HSCT clinical trials
Find out more about HSCT clinical trials
We want to see more research investigating stem cell transplantation in the UK. We’d particularly like to see trials comparing HSCT with the most effective disease modifying therapies (DMTs).
Right now we’re actively supporting researchers who are trying to make this happen.
MIST is a phase 3 clinical trial being run in the US and Sheffield. It involves 110 people with relapsing MS. All 110 people have continued to have relapses despite being on DMTs like beta interferon and Copaxone.
Researchers are testing the benefits of lower intensity chemotherapy before stem cell transplantation and comparing it to existing DMTs. They will measure if HSCT can reduce relapses and slow progression, as measured by the Expanded Disability Status Scale (EDSS).
The trial is expected to finish in 2018.
What we know so far - clinical trials
There have been a number of different trials investigating the potential of HSCT for people with MS. These trials have tested the benefits of different levels of chemotherapy and involved people with both relapsing and progressive MS.
Some trials have used lower intensity (non-myeloablative) chemotherapy to remove certain immune cells. Others have tested more aggressive (myeloablative) chemotherapy to wipe out all of the immune cells in the body.
The level of chemotherapy used will affect the risks involved in the procedure. More aggressive chemotherapy carries higher risks and more severe side effects.
The results of several clinical trials have been published. Some of these trials 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.
Canadian HSCT trial
This 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.
This 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 trial used high-intensity (myeloablative) chemotherapy.
The ASTIMS trial
The ASTIMS trial compared HSCT with mitoxantrone in 21 people with relapsing or secondary progressive MS.
To be involved in the trial, people had to have:
- experienced an increase in disability despite taking a conventional disease modifying therapy (DMT)
- and active lesions on an MRI scan.
The trial used high-intensity (myeloablative) chemotherapy.
Results showed HSCT was more effective at reducing relapses than mitoxantrone. HSCT was not shown to reduce disability in the four year follow-up.
The Chicago Experience
This trial involved 123 people with relapsing MS. All of those involved:
- had experienced relapses in the year before the trial started
- and were able to walk 100m using a walk stick without rest.
28 people with secondary progressive MS were also treated as part of the trial.
The trial used low-intensity (non-myeloablative) chemotherapy.
International HSCT registries Since 1996, the European Group for Blood and Marrow Transplantation (EBMT) has maintained a database of all people undergoing HSCT for autoimmune conditions. EBMT is an organisation focused on reporting and improving HSCT technology.
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 results were published in 2017.
Understanding how HSCT affects the immune system
We funded a project that investigated the impact HSCT has on the immune system. Researchers looked at how people’s immune systems changed after stem cell transplantation.
This project has helped us to better understand how the treatment can reduce the damage seen in MS. It has also given us a better understanding of the long-term benefits and risks of HSCT for people with MS.