Abstract

Wellcome Trust has awarded a grant to TREVENTIS Corporation to discover a disease-modifying drug for the treatment of Alzheimer’s disease (AD). Numerous studies support the causative role of ß-amyloid (Aß) and tau in the aetiopathogenesis of AD.These proteins tend to abnormally “clump” (protein misfolding) and give rise to neurotoxic aggregates of ß-amyloid (plaques) and tau (“tangles”), the pathological hallmarks of AD. In vitro studies have identified that Aß can be neurotoxic when in small aggregates. Since disease-modifying drugs represent the most desirable therapeutic approach to AD, protein misfolding of Aß and tau represents a potential target in the rational design of a drug. The ultimate goal of this research is to discover a disease-modifying new chemical entity for the treatment of AD that is efficacious and safe. This goal will be achieved by optimizing a class of small organic molecules capable of binding to both ß-amyloid and tau, blocking their misfolding. A new series has recently been identified that are drug-like, potent anti-aggregants and penetrate the Central Nervous System. Funding under this award will enable the discovery of a lead candidate with completed pre-clinical pharmacokinetic and toxicology package.