Groundbreaking stem cell therapy for multiple sclerosis recommended for use
Giving hope to thousands of people with Multiple Sclerosis, the Scottish Health Technologies Group (SHTG) has announced that is believes there is now sufficient evidence for it to recommend making ground-breaking HSCT available on the NHS in Scotland.
Treatment would be offered to patients who have the relapsing-remitting form of the disease – where relapses last at least 24 hours – and who were not responding to treatments.
Lucy Clarke from the Scottish HSCT Network said the recommendation was “a huge step forward” for people. The Scottish Health Technologies Group (SHTG) is a national health technology assessment agency. They provide evidence, support and advice to NHSScotland on the use of new and existing health technologies which are not medicines and which are likely to have significant implications for people’s care.
SHTG independently examine health technologies; these might be working as an intervention, product or service developed to prevent, diagnose or treat medical conditions; or promoting health; providing rehabilitation; or organising healthcare delivery. The health technology could be a test, device, procedure or patient pathway.
The assessments that SHTG carry out are multidisciplinary processes that use explicit methods to assess the value of a health technology at points in its lifecycle. The process is comparative, systematic, transparent and involves multiple stakeholders. The purpose is to inform health policy and decision-making to promote an efficient, safe, sustainable, equitable and high-quality health system. You can find out more SHTG here: http://www.healthcareimprovementscotland.org/
Lucy Clarke underwent HSCT in Russia and credits it with substantially reversing her disability, she said; “This important decision supports HSCT as a treatment option where other treatments have failed. We will continue to push so that this treatment is available to people in Scotland who need it.”
Scotland has one of the highest rates of MS in the world. Until now Scottish patients seeking HSCT have had to travel overseas while bankrolling their own private treatment at a cost of around £40,000 to £60,000. It is also available privately in London but costs around £100,000.
Haematopoietic stem cell transplantation (HSCT) is an intense chemotherapy treatment for MS. It aims to stop the damage MS causes by wiping out and then regrowing your immune system, using your stem cells. Results have shown that HSCT is most effective for people with the following symptoms; With signs of active inflammation, as seen by frequent relapses alongside new or active lesions on an MRI scan; Who are early on in their disease course; Without
The Multiple Sclerosis Society recommends that it will mainly include people with highly active relapsing MS who are still having relapses despite taking disease modifying therapies (DMTs).
If someone has progressive MS and still has active inflammation (either relapses or lesions on an MRI), HSCT may be able to stop or slow this.
But HSCT can’t regrow nerves or repair damaged myelin, so it can’t help those with advanced progressive MS who are no longer having relapses and don’t shown signs of inflammation on an MRI.
Researchers are working hard to find effective treatments for people with progressive MS.
HSCT aims to ‘reset’ the immune system to stop it attacking the central nervous system. It uses chemotherapy to remove the harmful immune cells and then rebuilds the immune system using a type of stem cell found in your bone marrow, called haematopoietic stem cells.
The stem cells used in the treatment can produce all the different cells in your blood, including immune cells. But, they can’t regenerate damaged nerves or other parts of the brain and spinal cord.
Iain Robertson, chairman of the SHTG, said; “Our committee members were able to advise that this treatment should be considered for those with this particular type of MS who have not responded to treatment with disease modifying therapies. We hope that our advice will be of use in helping decide the best course of treatment for these patients. It uses chemotherapy to wipe out patients’ “faulty” immune systems before replenishing it with a transplant of stem cells harvested from their own bone marrow. It puts patients at high risk from infections, which can be fatal.”
But the theory is that the treatment works by enabling patients to “reset” their immune system to stop it attacking the central nervous system – as is the case in MS.
HSCT is not considered an effective treatment for patients with the progressive form of MS.
However, as stem cells cannot regrow nerves or repair damaged myelin – the protective sheath coating nerves.
It will also be unavailable to patients with relapsing-remitting MS who no longer show signs of inflammation on an MRI brain scan.
Morna Simpkins, director of MS Society Scotland, said: “The decision from SHTG to approve HSCT for the treatment of MS is good news and could help in the development of a clear pathway, for people who could potentially benefit, to access it. We will push to ensure this decision leads to real change for people with MS by continuing to engage with other groups to offer the treatments, including HSCT, which are right for them.”
The SHTG said eligible patients must have equal access to the procedures regardless of where they live, but it is unlikely all health boards will be able to provide it.