Open source drug development

January 2018

Pharmacist using laptopOpen source doesn’t start and finish with software. Dr Alice E Williamson explains how an open, collaborative and community-based philosophy can benefit drug development too.

Since the discovery of penicillin in 1928, there have been huge advances in the treatment of infection and disease leading to improved outcomes for patients and longer life expectancy.

And yet, we still don’t have curative medicines or treatments for every disease and we haven’t even managed to eradicate diseases that are entirely treatable or preventable, including one of the world’s very oldest diseases: Malaria.

First documented almost 5,000 years ago, there are still over 200 million cases of malaria every year and around 429,000 fatalities, most of whom are young children (WHO World Malaria Report 2016 bit.ly/2gwezYl)

Recent reports of resistance to the best malaria medicines threaten to increase these figures dramatically, unless a new drug is discovered and distributed efficiently. And of course, Malaria is just one disease in urgent need of the very latest medicines.

A new way is needed

Finding new medicines is difficult and expensive. Once a good candidate has been discovered - often after years of research and synthesis and testing of 100s or even 1,000s of potential molecules - there are still several hurdles to clear and failure can strike very late in the race.

Because of multibillion dollar investments and the risks involved with drug discovery, the pharmaceutical industry is cloaked in secrecy in order to protect investors and maximise competition.

This secrecy is problematic. Competing companies could be working on identical molecules at the same time. This is wasteful even when drugs turn out to be successful, but in cases where the research fails this is clearly uneconomical and inefficient.

Additionally an industry that relies on considerable profits lacks the market incentive to work on cures for diseases that affect the world’s poor.

Going open source

Open source drug discovery aims to remove secrecy surrounding the invention and testing of new medicines and is emerging as a possible competitor to the pharmaceutical industry.

The Open Source Malaria (OSM) Consortium was founded by Associate Professor Matthew Todd in 2011, at the University of Sydney. OSM are looking for a new medicine for the treatment of malaria using open source principles.

Researchers from institutions across the globe are contributing to OSM and publishing their results and discussions on open internet platforms in real time. All data that is generated by the project is shared and there will be no patents.

Open Source Malaria aims to show that by removing secrecy and working openly it may be possible to find life saving medicines more quickly and cheaply, and hence provide low cost medicines for those who need them most.

The lack of secrecy has also lowered the barrier to participation in a drug discovery project. Undergraduates and even high school students have worked with OSM as part of The Breaking Good project to create new or existing medicines as part of an educational research project.

In recent years, news reports from countries including USA, UK and Australia have demonstrated that accessibility to medicines also affects patients in developed countries.

Sometimes, in diseases such as Alzheimer’s, for example, this is because no effective medicines exist, but all too often problems with accessibility are linked to unaffordable treatments.

Financial considerations

There are some medicines that are simply too expensive for patients and/or public or private healthcare systems. In particular, price-hiking of essential treatments such as Daraprim or EpiPen has captured the attention of the media, patients and politicians.

In 2016, a group of Sydney High School students worked with OSM to recreate Daraprim - a drug hiked in price from $13.50 to $750 in the USA - all in their high school lab and for next to nothing.

OSM are pioneering open source drug discovery as a viable alternative to the traditional model. The hope is that transparent communication and open data might pave the way for universal access to essential medicines.

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