On target for a new era of drug discovery?

Almost half a century ago, the world entered an apparent golden era of drug discovery. Driven by a rapidly advancing understanding of biological processes in the body and medicinal chemistry, a generation of game-changing new medicines emerged – many for previously untreatable illnesses – and life expectancy around the globe increased dramatically as a result.

This period of unprecedented R&D productivity was not destined to last and despite our best efforts, there were fewer scientific breakthroughs over the course of the 1990s and early 2000s than had been expected. Coupled with this, early advances in our understanding of genomics – the field of science focusing on DNA and genes – were not transformational in improving our ability to develop new medicines, as many experts had predicted.

Right now, only 10% of medicines in development ever reach patients. The vast majority of potential medicines fall by the wayside because we have not properly understood the link between the biological process in the body that our drug is targeting and the disease we want to treat. [1]

But that is beginning to change. The number and scope of diseases that scientists are tackling is increasing, and a wave of exciting new therapies is emerging. This includes medicines that fundamentally alter the course of – and in some cases, offer the potential to cure – disease.

It’s in our genes

Driving this change is a transformation in our understanding of our genes and what they tell us about our body – and why we get ill.

By studying in detail the genetic profiles of patients with disease, scientists are gaining a better understanding of the biological processes in the body that are connected to disease. This increased knowledge of human genetics is helping scientists to better predict whether a particular process in the body is involved with a disease – and target their research accordingly.

Lon Cardon, GSK’s Senior Vice President, Target Sciences, said: “Discovering and developing new medicines is exciting and innovative but also time consuming, high risk, and incredibly difficult. Anything we can do to choose better starting points in that journey can have a massive positive impact on our success rate in developing innovative new medicines for patients.”

Teamwork keeps us on target

To harness the potential of genetic understanding in targeting our research, we’ve created a dedicated team of scientists – the Target Science team, led by Lon. They work in partnership with our own Therapy Area aligned scientists and with external academic researchers too, to inform our development of new medicines. 

This teamwork is vital to capitalising on these scientific advances. As part of a collaboration called Open Targets, which we established with the European Bioinformatics Institute, the Wellcome Trust Sanger Institute and biotech company Biogen, we’ve launched an open access “Google”-type search engine that extensively searches, evaluates and integrates the mountain of genetic and biological data now being generated.

“We believe that harnessing the potential of “big data” and genome sequencing through this collaboration could help us dramatically improve our success rate for discovering new medicines,” said Lon.

We have also established a groundbreaking research institute in Seattle with leading professor of genome sciences, Dr John A. Stamatoyannopoulos. Known as the Altius Institute, this is another important initiative pioneering the latest genetic technologies to help us see inside cells’ operating systems at unprecedented scale and pace.

And most recently we established a major research initiative with UK Biobank and Regeneron to begin sequencing genetic data from 500,000 anonymised volunteers in UK Biobank’s healthcare resource. This sort of genetic evidence is revolutionizing scientific discovery and drug development and our hope is that the research initiative will help us to identify new starting points for our medicines of the future.

“This new era of drug discovery should be defined by openness,” said Lon. “Joining together as a scientific community to discover new targets – in collaborations like Open Targets, Altius and the UK Biobank – will help us create a fertile landscape for creating new medicines.”

[1] Validating therapeutic targets through human genetics; Nature Reviews Drug Discovery 12, 581–594 (2013)

[2] The support of human genetic evidence for approved drug indications; Nature Genetics 47, 856–860 (2015)