Autologous haematopoietic-stem-cell transplantation for multiple sclerosis

Lancet Neurol. 2005 Jan;4(1):54-63. doi: 10.1016/S1474-4422(04)00966-4.


Intense immunosuppression followed by autologous haematopoietic-stem-cell transplantation (HSCT) is being assessed as a potential treatment for patients with severe multiple sclerosis (MS). The treatment was developed from research that showed autologous HSCT was as effective as allogeneic HSCT in the treatment of experimental autoimmune encephalomyelitis. The treatment is thought to eradicate the defective immune system, and the infused haematopoietic stem cells reconstitute an immune system that is more tolerant to the nervous system. About 250 patients with MS have been treated with autologous HSCT as part of phase I and phase II open trials. Autologous HSCT seems feasible in MS and assessment with clinical and MRI measures suggests it induces a profound and long-lasting suppression of inflammation. The course of MS seems to be stabilised after autologous HSCT, especially in ambulatory patients with evidence of active disease. Autologous HSCT deserves further study in randomised controlled trials.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Clinical Trials, Phase I as Topic
  • Clinical Trials, Phase II as Topic
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Hematopoietic Stem Cell Transplantation* / mortality
  • Humans
  • Magnetic Resonance Imaging
  • Multiple Sclerosis / surgery*
  • Transplantation, Autologous