What does it take to discover a new medicine?

The views and opinions expressed in this article are those of the author and do not necessarily reflect the official policy or position of the University of Birmingham

“The fundamental issue here is that early research into new targets and drugs often involves some fairly routine and repetitive work, which is a long way away from the excitement of finding novel targets and novel chemistry leads.”

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As the USA FDA website states, ‘Research for a new drug begins in the laboratory’. Before the turn of the century, the majority of this research took place in the extensive R&D facilities of global pharmaceutical companies. However, since the mid-noughties, the pharmaceutical drug discovery model has changed, with an evolution towards academic partnerships. Why is this? Well, as was always the case, much of the “blue-skies” innovation in understanding disease mechanisms and defining target biology takes place in our academic institutions. However, the translation of the best academic knowledge into new, value-adding medicines isn’t straight forward with roadblocks to progress occurring at all stages in the journey from bench to bedside.

One key obstacle to translation is that most academic research is supported by grant funding with the primary goal of generating new knowledge. Success is measured by high impact publications, which are themselves critical to secure the next round of grant funding.

The fundamental issue here is that early research into new targets and drugs often involves some fairly routine and repetitive work, which is a long way away from the excitement of finding novel targets and novel chemistry leads. For example, a thorough evaluation that identifies and links specific molecular targets to a given disease, the likely physical and pharmaceutical properties of any potential drug, the potential safety profile, and an idea of which cohort of patients would benefit from the intervention, is usually required. The issue emerges when trying to get this vital work funded since such investigations are too routine and therefore often unattractive to grant-funding bodies.  On the other hand, with many of these questions unanswered, it is often too early or too risky for pharmaceutical companies to take a serious interest in a project.

So how are things changing? At the University of Birmingham, we realised there was a real need for funding to get scientific innovations out of the lab and into the drug development pathway and so we did a thorough search of all potential drug-opportunities across the whole of the University. We then worked out which were the most viable and what early work was needed to develop them to a point where they would be of potential interest for funding from pharmaceutical companies. The work we did was as varied as the projects themselves, so we devised a map for identifying successful projects called ‘TCP’: is it the right Target, is it the right Chemistry and are these the right Patients? The answers to these questions have guided our selection processes.

Following the TCP map, we have identified a portfolio of really interesting new drug projects. We are attracting interest from several international pharmaceutical companies with more companies in the queue to take a look at what we have. And, of course, we are delighted to help them with whatever they are looking for, whether it’s new ideas in cancer, arthritis, pain management or in womens’ health, to name just a few.

Seeing all this come together in the Birmingham Drug Discovery Hub makes me very proud to be working here, alongside outstanding academics, clinicians and research facilitators. And it’s also exciting to note that we are now collaborating with our colleagues at the Universities of Dundee, Nottingham and Edinburgh to ensure we throw the net even wider in our search for great ideas to translate into potential new drugs that will deliver patient benefit.

So, in answer to the question, what does it take to discover a new medicine, the answer for me is great scientific ideas that are rigorously screened to enable sound decision making, before committing the valuable resources needed to move projects towards clinical development.

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