March 22, 2018

International stem cell clinical trial shows stabilized disease and improved disability in MS

The Treatment:

Autologous hematopoietic stem cell transplantation (AHSCT) is an aggressive procedure which involves collecting stem cells from an individual’s own bone marrow and then exposing them to chemotherapy to deplete the immune system. The stem cells are then reintroduced into the body where they mature into new immune cells. In multiple sclerosis, the goal for this type of transplant is to reboot the immune system which is thought to be causing damage the nerve cells in MS.

The Clinical Trial:

Dr. Richard Burt of Northwestern University in Chicago and a team of researchers conducted an international trial of AHSCT with 110 people with active relapsing-remitting MS (RRMS). The results of this study were recently reported at the European Society for Bone and Marrow Transplantation in Lisbon. Participants of this study had frequent relapses and were unresponsive to standard disease-modifying therapies (DMTs). Half of the people in the trial were randomized to receive the transplantation procedure while the other half were treated with a standard DMT. The researchers reported changes in disability using the Expanded Disability Status Scale (EDSS) and tracked the number of relapses. The EDSS score of people receiving the treatment improved from an average of 3.5 to 2.4 while those on DMTs declined from an EDSS of 3.3 to 3.9. Furthermore, within the first year of the trial, only one participant experienced a relapse compared to 39 relapses reported in the DMT group. The trial is still active, but not recruiting. Additional details about this trial will be available when the results have been formally published.


The results from this study are similar to those reported from the Canadian Bone Marrow Transplant (BMT) trial.The Canadian BMT trial, funded by the MS Society of Canada and the MS Scientific Research Foundation, was led by Drs. Mark Freedman and Harry Atkins from the Ottawa Hospital Research Institute and published in the renowned medical journal The Lancet in 2016. Similar to the trial led by Dr. Burt reported above, the BMT trial resulted in a complete elimination of clinical disease activity, meaning no new relapses, or brain lesions were observed in all 23 participants during the entire follow-up period. Together, these studies support AHSCT as a potential treatment for those living with aggressive, highly inflammatory relapsing-remitting MS who have not responded to DMTs. For information on the Canadian BMT trial, check out the Quick Facts found here. For more information about this procedure in Canada, visit the Ottawa Hospital Research Institute website.