One of the things that tripped across my news feed then was an HSCT MS study in Canada that seemed very similar to Dr. Burt's procedure. I asked Dr. Burt about it when he stopped by on his daily rounds to visit me. He said the Canadian study was a similar procedure, but that he believed the chemo being used there was unnecessarily harsh. The Canadian study, managed by Dr. Mark Freedman, was an early-stage effort with only 24 patients. Dr. Burt's ongoing Phase 3 study will enroll hundreds of patients.
A March 2013 Science Daily article summarized some of the results of that Canadian study. These appear to support Dr. Burt's hypothesis about how HSCT resets the immune system back to a balanced, self-tolerant state. The article, posted below, talks about a lasting reduction of Th17 immune cells. (An overabundance of Th17 cells appears to set off a chain reaction of other immune cells that begin attacking the central nervous system.)
Anyway, it's reassuring to have more confirmation that HSCT works to stop MS, and also about how and why it works at the molecular level. I certainly don't understand it all, but it amazes me that scientists are not only figuring out the complex workings of the human immune system, but also finding ways to fix it when it's broken, like mine was.
And, having done HSCT myself, I have a different perspective on the concept of "risky" as stated in the article below. Risky compared to what? A life in a wheelchair while taking poorly understood immune-modulating drugs with their own risks and side effects? I'm so glad I did HSCT, and grateful that Dr. Burt's experience with the procedure, along with his professional team of nurses and specialists at Northwestern Memorial Hospital, minimized that risk significantly.
Best wishes and good luck to my new friends and acquaintances who are preparing to undergo the HSCT procedure in Chicago and elsewhere in the world! And especially good wishes to all those suffering from MS and other autoimmune diseases who are still in "wait and see" mode in regard to HSCT. Science keeps learning more as Dr. Burt's Phase 3 study continues. Dr. Burt's Phase 1 and 2 results continue to be very positive, with lasting results for most treated patients who remain in remission years later. The early stage Canadian study posted below also looks positive for HSCT.
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Major advance in understanding risky but effective multiple sclerosis treatment
Date:
March 26, 2013
Source:
McGill University
Summary:
A new study by multiple sclerosis researchers addresses why bone marrow transplantation has positive results in patients with particularly aggressive forms of MS.
new study by multiple sclerosis researchers at three Canadian centres addresses why bone marrow transplantation (BMT) has positive results in patients with particularly aggressive forms of MS. The transplantation treatment, which is performed as part of a clinical trial and carries potentially serious risks, virtually stops all new relapsing activity as observed upon clinical examination and brain MRI scans. The study reveals how the immune system changes as a result of the transplantation. Specifically, a sub-set of T cells in the immune system known as Th17 cells, have a substantially diminished function following the treatment.
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The finding to be published in the upcoming issue ofAnnals of Neurology and currently in the early online version, provides important insight into how and why BMT treatment works as well as how relapses may develop in MS.
"Our study examined why patients essentially stop having relapses and new brain lesions after the bone marrow transplant treatment, which involves ablative chemotherapy followed by stem cell transplantation using the patient's own cells," said Prof. Amit Bar-Or, the principle investigator of the study, who is a neurologist and MS researcher at The Montreal Neurological Institute and Hospital -The Neuro, McGill University, and Director of The Neuro's Experimental Therapeutics Program. "We discovered differences between the immune responses of these patients before and after treatment, which point to a particular type of immune response as the potential perpetrator of relapses in MS."
"Although the immune system that re-emerges in these patients from their stem cells is generally intact, we identified a selectively diminished capacity of their Th17 immune responses following therapy -- which could explain the lack of new MS disease activity. In untreated patients, these Th17 cells may be particularly important in breaching the blood-brain-barrier, which normally protects the central nervous system. This interaction of Th17 cells with the blood-brain barrier can facilitate subsequent invasion of other immune cells such as Th1 cells, which are thought to also contribute to brain cell injury.
Twenty-four patients participated in the overall clinical trial as part of the 'Canadian MS BMT' clinical trial, coordinated by Drs. Mark Freedman and Harry Atkins at the Ottawa General Hospital. The new discovery, made in a subset of patients participating in the clinical trial, was based on immunological studies carried out jointly in laboratories at The Neuro and the Université de Montréal. Results of this study not only show the clinical benefits of BMT treatment, but also open a unique window into the immunological mechanisms underlying relapses in MS. Th17 cells could be the immune cells associated with the initiation of new relapsing disease activity in this group of patients with aggressive MS. This finding deepens our understanding of MS and could guide the development of personalized medicine with a more favourable risk/benefit profile.
Among the patients treated in the Canadian MS BMT clinical trial, was Dr. Alexander Normandin, a family doctor, who was a third- year McGill medical student getting ready for his surgery exams when he first learned he had MS, "I was so engrossed in my studies that I didn't pay attention to the first sign but within a few days of waking up with a numb temple, my face felt frozen. I learned that I had a very aggressive form of MS and would probably be in a wheelchair within a year. It was a brutal blow. I became patient #19 -- of only 24 for this experimental treatment. My immune system was knocked out and then rebooted with my stem cells. Today, my MS has stabilized. I now have this disease under control and I take it one day at a time."
Both the clinical and biological studies were supported by the Research Foundation of the Multiple Sclerosis Society of Canada.
Multiple Sclerosis
MS is a disorder of the brain and spinal cord that causes fatigue, disequilibrium, sensory problems and muscle paralysis. The cause of MS is unknown, but evidence suggests that it is an auto-immune disease that destroys myelin, a substance coating axons, the thin strands that carry signals between brain cells.It usually strikes between the ages of 15 and 40 but can begin as early as age two. Women have twice the probability of developing MS than men. Canada has one of the world's highest national rates -- about 1,100 new cases each year. Some 50,000 Canadians have MS. More than 1 in 5 lives in Quebec. The most common form of MS is relapsing-remitting, in which acute symptoms alternate with periods of remission. Primary progressive MS, the least common form, develops continually without remission. Secondary progressive MS begins as relapsing-remitting, then becomes steadily progressive.
Story Source:
The above story is based on materials provided by McGill University. Note: Materials may be edited for content and length.
Journal Reference:
- Peter J. Darlington, Tarik Touil, Jean-Sebastien Doucet, Denis Gaucher, Joumana Zeidan, Dominique Gauchat, Rachel Corsini, Ho Jin Kim, Martin Duddy, Farzaneh Jalili, Nathalie Arbour, Hania Kebir, Jacqueline Chen, Douglas L. Arnold, Marjorie Bowman, Jack Antel, Alexandre Prat, Mark S. Freedman, Harold Atkins, Rafick Sekaly, Remi Cheynier, Amit Bar-Or. Diminished Th17 (not Th1) responses underlie multiple sclerosis disease abrogation after hematopoietic stem cell transplantation. Annals of Neurology, 2013; DOI: 10.1002/ana.23784
