New Trial of Hematopoietic Stem Cell Transplant for Aggressive Multiple Sclerosis

Hematopoietic stem cell transplant (HSCT) essentially resets the immune system by mobilizing stem cells, ablating the immune system, and reconstituting the immune system with autologous cells. HSCT is being tested as a treatment for multiple sclerosis (MS; NEJM JW Neurol Apr 2015 and JAMA 2015; 313:27). Now, researchers have conducted a phase II trial, enrolling patients from three centers who had poor prognostic features (e.g., expanded disability status scale [EDSS] ≥3.0 within 5 years after disease onset, ongoing disease activity after 1 year of standard MS therapy, or a cerebellar/pyramidal functional system score of ≥3). Median age was 34 years (range, 24–45) and median disease duration was 6 years (range, 1–11). Of 39 patients screene…