Breakthrough Treatment for Guillain-Barre Syndrome in Clinical Trials


The first new treatment for Guillain-Barre Syndrome in 20 years is entering a Phase II clinical trial. It’s called eculizumab, which is a humanized monoclonal antibody first approved by the Food and Drug Administration in 2007 to treat a rare blood disorder.

In Guillain-Barre Syndrome or GBS, the body’s immune system attacks part of the peripheral nervous system and often causes acute neuromuscular weakness. People with GBS may also experience numbness, tingling, and blurred vision. Because this disease can affect respiratory muscles, some patients have been placed on a ventilator. Up to 30 percent of patients are left with a permanent disability, including some who cannot walk unassisted.

Two treatments, plasmapheresis and intravenous immunoglobulin are currently used on patients with GBS, but medical experts continue to look for even better options, including eculizumab.

Neurologists believe controlling inflammation during the acute phase of GBS is key to reducing nerve injury and long-term neurological problems. Eculizumab may help do that by inhibiting activation of the body’s complement system. This component of the immune system may become overactive in GBS and damage nerve fibers. The Phase II clinical trial is being conducted by researchers at the University of Glasgow in Scotland and is expected to be complete by March of 2016.

The exact cause of GBS is not known, but it usually occurs after a respiratory or gastrointestinal infection. In some cases, the syndrome can be triggered by an immunization, including the influenza vaccine.

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