Chemotherapy with autologous hematopoietic stem cell transplantation (AHSCT) is occasionally being used to treat aggressive forms of multiple sclerosis (MS). The chemotherapy kills the immune cells that attack the brain and spinal cord in MS. The stem cells are then transplanted to regrow a new immune system that is more tolerant.
However, it is unclear how effective AHSCT is compared to standard MS therapies.
In this project, Professor Tomas Kalincik will use one of the largest international datasets to compare the effectiveness of stem cell transplantation with the most effective MS therapies.
The project will compare treatment outcomes in AHSCT versus B-cell depleting therapies (ocrelizumab, rituximab) and treatments that can ‘reset’ the immune system (immune reconstitution therapies) such ascladribine and alemtuzumab.
The team will also explore individual factors that determine the response to AHSCT.
Finally, they will compare outcomes of different AHSCT “conditioning regimens” (chemotherapy mixes).
This knowledge will allow for informed use of stem cell transplantation in the treatment of MS.
Professor Tomas Kalincik established the RESCUE-MS international collaboration of 7 AHSCT MS centres for this project. His team has collected and analysed data from these centres in Melbourne, Sydney, Prague, Uppsala, Sheffield, and Ottawa and also the MSBase registry. Comparison studies found that AHSCT is significantly more effective than fingolimod and slightly more effective than natalizumab in treating relapsing-remitting MS.
However, in progressive MS, AHSCT was not better than natalizumab in controlling relapses and disability. Therefore, AHSCT is not suitable for people with progressive MS who have few or no relapses. These findings have been presented at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) and published in reputable journals such as JAMA Neurology and the Journal of Neurology, Neurosurgery & Psychiatry.
Compared to immune reconstitution therapies, AHSCT was considerably more effective than cladribine and slightly more effective than alemtuzumab at suppressing MS relapses and helping recovery from disability in relapsing MS.
The team is now analysing the effect of different preparation treatments, known as conditioning regimens, and the influence of individual factors on the effectiveness and safety of AHSCT. These factors include age, gender, time from MS onset to therapy, disability level, relapse history, and MRI activity before treatment.
This research continues to provide valuable insights into MS treatment, potentially improving therapeutic strategies for people living with MS worldwide. Thanks to this work, decisions about using AHSCT are now more strongly backed by evidence.
With AHSCT now supported by stronger evidence, Professor Kalincik has been awarded an NHMRC Investigator Grant to expand his research. He and the RESCUE-MS group will continue the research on how best to use AHSCT in the treatment of MS.
Kalincik, T., Sharmin, S., Roos, I., et al. (2023). Comparative effectiveness of autologous haematopoietic stem cell transplantation vs fingolimod, natalizumab, and ocrelizumab in highly active relapsing-remitting multiple sclerosis. JAMA Neurology, 80(6), 702-713.
Kalincik, T., Sharmin, S., Roos, I., et al. (2024). Effectiveness of autologous haematopoietic stem cell transplantation versus natalizumab in progressive multiple sclerosis. Journal of Neurology, Neurosurgery & Psychiatry 95(5): 454-60
Updated 31 March 2025
$250,000
2023
3 years
Current project
