Stem cell transplant trial ‘has miraculous effect’ on multiple sclerosis sufferers
Doctors are hailing a new stem cell treatment for the degenerative disease multiple sclerosis, after trials showed it to reboot patients’ immune systems, halting the disease.
Patients said the results were “a miracle” and had seen them return to normal life after the disease left them in a wheelchair or unable to read.
Around 100,000 people in the UK have multiple sclerosis, a condition where the immune system attacks the nerves of the brain and spinal cord causing problems with vision, movement, and balance.
Early results from a clinical trial run from four international centres show that wiping out the patients’ immune systems with chemotherapy, and restoring them with the new stem cell treatment, appears to halt the disease and improve symptoms.
“We are thrilled with the results – they are a game-changer for patients with drug-resistant and disabling multiple sclerosis,” Professor John Snowden, director of blood and bone marrow transplantation at Sheffield’s Royal Hallamshire Hospital, which led the UK part of the trial, told the BBC.
Independent experts also welcomed the trial, and called on the NHS to ensure everyone who could benefit from stem cell transplantation can access it.
The treatment, called haematopoietic stem cell transplantation (HSCT), was trialled in a group of 100 patients with relapsing remitting multiple sclerosis – the most common form of the disease.
This type of MS usually strikes in a patient’s twenties or thirties, with new symptoms appearing (relapsing) followed by a period of remission where symptoms may improve or remain stable for months, or longer. Some patients do not respond to drug treatments intended to slow the disease and these relapses become more common.
In the trial, 110 patients who had two periods of relapse in the past year were registered at hospitals in Sheffield and Chicago, as well as Sao Paulo in Brazil and Uppsala in Sweden.
All patients underwent chemotherapy to wipe out their defective immune cells, then half were given a boost of stem cells taken from their blood and bone marrow while the rest underwent conventional drug therapies. In the trial’s first year only one patient in the stem cell group experienced a relapse in their symptoms, compared with 39 in the drug group.
The patients were followed up with after three years, on average, the stem cell transplant only failed in three of the 52 original patients (6 per cent) compared with a failure rate of 60 per cent in the drug group. Those patients who continued to deteriorate were allowed to switch to the stem cell treatments. Around 30 did, and their condition also improved.
Two years ago Louise Willetts’s MS had become extremely severe. She was in a wheelchair and struggling to read, and had given up on her family and career ambitions.
“It does feel like a miracle. I almost have to pinch myself and think ‘Is this real l? Is it really gone, is it ever going to come back?’” she told the BBC in an interview.
Since she became one of the trial participants at Royal Hallamshire Hospital, Louise is symptom-free and there is no sign of the disease attacking her brain. “It feels like my diagnosis was just a bad dream because I have just gone back to how I was before I got diagnosed,” she said.
The results were presented at the annual meeting of the European Society for Blood and Bone Marrow Transplantation on Sunday, but they have yet to be published in a peer-reviewed journal.
However, scientists said the reported results were more impressive than anything seen in previous trials.
Professor Basil Sharrack, who was also part of the Sheffield team, said: “Almost all patients receiving HSCT showed no signs of their disease being active a year on from having the treatment. More importantly, their level of disability improved significantly.”
Dr Susan Kohlhaas, director of research at the Multiple Sclerosis Society, said the next step was to compare this stem cell transplant with less drastic treatments and to make it available to as many people
who could benefit as possible.
“The trial results are important and show this area needs further research. While HSCT appears to be effective for some people with MS, it remains a high-risk treatment that won’t be right for everyone.
“HSCT will soon be recognised as an established treatment in England. And when that happens our priority will be making sure those who could benefit can actually get it. We’ve seen life-changing results for some people and having that opportunity can’t depend on your postcode.”