Multiple sclerosis is a deeply personal disease. No two people experience MS in exactly the same way, and while the underlying autoimmune event that attacks myelin is consistently at the core of the MS disease process, the signs, symptoms, and progression of the disease can vary enormously from person to person.
In the same vein, every person living with MS responds to treatments in her or his own way. A little over twenty years ago, there were no therapies available that could alter the course of the disease and reduce the number of relapses and brain lesions; today, 11 disease-modifying therapies are approved for relapsing-remitting MS in Canada with several others in the pipeline. A wide selection of disease-modifying therapies is ideal in that it means more options that can manage the individual needs of people living with MS. Despite the crucial advancements in treatment options for MS, some people do not respond to the treatments that are available, which again speaks to the varying nature of the disease.
Ongoing research is helping to expand the arsenal of treatment options for MS, while placing greater emphasis on a more personalized approach to treating the disease. The publication of the results from the Canadian Bone Marrow Transplantation (BMT) Trial in The Lancet represents the culmination of an extensive and collaborative effort funded by the MS Society of Canada’s affiliated Multiple Sclerosis Scientific Research Foundation (MSSRF) to identify a potential treatment for MS involving stem cells. The trial involved a procedure in which selected volunteers living with MS were given high-dose chemotherapy to dismantle the disease-causing immune system, followed by transfusion of their own stem cells to rebuild a healthy immune system that no longer attacks myelin. Given the risks associated with the procedure, individuals who were selected for the trial were those experiencing highly aggressive, inflammatory relapsing-remitting MS that did not respond to available treatments.
The study, titled “Immunoablation and autologous haemopoietic stem-cell transplantation for aggressive multiple sclerosis: a multicentre single-group phase 2 trial”, was led by Drs. Harry Atkins and Mark Freedman at The Ottawa Hospital. Drs. Atkins and Freedman noticed that, following transplantation of the stem cells, the participants showed remarkable improvements in disease course which were maintained over a long period of time. These improvements included the absence of new relapses and inflammatory brain lesions and, in some cases, lasting recovery of function.
The findings from the Canadian BMT Trial represent an important breakthrough in research exploring cell-based therapies for MS. The trial is the only one of its kind in Canada, and although there are have been other stem cell trials using similar techniques conducted abroad, the Canadian BMT Trial boasts the longest follow-up period of any stem cell trial in MS to date, in turn providing a long-term window into the benefits of this procedure. Some of the accompanying studies that branched out from the Canadian BMT Trial provided additional insights into why trial participants might have experienced improvements. These included a study that described the influence of the procedure on preventing disease-causing immune cells called Th17 cells from becoming re-established in the rebuilt immune system, and another that captured detailed imaging scans that mapped out how myelin was being repaired. The publication of these findings in a respected medical journal will likely serve to heighten the awareness surrounding stem cell therapies for MS now that data about the procedure’s efficacy is accepted by the medical community.
What does this mean for people living with MS? While the stem cell treatment is intended for a select group of people with active MS disease, it adds to the growing list of options that are available for different types of MS, which is a positive step towards personalized disease management. Ongoing MS clinical trials around the world involving hematopoietic stem cells may help to address a number of remaining questions, such as, “can the procedure provide further benefits to more people with other types of MS”, or “Is there an alternative version of the procedure that is less risky, but still effective?”
Ultimately, the results of the Canadian BMT trial are exciting because they position stem cells paired with dismantling the disease-causing immune system as a potential treatment option for people living with aggressive, highly active MS who would be otherwise unlikely to benefit from existing disease modifying therapies. The risks associated with this particular chemotherapy regimen remain an important factor in deciding who can undergo the treatment, but the good news is there is still a host of highly efficacious treatment options for relapsing-remitting MS, and a number of promising candidates in development for progressive MS. Additionally, Dr. Freedman continues to lead MESCAMS, a clinical trial looking at a different type of stem cell treatment that does not involve chemotherapy. Results of this work have the potential to make stem cell-based treatments both safer and more accessible to people living with MS.
One person who participated in the Canadian BMT trial and whose story serves as a beacon of hope for certain people living with MS is Jennifer Molson. After being diagnosed with an aggressive form of MS at the age of 21, Jennifer’s disability began to rapidly mount and she went from being a healthy, active adult to being unable to work and relying on others to help her carry out everyday tasks. Dr. Freedman, who also happened to be her neurologist at the time, suggested that Jennifer undergo the chemotherapy and stem cell transplantation procedure as part of the trial. Although Jennifer recounts that her recovery from the procedure was slow and difficult, today she is nearly free of her MS symptoms: she downhill skis and walks in high-heels, works full time and is living her life to its fullest potential. Watch the video below to hear about Jennifer’s remarkable personal journey.