIT/HCT therapy was administered to a group of 25 participants, 24 of whom ultimately received the experimental treatment.

NAshRHematopoietic Cell Transplantation for Relapsing-Remitting Multiple Sclerosis (HALT-MS) is an ongoing, multicenter, single-arm, phase 2 clinical trial of HDIT/HCT for patients with RRMS who have experienced relapses involving neurologic function loss during treatment with other disease-modifying therapies within 18 months prior to enrolment. Participants are to be evaluated through five years after receiving the HCT treatment, and this report is a scheduled three-year interim analysis of the trial in which autologous peripheral blood stem cell grafts were CD34+ selected, after which participants received high-dose treatment with carmustine, etoposide, cytarabine, and melphalan as well as rabbit antithymocyte globulin before receiving autologous HCT.

The main end point objective of HALT-MS is “event-free survival,” which is defined as survival without death or disease activity from any of specified outcomes: 1) confirmed loss of neurologic function, 2) clinical relapse, or 3) observation of new lesions using magnetic resonance imaging (MRI). Any toxic effects associated with the experimental treatment are to be reported using National Cancer Institute Common Terminology Criteria for Adverse Events. The median follow-up interval was 186 weeks, and overall event-free survival averaged 78.4% (ranging 90% CI, 60.1%-89.0%) at the three year mark. Progression-free survival and clinical relapse-free survival were 90.9 percent and 86.3 percent, respectively, at three years. The researchers report that adverse events observed were consistent with what had been expected with HDIT/HCT, and no acute treatment-related neurologic adverse events were observed. Improvement was observed in subjects’ neurologic disability, quality-of-life, and functional scores.

The scientists conclude that at the three-year mark in the trial, HDIT/HCT without and maintenance therapy was observed to be effective at inducing sustained remission of active RRMS and also associated with neurologic function improvements, and only a few serious early complications or unexpected adverse events.

The JAMA Neurology report’s coauthors note that researchers have been investigating autologous hematopoietic cell transplant (HCT) therapy for more than 20 years — the hoped-for objective being removal of disease-inducing immune cells and an immune system reset. However, many patients with advanced disabilities and progressive forms of MS who participated in early clinical trials of high-dose immunosuppressive therapy (HDIT)/HCT continued to lose neurologic function, consistent with the noninflammatory factors and progressive neurodegeneration associated with these stages of the disease. However in 15 years of monitoring, patients with active central nervous system inflammation before HDIT/HCT enjoyed significantly better outcome compared with those without active inflammation before HDIT/HCT, indicating that the therapy may achieve greater success if received in earlier MS inflammatory stages.

The researchers hypothesize that inflammation control applied in the earlier RRMS stages may confer prolonged remission and even potential for reversal of neurologic dysfunction. To that end, the Hematopoietic Cell Transplantation for Relapsing-Remitting Multiple Sclerosis (HALT-MS) study is investigating the effectiveness of early HDIT/HCT intervention in RRMS patients with breakthrough disease. However, they also note that because HDIT/HCT is also associated with significant risks, the treatment would have to be proved highly effective in order for it to be considered a reasonable alternative to non-HCT MS therapies. The report observes that in the HALT-MS study of subjects with RRMS, event-free survival in with was estimated to be 82.8% at two years and 78.4% at three years subsequent to the HCT treatment. The study coauthors caution that comparisons across clinical trials can be problematic, but note that in another study of patients who received natalizumab treatment or a placebo, only 37% and 7%, respectively, remained free from both clinical and MRI-documented disease activity after two years.

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Moreover, in the HALT-MS study, improvement was observed from baseline in the MSFC score and neurologic function (EDSS score) in contrast to observations for interferon therapy, and since continued breakthrough disease activity associated with currently approved non-HCT treatments indicates poor prognosis, and may justify the risks associated with administration of a more intensive approach that may improve long-term outcome, and consequently represent a potential therapeutic option particularly for patients with MS in whom conventional immunotherapy fails, as well as for other severe immune-mediated diseases of the central nervous system.

The scientists report that most adverse events (AEs) observed so far in the HALT-MS study have been hematologic or gastrointestinal, and are both anticipated and reversible after high-dose immunochemotherapy, and that no early treatment-related mortality or organ failure has been observed.

The researchers say longer follow-up will be needed to determine durability of the response, and careful comparison of results of this and other ongoing investigations will be needed in order to identify the most beneficial approaches for high-dose immunosuppressive therapies for MS, and plan more clinical studies.

In a related JAMA Neurology editorial entitled “Moving Targets for Stem Cell Transplantation for Patients with MS” (JAMA Neurology, published online December 29, 2014; DOI: 10.1001/jamaneurol.2014.3831), Dr. M. Mateo Paz Soldán, MD, PhD of the University of Utah Department of Neurology at Salt Lake City, brianweinshenkerand Dr. Brian G. Weinshenker, MD of the Mayo Clinic Department of Neurology at Rochester, Minnesota, observe that stem cell transplant therapy for patients with multiple sclerosis has been of great interest to scientists, physicians, and patients, the latter whom they say frequently enquire about the status of stem cell therapy during annual MS check-up visits, usually as a parting query. The authors note that most patients have a hazy concept of what stem cell therapy is, partly derived from a popular perception that stem cell therapy is regenerative, even though actual therapies that promote regeneration of lost myelin and axons are almost completely absent from current treatments administered by neurologists.

Drs. Soldán and Weinshenker note that the JAMA Neurology study as well as another phase 2 single-arm study leave little doubt that high-dose immunotherapy is able to substantially suppress inflammatory disease activity in patients with MS who have active disease in the short term and provide some evidence of long-term MS suppression. Additionally, lessons have been learned about how to manage and reduce treatment-related morbidity and mortality, although deaths have occurred and aggressive therapeutic regimens have occasionally resulted in development of lymphomas associated with Epstein-Barr virus, and the jury is still out regarding the appropriateness and indication of HCT for MS, the authors conclude.

JAMA Neurology

Image Credits:
Colorado Blood Cancer Institute
Mayo Clinic

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  1. Bill Feeney says:

    I am very interested in having this procedure done. I am a 63 YO male who has a severe neurological disorder (it is similar to MS). I live in the Tampa area. I understand that there is a practice down near Miami (Delray Beach?) that does this procedure. I want to get in touch with them. I would be VERY appreciative if you can tell me where I can have this procedure done.

    • Carol says:

      Hi Bill, Stem Cell treatment and HSCT are not the same thing, to HALT the progression the HSCT must include chemo. I am an HSCT (non myeloablative) veteran, I had HSCT in Feb 14.. not on this study though. It is available in many countries some of are German, Italy, Russia, Australia, England, Israel, Mexico, Singapore and many more countries. Dr Burt at Northwestern in Chicago is also a leader in this field if you are in the states. I now have no active or new lesion in the last 15 months. Search FB for the Hemotopoetic Stem Cell Transplant page to research where you can have this autoglous HSCT transplant to HALT progression of MS.

  2. Tammy says:

    How can I receive this treatment for my RRMS? I would be very interested in resetting my Immune System and remain in remission.

  3. B. Vaishnavi says:

    Housewife suffering from MS since 2008 had been many hospitals and taken medicines no improvement. un-able to walk and giddiness always. Is there cure and shall I become normal human.what is the best remedy to recover normal.

  4. SARA McCLOSKEY says:

    I AM 60 YR.OLD WT.FEMALE DX 2010
    HELP ME!!!

    • Carol says:

      Hi Sara I am an HSCT (non myeloablative) veteran, I had HSCT in Feb 14.. not on this study though. It is available in many countries some of are German, Italy, Russia, Australia, England, Israel, Mexico, Singapore and many more countries. Dr Burt at Northwestern in Chicago is also a leader in this field if you are in the states. I now have no active or new lesion in the last 15 months. Search FB for the Hemotopoetic Stem Cell Transplant page to research where you can have this autoglous HSCT transplant to HALT progression of MS.

    • Lois Penler says:

      I am 61 was fx six years ago. Was a working nurse. Now walk with a walker, poorly but at least able to walk very short distances. Hope things change for you. Whoops I put fx meant dx

  5. Martin Matko says:

    Multiple Sclerosis is an unproven autoimmune theory, many feel and know MS is a mechanical issue. CCSVI is a treatable recognized medical condition scientifically established in 42 neurological afflictions and MS !

  6. Jason Guarino says:

    Hi My name is Jason Guarino, I am 41 Years old, I have Remitting/Relapsing Multiple Sclerosis for over 12 years now. Been on all Medications. Currently on Tysabri. But I am experiencing so many other problems due to the MS , My life is so difficult.. I used to work as a Director of Information Technology and went through intense Schooling and Training. I used to be able to enjoy life.. Now all I do is get Collection agency calls, Eviction Notices & yesterday My Gas got turned off, Now I can’t cook , Take a Shower , cause the cold water makes me go into shock. I can’t wash my clothes cause the dryer is gas … I am always broke , I can’t even enjoy life. Now on my second Marriage and it’s getting even harder for me and my wife because of all these problems… Looks like I’m heading for another Divorce… Then I’ll be all alone.
    I won’t have anyone. I fear everyday that my wife will cheat on me cause I cannot even get an erection to have sex, due to MS , This just isn’t fair… I ask myself everyday, Why don’t I go to sleep and not wake up.. The pain and suffering that I go through just isn’t worth me being here anymore… I can’t find anyone to help me… My wife complains that I have so many insecurities and she’s fed up with the way I treat her… I don’t mean it, I’m just so unhappy… Isn’t their someone out there who can help me ?? PLEASE My email is
    If anyone can help me ….. I’m begging , I don’t want to die or loose my wife, She’s my best friend , my Life , My everything… We used to be so close ….

    • Kimn Nierman says:

      Jason, I am not a doctor but I was able to get my life back. I cut ALL- dairy, sugar and gluten. And I mean none. No “non-gluten” label stuff so nothing with rice, corn and any kind of grain. And I started eating 9 cups of dark green, leafy vegitable and tons of fresh veggies preferably organic but that is expensive. Got 30 minutes of sunshine (you need vitamin D which is cheap, sun). I put essential oils on my feet and neck but that can also be expensive. I drink fresh clear water. NO SODA, period. If you can walk get exercise. I take a lot of vitamins and try to clean your digestive system. I am 59 and have lost a lot of everything I had. I lost a six figure income and business because he stole my business. He sued my wife and I as well as my three kids. It has been a real trial for us. We settled because I just couldn’t handle the stress and lost a lot. I have not had an income for about a year and I am trying to start a new business. It is easy to just look at what you don’t have especially with this disease. DO NOT GIVE IN. THINGS WILL GET BETTER. Go to the internet and search Dr. Terri Wahl and she was in a wheel chair for 7 years and now she ran a marathon. Again, I am not a Doctor (almost worthless sometimes) I just know what worked for me. I accepted Christ as my Lord and I pray a lot and the Lord helped me to research and fought. Please fight. What you eat does matter. When I was not eating right I just got sicker and sicker and most of my body was fading away. From my neck all the way to my toes. My tongue was numb. You can do it but you have to fight.
      God bless!

  7. Lin says:

    I have had 2 Drs say I have MS and 2 that will not confirm it. I have at last count 5 lesions on my brain. I am losing cognizance and mobility. Some days I am good but most days it is like looking at life through water. I have no idea who to turn to for help. The MS Center in Baltimore did nothing and never called me back. I am losing my life. I am a horsetrainer and now I cannot function.Oh crap I just lost function in my arm and spilled coffee all over me.
    Someone please direct me. Thanks

  8. Shirley says:

    Funny how these articles come out on a very regular basis … In hopes to shut us up. Notice the writer of this article is not responding to any of you. Just us trying to help each other. What a shame that there is nothing that we can do that doesn’t involve mortgaging our homes in desperation that this treatment works.

    Wake up people … these MS Societies and their researchers are not interested in helping us but, in living off of us for their Quality of Life. There is no money in even giving us relief in the meantime.

  9. M.Sedita says:

    HSCT does work and there are many facilities world wide. The JAMA report from 21Jan 2015 gives the 5 year results with 87% progression free and 80% quality of life improvement. It’s not a walk in the park, it is serious but manageable treatment that gives a person better odds than doing nothing. It does involve chemo, in fact, it MUST involve chemo or it will not destroy the underlining bad cells. I was the care taker for my spouse and stayed with him while he went through this procedure.
    It is available in many places and there are many veterans willing to share their stories. Here is a great resource:

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