Scientists have announced a breakthrough in treating an aggressive type of multiple sclerosis after successfully replacing the immune system and regenerating a new one using blood stem cells.
The Canadian study – the first to show the complete, long-term suppression of all inflammatory activity in people with MS – revealed that the intensive procedure can eliminate all signs of damaging brain inflammation in people with early, aggressive MS. It also helps with recovery.
Led by Dr Harold Atkins and Dr Mark S. Freedman of The Ottawa Hospital and the University of Ottawa, the trial included 24 participants, all of whom were followed for up to 13 years.
Dr Atkins, a stem cell transplant physician and scientist at The Ottawa Hospital, and associate professor at the University of Ottawa, cautioned that while the findings were exciting, the procedure works only with a specific group of 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,” he said. “People with MS who have had significant disability for a long time would likely not benefit.”
The trial, details of which are published in the latest edition of The Lancet, evaluated immunoablation and autologous hematopoietic stem cell transplantation (IAHSCT) among 24 participants with aggressive relapsing MS and they were followed for between four and 13 years after treatment.
After they had undergone the therapy, none reported a clinical relapse – prior to the treatment each person had an average of 1.2 relapses per year – and no new active inflammatory lesions were detected in any of their brains.
None needed MS-specific drugs to control their disease and 70% reported a complete halt in disease progression. The average rate of brain shrinkage, typically a measure that correlates with MS progression, returned to levels that are associated with normal ageing.
However, 40% experienced some lasting reversal of symptoms such as vision loss, muscle weakness and balance problems. One participant died of liver failure due to the treatment and another required intensive care for liver complications.
The researchers said that the treatment regimen was modified to reduce toxicity, but all participants developed fevers, which were frequently associated with infections.
Dr Freedman, a neurologist and senior scientist at The Ottawa Hospital and professor at the University of Ottawa, said the follow up time helped to solidify the trial results.
“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,” he said.
“However, this is a very complex procedure that should only be performed at very specialised centres with expertise in both the management of MS patients and blood stem cell transplantation.”
Trial participant Jennifer Molson, who was diagnosed with MS in 1996 at the age of 21, said:
“Before my transplant I was unable to walk or work and was living in assisted care at The Ottawa Hospital Rehabilitation Centre,” she said.
“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 Atkins said that a variation of the procedure has been used to treat leukaemia for decades, but its use for auto-immune diseases is relatively new.
Atkins HL, Bowman M, Allan D et al, Immunoablation and autologous haemopoietic stem-cell transplantation for aggressive multiple sclerosis: a multicentre single-group phase 2 trial. Lancet 9 June 2016. [abstract]
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