Translational research--from gene to treatment: lessons from cystic fibrosis

Biomedical research is identifying a bewildering number of new targets for treatment of human disease. In order to translate this new knowledge into useful treatments, academics, charities and research councils will need to learn from the experience of large pharmaceutical companies who have been responsible for the majority of drug development over the past 50 years. Cystic fibrosis provides an ideal case study for the development of new treatments as there have been five examples in recent years and there are currently too many therapeutic targets for all to be pursued. Traditional patterns of drug discovery may need to be replaced by new models of funding and collaboration between academics, biotech, big pharma and charities. In particular, collaboration rather than competition between different interested groups should make the process of drug discovery cheaper and quicker. This applies particularly to gene therapy where new models of research organisation are emerging.