Results from two trials of an aggressive form of stem cell therapy indicate the treatment may have long-term benefits.
A Canadian study reported in the Lancet involved 24 people aged between 18 and 50. All had highly active MS (12 had relapsing remitting MS, 12 with secondary progressive MS) that hadn't been controlled with disease modifying drugs.
Treatment involved harvesting stem cells from participants then destroying their immune system with chemotherapy drugs. Their stem cells were then returned to them to restore the immune system to stop it attacking the body.
Participants received the treatment between 2001 and 2009. The average follow up period ranged from four years to twelve and half.
The main aim of the study was to monitor 'activity-free survival' after three years. This was defined as no relapses, no new brain lesions seen on MRI scans and no progression in EDSS scores. Almost seven out of ten participants achieved this.
During the follow up period, none of the participants had a relapse and eight showed improvements in their EDSS score seven and a half years after treatment.
The treatment is not without risks and one of the trial participants died as a result of the chemotherapy.
Dr Mark Freedman, who led the trial, pointed out that 24 people was a very small sample size and that further research would be necessary to reduce the risks of treatment and to better understand which people with MS would benefit from the approach.
The results reflect another trial that was reported at the CMSC (Consortium of MS Centers) conference. This study in Seattle also involved 24 people receiving high-dose immunosuppressive drugs followed by a stem cell transplant. After five years, seven out of ten showed no relapses, new lesions or increasing EDSS scores. Three people died in this study but all for reasons unrelated to their treatment.
Amy Bowen, MS Trust Director of Service Development, said,
"These results are extremely interesting and encouraging, though sadly highlight just how risky stem-cell treatment could be.
"Stem cell therapy holds great promise for people living with MS but it’s a long way from being a routine treatment. We still need more clinical trials to understand who is most likely to benefit from treatment, to develop safer treatment procedures and understand what the long-term effects of treatment might be. We also need to understand the most effective ways to provide stem cell therapy safely to the people most likely to benefit.
"This is why the MS Trust is working in conjunction with a pan-London group of experts in MS, and in stem cell treatment for other conditions, to conduct the first ever audit of a real-world UK MS stem cell service run outside of a clinical trial. From this audit we hope to understand how people with MS can be assessed and selected for stem cell treatment on the NHS, and what is needed to develop safe, high quality services for the future."
At the moment stem cell therapy is only available on the NHS in very limited circumstances, and the criteria for eligibility varies.