Open source: the future of medical discovery

There is growing recognition that no single research body or group has the know-how or resources to tackle the most widespread and persistent diseases. Fortunately, a growing number of scientists are engaged in open source innovation that removes barriers and creates a more transparent environment for medical discovery.

Based on the recognition that the whole can be greater than the sum of its parts, open source innovation is an interconnected way of working, in which sharing is everything. The benefits of this alternative approach are being most realised in diseases affecting the developing world, such as malaria and tuberculosis, where the science is complex and the commercial opportunity limited.

Among the pioneers of this new model is Associate Professor Matthew Todd, lead researcher at the University of Sydney and founder of the Open Source Malaria consortium. He believes a collaborative approach will help find medicines that will reduce the 200+ million new cases of malaria each year that cause an estimated 438,000 deaths annually, the majority of which are recorded in the developing world.

“Open source is a radically different approach that is likely to accelerate the discovery and development of new treatments. Using an open source model, the team has been able to access expertise, knowledge and equipment that we wouldn’t normally have access to, which is very valuable to the research,” said Professor Todd.

Professor Todd believes that reversing the tide on malaria requires the pooling of resources combined with bringing together the experience and expertise of scientists from different backgrounds and specialties. As part of his work, he has collaborated with the Tres Cantos Open Lab Foundation that gives independent researchers access to GSK resources, expertise and facilities to help research into diseases of the developing world.  GSK also opened to the public the structures of 13,500 chemical compounds that are capable of killing the parasite that causes malaria. 

GSK hopes that sharing information and working together will lead scientists to develop a drug for treating the mosquito-borne disease faster than the company could on its own.

Using an open source model allows scientists from different institutions and backgrounds to work together, both physically and remotely, drawing on each other’s strengths and know-how.

“Tres Cantos gave us existing data on malaria, which really helped as a starting point and gave us a head start. Medical researchers want to work with the best people, the latest equipment and have access to data. To support future research our own data is available to others in real time, which we hope will encourage debate and the discovery of new medicines,” said Prof Todd.

Set up in 2010, the Tres Cantos Open Lab Foundation is considered an unprecedented step in tackling the diseases of the developing world. Supported by GSK, it was the first time a global pharmaceutical company had helped set up an open lab that would unlock commercial resources and expertise to the broader scientific community. Six years on and the Tres Cantos Open Lab Foundation is an emerging success story and a thriving international hub for research into diseases of the developing world.

Scientists who visit the open lab in Spain often cite access to world-class facilities and the opportunity to collaborate with scientists working in the drug discovery field as the key benefits of this unique approach. The lab operates with the support and advice of a broad range of actors, including GSK, the Wellcome Trust, the European Union, and Medicines for Malaria Venture, as well as various other product-development partnerships and academic centres.

More than 100 scientific staff work at the Tres Cantos campus in a range of areas including medicinal chemistry, parasitology, mycobacteriology, pharmacology and toxicology. The combination of these facilities and expertise enables Tres Cantos to take care of all scientific needs of drug discovery and development from screening campaigns to clinical Proof of Concept studies (Phase IIb).

It is hoped that the goals of the Tres Cantos Open Lab Foundation will be achieved through collaborations where the complementary expertise and capacity, currently residing in the pharmaceutical industry as a whole, is made accessible to academic, biotech and other pharmaceutical industry scientists.

Dr Andrew Weekes, medical director at GSK Australia, believes open source innovation will increase in popularity as more pharmaceutical companies adopt the model.

“The use of open source models in medicine is now a reality that the scientific community is embracing. There is a growing consensus that an open source approach, with greater collaboration and transparency between the pharmaceutical industry and independent researchers, is the key to tackling diseases of the developing world,” said Dr Weekes.

With over 50 projects in the portfolio, Tres Cantos activities are starting to bear fruit in terms of publications, validation of novel therapeutic modalities, promising lead optimisation programs and leveraged funding from third party agencies.

This bold and flexible approach has positioned the Tres Cantos Open Lab Foundation as a dynamic research hub, helping to stimulate innovative research that could ultimately result in the discovery and development of new medicines.

“GSK is committed to researching new treatments for diseases that affect millions of people. There is still work to be done, but increasing openness and collaboration among scientists researching and developing medicines for the developing world is paving the way for further progress,” said Dr Weekes.

While it's still too early to evaluate the success of the Tres Cantos Open Lab Foundation in terms of drug approvals, this new approach has encouraged research into diseases of the developing world. There is palpable excitement that open source ways of working could herald a new age of research and development.

Proposals to the Tres Cantos Open Lab Foundation can be submitted at any time via www.openlabfoundation.org. Each submission is reviewed by the Foundation’s Governing Board and Trustees.

------------------------------------------------

This article was first published in Research Australia's Spring 2016 Inspire Magazine https://issuu.com/researchaustralia/docs/research_australia_inspire_spring20 

* Professor Matthew Todd’s research into malaria is being funded by the Australian Research Council and Medicines for Malaria, based in Geneva. He received an Open Lab Foundation grant for his research towards new medicines for tuberculosis.