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Stem Cell Transplants May Safely Slow MS Progression, Suggests Study

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A new study has suggested that stem cell transplants using a patient’s own stem cells to “reset” their immune system can safely slow the progression of relapsing–remitting multiple sclerosis (MS) and should be considered as the standard-of-care for severe disease. The research is published in The Journal of Neurology Neurosurgery & Psychiatry.

Identifying more treatments to benefit MS patients

MS is an inflammatory disease affecting almost 3 million people worldwide. It is thought to be an autoimmune disorder – in which the body’s immune system attacks its own tissues – and affects the central nervous system, attacking the protective myelin sheath that covers nerve fibers, leading them to deteriorate.

The disease has many symptoms, such as fatigue, difficulty walking and vision problems. But not everyone with MS is affected in the same way. The most common presentation is relapsing–remitting MS (RRMS), characterized by flare-ups of the disease followed by periods of recovery. However, RRMS over time can progress to secondary progressive MS (SPMS), which is more severe.

MS has no cure, but disease-modifying treatments (DMTs) can be used to help curb inflammation and delay relapses. However, a treatment called autologous hematopoietic stem cell transplantation (aHSCT) – commonly used to treat blood cancers – was first used to treat MS in the 1990s. aHSCT essentially “resets” a patient’s immune system, first wiping it out with chemotherapy and then rebuilding it using their own blood stem cells harvested before treatment. This is thought to eradicate the self-reactive immune system and rebuild one with better control over disease-causing cells.

“Another name for aHSCT is high-dose chemotherapy with stem cell support, which perhaps better describes the procedure,” said the study’s senior author Dr. Joachim Burman, an adjunct senior lecturer at Uppsala University, speaking to Technology Networks.

aHSCT was recognized in Sweden as a treatment for MS in 2016 but has yet to be implemented in clinical guidelines in many countries. In the current study, Burman and colleagues analyzed data from a Swedish MS registry, investigating the safety and efficacy of aHSCT when used in routine healthcare settings – which are more representative of the general population – rather than clinical trials.

A “significant advantage” over DMTs

The researchers analyzed data from 174 RRMS patients treated using aHSCT prior to 2020 – the average age was 31 years and 64% were women.

In the first three years after the procedure, 20 patients (11%) received DMTs – however, nearly three-quarters of patients showed no evidence of disease activity after 5 years and almost two-thirds showed no activity after 10 years.

Looking at the level of disability among patients, of the 149 patients who displayed disability prior to treatment, 54% improved, 37% remained stable and 9% worsened.

“Another important finding is that the treatment effect is durable. Ten years after the procedure only 35% had evidence of disease activity and only 11% needed to restart some other treatment. It is likely that some of the patients will never need treatment for MS again,” Burman added.

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The safety of the therapy is also a considerable concern, as it is perceived as a high-risk procedure – five patients required treatment in intensive care, but none died as a result of treatment. Infections were common – 61 contracted a bacterial infection within 100 days of the procedure and 23 experienced a viral infection, with 3 patients developing shingles. The most common side effect observed in 68% of patients was febrile neutropenia – a high fever and low white blood cell count.

“Patients treated in routine healthcare come in all flavors and are more complicated than patients in clinical trials, which are generally more homogenous,” Burman explained. “Prior to this study, one concern had been that the number of adverse events would be higher in routine care. Reassuringly, there were very few serious adverse events and no treatment-related mortality, so I think it is fair to say that this procedure can be performed quite safely.”

Consideration for standard-of-care?

The authors suggest that these findings support the idea that aHSCT could benefit a greater number of MS patients, and suggest that it should be considered for addition as the standard-of-care for highly active MS. Additionally, more studies are needed to identify which patients may benefit the most from aHSCT.

Nevertheless, Burman also acknowledges some of the limitations of these findings that come with the absence of a comparison group: “This was an observation study, so we cannot learn how this type of treatment compares with conventional disease-modifying drugs,” he explained.

Burman also elaborated on the research team’s future plans: “We are currently trying to find out why some of the patients relapse after treatment, while others can stay in remission [and] off drugs for 20 years or more.”

Reference: Silfverberg T, Zjukovskaja C, Ljungman P, et al. Hematopoietic stem cell transplantation for treatment of relapsing-remitting multiple sclerosis in Sweden: an observational cohort study. JNNP. 2023. doi: 10.1136/jnnp-2023-331864

Dr. Joachim Burman was speaking to Dr. Sarah Whelan, Science Writer for Technology Networks.