An Open Pharma Approach Targets a Terrible Disease

Mycetoma patients
Image credit
Soad Abdelrahman, a medical lab technician, checks on the amputated stump of patient infected by mycetoma. Image by Neil Brandvold.

One reason neglected diseases like mycetoma are so neglected: The lack of market incentives for pharmaceutical companies to develop new drugs.

A new project targeting the flesh-eating, bone-destroying disease mycetoma throws out the traditional market-driven approach and opens the discovery process to scientists worldwide. Launched on Tuesday by the University of Sydney, Erasmus MC, and the Drugs for Neglected Diseases initiative (DNDi), the MycetOS (Mycetoma Open Source) project relies on an Open Pharma approach to “discover compounds that could lead to new treatments for patients suffering from fungal mycetoma (eumycetoma), a devastating disease for which current treatments are ineffective, expensive, and toxic.”

Tossing out the Big Pharma model of closed research and steeply priced drugs that recover the R&D costs, MycetOS leads drug discovery efforts through “community-driven, in-kind scientific contributions and a robust, fully transparent online presence,” say the organizers. Ideas and testing results will be open and shared in real time via an open-access database.

Simultaneous with the launch, the project announced encouraging results from its efforts to screen 800 compounds for potential drugs targeting eumycetoma.

In an interview with GHN, Matthew Todd, a MycetOS leader and a University of Sydney associate professor, explains the project’s novel approach, its risks and its applicability to other diseases.

Mycetoma was selected as the Untold Global Health Story of 2015 and covered in "The Most Neglected Disease," a GHN series by journalist Amy Maxmen.


Why did you and your colleagues decide to take an Open Pharma approach toward new treatments for fungal mycetoma?

Science is at its best when it is shared and anyone can contribute. By opening up the project, it becomes possible for a truly formidable team to assemble—those researchers already working on the disease alongside others who might be a perfect fit for the research but are unaware of the disease. An open source approach welcomes everyone—students through to professors, researchers in big pharma through to philanthropists. If you want to beat a tough disease like mycetoma, this is the way to do it.

So, you’ve already screened 800 compounds against the fungus that causes eumycetoma. Tell us about the promising hits. What’s the next step for those?

The hits are very nice—based on known compounds, but with plenty of room for improvement. Wendy van de Sande's results suggest that molecules that work in simple assays ("in vitro") are not necessarily what we need for good treatments in more realistic assays ("in vivo"), and we really want to act on that discovery (for any drug discoverers out there we need to lower the logD). Generally though, 3 things are needed: a) to take a look through the database of compounds we have access to and see what we should screen next; b) to see whether anyone would like to make a molecule or two, perhaps as part of an undergrad lab program; and c) to see whether anyone has any promising compounds stored away in a fridge somewhere.

What risks are involved in the Open Pharma approach? A lot of researchers might be worried about opening up their research so much, not to mention using reddit and github for sharing data.

Just like Wikipedia, anyone can use anything disclosed by MycetOS for any purpose (including to make money) provided the original project is cited. This creates a community with enormous momentum where there is this impetus to share and contribute, rather than take for one's own gain. People participating will disclose their results in real time online, which is the fastest method of publication I know of, and one that clearly associates a result with an author. So, there's no real possibility of scooping, and the team can write up their results quickly together in a way that welcomes all authors. Open source software has been working in this way, remarkably productively, for decades.

There are 2 risks. First, we as MycetOS must ensure the project's sustainability, to ensure that everything that we build together, lasts. Second, the openness means you can no longer rely on patenting and secrecy to bring a medicine to market in the usual way. There are ways around that (that I and others have described, and DNDi are pioneers in this area), but we're short on precedents.

How successfully can this model be translated to other diseases?

Well, I've been involved in a project that has been running successfully using this ideas, Open Source Malaria, that recently published its first paper. The approach is ideally suited to neglected diseases, given the so-called “market failure” associated with some potential medicines. Open source is well-suited to rare diseases where we'd be looking at patient cohorts that are about the same size as the clinical trial cohorts. Al Edwards [CEO of M4K Pharma] is making great strides in this area, for example. In the coming years, I'm excited to test the applicability to some of the great threats we face in society—in cancer, or dementia. Areas that are so big that they require a genuinely new approach to compete with that adopted by the pharmaceutical industry.

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