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 ExperimentApp for rare and neglected diseases

ShareData and molecules

Inspiration:P4C2011Scio2012

Instigation:PA Dragons Den 2012

Crowdfunding visibilityPublications, posters, talks

biotech

Mol Informatics, 31: 585-597, 2012Drug Discovery Today, In press.

TB Mobile: Appifying data

Not a diagnostic, not a cure for the disease - a research tool

Funded by NIAID 2R42AI088893-02

Same data - different delivery >700 molecules screened versus Mtb and their targets

Similarity searchInfer targets

Problem addressed. Increase in whole cell screening – no idea of target

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..)