A therapy normally used to treat blood cancers by “resetting” the immune system could slow the progression of a common form of multiple sclerosis (MS), a study suggests.
Researchers said they hope the treatment could be “included as a standard of care” in treating MS in the future.
Autologous haematopoietic stem cell transplantation (aHSCT) aims to kickstart a patient’s immune system.
Blood cells are harvested from their own bone marrow or blood and are then reintroduced following chemotherapy.
The process could help slow the progression of relapsing remitting MS, according to a Swedish study.
People with the condition have spells where their symptoms get worse but is followed by a period of recovery.
The results indicate that patients would benefit from aHSCT and that this form of stem cell therapy can be safely performed in a clinical setting, representing a powerful clinical tool to successfully treat MS and restore function and quality of life to patients— Dr Alena Pance, University of Hertfordshire
However, after each relapse, their disability can end up worse than before.
The research team recruited 231 patients, 174 of which had been treated with aHSCT before 2020.
Those included in the study had had MS for more than three years and had received two lots of standard treatment.
The study found no evidence of disease activity in 73 per cent of those treated after five years and in 65 per cent after 10 years.
Among the 149 patients who had some disability to begin with, 54 per cent improved, 37 per cent remained stable and nine per cent got worse.
On average patients were relapsing 1.7 times in the year before the aHSCT treatment.
Following the therapy, the average relapse rate was one every 30 years.
The team said its findings, published in the Journal of Neurology Neurosurgery and Psychiatry, “demonstrate that aHSCT for (relapsing-remitting MS) is feasible within regular healthcare and can be performed without compromising safety”.
The researchers added: “Our study corroborates the results observed in the only randomised controlled trial conducted to date. We believe that aHSCT could benefit a greater number of MS patients and should be included as a standard of care for highly active MS.”
According to the MS Society UK, there are more than 130,000 people with the condition in the UK and 7,000 are newly diagnosed each year.
Symptoms include fatigue, muscle spasms and pain, as well as problems with vision, mobility, thinking, talking and swallowing.
Dr Alena Pance, a senior lecturer in genetics at the University of Hertfordshire, said: “The disability caused by MS is the result of damage or degradation of the myelin that envelops and protects the nerves of the central and peripheral nervous system.
“Though aHSCT can generate only blood cells (of all types) and not cells of the neuronal system, resetting the immune system stops the constant attack on myelin, which then gives a chance to the specialised repair cells called oligodendrocyte precursors to regenerate the damaged myelin.
“However the potential and extent of repair depends on the severity of the disease, which is why, as the study indicates, the effect on disability is greater the lower the severity of the disease is.”
Dr Pance added that a limitation of the Swedish study is the “absence of control groups treated only with regular therapies” which “makes it difficult to assess the magnitude of the effect of aHSCT”.
However, she cited a trial comparing aHSCT and disease-modifying therapy (DMT), which showed “similar positive effects of aHSCT in terms of prolonged time to disease progression and better outcomes than DMT”.
“The results indicate that patients would benefit from aHSCT and that this form of stem cell therapy can be safely performed in a clinical setting, representing a powerful clinical tool to successfully treat MS and restore function and quality of life to patients,” she added.
Dr Sarah Rawlings, executive director of research and external affairs at the MS Society, said: “HSCT on the NHS is subject to strict eligibility criteria and can be difficult to access.
“We know HSCT doesn’t work for everyone with MS, but it has been an important development in MS treatment and some people see life-changing results. This study provides further evidence as to how it can help slow progression of symptoms.
“More than 130,000 people live with MS in the UK and there are multiple treatments available, but we still don’t know enough about how the effectiveness of HSCT compares with the most effective DMTs.”
Dr Rawlings also urged patients to consider the StarMS study, which is looking at the effectiveness of stem cell transplants compared to the latest DMTs.
Some patients involved in the trial will be given stem cell transplants if they have “highly active multiple sclerosis failing drug treatment” or as a first-line treatment for patients with the aggressive multiple sclerosis.
Others will be given drug treatments which have shown promise in clinical trials – alemtuzumab, ocrelizumab, ofatumumab or cladribine.
Dr Rawlings added: “These new results are encouraging but only observe how disability progresses after HSCT and so reinforce the need for randomised controlled trials comparing HSCT with the most highly effective DMTs.
“The new StarMS trial, currently taking place in hospitals around the UK, will do this. We would urge anyone considering HSCT or interested in taking part in the StarMS trial to discuss this with their neurologist.”
