A large body of clinical and experimental data indicate that complement 
activation is an important mechanism for neuronal and glial injury in 
Guillain-Barré syndromes. Inhibition of complement activation therefore might be 
expected to limit the progression of the disease. Using in vitro and in vivo 
models of the Guillain-Barré syndrome variant, Miller Fisher syndrome, we have 
shown previously that anti-GQ1b ganglioside antibodies target the presynaptic 
motor nerve terminal axon and surrounding perisynaptic Schwann cells, thereby 
mediating destructive injury through deposition of membrane attack complex. 
Here, we have used this model to investigate the effects of a novel therapeutic 
inhibitor of complement activation, APT070 (Mirococept), both in vitro and in 
vivo. In these models, APT070 completely prevents membrane attack complex 
formation, and thereby has a major neuroprotective effect at the nerve terminal, 
as assessed by immunohistology of perisynaptic Schwann cell and axonal 
integrity. These data provide a rationale for considering clinical trials of 
APT070 in Guillain-Barré syndrome, its variant forms, and other complement 
dependent neuromuscular disorders.