#ECTRIMS2018 – Stem Cell Transplant Effectively Lessens Disability in Patients with Aggressive MS, Study Finds

#ECTRIMS2018 – Stem Cell Transplant Effectively Lessens Disability in Patients with Aggressive MS, Study Finds

Treatment with autologous hematopoietic stem cell transplant (aHSCT) led to a safe and rapid lessening of disability and no clinical relapses in patients with aggressive multiple sclerosis (MS), according to a new study.

The research, “The use of autologous hematopoietic stem cell transplantation as a first line disease modifying therapy in patients with ‘aggressive’ multiple sclerosis,” was presented at the recent 34th congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), which concluded in Berlin, Germany last week.

The data was presented by Joyutpal Das from the department of neurology, Sheffield Teaching Hospitals NHS Foundation Trust, United Kingdom.

aHSCT is a procedure that involves collecting stem cells from a patient’s bone marrow, peripheral blood or umbilical cord blood, followed by partial or total impairment of the immune system with a conditioning regimen (called immunoablation), and then infusing the stem cells back into the patient to reconstitute and reactivate the immune system.

This method has been very effective in patients with highly active relapsing MS, who had failed to respond to standard disease modifying therapies (DMTs). The European Society for Blood and Marrow Transplantation guidelines recommend its use as a first-line therapy in patients with aggressive MS.

Aiming to address the procedure’s safety and effectiveness as a first-line strategy among these patients, the study focused on 20 with aggressive MS who received aHSCT at five centers — seven patients in Sheffield, U.K., seven in Uppsala, Sweden, four in Ottawa, Canada, one in Florence, Italy, and one in Chicago, Illinois.

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No patient received any standard DMT for more than three months before aHSCT. Patients were treated with either BEAM (carmustine, etoposide, cytarabine, melphalan) chemotherapy plus antithymocyte globulin (ATG) to lessen transplant rejection, cyclophosphamide with ATG, or a triple combination of cyclophosphamide, ATG and busulphan were used as conditioning regimens.

Patients had a mean age at diagnosis of 28 years (range 19-52 years), and a median pre-aHSCT Expanded Disability Status Scale (EDSS) score of 6.5, which means requiring two walking aids to walk about 20 meters (almost 22 yards) without resting. (The higher the EDSS score, the greater the patient’s disability.) Median follow-up duration was 29.5 months.

Patients showed a statistically significant median EDSS score improvement (reduction) of 2.5 between pre-aHSCT and the last follow-up visit. The median EDSS score at the last visit was 2.0, which indicates a minimal disability level. Das also noted that “no patients had post-transplant disability progression.”

No patients experienced a clinical relapse following aHSCT.

New brain/spinal cord lesions were found in three patients during the first follow-up using magnetic resonance imaging (MRI) scan, but no further lesions were found in subsequent scans.

Treatment with aHSCT led to routine toxicities, but did not cause death.

To further support the successful approach with aHSCT, Das reported that among the patients treated “one female naturally conceived and gave birth to a healthy baby,” and “one male patient fathered a healthy baby.”

Overall, the team concluded: “aHSCT was safe and highly effective in inducing rapid and sustained remission in this cohort and was associated with a significant improvement of patients’ level of disability,” the researchers wrote. “This demonstrates the potential role of aHSCT as first line therapy in ‘aggressive’ MS,” they added.

Of note, these results build on data presented in April at the 2018 Annual Meeting of the American Academy of Neurology.

José is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has studied Biochemistry also at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario, in London, Ontario. His work ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.
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José is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has studied Biochemistry also at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario, in London, Ontario. His work ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.
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27 comments

  1. kathy girouard says:

    I would like more info on this procedure. You mention a location in Chicago. Are more procedures being done and how can I contact them. Phone #586-294-8371

    • Ally Grant says:

      Hi Kathy 🙂 this procedure was being done by Dr Richard Burt at Northwestern Hospital in Chicago so you would need to contact them to get more information regarding criteria for the AHSCT trials there.

    • Helena says:

      Hello everyone. I have MS and I had my stem cell transplant in July 20, 2018. The stem cell was done by placenta. I feel great. My doctor is Douglas Spiel TEL: 732-548-2000

      • Susan says:

        Just wondering your disability level before and after since I have read about HSCT in only RRMS- I have also read it is considered usually used only if DM meds don’t work.

  2. Getty Bailey says:

    Again, only relapsing remitting! It’s as if those of us with primary progressive don’t exist. Aggressive MS, I would say that mine is very progressive! I suppose because we now have Ocrevus we don’t need something that might help us walk again.

  3. Scott says:

    I have PPMS and had aHSCT abroad at 52. It’s a mystery as to why they’ve been doing this for twenty years with amazing success and still can’t figure out if it good line of treatment? Considering time is of the essence this should be a serious consideration for people like Debbie.

  4. Larry williams says:

    I thought this procedure was not allowed in the US and that prior treatments had resulted in deaths or a long recovery period, plus the costs in $ in extremely high! Why stop at 58? What is wrong with someone in their 70’s?

    • Ally Grant says:

      Dr Richard Burt has been doing trials at Northwestern Hospital Chicago for a few years now and most people had the cost covered by their US insurance companies. Deaths and long recovery periods are also now a thing of by the past. I was mainly tired/nauseous during the chemotherapy stage of my own treatment and it took me about 3 months to recover sufficiently enough to commence working with a physiotherapist.3 years later and here I am and I’m just fine🙂 There are many groups on the internet /social media where you can find more information about HSCT .

  5. Scott says:

    There more than 1000 people who have done HSCT abroad. There are many HSCT Facebook forums with many hundreds of testimonies for all forms of MS. I could go on forever about an opportunity that was given to me that I could not get here in the states because there’s no incentive. I can only say good things about the professionalism and my outcome as of today. If you feel that you have no other choice HSCT is real medicine an because of self-preservation, anyone with multiple sclerosis has to give HSCT consideration if the future is grim as with me.

  6. Helene says:

    This treatment can help all forms of MS. I had stem cells deployed into my spinal cord two years ago. I have spms and it helped me. My fatigue lessened, simple things like putting on my pants or getting of the toilet were a challenge. After the stem cells I could do these things and still can. Stems cells help MS, I believe they do not research stem cells for MS because it would hurt the pharmaceutical companies dollar. I had to pay for the treatment myself.

  7. Ally Grant says:

    I had AHSCT in 2015 and though it cost a pretty penny to travel overseas because the treatment is not yet readily available in my home country (Australia) it was well worth every cent ! I went from walking with a cane and being exhausted to the point of being bedridden most days, 3 years later and you would be hard pressed to see that I’d ever had MS … This is an interview that I did with our local TV station upon my return from having treatment.
    https://www.facebook.com/7NewsWideBay/videos/1675475742731670/

  8. Jill says:

    I had HSCT in Russia June of 2017. I am an American citizen who was denied access to treatment by Dr. Burt in Chicago because he said that for me the risk outweighed the benefit. My EDSS was 4.5, I had a light lesion load with no active lesions, but was continuing to progress. They still considered my MS relapsing/remitting. Thank god there was an alternative. Having HSCT has changed my life in so many positive ways. My EDSS score is 2 now.
    I understand that this article is reporting on research. But the fact that we have only been studying “aggressive MS” patients is a disservice to all patients with MS. Most MS patients can benefit from HSCT. The decision of the risks outweighing the benefits, should be left to the patient. We have been providing similar treatments for cancer patients for a long time. It’s not new treatment. It’s only the diseases being treated that is new. We KNOW it works.
    HSCT NEEDS to become a mainstream option.

  9. Helena says:

    Hello everyone. I have MS and I had my stem cell transplant in July 20, 2018. The stem cell was done by placenta. I feel great. My doctor is Douglas Spiel TEL: 732-548-2000

    • Brian Melton says:

      If there isn’t any chemo the disease will continue to progress. You have to put out the fire first and the only way is HSCT. Be your own advocate and research.

    • Cathy says:

      Hi, I was dia. In 95, and moved to nm. No one would help me here, now it’s all down my spine, along with brain . I need help. Could you tell me where in colorado ….

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