New treatment can ‘halt’ multiple sclerosis, says study.
Aggressive chemotherapy followed by a stem cell transplant can halt the progression of multiple sclerosis , a small study has suggested.
The research, published in The Lancet, looked at 24 patients aged between 18 and 50 from three hospitals in Canada. For 23 patients the treatment greatly reduced the onset of the disease. An MS Society spokeswoman said this type of treatment does “offer hope” but also comes with “significant risks”.
Around 100,000 people in the UK have MS, which is an incurable neurological disease.
‘No Relapses’
The condition causes the immune system to attack the lining of nerves in the brain and spinal cord. Most patients are diagnosed in their 20s and 30s.One existing treatment is for the immune system to be suppressed with chemotherapy and then stem cells are introduced to the patient’s bloodstream – this procedure is known as an autologous haematopoietic stem cell transplant (HSCT). But in this study, Canadian researchers went further – not just suppressing the immune system, but destroying it altogether.
It is then rebuilt with stem cells harvested from the patient’s own blood which are at such an early stage, they have not developed the flaws that trigger MS. The authors said that among the survivors, over a period of up to 13 years, there were no relapses and no new detectable disease activity. All the patients who took part in the trial had a “poor prognosis” and had previously undergone standard immunosuppressive therapy which had not controlled the MS – which affects around two million people worldwide. One person died as a result of the strong effects of the chemotherapy, the authors said.
‘Disease Free’
Lead author Dr Mark Freedman admitted there were limitations to the study – such as the small sample size – and there was no control group used for comparison with those who were treated. He said: “Larger clinical trials will be important to confirm these results. “Since this is an aggressive treatment, the potential benefits should be weighed against the risks of serious complications associated with HSCT and this treatment should only be offered in specialist centres experienced both in multiple sclerosis treatment and stem cell therapy, or as part of a clinical trial.”
Dr Emma Gray, who is head of clinical trials at the MS Society, said: “This type of stem cell transplantation is a rapidly evolving area of MS research that holds a lot of promise for people with certain types of MS. “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 HSCT we’d recommend they speak to their neurologist.” Prof Siddharthan Chandran at the University of Edinburgh described the work as “important and carefully conducted”.
“…[It] demonstrates that powerful chemotherapy-based treatment for a selected subset of MS patients with very aggressive disease is effective in preventing further disabling relapses and, in a proportion, appears to render them effectively disease-free,” he said. Meanwhile, Dr Stephen Minger, a stem cell biologist and independent consultant, described the study as “truly impressive”
He said: “It’s important to stress that this is a very early study, though with impressive long-term follow-up of treated patients.
“Nevertheless, 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.”
Stem Cell Transplants Improve on Current MS Treatments
A one-time stem cell transplantation treatment for multiple sclerosis showed improvements over the current therapies, according to a preliminary trial published in the Journal of the American Medical Association (JAMA).
The process, called hematopoietic stem cell transplantation (HSCT), temporarily shuts down and reboots patients’ immune systems, allowing the body to rebuild damaged nerve cells. The success of this trial serves as a proof-of-concept for the treatment, according to Richard Burt, MD, chief of Immunotherapy and Autoimmune Diseases in the Department of Medicine and lead author of the study.
“This opens the door for this therapy,” Burt said. “Now we can work on improving it, refining it and making it safer.”
MS is an immune-mediated disorder of the central nervous system often arising in young adulthood. It damages the nerve cells in the brain and spinal cord, causing symptoms that range from fatigue to paralysis throughout life.
The current disease-modifying therapies are intense and personalized drug regimens that patients must take indefinitely, even as its effectiveness can eventually fade, according to Burt.
HSCT, however, relies on a different strategy: MS patients are given five days of immune-specific drugs to shut down their self-destructive immune system, then stem cells are transplanted into the patient’s blood. This produces new immune cells that several previous studies suggest may reset the immune system.
“When the immune system restarts in a non-inflammatory environment, it appears to default back to tolerance and stops the attack, allowing, depending on the stage of disease, for the body to repair itself to some extent,” Burt said.
Previously, HSCT found success in non-randomized patients in trials at Northwestern and elsewhere — including in Sweden and Canada, and at the Fred Hutchinson Cancer Research Center and Duke University — but the current study is the first fully-randomized trial that tested relapse-remitting MS (RRMS) against the current treatment, Burt said.
In the trial, 110 patients were randomized to receive either HSCT or the traditional drug therapy. At last follow-up, disease progression occurred in 34 patients in the drug therapy group, but in just three patients in the HSCT group.
Patients in the drug therapy group had worsening scores on the Expanded Disability Status Scale (EDSS) — a method of quantifying disability in patients with MS — while patients in the HSCT group actually saw improvements in their scores.
In addition to being more a more efficacious treatment, HSCT has the potential to be more cost-effective than the current drug therapy, according to Burt.
“A full cost analysis needs to be performed, because the current drugs are very expensive,” Burt said. “I would anticipate, since this is just one treatment, that it may be a win-win: patients undergo a one-time treatment and get off of the drug regimen, while possibly saving money from a public health perspective.”
However, Burt cautions that further investigations are still needed to replicate these findings, and that the treatment needs to be used in the right type of RRMS.
Other Northwestern authors include Borko Jovanovic, PhD, associate professor of Preventive Medicine in the Division of Biostatistics; Roumen Balabanov, MD, associate professor in The Ken & Ruth Davee Department of Neurology, in the Division of Multiple Sclerosis & Neuroimmunology; Xiaoqiang Han, MD; Irene Helenowski, PhD; Amy Morgan; Kathleeen Quigley; Kim Yaung; Carri Alldredge; Allison Clendenan; Michelle Calvario and Jacquelyn Henry.
New treatment can ‘halt’ multiple sclerosis, says study.
Stem Cell Transplants Improve on Current MS Treatments
