Apps for Rare & Neglected Diseases Sean EkinsCollaborative Drug Discovery, Inc
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..
ODDT as an App Experiment App for rare and neglected diseasesInspiration:P4C2011Scio2012Instigation: SharePA Dragons Den 2012 Data and molecules Publications, posters, talks Crowdfunding visibility biotech Mol Informatics, 31: 585-597, 2012 Drug Discovery Today, In press.
TB Mobile: Appifying data Problem addressed. Increase in whole cell screening – no idea of targetSame data - different delivery >700 molecules screened versus Mtb and their targets Similarity search Infer targetsNot a diagnostic, not a cure for thedisease - a research toolFunded by NIAID 2R42AI088893-02
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
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..)