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HSCT the truth

On 19 March, BBC Breakfast featured a short piece on a HSCT trial with relapsing remitting multiple sclerosis (RRMS) patients taking place in Sheffield. It provoked a strong reaction from the MS community and a lot of questions, so we’ve tried to answer some of them…SJL.png

What is it and how does it work?
A variety of clinics and hospitals across the world, including Sheffield and London are trialling and practicing HSCT treatment.

This particular Hematopoietic stem cell transplantation (HSCT) trial, which has been taking place in Sheffield, America, Sweden and Brazil, involves the patient having stem cells extracted from their bone marrow. Next they are given chemotherapy treatment, which strips back their immune system to almost that of a baby and then the healthy stem cells are transplanted back into their body.

The trial was set up to test the efficacy of HSCT treatment versus FDA approved MS drugs, such as interferon, glatiramer acetate, mitoxantrone, natalizumab, fingolimod, or tecfidera.

The findings
Just over 100 patients have taken part in the trial, in hospitals in Chicago, Sheffield, Uppsala in Sweden and Sao Paulo in Brazil.

Scientists conducting the research claim they have made a significant breakthrough with this type of treatment in patients with highly active relapsing remitting multiple sclerosis (RRMS).

Patients received either HSCT or drug treatment. After one year, only one relapse occurred among the stem cell group compared with 39 in the drug group.

After an average follow-up of three years, the transplants had failed in three out of 52 patients (6%), compared with 30 of 50 (60%) in the control group.

Those in the transplant group experienced a reduction in disability, whereas symptoms worsened in the drug group.

The interim results were released at the annual meeting of the European Society for Bone and Marrow Transplantation in Lisbon.

Click here to read the study’s abstract - Hematopoietic Stem Cell Therapy for Patients With Inflammatory Multiple Sclerosis Failing Alternate Approved Therapy: A Randomized Study.

What is the inclusion criteria?
Participants have to be aged 18-55 and have a clinically defined MS diagnosis using the revised McDonald criteria.

Their Expanded Disability Status Score (EDSS) should be 2.0 to 6.0.

The must show inflammatory disease despite treatment with standard disease modifying therapy, including at least six months of interferon or copaxone.

Inflammatory disease is defined based on both MRI (gadolinium enhancing lesions) and clinical activity (acute relapses *treated with IV or oral high dose corticosteroids and prescribed by a neurologist). Minimum disease activity required for failure is defined as: a) two or more *steroid treated clinical relapses with documented new objective signs on neurological examination documented by a neurologist within the year prior to the study, or b) one *steroid treated clinical relapse within the year prior to study and evidence on MRI of active inflammation (i.e., gadolinium enhancement) within the last 12 months on an occasion separate from the clinical relapse (three months before or after the clinical relapse).

A steroid treated relapse will include a relapse that was severe enough to justify treatment but due to patient intolerance of steroids, or a history of non-response to steroids, they were offered but not used.

More information about inclusion and exclusion criteria can be found here.

Can I get on the trial?

Unfortunately you cannot. This is because although the trial is still active they are not recruiting.

Will it really be available on the NHS within a few months?
Dr Susan Kohlhaas, director of research at the MS Society, said the stem cell transplant HSCT "will soon be recognised as an established treatment in England”, but will it?
While this is a phase III trial testing the efficacy of the HSCT, which will be incredibly significant when it comes to gaining licensing approval, the treatment has only been formally assessed for use in the NHS within clinical trials.

There will also be a few more hoops to jump through, such as gaining approval from NICE (The National Institute for Health and Care Excellence). Although NICE does now have a fast track criteria, which enables certain drugs and treatments with the right evidence to pass through the system much quicker than we have seen in the past. Cost will almost definitely be a deciding factor. HSCT comes with a price tag of £30,000, but there are already some approved DMTs with a similar costing available to patients, so this could help justify the expenditure, especially if the treatment can halt the MS for a long period of time. We should also be mindful that a higher price point can often lead to drugs and treatments being allocated to minorities with strict criteria, rather than being rolled out for everyone.

When does the trial end?
The trial is still ongoing and its estimated end date is December 2018.

Is it really a “game changer”?
Well, it’s a great step forward for people with RRMS and it does mean there is a potential highly-successful treatment that could halt MS in its tracks on the horizon.
However, HSCT treatment in secondary and primary progressive patients doesn’t tend to be as effective and you tend to see less improvement in disability because the nerve damage by this point has become permanent.

There are still a number of questions we do not have the answer to, such as how long does the treatment last?

But maybe the biggest questions of all is if MS is genetic, the person will still have the same gene and what’s to stop the gene being triggered again and the MS returning if we do not know the true cause?

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