From the Director | What's New at NCATS? | Research Opportunities Volume 02 • Issue 03 • August 13, 2013

Director's Message

Christopher Austin

A major NCATS mission is to develop new technologies and models that scientists in many different fields can use to advance translational research and ultimately improve health. Given the enormous opportunities and needs in translational science, we are particularly interested in "game-changing" technologies that could revolutionize the field. Extracellular RNA (exRNA) communication, a recently discovered type of cell signaling, is one such new area. Scientists previously believed that RNA worked mostly inside the cell that produced it, but recent findings show that cells release certain types of RNA that travel through body fluids to affect other cells. ExRNA appears to have many functions in the body and may play an important role in health as well as in a wide range of diseases.

Though still in its early days, exRNA looks to be another of those remarkable discoveries of a naturally occurring process that has enormous translational potential — like RNA interference (RNAi) and stem cells. Stem cells can renew themselves and transform into many different cell types, and they are being used in many of NCATS' programs to develop drugs more safely and effectively. RNAi is a process by which the activity of genes can be selectively decreased; scientists use RNAi in the laboratory to study the effects of individual genes and are developing RNAi-based therapies, particularly for cancer. Here at NCATS, our program in genome-wide RNAi screening has developed new methods that are driving reliable large-scale studies of gene function and identification of new therapeutic targets.

ExRNA appears to hold the same  potential for transforming translational research, but scientists still know very little about its basic biology, functions and roles in disease. To address this need, NIH developed a new trans-NIH program funded by the NIH Common Fund called Extracellular RNA Communication. Today, NIH announced that it will award $17 million this year for 24 research projects designed to improve scientists' understanding of exRNA and investigate its role in a variety of diseases, including many types of cancer, bone marrow disorders, heart disease, Alzheimer's disease and multiple sclerosis. NCATS will administer 18 of these awards to develop biomarkers from exRNA found in body fluids and to create new therapeutic strategies for using exRNA in developing and delivering treatments.

The ExRNA program also illustrates a point I make often, about the intersection of fundamental and translational research and how each benefits the other. Discoveries in basic science feed forward to translation, just as translational discoveries often illuminate fundamental mechanisms. ExRNA is both a type of cell-to-cell communication and a route to disease understanding, diagnostics, biomarkers and therapeutics. Like the DNA formerly known as "junk" (the DNA residing outside of genes that we now know has important functions), exRNA was formerly thought to be functionless cellular debris. There are undoubtedly many other such discoveries waiting to be made that will transform both our understanding of ourselves and our ability to intervene when things go wrong. Read more about this exciting new area of science as well as many other NCATS initiatives and achievements in this issue of the NCATS e-newsletter.


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

What's New at NCATS?

NCATS Supports Research on Newly Discovered Type of Cell-to-Cell Communication

NIH Funds Academic-Industry Collaborations to Identify New Uses for Existing Compounds

IOM Releases Report on CTSA Program

CTSA Resources Support Largest U.S. Study of Newborn Screening for Fragile X Mutations

NCATS and Johns Hopkins Researchers Identify New Therapeutic Strategy for Eye Diseases

NCATS Spotlight: Tox21 Program

NCATS Teams Receive NIH Director's Awards

NCATS Council and CAN Review Board Meetings

Upcoming Events

Collaborate with NCATS Scientists

NCATS in the News

encapsulated extracellular rna

NCATS Supports Research on Newly Discovered Type of Cell-to-Cell Communication

On August 13, NIH announced $17 million in awards for the Extracellular RNA (exRNA) Communication program. This trans-NIH initiative is designed to advance research in a newly discovered type of cell-to-cell communication that may play a role in the diagnosis and treatment of various health conditions.

Through 24 awarded research projects, scientists will explore basic exRNA biology and develop tools and technologies that apply new knowledge about exRNA to the research, diagnosis and treatment of diseases, including many types of cancer, bone marrow disorders, heart disease, Alzheimer's disease and multiple sclerosis. NCATS will administer 18 of these awards, through which researchers will develop biomarkers from exRNA and design new ways to use exRNA in treatments.

The program is supported by the NIH Common Fund and led by a trans-NIH team that includes NCATS; the National Cancer Institute; the National Heart, Lung, and Blood Institute; the National Institute on Drug Abuse; and the National Institute of Neurological Disorders and Stroke.

Read the full news release to learn more. See the NCATS-administered projects on our website or view the complete list of awards on the NIH Common Fund website.

Scientist working in lab

NIH Funds Academic-Industry Collaborations to Identify New Uses for Existing Compounds

On June 18, NIH awarded $12.7 million to match nine academic research groups with a selection of pharmaceutical industry compounds. The awardees are exploring new treatments for patients in eight disease areas, including Alzheimer's disease, Duchenne muscular dystrophy and schizophrenia. The collaborative pilot initiative, called Discovering New Therapeutic Uses for Existing Molecules, is led by NCATS and funded by the NIH Common Fund.

This innovative program addresses one of the many translational research problems that NCATS strives to overcome: the process of developing a new therapeutic. On average, this process can take more than 13 years from target discovery to approval of a new treatment — and more than 95 percent of the time, these treatments fail. However, this failure rate offers opportunities: Advancing partially developed compounds to clinical trials is faster than starting from scratch, because the compounds have undergone significant research and development by industry, including safety testing in humans. Because thousands of human diseases still have few or no treatments, NCATS strives to accelerate the pace at which discoveries are transformed into new therapies for patients.

Eight pharmaceutical companies are participating in the pilot initiative: AbbVie (formerly Abbott); AstraZeneca; Bristol-Myers Squibb Company; Eli Lilly and Company; GlaxoSmithKline; Janssen Research & Development, LLC; Pfizer; and Sanofi.

Read more about the awards in the NIH news release, or visit the NCATS website for details about the projects, including videos by several of the award recipients.

IOM Report Cover on the CTSA Program at NIH

IOM Releases Report on CTSA Program

On June 25, the Institute of Medicine (IOM) released the findings from its in-depth review of the Clinical and Translational Science Awards (CTSA) program. Established in 2006, the CTSA program was designed to provide academic homes for clinical and translational research. The institutions supported by the program have been transformative to the science and culture of those academic centers across the country, providing expertise, capacities, training and collaborations to advance clinical translational science as a discipline across the translational spectrum.

In December 2011, the CTSA program became a part of NCATS, and in July 2012, at the urging of Congress, NIH commissioned a study by the IOM to evaluate the program and recommend possible changes to its mission and operation. The thoughtful and measured review included input from a broad spectrum of internal and external stakeholders. The resulting report is now available on the IOM website.

The report recommends seven steps NCATS can take to build on the successes of the CTSA program and realize its full potential for transforming clinical and translational science to benefit human health. Learn more about these recommendations and NCATS' response in a statement issued by NCATS Director Christopher P. Austin, M.D.

Baby's feet with blood spot screen for Fragile X

CTSA Resources Support Largest U.S. Study of Newborn Screening for Fragile X Mutations

Dramatic advances in genetics and DNA sequencing technologies have increased clinicians' ability to accurately diagnose inherited diseases. Scientists also now know that many disease-causing gene changes, or mutations, are more common than previously thought.

A team of researchers at the University of California, Davis (UC Davis), studying the rare disease Fragile X syndrome (FXS) found that more people have genetic changes linked to this disease than anticipated. FXS is an inherited condition caused by changes in a gene on the X chromosome that lead to intellectual disability, behavioral and learning challenges, and various physical characteristics. Previous estimates suggested that FXS affects one in every 2,500 to 8,000 individuals, but it is not known how many people in the general population have the associated genetic changes.

The UC Davis team set out to show that large-scale newborn screening for FXS mutations was technically and logistically possible and could fill this crucial knowledge gap. This screening could provide a way to determine the prevalence of FXS mutations in the general population before symptoms appear.

After five years of intensive work with support from the university's Clinical and Translational Science Center (CTSC), these researchers made their goal a reality and published findings in the Dec. 21, 2012, issue of Genome Medicine.

NIH provides funding to the UC Davis CTSC through the Clinical and Translational Science Awards (CTSA) program, which is administered by NCATS. NIH's Eunice Kennedy Shriver National Institute of Child Health and Human Development funded the project with additional support from the Centers for Disease Control and Prevention and the Association for Prevention Teaching and Research.

Read the full feature to learn more about this collaborative research project.

Optic disc from a normal eye (left) and optic disc from an eye in which the optic nerve has been damaged by glaucoma (right).

NCATS and Johns Hopkins Researchers Identify New Therapeutic Strategy for Eye Diseases

Retinal diseases like glaucoma damage the vision of millions of people worldwide, but they have limited treatment options. Although researchers possess a breadth of knowledge about the cause of glaucoma, they have struggled to identify molecular targets that could lead to new and more effective treatments. Scientists at NCATS are experts at this first step in the translational process, determining targets for disease intervention. However, to be effective in addressing specific diseases, NCATS' translational expertise must be matched with expertise in those diseases. NCATS custom-assembles a team for every project, each built to solve a particular roadblock to translation.

To investigate new ways to treat glaucoma, NCATS partnered with the Johns Hopkins School of Medicine in Baltimore. Led by Don Zack, M.D., Ph.D., a glaucoma specialist and molecular biologist at Hopkins' Wilmer Eye Institute, the Hopkins team brought extensive knowledge and robust animal models of retinal degenerative diseases. The NCATS team, led by Jim Inglese, Ph.D., director of NCATS' Assay Development and Screening Technology Laboratory, contributed expertise in assay development and RNA interference. Together, the investigators identified many promising new compounds, determined which gene a successful treatment would need to target and zeroed in on one compound in particular — developing multiple new techniques along the way that could be applied to research on other diseases.

The team's work was published in the March 5, 2013, issue of the Proceedings of the National Academy of Sciences (PNAS). Although hurdles remain before their approach can be used in the clinic, these studies offer a crucial new target for the treatment of widespread diseases that cause blindness. "This remarkable story illustrates NCATS principles and impact beautifully," said NCATS Director Christopher P. Austin, M.D. "The collaborative research team came together to solve a problem neither could solve alone."

Read the full feature for more about this advance.

Graphic of DNA and associated code

NCATS Spotlight: Tox21 Program

We are exposed to thousands of different chemicals during our lifetimes, from consumer products to food additives to pharmaceutical drugs. Yet scientists lack the data to predict how these chemicals affect our bodies and their potential for harm. New system-wide methods for assessing chemical toxicity can improve the way scientists evaluate environmental chemicals and develop new medicines.

The Toxicology in the 21st Century (Tox21) program, a collaborative initiative among NIH, the Food and Drug Administration, and the Environmental Protection Agency, aims to test 10,000 chemicals and evaluate their potential to cause health problems. The compounds undergo testing in the high-throughput robotic screening system at the NCATS Division of Pre-Clinical Innovation. Program scientists design innovative methods to quickly and efficiently predict whether certain chemicals can disrupt processes in the human body, leading to adverse health effects.

Read more about the program's progress thus far and new collaboration opportunities in the full feature.

John McKew receives award from the NIH Director Francis Collins

NCATS Teams Receive NIH Director's Awards

NIH Director Francis S. Collins, M.D., Ph.D., presented NCATS with two prestigious awards at a ceremony in June on the NIH campus in Bethesda, Maryland. The NIH Director's Award recognizes superior performance or extraordinary efforts that help NIH fulfill its mission.

The Niemann-Pick Disease Type C Therapeutic Development Team, which includes scientists from NCATS' Therapeutics for Rare and Neglected Diseases program and collaborators from three other NIH institutes, three academic institutions, and a pharmaceutical company, was honored for its work to identify and develop a potential treatment for Niemann-Pick disease type C, called cyclodextrin. NIH launched a clinical trial of cyclodextrin in January. Learn more about this project in a recent feature story.

This progress is the result of the collaborative efforts of an award-winning, multidisciplinary team of experts from NCATS, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Institute of Neurological Disorders and Stroke, and the National Human Genome Research Institute; Janssen Research & Development, LLC; Washington University in St. Louis School of Medicine; Albert Einstein College of Medicine, New York City; and the University of Pennsylvania, Philadelphia.

The Center's Matrix Group was recognized for developing a platform that allows large-scale testing of combinations of drugs. The new system will aid in the search for next-generation therapeutics. The team includes eight investigators from NCATS and one from the National Cancer Institute.

During the awards ceremony, Collins thanked the recipients for their dedication and talent, which he said have greatly enhanced NIH's efforts to turn discovery into health: "Because YOU serve, NIH is truly at its best!" He also thanked recipients' families, friends and coworkers for their support. To learn more, read the 2013 NIH Director's Awards Ceremony brochure, or visit the NIH Office of Human Resources website to find out about the awardee selection process and criteria.

Christopher Austin speaks to NCATS advisory council.

NCATS Council and CAN Review Board Meetings

On May 17, NCATS Director Christopher P. Austin, M.D., led a joint meeting of the NCATS Advisory Council and Cures Acceleration Network (CAN) Review Board on the NIH campus in Bethesda, Maryland. The NCATS Council and CAN Review Board members provide Austin with guidance and direction on the Center's initiatives, programs and policies. During the meeting, Austin provided brief updates on the NCATS budget and hiring plans and an overview of NIH's new bioinformatics initiative, Big Data to Knowledge. Read more about the May meeting or view the archived videocast.

NCATS will hold the next joint meeting of the NCATS Advisory Council and CAN Review Board on September 16 in Conference Room 6 of Building 31C on the NIH campus. Details will be posted on the upcoming events page of the NCATS website.

Upcoming Events


NCATS Research and Development Day Planned for September 12

On Sept. 12, 2013, NCATS will hold a research and development day at the Novartis Institutes for Biomedical Research in Cambridge, Massachusetts, from 8:30 a.m. to 4:30 p.m. Staff from NCATS' Division of Pre-Clinical Innovation will provide an overview of the Center's pre-clinical projects portfolio. The goal of the event is to introduce potential investors to NCATS' external partners and help transition these projects into clinical development. Links to the event website, registration and agenda are available on the events page of the NCATS website.

NCATS Advisory Council/Cures Acceleration Network (CAN) Review Board Set to Meet September 16

On Sept. 16, 2013, NCATS will hold a joint meeting of the NCATS Advisory Council and the CAN Review Board on the NIH campus in Bethesda, Maryland. The meeting will feature reports from NCATS Director Christopher P. Austin, M.D., and others about the Center's initiatives, policies, programs and future direction. For more information, visit the NCATS Advisory Council page and the CAN Review Board page of the NCATS website.

Collaborate with NCATS Scientists

NCATS researchers actively are seeking collaborators in the following areas:

Therapeutics for Rare and Neglected Diseases (TRND) Program

TRND, which seeks to improve and accelerate the development of treatments for rare and neglected diseases, is accepting proposals for collaborative projects that focus on pre-clinical and early clinical development of new drugs for rare and neglected tropical diseases. Letters of intent are due by Sept. 16, and final proposals are due on Sept. 30, 2013. Visit Apply to TRND to get started.

Bridging Interventional Development Gaps (BrIDGs) Program

The BrIDGs program makes available, on a competitive basis, certain critical resources needed for the development of new therapeutic agents. The next opportunity to apply to the BrIDGs program tentatively is scheduled for January/February 2014. Information will be available here.

NCATS Chemical Genomics Center (NCGC)

NCGC is one of the centers in the Molecular Libraries Probe Production Centers Network (MLPCN), which is an NIH Common Fund Initiative. Through the MLPCN, NCGC offers biomedical researchers access to large-scale screening capacity, along with the medicinal chemistry and informatics necessary to identify chemical probe molecules and to study the functions of genes, cells and biochemical pathways. For inquiries or to obtain NCGC probe molecules, contact Ajit Jadhav.

NCGC researchers also seek collaborators for assay development and high-throughput screening, chemistry and chemistry technology, automation, and informatics. Learn more.

NIH RNA Interference (RNAi) Initiative

The NIH RNAi Initiative, administered by NCATS, provides state-of-the-art, high-throughput RNAi genome-wide screens for both humans and mice. This resource is available only to researchers at NIH. NIH scientists interested in performing a high-throughput RNAi screen can contact Scott Martin, Ph.D., for more information.

Toxicology in the 21st Century (Tox21) Program

The Tox21 program aims to test 10,000 chemicals and evaluate their potential to cause health problems. Any investigator may propose the development of biological assays for high-throughput screening.

To suggest an assay, submit an assay nomination form to Dr. Menghang Xia. Proposed assays must be compatible with the high-throughput screening guidelines as described in the assay guidance criteria.

NCATS in the News

Research Opportunities and Announcements

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

Centers of Excellence for Big Data Computing in the Biomedical Sciences (U54) • RFA-HG-13-009

Opportunity to Submit Proposals for the Therapeutics for Rare and Neglected Diseases (TRND) Program • NOT-TR-13-007

Extramural Loan Repayment Program for Clinical Researchers (LRP-CR) • NOT-OD-13-081

Extramural Clinical Research Loan Repayment Program for Clinical Researchers from Disadvantaged Backgrounds (LRP-IDB) • NOT-OD-13-082

Extramural Loan Repayment Program for Pediatric Research (LRP-PR) • NOT-OD-13-083

Extramural Loan Repayment Program for Contraception and Infertility Research (LRP-CIR) • NOT-OD-13-084

Extramural Loan Repayment Program for Health Disparities Research (LRP-HDR) • NOT-OD-13-085

HHS Reissues PHS 2013-02 SBIR and STTR Omnibus Grant Solicitations Implementing Venture Capital Provision and SBA Company Registry Requirement of the SBIR/STTR Reauthorization Act of 2011 • NOT-OD-13-071

Reissue PHS 2013-02 Omnibus Solicitation of the NIH, CDC, FDA and ACF for Small Business Innovation Research Grant Applications (Parent SBIR [R43/R44]) • PA-13-234

Reissue PHS 2013-02 Omnibus Solicitation of the NIH for Small Business Technology Transfer Grant Applications (Parent STTR [R41/R42]) • PA-13-235

Research on the Role of Epigenetics in Social, Behavioral, Environmental and Biological Relationships, throughout the Life-Span and across Generations (R21) • RFA-TW-13-002

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