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Director’s Corner

December 21, 2016: Celebrating Five Years of Innovation in Translational Research

Christopher Austin

NCATS celebrates its fifth birthday this month, providing us the opportunity to reflect on the remarkable progress of the last five years and share our plans for an even more transformative future. On Dec. 23, 2011, NCATS was officially established. It was conceived as a catalyst for innovation, with the audacious mission to transform translation from an empirical process to a predictive science and thereby get more disease treatments and cures to more patients more quickly. The NCATS team-based “3Ds” paradigm of developing, demonstrating and disseminating technologies and approaches that improve our scientific understanding and operations of translation has produced insights and advances well beyond what I thought possible five years ago. I will briefly reprise a few of these advances before explaining why I think this has happened and why it makes me so hopeful for the future.

Our first accomplishments were existential: As a new Center with a unique mission, we first defined translation and the new field of translational science, which is NCATS’ focus. We defined the translational science spectrum, from basic discovery to public health. Our next accomplishments were cultural, establishing that “translation is a team sport” and that translational scientists focus on how their work is “carried across” to other disciplines or stages in the development process. Scientific and operational advances followed in rapid succession, many of which are featured on our fifth anniversary website page. Among those:

  • In the pre-clinical area, NCATS chemists working on a multidisciplinary team identified a specific ketamine metabolite that likely holds the secret to ketamine’s rapid antidepressant action without the drug’s dissociative, euphoric and addictive properties. The findings could lead to the development of a safer and more practical ketamine-derived antidepressant that could lift depression within hours instead of weeks.
  • NCATS’ Clinical and Translational Science Awards (CTSA) Program has evolved rapidly to increase the effectiveness of clinical research, including networking efforts to achieve increased quality and efficiency in the conduct of multisite studies, improved trial recruitment, and innovative training of translational science researchers. Examples include the NCATS Streamlined, Multisite, Accelerated Resources for Trials (SMART) Institutional Review Board (IRB) Reliance Platform to harmonize and streamline ethical review for multisite studies and the case study of a trainee at the University of Miami CTSA Program hub who developed a new technology to improve cochlear implant surgery.
  • We began bold efforts to dramatically increase the pace of translational progress in rare diseases, which are disproportionately devastating and costly for patients, families and the nation as a whole due to the severity of these diseases, the difficulty of obtaining a timely diagnosis, and the lack of treatments. NCATS’ efforts to speed drug development have focused especially on rare disease patients. For example, NCATS investigators developed a sophisticated combination drug screening platform to more quickly narrow down a long list of potential drug combinations and find those with the most potential to help patients. Our new patient and caregiver videos help spread awareness of these diseases and what it’s like to live with them.
  • As part of our efforts to provide the research community with tools and resources to facilitate the process of translation, NCATS launched its Biomedical Data Translator program earlier this year. The goal is to develop a groundbreaking, publicly available resource that brings together all biomedical and health data types using an informatics platform approach. Ultimately, researchers will have access to a powerful system that will enable discovery of complex relationships among data, helping scientists better understand disease and develop new treatments for patients.

As I reflected on this remarkable progress, I recalled that five years ago, the problems that NCATS is now conquering were often called “intractable.” More than anything, what NCATS’ first five years have shown us is that such problems are not intractable but that solutions do require a fundamentally different approach that is unfailingly team based, innovation centered and patient focused. But our work has just begun. In our first five years, we have built a cultural, scientific and operational foundation for the myriad opportunities and challenges to come. Thank you for your partnership on this journey, and please stay with us as we aim to further accelerate the pace of progress, improving health through smarter science.

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