Future treatment for Guillain–Barré syndrome

Abstract

Plasma exchange and intravenous immunoglobulin were established to be effective treatments for Guillain–Barré syndrome (GBS) in the 1980s and 1990s, respectively. However, even when treated with the currently best therapy available, the mortality rate is still approximately 5%, and approximately 20% of patients cannot walk unaided 6 months after onset. Therefore, many GBS patients still develop severe weakness and have a long disease course, often with pain and fatigue as a result of persistent axonal loss. A better treatment is required. A clinical trial of a second immunoglobulin is ongoing. Eculizumab, a humanized monoclonal antibody against complement C5, is a promising novel agent, and phase 1/2 trials are ongoing in Japan (Japanese Eculizumab Trial for GBS) and Scotland (Inhibition of Compliment Activation for GBS). Immunoglobulin G-degrading enzyme of Streptococcus pyogenes is another promising agent, and a phase 2 trial is planned. Prevention of severe axonal injury with new treatments early in the course of this disease remains a major focus, because axonal degeneration is an important limiting factor in achieving a good outcome.