56 What does the research say?

I thought I’d share with you some of the research into HSCT that I have been reading.  There are several studies showing the effectiveness and safety of HSCT and many thoughtful accounts from individuals who have accessed the treatment. 

Before talking further about the successes, I want to make it clear that I am fully aware that HSCT does not work for everyone.  For somewhere around 20% of patients, HSCT does not halt progression of the disease and there seems to be no way of predicting who it won’t be successful foror working out why.  It’s a gamble everyone pursuing HSCT must take. 

To put this into perspective, it’s worth noting the success rate of currently available DMD (Disease Modifying Drugs).  As a first line treatment, most MSers are offered drugs with a quoted effectiveness rate of just 30%.  If these fail to prevent relapses, there are various options, eventually leading to drugs with about 60% effectiveness.  These drugs must be taken long term and have various effects on the body.  The most effective drugs have the most severe side effects and higher risk of contributing to other medical problems.  Many neurologists do not believe that in the long term accumulation of disability is reduced by taking DMDs and the truth is, there isn’t universal agreement on their safety, effectiveness, long term risks and benefits. 

So, conventional treatment is a gamble too. 

In April of this year, an interim report on an international trial known as the MIST trial was published.  This relates to an ongoing study of MS patients treated with either HSCT or an alternative treatment in Chicago, Sheffield, Uppsala (Sweden) and Sao Paulo (Brazil).

Key Findings:

• 110 people with active relapsing remitting MS despite been treated with disease modifying drugs were randomised to receive either the best available drug treatment or AHSCT.
• During the treatment follow up period, disability improved significantly after AHSCT.
• The EDSS score of patients receiving the transplantation improved from an average of 3.5 to 2.4, which is unprecedented in MS treatment trials. This contrasted significantly with those receiving standard drug treatment whose EDSS scores declined from an average score of 3.3 to 3.9.
• Within a year of joining the trial, only one patient in the transplant arm of the trial suffered a relapse compared to 39 relapses observed in the drug treatment arm.
• With a mean follow up of 3 years, treatment failure measured by disability progression was 6% in the HSCT arm and 60% in drug treatment arm.
• 30 people who were originally randomly allocated into the drug treatment arm of the trial were moved over to the transplant arm during the trial period after they had a decline in their EDSS scores. After AHSCT their scores improved from 5.2 to 2.6.
• No person in the AHSCT arm suffered any significant side effects.

MIST Trial Findings

Neurology April 9th 2018

“Non-myeloablative hematopoietic stem cell transplantation (HSCT) is superior to disease modifying drug (DMD) treatment in highly active Relapsing Remitting Multiple Sclerosis (RRMS): interim results of the Multiple Sclerosis International Stem cell Transplant (MIST) Randomized Trial”

I found this information interesting “Five control patients were withdrawn after soliciting transplants at other centers.”  I imagine if you are a patient on the least successful side of a treatment trial, you are likely to want the successful treatment as soon as you become aware of it! 

This week there has been a report published from a separate Australian HSCT trial, where similar positive outcomes were revealed. 

Australian AHSCT Trial Results Released

Prospective phase II clinical trial of autologous haematopoietic stem cell transplant for treatment refractory multiple sclerosis

“Conclusions The EFS in our MS cohort is significantly greater than other high-efficacy immunosuppressive therapies and similar to other AHSCT studies despite a more heavily pretreated cohort.”

Research into HSCT is in its infancy and this is the reason that it is not yet being embraced in many countries as a mainstream treatment.  There are many medical professionals who are reluctant to recommend HSCT at the moment, but who feel it could well be the treatment of the future. 

This is great for MS patients going forward …but I don’t have time to wait. 

EDSS – Expanded Disability Status Scale (a measure of disability due to MS)

AHSCT – Autologous Haematopoietic Stem Cell Transplant (Autologous = Transplant using the individuals’s own tissues)

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