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2016 Director’s Messages

Select a 2016 message from the list below:


Jan. 28, 2016: A New Year’s Resolution for Accelerating Progress in Translational Science

Some of the most common New Year’s resolutions are health-related, and NCATS is no exception — but we aim for resolutions that will be more durable than average, since evidence shows that most resolutions are broken by February! 2016 began on a wonderful note, with a fiscal year budget boost from Congress that will help greatly in our efforts to translate scientific discoveries into tangible interventions that improve human health. A few NCATS resolutions follow to presage an exciting year ahead.

Our first New Year’s resolution is to expand successful initiatives, such as our Tissue Chip for Drug Screening program. Since the launch of this program in 2012, collaborating researchers have successfully developed tissue chips representing over a dozen organs and are now working to integrate them into multi-organ systems. The next step will be to establish testing centers where researchers can use select groups of compounds and drugs to validate the tissue chips’ performance in mimicking drug responses in humans. This testing will bring the tissue chip program closer to the goal of creating therapeutic development models that more accurately reflect human physiology and thus serve as more reliable testing systems to determine safety and efficacy of new compounds.

NCATS also resolves to continue building local, collaborative and network capacity in the Clinical and Translational Science Awards (CTSA) Program. Following up on our 2015 funding opportunity announcements, we plan to issue new awards this year to support greater quality and efficiency in multisite clinical research, improved clinical trial recruitment, and collaboratively developed approaches toward accelerating the clinical and translational research process.

Another 2016 NCATS resolution is to ramp up research on rare diseases, through collaborative efforts to study the commonalities and underlying molecular causes across related disorders. Rare diseases are devastating and costly for patients, families and the nation as a whole, partly due to the severity of these conditions but also because diagnosis can be difficult and is often possible only well after symptoms have appeared. Our new approach to rare diseases research, including our flagship Rare Diseases Clinical Research Network, promises to accelerate the pace of progress in rare diseases.

Brand-new initiatives are on the horizon as well, including 3-D bioprinting of human tissues for drug screening and increasing access to compounds and toxicity data to make identification of new therapeutics easier and more predictable.

We’re already working on our resolutions, and we expect 2016 to be an exciting year for NCATS! Please stay in touch to learn more about and take part in our transformation of translational research as we work to get more treatments to more patients more quickly.

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


Feb. 23, 2016: Celebrating Progress at Rare Disease Day 2016

During the weeks leading up to the annual Rare Disease Day at NIH, I have been reflecting on my career path in clinical medicine and therapeutics development. Three decades ago, when I was training to be a neurologist, I too often had to deliver the disheartening news to my patients with rare neurological diseases that I couldn’t do much, if anything, for them. We simply didn’t know enough about the diseases, and very few had any treatment. My desire to change that status quo led me from the clinic into the basic research laboratory, and then to the field that connects them: translational research.  

Like other young physician-scientists at the time, I suspected that genetics was the key to deciphering rare diseases and discovering therapeutics for them. Thirty years later, that hunch has turned out largely to be true, albeit with caveats. As exemplified by the Human Genome Project, we know exponentially more about genetics, biology and physiology than ever before. But most of the diseases that were untreatable when I was in training remain so today, so the promise of this spectacular science has yet to reach most patients in need.

Translation of fundamental research into interventions has lagged, especially for the more than 6,500 rare diseases that affect humans. Today, only a few hundred rare diseases have any FDA-approved treatment. Though the pace of rare disease therapeutic development has increased in recent year — of the drugs approved by FDA in 2015, almost half were for rare disorders — the large number of currently untreatable diseases calls for a fundamentally different approach to rare disease translation.  The longstanding “one disease at a time/one organ at a time” therapeutic development model is both inadequate and unnecessary given the science of 2016.

Through its programs and initiatives, NCATS is focusing on rare disease commonalities and shared underlying molecular causes. By identifying commonalities across diseases, scientists have the potential to accelerate the development and demonstration of treatments for multiple diseases at once. 

We also must disseminate the knowledge gained and support awareness, advocacy and collaboration among researchers in the public and private sectors, patients, patient advocates, and policymakers. That’s the purpose of Rare Disease Day at NIH, which is sponsored by NCATS and the NIH Clinical Center. This year’s February 29 event — Leap Day is rare and special, as are rare disease patients — will feature presentations from NIH leadership, leading scientists, government representatives, patient advocacy groups, and caregivers; poster sessions and exhibits; an art show; and tours of the NIH Clinical Center. I urge you to register today to join us!

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


March 21, 2016: A New Horizon for Discovering Rare Disease Therapies

Last month, in anticipation of Rare Disease Day at NIH, I wrote about the importance of rare diseases research and NCATS’ multifaceted approach to tackling these conditions. I’m delighted to report that the NIH event was a standing-room-only, record-breaking success and that the unprecedented number of participants included members of Congress as well as leaders in academia, industry, government and patient advocacy.

The increasing interest in Rare Disease Day speaks to the research community’s growing commitment to understanding rare diseases and the process by which treatments for them are discovered and deployed. This month, I will focus on specific examples that illustrate this encouraging trend.

I often speak about the number of rare conditions that have no Food and Drug Administration (FDA)-approved treatment, so the movement of a disease into the “treatable” category for the first time is always a cause for celebration. This occurred recently with the first FDA approval of a drug therapy for lymphangioleiomyomatosis (LAM), a rare, progressive, often fatal lung disease that primarily affects women of childbearing age. It is characterized by buildup of abnormal tissue in the lungs that blocks airflow and makes breathing increasingly difficult. With support from NCATS’ Rare Diseases Clinical Research Network, a team led by researchers at Cincinnati Children’s Hospital Medical Center demonstrated in a clinical trial that sirolimus (Rapamune), a drug already approved to prevent organ rejection in patients receiving transplants, stabilized LAM patients’ lung function, reduced their symptoms, and improved measurements for quality of life. Those results led to the FDA’s approval of sirolimus as the first drug indicated for the treatment of LAM.

The approval of sirolimus for LAM is another success for repurposing, which is the process of using existing therapies to treat new diseases based on commonalities in the molecular causation of clinically disparate conditions. This approach is a central theme of Recursion Pharmaceuticals, a company being supported by NCATS’ Small Business Innovation Research and Small Business Technology Transfer programs. Recursion scientists use automation and robotics to carry out high-throughput drug repurposing screens for multiple rare diseases in parallel, and currently they are working to validate 12 promising repurposed drug candidates for several medical conditions.

Translation is a team sport, and NCATS’ goal of getting more treatments to more patients more quickly will be realized most effectively by teamwork with fellow rare disease researchers all over the world. This vision was the genesis of the International Rare Diseases Research Consortium (IRDiRC), founded in 2010. This month, I became the new chair of the IRDiRC executive committee; in fact, I am writing this message on a plane coming back from a tremendously exciting meeting of that committee in Lyon, France. I look forward to keeping you informed about our projects and progress, both domestic and international, in the months to come.

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


April 28, 2016: Training the Translational Science Leaders of the Future

Biomedical scientists likely remember their graduate school days as being filled with learning the basics of their scientific disciplines, how to critically read journal articles and write their own, and other best practices for contributing new knowledge to their fields. However, too few learn how to translate their discoveries into interventions that improve health.

This is one key reason translation is currently a slow and inefficient process. A critical part of NCATS’ mission is to strengthen the translational science workforce through innovative education and training initiatives, mentored research programs and career development support for translational investigators.

One exciting initiative is the Translational Science Training Program (TSTP) at NIH. This collaborative effort between NCATS and the NIH Office of Intramural Training and Education offers NIH intramural postdoctoral fellows and graduate students the opportunity to learn more about the science and operation of the full spectrum of translation. The annual program features a mixture of classroom-based instruction, small group discussions and a hands-on workshop to introduce NIH trainees to fundamentals of the therapeutic development process, professional skills development, clinical trial terminology and career exploration in translational science.

Follow-up participant surveys show that the program has been very successful, with multiple alumni going on to establish successful careers in translational science. In the NCATS “3Ds” vernacular, we developed the TSTP, demonstrated its utility and, as will be outlined in a forthcoming publication, plan to disseminate the course as a model to be used by academic institutions and other translational research organizations to educate and train tomorrow’s translational scientists.

The NCATS Clinical and Translational Science Awards (CTSA) Program supports two formal clinical research training awards at CTSA Program institutions, as well as a wealth of training resources available to all investigators interested in translational research. You can read about a scientist who used some of those resources to open new doors at the University of Miami Clinical and Translational Science Institute (CTSI), a CTSA Program hub. As a new assistant professor at Miami, Suhrud Rajguru, Ph.D., attended a “boot camp” similar to the TSTP offered through the CTSI and used several CTSI resources to develop a device to improve cochlear implant surgery. To help fellow researchers at Miami benefit from the knowledge he gained in moving his device to the clinic, he is participating in the NCATS-supported “train-the-trainer” program to bring an Innovation Corps (I-Corps) short course to Miami to provide entrepreneurship training to other translational scientists.

These young scientists — TSTP alumni, CTSA Program trainees and leaders like Rajguru — are paving the way to a new era in which biomedical researchers will begin their careers equipped with the tools and knowledge to more effectively translate basic science into interventions to improve human health.

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


May 24, 2016: A Biomedical Data Revolution for Accelerating Translation

NCATS accelerates translational science through initiatives that push the boundaries of biomedical research to revolutionize the way we study diseases and discover treatments. I am very excited about a new such signature initiative: NCATS’ Biomedical Data Translator program, called Translator for short.

As I have previously discussed in this space, one of the roadblocks to translation is the siloed nature of biomedical research across diseases and disciplines, which impedes discovery of commonalities across diseases and the teamwork required for successful translation. The ultimate vision for Translator is an informatics platform enabling interrogation of relationships across the full spectrum of data types, from disease names, clinical signs and symptoms, to organ and cell pathology, genomics, and drug effects. Collaborative teams of innovators of unprecedented scope are being summoned to make this vision a reality in our Center’s recently released funding opportunity announcement (PDF - 134KB).

Currently, a tremendous amount of data — from biomedical research, disease classifications, health records, clinical trials and adverse event reports — is available and useful for understanding health and disease and for developing and identifying treatments for diseases. Unfortunately, these very rich data types are individually incomplete and exist in different locations and are often in different scientific languages that are not compatible or “translatable” to others. NCATS’ goal with Translator is to merge all this information together in order to provide new ways of understanding the basis of diseases and new approaches to treatment.

This approach will enable the bridging of the current symptom-based diagnosis of disease classification with research-based molecular and cellular characterizations that can be targeted by various preventative and therapeutic interventions. This multiyear, iterative effort will produce a publicly available, comprehensive, relational, N-dimensional Biomedical Data Translator that will be useful for researchers worldwide to facilitate their translational efforts.

For the first step of the program, as outlined in the FOA, NCATS is using its Other Transactions Authority (through the Cures Acceleration Network) to invite innovative proposals for addressing the architecture needs to build the Translator and to assess its technical feasibility.

Translator is an unprecedented effort to push beyond the “business-as-usual” incremental approach to translational science and bring about rapid, high-impact change that will get more treatments to more patients more quickly.

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


June 21, 2016: Streamlining the Institutional Review Board Approval Process to Accelerate New Treatments for Patients

A colleague here at NCATS received devastating news last year: Her 4-month-old grandson had been diagnosed with a rare brain tumor. The cancer failed to respond to first- and second-line chemotherapy, but molecular testing of the tumor revealed a target for which there was a potential treatment. The drug, however, was not widely available to children so young, so the boy would need to be enrolled in a clinical trial. The good news came that there was such a trial, raising the family’s hopes for an effective treatment. But then they learned the bad news: The trial was stalled due to problems getting multisite institutional review board (IRB) approvals. The child’s mother crystalized the issue with her response: “I don’t understand. Don’t they know brain tumors don’t wait for IRBs?”

One of the longstanding challenges for clinical research is the process of obtaining human-subjects approval from multiple IRBs for clinical studies. Researchers cannot begin recruiting participants until an IRB reviews and approves the protocol for conducting research on human subjects. When the same protocol is reviewed by many different IRBs in a multisite study, the redundancy of this process can lead to long delays. No disease waits for IRBs or other obstacles that slow clinical research. Time, for my colleague and her family — and so many others — really can mean a life.

NCATS’ mission is to develop solutions to these kinds of systemic, previously intractable translational science problems. Through its Clinical and Translational Science Awards (CTSA) Program, NCATS develops, demonstrates and disseminates approaches to improve and streamline clinical studies. One way to do this and to provide more consistent, high-quality IRB reviews is for all sites participating in a multisite clinical study to rely on the review of a single IRB. Such “IRB reliance” would mitigate a major roadblock to the translation of research discoveries into health benefits.

Given its potentially transformational impact, we at NCATS made the creation of a national single-IRB platform a top priority, and I am now proud to announce the fruit of these labors: the launch of the NCATS Streamlined, Multisite, Accelerated Resources for Trials (SMART) IRB Reliance Platform. The SMART IRB Reliance Platform is designed to provide a flexible prototype that can easily be used by any clinical research network or even a single investigator wishing to conduct a multisite clinical study. It will serve as a roadmap to help implement NIH’s requirement that all NIH-funded multisite clinical studies use a single IRB.

SMART IRB Reliance Platform builds on the successful experiences of several NIH central IRB initiatives and those of multiple regional CTSA Program networks, including a CTSA Program demonstration project called IRBrely. The bold, game-changing SMART IRB concept never could have come to fruition without the collaborative, team and trust-based relationships that CTSA Program investigators have built over many years in their efforts to improve clinical and translational science. As we like to say around here, “translation is a team sport,” and never was this more true than in the development of SMART IRB.

NCATS will continue to collaboratively develop, test and disseminate SMART IRB as a trusted national standard for single IRB reliance for clinical studies. This advance promises to help the countless patients — such as my colleague’s grandson — who so desperately need new therapies.

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


July 15, 2016: Engaging Patients Is Crucial to Improving the Translational Process

Engaging patients at all stages of translation is crucial: Their input and perspectives as members of the research team help provide insights, focus, urgency and connectivity that can be instrumental in making the development, testing and deployment of new interventions more efficient and effective.

NCATS views the science of patient engagement as a key area for exploration and innovation. As a related communications endeavor to help make researchers and the public more aware of the challenges that rare disease patients and their families face, NCATS has produced 10 video stories that I hope you will take the time to view and share.

In the pre-clinical arena, James Inglese, Ph.D., director of NCATS’ Assay Development and Screening Technology Laboratory, forms collaborative relationships with numerous rare disease organizations to launch early-stage drug discovery efforts. These agreements involve foundation support for postdoctoral researchers with expertise in a particular disease to work with Inglese, a world expert in developing drug testing systems for novel therapeutic targets. Most recently, Inglese announced such an agreement with the Global Foundation for Peroxisomal Disorders and the Wynne Mateffy Research Foundation, which fund research on a group of rare genetic conditions that appear at birth or in early life, causing multi-organ dysfunction and life-threatening complications. The aim is to identify a promising potential treatment.

On the clinical side, NCATS requires all Rare Diseases Clinical Research Network (RDCRN) consortia to include patient groups as full partners on their research teams, an innovative approach that helps achieve greater success, including in design and enrollment of clinical studies. The RDCRN Coalition of Patient Advocacy Groups develops and shares best practices, and the RDCRN website includes a web-based contact registry for patients who may be interested in participating in RDCRN clinical studies. NCATS also engages patients through its Genetic and Rare Diseases Information Center (GARD), which provides comprehensive information about rare and genetic diseases to patients, their families, health care providers, researchers and the public. GARD provides accurate, up-to-date information about ongoing research, symptoms, treatment options and other details.

In addition, NCATS continues to collaborate with organizations that share our commitment to understanding and implementing the science of patient engagement. For example, NCATS Health Scientist Shelley Brown, Ph.D., recently spoke at a FasterCures workshop focused on better understanding the influences that are helping or hindering patient-centered activities, identifying and prioritizing tools and templates to reduce resistance and remove practical barriers to patient engagement, and shaping the future agenda of collaborative activities to enable greater patient centricity.

These are just some of the ways in which NCATS is committed to engaging patients, their families and the advocacy organizations that serve them, to form the teams that will get more treatments to more patients more quickly.

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


Aug. 19, 2016: Tackling the Adherence Challenge at the Culmination of the Translational Odyssey

An oft-cited component of NCATS’ mission is getting more treatments to more patients more quickly. It’s tempting to assume that once a potentially life-saving therapy is developed in the laboratory, shown to be safe and effective in human trials, and enters clinical use, its health benefits are assured. But in fact, getting an intervention — a new drug, for example — to those who need it can take more than a decade, with many lives unnecessarily lost as a result. One reason for this is that patients often don’t take drugs as prescribed. This problem — called medication nonadherence — can be a major hurdle at the far end of the translational spectrum.

Patients struggle with adherence for numerous reasons, such as unpleasant side effects, hard-to-understand instructions, complex dosing schedules, and high prescription costs. Experts estimate that,  on average, medication adherence is around 50 percent. Approximately 20 percent to 25 percent of prescriptions are never filled; of those that are, 20 percent are not consumed.

The potential consequences of nonadherence are serious. The resulting hospitalizations and poor health outcomes cost the U.S. approximately $290 billion each year. In addition, poor adherence prevents researchers from properly assessing drugs in clinical trials, contributing to a high failure rate for these studies. Clinical trials can cost $1 billion or more, and they most often fail because researchers cannot prove a therapy’s effectiveness or safety. Just as concerning, noncompliance during a trial may cause researchers and regulators to miss harmful side effects and allow unsafe drugs to enter the market.

As in all other areas of translation, NCATS is developing, demonstrating and disseminating new technologies and approaches to improving medication adherence. For example, through its Small Business Innovation Research (SBIR) program, NCATS funded the work of a startup company, AiCure, to develop a remarkable artificial intelligence smartphone application that visually confirms a patient’s identity and medication intake. In other words, the technology helps ensure that the right patient takes the right medication at the right time. This video shows how the app works. AiCure now is under contract with five of the top 12 U.S. pharmaceutical companies to use the application to improve medication adherence in clinical trials. The team also intends to offer the technology for outpatient clinical care.

Another example is from the South Carolina Clinical and Translational Research Institute at the Medical University of South Carolina (MUSC). With support from NCATS’ Clinical and Translational Science Awards (CTSA) Program, MUSC researchers are developing, studying and implementing mobile technology to improve patient adherence to medication and therapy. Their work includes the design of special electronic pill bottle caps, medication dispensers and blood pressure monitors that use Bluetooth technology paired with smartphone apps to monitor and enhance adherence to treatment regimens.

Through these and other efforts to increase the efficiency of translation, NCATS is working to ensure that all patients receive the full benefits of promising new interventions as rapidly as possible.

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


Sept. 29, 2016: A New Phase for NCATS’ Tissue Chip Program

Just five years ago, the idea of engineering a human-body-on-a-chip to predict drug safety and efficacy seemed more like science fiction than reality. But today, NCATS’ Tissue Chip for Drug Screening program, a collaborative initiative with the Defense Advanced Research Projects Agency and the Food and Drug Administration (FDA), is closer than ever to attaining that vision.

In the first two years of the program, researchers developed individual tissue chips that could mimic human organ structure, function, and physiological and drug responses more accurately than traditional cell- and animal-testing methods. Currently, there are tissue chips for the heart, liver, blood-brain barrier, blood vessels, kidney, gastrointestinal system, nervous system, and female reproductive system, as well as models of adipose (fat) tissue, tumors and metastasis.

During the next three years, teams of scientists joined forces to connect individual organ chips. Researchers collaborated to refine the chips and integrate them into systems that can mimic the complex interactions and diseases of the human body. This integration promises to enable real-time measurement of drug activity within and across various organs and tissues, such as in the liver and digestive system. Ultimately, the goal is to create an integrated body-on-a-chip that accurately models and predicts responses to new drugs, addressing and resolving common causes of drug development failure.

Now, I’m delighted to introduce the Tissue Chip program’s newest phase, in which laboratories other than other than those that created the chips will “test drive” them to determine if they perform as intended; this wider testing is critical to any new technology intended for widespread use, to ensure reproducibility, accuracy and reliability. Tissue Chip Testing Centers (TCTCs) at the Massachusetts Institute of Technology and Texas A&M University will serve as testing sites, and a TCTC at the University of Pittsburgh will develop and maintain a database collating and sharing  methods, protocols and chip performance data among the TCTCs and the Tissue Chip Consortium, which consists of tissue chip technology developers; government experts representing the FDA and more than 15 NIH Institutes, Centers and Offices; and industry partners. In addition to conducting tests of technical functionality and robustness,  TCTC scientists will use drugs and investigational compounds vetted by pharmaceutical partners to determine biological validity — that is, whether the tissue chip platforms truly reproduce the effects that have been seen in humans who have taken these drugs.

Over the past five years, NCATS has catalyzed the development of this potentially revolutionary translational technology and has demonstrated its utility for accurately modeling human physiology, diseases and drug responses. This has been possible due to the application of the core NCATS values of collaboration, utility and exponential improvement in translational effectiveness. As the dissemination phase of the program begins, I am optimistic that tissue chips will one day be as commonplace as computer chips and will have an equally large impact, fundamentally improving the efficiency and effectiveness of the translational process and helping to get more treatments to more patients more quickly.

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


Oct. 24, 2016: Translator Comes Alive!

This past May, I wrote about the new NCATS Biomedical Data Translator program. I am delighted to report that we now have issued five awards totaling just over $5 million in Cures Acceleration Network (CAN) funds to researchers at 11 institutions who will collaborate with NCATS staff to design the architecture and test the feasibility of the Translator.

For these inaugural Translator awards, NCATS used its CAN Other Transaction authority (PDF - 400KB), a flexible research funding mechanism by which we were able to assemble a scientific team of unprecedented scope to spur innovation and collaborative problem solving. This is a new way of doing business that aligns perfectly with NCATS’ mission to speed translation via innovative collaborative approaches. Last week, I attended the Translator kickoff meeting, at which the funded scientists shared their ideas and jointly devised a plan to create a whole that will be greater than the sum of their individual parts. It was exhilarating to experience the collaborative brainstorming atmosphere and the excitement with which the scientists are approaching this groundbreaking project.

The new awardees bring together complementary expertise in informatics, computer science, pre-clinical development, disease biology, environmental health, and clinical medicine, among other areas. Over the next two years, they will work to develop a prototype informatics platform allowing interrogation of relationships across the full spectrum of biomedical data types to provide new ways of understanding the basis of diseases and to develop new approaches to treatment. NCATS staff will work closely with the team to modify, combine, initiate or discontinue certain activities based on data, emerging methods and technologies, and availability of funds.

Two hundred years ago, chemists created a comprehensive enumeration of the elements and systematic relationships among them. This periodic table transformed chemistry by placing it on a firm scientific footing. I envision the Translator as doing the same for translational science. I encourage you to share in our excitement about this transformational NCATS signature initiative via the links above, and stay tuned for updates on our progress.

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


Nov. 29, 2016: NCATS’ Strategic Plan Sets Stage for Future Translational Science Advances

Five years ago, NCATS was established to catalyze a transformation in the way health interventions are developed and to bring more treatments to more patients more quickly. This required new fundamental principles and approaches to define the burgeoning field of translational science. Just as the understanding of cells and organs created a basis for the development of interventions for the diseases that affect them, so will the understanding of the translational process create a basis for more science-driven, predictive and effective intervention development for the prevention and treatment of all diseases.

This new science of translation is distinct in content, operations and culture. While this presents unique challenges in uncharted territory, it also provides us with unprecedented opportunities within the larger context of the biomedical enterprise. Encompassing many disciplines of science and operations, including biology, chemistry, informatics, pharmaceutics, engineering, medicine, public health, project management, team science, collaboration development and patient engagement, translational science defines the scientific and operational relationships among these fields, builds bridges and creates a network that leverages all stakeholders to more effectively develop and deliver interventions that benefit the health of the public.

NCATS has engaged in a broad internal and external consultation process to establish a strategic plan that conveys not only what we aspire to accomplish, but also the enormous potential and excitement of our field.  This has itself been a learning process, and our Center is much richer for the hundreds of contributors who have given us their best ideas, many of which you will read here.

We have organized the NCATS Strategic Plan according to conceptual themes of translational science, collaborations, training and stewardship. Within these themes are NCATS’ goals and specific objectives and strategies that exemplify best approaches. We are proud of what has been accomplished during the first five years of NCATS, as described in our Annual Reports. Our new strategic plan sets the stage for the Center’s future and will be a living document that will be adapted over time and as relevant to the changing needs in translational science.

NCATS is by design a different kind of scientific organization with a different kind of mission, epitomized by the word “translation” in our name, which is derived from Latin meaning “to carry across.” The science of carrying across from one field to another, from one part of the research ecosystem to another, to bring more treatments to more patients more quickly — this is the NCATS mission. We welcome all to join us in making this bold, collaborative and forward-looking plan a reality.

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


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

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.
Director
National Center for Advancing Translational Sciences

 
Last updated: 08-22-2017
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