Stem-Cell Transplants Delayed Disease Progression of Multiple Sclerosis

January 25, 2019—As published in JAMA, interim results of the phase 2 MIST trial (NCT00273364) show that stem-cell treatment resulted in a significantly prolonged time to disability progression (Table)(hazard ration [HR] = .07) for patients with MS who had 2 or more relapses while using disease-modifying treatments (DMTs) prior to study enrollment. Disability progression was defined as an increase in Expanded Disabiity State Scale score of 1 or more at 2 or more consecutive evaluations done every 6 months. In the first year, 1 of 51 (2%) patients treated with stem-cell transplant had a relapse, compared with 36 of 52 (69%) of patients treated with a DMT that was of higher efficacy or a different class. In the first year, patients treated with stem-cell transplant also had improvements in EDSS, Neurologic Rating Scale (NRS), and T2-weighted lesion volume compared with patients treated with DMT (p <.001 all measures). In the group treated with DMT, EDSS and NRS scores worsened and T2-weighted lesion burden increased.

The stem-cell transplants in this study were autologous hematopoetic stem cells (the patient’s own blood cell precursors) and the transplants were nonmyeloablative, that is done without bone-marrow--destroying doses of radiation.