New class of anti-amyloid drugs reported in PNAS

Australia-based Prana Biotechnology, a biopharmaceutical company focused on the R&D of treatments for neurodegenerative disorders, has announced a publication in the Proceedings of the National Academy of Sciences journal, describing a completely new class of amyloid inhibiting drugs. The reported drugs are directed against the amyloid present in Alzheimer's disease, composed of the Abeta protein. By specifically targeting and blocking the metal binding site on the Abeta protein, these agents change the intrinsic structural properties of Abeta and its oligomers, stopping neuronal toxicity and the subsequent formation of ABeta fibrils.

The PNAS paper highlights several key characteristics of these amyloid inhibitors, which upon binding to Abeta change the protein's structural properties and consequent behaviour including: the ability to stop Abeta aggregation progressing into amyloid fibrils; shut down of toxic free radical production by Abeta; and rescue of synapses from Abeta-induced toxicity, as measured by long-term potentiation, a measure of synaptic strength which is commonly viewed as a biochemical model of memory.

Prana's chemistry program is building upon the structural template provided by these proof-of-concept compounds to create a library of novel drugs that are orally available. To date, the firm's lead anti-amyloid compound has been designed to penetrate the brain, and is able to reduce both Abeta and tau protein biomarkers in the brain of Alzheimer's disease transgenic mouse models.