An exciting new stem cell injection treatment could stop multiple sclerosis in its tracks.
A collaborative science study involving experts in Europe and the US found the treatment appears to protect the brains of MS patients from further damage.
In the first-ever clinical trials in humans, the researchers found patients injected with the stem cells exhibited no increase in disability or worsening of symptoms.
The promising study, published in the journal Cell Stem Cell, is hoped to lead to further clinical trials that could provide treatment for progressive MS.
More than two million people live with MS across the globe and, whilst some treatments currently available can reduce the severity and frequency of relapses, two-thirds of patients still transition into a debilitating secondary progressive phase of the disease within 25 to 30 years of diagnosis.
In MS, the body’s immune system attacks and damages myelin – the protective sheath around nerve fibers – causing disruption to messages sent around the brain and spinal cord.
Key immune cells involved in the process are called macrophages – meaning ‘big eaters’ – which ordinarily attack and rid the body of unwanted intruders.
However, a particular type of macrophage found in the brain and spinal cord, known as a microglial cell, can attack the central nervous system (CNS) in progressive forms of MS, causing chronic inflammation and damage to nerve cells.
Recent scientific advances involving the transplantation of stem cells have raised expectations that therapies could be developed to help ameliorate this damage.
These involve the transplantation of ‘master cells’, which can be programmed to develop into almost any type of cell within the body.
Previous experiments in mice from the team at the University of Cambridge have shown that skin cells reprogrammed to be brain stem cells and transplanted into the CNS can help to reduce inflammation, and may even be able to help repair damage caused by MS.
The research team behind the latest study, incorporating experts from the UK, US, Switzerland and Italy, completed a world-first early-stage clinical trial in which neural stem cells were injected into the brains of 15 patients with secondary MS recruited from two Italian hospitals.
The trial was undertaken by teams at the University of Cambridge, the University of Milano-Bicocca, the Casa Sollievo della Sofferenza and Santa Maria Terni hospitals in Italy, the Ente Ospedaliero Cantonale hospital in Lugano, Switzerland, and the University of Colorado in the United States.
The stem cells used in the trial were derived from cells from the brain tissue of a single, miscarried fetal donor.
The researchers had previously shown that it would be possible to produce an almost limitless supply of these stem cells from a single donor and, in the future, it may be possible to derive these cells directly from the patient – helping to overcome practical problems associated with the use of allogeneic fetal tissue.
After their injections, the 15 patients – recruited from the two Italian hospitals involved in the study – were followed over the proceeding year.
No treatment-related deaths or serious adverse events were recorded, and, though some side effects were observed, all were either temporary or reversible.
All patients exhibited high levels of disability at the beginning of the trial, with most requiring a wheelchair.
However, during the 12-month follow-up period, none showed any increase in disability nor any worsening of symptoms.
None of the patients reported symptoms that suggested a relapse, and neither did any of their cognitive function worsen significantly.
Overall, the researchers suggest this points to the substantial stability of the disease without signs of progression – though they admit the high levels of disability at the start of the trial make this difficult to confirm.
A subgroup of patients was also assessed for changes in the volume of brain tissue associated with disease progression, which found that the larger the dose of injected stem cells, the smaller the reduction in this brain volume over time.
The researchers speculate that this may be down to the stem cell transplant dampening inflammation.
They additionally looked out for signs that the stem cells were having a neuroprotective effect in protecting nerve cells from further damage.
Previous work has shown how tweaking metabolism – the process of how the body produces energy – can reprogram microglia from ‘bad’ to ‘good’.
In their new study, the researchers looked at how the brain’s metabolism changes after the treatment.
They measured changes in the fluid around the brain and in the blood over time and found signs linked to how the brain processes fatty acids which were connected to how well the treatment works and how the disease develops.
The higher the dose of stem cells, the greater the levels of fatty acids, which also persisted over the 12-month period.
Professor Stefano Pluchino, a co-leader of the study from the University of Cambridge, admitted that though the research had limitations, the findings were extremely promising.
“We desperately need to develop new treatments for secondary progressive MS, and I am cautiously very excited about our findings, which are a step towards developing a cell therapy for treating MS,” he said.
“We recognize that our study has limitations: it was only a small study and there may have been confounding effects from the immunosuppressant drugs, for example – but the fact that our treatment was safe and that its effects lasted over the 12 months of the trial means that we can proceed to the next stage of clinical trials.”
Professor Angelo Vescovi, another co-leader of the study from the University of Milano-Bicocca, added: “It has taken nearly three decades to translate the discovery of brain stem cells into this experimental therapeutic treatment.
“This study will add to the increasing excitement in this field and pave the way to broader efficacy studies, soon to come.”
Caitlin Astbury, Research Communications Manager at the MS Society, says:
“This is a really exciting study which builds on previous research funded by us.
“These results show that special stem cells injected into the brain were safe and well-tolerated by people with secondary progressive MS.
“They also suggest this treatment approach might even stabilise disability progression.
“We’ve known for some time that this method has the potential to help protect the brain from progression in MS.
“This was a very small, early-stage study and we need further clinical trials to find out if this treatment has a beneficial effect on the condition.
“But this is an encouraging step towards a new way of treating some people with MS.”
Produced in association with SWNS Talker
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