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Crowdsourcing Therapeutic Molecules for Drug Discovery

Posted on by Dr. Francis Collins

An assortment of pills, vials, and bottles containing liquid, against white background

Caption: The Discovering New Therapeutic Uses for Existing Molecules pilot program matches researchers with pharmaceutical compounds to explore new treatments for patients

Developing a drug takes time and money: on the average, around 14 years and $2 billion or more. More than 95 percent of the drugs fail during development. Even those that go all the way to large and expensive clinical trials in humans frequently don’t make the cut—perhaps because they weren’t quite as effective as they were supposed to be, had undesirable side effects, or didn’t align with the developer’s business priorities. But some of these compounds may have surprising therapeutic properties that have not yet been fully exploited. It would be a wasted opportunity not to take another look at them and test them for effectiveness in other conditions.

For that reason, our National Center for Advancing Translational Sciences (NCATS), with financial support from the NIH Common Fund, launched a pilot program to discover new therapeutic uses for existing molecules. Today we are awarding $12.7 million to nine academic institutions to reexamine a collection of compounds developed by major pharmaceutical companies and test them as treatments for diseases, both common and rare: from alcoholism and Alzheimer’s disease to Duchenne muscular dystrophy and schizophrenia.

This new program is a collaboration between NIH, academia, and industry. Industry collaborators—AbbVie (formerly Abbott), AstraZeneca, Bristol-Myers Squibb Company, Eli Lilly and Company, GlaxoSmithKline, Janssen Pharmaceutical Research & Development, L.L.C., Pfizer, and Sanofi—made 58 of their compounds, all extensively researched and tested for safety, available. Relevant data about these 58 molecules was shared with biomedical researchers and, as we had hoped, scientists from across the country recognized how these molecules might interact with various disease pathways or disorders they are studying. They submitted some remarkably innovative ideas; NIH carried out a rigorous review and is now distributing the first awards. With this approach, we are hopeful that we can give these compounds a second chance, while making important discoveries for several diseases.

I am very excited about this pilot program for several reasons. As part of the design of the program, NIH developed template agreements to streamline the legal and administrative process for academic-industry collaboration—and already it’s clear that that strategy is saving months if not years of negotiations. In addition, this is our first experiment with “crowdsourcing” of therapeutic opportunities—giving the entire biomedical community access to highly active compounds and related data, and enabling anyone to make new connections to disease. I expect this model to yield some great science, and I’m optimistic it will also speed the development of new drugs to patients.

Who knows what therapeutic talents these 58 molecules possess? These “Therapeutics Discovery” awards will help us find out.


Discovering New Therapeutic Uses for Existing Molecules

Library of Industry-Provided Agents

Therapeutic Development Process

Template Agreements

NIH support: National Center for Advancing Translational Sciences; Common Fund


  • Kevin Engholdt says:

    Great progress and great note. May be need to check numbers on “2 Billion” for new drug – it’s more on the end of $800 million (

    I’d like to see if there is an opportunity to allow for natural ingredients to received funding for clinical trial work. The funding would be dramatically smaller with lower safety concerns with more recent data available and anecdotal data from different historical cultures using the natural ingredients. $12.7 million will go no where in terms of a daunting funding history like $2B or $800 million, no matter where your financial source comes from. With a natural ingredient, small startups with evidence based medicine could dramatically prove out their ingredients with $12.7 million. There would need to be a better IP protection system of course, but that money would be highly valued in the natural realm rather than in the pharmaceutical realm.

    Truly, I think it’s a great program, room for more improvement, but still a great program. Best of luck to all of you!

  • Sery Kim says:

    VERY cool Francis! Good luck!

  • Kirk Maxey says:

    Great idea – but one practical question, and one way to build on this program’s momentum:

    1. Where will NIH get the compounds? Most pharma companies will not be willing to go back and prepare a couple of kilos of their long-abandoned clinical candidate for you, so…who makes them for this program?

    2. For the future, simply make it a requirement of any Phase II Clinical Trial, that the applicant shall, in addition to all of it’s other IND obligations, prepare and submit to the FDA 100g of their small molecule candidate or 1 gram of any biologic. Multiply X10 for Phase III.

    NIH should contract to have this growing collection of compounds plated and sold as a screening library. The revenue generated could be used to offset the sequester.

    • Moderator says:

      Mr. Maxey, thanks for your question and feedback on the New Therapeutic Uses program. The pharmaceutical industry partners are providing the compounds, as well as the placebos, needed for project clinical trials.

  • JM says:

    Really interesting, thanks!​

  • Sunil Aggarwal, MD, PhD says:

    Dear Dr. Collins,

    Why not do something similar for herbal cannabis? … Empiric treatment trials, controlled trials, and comparative effectiveness studies could all be done with a very cheaply producable herbal medicinal plant. Pain, neurodegenerative diseases, seizure disorders, cancer, many targets are out there. We just need NIH to give some support to the botanical CAM community, rather than always looking down the pharma pipeline.


  • BFP says:

    It’s very useful. Thanks you.

  • Billy Bonilla says:

    Hi! Great reading your blog.

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