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MS Stem Cell Therapy Shows Promising Results In Early-Stage Clinical Trial

The trial was small-scale but illustrates the potential of treating the disease with stem cell therapy.

Holly Large - Editorial Assistant

Holly Large

Holly Large - Editorial Assistant

Holly Large

Jr Copy Editor & Staff Writer

Holly is a graduate medical biochemist with an enthusiasm for making science interesting, fun and accessible.

Jr Copy Editor & Staff Writer

close up of a person using a wheelchair

Patients with MS injected with neural stem cells showed no worsening of symptoms over the following year.

Image credit: Freedom Studio/

An early-stage clinical trial of an injectable stem cell therapy for multiple sclerosis (MS) has shown that the treatment is not only safe, but may also show promise in stopping the disease from progressing.

The trial recruited 15 patients with secondary MS – the progressive phase of the disease – aged from 38 to 57 years old, all of whom showed high levels of disability as a result of their condition at the start of the trial. The patients then had neural stem cells injected directly into their brains, and over the next 12 months, were carefully observed for any side effects, change in symptoms, and disease progression. They also had to take drugs to suppress their immune systems for half of the follow-up period.


The following year revealed that the treatment was safe and well-tolerated; there were no deaths or serious adverse events and when side effects did occur, they were either temporary or reversible. The main result, however, was that none of the patients showed an increase in disability or a worsening of symptoms.

Whether this was solely down to the neural stem cells needs to be confirmed by further trials, but the researchers believe their other observations suggest that the treatment may well impact disease progression. For example, some patients were assessed for a reduction in brain volume that’s well-associated with disease progression; the researchers found that the larger the dose of stem cells, the smaller this reduction was over time. 

The team also monitored changes in the brain’s metabolism – aka how it produces energy – over the 12 months, as previous research had found that altering metabolism could reprogram the immune cells that attack the central nervous system in MS. They found signs that the stem cell therapy had affected metabolism and thus may have had an anti-inflammatory effect; the higher the dose of stem cells, the greater the levels of fatty acids, which are key molecules in metabolism. 

“I am cautiously very excited about our findings, which are a step towards developing a cell therapy for treating MS,” said Stefano Pluchino, who co-led the study, in a statement. The author also explained, however, that there is reason to be mindful when making conclusions about the results.


“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.”

It’s estimated that over 2 million people worldwide live with MS, of which two-thirds will move into the secondary, progressive phase of the disease within 25 to 30 years after their diagnosis. Whilst the current study is small, it demonstrates promise in stem cell therapy-based approaches to treating this highly debilitating disease.

“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,” concluded Caitlin Astbury, research communications manager at the MS Society.

The study is published in Cell Stem Cell.


healthHealth and Medicinehealthmedicine
  • tag
  • medicine,

  • brain,

  • stem cells,

  • multiple sclerosis,

  • MS,

  • stem cell therapy,

  • central nervous system