Injecting stem cells into the brains of patients with multiple sclerosis (MS) is safe and may stop the disease causing further damage, according to an early-stage clinical trial.
An international team of scientists from the UK, Italy, Switzerland and the USA directly injected neural stem cells into the brains of 15 patients with secondary progressive MS. Throughout the 12-month follow-up period, the treatment caused no serious adverse events, with only temporary or reversible side-effects reported.
Professor Stefano Pluchino from the University of Cambridge, who co-led the study, said: '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.'
In a healthy brain, a subset of immune cells called microglial cells protects the brain from infection and removes damaged neurons. When MS occurs, microglial cells attack and damage myelin, the sheath that protect nerve fibres, disrupting vital signalling between the brain and the spinal cord, causing chronic inflammation.
Over two million people worldwide have MS, with two-thirds of patients eventually advancing to the secondary progressive stage of the disease. During this stage neurologic function worsens and disability increases.
During the first-in-human, early-stage clinical trial, all patients participating showed high levels of disability, with most requiring a wheelchair. During the 12-months following treatment no patients reported any increase in disability or symptoms deterioration.
Furthermore, patients' brain scans revealed those who received higher dose of stem cells experienced less brain shrinkage, which the researchers suggesting this was due to the transplanted stem cells dampening the autoimmune response and reducing inflammation that drives MS, rather than rebuilding the damaged tissues. These patients also had higher levels of metabolite compounds called acylcarnitines in their cerebrospinal fluid, which are associated with neuroprotection.
The findings, published in Cell Stem Cell, point to a 'substantial stability of the disease, without signs of progression, though the high levels of disability at the start of the trial make this difficult to confirm'.
This current study used neural stem cells from the brain tissue of a miscarried fetal donor. However, the researchers hope to be able to derive stem cells directly from the patient in future studies to avoid complications associated with the use of fetal tissue. Stem-cell transplantation using patients' own stored stem cells has been shown effective in another trial on patients with early, relapsing-remitting MS (see BioNews 1210).
The scientists involved in this current study recognise the trial's limitations. Professor Pluchino explained 'it was only a small study and there may have been confounding effects from the immunosuppressant drugs, for example,'. The patients required these drugs before transplantation to prevent their immune systems rejecting the transplanted stem cells.
'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.' Caitlin Astbury, research communications manager at the MS Society, commented '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.'
Sources and References
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Early-stage stem cell therapy trial shows promise for treating progressive MS
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Phase I clinical trial of intracerebroventricular transplantation of allogeneic neural stem cells in people with progressive multiple sclerosis
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Doctors encouraged by early-stage trial of MS stem cell therapy
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MS could be cured by injecting stems cells into brain, Cambridge scientists find
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Multiple sclerosis stem cell treatment breakthrough could stop disease in its tracks, study finds
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Stem cell therapy shows promise for treating multiple sclerosis – new study
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