An approach that could reduce the chances of drugs failing during the later stages of clinical trials has been demonstrated by a collaboration between the University of Cambridge and pharmaceutical company GlaxoSmithKline (GSK).
This further suggests that human genetics can support the development of new therapies, and can offer insights into their safety profile early in the development process
The technique involves identifying genetic variants that mimic the action of a drug on its intended target and then checking in large patient cohorts whether these variants are associated with risk of other conditions, such as cardiovascular disease.
When developing a new drug for market, pharmaceutical companies must not only demonstrate that it is effective at treating a particular condition, but also that the drug does not have any adverse side-effects in patients. For example, the Food and Drug Administration, which approves all new medicines for use in the USA, has defined that any new anti-diabetic medicines need to demonstrate cardiovascular safety. However, in many cases adverse safety profiles do not become apparent until late in the drug development process, by which point millions – possibly even billions – of pounds will have been invested.
In a study published today in the journal Science Translational Medicine, scientists have provided a proof of concept that it is possible to use genetic analyses to demonstrate systematically at a very early stage whether a drug will alter the risk of developing other conditions.