High-dose chemotherapy and multiple sclerosis

Purpose of review: Immunomodulatory medications for multiple sclerosis provide only modest control of this potentially debilitating auto-immune disease of the central nervous system. The immunosuppression provided by high-dose chemotherapy has been studied to address treatment-refractory disease. In this review, we discuss the recent significant work in this field and its associated controversies. Recent findings: Conclusive evidence for the efficacy of high-dose chemotherapy with stem cell rescue is lacking given the lack of uniform patient populations and varying treatment protocols. Moreover, the significant toxicity associated with this procedure has dampened enthusiasm for its widespread use. High-dose chemotherapy without stem cell rescue has been trialed as a less toxic approach that eliminates the possibility of re-infusing autoreactive lymphocytes found in the stem cell product. Summary: Before high-dose chemotherapy with or without stem cell rescue can be adopted for clinical practice, both approaches require testing in randomized clinical trials. Both procedures have the possibility of decreasing disease activity but high-dose chemotherapy without stem cell rescue having a more favorable safety profile, may prove a more significant advance in the field of high-dose therapy for multiple sclerosis.