Interim AHSCT trial results released - MS Research Australia

Interim AHSCT trial results released

19 March, 2018

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 overnight.

Scientific conferences like these enable researchers to discuss preliminary results and get feedback from their colleagues before the study is completed and published in scientific journals.

AHSCT involves using cancer chemotherapy to completely or partially remove the patient’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 patient’s own haematopoietic stem cells back into the body. More information can be found on the MS Research Australia website.

The clinical trial discussed at the conference in Lisbon is known as the MIST study, led by Professor Richard Burt based in Chicago, USA. It involved 110 patients with active relapsing remitting MS (more than two relapses in the preceding year) who 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 of the patients were treated with approved MS medications which included mitoxantrone, glatiramer acetate, intereferon-beta, fingolimod, dimethyl fumarate and natalizumab.

While we do not have access as yet to the full trial results the conference abstract provides a summary of the interim results presented at the conference.

The abstract indicates that, so far the patients have been followed for an average of 3 years following treatment, ranging from a minimum of 12 months up to 5 years. During the first 12 months following treatment there was only one relapse 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 had a decrease in disability of on average 1.1 point on the EDSS scale. The EDSS is a standard measure used by neurologists to rate disability in MS. People receiving standard medications had an average increase in EDSS disability of 0.6 points.

Treatment was deemed a failure if a person had a confirmed disability increase of 1 EDSS point or more in six months. 30 of the 50 patients in the standard MS therapy group met this criterion and were then treated with AHSCT. Three of the 52 receiving 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 that “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”.

She added, “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 at

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


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.

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