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HSCT clinical trials

HSCT research evidence

HSCT is an intense chemotherapy treatment for MS. It aims to stop the damage MS causes by removing harmful immune cells. The person’s own stem cells are then used to regrow the immune system.

If you are interested in taking part in clinical trials please have a look at our Be in a study page.

In Europe, all clinics offering HSCT should have Joint Accreditation Committee ISCT-Europe & EBMT (JACIE) accreditation. You can find out more about JACIE on the JACIE website.

Current HSCT clinical trials

Current clinical trials are looking at where HSCT fits with other disease modifying therapies (DMTs) for MS. This means working out when people could most benefit from it as well as who.

Because of the intense nature of the procedure, all of the trials are open label. This means the people involved in the trial know they were having HSCT.

RAM-MS trial

RAM-MS is an international phase 3 trial comparing HSCT with alemtuzumab (Lemtrada), which is one of the most effective disease modifying therapies (DMTs) currently available.

This follows results from the MIST trial, which showed that HSCT can benefit people whose MS is not being controlled by a range of DMTs. Results are expected in spring 2022.

Read about RAM-MS on the ClinicalTrials.gov website

BEAT-MS trial

BEAT-MS is a phase 3 clinical trial in the US (a site may open in London at a later date). It will compare HSCT to the best available DMT for the participants in the study. The researchers will follow their participants for 6 years and will be due to complete in 2029.

Read more about the BEAT-MS study on the Clinicaltrials.gov website

StarMS trial

StarMS is a planned phase 3 trial comparing HSCT with alemtuzumab (Lemtrada) and ocreliuzmab (Ocrevus). Recruitment is expected to open in 2021. We played a key role in making this trial a reality.

Read about StarMS on the Sheffield University website

Our HSCT research

StarMS add-on study

Current highly effective DMTs can reduce nerve cell death. But the use of chemotherapy in HSCT means it might cause damage to nerve cells. In this add-on study to STAR-MS, researchers will compare neurofilaments (a marker of nerve damage in MS) in people taking alemtuzumab and ocrelizumab and those who have HSCT.

Knowing more about the safety and effectiveness of different treatments will allow people with MS to make more informed choices about their treatment.

How HSCT affects the immune system

We funded researchers to investigate the impact HSCT has on the immune system. Researchers found that HSCT can change the balance of different immune cells in the body.

This gives us a better understanding of the long-term benefits and risks of HSCT for people with MS.

Explore past HSCT research

Randomised controlled trials

Until recently, HSCT studies have generally been small and most did not have a control group. But now large-scale clinical trials are beginning to publish results. 

In randomised controlled trials, people are randomly assigned to have either HSCT or an existing MS treatment. This type of trial gives the most reliable answer to whether a treatment is safe and effective.

MIST trial

MIST was an international phase 3 clinical trial involving doctors at Sheffield Royal Hallamshire Hospital. It involved 110 people with relapsing MS.

Everyone in the study had experienced at least two relapses in the past year despite being on a disease modifying therapy (DMT) like beta interferon and Copaxone. Half underwent HSCT and the other half took licensed DMTs recommended by their neurologists.

The results were published in 2019. Almost all people who had HSCT showed no signs of their condition getting worse a year after the treatment, and some experienced improvement. Only one person who had HSCT suffered a relapse, compared to 39 relapses in people taking DMTs.

Read the results of the trial on the JAMA Network website

The ASTIMS trial

The small-scale ASTIMS trial compared HSCT with mitoxantrone (Novantrone) in 21 people with relapsing or secondary progressive MS. Mitoxantrone is not licensed for MS in the UK.

Everyone taking part had experienced an increase in disability despite taking a conventional disease modifying therapy (DMT) and also showed active lesions on an MRI scan.

The trial used high-intensity (myeloablative) chemotherapy.

Results published in 2015 showed HSCT was more effective at reducing relapses than mitoxantrone. But HSCT was not shown to reduce disability progression in the four year follow-up.

Read about the ASTIMS trial on Neurology.org

Prospective studies with no control group

We can also learn valuable information from studies where everyone has HSCT and there is no 'control group'. In ‘prospective’ studies, people are recruited to join a study that’s been designed to answer a specific question.

Clinica Ruiz, Mexico

In 2019, researchers from Mexico published a study with data on 617 people with all forms of MS treated at their centre. 240 of them were followed up for at least one year after treatment.

The researchers measured the effectiveness of HSCT using a version of the EDSS where people with MS rate themselves. They acknowledge that asking people to rate themselves may have caused bias.

After one year, 188 people reported either an improvement or stabilisation. This ranged from 83% in relapsing MS to 73% in secondary progressive MS. Overall, there was a significant reduction in the EDSS score (indicating an improvement) from an average score of 5.1 to 4.5.

The researchers say their results show a potential trend of better outcomes in relapsing MS.

They've also published a large number of papers discussing HSCT safety at their centre. And in 2021 they published a review describing their experiences providing HSCT to 978 people with MS since 2006.

Read the 2021 review article on frontiers website

Read the full 2019 study on pubmed

HALT-MS trial

The Canadian trial involved 24 people with highly active MS, all of whom had evidence of inflammation and were experiencing relapses.

The trial used one of the most aggressive types of chemotherapy available. Over two thirds of people experienced no relapses or disability progression after completing the procedure.

Read about the HALT-MS trial on ClinicalTrials.gov

The Chicago Experience

This study involved 123 people with relapsing MS and 28 people with secondary progressive MS. All of those taking part had experienced relapses in the year before the study started and were able to walk 100m using a walk stick without rest.

28 people with secondary progressive MS also received low-intensity (non-myeloablative) chemotherapy.

Results showed that people with active relapsing MS who had the condition for less than 10 years were most likely to respond to the treatment. People with secondary progressive MS were unlikely to respond.

Read the full study on the Jama Network website

The Russian centre

Researchers from Russia have published data on 99 people with MS receiving HSCT at their centre in Moscow.

In 2015, they reported that 64 out of 99 had not experienced relapse or progression within three years following treatment. But some were excluded “due to disease progression or relapses” meaning the outcomes might not represent how successful the treatment will be for other people. 

The conclusion of the study was that the best candidates for HSCT were people with MS who are relatively young (mean age was 35) with low levels of disability (median EDSS was 3.5), experiencing active rapidly progressing MS that is unresponsive to DMTs.

Read the full study on the journal website

Retrospective studies with no control group

We can also learn valuable information from studies where everyone has HSCT and there is no 'control group'. In ‘retrospective’ studies, researchers look back at people who've had HSCT in the past, not necessarily as part of a trial. These are sometimes the best way to gather evidence from large numbers of people.

EBMT HSCT registry

The European Group for Blood and Marrow Transplantation (EBMT) is an organisation focused on reporting and improving HSCT technology.

Since 1996, EBMT has maintained a database of all people receiving HSCT for autoimmune conditions from its members. Researchers can learn from the data they collect by using it to monitor how people do after HSCT.

One study monitored 281 people who had HSCT for any type of MS from 1995 to 2006. The study showed that HSCT is most effective in people with MS who have ‘active inflammation’ in their brain and spinal cord. Five years after HSCT, 33% of people with secondary progressive MS had not progressed, whereas 73% of people with relapsing MS had not progressed.

Imperial College and King’s College 

In June 2021, researchers from London reported results of an observational study including data from 120 people who had HSCT. Almost everyone had MRI inflammation. 58 people had relapsing MS, 40 had secondary progressive MS and 22 had primary progressive MS.

Across the whole group, rates of relapses and new MRI lesions were lower in the years following HSCT than the years before it. And two thirds of people followed up at four years showed no worsening of disability since having HSCT.

Experimental stem cell therapies

Stem cells are an exciting area of research for MS. A number of different procedures are being investigated including MSCT, but only HSCT has been proven to be effective so far.

Scientists have warned against unproven stem cell treatments being offered outside of clinical trials.