Encouraging preliminary results of one of the first randomised controlled trials comparing autologous haematopoietic stem cell transplant (AHSCT) to other available MS therapies have been presented at the Conference of the European Society for Bone Marrow Transplant (EMBT) in Lisbon.

The trial, known as the MIST study, was led by US Professor Richard Burt and involved 110 people with active relapsing remitting MS.


AHSCT involves using cancer chemotherapy to completely or partially remove the individual’s immune system. The extent of immune system destruction depends on the chemotherapy regime used. The regrowth of the immune system is then supported by transfusing the person’s own haematopoietic stem cells back into their body.

The 110 participants in the MIST study were treated at sites in the USA, UK and Europe. The patients were randomly split into two groups. Half were treated with AHSCT using the cyclophosphamide chemotherapy regime, which removes the circulating immune cells but leaves the bone marrow relatively unscathed. The other half were treated with approved MS medications including mitoxantrone, glatiramer acetate, intereferon-beta, fingolimod, dimethyl fumarate and natalizumab.

Whilst the full trial results are not yet available, the conference abstract does provide a summary of the interim results. The abstract notes that the participants have so far been monitored for an average of three years following treatment, ranging from a minimum period of 12 months up to five years. During the first 12 months following treatment, only one relapse was observed in the group receiving AHSCT, whereas 39 relapses were identified for those who received the other treatments. The abstract does not describe the number of relapses observed in subsequent years.
Participants treated with AHSCT showed a decrease in disability of, on average, 1.1 point on the Expanded Disability Status Scale (EDSS).

The EDSS is a standard measure used by neurologists to rate disability in MS. People receiving standard medications had an average increase in disability of 0.6 EDSS  points.

Treatment was deemed unsuccessful if a person had a confirmed disability increase of one EDSS point or more in six months. Among the 50 people in the standard MS therapy group, 30 met this criterion and were then treated with AHSCT. Three of the 52 who received AHSCT were considered to have treatment failure. The research team reported no deaths and there were no very serious adverse events as a result of the AHSCT treatment.

It is important to note that this is an interim analysis of the trial results and we will need to await the publication of the full trial results for more details and more certainty about the outcomes.

Dr Lisa Melton, Head of Research at MS Research Australia commented:
‘The global clinical community have been eagerly awaiting the results of clinical trials such as this to help inform their use of this treatment for people with MS. These results, together with the international data that has been accumulating on this treatment for MS, indicate that AHSCT is a potentially important treatment option for people with active relapsing MS who fail to respond to other MS therapies’.

‘The publication of these results and those of other ongoing studies will help support hospitals and clinicians to be able to safely provide this treatment to people with MS who need it.’

In Australia, AHSCT for MS is currently provided as part of observational clinical trials at the Austin Hospital in Melbourne and St Vincent’s Hospital in Sydney. Patients must be referred by a neurologist and have failed to respond to other MS therapies to be eligible. More details on these studies can be found here.

For more details on AHSCT for MS and the results other international studies please visit our website here.

With thanks to MS Research Australia – the lead provider of research summaries on our website.

Note: MS Research Australia does not endorse any particular treatment for MS. MS is an extremely variable condition and people can respond differently to treatments. Treatment decisions need to be taken in conjunction with a neurologist and should take into consideration the unique health and life circumstances of each individual.