Genetics and stem cells

MS stem cell therapy trialled

“A controversial trial using bone marrow stem cell therapy for MS patients has helped stabilise the disease,” reported the Daily Mail .

The research was a phase I clinical trial in six people with chronic multiple sclerosis (MS), investigating whether it was safe to treat them with stem cells from their own bone marrow. Though the newspaper described the trial as controversial and mentions a commercial service that offers stem cells from umbilical cords, this service is seemingly unrelated to this research, and it is unclear where the controversy might stem from.

The results were promising in that there were no serious side effects up to a year after treatment. Although the patients’ deterioration appeared to halt over this period, there was no control group and the trial involved only six people. It is therefore too early to know how effective it is.

Despite being preliminary, these results are encouraging and need further investigation. The next stage would be to test the treatment in a larger patient population, comparing it to a placebo or an existing treatment to see whether it stabilises or improves MS symptoms over time.

Where did the story come from?

The study was carried out by researchers from the University of Bristol and Imperial College London. Various charitable trusts funded the research. The study was published in the peer-reviewed medical journal Clinical Pharmacology and Therapeutics.

The BBC and The Daily Telegraph covered the story well, highlighting the preliminary nature of the trial. The Daily Mail suggests that this was a “controversial stem cell trial” and refers to a commercial service in Rotterdam that offers cells taken from umbilical cords. The stem cells in this study came from the patients' own bone marrow. However, this point is not made clear in the news report, and it seemingly removes any controversy from the research.

What kind of research was this?

This was a phase I clinical trial investigating whether it is safe and feasible to give people with multiple sclerosis (MS) bone marrow stem cell therapy. MS is the most common neurological condition among young adults in the UK, affecting approximately 85,000 people. The condition affects the central nervous system (the brain and spinal cord), which controls the body's actions and activities, such as movement and balance.

Each nerve fibre in the central nervous system is surrounded by a substance called myelin. Myelin helps the messages from the brain travel quickly and smoothly to the rest of the body. In MS, the myelin becomes damaged, which disrupts the transfer of these messages.

Myelin is made by a type of brain cell called an oligodendrocyte. Bone marrow contains stem cells that can develop into brain cells. Research in animal models of MS indicates that bone marrow stem cells encourage the repair of myelin and help to prevent the loss of oligodendrocytes and damage to nerve cells. Bone marrow stem cell therapy has been used in patients for other conditions.

What did the research involve?

The study recruited six people who had chronic multiple sclerosis for more than five years. Bone marrow was harvested from the patient’s pelvis under general anaesthetic. The bone marrow (containing a mixture of cell types including stem cells) was then filtered and transfused back into the patient over one to two hours.

The patients were followed-up for 12 months after the transfusion. Their disease progression was assessed using the Extended Disability Status Score (EDSS) and the MS Functional Composite, which looks at factors such as the patients’ walking speed and their dexterity.

The patient’s brainwave patterns were also examined using electrophysiological electrodes placed on the surface of the patient’s scalp. This looked at how quickly the brain responded to visual, auditory and tactile stimuli (people with MS may have a longer interval between the stimulus and the brain response, indicating that the nerve signals are being impeded). MRI scans were also taken to count the number of lesions (areas where the nerve cells did not have myelin insulation).

The patients were also assessed to see whether the treatment had any side effects.

What were the basic results?

None of the six patients experienced any severe side effects. However, three patients had moderate side effects, such as a temporary increase in the spasticity of their legs and a temporary inability to pass urine.

One patient had a relapse of MS within two months of the treatment, which was resolved when treated with steroids. The other five patients showed no sign of disease progression, and their EDSS disability score remained unchanged. Improvements were seen in the MS Functional Composite but these changes were not significant.

The recordings of brainwave patterns showed an improvement compared to pre-treatment. This improvement was apparent within three months of the treatment (p=0.07) and was maintained at one year after the treatment (p=0.02).

There was a trend for an increased number of lesions at three weeks, but this was not statistically significant, and the trend disappeared by three months after treatment.

How did the researchers interpret the results?

The researchers suggest that the procedure, carried out as a day-case treatment in people with MS, was well-tolerated and not associated with any serious adverse effects. They say that “these results are preliminary evidence of the “possible benefit of bone marrow cellular therapy in patients with MS”. The researchers say that these findings warrant further investigation in a randomised placebo-controlled phase II/III clinical trial, with a longer follow-up period.


This very preliminary research has demonstrated that transfusing filtered bone marrow cells into people with MS did not cause serious side effects in this small group of six patients. The patients’ disability remained stable, and there was an improvement in their brain response to stimuli compared to before the treatments.

This study did not compare the stem cell treatment to a placebo group, and it was conducted in a very small group of individuals. As such, it is not possible to say whether these changes are due to the treatment, or if they would have happened anyway. It is also possible they are due to a placebo effect or they simply happened by chance.

Despite being at a preliminary stage, these results are encouraging and need further investigation. The next stage would be to carry out a further safety assessment in a larger patient population, and to compare the treatment to a placebo or an existing treatment to see whether it stabilises or improves MS symptoms over time.

NHS Attribution