FBLD and CNS disease

Challenges in CNS drug discovery have been two-fold:  identification of suitable targets for drug discovery and the added hurdle that compounds must be able to access the brain by crossing the blood brain barrier.

In the post-genomic era, target identification for CNS disease has been progressing at a very rapid rate.  Mutations have been identified and pathways for discovery of disease modifying therapies have been opened.

Because fragment-based lead discovery centers on identification of simple, small and ligand efficient compounds, it is ideally suited to generate leads that have chemical properties consistent with marketed CNS therapeutic agents and crossing the blood brain barrier.  These compounds may be identified relatively rapidly and can be used in early target validation and proof-of-concept studies. 

Other methods such as high-throughput screening are sufficient for identification of lead compounds, but they tend to be high molecular weight and have other chemical properties less suitable for CNS penetration.