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Jun 27, 2012

App Connects Rare Disease Researchers to Data

New mobile app aggregates chemistry data and other open science data to help advance research for a myriad of diseases.

App Connects Rare Disease Researchers to Data

With patient populations that are small and scattered around the world, tools such as mobile apps that facilitate global sharing of information could be invaluable for rare disease research. [© Aaron Amat - Fotolia.com]

  • Investigators of rare and neglected diseases can access some of the latest research in the field, plus data about the disorders themselves, through a recently launched free app for Apple devices.

    Sean Ekins, Ph.D., D.Sc., a consultant on computational drug discovery research, and Alex M. Clark, Ph.D., a designer of software for drug discovery scientists, have created Open Drug Discovery Teams (ODDT). The app aggregates chemistry data (such as molecular structure, reactions, and datasheets) and other open science data captured on a server through Twitter hashtags, as well as from RSS feeds, with each topic paired with a feed created via Google Alerts. ODDT also promotes crowd-sourced curation of that data through Twitter, allowing users to make annotations and assertions, as well as send tweets to endorse or disapprove.

    More than 7,000 rare and neglected diseases affect an estimated 350 million people worldwide, including more than 30 million Americans. ODDT collects data on a small but growing number that includes Chagas disease, HIV/AIDS, Huntington disease, leishmaniasis, malaria, Sanfilippo syndrome, and tuberculosis. The app also includes emerging topics such as drug repurposing and green chemistry.

    “I don’t think we could imagine it getting to the point where we have all 7,000 rare diseases in there. But there will be versions of the software ultimately in the future where people should be able to customize it and select their own topics,” said Dr. Ekins, a former Pfizer and Eli Lilly scientist, now VP of science for Collaborative Drug Discovery. “There’s a little bit of a method to our madness of putting some of these topics in there. We feel that there are places out there that will fund particular things.”

    Visibility will be a key challenge for ODDT. Of five foundations and individual researchers contacted by GEN, none were familiar with ODDT—including the National Organization for Rare Disorders (NORD), a nonprofit federation of voluntary healthcare organizations dedicated to helping rare-disease patients and groups serving them. At a NORD-co-sponsored forum on rare diseases during last week’s 2012 BIO International Convention, one highlighted topic was how patient organizations could partner with academic and industry researchers to speed therapy development.

    “New apps and other tools for sharing data hold a special significance for the rare disease community,” NORD spokeswoman Mary Dunkle told GEN. “Rare disease patient organizations are highly motivated to establish such partnerships. And, with patient populations that are small and scattered around the world, tools such as mobile apps that facilitate global sharing of information are potentially game-changers.”

    ODDT was launched April 12. Dr. Ekins began developing the app earlier this year for the “Dragons’ Den” session of the annual conference of the nonprofit Pistoia Alliance, on whose board he sits. The idea of a rare disease app arose after he met Jill Wood, who with husband Jeremy Weishaar co-founded Jonah’s Just Begun, a foundation created to cure Sanfilippo syndrome. Wood lamented how difficult she found it to learn more about her son Jonah’s inherited disorder, in which missing or defective enzymes prevent the body from breaking down glycosaminoglycans.

    After sketching out the app, he sent images to friends—one of whom was Dr. Clark. His two-year-old business Molecular Materials Informatics has developed the Mobile Molecular DataSheet and other chemistry mobile apps.

    Intent on covering rising costs of cloud server hosting, and on continuing to develop the app and improve its features, Drs. Ekins and Clark earlier this month turned to crowd-funding, presenting their project on Indiegogo. ODDT says most of the $5,000 it seeks will fund server hosting ($1,000 a year for three years), while the remainder will pay for additional features.

    “Future versions of the app will enable social networking features for organizing participants into teams, with various forms of communication and content management possible,” according to ODDT’s Indiegogo page. “We are also working on adding more sophisticated tools to enable drug discovery from the app, e.g., remote docking and chemistry searches.”

    Longer-term, Dr. Ekins said, ODDT hopes to develop disease-specific apps, as well as generate significant revenue by licensing the app to biopharma companies for internal collaboration, enabled within their firewalls on staffers’ and partners’ mobile devices. That makes sense since the companies carry out R&D increasingly through collaborations and for some diseases, through open-source partnerships.

    Another source of potential licensees would be rare-disease organizations ranging from disease-specific groups. To date ODDT has connected with Hannah’s Hope Fund for Giant Axonal Neuropathy, in addition to Jonah’s Just Begun. ODDT needs to continue outreach to these groups and others fighting rare diseases, and would well benefit from partnering with NORD, whose education, advocacy, research and patient assistance has been invaluable to rare-disease researchers.


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