Blood Stem Cell Transplants Improve RRMS Patients’ Disability, Phase 3 Trial Shows

Blood Stem Cell Transplants Improve RRMS Patients’ Disability, Phase 3 Trial Shows

Blood stem cell transplants lead to significant improvements in relapsing-remitting multiple sclerosis patients’ disability, a Phase 3 clinical trial shows.

The 110 patients who took part in the MIST study (NCT00273364) were having relapses after receiving standard therapies such as beta interferon, Copaxone (glatiramer acetate), Novantrone (mitoxantrone), Tysabri (natalizumab), Gilenya (fingolimod), and Tecfidera (dimethyl fumarate).

Half the participants were randomly assigned to continue with standard treatment. The other half received blood stem cells transplants. Doctors call the transplant procedure autologous haematopoietic stem cell transplantation, or AHSCT.

AHSCT is an intensive therapy that rebuilds a patient’s immune system. The first step is  harvesting and storing a patient’s blood stem cells. The next is using chemotherapy to kill any immune cells in the stem cells. And the final step is returning the stem cells to patients to rebuild their immune system.

The MIST trial’s primary goal was to see if the transplants could do a better job than standard treatment at stopping the progression of RRMS. The main yardstick of progression was patients’ scores on the Expanded Disability Status Scale, or EDSS.

A year after the transplants, the average EDSS score of treated patients had improved significantly. A decline in an EDSS score indicates less disability, and treated patients’ average score fell from 3.5 to 2.4. This contrasted with an average increase in the score of those on a standard therapy from 3.3 to 3.9.

Only one patient in the transplant group experienced a relapse during the year, compared with 39 in the standard therapy group.

After three years, all but 6 percent of the transplant group had shown an improvement in disability progression, versus 60 percent in the standard therapy group.

Researchers shifted 30 patients who had initially been receiving standard therapy to a transplant after their disability worsened. This led to their average EDSS scores improving from 5.2 to 2.6.

In terms of safety, transplant patients showed no signs of significant side effects.

“We are very excited by these hugely encouraging findings, which are the first to assess the long-term effectiveness of AHSCT [transplants] in people with the active relapsing remitting form of the disease in a Phase 3 randomized trial,” Basil Sharrack of Sheffield’s Royal Hallamshire Hospital said in a press release.

“In the study, almost all patients receiving autologous haematopoietic stem cell transplantation showed no signs of their disease being active a year on from having the treatment and, more importantly, their level of disability improved significantly. It is also really important to emphasize that these are still early days, and the patients will be followed for five years,” Sharrack added.

“The initial results of the MiST trial show that this type of stem cell transplant can be delivered with acceptable safety to people with highly active relapsing remitting MS,” said John Snowden, who directs the bone marrow transplants program at Sheffield Teaching Hospitals NHS Foundation Trust.

“It is important to stress that this treatment is unfortunately not suitable for every person with MS,” added Snowden, a co-investigator of the trial. “This type of stem cell transplant targets the inflammatory phase of MS.”


  1. Lary williams says:

    Looking forward to more reports and updates, my wife rid this procedure in the US three years ago, very expensive and zero results, but do not believe they cleaned the stem cells as this test did, which I feel is a key, good luck, stem cells are the answer, just need to ge5 approved for use and how in the world will it get done I;the US, when big pharm cannot get their hands into the action?

  2. Joshua Kalnitsky says:

    I still suffer from that terrible MS, Will a cure ever be found??? Probably not in my life time!!


  3. Jill says:

    AHSCT should be available to all patients with MS! After having been rejected at Northwestern for HSCT because Dr. Burt said the risk outweighed the benefit because, in his opinion, my MS was not bad enough, I had AHSCT in Russia. My EDSS pre transplant was at its worst 4.0. I had a mild lesion load in my brain. The US radiologists said I had no involvement in my spine. But on my MRI done in Russia, there was clear evidence of spinal involvement. I had superior care in Russia! The belief by Dr. Fedorenko and his team is that we need to do AHSCT before their is great impairment. In my past life I was a runner; 1/2 marathons were my go to. 9 months post HSCT and I am running again. Today I ran 3.5 miles. I am beyond ecstatic that my life is mine again! Thanks to AHSCT and the great Drs at Aa Maximov Hospital. So lets think a little further outside the box!

    • yogesh mishra says:

      ahsct is not fda approved but being done in many centers worldwide with excellent results.after good researchwork i ve taken appointment from aa maxmove hospital russia i ve met nd talked to many patients who ve recd this treatment nd leading a disease free life with much less disabilities nd improved quality of medical life.

  4. The stem cell technique described in this article would for sure make this a biologic drug….requiring a USA company or doctor to have an Investigational Drug License and Biologic License Application (IND/BLA) approved with the FDA before doing this to people. Basically no strip mall clinic or chiropractor office selling “stem cells” out of a bottle is doing anything close to what is described in this research study. This is one of the major problems….unethical people will use this study as proof that there are stem cells in peripheral blood (ala Lung Institute) for same day clinical use….and that they can give stem cells IV for anything….which is ok only if the FDA has approved an IND/BLA or a RMAT ….which the FDA has not done for any neurologic disorders in teh outpatient clinical setting to myh knowledge.

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