ABSTRACT
High-throughput screening is the random testing of large numbers of compounds on a biological system in order to identify those that affect the system in a desired way. In principle, a compound can be a small organic molecule, a natural product extract, a polypeptide, cDNA, siRNA, or other reagent that can provide the clues necessary to advance the hypothesis or objective in mind. The drug-screening process could include all of these strategies. However, this chapter is focused on the screening of small organic molecules with the objective of discovering and developing an orally bioavailable drug that can be taken chronically by patients suffering from ALS or other neurodegenerative diseases. The challenge is to develop a relevant biological system that has the potential to identify small molecules with activities likely to intervene in the disease process. Following that, the goal is to transform these chemical entities into safe and effective drugs.
