New data reveal the power of stem-cell transplantation in slowing or even halting disease progression in patients with RRMS, thereby reinvigorating further exploration of cellular treatments in MS.
The findings come from a small, randomised, controlled clinical trial led by an international panel of experts in the USA, Sweden, the UK and Brazil. A total of 110 RRMS patients aged 18–55 whose disease was not effectively controlled by an existing disease-modifying therapy (DMT) were assigned to either haematopoietic (blood-producing) stem cell transplantation (HSCT; n = 55) or an alternative/stronger DMT than previously received (n = 55).
HSCT utilises cells derived (primarily) from a patient’s own bone marrow. Following a regimen of immunosuppressant therapy (in this case cyclophosphamide, 200 mg/kg and antithymocyte globulin, 6 mg/kg), the stem cells are deployed to effectively “reboot” the immune system.
In line with the study’s primary endpoint of disease progression (increase in Expanded Disability Status Scale ≥ 1.0 point* at 1 year), results revealed significantly fewer incidences of disease progression in the HSCT group (3/52) compared with DMT (34/51).
In brief, these results suggest that HSCT slows or even halts disease progression in RRMS patients who have not benefited from conventional DMT, and provide an initial stepping stone to further the research of stem-cell therapies in the treatment of MS.
Read the paper and the accompanying editorial here.
*Minimal clinically important difference: 0.5. Two evaluations 6 months apart, with differences in time to progression estimated as hazard ratios.