Where do you start when developing a new medicine?

A pioneering public-private research initiative between GSK, the European Bioinformatics Institute (EMBL-EBI) and the Wellcome Trust Sanger Institute is to harness the power of ‘big data’ and genome sequencing to improve the success rate for discovering new medicines. The new Centre for Therapeutic Target Validation (CTTV) will aim to address a wide range of human diseases and will share its data openly in the interests of accelerating drug discovery.

The CTTV aims to use the almost daily advances in cutting-edge genetic research to help researchers in that crucial first step in exploring new medicines – finding where to start. Target validation is about clearly defining the role that a biological process plays in disease before developing a new drug to tackle it. Currently, an estimated 90 per cent of compounds entering clinical trials fail to demonstrate the necessary efficacy and safety requirements, never reaching patients as medicines. This is often because the biological target for a drug is not well understood.

Dr Ewan Birney, Associate Director and Senior Scientist at EMBL-EBI, has been appointed as Interim Head of the CTTV. Dr Birney, who brings with him significant knowledge about genomics and bioinformatics, will develop a work programme to steer the research activities of the centre’s scientists.

“The Centre for Therapeutic Target Validation is a transformative collaboration to improve the process of discovering new medicines,” says Dr Birney. “The pre-competitive nature of the centre is critical: the collaboration of EMBL-EBI and the Sanger Institute with GSK allows us to make the most of commercial R&D practice, but the data and information will be available to everyone. It is truly exciting to apply so many different areas of expertise, from data integration to genomics, to the challenge of creating better medicines.

CTTV scientists will combine their expertise to explore and interpret large volumes of data from genomics, proteomics, chemistry and disease biology. The new approach will complement existing methods of target validation, including analysis of published research on known biological processes, preclinical animal modelling and studying disease epidemiology.

This new collaboration draws on the diverse, specialised skills from scientific institutes and the pharmaceutical industry. Scientists from the Wellcome Trust Sanger Institute will contribute their unique understanding of the role of genetics in health and disease and EMBL-EBI, a global leader in the analysis and dissemination of biological data, will provide bioinformatics-led insights on the data and use its capabilities to integrate huge streams of different varieties of experimental data. GSK will contribute expertise in disease biology, translational medicine and drug discovery.

A cornerstone of the collaboration is an agreement among the collaborators that sequence data and information gathered within the CTTV will be shared to benefit the broader scientific community, after basic quality control checks to ensure consistency with the data-sharing guidelines of both institutes. The centre will also seek publication of data and information arising from CTTV projects in peer-reviewed scientific journals. Once the centre is fully established, the collaborators will actively seek to attract new interest from other companies and academic institutions in the centre with the aim of expanding its activities.

The CTTV will be supported by up to 50 researchers drawn from the three founding organisations and will be based on the Wellcome Trust Genome Campus near Cambridge, UK. Researchers will use EMBL-EBI’s Innovation and Translation suite, which received substantial support from the UK government in 2012, and the laboratories of the Wellcome Trust Sanger Institute. The CTTV has been established with significant contributions of resource, skills and platform technologies from each of the three founding organisations and a multi-million pound contribution by GSK to fund an initial wave of projects.

Comments by the founding organisations:

Patrick Vallance, President of Pharmaceuticals R&D at GSK, says: “Target validation is one of the greatest challenges in drug discovery. We need to understand better the mechanisms in our body related to disease to improve how we can develop the most effective medicines.

“By changing our business model, taking a more open-minded approach to sharing information and forging collaborations like the Centre for Therapeutic Target Validation, we believe there is an opportunity to accelerate the development of innovative new medicines.

“The UK leads the fields of genome sequencing and bioinformatics and I am delighted that GSK has the opportunity to collaborate with two of its premier research institutions.”

Professor Sir Mike Stratton, Director of the Wellcome Trust Sanger Institute, says: “Advances in genomics have led to a rapid increase in the availability of drug targets, providing enormous opportunity but also posing the problem of how we best convert this new knowledge into medicines. The challenge we now address is to identify those new targets with the greatest relevance to human disease which, in turn, will undoubtedly increase the speed and efficiency in which new medicines can be developed.”

Professor Dame Janet Thornton, Director of the European Bioinformatics Institute, says: “Maximising our use of ‘big data’ in the life sciences is critical for solving some of society’s most pressing problems. This collaboration gives us an opportunity to translate this complex information into biomedical discoveries.

“As biology becomes increasingly data-driven, pre-competitive collaborations such as the Centre for Therapeutic Target Validation have become crucial for improving efficiencies, driving down costs and providing the best opportunities to deliver beneficial results.”