| ID | Title | Body | Facebook Description | Facebook image | Facebook Title | Facebook Video | Twitter Description | Twitter Image | Twitter Title | Meta tags |
|---|---|---|---|---|---|---|---|---|---|---|
| 8046 | News Brief: CTSA Program Supports Research Network in Geographically Isolated Region | With support from NCATS’ Clinical and Translational Science Awards (CTSA) Program, the University of New Mexico Clinical and Translational Science Center (UNM CTSC) was a founding partner in the Mountain West Research Consortium (MWRC), currently a network of 11 universities spanning seven states. The MWRC’s goal is to build and enhance the geographically isolated region’s clinical and translational research capacity. “A challenge in these states is building infrastructure for clinical and translational research,” said Richard Larson, M.D., Ph.D., principal investigator at the UNM CTSC. “The network we’ve created provides support to arm Mountain West researchers with training, data and knowledge that helps them collaborate with basic research colleagues, successfully apply for funding, and launch their first clinical trials.” The UNM CTSC serves as a biostatistics support core for the MWRC, featuring services and training for consortium statisticians to build biostatistics capabilities locally. Investigators share a broad range of knowledge, expertise and resources, and they host an annual summer immersion program in clinical and translational science for undergraduate students from consortium institutions. UNM CTSC representatives also provide grant writing mentoring, and in 2013 they worked with other MWRC representatives to collectively apply for and receive NIH support through the National Institute of General Medical Sciences’ Institutional Development Award (IDeA) program. The IDeA program fosters health-related research and enhances the competitiveness of investigators at institutions in states with historically low success rates for NIH applications. The IDeA funding is supporting MWRC’s clinical and translational research on diseases that often affect medically underserved populations. Posted March 2016 | asdfasfdasdf | asdfasfdasdf | ||||||
| 8044 | News Brief: CTSA Program Collaborations Spark Broad Public Engagement in Common Rule NPRM | Clinical and Translational Science Awards (CTSA) Program representatives recently collaborated with patient advocacy groups to host four national meetings promoting public engagement in the Notice of Proposed Rulemaking (NPRM) on the Federal Policy for the Protection of Human Subjects. The NPRM was released by federal agencies, including the U.S. Department of Health and Human Services, in September 2015. The goal was to garner public comment on proposals to improve and modernize what is known as the Common Rule, which is a set of federal regulations to protect human subjects involved in trials while facilitating valuable research and reducing burden, delay and ambiguity for investigators. In coordination with FasterCures, the Center for Information & Study on Clinical Research Participation, Genetic Alliance and other organizations, CTSA Program representatives planned and executed the meetings within a two-month period in fall 2015 to coincide with the Common Rule public comment period. The meetings were held in Nashville, San Diego, Chicago and Philadelphia, and each was thematically organized to focus on a different aspect of the proposed changes. The goals were to: Enhance understanding of the NPRM. Facilitate a robust exchange of ideas among a broad range of research stakeholders about the proposed changes and their implications. Promote informed public engagement in the policy making process. Following extensive outreach to more than 170 stakeholder groups, the combined attendance for all four meetings exceeded 1,400, with 240 attending in person and more than 1,100 participating via webinar. Every effort was made to achieve diversity among participants and to ensure the attendance and active participation of all stakeholder groups, particularly patient advocacy organizations, underrepresented minorities, underserved groups and vulnerable populations. The majority of attendees were from academic medical centers, and private citizens composed the next largest group. Evaluation surveys indicated an increased level of understanding of the NPRM following each event. Posted March 2016 | asdf | asdf | ||||||
| 8042 | News Brief: CTSA Program Collaboration in Action | Improving Patient-Reported Outcome Data for Research through Seamless Integration of the PROMIS Toolkit and Computer-Adaptive Testing Modules into EHR Workflow CTSA Program Hub: Northwestern University Dr. Stephan Schuele (right) leads the Northwestern University Comprehensive Epilepsy Center based at Northwestern Memorial Hospital. The center's team of neurologists, neurosurgeons, radiologists, neuropsychologists, electrophysiologists, and nuclear medicine specialists apply the latest advances in molecular biology, imaging, and pharmacology to diagnose and treat epilepsy. Patient-reported outcomes (PROs) reflect the experience of health and health care as reported directly by the patient. There is increasing evidence that capturing PROs is an essential component of quality measurement, quality improvement, and patient engagement in care and research. The Patient-Reported Outcomes Measurement Information System (PROMIS) toolset is a PRO survey system that utilizes computer-adaptive testing (CAT) to provide precise measurements with a minimum number of questions, often shortening conventional PRO surveys 10-fold or more. Unfortunately, previous attempts to integrate PROMIS into electronic health records (EHRs) have not been entirely successful. Working with the PROMIS software team, NUCATS has developed a seamless integration of the PROMIS toolset into our local Epic EHR installation. This experience has convinced us that tight workflow integration brings many benefits and greatly facilitates incorporation of PROs into both quality and clinical research projects, while minimizing the burden on patients and research participants. The response to our presentation of this work has also demonstrated that there is a need for similar integration at many CTSA Program sites. This project represents a collaboration of nine CTSA Program sites, including Northwestern, University of Chicago, University of Illinois at Chicago, University of Alabama at Birmingham, University of Kentucky, University of Florida, University of Utah, Harvard Catalyst CTS, and Southern California CTSI. These sites utilize a variety of different EHR platforms. The team includes the developers of the PROMIS toolkit software, experts in EHR integration, and experts at SMART and FHIR). The goal of this project is to develop and evaluate a suite of software tools that will allow all CTSA Program sites to integrate PROMIS tools directly into their EHRs. To achieve this, we will develop software to support tight integration into the two most common academic medical center EHRs — Epic and Cerner. We will develop a generalized integration of the PROMIS toolset, utilizing the SMART-on-FHIR standard, that can be implemented in multiple EHR platforms. Finally, we will implement and evaluate these software solutions across a number of diverse CTSA Program sites, both within and outside of the project team sites. Optimizing Translational Veterinary Trials to Advance Human Outcomes CTSA Program Hub: Ohio State University Caroline Whitacre, Ph.D., one of the world’s most acknowledged authorities on autoimmune disease, is among hundreds of scientists at The Ohio State University Medical Center who translate innovative research findings into personalized patient care. The movement of novel discoveries into clinical practice follows a relatively long and inefficient path. More than 50 percent of human clinical trials fail to meet critical endpoints, often late in the course of development, resulting in both financial loss and the lack of additional treatment options for affected patients. Although there are several reasons for these failures, the primary driver is typically lack of efficacy. It is now evident that while induced animal models provide substantial data regarding disease mechanisms, those models likely do not completely recapitulate the biology of a variety of spontaneous conditions that occur in people, so the impact of therapeutic intervention in many models overestimates potential clinical value in human patients. In contrast, there is increasing evidence that spontaneous diseases in veterinary patients represent unique models that more closely parallel the biology of comparable human diseases, including heterogeneity of clinical presentation and response and ensuing resistance to therapy. Importantly, spontaneous diseases in veterinary patients are an inherently less biased setting for therapeutic evaluation than those involving induced disease, in which all affected animals are free of variables and comorbidities that may substantially influence treatment outcome. Therefore, studies incorporating veterinary patients represent a unique opportunity to generate critical data regarding the safety and efficacy of novel drugs and devices that can serve to de-risk subsequent human clinical trials. To this end, the CTSA One Health Alliance (COHA), an affiliation of 11 veterinary academic centers, is working to facilitate therapeutic success in humans by incorporating large animal models of spontaneous disease into the pre- and post-IND process. COHA has established a primary mission of creating an organized network of veterinary academic centers that will conduct translational clinical trials in veterinary patients with spontaneous disease. To accomplish this goal, a set of standard operating practices and procedures for veterinary trials initially developed at the Ohio State University will be optimized and implemented across the network of 11 COHA institutions, ultimately creating a highly trained veterinary clinical trials consortium that operates under a single set of guidelines. This will be accomplished by (1) optimizing a set of operating practices for the performance of veterinary trials; (2) generating a standard veterinary GCP training module for certification of trials staff; (3) establishing REDCap as the primary mechanism for clinical trial management and reporting across the COHA consortium; (4) forming a data safety management board to oversee trials and facilitate institutional approval; and (5) developing a coordinated outreach effort to ensure adequate enrollment in studies. Successful completion of these goals will enable seamless initiation of veterinary trials over multiple sites, ensure a high level of training of all involved partners, and provide a single platform for data reporting, thereby establishing a well-organized, proficient nationwide network that will generate critical information to accurately inform human translational efforts. SPARC: The Strategic Pharma-Academic Research CTSA Program Hub: Indiana University Melissa Kacena, Ph.D., right, associate professor of orthopaedic surgery at the Indiana University School of Medicine, works with members of her team to advance research funded by NIH and the U.S. Department of Defense on a compound that could act as a potential new, more effective treatment for broken bones. The project’s early phase received critical support from the Indiana CTSI. The Strategic Pharma-Academic Research Consortium (SPARC) is an organization to spark innovative collaborations in discovery and “pre-competitive” target discovery stages between academic research institutions who are members of the NIH CTSA Program network and the biopharmaceutical industry. The consortium was established by four Midwestern CTSA Program hubs as founding partners in 2014, along with two large pharmaceutical companies located in the Midwest: Eli Lilly and Company and Takeda Pharmaceuticals. The consortium is housed in the Indiana Clinical and Translational Sciences Institute. SPARC is a platform for research that utilizes the unique strengths of academic and industry for basic discovery, target identification, and testing tool molecule chemical biology and translational research. The vision is to establish a public-private partnership for patient-focused discoveries that generate greater knowledge and better approaches to next-generation and targeted therapies. The intent is also to conduct patient data and samples toward understanding of human physiological traits of different diseases of interest. The current members include Indiana University, the Ohio State University, Northwestern University, University of Chicago, and Washington University in Saint Louis, along with the two major pharmaceutical companies, Eli Lilly and Takeda. In the first round of funding, the consortium focused on autoimmune diseases as the major theme for selecting projects. To date, the consortium has funded five projects involving multiple institutions and 12 faculty investigators and several industry scientists. Posted February 2017 | CTSA Program hubs are working to improve patient-reported outcome data, optimize translational veterinary trials, and spark innovative collaborations | /sites/default/files/ctsa_indiana.jpg | CTSA Program Collaboration in Action | CTSA Program hubs are working to improve patient-reported outcome data, optimize translational veterinary trials, and spark innovative collaborations | /sites/default/files/ctsa_indiana.jpg | CTSA Program Collaboration in Action | ||
| 8041 | CTSA Program Good Clinical Practice Standards Published | To improve the translational science process, clinical trial personnel must be qualified and have the necessary skills to implement safe and efficient studies. To help meet this goal, Clinical and Translational Science Awards (CTSA) Program hub investigators collaborated to establish ethical and scientific quality standards and competencies — called good clinical practice (GCP) — for the design, conduct, recording and reporting of clinical research involving human subjects. GCP is composed of three phases, which the investigators described in three recent papers in the Journal of Clinical and Translational Science. In the first phase, the investigators developed a consensus across the CTSA Program national network about training guidelines and standards in the implementation of clinical trials. The second phase focused on developing competencies and minimum qualifications for a training curriculum for researchers involved in clinical studies. During the third phase, the investigators established best practices for conducting social and behavioral research, with the goal of creating learning modules. Hub personnel are currently testing the effectiveness of the GCP training standards at their respective institutions. View the article published online on Jan. 13, 2017: Education and training of clinical and translational study investigators and research coordinators: A competency-based approach View the article published online on Jan. 13, 2017: Enhancing Clinical Research Professionals’ Training and Qualifications (ECRPTQ): Recommendations for Good Clinical Practice (GCP) training for investigators and study coordinators View the article published online on Feb. 6, 2017: Best practices in social and behavioral research: report from the Enhancing Clinical Research Professional’s Training and Qualifications project Posted February 2017 | CTSA Program investigators collaborated to establish ethical and scientific quality standards and competencies for clinical research involving human subjects. | /sites/default/files/ctsa-gcp_0.jpg | Good Clinical Practice Standards Published | CTSA Program investigators collaborated to establish ethical and scientific quality standards and competencies for clinical research involving human subjects. | /sites/default/files/ctsa-gcp_0.jpg | Good Clinical Practice Standards Published | ||
| 8040 | News Brief: CTSA Program Collaboration Provides Investigators with Access to High-Throughput Screening and Drug Discovery Expertise | Clinical and Translational Science Awards (CTSA) Program hub researchers at the University of New Mexico (UNM), University of Kansas Medical Center (KUMC), and University of North Carolina (UNC), Chapel Hill, are collaborating to more rapidly test existing drugs and advance those showing promise into clinical trials. The Drug Rescue, Repurposing and Repositioning Network (DR3N), led by UNM Principal Investigator Richard Larson, M.D., Ph.D., and component leader Larry Sklar, Ph.D., provides CTSA Program investigators with access to state-of-the-art technology, innovative tools, and guidance in translating pilot projects from preclinical to clinical stages via drug repurposing. Each DR3N hub offers unique drug-discovery expertise, resources and capabilities. UNM has drug libraries, high-throughput small-molecule screening and analytic capabilities; KUMC specializes in chemistry and formulation; and UNC offers a number of specialized receptor assays and libraries. DR3N launched last year, and several early-stage projects — both within the network institutions and with outside hubs — are already underway. Through this integrated, collaborative network, the investigators aim to accelerate drug discovery to bring more treatments to more patients more quickly. Posted February 2016 | test | test | ||||||
| 8030 | Audio | These audio clips feature funded scientists describing research goals as part of the Discovering New Therapeutic Uses for Existing Molecules program. Clip Description Preview The Efficacy and Safety of a Selective Estrogen Receptor Beta Agonist (LY500307) Alan Breier, M.D., Indiana University, Indianapolis For this project, the research team will test a drug candidate that mimics estrogen’s action in the body — but without its negative side effects — to see if it is safe and relieves symptoms in patients with schizophrenia. Runtime: 0:59 Date: June 2013 Right-click to download this clip (MP3 - 2MB) Clip courtesy of Indiana University. Your browser does not support the audio element. Right-click to download a transcript (1 KB) Medication Development of a Novel Therapeutic for Smoking Cessation Darlene H. Brunzell, Ph.D., Virginia Commonwealth University, Richmond For this project, the team of researchers will conduct studies in rodents followed by clinical trials with cigarette smokers to test an existing compound as a potential treatment for tobacco cessation. Runtime: 0:42 Date: June 2013 Right-click to download this clip (MP3 - 659KB) Clip courtesy of Virginia Commonwealth University. Your browser does not support the audio element. A Novel Compound for Alcoholism Treatment: A Translational Strategy Lorenzo Leggio, M.D., Ph.D., M.Sc. (NIAAA/NIDA), partner with the University of Rhode Island, Kingston For this project, the research team will test an oral medication that blocks a hormone called ghrelin to see if the drug is safe and if it reduces alcohol dependence. Runtime: 0:27 Date: June 2013 Right-click to download this clip(MP3 - 427KB) Clip courtesy of NIAAA/NIDA. Your browser does not support the audio element. Reuse of ZD4054 for Patients with Symptomatic Peripheral Artery Disease Brian H. Annex, M.D., University of Virginia, Charlottesville For this project, researchers will test an investigational drug for its ability to improve blood flow in the legs of patients with peripheral artery disease, which can cause severe leg pain. This drug may relieve this pain, help patients walk further and longer, and reduce the costs of hospitalization and treatment. Runtime: 0:44 Date: June 2013 Right-click to download this clip (MP3 - 2MB) Clip courtesy of the University of Virginia. Your browser does not support the audio element. Therapeutic Strategy for LAM N. Tony Eissa, M.D., Baylor College of Medicine, Houston For this project, the research team aims to determine if blocking Src kinase activity using an investigational drug is safe and can reduce the growth and spread of lymphangioleiomyomatosis (LAM), a progressive lung disease. Runtime: 0:43 Date: June 2013 Right-click to download this clip (MP3 - 2MB) Clip courtesy of Baylor College of Medicine. Your browser does not support the audio element. Therapeutic Strategy to Slow Progression of Calcific Aortic Valve Stenosis Jordan D. Miller, Ph.D., Mayo Clinic, Rochester, Minnesota For this project, the researchers will test the safety of an investigational drug in heart valve disease patients in slowing progression of stenosis, an abnormal narrowing of vessels or other structures. Runtime: 0:34 Date: June 2013 Right-click to download this clip (MP3 - 670KB) Clip courtesy of Mayo Clinic. Your browser does not support the audio element. Right-click to download a transcript (1KB) Translational Neuroscience Optimization of GlyT1 Inhibitor John H. Krystal, M.D., Yale University, New Haven, Connecticut For this project, the researchers will use brain imaging studies to determine the safest dose of an experimental drug in healthy subjects and, ultimately, whether it is effective in combination with mind exercises to treat cognitive deficits in schizophrenia patients. Runtime: 0:37 Date: June 2013 Right-click to download this clip (MP3 - 1MB) Clip courtesy of Yale University. Your browser does not support the audio element. Right-click to download a transcript (1KB) | Audio | Audio | ||||||
| 8029 | Genesis: Modern Chemical Library to Enable Large-Scale Deorphanization of Novel Biological Mechanisms | Contact: Early Translation Branch Library Size: 100K compounds Format: 1536-well plates in dose-response format Availability: Accessed through collaboration with NCATS Description: Genesis Chemical Library: NCATS has been assembling a high-value, modern chemical library that contains more than 100,000 compounds and will grow over the course of the next few years. The collection is plated in qHTS format, enabling large-scale deorphanization of unprecedented mechanisms. Bioactives identified from Genesis screening provide a high-quality starting point for medicinal chemistry optimization of proof-of-concept tool compounds or therapeutic leads. The library consists of more than 1,000 scaffolds that vary in representation from 20 to 100 compounds per chemotype. The modern nature of the library incorporates years of valuable lessons learned on chemical library design. A portion of the library includes many sp3-enriched chemotypes that are inspired by naturally occurring compounds. Such chemotypes retain the pharmacophore found in natural products while reducing the extreme complexity, making them synthetically tractable. The chemotype includes spirocyclic compounds as well as non-spiro novel chemotypes that either do not exist in literature or patent space or are scarcely found. This novelty provides a distinct advantage in the development of potential intellectual property, as well as providing opportunities for first-in-class compounds. Yet another feature of Genesis is that, given the need for rapid derivatization in medicinal chemistry, the library has been designed with core scaffolds that are purchasable commercially. This allows for optimization around what would otherwise be complex synthetic cores. Genesis’ compound space is largely non-overlapping with PubChem or any publicly available chemical library. Its overall library composition provides shape and electrostatic diversity while keeping known drug-like properties, such as solubility, lipophilicity, hydrogen bond donor/acceptor status, and molecular weight, in check. | Modern Chemical Library to Enable Large-Scale Deorphanization of Novel Biological Mechanisms | Genesis | Modern Chemical Library to Enable Large-Scale Deorphanization of Novel Biological Mechanisms | Genesis | ||||
| 8028 | Leadership Videos | Description Preview Christopher Austin speaks at Partnering for Cures NCATS Director Christopher P. Austin, M.D., speaks about teamwork in translation at Partnering for Cures, Nov. 28-30, 2012, in New York City. Runtime: 6:01 Date: November 2012 Right-click to download this video (MP4 - 67MB) Video courtesy of FasterCures. YouTube embed video: https://www.youtube-nocookie.com/embed/Ud9YnviTxcM Christopher Austin speaks at Partnering for Cures (with introductions) With introductions from NIH Director Francis S. Collins, M.D., Ph.D., and NCATS Advisory Council member Louis DeGennaro, Ph.D., NCATS Director Christopher P. Austin, M.D., speaks about teamwork in translation at Partnering for Cures, Nov. 28-30, 2012, in New York City. Runtime: 8:16 Date: November 2012 Right-click to download this video (MP4 - 96MB) Video courtesy of FasterCures. YouTube embed video: https://www.youtube.com/watch?v=zlx0nO-3DlQ | Leadership Videos | Leadership Videos | ||||||
| 8027 | Collaborate with NCATS Scientists | Biomedical Data Translator NCATS’ Biomedical Data Translator program aims to accelerate biomedical translation by developing a biomedical “data translator” for the research community. Through this program, NCATS will integrate multiple types of existing data sources and reveal potential relationships across the spectrum of data types. For more information, send an email to translator-questions@nih.gov. Bridging Interventional Development Gaps (BrIDGs) Through its BrIDGs program, NCATS assists researchers in advancing promising therapeutic agents through late-stage preclinical development toward an Investigational New Drug application and clinical testing. To discuss a potential collaboration with BrIDGs scientists, contact askTDB@nih.gov. Functional Genomics Lab The Functional Genomics Lab, administered by staff in NCATS’ Division of Preclinical Innovation, is designed to help NIH investigators use the latest functional genomics technology to advance drug discovery and scientific knowledge about health and disease. To learn more, contact Ken Cheng, Ph.D. Licensing, Technology Transfer & Patents NCATS’ strategic alliances team helps industry and academic researchers interact and partner with Center laboratories and scientists. The Center’s support for technology development and partnership includes day-to-day negotiations of transactional agreements and licenses between NCATS and outside parties, including universities, pharmaceutical companies and biotechnology companies. For more information, email NCATSPartnerships@mail.nih.gov. NCATS Early Translation Branch (ETB) NCATS’ ETB program staff offer biomedical researchers access to large-scale screening capacity and medicinal chemistry and informatics expertise to develop chemical probe molecules. These resources can help scientists study the functions of genes, cells and biochemical pathways. The program also features assay development and high-throughput screening, chemistry and chemistry technology, automation, and informatics. To learn more and to obtain ETB probe molecules, contact ETB. Pfizer's Centers for Therapeutic Innovation (CTI) for NIH Researchers NCATS is facilitating Pfizer's CTI program at NIH, which pairs NIH intramural researchers and clinicians with Pfizer resources to pursue scientific and medical advances through joint therapeutic development of biological compounds. For more information, contact NIH-PfizerCTI@mail.nih.gov. Stem Cell Translation Laboratory (SCTL) Through the SCTL, NCATS provides researchers across various disciplines and organizations with the ability to establish collaborations to advance the translation of regenerative medicine applications. NCATS seeks stem cell research collaborators from the biotechnology, pharmaceutical, and NIH intramural and extramural communities on an ongoing basis. Proposal receipt dates are July 1, November 1 and March 1. To learn more, contact Carlos A. Tristan, Ph.D. Therapeutics for Rare and Neglected Diseases (TRND) The TRND program provides collaborators with access to significant in-kind resources and expertise in the preclinical and early clinical development of new therapeutics for rare conditions and neglected tropical diseases. To discuss a potential collaboration with TRND scientists, contact askTDB@nih.gov to learn more. Toxicology in the 21st Century (Tox21) The goal of the Tox21 program is 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. Proposed assays must be compatible with the high-throughput screening guidelines described in the assay guidance criteria. To suggest an assay, submit a nomination form (PDF - 44KB) to Menghang Xia, Ph.D. Collaborate on Other Intramural Projects Connect with our intramural researchers to explore collaboration opportunities. To view existing project collaborations, visit the NIH Intramural Database and select “National Center for Advancing Translational Sciences (NCATS)” in the “Select Institute or Center” drop-down menu. | Learn more about the many ways to collaborate with NCATS scientists to advance translational research. | Collaborate with NCATS Scientists | Learn more about the many ways to collaborate with NCATS scientists to advance translational research. | Collaborate with NCATS Scientists | ||||
| 8026 | Features and Briefs | NCATS programs and initiatives are enabling scientists to transform the way research discoveries are turned into new tools, methods and treatments. A collection of these stories and other items of interest are featured below. View NCATS features by year: 2023 stories 2022 stories 2021 stories 2020 stories 2019 stories 2023 Better Method for Growing Stem Cells Could Speed Gene-Editing Therapies Scientists have published step-by-step instructions on how to use a four-part cocktail of drugs and compounds to grow stem cells from a single cell. NCATS Support: Division of Preclinical Innovation Rare Disease Day at NIH 2023: Putting Hope into Action Energy, excitement and hope guided Rare Disease Day at NIH 2023, which included inspiring stories from people with rare diseases, research updates, exhibits and poster sessions, and networking. NCATS Support: Rare Disease Research Rural Hospital Closures Fuel Rising Demand and Costs at Nearby Hospitals CTSA Program–supported research reveals that rural hospital closures nationwide put a strain on the financial and staffing resources of surrounding hospitals, which may further jeopardize access to health care in rural communities. NCATS Support: Clinical and Translational Science Awards Program Analysis Reveals Nationwide Spread of Three Historically Regional and Potentially Life-Threatening Fungal Infections CTSA Program–supported research found that three fungal infections spread outside their usual regions and now appear in at least half of the United States. These findings could speed recognition and treatment outside typical areas of infection. NCATS Support: Clinical and Translational Science Awards Program 2022 Efficient Registry Approach Powers Practice-Shaping Infant Heart Surgery Trial A clinical trial of 1,200 infants undergoing congenital heart disease surgery found that an anti-inflammatory steroid did not perform better than a placebo at reducing risk of severe outcomes, except for some subgroups of infants. NCATS Support: Clinical and Translational Science Awards Program COVID-19 Hospitalization May Increase Risk of Heart Failure A large, retrospective study using National COVID Cohort Collaborative (N3C) data revealed that people who had been hospitalized with COVID-19 were more likely to develop heart failure during their recovery than people who had been hospitalized without COVID-19. NCATS Support: Clinical and Translational Science Awards Program Previous Common Colds Could Boost Risks of More Severe COVID-19 Having a common cold caused by seasonal coronaviruses may cause immune distraction, making some people’s immune systems respond less effectively to SARS-CoV-2 infection and increase their risk of more severe COVID-19. NCATS Support: Clinical and Translational Science Awards Program N3C Data Reveal More Severe COVID-19 Outcomes in Rural Communities After examining patient health records from the National COVID Cohort Collaborative (N3C), researchers found that people with COVID-19 who live in rural areas are more likely to be hospitalized than those who live in urban areas. NCATS Support: Clinical and Translational Science Awards Program Nationwide IRB Reliance Agreement Aimed at Speeding Research Reaches 1,000 Signatories The Streamlined, Multisite, Accelerated Resources for Trials (SMART) Institutional Review Board (IRB) agreement has reached 1,000 participating sites, making it one of the largest medical research study reliance agreements in the United States. NCATS Support: Clinical and Translational Science Awards Program Study Finds Immune Dysfunction Is a Significant Risk Factor for COVID-19 Breakthrough Infection The National COVID Cohort Collaborative Data Enclave helps researchers identify a link between COVID-19 breakthrough infections and people with abnormal or impaired immune systems. NCATS Support: Clinical and Translational Science Awards Program Researchers Use Cutting-Edge EHR Data Resource to Find Risk Factors for Severe COVID-19 in Children Researchers used data from NCATS’ National COVID Cohort Collaborative Data Enclave to discover why some children with COVID-19 developed a dangerous condition called multisystem inflammatory syndrome in children, also known as MIS‑C. NCATS Support: Clinical and Translational Science Awards Program Rare Disease Day at NIH 2022: Shining a Light on Patient Perspectives For the second year, the annual Rare Disease Day at NIH 2022 event was held virtually due to the pandemic, but that didn’t stop patients and researchers from coming together to share their stories and highlight research efforts. NCATS Support: Rare Diseases Research Robots Could Spur Use of Stem Cells in the Laboratory and Clinic NCATS scientists used robots to automate the culturing of stem cells, thus speeding up the process and bringing stem cells one step closer to clinical use. NCATS Support: Division of Preclinical Innovation International Registry Reveals Risks COVID-19 Poses with Sickle Cell Disease NCATS-funded CTSA Program researchers developed a collaborative registry that collects data on COVID-19 illness in people with sickle cell disease. NCATS Support: Clinical and Translational Science Awards Program 2021 Drug Repurposing Approaches Reveal HIV Drug Holds Promise Against a Rare, Disabling Muscle Disorder Using drug repurposing approaches, researchers from NCATS, Duke University and the University of Florida discovered that an existing antiviral drug used against HIV infection may be helpful in treating a form of dystonia. NCATS Support: Division of Preclinical Innovation 10,000 Compounds and Counting: Scientists Create Extensive Chemical Library to Study Toxic Substances, Share Data NCATS scientists and collaborators created the Tox21 10K chemical library, a set of approximately 10,000 compounds and FDA-approved drugs, to help researchers rapidly conduct toxicity testing. NCATS Support: Toxicology in the 21st Century (Tox21) Lung Tissue Chips Could Help Predict Future Infectious Influenza Disease Variants NCATS-funded researchers at the Wyss Institute for Biologically Inspired Engineering at Harvard University used lung tissue chips to replicate how influenza viruses evolve, potentially sharpening predictions about future infectious influenza disease variants. NCATS Support: Tissue Chips for Drug Screening Genetic Analysis Suggests Dilated Cardiomyopathy Therapies May Work for Rare Peripartum Cardiomyopathy Analyzing genetic information drawn from patients and multiple databases, CTSA Program researchers discovered similarities between nonischemic dilated cardiomyopathy (DCM) and peripartum cardiomyopathy (PPCM). NCATS Support: Clinical and Translational Science Awards Program National EHR Data Resource Reveals COVID-19’s Stark Mortality Risk in People with COPD Northwestern University researchers using N3C data found that individuals with COPD were more likely to be hospitalized and die of COVID-19 than those without COPD. NCATS Support: Clinical and Translational Science Awards Program NCATS Researchers’ New Approach to Measure Gene Activity Could Speed Search for Rare Disease Therapies NCATS scientists design an innovative test to find potential treatments for Charcot-Marie-Tooth disease (CMT), one of the most common inherited neurological diseases, opening new pathways for rare diseases research. NCATS Support: Assay Development and Screening Technology Low Vitamin D Levels May Boost COVID-19 Risk in Black People CTSA Program researchers find a link between low vitamin D levels and a higher risk of testing positive for COVID-19 in Black people. NCATS Support: Clinical and Translational Science Awards Program Existing Drugs That Safeguard Endoplasmic Reticulum Could Point to Therapies for Many Diseases NIH researchers identified five U.S. Food and Drug Administration–approved drugs that help maintain healthy calcium levels in the endoplasmic reticulum and prevent proteins from fleeing, which could point to therapies for many diseases. NCATS Support: Division of Preclinical Innovation CTSA Program-Supported Researchers May Turn Brown Fat into an Ally Against Obesity CTSA Program researchers discover how brown fat may help reduce the risk of heart disease, type 2 diabetes and other cardiometabolic diseases, particularly in people who are obese. NCATS Support: Clinical and Translational Science Awards Program 2021 Rare Disease Day at NIH: Finding Hope Within Uncertainty The COVID-19 pandemic has profoundly affected nearly every aspect of the rare disease world. The greatest impacts of the pandemic, however, have been felt by the patients themselves and their families. NCATS Support: Rare Diseases Research Automated Retinal Screening Opens a Window into People’s Health Eyenuk, Inc., received NCATS small business funding to develop EyeMarkTM and support from the NCATS Commercialization Readiness Pilot program to conduct clinical trials needed for regulatory approval of EyeMark in the United States. NCATS Support: Small Business Innovation Research Building Lung Tissue Models to Speed Virus Drug Testing NCATS scientists are developing 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, potentially speeding up the testing of new treatments. NCATS Support: 3-D Tissue Bioprinting Improving Manufacturing Techniques to Deliver High Quality Biotherapeutics to the Clinic Faster Intabio, Inc., used funding from NCATS’ Small Business Innovation Research program to create an automated instrument that streamlines the production of cellular and gene-based medicines. NCATS Support: Small Business Innovation Research Tissue Model Approach Provides Clues to SARS-CoV-2 Brain Infections A team of researchers used organoids, tiny 3-D tissue models of human brain development, to better understand brain infections caused by SARS-CoV-2. NCATS Support: Division of Preclinical Innovation 2020 Tissue Chip Trio to Orbit Earth for Answers to Osteoarthritis and Muscle Diseases Three tissue chip experiments exploring osteoarthritis, muscle wasting and heart tissue damage will launch into space for the Tissue Chips in Space initiative. NCATS Support: Tissue Chips in Space NCATS Patent Book Showcases Opportunities for Collaborations and Licensing NCATS’ online book, Innovation at NCATS, describes 22 patents issued to NCATS and its collaborators between October 2018 and July 2020. NCATS seeks commercial partners interested in licensing and using the patents to accelerate biomedical research. NCATS Support: Office of Strategic Alliances, Division of Preclinical Innovation Target Watch Sheds Light on Overlooked Proteins The Illuminating the Druggable Genome program, supported by the NIH Common Fund and administered through NCATS and the National Institute of Diabetes and Digestive and Kidney Diseases, has partnered with Nature Reviews Drug Discovery to create Target Watch, which highlights understudied protein targets. NCATS Support: Illuminating the Druggable Genome Teaching Children About Translational Science and Clinical Trials University at Buffalo’s CTSA Program researchers created “Sofia Learns About Research,” a coloring and activity book that introduces children and their guardians to translational science and teaches them about the importance of clinical trials. NCATS Support: Clinical and Translational Science Awards Program High-Throughput Screening Helps Advance Melanoma Therapy Finding the right compounds for new uses in treating a disease or improving a therapeutic approach can be time consuming. To speed up the process, NCATS scientists used high-throughput screening to help advance a therapy for melanoma. NCATS Support: Early Translation Branch The Beat Goes On — Even 258 Miles Above Earth By using tissue chips to model cardiac dysfunction in space, researchers can advance heart disease research on Earth. NCATS Support: Tissue Chips in Space Rare Disease Day at NIH Fosters Connections Across the Community The annual event — held this year on Feb. 28, 2020 — focused on forging connections in the rare diseases community, shortening the journey toward diagnosis and pursuing personalized medicine as a therapeutic approach for treating rare diseases. NCATS Support: Rare Diseases Research Easing the Way to Diagnosing Alzheimer’s and Rare Syndromes Contributions from the NCATS-led Rare Diseases Clinical Research Network helped develop a new blood test that may make it easier to diagnose Alzheimer’s disease and rare neurodegenerative syndromes. NCATS Support: Rare Diseases Clinical Research Network A Multipronged Approach Toward Treating DMD Experts in high-throughput screening and lysosomal biology collaborate on a multipronged approach to lessen the symptoms of Duchenne muscular dystrophy. NCATS Support: Division of Preclinical Innovation, Early Translation Branch, Rare Diseases Research Making Daily Pills a Thing of the Past to Improve Medication Adherence About half of all patients in the developed world don’t take medicines as prescribed. A star-shaped capsule manufactured with support from NCATS’ Small Business Innovation Research program could help keep people from missing a dose. NCATS Support: Small Business Innovation Research Grants Help Move Promising Rare Disease Interventions to Clinical Trials To close the gap between development of candidate interventions for rare diseases and clinical testing, NCATS and NICHD created rare-disease Clinical Trial Readiness grants. Funds support projects that collect data needed to move on to clinical trials. NCATS Support: Rare Diseases Clinical Research Network Harnessing the CTSA Program to Advance Telehealth CTSA Program-supported researchers are harnessing their resources and expertise to evaluate access to high-quality telehealth care for rural and underserved children. NCATS Support: Clinical and Translational Science Awards Program 2019 View Slideshow of NCATS’ 2019 Successes and Activities Collaboration is integral to our approach for breaking down barriers in the translational science process, as illustrated in these highlights from 2019. NCATS-Supported Consortium Plays Pivotal Role in New Porphyria Drug Years of NIH-supported studies contributed to the U.S. Food and Drug Administration approval of a new drug for acute intermittent porphyria—a rare, debilitating condition. The NCATS-supported Porphyrias Consortium played a pivotal role in its development. NCATS Support: Rare Diseases Clinical Research Network Small Business Awards Propel "One-of-a-Kind" Technology A new tissue chip technology that can predict the effects of drugs on the human nervous system is the latest discovery advanced by a small business innovation research award. Nerve-on-a-Chip® is considered a first-of-its-kind tissue chip technology. NCATS Support: Small Business Innovation Research, Tissue Chip for Drug Screening Ketogenic Diet Leads to Cognitive Improvement in Patients with Mild Alzheimer’s Disease CTSA Program-supported researchers tested the hypothesis that ketones could provide a new source of energy for the brain in people with Alzheimer’s disease (AD). The study found improved brain function in people with mild AD after a three-month ketogenic diet. NCATS Support: Clinical and Translational Science Awards Program Following a Translational Science Path NCATS supports several training and career development programs that not only inspire students to become scientists but also help grow the translational science workforce. Read about one student’s journey to becoming a translational scientist. NCATS Support: Translational Science Education & Training; Early Translation Branch NCATS BioPlanet: A Resource for Discovery Researchers can better study how compounds and drugs affect cells with an NCATS resource that combines all pathways in human cells into one database. BioPlanet lets users easily browse, retrieve and analyze the pathways and explore pathway connections. NCATS Support: Division of Preclinical Innovation - Early Translation Branch and Toxicology in the 21st Century (Tox21) Research Speeds Diagnosis of Genetic Diseases in Seriously Ill Children NCATS-supported researchers have developed an automated approach to diagnosis of genetic diseases in seriously ill children to allow faster diagnosis and initiation of treatment, and, ultimately, better outcomes. The study appeared in the April 24, 2019, issue of Science Translational Medicine. NCATS Support: Clinical and Translational Science Awards Program An Itch to Scratch: Scientists Identify a Potential New Approach to a Chronic Problem People with constantly itchy skin, take heart. NCATS and National Institute of Dental and Craniofacial Research scientists report that blocking a receptor on the surface of spinal cord neurons may help relieve the chronic need to scratch. The study appeared online on July 10 in Science Translational Medicine. NCATS Support: Assay Development and Screening Technology CTSA Program Researchers Aim to Improve Health Care from All Sides A research framework enables the recruitment of hospitalized patients for a wide range of studies to identify optimal care approaches during and after hospitalization. Undergraduate students play an integral role in the research as they recruit patients, conduct follow-up activities, and learn about clinical research. NCATS Support: Clinical and Translational Science Awards Program NCATS Innovation Continues Through Latest HHS Ignite Team Selections Three NCATS teams selected through the latest round of HHS’ Ignite Accelerator IDEA incubator program will begin projects to emphasize happiness as an essential component of health, establish a system to track and coordinate clinical and translational science best practices, and optimize NCATS’ Genetic and Rare Diseases (GARD) Information Center. Teams will receive support and training to develop their innovations. NCATS Support: Division of Clinical Innovation and Office of Rare Diseases Research CTSA Program Support Enables Development of Life-saving Blood Loss Monitor NCATS-supported researchers have developed an innovative device that detects internal bleeding and monitors a patient’s response to blood loss. The monitor guides appropriate treatment before a patient goes into life-threatening shock. The device was approved by the U.S. Food and Drug Administration in 2018 and is in clinical use today. NCATS Support: Clinical and Translational Science Awards Program New Method Could Help Find Potential Treatments for Rare Diseases Scientists in NCATS’ Assay Development and Screening Technology Program are developing new methods for screening small molecules that could help researchers test possible drugs and develop new treatments for many diseases. NCATS Support: Assay Guidance Manual Working Together Is the Name of the Game at Rare Disease Day at NIH Rare Disease Day at NIH, held on Feb. 28 in 2019, exemplifies NCATS’ approach to rare diseases research: involving patients as partners throughout the translational research process to ensure that research and interventions address patients’ needs. NCATS Support: Rare Diseases Research NCATS Assay Recognized in Top Ten List of Innovative Scientific Achievements NCATS developed a screening test to identify molecules that block the process of growing new blood vessels. With the test, NCATS scientists found both known and potential new anticancer drugs. The work earned a spot on the Society for Laboratory Automation and Screening’s top 10 innovative scientific achievements list for 2018. NCATS Support: Toxicology in the 21st Century (Tox21) Brigham and Women’s Hospital and NCATS Scientists Dial Up an Approach Against Out-of-Control Inflammation Scientists at Brigham and Women’s Hospital collaborated with NCATS high-throughput screening experts to investigate a different approach against chronic inflammation. The team identified four compounds, including two novel synthetic molecules. This finding provides a potential opportunity to develop a new kind of drug to reduce inflammation. NCATS Support: Division of Preclinical Innovation; Early Translation Branch Opioids Increase the Risk of Pneumonia NIH-supported research suggests a new danger associated with opioids: They can increase a person’s risk for pneumonia that is severe enough to warrant hospitalization. NCATS Support: Clinical and Translational Science Awards Program Researchers Convene to Take Stock and Take Action in the Opioid Crisis In February, NCATS co-hosted a two-day symposium for pain and addiction researchers as part of The NIH Helping to End Addiction Long-termSM Initiative, or NIH HEAL InitiativeSM. The event highlighted key challenges and innovative approaches to finding better treatments for addiction and pain. NCATS Support: NIH HEAL Initiative, Division of Preclinical Innovation, Assay Guidance Manual NCATS Spearheads a New Resource for Natural Products Canvass, a screening library for natural products, was created by NCATS in collaboration with academic and industry researchers to uncover the potential medical use of these compounds and enable the creation of new treatments for diseases. The researchers have already found some compounds with unexpected biological activities. NCATS Support: Division of Preclinical Innovation CTSA Program Supports Emerging Research on Health Effects of Plastics An early-career investigator has developed techniques to study how chemicals used in medical devices affect the still-developing hearts of pediatric patients. NCATS Support: Clinical and Translational Science Awards Program NCATS Releases Updated Translational Road Maps An expanded, interactive beta version of the Drug Discovery, Development and Deployment Maps provides a more dynamic map interface for enhanced usability compared with the static version. Users can zoom into and out of areas of the process to view different levels of complexity, find information and best practices, and connect to relevant NCATS programs and resources. NCATS invites the public to explore the map and provide feedback on the current version. NCATS Support: Office of the Director NCATS-Supported Researchers Find Cell Source Matters for Tissue Chips Researchers from the Tissue Chip Testing Center at Texas A&M University have evaluated a kidney-on-a-chip developed by researchers at the University of Washington and Nortis Bio. The kidney chip was able to perform the same functions as real kidneys, helping to validate this innovative research model. NCATS Support: Tissue Chip for Drug Screening NCATS-Supported Researchers Find Exercise May Help Protect DNA Researchers supported by NCATS’ Clinical and Translational Science Awards (CTSA) Program who studied older caregivers found that those who exercised had longer telomeres (the caps that protect the ends of DNA). These findings may lead to better health outcomes for older adults as they age. NCATS Support: Clinical and Translational Science Awards Program | Features and Briefs | Features and Briefs |
Last updated on