From the Director | What's New at NCATS? | Research Opportunities Volume 01 • Issue 04 • September 24, 2012

From the Director

Christopher Austin

As the Center’s new director, I am excited to lead NCATS on its mission to catalyze innovations aimed at enhancing the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions. Advances in these areas will enable others in the public and private sectors to develop drugs and diagnostics more efficiently for any number of diseases, ultimately accelerating the pace at which new treatments are delivered to the patients who need them.

Already, NCATS has become a hub of innovation for translational sciences. Since December 2011, the Center has launched several major research initiatives, cultivated promising strategic partnerships, and established a presence at NIH and in the community — all while standing up a brand new center. Many of these milestones are highlighted on the NCATS timeline, including:

These accomplishments would not have been possible without extraordinary leadership from my predecessor, Thomas R. Insel, M.D., who has served as NCATS acting director since our launch. He established a strong foundation on which to build, and I look forward to working with a tremendously talented and supportive team to continue advancing our translational medicine programs and initiatives.

In this issue, you can read more about our first Advisory Council and CAN Review Board meetings, an award for excellence in technology transfer, recent science advances, news from the Institute of Medicine, and upcoming events and deadlines.

Thank you for your continued support and interest in our work!


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

What's New at NCATS?

Christopher P. Austin Named New NCATS Director

NCATS Holds First Council, CAN Review Board Meetings

NCATS Welcomes New Advisory Council, CAN Board Members

NCATS Collaborative Project Wins Award for Excellence in Technology Transfer

NCATS Part of Team that Finds Activating Key Enzyme, Blocks Tumor Growth in Mice

NIH Launches Trial for Rare Degenerative Muscle Disease Treatment

Institute of Medicine Summary on June Cures Acceleration Network Workshop Now Available

NIH Commissions Institute of Medicine Study on Clinical and Translational Science Awards Program

NIH Celebrates Science at Collaborative September Event

Upcoming Events

NCATS in the News

Christopher Austin and Robert Beall

Christopher P. Austin Named New NCATS Director

Christopher P. Austin, M.D., was named director of NCATS earlier this month by NIH Director Francis S. Collins, M.D., Ph.D., at the inaugural meetings of the NCATS Advisory Council and Cures Acceleration Network (CAN) Review Board. Austin succeeded NCATS Acting Director Thomas R. Insel, M.D., on Sept. 23, which also happened to be Austin’s 52nd birthday.

Austin had been serving as the director of the NCATS Division of Pre-Clinical Innovation since NCATS’ launch in December 2011.

"Dr. Austin’s accomplishments in virtually every stage of the translational science spectrum make him an ideal choice to continue building on NCATS’ momentum and successes," Collins said. "From his clinical experience to his work in the public and private sectors, he is poised to lead the Center in revolutionizing the science of transforming laboratory discoveries into new therapies for patients."

Austin will lead NCATS’ innovative initiatives in Discovering New Therapeutic Uses for Existing Molecules and Tissue Chip for Drug Screening. He also will spearhead NCATS’ research efforts, including the Clinical and Translational Science Awards, which support a national consortium of medical research institutions to enhance the efficiency and quality of translational research. The Office of Rare Diseases Research, the NCATS Chemical Genomics Center (NCGC) and the Therapeutics for Rare and Neglected Diseases (TRND) efforts also are among NCATS’ programs.

In 2002, Austin came to NIH from Merck, where his work had focused on genome-based discovery of novel targets and drugs. He began his NIH career as the senior advisor to the director for translational research at the National Human Genome Research Institute, where he initiated the Knockout Mouse Project and the Molecular Libraries Roadmap Initiative. While at NIH, he also has served as director of the TRND program and NCGC as well as scientific director of the NIH Center for Translational Therapeutics.

"In its first months, NCATS has made great strides in addressing a multitude of translational science challenges," Austin said. "I feel privileged to have this opportunity to continue serving the NIH mission by leading NCATS’ innovative efforts to transform basic discoveries into improved patient care."

Read the NIH news release.

Photo by Bill Branson

Christopher Austin and members of the NCATS Advisory Council and CAN Review Board

NCATS Holds First Council, CAN Review Board Meetings

Sept. 14, 2012, marked the first meetings of the NCATS Advisory Council and Cures Acceleration Network (CAN) Review Board on the NIH campus in Bethesda, Maryland. Both groups are providing the NCATS director with guidance on the Center’s initiatives, programs and policies. They also will make recommendations for achieving goals and overcoming significant barriers to successful translation of basic science into clinical application.

Former NCATS Acting Director Thomas R. Insel, M.D., shared the gavel with CAN Review Board Chair Freda C. Lewis-Hall, M.D., who also is an advisory council member, to open the joint meetings and welcome new members, respectively. Insel presented an overview of NCATS’ mission and its role in translational research at NIH, budget details, recent science advances and major Center milestones.

NIH Director Francis S. Collins, M.D., Ph.D., then joined the event to announce Christopher P. Austin, M.D., as the new NCATS director, effective Sept. 23, 2012. He heralded Austin as a visionary who will engage in disruptive innovation. Collins also lauded the progress NCATS has made thus far. "This team has been amazing in what they have accomplished in such a short time," he said in thanking the acting leadership team for their dedicated service and contributions. "I am looking forward to seeing how NCATS will continue to develop and grow."

Read the full story.

Photo by Bill Branson

Members of the NCATS Advisory Council and CAN Review Board

NCATS Welcomes New Advisory Council, CAN Board Members

Comprising nearly two dozen leading experts from patient advocacy groups, pharmaceutical companies and biomedical research organizations, the NCATS Advisory Council and Cures Acceleration Network (CAN) Review Board met for the first time on Sept. 14, 2012. 

Announced in August, these members will provide the NCATS director with guidance on its programs, policies and initiatives, advise on CAN activities, and help identify barriers to translational research.

For more information about their roles and meeting schedules, visit the NCATS Advisory Council and CAN Review Board pages of the NCATS website. Clicking on each name on the Advisory Council roster and CAN Review Board roster will bring up a biographical sketch and picture of each new member. A videocast archive of the Sept. 14 meetings also is available.

Photo by Bill Branson

Lili Portilla, Elizabeth Ottinger, Alan Hubbs, Forbes Porter, and Steven Silber

NCATS Collaborative Project Wins Award for Excellence in Technology Transfer

A collaborative research team, including nine experts from NCATS, was honored last month for its work on an investigational treatment for Niemann-Pick disease type C1, a rare genetic disease of cholesterol storage that eventually leads to neurodegeneration. Comprising investigators from four NIH institutes and one pharmaceutical company, the team won the Excellence in Technology Transfer Award for its work with 2-hydroxypropyl-β-cyclodextrin (HPβCD) as a potential treatment for Niemann-Pick type C1 ― a disease for which there are no Food and Drug Administration-approved therapies.

It is the first award of its kind to NCATS, recognizing laboratory employees and their partners who have outstanding accomplishments in transferring federally developed technology to the marketplace. The Federal Laboratory Consortium (FLC) for Technology Transfer of the mid-Atlantic region presented the award to the investigators at a ceremony on Aug. 30, 2012, in Cambridge, Maryland.

Read the full story.

PKM2 enzyme

NCATS Part of Team that Finds Activating Key Enzyme, Blocks Tumor Growth in Mice

Scientists have known for decades that cancer cells use more glucose than healthy cells, feeding the growth of some tumors. Now, a team that includes NCATS researchers has identified molecular compounds that delay the formation of tumors in mice by targeting a key enzyme. This enzyme, known as pyruvate kinase, governs how cancer cells use glucose.

Christopher P. Austin, M.D., NCATS’ new director, was an author of the study, which was published online in the August 26, 2012, advance issue of Nature Chemical Biology. The study was led by biologists from the Koch Institute for Integrative Cancer Research at the Massachusetts Institute of Technology (MIT), Cambridge, and included researchers from NCATS, the Structural Genomics Consortium at the University of Toronto, and Harvard Medical School in Boston.

"The last several years have brought an avalanche of new discoveries that have begun to explain a phenomenon of altered cancer cell metabolism first described almost 90 years ago," said Austin. "This work provides a wonderful example of how molecular compounds can be used as tools to probe and understand biological processes, and at the same time explore new drug targets in the fight against cancer."

Read the full story.

Photo by Dimitris Anastasiou, Will Israelsen and Andrea Howell, MIT

NIH Launches Trial for Rare Degenerative Muscle Disease Treatment

NIH researchers have launched a clinical trial to evaluate the drug candidate DEX-M74 as a treatment for patients suffering from a rare degenerative muscle disease called hereditary inclusion body myopathy (HIBM). NIH scientists from NCATS and the National Human Genome Research Institute will conduct the study at the NIH Clinical Center in Bethesda, Maryland.

HIBM, also known as GNE myopathy, has no available therapy. Disease symptoms emerge in adulthood and slowly lead to progressive muscle weakness. Most patients develop symptoms while in their early 20s and eventually require a wheelchair as their arm, hand and leg muscles weaken. Mutations in the GNE gene cause HIBM by producing low sialic acid levels in muscle proteins, which scientists think contribute to the symptoms of muscle weakness. Normally, GNE produces an enzyme that produces sialic acid in the body, a sugar important to muscle development and kidney function.

"This study marks an important milestone toward developing a treatment for an underserved patient population, and we would not be this far along had it not been for the teamwork and dedication of the researchers working on this collaboration," said Christopher P. Austin, M.D., the newly appointed NCATS director.

Read the NCATS news release.

IOM report cover

Institute of Medicine Summary on June Cures Acceleration Network Workshop Now Available

The Institute of Medicine recently released a summary of its two-day workshop on the Cures Acceleration Network (CAN) held in June 4–5, 2012, titled "Accelerating the Development of New Drugs and Diagnostics: Maximizing the Impact of the Cures Acceleration Network." Authorized by the Affordable Care Act and housed within NCATS, CAN is designed to advance the development of high-need cures and reduce significant barriers between research discovery and clinical trials. The purpose of the workshop was to inform the community and the NCATS CAN Review Board about ways to increase CAN’s impact on developing cures and accelerating the translational research process. 

Read the workshop summary.

NIH Commissions Institute of Medicine Study on Clinical and Translational Science Awards Program

In response to a congressional request, NIH has commissioned the Institute of Medicine (IOM) to assemble an ad hoc expert committee to evaluate the Clinical and Translational Science Awards (CTSA) program and provide advice related to its implementation at NCATS. Committee members will review existing evaluations and stakeholder input; they also will seek input through workshops and other means.

The IOM study will explore the contributions of CTSAs in accelerating the development of new therapeutics, the CTSA program’s role in facilitating disease-specific and pediatric research, and its role in enhancing the integration of other NIH Institute and Center programs.

In June 2013, the committee will release a report with recommendations on the CTSA program’s current mission and goals and whether changes are needed.

Visit the IOM website to learn more.

Celebration of Science

NIH Celebrates Science at Collaborative September Event

Star-studded appearances, inspiring presentations, research success stories and lively discussions marked the start of the Celebration of Science initiative during a three-day kick-off event Sept. 7–9, 2012. Spearheaded by FasterCures and the Milken Foundation, the event affirmed the importance of bioscience by showcasing some of the remarkable accomplishments scientists have made over the past decade.

NIH’s main campus in Bethesda, Maryland, was one of three Washington, D.C., area locations for the celebratory weekend. The event convened more than 1,000 scientific and policy leaders, who collaborated to rally support for the initiative.

During panel discussions on Sept. 8 ― "NIH Day" ― both U.S. House Majority Leader Eric Cantor and NCATS Cures Acceleration Network Review Board Chair Freda Lewis-Hall, M.D., of Pfizer lauded NIH and its creation of NCATS. NIH experts discussed advances in HIV/AIDs research, precision medicine, protein folding, neuroscience and rehabilitation medicine, and also highlighted the economic benefits of research. Moving patient testimonials were woven in throughout the agenda to demonstrate on a personal level how medical research is crucial to improving and saving lives.

View the NIH videocasts of the morning and afternoon sessions, watch highlights in a brief video, or visit the Celebration of Science website for additional video coverage.

Upcoming Events


Third Annual Conference on Clinical Research for Rare Diseases
On Oct. 2, 2012, the Rare Diseases Clinical Research Network and the Clinical and Translational Science Awards (CTSA) Consortium will host a one-day conference in Rockville, Maryland, focused on conducting clinical research in rare diseases. Speakers will include expert clinical investigators who represent a spectrum of medical diseases and disciplines and who are actively engaged in rare disease research, as well as NIH and industry representatives. The registration fee is $100. Visit the Events page for more information.

2012 Sleep Research Network Conference
Supported in part by an NCATS conference grant, the primary goal of the Sleep Research Network conference, Oct. 22–23, 2012, in Bethesda, Maryland, is to provide an ongoing opportunity for investigators and trainees from across the United States to meet for the purpose of building a Sleep Research Network, evaluating diagnostic and therapeutic technologies, identifying opportunities for new lines of collaborative research and training, and leveraging resources available through local CTSAs to promote inter-institutional sleep and circadian medicine research. Visit the Events page to learn more.

Mercedes Taylor and student

Mercedes Taylor, an NIH post-baccalaureate intramural research training awardee, demonstrates chemistry activities at the 2011 Frontiers in Science and Medicine Day. (Johns Hopkins University Photo)

Frontiers in Science and Medicine
On Oct. 26, 2012, researchers from NCATS will join more than a dozen universities, health care organizations, private companies and research centers at various locations in Rockville, Maryland, for the fourth annual Frontiers in Science and Medicine Day. The event is an opportunity to introduce students to the possibilities of careers in science and medicine. Several hundred middle school students from Montgomery County public schools will participate in hands-on science activities and tours throughout the day. NCATS researchers from the Division of Pre-Clinical Innovation (DPI) will give tours of the biology laboratory and high-throughput robotic screening platform. In addition, DPI researchers will host a hands-on activity demonstrating how to turn a liquid (school glue) into a solid (slime) using some basic chemistry techniques.

NCATS Policy Workshop
NCATS will hold a one-day workshop on Oct. 29, 2012, in Rockville, Maryland, to begin building a solid policy agenda for the Center. Given NCATS’ mission to serve as a hub for catalyzing innovations and creative partnerships in translational science, this workshop provides an opportunity to explore key policy issues that affect translational research. The goal of the workshop is to better serve NCATS’ stakeholders by expanding the Center’s understanding of relevant policy issues and becoming a resource for solving complex issues across the continuum of translational research. Registration information is forthcoming. Be sure to check back soon on the Events page for details.


FDA-NIH Workshop: The Science of Small Clinical Trials
The Food and Drug Administration’s Office of Orphan Product Development, in collaboration with NCATS’ Office of Rare Diseases Research at NIH, will hold a two-day public workshop titled “The Science of Small Clinical Trials,” Nov. 27–28, 2012, in Silver Spring, Maryland. The workshop will present an overall framework and provide training in the scientific aspects of designing and analyzing clinical trials based on small study populations. Participants will discuss examples of small clinical trials and identify strategies and trial designs that are conducive to overcoming the challenges involved in executing clinical trials using small study populations. Visit the Events page to learn more.

NCATS in the News

NCATS and its programs are in the news frequently. Below are a few examples of recent media coverage:

Be sure to visit our News & Events page to learn more about these stories and other NCATS programs in the news.

Research Opportunities and Announcements

Visit the NCATS Open Opportunities page for a complete list of funding and program announcements.

We Want to Hear from You

We welcome your feedback to ensure that we are meeting the needs of all of our stakeholders. Please e-mail us directly at, follow us on Facebook and Twitter, view our YouTube channel, and join the NCATS e-mail list for other Center announcements.

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