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.
NCATS is all about getting more treatments to more patients more quickly. The Center is distinct in many ways; it focuses not on specific diseases, but on what is common among them and the translational science process.
NCATS develops new approaches, technologies, resources and models; demonstrates their usefulness; and disseminates data, analyses and methodologies to the community. It complements 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 — NCATS solicited applications for institutional CTSAs.
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 Food and Drug Administration (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., M.Sc.(Dent.) 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.
February 2014 — NCATS and the NIH Clinical Center hosted Rare Disease Day to spotlight the challenges encountered by those affected and the significant research and collaboration activities that are helping to make a difference in the development of new diagnostics and treatments.
April 2014 — NCATS released its first annual report (2012-2013).
May 2014 — Petra Kaufmann, M.D., M.Sc., joined NCATS as the director of the Division of Clinical Innovation.
May 2014 — NCATS Advisory Council working group released findings on the Institute of Medicine’s 2013 report about the CTSA program.
May 2014 — Within the CTSA program, institutions participating in institutional review board (IRB) reliance networks have shown that efficient and centralized oversight can accelerate translational science.
June 2014 — NIH and NSF collaborated on an I-Corps pilot program to train business-minded biotech researchers. NCATS, the National Cancer Institute, the National Heart, Lung and Blood Institute, and the National Institute of Neurological Disorders and Stroke participated in the program.
July 2014 — The first drug candidate from an NIH program was acquired by a biopharmaceutical company. Baxter International acquired Aes-103, a potential treatment for sickle cell disease advanced by NCATS’ TRND program researchers and collaborators. This is the first time a company has acquired a drug candidate developed with TRND resources. View Image.
July 2014 — NCATS launched a chemical toxicity data model competition. The Toxicology in the 21st Century (Tox21) Data Challenge 2014 was a crowdsourcing competition to develop computational models that can better predict chemical toxicity.
July 2014 — NIH announced a three-year pilot project called Illuminating the Druggable Genome (IDG). The awards primarily are funded through the NIH Common Fund. Other NIH ICs including NCATS are supporting this effort.
July 2014 — The Michael J. Fox Foundation funded a research project that showcases how NCATS' chemical screening resources can advance development of potential therapeutics for a broad range of diseases.
August 2014 — NCATS and National Human Genome Research Institute (NHGRI) scientists developed a potential treatment for patients with Gaucher disease, a rare, inherited condition marked by enlargement of the liver and spleen, anemia, nose bleeds, easy bruising and bleeding, bone problems, and occasionally neurological problems.
September 2014 — NCATS solicited applications for institutional CTSAs.
September 2014 — NIH funded the next phase of the Tissue Chip for Drug Screening program to integrate tissue chips and test drug effects.
October 2014 — NCATS announced new research projects to improve treatments for rare blood disorders and infectious diseases. The research, supported through the Center's TRND program, also is designed to provide insights that will broadly improve and accelerate the translational science process.
October 2014 — NIH announced three five-year grants for frontotemporal degeneration research. The projects are being funded by NCATS, the National Institute of Neurological Disorders and Stroke, and the National Institute on Aging.
October 2014 — NCATS supported two new BrIDGs projects to develop treatments for diabetes-related blindness and severe heart attacks.
November 2014 — NCATS experts, academic researchers, a patient advocacy group and a pharmaceutical company collaborated to identify a potential drug target for an inherited neurological disorder called Charcot-Marie-Tooth disease.
November 2014 — NCATS repurposing test identified 53 drugs that may block Ebola infection.
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.
Christopher P. Austin, M.D., is director of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH). Austin leads the Center’s work to improve the translation of observations in the laboratory, clinic and community into interventions that reach and benefit patients — from diagnostics and therapeutics to medical procedures and behavioral changes. Under his direction, NCATS researchers and collaborators are developing new technologies, resources and collaborative research models; demonstrating their usefulness; and disseminating the data, analysis and methodologies for use by the worldwide research community.
Austin’s career has spanned the spectrum of translational research in the public and private sectors. He joined NIH in 2002 as the senior advisor to the director for translational research at the National Human Genome Research Institute (NHGRI), where he was responsible for conceptualizing and implementing research programs to derive scientific insights and therapeutic benefits from the results of the newly completed Human Genome Project. While at NHGRI, Austin founded and directed the NIH Chemical Genomics Center (now the NCATS Chemical Genomics Center), Therapeutics for Rare and Neglected Diseases program, Toxicology in the 21st Century initiative, and NIH Center for Translational Therapeutics. When NCATS launched in late 2011, Austin became the inaugural director of the Center’s Division of Pre-Clinical Innovation, and then was appointed as the NCATS director in 2012. Before joining NIH, Austin worked at the pharmaceutical company Merck, where he directed programs on genome-based discovery of novel targets and drugs, with a particular focus on treatments for schizophrenia and Alzheimer’s disease.
Austin is trained as a clinician and geneticist. He earned an M.D. from Harvard Medical School and an A.B. summa cum laude in biology from Princeton University. He completed a research fellowship in developmental neurogenetics at Harvard, studying genetic and environmental influences on stem cell fate determination. Austin also trained in internal medicine and neurology at the Massachusetts General Hospital in Boston, after which he practiced medicine in academic and community hospitals, providing primary care in urban settings and in rural Alaska and Africa.
|Name||In Office from||To|
|Thomas R. Insel (Acting)||December 23, 2011||September 22, 2012|
|Christopher P. Austin||September 23, 2012||Present|
NCATS’ programs and initiatives span the entire spectrum of translational science.
- Bridging Interventional Development Gaps (BrIDGs) program. BrIDGs provides targeted contract access to drug development resources that allow academic, not-for-profit and small business collaborators to generate the data they need to submit an Investigational New Drug application to the FDA.
- Clinical and Translational Science Awards (CTSA) program. This innovative national network of medical research institutions — called “hubs” — helps NCATS tackle system-wide and operational problems to make the clinical and translational research enterprise more efficient. CTSA hubs collaborate locally, regionally and nationally to foster innovation in all aspects of translational science, including training the workforce, patient and community engagement, multisite clinical trials, and new methodologies such as cutting-edge informatics.
- Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) program. Using a crowdsourcing approach, the program supports collaborations between pharmaceutical companies and academic researchers to rapidly test new ideas for existing investigational compounds.
- Extracellular RNA (exRNA) Communication program. 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 that holds enormous promise for improving our understanding of a wide variety of diseases.
- Genetic and Rare Disease Information Center (GARD). This collaboration with the National Human Genome Research Institute offers comprehensive information on rare and genetic diseases to patients, their families, health care providers and the public.
- NIH/NCATS Global Rare Diseases Patient Registry Data Repository/GRDR® program is designed to advance research for rare diseases and, through application of scientific insights gained, to further research for common diseases as well.
- Pfizer’s Centers for Therapeutic Innovation (CTI) for NIH Researchers. This innovative collaboration is led by NCATS and designed to help bridge the gap between early scientific discovery and its translation into new medicines through public-private resource sharing. Pfizer’s CTI program pairs leading researchers with Pfizer resources to pursue scientific and medical advances through joint therapeutic development. Partners in the CTI network include 25 academic institutions, four patient foundations and now NIH.
- Rare Diseases Clinical Research Network (RDCRN). This initiative, which involves researchers and patient engagement groups, aims to address many of the challenges in studying and developing treatments for rare diseases, including difficulties in diagnosis, widely dispersed patients and scientific experts, and a perceived high risk and cost for developing such therapies.
- RNA interference (RNAi). Gene silencing through RNAi 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.
- Therapeutics for Rare and Neglected Diseases (TRND) program. TRND supports research collaborations among NIH and academic scientists, patient organizations and biopharmaceutical companies to encourage and speed the discovery of new drugs for rare and neglected diseases.
- Tissue Chip for Drug Screening (Tissue Chip) initiative. This partnership with the Defense Advanced Research Projects Agency and the FDA is designed to develop 3-D human tissue chips that model the structure and function of human organs, such as the lung, liver and heart, and then combine these chips into an integrated system that can mimic complex functions of the human body.
- Toxicology in the 21st Century (Tox21) initiative. Tox21 is a collaborative effort among NIH — including NCATS and the National Toxicology Program at the National Institute of Environmental Health Sciences — the Environmental Protection Agency and the FDA. Through Tox21, researchers are testing 10,000 drugs and environmental chemicals for their potential to affect molecules and cells in ways that can cause health problems. The compounds undergo testing in NCATS’ high-speed robotic screening system.