Small molecule clears amyloid

Amyloidosis is caused by the deposition of proteins such as serum amyloid P component (SAP), and contributes to the pathogenesis of a range of debilitating disorders from type II diabetes to Alzheimer's disease. There is currently no treatment available for these diseases. But in 16 May Nature, Pepys and colleagues from Royal Free and University College Medical School, London, describe a novel therapeutic approach that could be used to treat human amyloidosis.

Pepys et al. used a high-throughput screen to test a suite of chemicals for their ability to inhibit the binding of SAP to the Aβ peptide, and therefore prevent the development of the insoluble senile plaques characteristic of Alzheimer's disease. Using a murine model they identified a drug that acts as a competitive inhibitor of SAP binding to amyloid fibrils. "This palindromic compound also crosslinks and dimerizes SAP molecules, leading to their very rapid clearance by the liver, ...