ABSTRACT

Discovery and development of therapeutically useful drugs requires the concentration of resources. Traditionally, models of human disease are utilized for drug screening

purposes. Most frequently, these are animal or cell-based screening models representing a well-defined disease phenotype. Drug candidates are tested in these models in order to identify compounds that alter the disease phenotype. The majority of the therapeutically available drugs used in the Western world were developed along these “traditional” avenues. The main advantage of this screening method is that it uses a full biological system, and compounds passing this test are highly likely to be useful as therapeutic agents. Parallel to this, serious toxicity can also be ruled out. The major disadvantage of this approach rests in its inability to pinpoint target molecules, therefore, one cannot predict and dissect mechanisms of action and side effects. As a consequence, there are still a number of widely used drugs with obscure molecular mechanism of action.