Being one of the over 2.3 million people in the world who live with MS can bring about significant challenges. Currently, there is no sure fire cure for the autoimmune condition, however, many of the worlds greatest minds are dutifully at work learning about MS. One such set of scientists includes Dr. Harold Atkins and Dr. Mark S. Freedman of the Ottawa Hospital and the University of Ottawa. The two doctors have spent the last few decades working on a clinical trial just recently published in The Lancet, a top medical journal. While their results may be the first of their kind, they do have limitations.
Dr. Atkins and Dr. Freedman led a $6.47 million clinical trial that included 24 participants, ages 18-50 years old. The research, funded primarily by the MS Society of Canada and its affiliated Multiple Sclerosis Scientific Research Institute, followed a very distinct subset of individuals with MS who were categorized as having a poor prognosis, ongoing and aggressive disease activity, and significant disability (an Expanded Disability Status Scale of 3.0-6.0).
The trial utilized a treatment known as immunoablation and subsequent autologous hematopoietic stem cell transplantation, or aHSCT. In basic terms, the researchers administered medication to participants that essentially “pulled” out their hemapoietic stem cells from their bone marrow, and harvested it from their blood. Once the stems cells were collected, they were purified, frozen, and stored while the participant underwent high doses of chemotherapy meant to eliminated their entire malfunctioning immune system. After their immune system is destroyed and has no “memory” of its previous function, the stem cells were then reintroduced to the participant to generate an entirely new, (hopefully) properly functioning central nervous system. This type of therapy can be incredibly risky. In Atkins and Freedman’s study, one patient passed away due to liver failure as a result of the aHSCT, and many others experienced serious infection. The treatment, previously only used in severe cases of leukemia, has a 5% mortality rate, making it incredibly risky.
The researchers followed up with participants over the course of the next 3-13 years depending on the availability of each participant. It was found that none of the patients required further MS-specific drugs to control their disease, experienced no more relapses of MS, nor any more lesions in their brains (both of which were constantly being experienced before the treatment began in the participants), nearly 70% of participants experienced a halt in MS progression, and 30-40% experienced an improvement or lasting reversal of disabling symptoms.
“Our trial is the first to show the complete, long-term suppression of all inflammatory activity in people with MS. This is very exciting. However, it is important to note that this therapy can have serious side effects and risks, and would only be appropriate for a small proportion of people with very active MS. People with MS who have had a significant disability for a long time would likely not benefit.”
-Dr. Atkins, stem cell transplant physician and scientist at The Ottawa Hospital and associate professor at the University of Ottawa
“This procedure should be considered as a treatment option for people with early, aggressive MS. Although this trial was relatively small, it was intensive, with the longest prospective follow-up of any such treatment group to date, and that is what makes the results so convincing. However, this is a very complex procedure that should only be performed at very specialized centres with expertise in both the management of MS patients and blood stem cell transplantation.” – Dr. Freedman, neurologist and senior scientist at The Ottawa Hospital and professor at the University of Ottawa
As both professors state, the expansion of this study is limited. There was no control or placebo group, and participants had to have an early, and very aggressive form of MS to even be considered, which doesn’t represent all MS patients. While the study does utilize stronger drugs to eliminate participants’ immune systems, followed up with participants over the course of a long time, and the participants did show long-lasting responses to the treatment, it is important to note that there is much more work to be done. Critics have suggested that while this study may not change the way MS is treated in the short term, it can certainly be built upon, and made safer to be expanded upon in research in the longer term.1-3
Atkins HL et al. “Immunoablation and autologous haemopoietic stem-cell transplantation for aggressive multiple sclerosis: a multicentresingle-group phase 2 trial.” Available at: http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(16)30169- 6/abstract [Accessed 22 June 2016.]
Dörr J. “Haemopoietic stem-cell transplantation for multiple sclerosis: what’s next?” Available at: http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(16)30377- 4/abstract [Accessed 22 June 2016.]
ScienceDaily. “MS breakthrough: replacing diseased immune system halts progression, allows repair.” Available at: https://www.sciencedaily.com/releases/2016/06/160609222439.htm [Accessed 22 June 2016.]