Bone marrow transplantation (BMT) is widely used in a number of neoplastic diseases, and there have been a number of reports suggesting that it may also be beneficial in several autoimmune diseases. The procedure consists of a severe immunosuppressive regimen provided by high-dose chemotherapy or total body irradiation followed by IV infusion (transplantation) of bone marrow-derived haematopoietic stem cells. During the first months after BMT, transplant recipients are exposed to many sources of complications, some of which are potentially lethal. The procedure has been shown to be effective in EAE, but observations in MS are only obtained in small studies and claims for therapeutic efficacy are based on anecdotal (and often poorly documented) reports of therapeutic efficacy in MS. First impressions suggest that the procedure appears to be ineffective for patients with progressive disease, whereas patients with low disability and more inflammatory disease activity might benefit. Recent studies suggest that bone marrow transplantation does not have an impact on demyelination and neurodegeneration, two of the most fundamental underlying pathologic features of MS.

In the opinion of the Committee, the rationale of BMT is plausible and warrants investigation in well-designed studies carried out in centres with experience in managing profoundly immunocompromised patients. BMT carries serious risk.