Why Open Source Drug Discovery Needs a “Champion” Sean Ekins, Ph.D., D.Sc. Collaborations In...

Why Open Source Drug Discovery Needs a “Champion”

Sean Ekins, Ph.D., D.Sc.

Collaborations In Chemistry, Fuquay Varina, NC

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

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.

The Big Ask

• Can we do something that helps people become champions of Open Source Drug Discovery

The Problem – Opposites Attract

• Neglected & Rare Diseases

A handful of diseasesBiology well knownMillions die – affects poorNo incentive for pharmaTreatments need to be cheap (cents)

Thousands of diseasesBiology not well knownAffects 10s- 1000s per disease – affects richPharma targeting bigger rare diseasesTreatments need to be expensive $1M/yr pp

Bacteria, Parasites etc Genetic mutations etc

Neglected Diseases

• BMGF funded these and now more research efforts ongoing

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

Rare Diseases

Rare Diseases

• 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

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

Scientists Chase Funding Through Crowdfunding

Ethan raised to $25K using crowdfunding to fund research on methamphetamineOther bigger team projects have raised $100’sK

Parents Funding Scientists To Cure Their Children Though Crowdfunding

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

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

Proposal

Why don’t we apply Open Source Drug Discovery to Rare diseases as

well as neglected diseases??

Examples of Open Science and Tools to Share Openly

Linked Open data cloud 2011 (Wikipedia)

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

Challenges of Open Source Drug Discovery

Tools, Systems, IP concerns, organizational support

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?

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

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

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?

A mashup of..

• Crowdfunding• Crowdsourcing• Open Innovation• Open Source• Open Science• Precompetitive Initiative• Open Collaboration

Evolution of Systems Support for Open Source Drug Discovery

• Extending ODDT – can we improve the model?

It could be:An appA websiteA companyA technologyConsultancy

Components Needed to Support Open Source Drug Discovery

Fund ResearchGet Research Done by

CrowdExternalize Research to

Companies

Share Research Openly Precompetitive Collaboration

Find Collaborators Openly

Share Research Openly

Fund Research Externalize to CompaniesCrowdsource Research

Find CollaboratorsPrecompetitive Collaboration

Components Needed to Support Open Source Drug Discovery

Fund Research Crowdsource Research Externalize to Companies

Share Research Openly Precompetitive Collaboration Find Collaborators

Fund Research

Fund Research

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

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

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

Components Needed to Support Open Source Drug Discovery

Fund Research Crowdsource research Externalize Research to Companies

Share Research Openly Precompetitive Collaboration Find Collaborators

Externalize Research to Companies

Externalize to Companies

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?

Components Needed to Support Open Source Drug Discovery

Fund Research Crowdsource Research Externalize to Companies

Share Research Openly Precompetitive Collaboration Find CollaboratorsShare Research Openly

Share Research Openly

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

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

Precompetitive Collaboration

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

Components Needed to Support Open Source Drug Discovery

Fund Research Crowdsource Research Externalize to Companies

Share Research Openly Precompetitive Collaboration

Find Collaborators Openly

Find Collaborators Openly

Find Collaborators

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

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