Emerging drugs for Guillain-Barré syndrome

Intravenous immunoglobulin (IVIg) and plasma exchange are proven effective treatments for Guillain-Barré syndrome (GBS). However, this treatment is insufficient for many patients as 1 - 5% die, 25% need artificial respiration, 20% are still unable to walk unaided after 6 months and 85% have residual symptoms, such as fatigue and pain.

Strategies to design and conduct trials with new compounds and individualized regimens of IVIg are discussed. The development of specific immunomodulators is set against a background of recent insights in the pathophysiological mechanisms of GBS. Patients with poor prognosis can be identified in the early phase of disease using predictors such as high age, severe disability, preceding diarrhea and possibly low increase in serum IgG after standard IVIg treatment. An ongoing trial with a second IVIg dose in this group and the preclinical development of potential new treatments and their mode of action are discussed.

GBS is a heterogeneous disease with considerable short- and long-term disability for which more effective and individualized treatments are required. Under investigation are new treatment strategies with adapted IVIg dosages based on prognostic factors and more specific immunomodulation, including complement inhibitors.