Is haematopoietic stem cell transplantation a treatment option for severe MS or not?

A number of treatments are now available to treat multiple sclerosis (MS) among them several beta interferons (IFN-β) and glatiramer acetate (GA) for relapsing–remitting MS (RR-MS), and mitoxantrone and natalizumab for aggressive forms of RR-MS and those patients who have failed the first line therapies IFN-β and GA. With mitoxantrone and natalizumab, higher efficacy comes at the price of more and more severe adverse events among them secondary leukaemias and compromise of cardiac function (mitoxantrone), and progressive multifocal leukoencephalopathy (PML)(natalizumab). Numerous other therapies that fall in the latter category are currently in development including alemtuzumab (Campath-1), fingolimode (FTY-720), cladribine and others. All these approaches share several characteristics. They all target the inflammatory phase of MS; with the exception of fingolimode, they all have to be injected; and their potential risks are higher than those of IFN-β and GA. Hence, their use needs to be weighed carefully, and patient involvement in treatment decisions is paramount.Haematopoietic stem cell transplantation (HSCT) appears to represent yet a further escalation. Autologous HSCT encompasses mobilization and preservation of autologous haematopoietic stem cells (HSCs); subsequently, in most cases, myeloablative immunosuppression by a combination of chemotherapeutic drugs and often total body irradiation (TBI); and later the reinfusion of HSCs (Tyndall …