Stem cells have the potential to develop into cells with special functions such as the nerve cells in the brain or the muscle cells of the heart. It is this potential that is a reason for researching their use in MS, as they might be able to repair damage to the nervous system or boost the immune system to prevent damage from happening.
Hematopoietic stem cell transplantation (HSCT) is a type of stem cell therapy that is being investigated as a treatment for MS. It is usually an aggressive and risky procedure that requires the immune system to be killed off prior to stem cells being transplanted. This study investigated a less intense form for HSCT treatment, where different drugs were used which only reduce the number of cells in the immune system making the person less vulnerable to infections and complications.
151 people with relapsing remitting MS (RRMS) or secondary progressive MS (SPMS) received HSCT treatment at the Northwestern University in the USA, between 2003 and 2014. The participants went through several tests and assessments before HSCT and for up to five years after treatment.
The study found that the HSCT treatment procedure used, appeared to be safe and after treatment there were significant improvements in disability as measured by the EDSS, where scores were an average of 4 before treatment and an average of 3, six months after treatment. People with RRMS and who were within 10 years of their diagnosis, were those who had a significant improvement in their EDSS score. Improvements were not seen in people with SPMS or who had lived with MS for longer than 10 years.
This study appears to show that HSCT treatment can improve disability in a subgroup of people with MS. The results are encouraging but there are a number of limitations to the study that should be taken into account when considering the results. The authors conclude that although the results suggest HSCT could improve disability, the findings are only preliminary and there is not sufficient evidence from this small study alone to confirm that it would be an effective treatment. Larger and randomised controlled trials would be needed to confirm the results.
Stem cells are different from all other kinds of cells in the body as they are unspecialised, which means they are not already dedicated to having a specific function in the body. They have the potential to develop into cells with special functions such as the nerve cells in the brain or the muscle cells of the heart. It is this potential that is a reason for researching their use in MS, as they might be able to repair damage to the nervous system or boost the immune system to prevent damage from happening.
Hematopoietic stem cell transplantation (HSCT) is a type of stem cell therapy that is being investigated as a treatment for MS. It is designed to reset the immune system, rather than just suppress it, as some current drugs used in the treatment of MS do.
The usual form of HSCT used is an aggressive procedure that involves several stages: first the stem cells are collected from the person with MS. These are either collected directly from their bone marrow using a needle and syringe to suck the cells from the bone marrow in the hip while under general anaesthetic. Or the person is given a drug that encourages the stem cells to leave the bone marrow and enter into the blood so they can be collected in a blood sample. The stem cells are then frozen. The person with MS is given high dose chemotherapy and sometimes radiotherapy to kill off their existing immune system. Their own stem cells are then defrosted and injected into the person with MS, where they multiply and recreate the immune system.
This study investigated a less intense form for HSCT treatment, where instead of killing off the immune system completely prior to transplantation, different drugs were used which only reduce the number of cells. This reduces the risk of person receiving the transplant contracting serious infections and not being able to fight them off.
How this study was carried out
151 people with MS received HSCT treatment at the Northwestern University in the USA, between 2003 and 2014. 123 had relapsing remitting MS (RRMS) and 28 had secondary progressive MS.
145 participants completed the study and all of the follow up assessments. All were aged between 18 and 60 years old, with an average age of 36 years old, 85 were female and 60 were male.
The participants were assessed before HSCT and six months after, then every year for up to five years. They completed several questionnaires and tests that assessed how their MS affected them, their EDSS score was determined and they had MRI scans taken.
What was found
The study found that the HSCT treatment resulted in a significant improvement in EDSS score from an average score of 4 before treatment to approximately a score of 3 six months after treatment. EDSS scores decreased for the group from the point of transplant up to the fifth year of assessment. Just over half (51%) of the participants had an improvement in their EDSS score of 1 or more, two years after treatment. After four years 64% of participants had improved EDSS scores and 80% had been without a relapse.
After treatment the participants also had less lesions on their MRI scans, had improvements in their physical health, mental health and quality of life.
The less intense HSCT treatment procedure also appeared to be safe to use, none of the participants died and there were few infections.
When the researchers compared different groups they found that people with RRMS and who were within 10 years of their diagnosis, were those who had a significant improvement in their EDSS score. Improvements were not seen in people with SPMS or who had lived with MS for longer than 10 years.
What does it mean?
This study appears to show that HSCT treatment can improve disability in a subgroup of people with MS, as seen through reduced EDSS scores, as well as lead to improvements in other measures, including number of relapses and lesions seen on brain scans. The results suggest treatment at an earlier stage works best as the procedure used did not appear to have the same positive effect on people with SPMS.
The results are encouraging and the authors do state that they believe that this is the first study to report a "significant and sustained improvement in EDSS score following any treatment for MS". However there are a number of limitations to the study that should be taken into account when considering the results. All of the participants were treated at the same location and there was no control group to compare them to, so how these results would apply to the general MS population around the world is not yet clear. Additionally many of the participants were treated with HSCT as they had not responded to other drug treatments and they had continued to have relapses. It may be that the improvement in function and EDSS score seen in the study was as a result of them continuing to recover from their earlier relapses and not as a consequence of the stem cell treatment. The study also only followed the participants for up to five years after the HSCT treatment, and only 27 participants were assessed at the five year point and so long term effects of treatment were not really studied.
The authors conclude that although the results appear encouraging and suggest HSCT could improve disability, the findings are only preliminary and there is not sufficient evidence from this small study alone to confirm that it would be an effective treatment. Larger and randomised controlled trials would be needed to confirm the results.
Burt RK, Balabanov R, Han X, et al.
Association of nonmyeloablative hematopoietic stem cell transplantation with neurological disability in patients with relapsing-remitting multiple sclerosis.
JAMA. 2015 Jan 20;313(3):275-84.
More about stem cells
Stem cells are naturally occurring cells that differ from other cells in the body, as they are unspecialised, which means they are not already dedicated to having a specific function in the body. They have the potential to develop into different types of cell and it is this which is driving the scientific interest in their use as a possible therapy in multiple sclerosis.
Three possible theories as to how stem cell treatment might work in MS are being explored, stem cells might be persuaded to:
- Develop into nerve cells to repair damage to the brain
- Develop into oligodendrocytes to repair damage to myelin
- Boost the immune system to prevent damage
Although work with stem cells is still at a very early stage, there have been small clinical trials in people with MS in both Europe and the United States.
Clinical trials in progress
Currently there are a number of stem cells trials ongoing in the UK. This includes a study part-funded by the MS Trust into a stem cell therapy at Frenchay Hospital in Bristol that aims to reduce the increase in disability that is commonly seen in people with a progressive form of multiple sclerosis. This trial started in 2012 and results are expected to be announced in 2017.
Interest in stem cell research is high and unfortunately, as the trials are only small scale at the moment, any opportunities are always massively over-subscribed. The MS Trust is not aware of any opportunities to get involved in stem cell research at the moment.
Stem cell treatments are being offered commercially on the internet, as are 'clinical trials' where the participants are expected to pay for treatment, these sites should be approached with great caution. In some instances, clinics have been found to be offering expensive treatments with no evidence that they work or are safe and providing little in the way of follow-up support for customers. The MS Trust is not currently aware of any ethical, authorised clinics using stem cells to treat MS outside clinical trials.
Hope or hype?
Stem cells do offer great potential but currently all stem cell treatments remain experimental and the consensus is that any treatment based on stem cells is still some years away. As well as studies to show that the stem cells can be used to repair or replace damaged parts of the body, a lot of work also needs to be done to ensure stem cells can be controlled and are safe to use.
Stem cell treatments for MS always seem to attract a great deal of media interest and not all of these reports are accurate accounts of what the studies have found. Prof Neil Scolding who is leading the stem cell research in Bristol wrote an article for Open Door examining stem cells, including the media's reporting of research. Sign up for Open Door here or call us on 0800 032 38 39 or 01462 476700
You can read more about MS research including how to tell good research from hype in our new information sheet Research and MS, which can be read online, downloaded as a PDF file or ordered as a printed version.
The NHS Choices website also has a helpful 'Behind the Headlines' section that takes a closer look at the research behind some of the top newspaper headings and examines what the study really found.
You can read more about stem cells in the A to Z of MS.
Research by topic areas...
Wolkorte R, Heersema DJ, Zijdewind I.
Muscle Fatigability During a Sustained Index Finger Abduction and Depression Scores Are Associated With Perceived Fatigue in Patients With Relapsing-Remitting Multiple Sclerosis.
Neurorehabil Neural Repair. 2015 Jan 20. [Epub ahead of print]
Causes of MS
Fabis Pedrini MJ, Seewann A, Bennett KA, et al.
Helicobacter pylori infection as a protective factor against multiple sclerosis risk in females.
J Neurol Neurosurg Psychiatry. 2015 Jan 19. [Epub ahead of print]
Disease modifying treatments
Clausi V, Giannecchini S, Magnani E, et al.
Markers of JC virus infection in patients with multiple sclerosis under natalizumab therapy.
Neurol Neuroimmunol Neuroinflamm. 2015 Feb;2(1):e58.
Read the full text of this paper
Salhofer-Polanyi S, Baumgartner A, Kraus J, et al.
What to expect after natalizumab cessation in a real-life setting.
Acta Neurol Scand. 2014 Aug;130(2):97-102.
Sanoobar M, Dehghan P, Khalili M, et al.
Coenzyme Q10 as a treatment for fatigue and depression in multiple sclerosis patients: A double blind randomized clinical trial.
Nutr Neurosci. 2015 Jan 20. [Epub ahead of print]
Deppe M, Marinell J, Krämer J, et al.
Increased cortical curvature reflects white matter atrophy in individual patients with early multiple sclerosis.
Neuroimage Clin. 2014;6:475-87.
Read the full text of this paper
Oh J, Sotirchos ES, Saidha S, et al.
Relationships between quantitative spinal cord MRI and retinal layers in multiple sclerosis.
Neurology. 2015 Jan 21. [Epub ahead of print]
Hubbard EA, Motl RW.
Sedentary behavior is associated with disability status and walking performance, but not cognitive function, in multiple sclerosis.
Appl Physiol Nutr Metab. 2014 Oct 28:1-4. [Epub ahead of print]
Guner S, Inanici F.
Yoga therapy and ambulatory multiple sclerosis Assessment of gait analysis parameters, fatigue and balance.
J Bodyw Mov Ther. 2015 Jan;19(1):72-81.
Read the full text of this paper
Motl RW, McAuley E, Sandroff BM, et al.
Descriptive epidemiology of physical activity rates in multiple sclerosis.
Acta Neurol Scand. 2015 Jan 18. [Epub ahead of print]
Pregnancy and childbirth
De Giglio L, Gasperini C, Tortorella C, et al.
Natalizumab discontinuation and disease restart in pregnancy: a case series.
Acta Neurol Scand. 2015 Jan 18. [Epub ahead of print]
Piccolo L, Kumar G, Nakashima I, et al.
Multiple sclerosis in Japan appears to be a milder disease compared to the UK.
J Neurol. 2015 Jan 22. [Epub ahead of print]
Symptoms and symptom management
Amtmann D, Askew RL, Kim J, et al.
Pain Affects Depression Through Anxiety, Fatigue, and Sleep in Multiple Sclerosis.
Rehabil Psychol. 2015 Jan 19. [Epub ahead of print]
Hughes AJ, Beier M, Hartoonian N, et al.
Self-Efficacy as a Longitudinal Predictor of Perceived Cognitive Impairment in Individuals with Multiple Sclerosis.
Arch Phys Med Rehabil. 2015 Jan 15. [Epub ahead of print]