Autologous hematopoietic stem cell transplant is better than disease-modifying therapies (DMT) at reducing the risk of disease progression in patients with relapsing-remitting multiple sclerosis (RRMS), results from the MIST clinical trial show.
The study “Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis – A Randomized Clinical Trial” was published in the journal JAMA.
While RRMS patients may respond to immune suppressive therapies, in order to be effective these need to be applied early in the disease course, i.e., while MS is mainly an immune-mediated and inflammatory disease.
Up to now, no therapeutic strategy has been able to prevent progressive disability. Hematopoietic stem cell transplant (HSCT) has shown potential to slow or prevent progressive disability in RRMS patients.
This strategy first collects a patient’s own (aka, autologous) healthy hematopoietic (blood cell-producing) stem cells from the bone marrow, followed by a much less aggressive combination of chemotherapy (non-myeloablative) that kills the rest of the patients’s immune cells.
The hematopoietic stem cells are then infused back into body where they generate a new, healthy immune system.
In the MIST Phase 2 clinical trial (NCT00273364), an international team of researchers evaluated how non-myeloablative HSCT compares to continuous DMT therapy.
The study enrolled 110 patients, ages 18 to 55, with aggressive RRMS. All participants had at least two relapses while undergoing treatment with DMTs in the previous year.
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