People with MS who were involved in a long-term clinical trial are out and about enjoying a full and normal life with no signs of the disease. This follows their recovery from the stem cell transplants involving aggressive chemotherapy, or aHSCT as the procedure is known.
The 24 multiple sclerosis patients recruited for the trial were severely disabled but, with the exception of one who died, are now walking, working, kayaking, and skiing.
The trial is the first in the world to show complete long-term stopping of relapses of the debilitating disease and formation of new brain lesions. It used the Autologous Hematopoietic Stem Cell Transplantation (aHSCT) method with strong chemotherapy drugs to kill off the patients’ immune systems before rebooting them with their own heathy stem cells.
Normal life includes marriage and skiing
So, let’s take a look at how things are going now, thanks to the Daily Telegraph’s science editor Sarah Knapton:
Jennifer Molson, who was diagnosed with MS in 1996, and received her stem cell transplant in 2002 said: “Before my transplant I was unable to walk or work and was living in assisted care.
“Now I am able to walk independently, live in my own home and work full time. I was also able to get married, walk down the aisle with my Dad and dance with my husband.
“I’ve even gone downhill skiing. Thanks to this research I have been given a second chance at life.”
Dr. Mark Freedman, of the University of Ottawa and Ottawa Hospital, where the trials were carried out, said: “Jennifer, she freaked me out one day when she came to the clinic wearing high heels. This was a girl who could barely walk.”
The trial included 24 participants with aggressive, relapsing MS who were followed for up to 13 years after treatment.
This how aHSCT works: First, a person is given medication to encourage their stem cells to migrate from their bone marrow into their blood. These stem cells are then collected from the blood, purified and frozen.
Next, high doses of chemotherapy drugs are used to destroy the person’s diseased immune system. This necessitates a stay in isolation.
The stem cells are then unfrozen and transplanted back into the same person, so that they can give rise to a new immune system that has no memory of the previous pattern of attacking the central nervous system.
Only for those with active MS
Ms. Knapton writes:
“Our trial is the first to show the complete, long-term suppression of all inflammatory activity in people with MS,” said Dr. Harold Atkins, a stem cell transplant physician and scientist at The Ottawa Hospital, and associate professor at the University of Ottawa.
“A variation of this procedure has been used to treat leukaemia for decades, but its use for auto-immune diseases is relatively new.
“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.”
It must be pointed out that during the trial one participant died of liver failure due to the treatment and another required intensive care for liver complications. However, comprehensive medical tests, at centers where this treatment is used worldwide, are used to screen out patients judged to be at risk.
The Telegraph continues:
Dr Emma Gray, Head of Clinical Trials at the MS Society, said: “This type of stem cell transplantation is a rapidly evolving area of MS research that holds a lot of promise for people with certain types of MS.
“This treatment does offer hope, but it’s also an aggressive procedure that comes with substantial risks and requires specialist aftercare.”
But experts said the results constituted a breakthrough in the treatment of MS.
Back to Ms Knapton;
Prof Siddharthan Chandran, MacDonald Professor of Neurology, MRC Centre for Regenerative Medicine, University of Edinburgh, said: “This is an important and carefully conducted proof of concept study that demonstrates that powerful chemotherapy based treatment for a selected subset of MS patients with very aggressive disease is effective in preventing further disabling relapses and, in a proportion, appears to render them effectively disease free.”
And while aHSCT is NOT a cure, Dr Stephen Minger, stem cell biologist and independent consultant, of SLM Blue Skies innovations Ltd is enthusiastic. Sarah Knapton explains:
He said: “The clinical results are truly impressive, in some cases close to being curative.
“For a life-long progressive disease like MS with few treatment options this is really exciting data. I would consider it a breakthrough therapy.”
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