Diagram of stem cell treatment

New study shows long term promise of stem cell therapy for MS

New research has shown aggressive autologous haematopoietic stem cell transplantation (AHSCT) can halt relapses for people with highly active, relapsing MS.

This trial involved 24 people and used high intensity chemotherapy prior to stem cell transplantation, with the aim of completely eliminating and then resetting the immune system.

High risk treatment

Researchers found the treatment effectively prevented MS-related immune attacks. No-one taking part in the trial had a relapse during the follow up period, which ranged from four to 13 years.

In addition, 70% of the people in the trial did not experience any worsening of disability after the treatment and 40% actually experienced an improvement in disability during the follow-up period. Worsening of disability was assessed using the Expanded Disability Status Scale (EDSS).

The aggressive nature of the procedure did however carry substantial risks. One person taking part in the study died from liver failure after undergoing AHSCT and another required intensive care as a result of the aggressive chemotherapy. A quarter of participants developed infections and all but one had a toxic response to treatment.

Larger trials needed

The trial reported sustained benefits for people with highly active, relapsing MS. However Dr Freedman, one of the researchers involved in the trial, highlighted the need to interpret the results with caution: “The sample size of 24 patients is very small, and no control group was used for comparison with the treatment group. Larger clinical trials will be important to confirm these results.

“Since this is an aggressive treatment, the potential benefits should be weighed against the risks of serious complications associated with AHSCT, and this treatment should only be offered in specialist centres experienced both in MS treatment and stem cell therapy, or as part of a clinical trial.

“Future research will be directed at reducing the risks of this treatment as well as understanding which patients would best benefit from the treatment.”

Treatment sweet spot

Professor John Snowden, who works on AHSCT at Sheffield Teaching Hospitals, said:

“There are different ways to deliver AHSCT, which vary in intensity and how the stem cells are manipulated. We need to balance how successful a treatment can be with its risks. Whilst this study protocol was remarkable in achieving such profound and sustained responses in patients with aggressive MS, its toxicity appears greater than with some other AHSCT techniques.

"More clinical trials are needed to determine the ‘sweet spot’ - where the AHSCT technique is able to achieve a long term response in the majority of patients with an acceptable safety profile.”

Balancing risks

Dr Emma Gray, our Head of Clinical Trials, said:

“This type of stem cell transplantation is a rapidly evolving area of MS research that holds a lot of promise for people with certain types of MS.

“In this latest trial patients were monitored post- treatment for a longer period than previous studies, providing valuable information about the long-term safety and effectiveness of HSCT as well as who might benefit.

“This treatment does offer hope, but it’s also an aggressive procedure with substantial risks and requires specialist aftercare. If anyone is considering AHSCT we’d recommend they speak to their neurologist.”