Investing in future drugs to defeat disease

Young children in sub-Saharan Africa are the main victims of the age-old killer malaria. Picture: Getty

Young children in sub-Saharan Africa are the main victims of the age-old killer malaria. Picture: Getty

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LIFE science experts working to help colleagues cross the ‘valley of death’ and deliver life-saving treatments can change face of medicine says Mike Ferguson.

Malaria kills more than half a million people each year, mostly children under five in sub-Saharan Africa.

Colleagues at the University of Dundee, working in our drug discovery unit, and their collaborators around the world, have just published research that describes a new class of antimalarial compound that may become a new medicine.

Named DDD10107498, it needs to undergo further study and testing in patients but, uniquely, it has the potential to prevent, cure and stop the transmission of malaria. These exciting properties warranted it being declared a candidate by the Medicines for Malaria Venture (MMV), the not-for-profit product development partnership organisation in Geneva that co-ordinates almost all malaria drug research and development worldwide. MMV recently partnered DDD10107498 with European pharmaceutical giant Merck Serono for development.

In this case, the scientists first made a drug that kills the malaria parasite and then figured out exactly its “drug target”, the precise component of a cell, in this case the malaria parasite, with which the drug engages and through which it exerts its beneficial effects. The alternative route to a medicine is to first select a potential “drug target” for a particular disease and then make a drug that engages it. Both approaches are equally valid and require largely the same science and technology, and both are taken in the drug discovery unit at Dundee.

Founded in 2006 at the university and led by Professor Paul Wyatt, the unit has almost 90 staff and has a turnover of about £6 million a year, funded through organisations such as the Wellcome Trust, The Gates Foundation, the Medical Research Council and MMV. Its remit is to tackle neglected tropical diseases like malaria, tuberculosis, African sleeping sickness, leishmaniasis and Chagas’ disease and other unmet medical needs closer to home, including cancer, cystic fibrosis, eczema, psoriasis, and rheumatoid arthritis.

All this sounds a bit like the activity of a pharmaceutical company so a good question is: “Why are you doing this in a university?” The answer is to help academic innovation cross the “valley of death”.

The “valley of death” is a phrase often used to describe all that can go wrong between ideas and their successful application, often referring to the gap between having a potentially exploitable discovery from fundamental research and providing enough validation or proof-of-concept to make it attractive enough to be developed for application.

The translation of an innovative new drug target into a new medicine is a long and expensive process with much attrition, right up to and including rejection in advanced clinical trials that may have cost hundreds of millions of pounds. Pharmaceutical companies have traditionally spotted their drug targets from the academic scientific literature. However, as the amount and complexity of life sciences research increases, picking the winning innovations, ironically, becomes harder.

Our view is that we should help de-risk some of the best life science research by having our drug discovery unit work closely with scientists, from Dundee and our sister Scottish and UK universities, who have discovered potential new drug targets. Through this close collaboration, exciting life sciences discoveries are taken through a stringent drug discovery process in order to translate potential drug targets into a smaller number of de-risked drug targets.

These can then be advanced into the clinic by pharmaceutical industry partners or through the creation of new spin-out companies. As a recent example, collaboration between Professor Irwin McLean, an expert in genetic diseases at the University of Dundee, and the drug discovery unit led to the partnering of an innovative drug target for cystic fibrosis with the multinational pharmaceutical company Pfizer.

In summary, universities are excellent at identifying the mechanisms of disease and identifying potential drug targets that could to lead to new therapeutics. There is a national, and moral, imperative to innovate by translating these opportunities towards patient benefit and job creation. In my opinion, universities themselves need to be part of this translation – not at the expense of their blue skies research, without which there is nothing to translate, but as well as. At the University Dundee, we have chosen to invest heavily in early stage drug discovery, in the form of a drug discovery unit, as a principal mechanism for translation. It sits inside one of the highest-ranked university life science departments in the UK and interacts with several more with significant successes to its name, not least the latest antimalarial describe above.

• Professor Mike Ferguson is Regius Professor of Life Sciences at the University of Dundee www.dundee.ac.uk

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