Neglected and Rare Disease Drug Discovery Needs Open Data

This short symposium consisting of four presentations was organized by Sean Ekins, Antony Williams and Joel Freundlich. The following three presentations (and one no show) were brought to the spring meeting attendees on Sunday afternoon, March 16, 2014 in Dallas.

The first talk was given by Sean Ekins (Collaborative Drug Discovery) entitled “Looking back at mycobacterium tuberculosis mouse efficacy testing to move new drugs forward.”  Sean Ekins described the collaborative efforts with co-authors, including co-chairs of this session, Antony Williams (Royal Society of Chemistry) and Joel Freundlich (Rutgers University),  to curate and analyze a dataset of mouse in vivo information for tuberculosis research. He described various machine learning models based on 773 molecules, and presented external testing and molecular descriptor analyses. In addition, Sean highlighted the development of open source fingerprints used in the new version of TB Mobile and to build models that could be shared openly.

The second talk by Antony Williams described “Royal Society of Chemistry developments to support open drug discovery.”  In particular, Tony discussed their cheminformatics support of the Indian Open Source Drug Discovery effort working on tuberculosis. In addition, he highlighted their involvement with PharmaSea to help to identify the classes of antibiotics by searching the oceans. Finally, Tony reviewed the acquisition by RSC of MarinLit, a database of marine natural products research. This complements their natural product portfolio which includes Natural Product Reports and Natural Product Updates, and represents over 27,000 molecules to be added to ChemSpider.

The final talk by Evan Bolton (NIH) explored “How can PubChem be leveraged for neglected and rare disease drug discovery?” Evan listed NIH resources for rare diseases like the National Center for Advancing Translational Sciences (NCATS), the Therapeutics for Rare and Neglected Diseases (TRND), Genetic and Rare Diseases Information Center (GARD) and other programs.  He pointed out that it is not easy to get disease information in PubChem and that they are considering how to improve it. Evan proposed that scientists working on open source rare and neglected disease research could upload their data in PubChem.

This session may be one that could be expanded in future to track the developments in data and tools for rare and neglected diseases.

Sean Ekins, Symposium Co-Organizer


Collaborative Computational Technologies for Biomedical Research; Technologies for the Pharmaceutical Industry Series.

Editors: Sean Ekins, Maggie A. Z. Hupcey, Antony J.Williams.

Hardcover ISBN: 9780470638033 July 2011, 576 pages, $146, Wiley.