Guest post: Opening up drug discovery for neglected diseases

Chemsitry and chemical biology associate professor Michael Pollastri uses a drug repurposing approach to identify new treatments for neglected tropical diseases. Photo by Brooks Canaday.
Chemsitry and chemical biology associate professor Michael Pollastri uses a drug repurposing approach to identify new treatments for neglected tropical diseases. Photo by Brooks Canaday.

Chemsitry and chemical biology associate professor Michael Pollastri uses a drug repurposing approach to identify new treatments for neglected tropical diseases. Photo by Brooks Canaday.

This post was written by associate professor Michael Pollastri.

Neglected tropical diseases are a collection of infectious diseases that primarily affect the poor in developing nations, and many have extremely subpar treatments. Since the patients can’t pay for expensive new drugs, the for-profit pharmaceutical industry cannot devote their research and development resources. I worked in the industry for a number of years, and decided to develop an independent academic operation that could focus on these diseases. Indeed, an increasing number of research groups in the academic and non-profit environment are putting their focus on NTD drug discovery.

I know from my experience working in the industry that the typical practice is to keep research internalized and secret. This is so that drug companies can keep a competitive advantage, file patents, recoup research costs and eventually generate profits. This practice, because of its long-ingrained nature in industry, becomes a habit, and as a result is often also applied even to NTD drug discovery in the non-profit environment. But I recently started asking whether this really makes sense. Does keeping trade secrets really benefit anyone when there are minimal profits to be made? We have experienced first-hand that these secretive practices inevitably can lead to duplication of effort. For example, after spending several months developing a project to discover new malaria drugs, we discovered (via casual conversation) that another research team had done exactly the same thing, and decided that the results that they were getting were not worth pursuing further. Had we known that this other team was working in the same space, we would have engaged with them to divide and conquer the work, and shared and compared our plans and results with them.

Unfortunately, this situation is common, and is truly unconscionable in a resource-poor research environment like NTD drug discovery. There is simply no money to waste.

However, it requires a totally different mindset to perform research out in the open, where new molecules are synthesized and tested for their utility as drugs, and all the data is shared in the public domain. Taking a page from the open source model of software development (think Linux, or Firefox…), more groups are starting to look at open models of collaboration. One such group is the Open Source Drug Discovery-Malaria (OSDD-Malaria), where a multinational team of collaborators agree to share the workload of synthesis, testing, and modeling, with the agreement that all data will be posted to the public as the data is generated, in real time. Anyone who wishes to participate can dial into regular project meeting teleconferences, and can contribute research efforts and expertise. It is an exciting model that is quickly gaining traction, and is sure to change the ways in which we think about how to do research in NTDs.

However, not everyone is totally comfortable with the fully open model of sharing, especially those of us who had formative experiences in pharma. I wondered whether there was another way to reduce redundancy and improve information sharing, and have started establishing an alternative. In contrast to the fully open model (where all data is shared with everyone, everywhere), we are forming a consortium of participants who agree to openly share data amongst each other. The consortium will be open to anyone who wishes to participate, provided that they agree to fully transparent data sharing within the consortium, and agree to keep each other’s data private. In this way, research teams can share information and results with people they know and trust, and keep some modicum of control over their own data. This will help reduce duplication of efforts, and will provide a mechanism for participants to easily release their data to the general public when they are ready to do so, expanding the value of their research results far beyond our consortium!

What next? We are currently recruiting research groups working on NTD drug discovery that would like to try out our “Hybrid Open” discovery model for research. Our Laboratory for NTD Drug Discovery will pre-populate the database with our own results in African sleeping sickness, malaria, leishmaniasis, and Chagas disease, and we invite other research teams like ours to participate.

In this way we hope to both make an impact on discovering drugs for these NTDs, and also (perhaps more importantly) influence the ways in which such research is done.

The Laboratory for NTD Drug Discovery is a component of the Northeastern University Integrated Initiative in Global Health. For more information about this initiative and how you can get involved, see www.northeastern.edu/globalhealth.