Multiple sclerosis is close to being cured after a pioneering treatment was found to stop and even reverse the disease, scientists said last night.
Results of a trial were hailed as remarkable, with the progression of the debilitating disease halted in almost all patients who had the treatment. A quarter of the MS sufferers had their condition effectively suppressed.
In the trial at the University of Ottawa in Canada, patients had aggressive chemotherapy combined with a transplant of their own cells. The chemotherapy destroyed the immune system instead of suppressing it as in standard treatment. It was then “reset” using blood stem cells.
The new technique is not without risk. Of the 24 patients treated, one died as a consequence, and the study was too small to assess the true dangers. Even so, the treatment was described as a breakthrough therapy which was “close to being curative”.
MS is a chronic inflammatory disease of the central nervous system. It is caused when the immune system attacks the body and can result in vision problems, memory loss, dizziness, fatigue and spasms. In severe cases it can lead to paralysis.
The condition affects about 100,000 people in the UK, most aged 20 to 40 when it is diagnosed. It has very few treatment options and often patients live for many years with the debilitating symptoms.
One of those treated in the Ottawa trial was Jennifer Molson, who was diagnosed with MS in 1996 when she was 21. She had an aggressive form of the condition and limited mobility when she began the trial, and received her transplant in 2002.
“Before my transplant I was unable to walk or work and was living in assisted care at the Ottawa hospital rehabilitation centre,” she said. “Now I am able to walk independently, live in my own home and work full time. I was also able to get married, walk down the aisle with my dad and dance with my husband. I’ve even gone downhill skiing. Thanks to this research I have been given a second chance at life.”
Researchers enrolled 24 people aged 18 to 50 with aggressive MS who had not responded to immunosuppressive therapy. They were followed for an average of six-and-a-half years to test the long-term effects of chemotherapy followed by the treatment, called autologous haematopoietic stem cell transplantation (aHSCT).
After treatment 70 per cent of patients experienced a complete stop in disease progression and 40 per cent saw lasting reversal of symptoms. Several were able to return to work or school and have children. None needed medication.
“[This shows with] unprecedented clarity a near-complete suppression of MS disease for several years following aHSCT,” Paolo Muraro, of Imperial College London, said. “None of the drugs that are currently approved or in late-phase clinical trials has been reported to achieve a similarly profound control of the disease.”
Stephen Minger, a stem cell biologist and independent consultant, said: “The clinical results are truly impressive, in some cases close to being curative, though we need longer-term follow-up to know for certain whether the patients continue to do well or if there is a chance of relapse. For a lifelong progressive disease like MS with few treatment options this is really exciting data.”
During treatment one of the patients died from hepatic necrosis and sepsis caused by the chemotherapy, which is one reason clinicians are approaching the results with caution.
Harold Atkins, a stem cell transplant physician at the Ottawa hospital and author of the study, said: “Our trial is the first to show the complete, long-term suppression of all inflammatory activity in people with MS. However, it is important to note that this therapy can have serious side-effects and risks, and would only be appropriate for a small proportion of people with very active MS.”
In Britain MS sufferers can receive aHSCT treatment only if they are chosen for a clinical trial. Emma Gray, a clinical lead at the MS Society, said that more research was needed. “This treatment does offer hope, but it’s also an aggressive procedure that comes with substantial risks and requires specialist aftercare. If anyone is considering aHSCT we would recommend they speak to their neurologist,” she said.
What is MS? It is a condition where the immune system starts attacking the body. It wrongly identifies myelin, the protective sheath coating nerve fibres, as a foreign body and eats away at it. This exposes nerves and disrupts their functioning.
What does it do? Damage to the nervous system affects the whole body. Symptoms are varied and pervasive, ranging from vision problems and spasms to impaired balance and memory.
What are the current treatments? Most treatments concentrate on managing the disease, emphasising diet, physiotherapy and exercise. Drugs are available to slow progression by suppressing the immune system. Blood stem-cell treatments have been tried before, but with lower doses of chemotherapy — and lower success rates.
What are blood stem cells? Known as hematopoietic stem cells and located in the bone marrow, these give rise to all other blood cells.
What does this treatment do? The procedure begins by giving a patient medication to coax the hematopoietic stem cells to migrate from bone marrow into the blood. These are then collected, purified and frozen. Chemotherapy is used to eliminate the diseased immune system. The stem cells are then transplanted back so that they can give rise to a new immune system that has no “memory” of the previous pattern of attacking the central nervous system.
Why are scientists excited about such a small study? The results are extremely impressive. The only explanation that makes sense is that the treatment turned round or halted their disease.
So is it a miracle cure? Not quite yet. The patients were chosen because they had failed to respond to immunosuppressive therapy, so they may not be representative. While the study could assess the treatment’s efficacy, it was not big enough to assess its risk. The single death may have been extremely bad luck but it may show that there is a high chance of the treatment killing patients.