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MS research update - Promising results from long term studies of stem cell transplants - 17 February 2017

Summary

There continues to be a great deal of interest in stem cell treatments for MS but only limited information has been available about how people do in the longer term following a transplant. Two recently published studies have reported data on levels of disability in people with MS five years after receiving stem cell transplants.

In one study, researchers requested data from 25 centres which had used a range of stem cell treatment methods to treat people with either relapsing or progressive MS between January 1995 and December 2006. Five years after treatment, just under half of the 281 people for whom data was available had not experienced an increase in their disability.

A second study has reported long-term results of stem cell therapy in 24 people with highly active relapsing MS. After five years, about two thirds continued to show no relapses, no progression and no new lesions.

While the results from these two studies are very promising, neither of these two studies included comparative groups receiving either placebo or an alternative treatment so it is not possible to say whether the people in these studies have done better (or worse) than people who did not have stem cell transplants.

Stem cell transplants are not without risks; although treatment regimens, patient selection and after-care have reduced risk, clinical trials since 2001 have still had treatment-related death rates of one or two people in every 100.

Both of these studies acknowledge that to fully assess the risks and benefits of stem cell treatments, understand where they fit into current treatment options for MS and make them more widely available on the NHS, what we really need now is evidence from controlled, randomised clinical trials directly comparing stem cell treatments against other treatments in both relapsing remitting and progressive MS.


Background

Stem cell transplants for people with MS have generated a great deal of interest but only limited information has been available about how people do in the longer term following a transplant. Two recently published studies have reported data on levels of disability in people with MS five years after receiving stem cell transplants.

In one study, researchers analysed data from 281 people with MS who had autologous haematopoietic stem cell transplants (HSCT) between January 1995 and December 2006. Five years after treatment, just under half were reported to be free of progression.

How this study was carried out

The investigators contacted 25 centres spread across 13 countries and asked for data on people with MS, both progressive and relapsing remitting, who had been treated with stem cell transplants between January 1995 and December 2006. People were included only if a minimum set of data had been recorded about them: type of MS, Expanded Disability Scale (EDSS) score at the start, details on the transplantation method used, and at least 1 follow-up appointment after the transplant. Data sets were pooled and analysed for 281 people with both relapsing and progressive types of MS and at different stages of their MS who had received transplants using different treatment methods.

What was found

Those who did the best following autologous HSCT were more likely to be younger, have relapsing remitting MS, have taken fewer disease modifying drugs and have lower levels of disability at the time of treatment. 73% of people with relapsing MS and 33% of people with progressive forms of MS were free of disability progression at 5 years.

Eight deaths (2.8%) occurred within 100 days of transplantation and were considered to have been caused by the treatment.

Analysis of the data did not find any difference in results between the different treatment methods used prior to stem cell transplantation.

Disability was looked at in more detail in a subset of 111 people for whom there was data on disability before as well as after treatment. For people with relapsing MS, EDSS increased an average of 1.4 points in the year before treatment and decreased by 0.76 points in the year after treatment. For people with progressive MS, there was an average increase of 0.73 points in the year before treatment and a decrease of 0.14 EDSS points in the year after treatment.

A second study has reported long-term results of stem cell therapy in 24 people with highly active relapsing MS. After five years, about two thirds continued to show no relapses, no progression and no new lesions.

How this study was carried out

The US-based HALT-MS clinical trial assessed autologous HSCT in 24 people with highly active relapsing remitting MS. The main measure of the study was a combination of no disability progression, no relapses, and no new lesions on MRI.

What was found

After five years, 69% continued to show no relapses, no progression and no new lesions.

All those in the study experienced severe side effects. Most of these happened within the first 30 days after transplant and were linked to low white blood cell counts and infections. No deaths were directly attributed to the treatment, however three patients died during the five years following the treatment. These participants had all experienced worsening of their MS.

What does it mean?

The results from these two studies show that stem cell therapy holds promise for people with MS. Neither of the studies included comparative groups taking placebo or an alternative treatment so it is not possible to say whether the people in these studies have done better (or worse) than people who did not have stem cell transplants.

Stem cell transplants are not without risks; although treatment regimens, patient selection and after-care have reduced risks, clinical trials since 2001 have still had treatment-related death rates of one or two people in every 100 (1.3%), according to analysis by the European Group for Blood and Bone Marrow Transplantation. The majority of deaths were due to infections.

Both of these studies acknowledge that to fully assess the risks and benefits of autologous HSCT, understand where it fits into current treatment options for MS and make it more widely available on the NHS, what is required now is evidence from controlled, randomised clinical trials directly comparing HSCT against other treatments in both relapsing remitting and progressive MS.

Muraro P, et al
Long-term outcomes after autologous hematopoietic stem cell transplantation for multiple sclerosis
JAMA Neurology 2017, Feb 20 [Epub ahead of print]
Abstract
Read the full text of the paper 

Nash RA, et al
High-dose immunosuppressive therapy and autologous HCT for relapsing-remitting MS
Neurology 2017, Feb 1 [Epub ahead of print]
Abstract
Read the full text of the paper 

More about autologous haematopoietic stem cell transplantation (HSCT)

Autologous haematopoietic stem cell transplantation (HSCT) uses high doses of cancer chemotherapy drugs to wipe out harmful cells in your immune system. Your own stem cells are used to “regrow” your immune system so that it no longer attacks myelin or causes inflammation in the brain and spinal cord.

The first part of the process requires taking a number of drugs to stimulate release of stem cells from your bone marrow into the blood stream. Stem cells are collected from your blood and kept frozen while the next stage of the treatment is carried out.

For the next part of the treatment you take cancer chemotherapy drugs which either completely eliminate (myeloablative or high intensity chemotherapy) or partially eliminate (non-myeloablative or low intensity chemotherapy) your bone marrow and immune system. Current treatment methods for people with MS have favoured lower intensity chemotherapy which carry a lower risk of complications and death.

Finally, your immune system is rebuilt using your own haematopoietic stem cells. Haematopoietic stem cells can develop into the different types of cells found in the blood including some cells which are part of the immune system.

Read more about autologous HSCT

Short guide to stem cell therapy

Stem cell therapy: balancing hope and hype - article in Open Door

Research by topic areas...

Assessment tools

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Disease modifying drugs

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Drugs in development

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Physical activity

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Prognosis

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Relapses

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Symptoms and symptom management

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Work

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