National Center for Advancing Translational Sciences (NCATS)

• Mission
• Important Events
• Legislative Chronology
• Director
• Major Programs

Mission

NCATS’s mission is to turn research observations into health solutions through translational science. The goal is more treatments for all people more quickly.

The Center works to enhance the development, testing and implementation of diagnostics and therapeutics for a wide range of diseases and conditions. Key approaches include understanding what’s similar across diseases to develop multiple treatments at a time, building models that better predict a person’s reaction to a treatment and enhancing clinical trials so the results reflect the full diversity of the population. NCATS collaborates with other government agencies, including other NIH ICs; industry; academia; and patient support organizations.

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.

March 2012 — NCATS teamed with Eli Lilly and Company to assess biological profiles of medicines and molecules that may enable biomedical researchers to better predict treatment outcomes, improve drug development, and lead to more specific and effective approaches.

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 creating partnerships between pharmaceutical companies and the biomedical research community.

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 — 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 Institutes and Centers (ICs).

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 — NCATS announced the members of its inaugural Advisory Council and Cures Acceleration Network Review Board.

September 2012 — NIH Director Francis S. Collins, M.D., Ph.D., announced the appointment of Christopher P. Austin, M.D., as director of NCATS.

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).

November 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.

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 (NPC1).

March 2013 — To investigate new ways to treat glaucoma, NCATS partnered with the Johns Hopkins School of Medicine in Baltimore. The 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.

June 2013 — As part of a pilot initiative, NCATS announced New Therapeutic Uses program awards to find new treatments for patients in eight disease areas.

June 2013 — The National Academies of Sciences, Engineering, and Medicine (formerly known as the Institute of Medicine) released The CTSA Program at NIH: Opportunities for Advancing Clinical and Translational Research.

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. Researchers at the National Institute of Neurological Disorders and Stroke (NINDS) 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.

December 2013 — Pamela M. McInnes, D.D.S., M.Sc.(Dent.) was named deputy director of NCATS, effective Jan. 12, 2014.

December 2013 — NCATS collaborated with Life Technologies Corporation to make gene-silencing data on the biochemical makeup of small interfering RNA molecules publicly available.

February 2014 — NCATS and National Cancer Institute (NCI) scientists published the first results from a test of a combination drug screening platform.

February 2014 — NCATS and the NIH Clinical Center hosted Rare Disease Day at NIH 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. Since 2012, NCATS has played a major role in each annual event.

April 2014 — NCATS released its first annual report (2012-2013).

May 2014 — NCATS Advisory Council working group released findings on a 2013 report about the Clinical and Translational Science Awards (CTSA) Program released by the National Academies of Sciences, Engineering, and Medicine (formerly known as the Institute of Medicine).

May 2014 — CTSA Program institutions participating in institutional review board (IRB) reliance networks showed that efficient and centralized oversight can accelerate translational science.

June 2014 — NIH and NSF collaborated on an Innovation Corps (I-Corps^TM) pilot program to train business-minded biotech researchers. NCATS, the NCI, the National Heart, Lung and Blood Institute (NHLBI), and NINDS participated in the program.

July 2014 — Baxter International acquired Aes-103, a potential treatment for sickle cell disease advanced by NCATS’ TRND program researchers and collaborators. This was the first time a company has acquired a drug candidate developed with TRND resources.

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 — NIH funded the next phase of the NCATS-led Tissue Chip for Drug Screening program to integrate tissue chips and test drug effects.

October 2014 — NIH awarded $29 million to expand the Rare Diseases Clinical Research Network (RDCRN) to study more than 200 rare diseases.

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 — An NCATS repurposing test identified 53 drugs that may block Ebola infection.

January 2015 — NCATS and the Eunice Kennedy Shriver National Institute of Child Health and Human Development entered into an agreement with biotechnology company Vtesse, Inc., to develop treatments for Niemann-Pick disease type C (NPC) and other lysosomal storage disorders.

January 2015 — NCATS announced the winners of the Tox21 Data Challenge 2014, a crowdsourcing competition that attracted contestants from 18 countries to design computational models to better predict chemical toxicity.

March 2015 — Supported in part by NCATS, scientists found that a compound originally developed as a cancer therapy potentially could be used to treat Alzheimer’s disease.

April 2015 — NCATS released two funding opportunity announcements for Collaborative Innovation Awards, designed to stimulate team-based research across the CTSA Program consortium.

April 2015 — NCATS launched an initiative to wash and reuse plastic plates used during high-throughput robotic drug screening.

April 2015 — NCATS-supported researchers found that an over-the-counter drug indicated to treat allergy symptoms limited hepatitis C virus activity in infected mice.

May 2015 — An NCATS Chemical Genomics Center project team constructed a 3-D model of ovarian cancer metastasis using cells from patients.

June 2015 — NCATS released two CTSA Program funding opportunity announcements, one for Recruitment Innovation Centers and the other for Trial Innovation Centers. Both are aimed at overcoming key roadblocks to multisite clinical trials.

July 2015 — NCATS released its 2014 annual report, featuring the Center’s major milestones, programs and initiatives.

July 2015 — NCATS awarded nearly $3 million to support four academic research groups in testing a selection of pharmaceutical industry assets for new therapeutic uses.

July 2015 — NCATS-supported research enabled five men with complete motor paralysis to voluntarily generate step-like movements thanks to a new strategy that non-invasively delivers electrical stimulation to their spinal cords.

August 2015 — The White House announced that NIH is expanding its I-Corps™ training program to accelerate the commercialization of biomedical technologies developed with federal Small Business Innovation Research (SBIR) and Small Business Technology Transfer funding.

August 2015 — Supported by NCATS’ Tissue Chip for Drug Screening program, a team of scientists from Northwestern University, Charles Stark Draper Laboratory and the University of Illinois at Chicago designed a miniaturized 3-D representation of the female reproductive tract and liver — called EVATAR™ — on a handheld, interconnected platform for use in drug testing and to study the basic biology of female reproduction.

September 2015 — NCATS researchers and collaborators from the NIH’s National Institute of Allergy and Infectious Diseases (NIAID), Georgetown University, and the University of California, San Francisco, released a large dataset of potential drug combinations for malaria.

September 2015 — NCATS announced it would spearhead the second phase of several NIH ExRNA Communication program projects to test and validate exRNA molecules for their potential as disease biomarkers and treatments. The ExRNA Communications program is supported through the NIH Common Fund.

September 2015 — An NIH initiative expanded NCATS’ Tox21 program to investigate the effects of environmental chemicals on human development using robotic screening of cultured cells.

December 2015 — An NCATS-supported study revealed new genetic clues to age-related macular degeneration.

January 2016 — NCATS, the U.S. Environmental Protection Agency, and NIH's National Toxicology Program within the National Institute of Environmental Health Sciences announced a challenge that would award up to $1 million to improve the relevance and predictivity of data generated from automated chemical screening technology used for toxicity testing.

March 2016 — NCATS’ small business funding helped launch a new platform for rare diseases drug discovery.

March 2016 — RDCRN researchers partnered with The LAM Foundation, culminating in the first U.S. Food and Drug Administration (FDA)–approved treatment for lymphangioleiomyomatosis (LAM).

May 2016 — NCATS introduced plans for its new single IRB reliance platform for multisite clinical studies.

May 2016 — An NIH-funded team, supported in part by NCATS, found ketamine lifts depression via a byproduct of its metabolism.

May 2016 — NCATS scientists contributed to an NIH study that visualized proteins involved in cancer cell metabolism with an approach that may have an impact on drug discovery and development.

June 2016 — NCATS-supported researchers developed a clinical-grade stem cell line with the potential to accelerate the advancement of new medical applications and cell-based therapies.

July 2016 — NCATS scientists collaborated with NHGRI and NINDS researchers to identify and test a molecule that shows promise as a possible treatment for the rare Gaucher disease and the more common Parkinson’s disease.

August 2016 — NCATS researchers identified compounds that potentially can be used to inhibit Zika virus replication and reduce its ability to kill brain cells.

September 2016 — NCATS released its 2015 annual report, featuring the Center’s major milestones, programs and initiatives.

September 2016 — NCATS announced the Bench-to-Clinic Repurposing initiative and related funding opportunity to support investigators in repurposing existing experimental drugs or biologics, as well as FDA-approved therapies already on the market.

October 2016 — NCATS and the Center for the Advancement of Science in Space announced a collaboration to use tissue chip technology for translational research at the International Space Station U.S. National Laboratory.

October 2016 — NCATS partnered with the Stimulating Peripheral Activity to Relieve Conditions program to support research on the peripheral nervous system in hopes of finding new ways to treat conditions such as asthma, diabetes and nausea.

October 2016 — Scientific experts from 11 institutions began collaborating to assess the feasibility of developing the Biomedical Data Translator. Through this effort, NCATS integrated existing biomedical data to help reveal new relationships within those data and identify novel opportunities for research.

October 2016 — NCATS announced approximately $6 million in new awards for fiscal year 2016 to establish three testing centers for the Tissue Chip for Drug Screening program.

October 2016 — An NCATS-supported study found that a wearable patch that delivers small amounts of peanut protein through the skin showed promise for treating children and young adults with peanut allergy.

October 2016 — NCATS-supported Zika research in male mice revealed reproductive implications for human males.

November 2016 — NCATS released the Center’s strategic plan, organized into four overarching themes: translational science, collaboration and partnerships, education and training, and stewardship. The themes are captured in the strategic goals and collectively provide an overview of what the Center plans to accomplish to achieve its mission.

November 2016 — NCATS and NIAID researchers created a rapid screening test to identify drugs and drug combinations that may potentially be useful in combating infections that are resistant to many different antibiotics.

January 2017 — NCATS scientists, working with collaborators from the University of New Mexico and the University of Miami as part of the IDG Knowledge Management Center, developed the Target Central Resource Database (TCRD), which collates many heterogeneous gene/protein datasets, and Pharos, a multimodal web interface that presents the data from TCRD.

January 2017 — An unprecedented trans-NCATS collaboration enabled the rapid advancement of a rare lung disease therapy to human trials.

March 2017 — NCATS and NINDS-supported scientists discovered a urinary biomarker that may help track amyotrophic lateral sclerosis.

March 2017 — NCATS announced that all CTSA Program sites signed on to the NCATS Streamlined, Multisite, Accelerated Resources for Trials (SMART) IRB authorization agreement. This agreement enabled all participating study sites to rely on the ethics review of one IRB for each study, making it possible to initiate multisite studies within weeks instead of months.

March 2017 — Patient-focused translational science collaboration among government and academic scientists, patients, and patients’ advocates advanced a potential treatment for the rare disease NPC1.

April 2017 — Teams led by NCATS and University of Tokyo scientists identified the first inhibitor of an enzyme long thought to be a potential drug target for fighting disease-causing parasites and bacteria.

April 2017 — NCATS-supported researchers found that an experimental treatment cured 100 percent of guinea pigs and rhesus monkeys in late stages of infection with lethal levels of Marburg and Ravn viruses, relatives of the Ebola virus.

April 2017 — NCATS CTSA Program mentoring support sparked an interdisciplinary translational research team that developed a potential therapy for brain cancer.

May 2017 — Researchers from NCATS, NHLBI and the University of Tokyo identified the first inhibitor of an enzyme long thought to be a potential drug target for fighting disease-causing parasites and bacteria.

May 2017 — The NCATS SMART IRB team launched an Online Reliance System to help streamline the ethics review conducted by IRBs.

June 2017 — NCATS and the University of Nevada, Reno School of Medicine researchers demonstrated that a drug originally targeted unsuccessfully to treat cancer may have new life as a potential treatment for Duchenne muscular dystrophy.

June 2017 — NCATS and the Center for the Advancement of Science in Space announced five grants to support research on human physiology and disease onboard the International Space Station U.S. National Laboratory.

June 2017 — NCATS hosted an inaugural event — NCATS Day — to help identify patients’ needs and brainstorm ways to foster engagement at every step of the translational science spectrum. The Center hosted the event in 2018 and 2019.

June 2017 — NCATS partnered with Eli Lilly and Company on the NCATS-Eli Lilly scholars’ externship program. The effort paired scholars, trainees and investigators from NCATS’ CTSA Program with an Eli Lilly project team for up to one year.

June 2017 — An NCATS-supported multidisciplinary team addressed translational research hurdles — including ethics review and informed consent processes — to add genetic tests to newborn screenings and improve early diagnosis of fragile X syndrome and other rare genetic diseases.

July 2017 — NCATS-supported scientists demonstrated how an investigational drug works against a rare, fatal genetic disease, NPC1.

July 2017 — Through its Small Business Innovation Research program, NCATS supported the development of a technology to use electronic health records data to advance value-based and enhanced patient care.

August 2017 — Supported in part by NCATS, a team of scientists examined the maturation of astrocytes in 3-D structures grown in culture dishes to resemble human brain tissue. This research addresses a significant gap in human brain research by providing an invaluable technique to investigate the role of astrocytes in both normal development and disease.

September 2017 — A collaboration between researchers from NCATS’ TRND program and Agilis Biotherapeutics, Inc., of Cambridge, Massachusetts, resulted in a gene therapy for the rare pediatric condition aromatic L-amino acid decarboxylase deficiency moving closer to market years ahead of schedule.

September 2017 — Through its Tissue Chip for Drug Screening program, NCATS awarded $15 million to support development of 3-D microphysiological system platforms that model human disease.

September 2017 — Supported in part by NCATS, researchers found that children exposed to high indoor levels of pet or pest allergens during infancy have a lower risk of developing asthma by 7 years of age. The findings may provide clues for the design of strategies to prevent asthma from developing.

September 2017 — Supported in part by NCATS, research conducted in mice provided evidence that highly lethal brain tumors, called high-grade gliomas, stop growing when deprived of a specific molecule naturally produced when brain cells fire.

November 2017 — Supported in part by NCATS, scientists identified a potential drug that could lead to new approaches for preserving brain cells after an ischemic stroke.

November 2017 — NCATS CTSA Program-supported investigators collaborated to validate biomarkers intended to monitor the response to therapy for NPC1.

December 2017 — A team of NIH-supported researchers explored the use of a highly sensitive technique to counteract hearing loss caused by cisplatin, a powerful drug used to treat cancer. The team, which included NCATS scientists, measured and mapped cisplatin build-up in the inner ear and found a region that could be targeted to prevent hearing loss.

December 2017 — NCATS-supported researchers demonstrated the value in a community-based research evaluation model of consultation services that is helping to translate clinical practice observations into research-supported treatment approaches.

January 2018 — NIH launched the Somatic Cell Genome Editing (SCGE) program aimed at removing barriers to slow the adoption of genome editing for treating patients. This program is supported by the NIH Common Fund and is managed by a trans-agency working group, led by NCATS.

January 2018 — NCATS’ TRND program collaborated with scientists at Duke University’s Clinical and Translational Science Institute to advance a gene therapy for a rare neuromuscular disorder toward clinical testing.

January 2018 — NCATS scientists and collaborators demonstrated that blocking the activity of a certain enzyme reduced the effects of Huntington’s disease in cell and animal models.

February 2018 — Scientists from NCATS and Sweden’s Karolinska Institutet developed a potential new approach to fighting cancer by breaking down a defense system used by cancer cells. The defense system involves an enzyme, thioredoxin reductase 1 (TrxR1), which supports cancer cell survival.

March 2018 — NCATS supported an innovative platform technology to precisely deliver nutrients and hormones to cells for a preclinical therapeutics testing program.

March 2018 — Tox21 program partners from NCATS and other collaborators published a new strategic and operational plan to broaden the scope of their research activities to address new challenges.

April 2018 — NCATS-supported researchers made significant strides in reliably engineering human heart muscle that mimics the intricacies of a human heart, which could serve as a better model for testing the effects of toxic substances than current tissue-engineered heart models.

April 2018 — NCATS-funded researchers used stem cells originally derived from a person’s skin to recreate interactions between blood vessels and neurons that may occur early in the formation of the fetal human spinal cord, which may potentially be used to test personalized treatments of neurological disorders.

April 2018 — Through its New Therapeutic Uses program, NCATS awarded funding for three new projects to repurpose compounds that have already undergone significant research and development by industry, including testing in humans.

May 2018 — Scientists at NIH, Northwestern University and their collaborative research partners identified a compound that blocks the spread of pancreatic and other cancers in various animal models. This research was in-part funded by NCATS.

May 2018 — NCATS Biomedical Data Translator teams implemented a unique and collaborative approach to advance biomedical data sharing.

May 2018 — NIH's RDCRN-supported research helped overcome a translational roadblock by charting how several rare, inherited disorders progress in patients over time, which enabled a potential rare disease gene editing treatment into clinical trials.

July 2018 — NCATS-supported researchers developed an innovative device that detects internal bleeding and monitors a patient’s response to blood loss.

August 2018 — Researchers at Cedars-Sinai Medical Center and the University of California, Los Angeles, part of NCATS’ CTSA Program, paired pharmacists with barbershops to help prevent heart attacks and strokes.

August 2018 — NCATS scientists created a simple, fast and automated process to make 3-D tissues transparent, which enables many types of observations within 3-D growth environments.

August 2018 — NCATS created a new online tool — NCATS Inxight: Drugs — that aggregates reliable, curated drug development data from multiple existing sources to help researchers find the information they need more easily.

August 2018 — NCATS and the FDA co-hosted a two-day workshop about gene therapy for rare diseases.

September 2018 — NCATS launched the Rare Diseases Are Not Rare! Challenge seeking creative ways to help raise awareness about rare diseases. Winners and honorable mentions were announced in December 2018. In February 2020, NCATS ran the Challenge again and announced winners and honorable mentions in August 2020. View the gallery of 2020 winning entries.

October 2018 — NCATS’ CTSA Program support enabled the early development and “de-risking” of an innovative device that may help patients avoid complications of heart valve replacement.

October 2018 — NIH awarded $86 million to support research aimed at improving methods to edit the human genome. These grants were the first to be awarded through the SCGE program, which is managed by staff from multiple NIH ICs, with leadership from NCATS.

November 2018 — Joni L. Rutter, Ph.D., joined NCATS as the Center’s new deputy director.

December 2018 — NCATS announced it would support three new funding opportunities and a prize challenge through the NIH Helping to End Addiction Long-term^® Initiative, or the NIH HEAL^® Initiative.

December 2018 — NCATS partnered with the ISSNL to send tissue chips into space for research initiatives leveraging the unique microgravity environment.

December 2018 — CTSA Program-supported investigators devised and piloted an innovative approach called Real-Time IRB that reduces the time between study submission to the institutional review board and final approval by 70 percent.

December 2018 — Researchers supported by NCATS’ Tissue Chip for Drug Screening program developed a 3-D working model of a human heart chamber.

December 2018 — NCATS researchers overcame a translational roadblock by developing tests to identify compounds that disrupt a driver of several cancers. Their work, which involved using NCATS’ high-throughput screening technology to evaluate thousands of compounds at once, provided a template for other scientists to discover new compounds that could be useful as therapies.

January 2019 — NCATS examined the biological activities of natural products to evaluate the compounds’ possible uses in treating a variety of diseases. The findings make up the Canvass library of natural products.

February 2019 — NIH launched the Platform Vector Gene Therapy (PaVe-GT) initiative, led by NCATS, to test whether it is feasible to use the same gene delivery system and manufacturing methods for multiple rare diseases in gene therapy clinical trials.

February 2019 — NCATS, the National Institute on Drug Abuse and NINDS co-sponsored The Opioid Crisis and the Future of Addiction and Pain Therapeutics: Opportunities, Tools, and Technologies Symposium.

April 2019 — Scientists from the Extracellular RNA Communication Consortium published a landmark collection of papers on the biology and possible clinical applications of exRNA. The ExRNA Communication program is funded by the NIH Common Fund and led by staff from the Common Fund, NCATS and other NIH ICs.

May 2019 — The fifth edition of the International Congress on Research of Rare and Orphan Diseases was co-organized in collaboration with NCATS to bring together scientists to share research and exchange ideas.

May 2019 — NCATS-supported researchers sent four tissue chip projects into space to speed the study of aging-related conditions and the development of treatments for them.

May 2019 — NCATS and other members of an international group called Translational Together identified qualities that distinguish translational scientists.

June 2019 — NCATS sponsored a Workshop on CNS Immunogenicity Considerations for adeno-associated virus (AAV)-mediated Gene Therapy that brought together the scientific and medical expert community for AAV-mediated therapies to focus on issues related to the central nervous system administration and immunogenicity of these products.

July 2019 — NCATS, the NCI and the National Institute of Biomedical Imaging and Bioengineering (NIBIB) co-sponsored a workshop to provide experts and the community the opportunity to share their perspectives on current issues associated with the incorporation of Artificial/Machine Intelligence systems into health care settings.

August 2019 — NCATS announced winners of the 2018 ASPIRE Design Challenges, which were launched as part of the NIH HEAL Initiative to encourage innovative and catalytic approaches toward solving the opioid crisis.

August 2019 — NCATS scientists and their colleagues created a one-of-a-kind database that details the body’s biological pathways, which can enable researchers to more readily study gene activity, gather insights on disease mechanisms, shed light on when and how compounds are toxic to cells and more.

September 2019 — Scientists from NIH, including NCATS and Cincinnati Children’s Hospital Medical Center devised a potential treatment against a common type of leukemia that could have implications for many other types of cancer.

September 2019 — NIH awarded approximately $89 million to advance additional projects for genome projects through the SCGE Program. The program is supported by the NIH Common Fund and led by NCATS.

September 2019 — NCATS collaborated with The Children's Inn at NIH, the Amateur Radio on the International Space Station and the International Space Station U.S. National Laboratory to host Ask an Astronaut: Biomedical Science Edition, which provided children receiving care at NIH the opportunity to talk to an astronaut in space about science.

November 2019 — NCATS and several other NIH ICs co-sponsored the NIH Inaugural Rural Health Seminar, which explored how innovations in clinical and translational science could improve rural health outcomes.

November 2019 — NCATS researchers and colleagues identified a drug pair that worked together to both kill cancer cells and counter the effects of a genetic mutation that causes the group of deadly childhood brain cancers.

December 2019 — NCATS hosted Repurposing Off-Patent Drugs: Research & Regulatory Challenges to discuss challenges around finding new uses for drugs that are already on the market but lack commercial and regulatory incentives for research and development.

February 2020 — Researchers from NCATS, NCI and Tuskegee University showed in laboratory experiments that a novel type of immune therapy can reprogram immune cells to kill cancer cells and halt tumor growth in several types of cancer.

March 2020 — NIH-supported researchers sent heart and gut tissue chip projects into space to speed the study of aging-related conditions and the development of treatments for them.

April 2020 — NCATS collaborated with NIAID, NIBIB and NCI to conduct a study to determine how many adults in the United States without a confirmed history of SARS-CoV-2 infection have antibodies to the virus.

May 2020 — NCATS scientists developed the COVID-19 OpenData Portal to share data from a large panel of experiments to test approved drugs against targets in or related to SARS-CoV-2.

May 2020 — The Rare Diseases Clinical Research Network, led by NCATS in collaboration with nine other NIH ICs, launched a research survey to find out how COVID-19 is affecting individuals with rare diseases, their families and their caregivers.

May 2020 — NCATS launched the OpenData Portal to share experimental data openly and quickly on how well new and existing therapeutics worked against SARS-CoV-2, including against emerging variants and mutations. It was a key resource for ACTIV Tracking Resistance and Coronavirus Evolution (TRACE) efforts.

June 2020 — Created through a collaboration between FDA and NCATS, CURE ID enabled the crowdsourcing of medical information from health care professionals to facilitate the development of new treatments using repurposed drugs for difficult-to-treat infectious diseases.

June 2020 — NCATS launched a centralized, secure enclave as part of the National COVID Cohort Collaborative (N3C) effort to enable researchers to study vast amounts of medical record data from people diagnosed with COVID-19. Researchers have used the resource to reveal risk factors for severe COVID-19 infection, detect trends in health outcomes among high-risk groups, and offer early insights into COVID’s long-term effects. To learn more about this collaboration, visit N3C In Action.

June 2020 — NCATS launched its first online translational science course, which focused on teaching key principles of translational science and how these principles have been operationalized in a real-world project. 

July 2020 — NCATS introduced the I Am Translational Science video series, which shares stories by researchers and others about what they do, what inspires their work and why they find translational science exciting. The initial stories featured people involved in COVID-19 efforts.

September 2020 — NIH awarded 10 grants to support researchers’ efforts in using tissue chips — tiny, bioengineered models of human tissues and organ systems — to study diseases and test drugs. The awards are administered through the new Clinical Trials on a Chip program, led by NCATS in conjunction with other NIH ICs.

October 2020 — NCATS and NIAID co-sponsored the NIH Virtual SARS-CoV-2 Antiviral Therapeutics Summit, which brought together experts from academia, industry and government to discuss the state of antiviral therapeutics development for COVID-19.

November 2020 — NCATS co-hosted the Virtual Workshop on Systemic Immunogenicity Considerations for AAV-Mediated Gene Therapy, which brought together scientists, therapeutic developers and patient representatives to focus on immunogenicity issues related to the systemic administration of AAV gene therapy products and identify solutions for those issues.

December 2020 — Three NCATS-supported tissue chip research projects were sent to the International Space Station to explore how to prevent osteoarthritis after joint injuries, what drives muscle wasting and whether heart tissue damage can be reversed.

January 2021 — A team of NCATS researchers and colleagues from other institutions used organoids, tiny 3-D tissue models of human brain development, to better understand brain infections caused by SARS-CoV-2.

February 2021 — NCATS scientists developed new 3-D tissue models to examine how respiratory viruses like the SARS-CoV-2 virus and influenza viruses can infect the lungs and other tissues and cause disease.

March 2021 — NCATS researchers developed an approach called biological activity-based modeling, which could speed the search for therapies for COVID-19 and other diseases.

March 2021 — NCATS Director Christopher P. Austin, M.D., announced his departure from NIH. Joni L. Rutter, Ph.D., was selected as acting director. 

April 2021 — NCATS oversaw a large, randomized, placebo-controlled Phase 3 clinical trial that evaluated the safety and effectiveness of several existing drugs to treat mild-to-moderate COVID-19 symptoms at home. The trial was part of the ACTIV public-private partnership.

May 2021 — Researchers in NCATS’ Stem Cell Translational Laboratory devised a four-part small-molecule cocktail called CEPT that can protect stem cells from damage when they are grown in the laboratory and, as a result, could make stem cells more useful for studying and treating diseases and testing drugs.

June 2021 — NCATS announced that, alongside key partners, it will play a pivotal role in the Antiviral Program for Pandemics, a multiagency initiative to develop safe and effective oral antivirals. 

June 2021 — NCATS scientists designed an innovative test to find potential treatments for Charcot-Marie-Tooth disease, one of the most common forms of inherited neurological disease. 

October 2021 — NCATS released the results of a pilot study led by the Center that indicate that individual medical costs for people with a rare disease are significantly higher than those for people who do not.

October 2021 — NCATS announced that it would co-lead the Bespoke Gene Therapy Consortium — a partnership among NIH, FDA and private organizations — to increase the number of effective gene therapies for rare diseases.

December 2021 — NCATS celebrated its 10-year anniversary, marking a decade of harnessing the power of translational science to speed solutions for many diseases at a time.

December 2021 — NIH-supported researchers sent blood-brain barrier tissue chips into space to help understand and treat neurodegenerative and immune disorders.

January 2022 — NCATS oversaw a Phase 3 randomized placebo-controlled clinical trial funded by NIH, which found that for people hospitalized with COVID-19, convalescent plasma taken from those who had recovered was not more effective than placebo in delivering clinical improvement.

March 2022 — Scientists at NCATS and Duke University discovered a compound that helps restore the brain’s normal dopamine levels, offering a potential approach to help treat addiction, Parkinson’s and age-related brain diseases.

April 2022 — NIH-supported researchers used the tissue chip model to show how a drug could potentially treat rare neuromuscular disorders.

May 2022 — A research team supported by NIH used data from the National COVID Cohort Collaborative to identify characteristics of people with long COVID and those likely to have it.

June 2022 — NCATS coordinated and oversaw a large randomized placebo-controlled clinical trial, which showed that treating adults hospitalized with COVID-19 with infliximab or abatacept did not significantly shorten time to recovery but did substantially improve clinical status and reduce deaths. The trial was part of the ACTIV public-private partnership.

August 2022 — An NIH-led team of scientists built and used a library of nanobodies to find promising new therapeutic leads for stopping viral activity.

August 2022 — The NCATS Streamlined, Multisite, Accelerated Resources for Trials (SMART) Institutional Review Board (IRB) agreement reached 1,000 participating sites. This milestone makes SMART IRB’s medical research study reliance agreement one of the largest in the United States.

September 2022 — Researchers at the Icahn School of Medicine at Mount Sinai in New York and NCATS reversed the effects of several life-threatening, inherited neurodegenerative diseases in patient cells and mice.

November 2022 — Joni L. Rutter, Ph.D., was selected as the permanent NCATS director.

December 2022 — NIH-funded researchers conducted a randomized clinical trial of 1,200 infants undergoing surgery for congenital heart disease, which found that the anti-inflammatory steroid methylprednisolone did not reduce the risk of death or other severe outcomes any more than a placebo.

January 2023 — NCATS, in partnership with several NIH institutes, developed a new approach to better understand the biology of polycystic kidney disease, an often-life-threatening genetic disorder that affects millions worldwide. Scientists combined two ways to model the disorder to show glucose’s role in forming PKD cysts.

February 2023 — A Clinical and Translational Science Awards (CTSA) Program–funded analysis of national health records redrew the maps used by clinicians to diagnose and treat three regional fungal infections, potentially speeding detection and treatment outside areas where the infections have historically been found.

May 2023 —An NCATS-supported study reported that nearly nine of 10 experts on rare diseases agreed that sequencing healthy newborns’ DNA to reveal treatable genetic disorders should be available for all infants.

June 2023 — NCATS scientists created an innovative process of turning human stem cells into millions of pain-sensing nerve cells that could help researchers speed up the search for new pain therapies.

July 2023 — NCATS scientists and their colleagues created human cell–based 3-D models of the midbrain in an ultra-rare neurological disease to provide new insights into the disease’s development and biology.

November 2023 — NCATS scientists helped create two inhibitors — FerroLOXIN-1 and FerroLOXIN-2 — that block a cell death process called ferroptosis. The process is linked to conditions from cancer to Alzheimer’s disease.

November 2023 — The NCATS-led Primary Immune Deficiency Treatment Consortium (PIDTC) showed that the newborn screening test they created for severe combined immunodeficiency — more commonly known as “bubble boy disease” — saved lives. When followed by early treatment, the five-year survival rate of children with the disorder increased from 73% before screening began to 87%.

December 2023 — Research by the NCATS-funded GEMINI study compared whole-genome sequencing with targeted testing in infants with a possible genetic disorder. This groundbreaking study showed the benefit of having genomic sequencing tests widely available and accessible.

February 2024 — Methotrexate has been the standard drug for treating blood cancers and rheumatoid arthritis, but it is known to have negative side effects. An NCATS-led research team designed a new way to build a better methotrexate that is less toxic and has fewer side effects.

February 2024 — NCATS co-funded a study with the National Institute of Neurological Disorders and Stroke that showed that understanding how a body processes prednisone may reveal which patients with Myasthenia gravis, a rare neuromuscular disorder, are more likely to respond to the drug.

February 2024 — The Nephrotic Syndrome Study Network (NEPTUNE) shared its Kidney Tissue Atlas, the most comprehensive human kidney cell and tissue catalog to date. NEPTUNE is part of the Rare Diseases Clinical Research Network and is jointly supported by NCATS and the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK).

February 2024 — New research into the drug auranofin showed promise in treating cancer, Alzheimer’s disease, and a host of rare conditions, such as inflammatory VEXAS syndrome, spinal muscular atrophy, and some types of lung cancer. Originally approved to treat rheumatoid arthritis, auranofin can increase cells’ ability to remove damaged proteins faster with fewer side effects. A research team from NCATS, NIDDK and the University of Maryland, Baltimore found that the drug can be repurposed.

March 2024 — NCATS’ Therapeutics for Rare and Neglected Diseases (TRND) program was a key partner in getting the drug vamorolone approved by the U.S. Food and Drug Administration to treat Duchenne muscular dystrophy (DMD). TRND scientists performed preclinical studies to show the efficacy and cost-effectiveness of this drug against DMD.

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.

December 13, 2016 — The 21st Century Cures Act changed NCATS annual report requirement to a biennial report and modified NCATS’ restriction on supporting clinical trials to allow support for all phase II clinical trials and support for phase III clinical trials on rare diseases.

March 27, 2020 — President Trump signed the Coronavirus Aid, Relief and Economic Security (CARES) Act, providing funding to NCATS and several other ICs to advance coronavirus research.

Biographical Sketch of NCATS Director Joni L. Rutter, Ph.D.

Joni L. Rutter, Ph.D., is the director of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH). Rutter oversees the planning and execution of the Center’s complex, multifaceted programs that aim to overcome scientific and operational barriers impeding the development and delivery of new treatments and other health solutions. Under her direction, NCATS supports innovative tools and strategies to make each step in the translational process more effective and efficient, thus speeding research across a range of diseases, with a particular focus on rare diseases. By advancing the science of translation, NCATS helps turn promising research discoveries into real-world applications that improve people’s health. In her previous role as the NCATS deputy director, Rutter collaborated with colleagues from government, academia, industry and nonprofit patient organizations to establish robust interactions with NCATS programs.

Prior to joining NCATS, Rutter served as the director of scientific programs within the All of Us Research Program, where she led the scientific programmatic development and implementation efforts to build a national research cohort of at least 1 million U.S. participants to advance precision medicine. During her time at NIH, she also has led the Division of Neuroscience and Behavior at the National Institute on Drug Abuse (NIDA). In this role, she developed and coordinated research on basic and clinical neuroscience, brain and behavioral development, genetics, epigenetics, computational neuroscience, bioinformatics, and drug discovery. Rutter also coordinated the NIDA Genetics Consortium and biospecimen repository.

Throughout her career, Rutter has earned a national and international reputation for her diverse and unique expertise via more than 50 publications in journals. She has received several scientific achievement awards, including a SmithKline Beecham Student Award in Pharmacology, a Janssen Research Foundation Young Investigator Award and a Fellowship Achievement Award from the National Cancer Institute (NCI).

Rutter received her Ph.D. from the Department of Pharmacology and Toxicology, Dartmouth Medical School, Hanover, New Hampshire, and completed a fellowship at NCI within the Division of Cancer Epidemiology and Genetics.

NCATS Directors

Name	In Office from	To
Thomas R. Insel (Acting)	December 23, 2011	September 22, 2012
Christopher P. Austin	September 23, 2012	April 15, 2021
Joni L. Rutter	April 16, 2021	Present

Major Programs

NCATS has several divisions and offices with diverse staff who guide the center’s research activities. Programs and initiatives administered within these divisions and offices span the entire spectrum of translational science.

Office of the Director
The Office of the Director develops and provides leadership for the center’s translational science programs, manages and directs executive level activities, and provides policy guidance and coordination for each of the center’s components.

Division of Clinical Innovation
The Division of Clinical Innovation plans, conducts and supports research across the clinical phases of the translational science spectrum.

Division of Extramural Activities
The Division of Extramural Activities advises NCATS leadership on issues related to policy and procedures for extramural activities. Additionally, it oversees and directs scientific review and grants management activities at NCATS. The division also manages the operations of the NCATS Advisory Council and Cures Acceleration Network Review Board.

Division of Preclinical Innovation
The Division of Preclinical Innovation plans and conducts collaborative research projects across the preclinical phases of the translational science spectrum, using both internal and contract resources to advance them.

Division of Rare Diseases Research Innovation
The Division of Rare Diseases Research Innovation facilitates and coordinates NIH-wide activities involving research for a broad array of rare diseases.

Office of Administrative Management
The Office of Administrative Management directs the administrative and financial operations management of the center.

Office of Drug Development Partnership Programs
The Office of Drug Development Partnership Programs promotes innovations that improve the efficiency of drug development from target identification — including expanding the target landscape — through early-stage clinical trials.

Office of Policy, Communications and Education
The Office of Policy, Communications and Education develops and communicates critical priorities for NCATS in a highly collaborative manner.

Office of Special Initiatives
The Office of Special Initiatives addresses translational problems with innovative solutions through the development and implementation of disruptive technologies using interdisciplinary approaches and novel public-private partnerships.

Office of Strategic Alliances
The Office of Strategic Alliances establishes and advances public-private partnerships, as well as develops innovative approaches, policies and methods to reduce, remove or bypass bottlenecks in translational science collaborations.

Office of Translational Medicine
The Office of Translational Medicine uses expertise across clinical and other relevant disciplines to amplify NCATS’ ability to foster innovative translational science and improve health.