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Stem cell therapy highly effective in active RRMS: Real-world study
Nearly 80% of patients in Denmark free of relapses after 2 years
by Steve Bryson, PhD |
Nearly 80% of adults with highly active relapsing-remitting multiple sclerosis (RRMS) were free of relapses or confirmed disability worsening two years after receiving stem cell transplant, according to a real-world study in Denmark.
Moreover, more than two-thirds (69%) of these RRMS patients achieved a clinical outcome called NEDA-3, or no evidence of disease activity — defined as the absence of relapses, disability worsening, or new or enlarging lesions on MRI scans — after two years.
While stem cell therapy in MS often is associated with severe side effects, no patients died and the procedure was deemed safe. According to the researchers, these outcomes may be largely attributed to a careful selection of patients and the use of a less toxic chemotherapy regimen prior to the transplant.
“Our findings based on the study of 32 RRMS patients supports the growing volume of evidence indicating that [stem cell therapy] is an effective and safe treatment with few serious [adverse events] and no mortality,” the team concluded.
The study, “Autologous hematopoietic stem cell transplantation of patients with aggressive relapsing-remitting multiple sclerosis: Danish nation-wide experience,” was published in the journal Multiple Sclerosis and Related Disorders.
32 patients undergo transplant in Denmark over decade
Autologous hematopoietic stem cell transplant (aHSCT), more commonly called stem cell therapy, is an invasive procedure intended to reset the immune system and stop the inflammatory attack that drives MS.
The treatment first involves collecting hematopoietic stem cells — those that give rise to mature immune cells — from a patient’s blood. Then, after the entire immune system is wiped out with a chemotherapy or radiation therapy regimen, the stem cells are infused back into the same patient. When a patient’s own stem cells are used, the procedure is called autologous; donor stem cells also can be used for the procedure.
The goal of all stem cell therapy in MS is to repopulate the immune system with healthy cells that won’t attack the nervous system.
Studies indicate that the best outcomes after aHSCT occur in RRMS patients with highly active disease, characterized by multiple relapses and/or MRI activity, despite treatment with disease-modifying therapies.
In this report, researchers at the Danish Multiple Sclerosis Center (DMSC), in Copenhagen, described their experience with aHSCT for RRMS patients in Denmark with highly active disease.
A total of 32 patients underwent stem cell therapy at the center in the decade from May 2011 to May 2021. Seven received the chemotherapy conditioning BEAM/ATG, and had a median follow-up of 49 months, or just longer than four years.
The other 25 were treated with a less aggressive chemotherapy regimen, called CY/ATG, that’s associated with a lower risk of side effects than BEAM/ATG. These patients were followed for a median of 39 months, or a little longer than three years.