HSCT clinical trials
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.
Find out more about HSCT clinical trials
Researchers are continuing to try and find the best HSCT procedure for MS. This often involves deciding what level of chemotherapy is least harmful to the patient while still properly tackling MS.
Current clinical trials are also 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.
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.
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 JACIE accreditation.
What we know so far - clinical trials
Several clinical trials of HSCT for MS have been published. Some of these compared HSCT with other treatments, but most did not have a control group.
Due to the intense nature of the procedure, all of the trials are open label. This means the people involved in the trial knew they were having HSCT.
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.
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.
Canadian HSCT trial
The Canada 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.
The HALT-MS trial involved 24 people with relapsing MS, who were experiencing severe relapses and worsening of disability before the treatment. This was despite taking disease modifying therapies (DMTs).
The ASTIMS trial
The 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 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.
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.
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.
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.
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.