Conference synopsis: hematopoietic stem cell therapy in autoimmune diseases, October 2001

Since 1996, patients with autoimmune diseases have been treated on single-arm investigational protocols with high-dose immunosuppressive therapy and autologous peripheral blood stem cell transplantation (HSCT). In a conference held in October 2001 at the City of Hope National Medical Center, participants discussed current laboratory studies in autoimmunity, the rationale of HSCT in autoimmune diseases, results of phase I-II studies, and the prospects for controlled trials. This conference synopsis summarizes major discussion points in clinical sessions and in sessions devoted to ethical and regulatory aspects of this investigational treatment. Protocols for controlled studies in multiple sclerosis (MS) and systemic sclerosis (SSc), originating in Europe and in the United States, have been designed or are in the final stages of design. The only controlled trial presently underway is for SSc in Europe (Autologous Stem Cell Transplantation International Scleroderma Trial [ASTIS]). There are current plans for a controlled trial for rheumatoid arthritis (RA) in Europe (ASTIRA) but not in the United States. Eventual cross-study analysis of the European and United States trials may give valuable comparative information on the different mobilization and immunosuppressive regimens used. Recognition of the importance of axonal degeneration in secondary progressive MS and the use of mitoxantrone as a rescue medication are two relatively recent developments now being considered in the design of controlled HSCT protocols in MS. The importance of informed consent and study accessibility was discussed as well as the continuing role of the US Food and Drug Administration in regulating these protocols in the United States.