- A stem cell treatment commonly used for blood cancer appears effective in treating one form of MS
- The treatment has been available since the 1990s but questions about safety and difficulty have limited its impact
- New research suggests it is safer and would benefit more people with MS than previously believed.
A stem cell therapy for multiple sclerosis (MS) appears to be generally safe and effective, but it isn’t widely utilized. Researchers in Sweden are trying to get the word out.
Multiple sclerosis (MS) is a devastating illness that has proven difficult to treat and impossible to cure. But for some MS patients there may be an option available that has been waiting in the wings for decades.
Autologous hematopoietic stem cell transplantation (aHSCT) is primarily used to treat cancers of the blood, but has been used in the treatment of MS since the 1990s.
However, questions about its safety and the difficult nature of the procedure have prevented it from becoming a standard part of MS care.
In an article published this week in the Journal of Neurology Neurosurgery & Psychiatry, researchers in Sweden found that aHSCT is a suitable treatment for a form of MS known as relapsing-remitting multiple sclerosis.
Because aHSCT has been studied predominantly in clinical settings, they wanted to see how the procedure worked in actual patients in traditional healthcare settings. They conclude that the treatment is not only safer and more effective than previously believed but that the procedure could potentially impact even more people living with MS.
“Our findings suggest that aHSCT could benefit a greater number of MS patients and it should be included as a standard of care for highly active MS. AHSCT has been viewed as a high-risk procedure in the past, but our findings challenge this view and we conclude that the procedure can be performed safely in experienced hands,” Dr. Joachim Burman, a member of the Department of Medical Sciences at Uppsala University in Sweden, and co-author of the research, told Healthline.
To investigate, Burman and his team pulled data from the Swedish MS registry and the European Society for Blood and Marrow Transplantation registry to find patients with relapsing-remitting multiple sclerosis who had been treated with aHSCT.
They initially identified 231 patients and the final analysis included 174 that met all of their criteria. This criteria included that they had to have relapsing-remitting multiple sclerosis and not another form of MS and had to have received their aHSCT treatment prior to 2020. Patients had a median follow-up time of 5.5 years.
The primary outcomes that the researchers were looking at were safety, as indicated by the occurrence of adverse events like infection or death; and disease progression.
The study showed “no evidence of disease progression” in 73% of patients after 5 years and 65% after 10 years. Of the 149 patients that had some form of disability at the beginning of the observation period, 54% showed improvement, 37% were stable, and only 9% showed worsening symptoms.
“This really is confirmatory because this study is quite consistent with many previous studies that have shown that aHSCT therapy can be beneficial for some people living with MS,” Bruce Bebo, Executive Vice President of Research at the National MS Society, told Healthline.
Patients did experience adverse events during the observation period.
The most common was
One death was reported but no treatment-related deaths were reported.
Adverse events were found to be manageable within traditional healthcare environments.
“This procedure has been perceived as a high-risk procedure, but as we have shown it can be performed quite safely,” said Burman.
Despite his findings though, aHSCT is still a serious procedure.
In MS, the immune system attacks part of the brain, spinal cord, and nerves; specifically, it damages the myelin sheath, a fatty substance that acts as insulation around nerves, and ultimately causes disability.
The goal of aHSCT is to “reset” the immune system and prevent it from further damaging the nervous system. The entire process requires blood stem cells to be collected from the patient and then frozen. The patient then undergoes chemotherapy to suppress the immune system. In this immunosuppressed state, the previously collected blood cells are then altered and reintroduced to the body.
The process involves weeks in the hospital, chemotherapy, and immunosuppressive medication that lower the body’s natural ability to fight off infection.
“Chemotherapy is used to ablate the bone marrow, and then the marrow is reconstituted with stem cells. This involves a 3-4 week stay in hospital, and patients are vulnerable to infection while the marrow is repopulating. “Historically there is about a 2% mortality rate with AHSCT, but there is more experience with the chemo regimens now and no deaths were seen in the trial,” said Dr. Christopher Lock, Clinical Associate Professor of Neurology & Neurological Sciences, and Clinical Trials Director for Multiple Sclerosis at Stanford University.
“It’s not a trivial treatment,” said Bebo, “While the protocols and techniques and drugs and things used over the last 10 or 20 years have been refined and the safety has been improved dramatically, it still comes with significant risks and side effects and needs to be considered carefully by anybody who is thinking about this.”
The National MS Society Guidelines recommend aHSCT for individuals who:
- Have relapsing-remitting multiple sclerosis
- Are less than 50 years old
- Have had MS for less than 10 years
- Are not responding to typical disease-modifying therapy
For some, the risk will likely be worth it. Nearly one million people live with MS in the United States and the disease affects three times as many women as men. It is the leading cause of permanent neurological disability in young adults. MS is also notoriously hard to diagnose. There are multiple forms of it as well. Relapsing-remitting multiple sclerosis is the predominant form of MS, and is characterized by bouts or “flare ups” that can cause vision problems, muscle weakness, numbness or tingling, fatigue, and more. These attacks clear up over time and are generally followed by a remission period. Flare-ups inevitably follow, and with each one the potential for worsening of symptoms.
Relapsing-remitting multiple sclerosis is sometimes followed by another form of MS called secondary progressive MS (SPMS). This type of MS is characterized by its “progressive” nature, meaning that neurologic function worsens and disability increases over time.
AHSCT treatment is only for individuals with relapsing-remitting multiple sclerosis and not other forms.
“We would generally reserve [aHSCT] for patients with aggressive forms of relapsing MS, and who are refractory to or have failed first- and second-line therapies. There are currently over 20 disease modifying drugs approved for MS, and we would try these first,” said Lock.
Disease modifying therapies (DMTs) are generally the first line of treatment for people with MS. DMTs help to prevent permanent damage to the central nervous system, control flare-ups, and improve every day symptoms.
But Burman believes that his work should help to inform more doctors about the procedure and help to open the door to more individuals with MS looking for an effective treatment.
“About 5-10% of MS patients have an aggressive disease with rapidly evolving disability. These patients should all be candidates for aHSCT. Some patients (maybe 20%) have very mild disease and should opt for regular treatment. There is a large middle group, which previously has not been considered for aHSCT, but I think that aHSCT could at least be considered in these cases,” he said.
Autologous hematopoietic stem cell transplantation (aHSCT) is effective and generally safe for treating relapsing-remitting multiple sclerosis.
New research contends that the procedure is safe and effective enough that it be included as part of the standard of care for relapsing-remitting multiple sclerosis.
AHSCT is still a serious procedure that requires hospitalization and immunosuppressant drugs. It is only recommended for certain individuals with relapsing-remitting multiple sclerosis.