At last, interim results of an international study show haematopoietic stem cell transplantation (HSCT) is an effective and safe treatment. It outperforms disease modifying therapies (DMTs) for people with highly-active relapsing MS.
Sadly, though, that’s as far as the study goes. Despite there being numerous people with progressive MS who say they have benefited from HSCT, the study only talks about the relapsing form of the disease.
However, notwithstanding its shortcoming, it is great that the first large scale, international. randomised study into HSCT has shown such promising results.
The trial, known as MIST, showed HSCT can stabilise relapsing MS and improve disability in people who were still experiencing relapses while taking DMTs.
Researchers Professor Basil Sharrack and Professor John Snowden, led the UK arm of the trial at Sheffield’s Royal Hallamshire Hospital. They describe the findings as ‘hugely encouraging’.
Sharrack said: “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.”
While that is good news, it is generally accepted that the recovery period for people after undergoing HSCT is two years. And that recovery period has many ups and downs, often referred to as a rollercoaster ride.
The trial involved 110 people who’d had at least two relapses in the past year while on a DMT. These were divided in two, with half having HSCT while the other half continued to take the DMTs recommended by their neurologist.
HSCT outstrips DMTs
The study analysed the results, in particular the number of relapses participants had and the progression of their disability. Just one of those who had HSCT suffered a relapse, which is tremendous when compared to people who stayed on DMTs. Of that group, 39 had relapses.
Under the rules of the study, anyone on DMTs who continued to have relapses could switch to the HSCT arm of the trial. Most of the 39 who had relapses, a total of 30 people, did switch. Encouragingly, their condition improved.
The study’s results have not yet been published. They were presented at the annual meeting of the European Society for Blood and Marrow Transplantation (EBMT). Next, they will need to be published in a peer-reviewed journal.
The UK’s MS Society director of research, Dr Susan Kohlhaas, cautiously praised the results. She said still more information is needed about HSCT’s relationship with DMTs:
On the society’s website, Kohlhaas said: “The MIST results are important and show this area needs further research. While HSCT appears to be effective for some people with MS, it remains a high-risk treatment that won’t be right for everyone. We now need to know how HSCT compares to existing, less aggressive, MS treatment options.”
Making sure HSCT is accessible for people who are eligible should also be prioritized, Susan said.
Promisingly, she continued: “HSCT will soon be recognised as an established treatment in England. And when that happens our priority will be making sure those who could benefit can actually get it. We’ve seen life-changing results for some people and having that opportunity can’t depend on your postcode.”
Let’s hope that, when the study’s final results are published, HSCT will be approved as a treatment for MS. That, of course, will be up to the FDA and other regulatory bodies worldwide.
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50shadesofsun.com is the personal website of Ian Franks, a freelance medical writer and editor for various health information sites. He enjoyed a successful career as a journalist, from reporter to editor in the print media. He gained a Journalist of the Year award in his native UK. Ian received a diagnosis of MS in 2002 and now lives in the south of Spain. He uses a wheelchair and advocates on mobility and accessibility issues.
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Note: Health-related information available on 50shadesofsun website is for your general knowledge only. It is not a substitute for medical advice or treatment for specific medical conditions. I am not a doctor and cannot and do not give you medical advice. You should seek prompt medical care for any specific health issues. Also, consult a doctor before starting a new diet or exercise programme. Any opinions expressed are purely my own unless otherwise stated.
2 thoughts on “HSCT ‘safe and effective’ for people with relapsing MS, international study”
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