There has been a widespread welcome for news that a new therapy involving stem cell transplants has halted or reversed the symptoms of advanced multiple sclerosis in a small number of patients.
Scientists have hailed the new research in Canada as exciting and remarkable. But one patient died, and the decision on whether to have the treatment can be a difficult and costly one for MS patients.
Multiple sclerosis is a debilitating condition and for patients in the advanced stages, known as “progressive” MS, there is no cure or drug which even delays its development.
MS occurs when there is a fault with the immune system which mistakenly attacks the protective layer surrounding nerve fibres in the brain and spinal cord, known as myelin.
Stem cell therapy
A therapy known as HSCT has been developed under which a patient’s stem cells are harvested followed by chemotherapy to wipe out the immune system.
The stem cells are then transplanted back into the body to reboot the immune system. It has proved effective on some patients in the early stages of MS.
But the latest study, published in The Lancet, is the first to suggest sustained improvement among some patients in the progressive phase, specifically people with highly active MS who had a poor prognosis and risk of significant rapid progression.
But one of the 24 patients being monitored died, and experts have warned that the aggressive form of chemotherapy creates significant risks for patients.
The scientists who welcomed the Canadian findings were quick to point out that there were safety concerns and that further studies with a larger sample size and control group were needed.
The MS Society is currently focusing its funding on finding disease-modifying treatments that can slow or stop disability progression by repairing myelin and protecting the nervous system against future damage.
Work on repurposing of existing drugs for other conditions such as depression and motor neurone disease, in a trial known as MS-SMART, is said to offer exciting potential. But translating this research into tested and approved drugs could take a few years yet.
Dr Emma Gray, head of clinical trials at the society, hailed the Canadian findings but noted that the treatment was “an aggressive procedure with substantial risks and requires specialist aftercare”.
She urged patients considering the HSCT to consult their neurologist.
And here’s the dilemma. HSCT is not widely available in the UK and many neurologists won’t recommend it. They regard it as unproven and with known risks which are not balanced by predictable benefits.
So patients with progressive MS have tough choices to make.
They can go to foreign clinics, for example in Israel, Mexico and Russia and take their chances with a risky therapy which does not come cheap and in clinics which may be unregulated. But for someone with progressive MS it at least offers the only chance of stabilising their condition.
The alternative to going down the HSCT route is to wait and hope that new treatments will emerge and that the potential now being discussed is realised. But this could take years and the wait will inevitably see continuing debilitation with worsening symptoms.
Much will depend now on funding for more extensive trials, and obtaining that will not be straightforward.
There are hopes that the government may provide more finance for research in the UK.
For those patients with progressive MS, who never had the chance to take drugs recently made available for those in the early stages of the condition, any sort of breakthrough can’t come a moment too soon.