#CMSC16 – RRMS Patients’ Five-Year Clinical Outcome Improved Through Combined Immunosuppresive Therapy, Cell Transplantation

#CMSC16 – RRMS Patients’ Five-Year Clinical Outcome Improved Through Combined Immunosuppresive Therapy, Cell Transplantation

Results from a Phase 2 clinical trial showed that five-year clinical outcomes of patients with highly active relapsing-remitting multiple sclerosis (RRMS) are significantly improved when patients are treated with high-dose immunosuppressive therapy combined with autologous hematopoietic cell transplantation.

The study, “Five-Year Outcomes of Halt-MS: High-
Dose Immunosuppressive Therapy and Autologous Hematopoietic Cell Transplantation for Severe Relapsing-Remitting Multiple Sclerosis,” was recently presented at the June 1-4 2016 Annual Meeting of the Consortium of Multiple Sclerosis Centers (CMSC).

Researchers investigated the outcome of a combined treatment based on high-dose immunosuppressive therapy (including BCNU, etoposide, ara-C, melphalan, and antithymocyte globulin) and autologous hematopoietic cell transplantation in a cohort of patients with highly active RRMS who failed to respond conventional therapy. The team assessed the rate of sustained disease remission.

In total, 24 patients received high-dose immunosuppressive therapy/autologous hematopoietic cell transplantation. Patients were followed for a median period of 62 months.

In the first three years after high-dose immunosuppressive therapy, the team registered adverse events (with 121 grade 3, and 93 grade 4), in its majority of hematological and gastrointestinal nature. Between the third and fifth years after the transplant, there were 15 adverse events of grade 3, and none of grade 4.

In total, three deaths occurred during the study – one patient experienced progressive loss of neurological function (death occurred at month 32); two other patients died post-transplant (after three years). But importantly, none of the registered deaths were related to the treatment.

At five years, the probability of event-free survival was 69.2 percent; progression-free and relapse-free survival were 90.9 percent and 86.3 percent, respectively; and the probability of freedom from disease activity detected by brain magnetic resonance imaging (MRI) was 88.2 percent. Notably, MS disease burden (determined by T2 lesion volume measured through MRI) showed a significant reduction by six months (when compared to patients values at baseline), and was sustained for five years.

In conclusion, the results from the Phase 2 clinical trial with highly active RRMS patients showed that a treatment based on high-dose immunosuppressive therapy/autologous hematopoietic cell transplantation can induce disease remission. Most importantly, the remission was sustained for five years without the need for maintenance therapy. Additionally, treatment-related adverse events were consistent and, according to the transplant procedure, without unexpected complications.

“High-dose immunosuppressive therapy was well-tolerated with few serious early complications,” said Dr. James Bowen, the study’s lead researcher who presented the results at CMSC 2016. “High-dose immunosuppressive therapy was highly effective for inducing sustained remission of highly active RRMS through year 5.”


  1. Steve says:

    So there has been studies going on for almost a decade on this process but they never seem to make it past a phase II study. Why? Supposedly the treatment was used in ’07 in Chicago but we are still no closer to mainstreaming this procedure. I have read where they say they want to understand how the process works. I take tecfidera where they say the mechanism of treatment in not completely understood but it is still FDA approved. Come on, peoples lives and quality of life are going downhill as we speak.

    • Eddie Nash says:

      Things are moving along more rapidly now that the social media(Face Book)has gotten into spreading the word. Many more people are applying for all the different autoimmune conditions. 245 are being followed for Ms as of 7-2015. Dr Richard Burt who is the administrator of this trial which is also being coordinated by him in Brazil, the UK and Sweden, is indeed making progress. Dr Burt has to work carefully to make sure everything is done by the book. Drug companies are not supportive of this coming to the mainstream. The medical community is not supportive because it will cut into both those very powerful lobbying entities MONEY! The trial is expected to be completed for RRMS in 2020. Will it go mainstream? Will the resistance of the drug companies and medical community be overcome? Only if the public rises up and demands it to be so! Become active in the activist communities pushing for HSCT to become available for RRMS and the 20 other autoimmune conditions already in Dr Burts trials. #HSCT.

      • Steve says:

        Don’t get me wrong, I follow the HSCT Facebook page and have seen your name on it multiple times. It is just frustrating that there is an abundance of medical data pertaining to the procedure to include multiple years of follow up, specifically by Dr. Burt, and that the best estimation of the procedure going before the FDA is at least 2020 if not a couple years down the road. I have been diagnosed for a little over a year and I went from a minor back spasm when I ran to almost needing a cane. I would love to apply for the procedure but I know my insurance will not cover it and I do not have the means for fund raising, if even accepted into the trial. How much more data is needed to push for FDA approval? it’s just frustration and fear setting in.

        • Eddie Nash says:

          Do not automatically “KNOW” that your insurance will “NOT PAY!” My insurance policy spelled out IN WRITING. “Does NOT PAY FOR TRIALS. Yet we applied and within 10 days we were told YES! Happy days believe me. DO not talk to your insurance company directly. Let Dr Burts trained staff(who knows the proper “CODES” and things to say and not to say) do their best to get you approved. As you can I have lived and breathed this subject and do all I can to educate and assist others as I PAY IT FORWARD! God be with you Steve!

          • Inge Freeth says:

            dear eddie nash and steve, readings your comments with interest. gives me hope to continue believing their will come a day that the treatment is for more people with this horrible disease MS(with one you suffer ,does not matter).keeep on posting and making us known by the world.Greetings Inge.

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