The NIH Almanac
NCATS' mission is to catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions.
This newest NIH Center is a distinctly different entity in the research ecosystem. Rather than targeting a particular disease and fundamental science, NCATS focuses on what is common across diseases and the translational process. The Center emphasizes innovation and deliverables, relying on the power of data and new technologies to develop, demonstrate and disseminate improvements in translational science. In these ways, NCATS is serving as an adaptor to enable other parts of the research system to work more effectively. NCATS is complementing the work of other NIH ICs, the private sector and the nonprofit community.
Important Events in NCATS History
December 2011—NCATS was established on Dec. 23 as part of the Consolidated Appropriations Act, 2012 (P.L. 112-74), which amended the Public Health Service Act.
May 2012—NCATS and Eli Lilly and Company jointly released an online Assay Guidance Manual. The manual provides researchers with step-by-step guidance through the complex process of turning a basic research finding into an assay that will start the process of discovering pharmacological tools and drugs.
May 2012—NCATS launched the Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) program to advance therapeutic development by developing partnerships between pharmaceutical companies and the biomedical research community. View Image.
May 2012—A team of NIH-funded scientists supported in part by NCATS’ Clinical and Translational Science Awards (CTSA) program, developed a new method to change the way genes are regulated, effectively causing cancer tumors to shrink and die in the laboratory. View Image.
June 2012 — Five additional companies became New Therapeutic Uses program collaborators, bringing the total to eight.
July 2012—A research collaboration including scientists from NCATS and the University of Wisconsin–Madison helped identify three promising molecular compounds from a collection of approved drugs to pursue as potential treatments for Charcot-Marie-Tooth disease, a genetic neurological disease for which no treatments currently exist.
July 2012—NCATS solicited applications for institutional CTSAs. July 2012—NIH awarded 17 grants for projects designed to create 3-D chips with living cells and tissues that accurately model the structure and function of human organs, such as the lung, liver and heart. These awards are funded and administered by NCATS. In September 2012, NIH awarded two additional tissue chip grants, administered by NCATS but funded by other NIH ICs. View Image.
August 2012—A team that includes nine NCATS researchers identified compounds that delay tumor formation in mice. The compounds target a specific form of pyruvate kinase, called PKM2, that governs how cancer cells use glucose.
August 2012—A collaborative research team, including nine experts from NCATS, was honored 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. View Image.
August 2012—NCATS announced the members of its inaugural Advisory Council and Cures Acceleration Network Review Board.
September 2012 — NIH researchers, including those from NCATS, launched a clinical trial to evaluate the drug candidate DEX-M74 as a treatment for a rare degenerative muscle disease, hereditary inclusion body myopathy (HIBM).
October 2012—Researchers from NCATS designed a novel drug discovery method that uses two co-expressed reporter genes rather than one to increase the odds of identifying candidate compounds with true activity against biological or disease targets.
November 2012—NCATS reported that researchers from 13 universities and hospitals, including 10 CTSA institutions, partnered with the Cystic Fibrosis Foundation and the drug manufacturer Vertex Pharmaceuticals to conduct clinical trials and obtain FDA approval for the drug Kalydeco as a new treatment. View Image.
December 2012—The NIH Bridging Interventional Development Gaps (BrIDGs) program, administered by NCATS, announced new projects to develop potential treatments for cancers, spinal cord injury and a rare disease. View Image.
January 2013—Research in NCATS’ Therapeutics for Rare and Neglected Diseases (TRND) program led to an NIH clinical trial for a possible treatment for Niemann-Pick disease type C1. View Image.
February 2013—NCATS and the NIH Clinical Center hosted a symposium for 2013 Rare Disease Day.
February 2013—A team of scientists from NCATS’ Division of Pre-Clinical Innovation and the Laboratory of Viral Diseases at the National Institute of Allergy and Infectious Diseases developed a drug that blocks early-stage herpes simplex virus infections in cultured cells and prevents reactivation of latent virus in mice. The study was published in the Jan. 9, 2013, issue of Science Translational Medicine.
March 2013—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. Zack and his team identified several compounds that appeared to stop the death of retinal ganglion cells, the neurons in the back of the eye that, when damaged in glaucoma, lead to vision loss and blindness.
April 2013—M. Janis Mullaney, M.B.A., officially joined the NCATS team as the associate director for administration.
May 2013—NCATS published a Federal Register notice on proposed methods for avoiding duplication, redundancy and competition with industry activities.
June 2013—NCATS announced New Therapeutic Uses awards to find new treatments for patients in eight disease areas.
June 2013—The Institute of Medicine released The CTSA Program at NIH: Opportunities for Advancing Clinical and Translational Research.
July 2013—Supported in part by CTSA funding, researchers found that certain molecules in urine can provide an early sign of transplant rejection. The test could allow doctors to act early to protect transplanted kidneys.
July 2013—A team of CTSA-supported researchers at the University of California, Davis, studying the rare disease Fragile X syndrome found that more people have genetic changes linked to this disease than anticipated.
August 2013—NIH announced Extracellular RNA Communications awards designed to improve scientists’ understanding of a newly discovered type of cell-to-cell communication based on extracellular (outside-the-cell) RNA, also called exRNA. View Image.
September 2013—NIH announced new TRND projects to pursue new therapies for rare diseases.
October 2013—NIH announced 15 institutional CTSAs to support scientific translation.
November 2013—Scientists at NIH used RNA interference (RNAi) technology to reveal dozens of genes that may represent new therapeutic targets for treating Parkinson’s disease. The findings also may be relevant to several diseases caused by damage to mitochondria. Richard Youle, Ph.D., an investigator at the National Institute of Neurological Disorders and Stroke and a leader of the study, collaborated with NCATS researchers to discover a network of genes that may regulate the disposal of dysfunctional mitochondria, opening the door to new drug targets for Parkinson’s disease and other disorders. View Image.
December 2013—Pamela M. McInnes, D.D.S., was named deputy director of NCATS, effective Jan. 12, 2014.
December 2013—NIH launched three pre-clinical projects to advance potential new treatments for acute radiation syndrome, brain injury following cardiac arrest and a rare blood disorder called beta thalassemia. The projects are part of NCATS’ BrIDGs program, funded by the NIH Common Fund.
NCATS Legislative Chronology
December 23, 2011—President Obama signed into law the Consolidated Appropriations Act, 2012 (P.L. 112-74), enabling NIH to establish NCATS. This law also transferred authority over the Cures Acceleration Network (CAN) to NCATS. Authorized to reduce significant barriers to successful translation and accelerate the development of high-need cures, the CAN provides NCATS with flexibility in how it funds projects. Implementation of this authority is guided by the CAN Review Board.
Biographical Sketch of NCATS Director Christopher P. Austin, M.D.
In September 2012, Christopher P. Austin, M.D., was appointed the first permanent director of NCATS by NIH Director Francis S. Collins, M.D., Ph.D.
Austin, who had served as director of the NCATS Division of Pre-Clinical Innovation since the creation of the Center in December 2011, leads NCATS in its mission. Currently, many costly, time-consuming bottlenecks exist in the translational process. Austin is applying his experience across the spectrum of the research pipeline to develop, demonstrate and disseminate innovative technologies and paradigms that increase the efficiency of translation, thus speeding the discovery and delivery of interventions that improve human health.
Austin came to NIH in 2002 from Merck, where his work 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. Austin also has served as director of the TRND program and the NIH Chemical Genomics Center (NCGC) and as scientific director of the NIH Center for Translational Therapeutics.
Austin earned an A.B. summa cum laude in biology from Princeton University and an M.D. from Harvard Medical School. He completed clinical training in internal medicine and neurology at Massachusetts General Hospital and a research fellowship in genetics at Harvard.
|Name||In Office from||To|
|Thomas R. Insel (Acting)||December 23, 2011||September 22, 2012|
|Christopher P. Austin||September 23, 2012||Present|
Clinical and Translational Science
The Clinical and Translational Science Awards program supports a national consortium of medical research institutions that are transforming the way biomedical research is conducted. CTSAs strengthen and support the entire spectrum of translational research by developing and providing the expertise, tools, training and collaborations to conduct and drive improvements in human subjects research.
Rare Diseases Research and Therapeutics
- The Therapeutics for Rare and Neglected Diseases program aims to encourage and speed the development of new drugs for rare and neglected diseases. TRND stimulates drug discovery and development research collaborations among NIH and academic scientists, nonprofit organizations, and pharmaceutical and biotechnology companies working on these diseases.
- The Bridging Interventional Development Gaps program — supported by the NIH Common Fund — makes available, on a competitive basis, certain critical resources needed for the development of new therapeutic agents. Investigators do not receive grant funds through this program. Instead, successful applicants receive free access to NIH contractors who conduct pre-clinical services, such as toxicology studies, for therapeutic projects that have demonstrated efficacy in a disease model.
- The Office of Rare Diseases Research (ORDR) supports and coordinates rare disease research, responds to research opportunities for rare diseases, and provides information on rare diseases. ORDR serves the needs of patients who have any one of the thousands of rare diseases known today.
- The Genetic and Rare Disease Information Center helps people find useful information about genetic and rare diseases.
- The Global Rare Diseases Patient Registry and Data Repository makes de-identified patient data available to all investigators to enable analyses across many rare diseases.
The Rare Diseases Clinical Research Network helps support broader and collaborative clinical trials and information sharing.
Re-engineering Translational Sciences
- Discovering New Therapeutic Uses for Existing Molecules is a collaborative pilot program designed to advance therapeutic development by developing partnerships between pharmaceutical companies and the biomedical research community. This innovative program matches researchers with a selection of molecular compounds from industry to test ideas for new therapeutic uses, with the ultimate goal of identifying promising new treatments for patients.
- The Tissue Chips for Drug Screening initiative aims to develop 3-D human tissue chips that accurately model the structure and function of human organs, such as the lung, liver and heart. Researchers will be able to use these models to predict whether a candidate drug, vaccine or biologic agent is safe or toxic in humans in a faster and more cost-effective way than current methods.
- The NIH Chemical Genomics Center aims to translate the discoveries of the Human Genome Project into biological and disease insights and, ultimately, new therapeutics for human disease through small molecule assay development, high-throughput screening, cheminformatics and chemistry. The NCGC provides researchers with access to the large-scale screening and chemistry capacity necessary to identify compounds that can be used as chemical probes to validate new therapeutic targets.
- The Toxicology in the 21st Century program, a federal collaboration involving NIH, the Environmental Protection Agency, and the FDA, is aimed at developing better toxicity assessment methods. The goal is to quickly and efficiently test whether certain chemical compounds have the potential to disrupt processes in the human body that may lead to adverse health effects.
- Through the Extracellular RNA Communication program, scientists are beginning to understand the potential exRNA research may hold for improving understanding, diagnosis, prognosis and treatment of a wide variety of diseases and conditions, such as cancer, bone marrow disorders, heart disease, Alzheimer’s disease and multiple sclerosis. ExRNA communication is a recently discovered cell-to-cell signaling process.
- Gene silencing through RNA interference has emerged as a powerful tool for understanding gene function. Over the past several years, high-throughput RNAi screens have illuminated a wide variety of biological processes, ranging from genes that affect the activity of therapeutic agents to novel components of signaling pathways.