Translational research in drug discovery: Tiny steps before the giant leap

The recent years have witnessed science taking gigantic strides and achieving milestones in the areas which were previously rendered to be difficult. Advancements such as the culmination of the Human Genome Project and the successful use of gene therapy have shown how science can be used to alleviate human diseases and benefit society. While basic scientific knowledge is increasing at a remarkable speed, the use of this knowledge to address real-world problems is still to catch the pace. The traditional drug-discovery approach involves a lengthy process of studying basic life processes, identifying a suitable target, followed by various checks to warrant the development of a prudent and potent drug molecule. The entire process often takes around 10-15 years to bring a molecule from the laboratory to the community. In contrast, the translation research approach first identifies a problem of clinical relevance and then sets out to identify the underlying mechanism and suitable drugs to combat the conditions. In short, translational research refers to the process of converting laboratory-based scientific knowledge to applications useful to previously unmet clinical needs. This chapter envelops the basic understanding of translational research-based drug discovery with an emphasis on the opportunities and challenges present in this field. We have also discussed the successful advancements seen in the field of translational drug discovery.