presentation given at joint TM Forum and Pistoia Alliance meeting in Oct 2012

1. Apps for Rare & Neglected
Diseases
Sean Ekins
Collaborative Drug Discovery, Inc

2. Problems can be opportunities
• Smaller research communities
• Less investment than Cancer, CV etc
• Published data inaccessible to parents
• But..
• Apps existing for symptoms disease mgmt
• Tight knit communities
• Willing to collaborate
• Try different approaches
• Approaches generalizable, cheap, disruptive..

3. ODDT as an App Experiment
App for rare and
neglected diseases
Inspiration:
P4C2011
Scio2012
Instigation: Share
PA Dragons Den 2012 Data and
molecules
Publications,
posters, talks Crowdfunding visibility
biotech
Mol Informatics, 31: 585-597, 2012
Drug Discovery Today, In press.

4. TB Mobile: Appifying data
Problem addressed. Increase in whole cell screening – no idea of target
Same data - different delivery >700 molecules screened versus Mtb and their targets
Similarity search
Infer targets
Not a diagnostic, not a cure for the
disease - a research tool
Funded by NIAID 2R42AI088893-02

5. Logical next steps
• ODDT customizable by 7000 diseases
• Imagine each rare disease community as app – with access to
private and public data (DDT)
• Make more data available as apps or publish in apps.
• Reuse data – mash up – find new ways to use- apps facilitate
• e.g. GSK just announced 200 TB cpds to be public
• NCATS 58 molecules from Pharmas- no structures
• Show examples of drug discovery by mobile device
• Show how hypothesis from one disease can apply elsewhere
• Demonstrate repurposing by app

6. What is needed
• Break down paywalls to papers
– Neglected disease apps could increase visibility of
older content to parent advocates and researchers
• Breaks down walls to data
– Only recent data in PubChem etc..rediscovery of old
drugs published previously for TB..(e.g. PNAS Gold et
al.)
• Find sponsors who promote openness (Burroughs
Wellcome, small disease charities..)