T-CELL-DEPLETED AUTOLOGOUS HEMATOPOIETIC STEM-CELL TRANSPLANTATION FOR MULTIPLE-SCLEROSIS - REPORT ON THE FIRST 3 PATIENTS

Multiple sclerosis (MS) is a disease of the central nervous system cha racterized by immune-mediated destruction of myelin, In patients with progressive deterioration, we have intensified immunosuppression to th e point of myeloablation. Subsequently, a new hematopoietic and immune system is generated by infusion of CD34-positive hematopoietic stem c ells (HSC), Three patients with clinical MS and a decline of their Kur tzke extended disability status scale (EDSS) by 1.5 points over the 12 months preceding enrollment and a Kurtzke EDSS of 8.0 at the time of enrollment were treated with hematopoietic stem cell (HSC) transplanta tion using a myeloablative conditioning regimen of cyclophosphamide (1 20 mg/kg), methylprednisolone (4 g) and total body irradiation (1200 c Gy), Reconstitution of hematopoiesis was achieved with CD34-enriched s tem cells, The average time of follow-up is 8 months (range 6-10 month s), Despite withdrawal of all immunosuppressive medications, functiona l improvements have occurred in all three patients, We conclude that T cell-depleted hematopoietic stem cell transplantation can be performe d safely in patients with severe and debilitating multiple sclerosis, Stem cell transplantation has resulted in modest neurologic improvemen ts for the first time since onset of progressive disease although no s ignificant changes in EDSS or NRS scales are evident at this time.