This ability to reduce plasma SAP concentrations with CPHPC is pivotal in the rapidly advancing use of CPHPC for the treatment of systemic amyloidosis (Gillmore et al., 2010 ▶; Bodin et al., 2010 ▶; http://www.clinicaltrials.gov/ct2/show/NCT01777243?term=amyloid+gsk&rank=2) and also offers exciting potential in Alzheimer’s disease (Kolstoe et al., 2009 ▶). The gene discussed is APCS; the disease is early-onset autosomal dominant Alzheimer disease.