Why Open Source Drug Discovery Needs a “Champion”

Yesterday I attended the Southeast Venture Philanthropy Summit held in Chapel Hill. Attendees included VC, philanthropy types, disease foundations (big and small), bioscience organizations, scientists from all over the country. There was of course the usual 1:1 partnering sessions which I did not attend, instead sitting through panels on personalized medicine, philanthropy, investment in the southeast, diabetes/regenerative medicine, oncology, neuroscience, research, funding and product continuum.

What I learned was the following: Bioscience brings in a staggering  $59bn in NC volume, 237,665 jobs , 23% growth in life science in the last decade. There is also a huge chasm ($100M’s  vs less than a $1M) between the amount of money major disease foundation have at their disposal compared to the ultra rare diseases. It is staggering that some disease foundations can give big pharma huge amounts of money to do research on their diseases. Witness the Cystic Fibrosis Foundation paying Pfizer $58M. This has already been questioned by some. But there is certainly a case here of the haves and have nots..Money talks.
I also met several more folks from rare disease foundations including Josh Sommer from the Chordoma Foundation. Lori Sames from Hannah’s Hope Fund and Allison Moore from the Hereditary Neuropathy Foundation who also participated on panels.
 The night before there was a small discussion bringing together about a dozen scientists, rare disease advocates, patients and companies to discuss the development of a ‘Rare disease center’ in the Research Triangle Park area. There was definitely a divergence of opinion as to what could be done from a virtual institute to bricks and mortar  which could be a neutral Institute to do some of the relatively mundane things like support GLP animal labs..Some discussion surrounded avoidance of silozation. But it was pretty clear that any effort would be looking for support from venture philanthropists in the hope that we could change the way we do drug discovery collaboration across organizations. Several of us suggested that rare diseases need earlier work and the model provided by the recent rare disease competition might also work on a local scale with various groups, providing services. Perhaps the importance of having a rare disease institute might be missed by some, but there will likely be pressure on government to work on other diseases if some of the ongoing rare disease gene therapy studies get approval. There are also over 7000 rare diseases and to date there are treatments for just a few hundred diseases – so something has to be done to give the patients hope. Another advantage of having an institute of some kind is that there are economies of scale to be gained as all foundations are building similar infrastructure and doing similar things but for different diseases (e.g. biobanks, registries etc).  There is NO national rare disease institute, and perhaps no hospital for rare diseases like St Jude Childrens Hospital.

I have been thinking of ways to change the situation for quite a while and have even proposed an institute for rare diseases that should be informatics driven and require a group that could centralize and direct the various ongoing academic collaborations funded by the rare disease groups. It would use the various existing databases to mine for compounds as potential treatments for rare diseases. It would also call upon the HTS screening resources at local universities and would leverage existing infrastructure and postdocs. It would partner with any existing rare disease groups at local universities as well as those involved in gene therapy treatments. Rare disease patient groups would provide funding and access to their complete researcher networks. Pharmaceutical companies would provide access to compounds, and databases for mining. The NC Biotechnology Center would provide seed funding and commercialization expertize to spin out any resulting IP in the best interests of the patient led organizations, funders and the Universities who would be equal partners in this enterprise. We would partner on the clinical side with area hospitals to enable the rapid translation of any therapeutics. The goal would be the creation of a world class center for rare diseases in North Carolina, becoming a magnet for global researchers, clinicians, patients and companies, and be self-funding within 5 years. More importantly, our goal would be to find cures for multiple rare diseases in this time frame.

Another parallel track I have been pursuing is creating tools that could act as a champion for rare diseases.
The idea is a first iteration mashup of :
•Open Innovation
•Open Source
•Open Science
•Precompetitive Initiative
•Open Collaboration
So in the spirit of openess I am putting these ideas out there to the community. Because if it could help the rare disease groups, it could help neglected diseases and perhaps others too. I honestly think with so many diseases, the only way we are going to be able to impact trying to find more treatments is by some flavor of open source drug discovery and at its heart there will need for more collaboration.



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  1. Ethan Perlstein (@eperlste) says:

    Excellent post, Sean! How many other states/regions could aspire to be rare disease research hubs?

  2. sean says:

    Good Question Ethan, not so sure location is as important as just having a go to place that is world renowned for dealing with rare diseases and doing the research.

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