31 Dec Multiple Sclerosis: Pilot Study of Immunosuppression Plus Transplantation
Medical Research: What is the background for this study? What are the main findings?
Dr. Nash: Multiple sclerosis is an autoimmune disease of the central nervous system which causes significant disability and in some cases results in patients being wheel-chair bound or bed-ridden. It is a significant medical problem amongst young adults. We undertook this study because current Multiple sclerosis therapies were not adequate for effective long-term control of the disease in the majority of the patients. High-dose immunosuppressive therapy followed by autologous hematopoietic cell transplantation is an effective treatment for many hematological malignancies. It causes a profound immunosuppression. Based on this effect on the immunological system, we initiated a clinical trial of this treatment modified for autoimmune disorders. The study was supported by the Immune Tolerance Network and NIAID, NIH. In a phase 2 clinical trial of 25 patients all of whom were followed for at least 3 years, we demonstrated that 80% of patients had no evidence of disease activity. No other Multiple sclerosis treatments were given after the study treatment. Adverse events were similar to what we have observed for this treatment in patients with hematological malignancies. No significant acute neurological adverse events were observed.
Medical Research: What should clinicians and patients take away from your report?
Dr. Nash: We concluded that this treatment is promising. Long-term control of the disease is possible. Further follow-up is planned since this report is only an interim analysis. All patients will be followed to 5 years.
Medical Research: What recommendations do you have for future research as a result of this study?
Dr. Nash: We believe that the data is sufficiently promising that a randomized clinical trial is warranted. We have been in discussions regarding this.