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 screened, 26 were enrolled, 24 underwent HSCT, 21 completed 3-year follow-up, and 13 were followed longer-term. MS activity-free survival at 3 years was 70%. The 30% with disease activity primarily had sustained disease worsening attributable to disease progression, not necessarily inflammatory activity. Before treatment, 24 patients had 167 relapses during a median 6 years. After HSCT, among 23 patients with 179 patient-years of follow-up, no clinical relapses occurred and no gadolinium-enhancing lesions were found; one person had 4 new T2 lesions 1 month posttransplant that were not present 5 months earlier. Improvement on the EDSS was observed in 40% and appeared to occur for several years after transplant.