AHSCT is being investigated as a treatment for severe immune-mediated diseases particularly MS, here at Sheffield Teaching Hospitals NHS Foundation Trust.
The procedure involves removing the harmful immune system cells that attack the brain and spinal cord, and then uses a person’s own bone marrow stem cells to effectively re-grow these immune system cells. The aim is that re-booting the immune system in this way will prevent further damage in the brain and spinal cord.
The aim of AHSCT is to ‘reset’ the immune system to stop it from attacking the body. Haematopoietic stem cells are not the type of stem cells that would be expected to change into or regenerate permanently damaged parts of the brain and spinal cord.
The AHSCT treatment uses high doses of chemotherapy and monoclonal antibody treatments, and therefore is more intensive and higher risk than most other MS treatments and involves a number of steps:
Haematopoietic stem cells (HSCs) are a type of adult stem cell made in the bone marrow, which have the ability to produce the different cells found in the blood. AHSCT is being investigated as a treatment for severe immune mediated diseases, including MS. The aim is to remove the harmful immune cells that attack the brain and spinal cord, and then re-boot the immune system using a person’s own (autologous) stem cells.
Typically, the person receiving a transplant will be closely observed for about three to four weeks in an isolation room whilst receiving antibiotics and transfusions to support them through the procedure. Prior to any treatment decisions being made, our specialist Consultant Haematologists and Neurologists will carefully assess the general health of the person receiving treatment and their ability to tolerate this type of treatment.
Clinical trials conducted so far suggest that AHSCT may be able reduce relapses and to stabilise or reduce the level of disability for some people with relapsing remitting multiple sclerosis.
People with highly active relapsing-remitting MS seem to have the best response to the treatment, but results are varied and it may not be effective for all types of MS, especially primary and secondary progressive MS.
The most recent study showed that in 123 people with relapsing remitting multiple sclerosis, AHSCT was linked to reduction in level of disability in 64% of the people treated. 80% of the people treated who were followed for 4 years had no more relapses and 87% had no worsening of their level of disability. Participants reported an improved quality of life and had a reduced number of new brain lesions on MRI scans
Unfortunately in this study, HSCT was not effective for the 28 people with secondary progressive multiple sclerosis or those who had had relapsing remitting multiple sclerosis for more than ten yearsMore information on HSCT
AHSCT is an aggressive therapy involving intensive chemotherapy and its short term risks are higher than other MS therapies. Following AHSCT for any disease (including blood cancers), a small proportion of patients may be severely unwell and even require intensive care support.
In Sheffield Teaching Hospitals most patients are told that the procedure may expose them to potentially life threatening complications and, to reflect this degree of risk, are informed of the department’s current treatment related mortality rate of 1% across all indications.
However, there has been no mortality related to the use of this treatment specifically for MS in Sheffield Teaching Hospitals NHS Foundation Trust. Generally, the level of risk of AHSCT in MS depends on the patient (and their fitness), the experience of the institution and treatment regimen used. We advise patients to discuss these aspects with their neurologist before referral, and, if referred to a transplant centre, be ready for detailed discussions with a haematologist.
If you are thinking about undergoing AHSCT, it is important that it is administered in a highly regulated environment where safety is assured.
Any UK centre performing transplantations for MS or any other condition, must have a Joint Accreditation Committee-ISCT & EBMT (JACIE) certificate. In order to get a certificate, the centre must comply with a huge number of quality standards which are subject to regular inspection and audits.
There are also long term complications associated with AHSCT and if you are receiving the treatment outside of an accredited centre you may not receive the necessary aftercare.
Sheffield Teaching Hospitals NHS Foundation Trust is a JACIE accredited transplant centre and meets all the required quality standards. The Trust has an established MS service and is highly regarded in the field of the treatment and research.
Unfortunately the trials performed to date show that AHSCT does not work as well in primary and secondary progressive multiple sclerosis. In view of this data, at Sheffield Teaching Hospitals NHS Trust we are only treating people with relapsing remitting multiple sclerosis.
There are a number of very effective treatments for relapsing remitting multiple sclerosis (disease modifying drugs) which reduce relapses (sudden worsening of your neurological symptoms). If you and your neurologist or MS nurse feel that your relapses are controlled on your current treatment, then we would not recommend AHSCT. If you do not feel that your relapses are well controlled then it would be important to discuss this with your neurologist or MS nurse, but it may be that another treatment would be preferable.
If you and your neurologist feel that you have primary or secondary progressive multiple sclerosis it is also very unlikely that AHSCT will be helpful for you.
A small number of people have been treated at Sheffield Teaching Hospitals NHS Foundation Trust with AHSCT outside of a clinical trial setting. These were people with very aggressive relapsing remitting multiple sclerosis, who had very frequent and severe relapses which could not be controlled with disease modifying drugs, who we think have the best chance of success with this treatment.
If you are interested in AHSCT you should speak to your neurologist before contacting the team at Sheffield Teaching Hospitals NHS Foundation Trust. The procedure is high risk so if your neurologist is advising against AHSCT it may be because it is unlikely to be effective in your circumstances based on current research.
If you are considering a different centre for the treatment, please remember that any centre performing transplantations, for MS or any other condition, should have a Joint Accreditation Committee-ISCT & EBMT (JACIE) certificate. If you are referred by your neurologist to receive AHSCT, you should ask them about their experience of the centre and make sure it has the necessary accreditation.
Currently AHSCT is administered as a one off treatment. We are in need of longer duration studies that follow up on people that have received AHSCT in order to determine the long term effects of the treatment. As AHSCT is a relatively new treatment in MS, this data doesn’t currently exist so we don’t know whether AHSCT would need to be repeated down the line.
When administered in a clinical trial or otherwise through the NHS, AHSCT does not have any financial cost to the recipient.
We would strongly encourage people with MS thinking about undergoing AHSCT to discuss this carefully with a specialist healthcare professional such as their neurologist first to weigh up the potential risks and benefits.
If you are thinking about undergoing AHSCT, it is important that it is administered in a highly regulated environment. The gold standard of this is to be a part of a clinical trial which will be highly regulated and monitor participants long term. In the UK there is currently a clinical trial called MIST recruiting participants.
If you are thinking about participating in a clinical trial, it is important to consider and understand exactly what is involved, as well as thinking about the risks and benefits.
In some carefully assessed cases, including here at Sheffield Teaching Hospitals NHS Foundation Trust, people have also been treated on the NHS at a JACIE accredited centre outside of a clinical trial.
Prior to any treatment decisions being made a haematologist and neurologist will carefully assess the general health of the person receiving the transplant and their ability to tolerate this type of treatment.
In the UK the treatment has been delivered here at Sheffield Teaching Hospitals NHS Foundation Trust and King’s College Hospital, London and some other centres. However, before contacting these Centres you should discuss the treatment with your own Neurologist of GP in the first instance as the treatment is not suitable for everyone.
We have a limited capacity for overseas & fee paying patients. To discuss the possibility of this option, please contact our dedicated private patient team in the first instance.
Please get your GP or Neurologist to send a written referral to using the relevant referral forms and process found below:
Criteria will vary between trials but the MIST trial that is currently recruiting for people in the UK lists a number of inclusion and exclusion criteria on its clinical trial webpage.
If you have been excluded from the trial it is likely that you do not meet the study criteria. This could be as a result of a number of factors including your age, the type of MS that you have, your current level of disability (EDSS score), the number of relapses that you had over the previous 12 months and other health problems you may have.
As it is a high risk procedure, if the benefits are unlikely to outweigh the risks this must be taken into consideration when recruiting participants. As a result, clinical trials are often restrictive due to the criteria that have to be put in place.
HSCT is an aggressive treatment involving chemotherapy that carries risks and so it is important that it is administered in a highly regulated environment. Any centre performing transplantations, for MS or any other condition, should have a Joint Accreditation Committee-ISCT & EBMT (JACIE) certificate or equivalent.
Undergoing this treatment outside an accredited setting, including overseas units, can have additional implications for your health. If your neurologist is advising against AHSCT, it may be because it is unlikely to be effective for you. There are also long-term complications associated with HSCT and if you are receiving the treatment outside of an accredited centre you may not receive the necessary aftercare.
Until recently there has not been much evidence around the effectiveness and safety of AHSCT in people with MS. Following some small early studies, hospitals in Europe and USA that offer AHSCT including Sheffield Teaching Hospitals NHS Trust have monitoring people with MS who have AHSCT. It has only been in the past few months that results from some of these studies have been reported.
Further information about research into Autologous Haematopoietic Stem Cell Transplantation (AHSCT) can be found by visiting the MS Society page about AHSCT or the MS Trust page about the results of stem cell clinical trials in MS