1. Why Open Source Drug Discovery Needs a “Champion” Sean Ekins, Ph.D., D.Sc. Collaborations In Chemistry, Fuquay Varina, NC

2. Inspiration Jill Wood –Jonah’s Just Begun Lori Sames – Hannah’s Hope Fund Allison Moore – Hereditary Neuropathy Foundation Alex Clark – Molecular Materials Informatics Antony Williams – Royal Society of Chemistry

3. Insert disease of choice here Learning from OSDD TB and Malaria efforts Open Source Drug Discovery (OSDD) is the concept of drug discovery where all data and ideas are shared in real time, and anyone may participate at any level. This prior disclosure means that there will be no patents and that any technology is both academically and commercially exploitable by whoever wishes to do so.

4. The Big Ask Can we do something that helps people become champions of Open Source Drug Discovery

5. The Problem – Opposites Attract Neglected & Rare Diseases A handful of diseases Biology well known Millions die – affects poor No incentive for pharma Treatments need to be cheap (cents) Thousands of diseases Biology not well known Affects 10s- 1000s per disease – affects rich Pharma targeting bigger rare diseases Treatments need to be expensive $1M/yr pp Bacteria, Parasites etc Genetic mutations etc

6. Neglected Diseases BMGF funded these and now more research efforts ongoing

7. Neglected Diseases Many researchers chasing a relatively small amount of funding for a handful of diseases Difficult to treat, faster cures elusive Massive clinical trials needed

8. Rare Diseases

9. Very few researchers chasing >7000 diseases. Funding likely a long tail. Relatively easy to treat. At the forefront of gene therapy resurgence Only miniscule clinical trials possible

10. This is Jonah’s Story He needs a cure for MPS IIIC and fast Researchers just developed a KO mouse funded by Sanfilippo foundations globally Searching for small molecule therapy Working on gene therapy Treatment years away

11. Scientists Chase Funding Through Crowdfunding Ethan raised to $25K using crowdfunding to fund research on methamphetamine Other bigger team projects have raised $100’sK

13. Parents Funding Scientists To Cure Their Children Though Crowdfunding

14. The Rare Disease Parent Odyssey Diagnosis of child Try to find out about disease – papers behind paywall Try to connect with scientists Form not-for-profit Raise funds Fund Scientific research on disease Advocate for support from NIH, FDA etc Start a company Try to find a cure before its too late

15. Let me repeat….A Parent/ Patient Advocate with no Ph.D. can do all this Start a foundation Raise funds Become an expert on their/childs disease Set up a scientific advisory board Fund scientific projects Submit NIH grants Start a company Leverage social media, traditional media

16. Proposal Why don’t we apply Open Source Drug Discovery to Rare diseases as well as neglected diseases??

17. Examples of Open Science and Tools to Share Openly Linked Open data cloud 2011 (Wikipedia)

18. A Mobile App for Open Drug Discovery A flipboard for science #ODDT Embraced by rare disease advocates Getting people to share data openly is a challenge NIH SBIR reviewers want closed systems to preserve IP Developed with Alex Clark Open Drug Discovery Teams – brings data from Twitter and the internet together

19. Challenges of Open Source Drug Discovery Tools, Systems, IP concerns, organizational support

20. Benefits of Open Source Drug Discovery Same as for Open Source anything*?? $ Citations Kudos Awards Followers love * Admittedly it may be complex

21. What is needed? “Helpers” to take the weight off the parent/patient How to manage the complex data? How to make sense of the literature? How to foster collaboration? How to leverage what is learnt from other diseases? How scientists find funding or projects to work on? How parents find scientists to solve their problems? How to accelerate the process of discovery?

22. Support human Champions with software and systems Parents need expert advice Drug Discovery advice Preclinical advice Awareness of research outside of their disease This could be you here

23. Support human Champions with software and systems To share and store the data they are funding To network with others doing the same for other diseases Bring scientists and parents/patients together To rank scientists for funding their research Helpers

24. Extend from Rare to Neglected Diseases If we can do this for small diseases…. Why not do it for bigger diseases? Why not expand to common diseases? Could Pharma use as their open innovation platform?

25. A mashup of.. Crowdfunding Crowdsourcing Open Innovation Open Source Open Science Precompetitive Initiative Open Collaboration

26. Evolution of Systems Support for Open Source Drug Discovery Extending ODDT – can we improve the model? It could be: An app A website A company A technology Consultancy

27. Components Needed to Support Open Source Drug Discovery Fund Research Get Research Done by Crowd Externalize Research to Companies Share Research Openly Precompetitive Collaboration Find Collaborators Openly Share Research Openly Fund Research Externalize to Companies Crowdsource Research Find Collaborators Precompetitive Collaboration

28. Components Needed to Support Open Source Drug Discovery Fund ResearchCrowdsource Research Externalize to Companies Share Research Openly Precompetitive Collaboration Find Collaborators Fund Research

29. Fund Research functions Scientists post project ideas they want funded Parents / crowd / foundations fund research Parents post ideas for projects they want to see done Scientists and parents / foundations engage in dialog Go beyond the current crowdfunding sites – all money goes to science

30. Components of Champion Fund Research Get Research Done by Crowd Externalize to Companies Share Research Openly Precompetitive Collaboration Find Collaborators Get Research Done by Crowd Crowdsource Research

31. Crowdsource Research Functions Scientists and parents propose work they cannot do Basically a help wanted advert What expertise can people offer whether paid or free Innocentive but more open on challenges

32. Components Needed to Support Open Source Drug Discovery Fund ResearchCrowdsource research Externalize Research to Companies Share Research Openly Precompetitive Collaboration Find Collaborators Externalize Research to Companies Externalize to Companies

33. Externalize to Companies Functions So you want something beyond what the academics can do Contract Research Organization connection Can you connect to companies to get their compounds to screen?

34. Components Needed to Support Open Source Drug Discovery Fund ResearchCrowdsource Research Externalize to Companies Share Research Openly Precompetitive Collaboration Find CollaboratorsShare Research Openly

35. Share Research Openly Functions Blends ODDT with a database element Search by compounds, by text Store molecules Bring in open data from external sources Could imagine a closed component or some halfway point

36. Components Needed to Support Open Source Drug Discovery Fund Research Get Research Done by Crowd Externalize Research to Companies Share Research Openly Precompetitive Collaboration Find Collaborators Openly Precompetitive Collaboration

37. Precompetitive Collaborations Functions Why do we need an actual organization to do these things? Provide a point for discussion on the topic Get people to propose projects Project teams self organize Provide a means for delivering content/projects to be shared An index of collaborations

38. Components Needed to Support Open Source Drug Discovery Fund ResearchCrowdsource Research Externalize to Companies Share Research Openly Precompetitive Collaboration Find Collaborators Openly Find Collaborators

39. Find Collaborators Functions Can we involve preexisting similar tools? Enable people to search by topic, disease, Search grants, etc. Find scientists that can do the research Connect to them

40. Benefits of Integration “One stop shop” for all needs Crossover between topics and services Create a pipeline for Open R&D within an app Some users may have use for multiple features Cheaper to develop than 6 separate apps