see www.sth.nhs.uk/panorama or call 0114 2715934.
The results look impressive, but in the programme there was limited discussion about the risk benefit profile. The Haematopoeitic stem cells will form new white blood cells and hopefully re-boot the immune system such that MS is gone.
However, their benefit is not via stem cells that form new myelin and nerves and the benefit observed is likely to be due to getting the immune response switched off and allowing the bodies compensation and repair capacity to improve function. Similar types of benefit can be seem with some of the other current DMT, when active disease is turned off.
The MS Society comments
AHSCT is an aggressive therapy and its short term risks are higher than other MS therapies. Typically, the person receiving a transplant will be closely observed for about a month in an isolation room whilst receiving antibiotics and transfusions to support them through the procedure. Prior to any treatment decisions being made a haematologist and neurologist at the HSCT centre will carefully assess the general health of the person receiving the transplant and their ability to tolerate this type of treatment.
We would strongly encourage people with MS thinking about undergoing AHSCT to discuss this carefully with a specialist healthcare professional, such as their neurologist or MS nurse 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.
Any centre performing transplantations, for MS or any other condition, must have a Joint Accreditation Committee-ISCT & EBMT (JACIE) licence. In order to get a licence, the centre must comply with a huge number of quality standards which are subject to regular inspection and audits. As well as having a JACIE licence, in the UK centres are highly regulated by the Human Tissue Authority (HTA) and the Medicines and Healthcare products Regulatory Agency (MHRA). 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 licencing.
It is important to note that undergoing this treatment outside an accredited setting, including overseas units, can have implications for your health as standards differ between centres. There is also an emotional cost to consider, if your neurologist is advising against AHSCT, it may be because it is unlikely to be effective in your circumstances based on current research. It can be disappointing and frustrating if the treatment does not deliver the results that were promised as well as financially damaging if you are paying for the treatment abroad. 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.
ABN statement on autologous haematopoietic stem cell treatment of multiple sclerosis
The Association of British Neurologists welcomes recent research into autologous haematopoietic stem cell treatment of multiple sclerosis. Despite many advances in the treatment of this disease, for some people it is disabling and life-limiting. New therapies which combine high efficacy with acceptable side-effects are certainly needed. However, as a recent commentary put it “the jury is still out regarding the appropriateness and indications of haematopoietic stem cell treatment for multiple sclerosis” (Soldán & Weinshenker, 2015).
We would encourage neurologists to support controlled trials of autologous haematopoietic stem in established transplant centres. At present, it does not have a place in the routine treatment of multiple sclerosis (see ABN Guidelines ).
Paz Soldán MM, Weinshenker BG. Moving targets for hematopoietic stem cell transplantation for multiple sclerosis. JAMA Neurol. 2015 Feb;72(2):147-9.
Snowden et al. Haematopoietic SCT in severe autoimmune diseases: updated guidelines of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant. 2012 Jun;47(6):770-90.
Mancardi et al. Autologous hematopoietic stem cell transplantation in multiple sclerosis: a phase II trial. Neurology. 2015 Mar 10;84(10):981-8.