From genes to effective drugs for neurological and psychiatric diseases

Abstract The description of the human genome sequence represents a major step towards complete understanding of human biology and disease mechanisms. The impact on drug discovery will not be immediate because even the almost complete list of putative drug targets provided by the genome sequence does not improve our ability to select and validate targets, the real bottlenecks in drug discovery and development. In neuroscience, the impact will be through the completion of lists of likely drug targets, such as G-protein-coupled receptors and ion channels, the advancements in identifying disease genes, and, in combination with mRNA or protein array technologies, the identification of systems affected in intricate diseases such as depression and schizophrenia. The genome sequence data will also help to identify transporters that regulate drug access to the brain. The surprisingly small number of genes and putative drug targets is likely to increase the competition over intellectual property.