Drug development in the pharmaceutical industry is a chain of well-defined steps that should lead to bioactive molecules ready to enter clinical trials. The role of bioinformatics in this process will be briefly described. Bioinformatics has a role to play at any stage of the process but is essential at the initial phase of target selection and validation. The concept will be illustrated on a new emerging target.
Genome wide association studies have pointed to Fat mass and obesity associated (FTO) gene as one of the most relevant non monogenic genes implicated in the obesity phenotype. The FTO association to obesity has been replicated in numerous studies on various populations suggesting that FTO might become an attractive drug target. However before embarking on a costly drug development path, the target needs to be validated. While the association is statistically robust, the physiological role of FTO emerges only slowly and the biochemical mode of function remains elusive. Bioinformatics analysis provided crucial clues for establishing experimentally that FTO gene codes for a DNA processing enzyme. Further progress can be expected from pooling all available and dispersed information. Bioinformatics can generate unexpected hypothesis but their experimental testing will remain always essential.