June 18, 2013: Translation in Three Dimensions: The 3Ds of NCATS

Much has been written about scientific and operational problems that limit our ability to develop and deploy new and effective interventions to improve human health. Much less has been written about potential solutions to these problems — and that’s where NCATS comes in. We focus on developing these kinds of system-wide solutions.

However, we also know that a goal to develop technologies and models that might improve some aspect of the translational process is simply not enough. Rather, we must demonstrate that these solutions improve the efficiency or effectiveness of an intervention through real-world examples. And if we are successful, we cannot assume that others will adopt these new technologies; instead, we must disseminate the translational advances proactively so that the broader research community can understand, apply and benefit from them.

To illustrate these principles, consider the Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) program. Today, NIH and NCATS announced the first project awards for this crowdsourcing effort to explore potential treatments in eight disease areas using existing pharmaceutical compounds.

New Therapeutic Uses addresses several specific hurdles in the translational process:

  1. The urgent need for medicines to treat the several thousand known diseases that do not have effective therapies approved by the Food and Drug Administration (FDA).
  2. The large number of partially developed molecules that failed partway along the lengthy and difficult path to making a new medicine.
  3. The complicated process of negotiating agreements between parties that want to work together, particularly pharmaceutical companies and academic investigators.

To address these obstacles, we seek potential solutions by gathering broad input about New Therapeutic Uses from across the research community (see the new Request for Information to submit your feedback). New Therapeutic Uses is an outcome of a meeting (PDF - 114KB) of NIH, pharmaceutical and academic leaders in 2011. The proposed approach was for NIH to serve as a matchmaker between pharmaceutical companies, which had molecules that were safe but not effective for their original disease target, and academic scientists, who had new ideas to use these molecules to fight other diseases.

To realize this vision, NIH worked to develop (the first D) two components: (1) standardized template agreements to help NIH, academic scientists and pharmaceutical companies work together efficiently and (2) a mechanism to “crowdsource” ideas for new therapeutic uses from the broad academic community.

To demonstrate (the second D) that academic-industry matchmaking was feasible and effective, NIH worked with eight pharmaceutical companies that volunteered compounds as test cases in the New Therapeutic Uses pilot program, launched in May 2012. The companies made detailed information on 58 molecules available on the NCATS website, and they committed to releasing needed data and supplying each compound at no cost to NIH or the investigators.

Ultimately, nine proposals were chosen for funding and announced today. These awards, supported by the NIH Common Fund, go to investigators at academic institutions across the country. During the next two to three years, these scientists will collaborate with the pharmaceutical companies that developed the molecules to test new therapeutic uses in patients with common and rare diseases.

When these studies are complete in the next few years, we will know if these ideas truly could become new treatments. But already this bold experiment has produced general insights that we can disseminate (the third D) to the research community to improve the translational process.

By applying the 3Ds to the “wisdom of the crowd” in the New Therapeutic Uses program, NCATS has served as the crucial matchmaker for fundamentally new scientific partnerships to bring more treatments to more patients more quickly. Stay tuned for more exciting developments!

Christopher P. Austin, M.D.
National Center for Advancing Translational Sciences