Stem Cells to Prevent and Reverse Disability in Multiple Sclerosis

Professor Tomas Kalincik

University of Melbourne, VIC

February 2023

specialisation: Immunology

focus area: A cure via repair and regeneration

funding type: Project

project type: Investigator Led Research

Summary

Chemotherapy with autologous hematopoietic stem cell transplantation (AHSCT) is occasionally being used to treat aggressive forms of multiple sclerosis (MS). However, it is unclear how effective 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 vs. B-cell depleting therapies (ocrelizumab, rituximab) and immune reconstitution therapy (cladribine).   

The team will also explore individual factors that determine the response to AHSCT. Finally, they will compare different AHSCT conditioning regimens.   

This knowledge will allow for informed use of stem cell transplantation in the treatment of MS.  

Progress

The ongoing research, led by Professor Tomas Kalincik, has made significant progress. 

Over the past year, the team collected and analysed data from collaborating centres in Melbourne, Sydney, Prague, Uppsala, Sheffield, and Ottawa and the MSBase registry. Comparative studies were completed, showing that AHSCT is notably 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. 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. 

Moving forward, the research will focus on comparing AHSCT with immune reconstitution therapies like alemtuzumab and cladribine and exploring individual differences in responses to AHSCT. This research continues to provide valuable insights into MS treatment, potentially improving therapeutic strategies for people living with MS worldwide. 

publications

  • 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. (in press). Effectiveness of autologous haematopoietic stem cell transplantation versus natalizumab in progressive multiple sclerosis. Journal of Neurology, Neurosurgery & Psychiatry. 

Updated 31 March 2024 

lead investigator

total funding

$250,000

start year

2023

duration

3 years

STATUS

Current project

Stages of the research process

Fundamental laboratory Research

Laboratory research that investigates scientific theories behind the possible causes, disease progression, ways to diagnose and better treat MS.

Lab to clinic timeline

10+ years

Translational Research

Research that builds on fundamental scientific research to develop new therapies, medical procedures or diagnostics and advances it closer to the clinic.

Lab to clinic timeline

5+ years

Clinical Studies and Clinical Trials

Clinical research is the culmination of fundamental and translational research turning those research discoveries into treatments and interventions for people with MS.

Lab to clinic timeline

3+ years

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Stem Cells to Prevent and Reverse Disability in Multiple Sclerosis