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Cambridge Centre for Neuropsychiatric Research


Application of proteomics in the discovery phase could positively impact on target identification, target validation, lead compound prioritisation and in vivo or ex vivo efficacy screening in preclinical models. Potential applications in the development phase include the generation of surrogate protein markers for drug efficacy and for validation of preclinical models of human diseases. Perhaps most importantly, proteomics can be used to identify protein signatures for diseases and use these signatures to derive new biochemical assays for disease diagnosis and to monitor drug efficacy in clinical trials. Proteomics can also be used to decipher interconnecting protein pathways of physiological processes which can help to improve our understanding of disease mechanisms and pave the way for improved rational drug design.

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The ultimate application of proteomics in drug discovery is to identify biomarkers in a readily accessible body fluid such as serum, which can be correlated with drug efficacy or toxicity. Such biomarker signatures could also be used as surrogate markers to help predict the response of individuals to treatment and allow tailoring of the therapy to achieve optimal efficacy without toxic side effects. Likewise this approach could be used to facilitate identification of patient sub-populations who will respond favourably to a drug. Therefore, the use of proteomic profiling technologies will provide a better understanding of drug efficacy, metabolism and toxicity, reduce costs for drug manufacturers and result in safer drugs for patients.