Streamlining the drug discovery process
Successful mapping of the entire human genome with the ambitious Human Genome Project has created the potential for therapies targeted at the specific genes involved in a disease. However, the genomics healthcare revolution has fallen short of expectations in part due to the complexity of potential targets and to a lack of coordination among different gene technology platforms. Delay in drug development leads to increased costs of pharmaceuticals to end users and often unmanageable waiting time for new and more effective treatments on the part of patients. European researchers sought to combine the different expertise of several companies at specific steps in the drug discovery process to expedite the successful identification of gene targets for drug therapy. The ‘Disease therapeutic targets, multidisciplinary parallel validation, accelerated drug discovery’ (PONT) project was designed to exploit aptamers, or small strands of DNA that bind to specific target molecules with very high affinity. The overarching goal was to use aptamers to identify in a streamlined fashion compounds with promising pre-clinical activity at new or tricky gene targets. Application of the aptamer-based compound screening in identification of compounds for treatment of hepatocellular carcinoma (HCC) showed activity of selected compounds in cell culture and in mice. Commercialisation of PONT expertise should bring compounds with promising pre-clinical activity to clinical trials and eventually patients in a reasonable timeline at reduced costs to companies, consumers and healthcare institutions.
