Stem cell therapy trial shows 'safe and promising' early results for MS treatment

A 3D illustration compares normal nerves to ones affected by MS.
A 3D illustration compares normal nerves to ones affected by MS. Copyright Canva
By Oceane Duboust
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Scientists had promising results in the preliminary stage of a neural stem cell therapy trial for progressive multiple sclerosis.

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The injection of stem cells into the brains of patients living with multiple sclerosis (MS) could help protect the brain from further damage, researchers have found in the early stage of a new first-in-human clinical trial.

MS is a chronic condition that affects the central nervous system, which includes the brain and spinal cord.

This can lead to a variety of symptoms, such as fatigue, difficulty walking, numbness or tingling, muscle weakness, and problems with coordination and balance.

These can vary widely from person to person, and the symptoms may come and go or progress over time. There is no known cure for MS.

"We desperately need to develop new treatments for secondary progressive MS, and I am cautiously very excited about our findings, which are a step toward developing a cell therapy for treating MS," Stefano Pluchino from the University of Cambridge, who co-led the new study, said in a statement.

Led by researchers at the University of Colorado Anschutz, the University of Cambridge and the University of Milano-Bicocca, the first phase of a trial involving 15 patients showed promising early results.

The researchers injected neural stem cells directly into the patients’ brains and followed them for a year. The findings were published in the journal Cell Stem Cell.

Stem cells may mitigate damage

When someone has MS, the immune system mistakenly attacks the protective covering of nerve fibers called myelin which disrupts the normal flow of electrical impulses along the nerves.

In this process, macrophages - a type of white blood cell - are key as they typically fend off and eliminate undesirable invaders within the body.

Specifically, a type of macrophage called a microglial cell targets the central nervous system, causing persistent inflammation and harming nerve cells in progressive manifestations.

There has been increased optimism about the potential of stem cell therapies to mitigate such damage.

The stem cells for this first human trial came from a single, miscarried foetal donor.

'Safe but also promising'

Although certain side effects were noted, they were either temporary or reversible. Moreover, none of the patients, who had high levels of disability at the start of the trial, showed a worsening of symptoms.

This breakthrough approach turned out not only to be safe but also promising, with respect to the effects observed through medical exams.
Angelo D’Alessandro
Professor, Univerisity of Colorado’s School of Medicine

"This breakthrough approach turned out not only to be safe but also promising, with respect to the effects observed through medical exams and, as data generated in our lab suggest, molecular markers of neuroinflammation," said Angelo D’Alessandro, a professor at the Univerisity of Colorado’s School of Medicine.

Researchers also found that the larger the dose of injected stem cells, the smaller the reduction in brain volume over time. This may be because the stem cell transplant dampened inflammation, they speculated.

"We recognise 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," Pluchino added.

The phase 1 trial marks only an initial step on the journey toward clinical application. To establish the safety, efficacy, and broader applicability of this innovative approach, further research is needed.

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