| ID | Title | Body | Facebook Description | Facebook image | Facebook Title | Facebook Video | Twitter Description | Twitter Image | Twitter Title | Meta tags |
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| 26385 | 2022 CCIA Projects | Video Repositories for Clinical Research: Overcoming Barriers and Testing Utility Analytics & Machine-learning for Maternal-health Interventions (AMMI): A Cross-CTSA Collaboration PANDA-MSD: Predictive Analytics via Networked Distributed Algorithms for Multi-System Diseases Perioperative Precision Medicine: Translating Science to Clinical Practice to Improve Safety and Efficacy of Opioids in Neonates, Children and Nursing Mothers Video Repositories for Clinical Research: Overcoming Barriers and Testing Utility Virginia Commonwealth University Principal Investigator: Henry Rozycki, M.D. Grant Number: 1R21TR003994-01A1 Collaborating Institution: University of Pennsylvania Patient observation is key to provider assessment. In addition, observations are often included in clinical scoring systems, such as the Glasgow Coma Scale and the Apgar Score, that are used to classify patients and as surrogate outcomes in research — signs used in place of other measures to tell whether a treatment works. Most observation data, however, have significant problems with inter-rater reliability — the extent to which two or more people assessing information agree — which makes them less robust than objective, measurable data, such as blood test values. This unreliability is due in part to using limited, nontypical subject samples during their creation and validation. Using large data sets from a variety of sources is the recommended way to help overcome this challenge. This has led to the creation of data registries and biorepositories. To date, however, there are almost no examples of repositories that focus on observations made by video recording. Although the overall framework for the design and management of other registries and repositories is well established, concerns about privacy, security and liability risk have prevented the creation of video repositories. The primary objective of this study is to design solutions to these concerns and produce a guideline or manual for the creation of clinical video repositories. Learn more about this project in the NIH RePORTER. Analytics & Machine-learning for Maternal-health Interventions (AMMI): A Cross-CTSA Collaboration The University of North Carolina at Chapel Hill Principal Investigator: Javed Mostafa, Ph.D. Grant Number: 1U01TR003629-01A1 Collaborating Institutions: Duke University and Wake Forest University Health Sciences African American women across the United States experience alarmingly higher rates of maternal mortality than their white counterparts. Social determinants of health — such as education, housing, transportation and nutrition — might contribute to this disparity in maternal health outcomes, along with clinical risk factors, including high blood pressure and heart disease. However, the complex connections among these factors, along with the role they play in increasing the risk of maternal mortality, are not well understood. Further, a need exists for comprehensive health care interventions that take these combined factors into account to provide decision and communication support for patients, providers and community support workers. The Analytics and Machine-learning for Maternal-health Interventions (AMMI) initiative — a partnership among researchers at The University of North Carolina at Chapel Hill, Duke University, and Wake Forest University — aims to address these gaps by developing a machine learning–enhanced health technology framework to reduce the risk of maternal mortality in African American women. Learn more about this project in the NIH RePORTER. Note: This U01 was co-funded by NCATS and the NIH Office of Disease Prevention. PANDA-MSD: Predictive Analytics via Networked Distributed Algorithms for Multi-System Diseases University of Pennsylvania Principal Investigator: Yong Chen, Ph.D. Grant Number: 1U01TR003709-01A1 Collaborating Institutions: The University of Alabama at Birmingham; University of Florida; Vanderbilt University Medical Center; Global Healthy Living Foundation; Vasculitis Foundation Researchers will develop novel ways to combine data from different sources using electronic health records from multiple CTSA hubs to create predictive models of multisystem diseases — disorders that affect multiple body systems. The project directly addresses the areas of emphasis in PAR-19-099 to “engage new collaborators in pre-existing collaborations to solve a translational science problem no one hub can solve alone.” The researchers will develop the Predictive Analytics via Networked Distributed Algorithms (PANDA) framework, which will improve risk prediction to help health care providers reach accurate diagnoses earlier. The researchers’ proposed methods directly address two major barriers: (1) lack of predictive models for multisystem conditions, and (2) lack of algorithms that effectively combine data from multiple sites in a way that preserves privacy and makes communication more efficient. Learn more about this project in the NIH RePORTER. Perioperative Precision Medicine: Translating Science to Clinical Practice to Improve Safety and Efficacy of Opioids in Neonates, Children and Nursing Mothers University of Pittsburgh Principal Investigator: Senthilkumar Sadhasivam, M.D., M.P.H. Grant Number: 1U01TR003719-01A1 Collaborating Institutions: Johns Hopkins University; University of California, San Francisco; Indiana University; Washington University Severe surgical pain is still poorly managed, yet clinicians must also avoid unpredictable and life-threatening opioid adverse effects, as well as long-term opioid use and misuse. Opioid adverse effects that occur around the time of surgery (perioperative) — from postsurgical nausea and vomiting to respiratory depression and death — are preventable challenges in managing surgical pain. This study aims to develop a proactive clinical practice to optimize postsurgical pain control and decrease opioid-related adverse effects. Opioid metabolism and opioids’ pain-relieving (analgesic) and adverse effects are influenced by genetic variations. Due to translational bottlenecks, lack of infrastructure and knowledge gaps in how to personalize opioid use and dose precisely for the best results, presurgical genotyping and personalized analgesia are not practiced, despite evidence, regulatory warnings, Clinical Pharmacogenetics Implementation Consortium guidelines, cost effectiveness and insurance coverage for CYP2D6 testing. Personalizing analgesia based on genetic risks will reduce opioid use and adverse effects and accelerate value-based care opportunities, particularly in children and nursing mothers. However, these opportunities are constrained by a lack of translational platforms and major gaps in our understanding of how to personalize and precisely dose opioids. This collaborative CTSA project aims to develop an innovative perioperative precision analgesia platform to reduce serious adverse outcomes of opioids and improve the safety of opioids in (1) children undergoing painful surgery and (2) nursing mothers and their infants. Learn more about this project in the NIH RePORTER. | Listing of 2022 CCIA Projects | 2022 CCIA Projects | Listing of 2022 CCIA Projects | 2022 CCIA Projects | ||||
| 26015 | Bias Detection Tools in Health Care Challenge | × The Challenge closed on March 1, 2023. The Minimizing Bias and Maximizing Long-Term Accuracy, Utility and Generalizability of Predictive Algorithms in Health Care Challenge seeks to encourage the development of bias-detection and -correction tools that foster “good algorithmic practice” and mitigate the risk of unwitting bias in clinical decision support algorithms. Key Dates Background Challenge Goals Contact Key Dates Note: Dates subject to change as necessary Challenge Announcement: September 1, 2022 Registration and Submission Portal Opens: October 31, 2022 Submission Deadline: March 1, 2023 Technical Evaluation Phase: March 2023 Federal Judging Phase: March and April 2023 Winners Announced: April 2023 Demo Day: May 5, 2023 Background Although artificial intelligence (AI) and machine learning (ML) algorithms offer promise for clinical decision support (CDS), their potential has yet to be fully realized. Even well-designed AI/ML algorithms and models can become inaccurate or unreliable over time due to various factors, including changes in data distribution; subtle shifts in the data, real-world interactions and user behavior; and shifts in data capture and management practices. Over time, these changes and shifts can degrade the predictive capabilities of algorithms, which can negate the benefits of these types of systems for clinics. How do we detect these shifts or changes on a continual basis to maintain prediction quality? Monitoring an algorithm’s behavior and flagging any significant changes in performance may enable timely adjustments that ensure a model’s predictions remain accurate, fair and unbiased over time. This approach maintains the predictive capability of an algorithm in the real world. As AI/ML algorithms are increasingly used in health care systems, accuracy, generalizability and avoidance of bias and drift become more important. Bias primarily surfaces in two forms. Predictive bias is seen in algorithmic inaccuracies that produce estimates that significantly differ from the underlying truth. Social bias reflects systemic inequities in care delivery leading to suboptimal health outcomes for certain populations. To address these issues and improve clinician and patient trust in AI/ML-based CDS tools, this Challenge invites groups to develop bias-detection and -correction tools that foster “good algorithmic practice” and mitigate the risk of unwitting bias in CDS algorithms. Challenge Goals The goal of this Challenge is to identify and minimize inadvertent amplification and perpetuation of systemic biases in AI/ML algorithms used as CDS through the development of predictive and social bias-detection and -correction tools. For this Challenge, participants across academia and the private sector are invited to participate in teams, as representatives of an academic or private entity, or in an individual capacity to design a bias-detection and -correction tool. For most up-to-date information about the rules, submission requirements, judging criteria, prizes, how to enter and to register for the Challenge, please visit the ExpeditionHacks site. You also can visit the Challenge.gov site. Contact Have feedback or questions about this challenge? Please send your feedback or question to NCATSAIBiasChallenge@mail.nih.gov. Back to Top | This challenge aims to improve trust in artificial intelligence and machine learning through bias detection and mitigation. | /sites/default/files/AI_Challenge_promo_900x600.png | Bias Detection Tools in Health Care Challenge | This challenge aims to improve trust in artificial intelligence and machine learning through bias detection and mitigation. | /sites/default/files/AI_Challenge_promo_900x600_0.png | Bias Detection Tools in Health Care Challenge | ||
| 25979 | 2020 NCATS ASPIRE Reduction-to-Practice Challenge Winners, Stage 2, Milestone 2* | NCATS is happy to announce the winners of the 2020 NCATS ASPIRE Reduction-to-Practice Challenge, Stage 2, Milestone 2: Development of a Comprehensive Integrated Platform for Translational Innovation in Pain Opioid Use Disorder and Overdose. *According to the rules for these ASPIRE Design Challenges, non-U.S. citizens and non-permanent residents were not eligible to win a monetary prize (in whole or in part). Iterative Learning and Automated Modular Platform for Optimum Nonaddictive Analgesic Discovery Closed Loop Bio Assay-Chemputer for Next Generation Analgesics (BioChemputer) Development of a Comprehensive Integrated Platform for Translational Innovation in Pain, Opioid Abuse Disorder and Overdose Iterative Learning and Automated Modular Platform for Optimum Nonaddictive Analgesic Discovery Gaurav Chopra Ram Samudrala R. Graham Cooks Connor W. Coley Panayotis K. Thanos Jun-Xu Li Purdue University State University of New York (SUNY) Purdue University Massachusetts Institute of Technology University at Buffalo University at Buffalo Closed Loop Bio Assay-Chemputer for Next Generation Analgesics (BioChemputer) Babak Esmaeli-Azad Leroy Cronin Sara I. Walker Philip J. Kitson S. Hessam M. Mehr James Zapf Evan Snyder Larry H. Ellisman Shaochen Chen CiBots, Inc. University of Glasgow Arizona State University University of Glasgow University of Glasgow Visionary Pharmaceuticals Inc. Sanford Consortium for Regenerative Medicine University of California, San Diego University of California, San Diego Development of a Comprehensive Integrated Platform for Translational Innovation in Pain, Opioid Abuse Disorder and Overdose Jeffrey Skolnick Andre Ghetti Nicole Jung Hongyi Zhou Georgia Institute of Technology ANABIOS Corporation Karlsruhe Institute of Technology Georgia Institute of Technology Back to top Contact Dobrila D. Rudnicki, Ph.D. | NCATS announced the winners of the ASPIRE Reduction-to-Practice Challenge, Stage 2, Milestone 2. | ASPIRE Reduction-to-Practice Challenge Winners, Stage 2, Milestone 2 | NCATS announced the winners of the ASPIRE Reduction-to-Practice Challenge, Stage 2, Milestone 2. | ASPIRE Reduction-to-Practice Challenge Winners, Stage 2, Milestone 2 | ||||
| 26204 | NCATS Fact Sheets | .shadow { width: 90%; filter: drop-shadow(-5px 5px 10px #0e0e0e); } h2 { font-size: 22px; } h3 { color: #006478; margin-top: 10px; } .grid-row { padding: .9em; margin: 5px 0; } .grid-col-2 { padding-right: 20px; } .grid-row:nth-child(odd) { background-color: #f7f7f7; } .grid-row:nth-child(even) { background-color: #e7e7e7; } /* .grid-row:nth-child(1) { background-color: #662e6b; color: #fff; margin-top: 0; padding: 0; } */ .grid-row:nth-child(1) div h2 { margin-top: 10px; } Please click on the fact sheets below to learn about key NCATS activities.Resources for ResearchersResources for the PublicResources for Researchers Assay Guidance ManualThe Assay Guidance Manual is a free, best-practices online resource devoted to the successful development of robust, early-stage drug discovery assays.Chemical Genomics BranchThe goal of the NCATS Chemical Genomics Branch is to explore new technological and operational paradigms, ranging from new strategies for drug discovery and testing to improving the use of stem cells in research.Clinical and Translational Science Awards (CTSA) ProgramThe CTSA Program supports a national network of medical research institutions that work together to improve the translational research process to get more treatments to all people more quickly.Core TechnologiesNCATS uses state-of-the-art resources and core technologies to enable the ongoing operation of all translational research activities happening at the Center.Early Translation BranchThe goal of the Early Translational Branch is to uncover new small molecule therapeutics and advance the process of therapeutic development through a model of collaborative research.National COVID Cohort CollaborativeThe National COVID Cohort Collaborative (N3C) maintains one of the largest collections of secure and deidentified clinical data in the United States for COVID-19 research.NCATS OpenData PortalThe OpenData Portal offers real-time information about how individual SARS-CoV-2 variants may respond to known therapeutics. It also focuses on NCATS’ broader drug repurposing findings and other pandemic threats.Research Supplements to Promote Diversity and Re-entryThe CTSA Program diversity and re-entry research supplements opportunities promote diversity in health-related research and re-entry into biomedical and behavioral research careers.Small Business InnovationThe Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) programs engage U.S. small businesses and research organizations in research and development that has the potential for commercialization and public benefit.Therapeutic Development BranchThe goal of the Therapeutic Development Branch is to help close the gap between basic research and preclinical testing of drugs as well as cell-based and gene therapies.Resources for the PublicAbout NCATSNCATS is speeding the translation of scientific discoveries into health solutions. The Center supports a range of initiatives to achieve a future that offers more treatments for all people more quickly.Community EngagementCTSA Program hubs work hand in hand with community leaders to build strong relationships, understand community needs and improve community health.Congressional JustificationThe budget for NCATS is based on the fiscal year appropriation provided by Congress and the U.S. President. The Congressional Justification provides Congress detailed estimates and justifications for research.Economic Burden of Rare DiseasesA study led by NCATS estimates the direct medical costs of rare diseases and the lengthy journey some people with rare diseases travel to an accurate diagnosis.Rare DiseasesNCATS supports a range of innovative approaches for understanding and treating rare diseases, which affect about 30 million people in this country.Copyright and Reuse of GraphicsThe NCATS website uses a mix of copyrighted and copyright-free graphics (including illustrations and photos). If you want to reuse a graphic, please follow these guidelines:Copyrighted graphics will usually be credited to individuals or organizations. Permission to reuse these must be negotiated directly with the creators, and not NCATS.Graphics explicitly credited to NCATS are copyright-free and may be used without our permission. Please credit the National Center for Advancing Translational Sciences as the source.If you are not sure who created a graphic or have other questions about reusing a graphic on the NCATS website, email NCATS at info@ncats.nih.gov. Please include the URL and file name in your email. | NCATS’ Fact Sheets | NCATS’ Fact Sheets | ||||||
| 25598 | Director’s Message Series: NCATS’ Bold Goals | Our 10th-anniversary celebration highlighted the many accomplishments of NCATS’ scientists, grantees and collaborators across the translational science spectrum. In a series of Director's Corner messages, NCATS Director Joni L. Rutter, Ph.D., shares her vision and audacious goals for the Center over the next decade. VisionThe Decade Ahead: More Treatments for All People More QuicklyDr. Rutter shares her vision of a future with more treatments for all people more quickly. Achieving this vision, she notes, will require bold ideas, impactful programs and new partnerships.More TreatmentsA Decade to Deliver Five Times More TreatmentsIn this message, Dr. Rutter highlights three approaches NCATS is pursuing to significantly increase the number of treatments in the drug development pipeline: developing more predictive tools, targeting what’s common across diseases and honing data-driven solutions.All PeopleIncrease Inclusivity to Improve Health for AllDr. Rutter shares activities underway at NCATS to dramatically increase inclusivity across the research pipeline and in the research workforce. They alone, she acknowledges, will not achieve the goal of improving health for all, but they are steps in the right direction.More QuicklyNew Treatments Twice as FastIn the final installment of the series, Dr. Rutter describes how leveraging big data and streamlining processes can speed the development and delivery of new treatments.Learn more about:NCATS’ DirectorNCATS' Research ActivitiesNews and Events | This series of Director’s Messages outlines NCATS’ vision of more treatments for all people more quickly. | /sites/default/files/Joni-Rutter_900x1000px.jpeg | Director’s Message Series: NCATS’ Bold Goals | This series of Director’s Messages outlines NCATS’ vision of more treatments for all people more quickly. | /sites/default/files/Joni-Rutter_900x1000px.jpeg | Director’s Message Series: NCATS’ Bold Goals | ||
| 25553 | Mount Sinai, NCATS Scientists Uncover a Potential New Path Against Neurological Disease | Researchers at the Icahn School of Medicine at Mount Sinai in New York and NCATS identified compounds that reversed the effects of several neurodegenerative diseases called lysosomal storage disorders (LSDs) in patient cells and mice. LSDs are characterized by genetic defects that prevent the cell’s lysosomes from breaking down and recycling fats, sugars and proteins, which can accumulate in the liver and brain. This accumulation eventually causes a malfunction in the energy-producing mitochondria. The compounds increased the activity of TRAP1, a protein that helps the mitochondria work properly. Here, lipid-filled lysosomes (red) from Niemann-Pick disease type C1 are normalized when mitochondrial TRAP1 (green) is activated. (Ioannou lab, Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai) September 8, 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 called lysosomal storage disorders (LSDs) in patient cells and mice. LSDs are characterized by genetic defects that prevent the cell’s lysosomes from breaking down and recycling fats, sugars and proteins, which can accumulate in organs, including the liver and brain. This accumulation eventually causes a malfunction in the energy-producing mitochondria, leading to further damage in these organs. A team led by Mount Sinai’s Yiannis Ioannou, Ph.D., and NCATS translational scientist Juan Marugan, Ph.D., restored the proper function of both the mitochondria and lysosomes. The researchers suggest the findings could have implications for other neurodegenerative diseases that have similar underlying causes. They reported their results in iScience. The researchers identified novel compounds that increased the activity of TRAP1, a protein that helps the mitochondria function properly. “Researchers have always looked for drugs that could recover the function of lysosomes to try to impact lysosomal storage diseases,” Marugan said. “This is a new approach to these diseases. Increasing TRAP1 activity promoted mitochondrial protein folding and facilitated the cell’s proper balance. These first-in-class molecules activate TRAP1 in the mitochondria and reduce storage in lysosomal storage diseases.” “Our data demonstrate that mitochondrial TRAP1 is a potential novel therapeutic target for multiple disorders that affect the central nervous system,” Ioannou said. The Mount Sinai and NCATS teams began collaborating several years ago on a project involving the LSD Niemann-Pick disease type C1 (NPC1), a rare neurological disease. Ioannou and his colleagues developed a test to measure the effect of compounds on NPC1. Marugan and his NCATS team subsequently used the test with NCATS’ high-throughput screening facilities to rapidly sift through thousands of compounds. They discovered that some compounds that activated TRAP1 made the mitochondria work properly again and restarted the lysosome’s recycling ability, helping reduce fats in the lysosomes and in cells. The researchers chemically improved the compounds that appeared to work best and tested them further. The collaborative team showed that increasing the activity of TRAP1 in cells from people with NPC1 could correct their lipid storage disorder and restore normal cholesterol levels. In addition, increasing TRAP1 activity in cells from patients with other LSDs — such as Fabry, Farber and Wolman diseases — also corrected their respective lipid storage. In subsequent studies in mouse models of Fabry disease, the Mount Sinai researchers found similar results. Heightened TRAP1 activity in the animals resulted in a reduction in the abnormal amounts of lipids stored in the kidney, liver and heart. The researchers discovered that TRAP1 initiated a “cross-talk” between mitochondria and lysosomes, which, they determined, was important to restoring the internal cellular balance. “What is surprising is that TRAP1, when activated, initiates a cascade that leads to restoration of normal lysosomal function in lysosomal storage diseases,” Ioannou pointed out. “Keep in mind that the defect causing each lysosomal disease is still present, so this cross-talk bypasses the genetic defect.” The scientists would like to understand more about how the compounds can reverse characteristics of the lysosomal storage diseases, which will be instrumental in developing potential drug treatments. They also plan to continue to refine these compounds and examine their effects in various models, including their ability to affect more common neurological disorders, such as Alzheimer’s disease and Parkinson’s disease. “It’s known that similar dysfunctions in cell recycling and mitochondria health occur in other neurodegenerative disorders, such as Parkinson’s, amyotrophic lateral sclerosis and Alzheimer’s,” Marugan noted. “We believe that this approach could have therapeutic benefits for more mainstream disorders, as well.” NIH…Turning Discovery Into Health® | Scientists found compounds to reverse the effects of several neurodegenerative diseases in patient cells and mice. | /sites/default/files/Marugan_Ioannou_TRAP1_900x600.jpg | Uncovering a possible new drug target for neurological diseases | Scientists found compounds to reverse the effects of several neurodegenerative diseases in patient cells and mice. | /sites/default/files/Marugan_Ioannou_TRAP1_900x600.jpg | Uncovering a possible new drug target for neurological diseases | ||
| 25475 | Tiny Antibodies May Provide New Tool to Fight COVID-19 | An NIH-led team of scientists built a library of small antibodies, called synthetic nanobodies, and used it to find promising new therapeutic leads for halting SARS-CoV-2 infection. The SARS-CoV-2 spike protein is depicted in white, and its three receptor binding domains (RBDs) are highlighted in blue. The RBD is the portion of the viral spike protein that binds to the protein receptor, ACE2, on the surface of healthy cells. SARS-CoV-2 enters cells through ACE2. Three nanobodies (red) mask the binding portion of the RBDs, preventing the spike protein from recognizing ACE2. This prevents the virus from entering the cell. (Kedar Sharma, Ph.D., and Mario Borgnia, Ph.D., National Institute of Environmental Health Sciences.)August 30, 2022As SARS-CoV-2 continues to evolve, scientists are on the hunt for therapeutics to combat new variants. To speed the search, an NIH-led team of scientists built a library of small antibodies, called synthetic nanobodies, and used it to find promising new therapeutic leads for halting viral activity.The team from NCATS, the National Institute of Environmental Health Sciences and the Naval Research Laboratory reported their results in PLOS One.Nanobodies are found in shark and camelid species, which include camels, llamas and alpacas. They are one-tenth the size of a human antibody. Their small size lets them attach inside protein grooves on viruses. This feature could help point scientists to weaknesses on the SARS CoV-2 spike protein that might otherwise be inaccessible to full-sized antibodies.“Nanobodies make attractive building blocks for the design of new therapies,” said NCATS scientist Bryan Fleming, Ph.D., who helped lead the work, along with former NCATS scientist Ying Fu, Ph.D. “We’ve developed a way to rapidly and efficiently discover nanobodies against SARS-CoV-2.”The first step to building the library was making llama nanobodies appear more like human antibodies. The researchers “humanized” the nanobodies by using a U.S. Food and Drug Administration–approved nanobody drug backbone as the basis for the library.The researchers combined humanized “framework” regions, which act as support structures for nanobodies. These regions are shared among every nanobody in the library. Additionally, each nanobody has regions that recognize and stick to protein targets. These regions are like nanobody fingerprints and make each nanobody unique to a protein. Changing these regions creates new nanobodies capable of targeting new proteins.According to Fleming, the construction of a synthetic library can yield about 10 billion possible nanobodies. These nanobodies can be studied as individual therapies or can be combined to create combination therapies.“We hope that this approach for creating humanized nanobodies will accelerate the path to patients,” said NCATS translational scientist Matthew Hall, Ph.D., a co-author.The scientists made several other different libraries to have a wider variety of nanobodies to choose from. They used NCATS’ high-throughput screening facilities to search through their nanobody libraries to quickly find those that bound tightly to and potentially would work best against the SARS-CoV-2 receptor-binding domain (RBD). The virus uses the RBD to dock with a cell and gain entry.They identified three nanobodies that appeared to be most effective in blocking SARS-CoV-2 infection. The scientists tested the nanobodies against the B.1.1.7 (UK/alpha) SARS-CoV-2 variant, which was the dominant strain in the United States in early June 2020.They also tested the three nanobodies on different modified viruses that can enter a cell but not cause disease. They found that high nanobody concentrations prevented infection, whereas low concentrations did not.One of the nanobodies, RBD-1-2G, was especially strong in binding to the virus. The scientists modified RBD-1-2G, making different versions that were larger and closer to the structure of a more conventional antibody. They found that adding multiple RBD-1-2G nanobodies linked together into a single candidate therapeutic increased the effectiveness of blocking viral infection. A therapeutic composed of three nanobodies was found to be the most effective.In tests against other variants, RBD-1-2G was very active in blocking the UK/alpha variant, but did not work well against variants, such as delta, mu and omicron. The scientists plan to continue to use the library to evaluate other nanobodies against SARS-CoV-2 variants and other viruses.NCATS’ technology to develop a library of humanized nanobodies was among those NIH recently licensed to the Medicines Patent Pool through the World Health Organization COVID-19 Technology Access Pool. These licenses will enable manufacturers from around the world to use these technologies.A nanobody library could prove useful for noninfectious diseases, as well.“This is a huge library of antibodies that can be screened to identify drug candidates against almost any protein, related to almost any disease,” Hall said. “Given that there are over 10,000 rare diseases, most of which have no treatment, we hope this new platform will also prove useful in tackling that challenge. | Scientists built a library of synthetic nanobodies to search for new therapeutic leads that block SARS-CoV-2 infection. | /sites/default/files/Full2_900x600px_0.jpg | Humanizing Tiny Antibodies to Fight COVID-19 | Scientists built a library of synthetic nanobodies to search for new therapeutic leads that block SARS-CoV-2 infection. | /sites/default/files/Full2_900x600px_1.jpg | Humanizing Tiny Antibodies to Fight COVID-19 | ||
| 25250 | LitCoin Pilot Design Challenge | Winners Key Dates Background The Problem Challenge Goals Rules Contact Winners First prize Jonathon Keeney, Ph.D., and Patrick McNeely, Ph.D. Dr. Jonathon Keeney and his colleague, Dr. Patrick McNeely, detailed an approach to building LitCoin that would integrate seamlessly with current open publishing interfaces, such as bioRxiv, and would allow publishers some freedom to choose how they could be alerted about new LitCoin submissions. RTI International RTI International described a system with several unique layers of automation and user interaction that would make the system easy to use for all stakeholders during the process of creating LitCoin publications. Second prize Insilicom, LLC Insilicom, LLC submitted a unique modular approach to the LitCoin conceptual framework. This includes functionality for researchers to publish hypotheses, which currently is lacking in the world of biomedical research. Key Dates Note: Dates subject to change as necessary July 29, 2022: Challenge Announcement August 29, 2022, 9:00 AM EDT: Submission window opens October 31, 2022, 5:00 PM EDT: Submissions due December 16, 2022: Prize winners announced Background Following on the heels of the LitCoin Natural Language Processing (NLP) Challenge, the LitCoin Pilot Design Challenge seeks to spur innovation by rewarding the most creative and effective plans to construct the LitCoin submission platform. This platform will enable researchers to generate high-quality computationally-accessible knowledge and share it with the greater community, all in line with the mechanism they’re already familiar with, the manuscript publishing pipeline. This ideation challenge is part of a broader conceptual initiative at NCATS to change the “currency” of biomedical research. This Challenge brings together government, design experts, researchers and publishing houses to spur innovation in the design of an end-to-end LitCoin submission platform for further development of the LitCoin program. The winning solutions will be used to facilitate the design of further funding opportunities to implement the LitCoin Pilot Program. With a two (2) month development cycle of the Challenge, highly diverse and collaborative teams will be challenged to develop a plan for an end-to-end system for implementation of the pilot program. The Problem Increasing the accessibility and usability of biomedical knowledge is an important and very difficult goal which NIH has emphasized for years. In order to make strides toward this goal, researchers will need to have the ability to generate computationally-accessible knowledge at the time of publication, as opposed to relying on post-hoc data generation and expensive curation of knowledge by a third party. To advance the field of biomedical research by utilizing some of the most promising technology solutions built with knowledge graphs, NCATS and its collaborators have launched the LitCoin program. This program aims to 1) help data scientists better deploy their data-driven technology solutions towards accelerating scientific research in medicine, 2) ensure that data from biomedical publications can be maximally leveraged and reaches a wide range of biomedical researchers and drives the impact of the critical problems they aim to solve. To this end, we are launching the LitCoin Pilot Design Challenge. Challenge Goals The challenge will spur the creation of innovative strategies for building a system capable of accepting free text describing research findings, running NLP algorithms on that free text to generate knowledge graphs representing these findings, allowing the authors to curate these knowledge graphs, and presenting the authors with similar knowledge assertions extracted from previously-published research when such assertions exist. Finally, authors should have the opportunity to submit their research to specific publisher-partners along with the extracted knowledge graphs, to facilitate the review of both the free text submission and the associated knowledge graph. For this challenge, participants across academia and the private sector are invited to participate in teams, as representatives of an academic or private entity, or in an individual capacity by designing an end-to-end conceptual plan for the LitCoin submission platform which includes an explanation of the expected user interactions with the server, as well as requirements for the deposition of generated knowledge assertions into the open-access knowledgebase built from these assertions. This design plan should include requirements that ensure compatibility with a myriad of publishers and the submission and review platforms that they utilize. Participants should also include a description of business models that could be implemented for long-term sustainability of the program. Rules For information about the rules, submission requirements, judging criteria, prizes and how to enter, visit https://www.challenge.gov/?challenge=litcoin-pilot-design-challenge. Contact Have feedback or questions about this challenge? Please send your feedback or question to litcoin-questions@mail.nih.gov. Back to Top | LitCoin Pilot Design Challenge seeks to spur innovation by rewarding the most creative and effective plans to construct the LitCoin submission platform. | /sites/default/files/litcoin_meta_image_680w_0.png | LitCoin Pilot Design Challenge | LitCoin Pilot Design Challenge seeks to spur innovation by rewarding the most creative and effective plans to construct the LitCoin submission platform. | /sites/default/files/litcoin_meta_image_680w_1.png | LitCoin Pilot Design Challenge | ||
| 25166 | Additional Rare Diseases Research and Initiatives | NCATS supports rare diseases research by funding a variety of extramural research programs and taking part in initiatives and activities aimed at addressing the public health crisis presented by rare diseases.On this page:Extramural Rare Diseases Research ProgramsOther NCATS Rare Diseases Activities and InitiativesSee our Gene Therapies and Gene Editing Programs page to learn about NCATS’ role in accelerating gene-targeted therapies for rare diseases.Extramural Rare Diseases Research ProgramsBasket Clinical Trials of Drugs Targeting Shared Molecular Etiologies in Multiple Rare DiseasesResearch teams funded by Basket Clinical Trials of Drugs Targeting Shared Molecular Etiologies in Multiple Rare Diseases grants will adapt the shared molecular etiology approach from oncology to rare diseases.Contact: P.J. Brooks, Ph.D.Clinical Trial Readiness for Rare Diseases, Disorders and SyndromesThe Clinical Trial Readiness for Rare Diseases, Disorders and Syndromes grants support projects focused on collecting the data needed to move promising rare disease therapies and diagnostics to clinical trial.Contact: Alice Chen Grady, M.D.Multidisciplinary Machine-Assisted Genomic Analysis and Clinical Approaches to Shortening the Rare Diseases Diagnostic OdysseyNCATS is funding innovative research using a variety of tools and approaches to shorten the time it takes to identify and accurately diagnose rare diseases.Contact: Alice Chen Grady, M.D.Other NCATS Rare Diseases Activities and InitiativesImpact of Rare Diseases on Patients and Healthcare Systems (IDeaS) for ChangeThe IDeaS study, led by NCATS, provided new evidence of the potential effect of rare diseases on public health, suggesting that nationwide medical costs for individuals with rare diseases are similar to costs for people with cancer or heart failure.Download the IDeaS study fact sheet.Contact: Dominique C. Pichard, M.D., M.S.Shared Molecular Etiologies Task ForceNCATS participates in the International Rare Diseases Research Consortium (IRDiRC) Task Force on Shared Molecular Etiologies Underlying Multiple Rare Diseases. The Task Force will address and document challenges in adapting the basket trial approach used in molecularly targeted oncology clinical trials to drugs targeting shared molecular etiologies underlying multiple rare diseases.Contact: P.J. Brooks, Ph.D. | NCATS funds extramural research and participates in initiatives and activities addressing rare diseases. | /sites/default/files/NCATS_default_metaimage_tagline_FINAL_0.png | Additional Rare Diseases Research and Initiatives | NCATS funds extramural research and participates in initiatives and activities addressing rare diseases. | /sites/default/files/NCATS_default_metaimage_tagline_FINAL_0.png | Additional Rare Diseases Research and Initiatives | ||
| 25064 | Media Advisory: To Find Answers for People on Earth, NIH-Funded Tissue Chip Models Take Flight | Researchers at the University of California, San Francisco (UCSF) are using tissue chips, 3-D bioengineered models of living organs and tissues, to study the impact of an aging immune system on the body’s ability to heal injured tissue. Tissue chips allow scientists to examine the effects of culturing immune cells and cells called mesenchymal stem cells (MSCs) in space, imitating how they interact in the body. MSCs are important for making and repairing tissues such as cartilage and bone. (Sonja Schrepfer, M.D., Ph.D., UCSF)July 13, 2022WHAT: Two National Institutes of Health-funded research teams will send tissue chips — tiny, 3-D bioengineered models of living organs and tissues built from human cells — to the International Space Station National Laboratory (ISS-NL). The tissue chips will spend several weeks in orbit to collect information about why the aging process causes muscles to lose mass and strength and why the immune system works less effectively. The projects will be aboard the SpaceX CRS-25, which is scheduled to lift off no earlier than July 14 from NASA’s Kennedy Space Center in Cape Canaveral, Florida.Scientists have been using the low-gravity conditions of space as a laboratory for decades. When astronauts spend time in space, their bodies change. Many of these physiological changes are like the effects seen in aging. Studies using tissue chips on the ISS provide a unique opportunity for researchers to model and study conditions related to diseases and aging over weeks, rather than the years that it would take for these conditions to develop on Earth. In many cases, investigators also are using this unique environment to test potential therapies to mitigate aging effects.The projects are funded through the Tissue Chips in Space program, a collaboration among NIH’s National Center for Advancing Translational Sciences (NCATS) and National Institute of Biomedical Imaging and Bioengineering (NIBIB) and the ISS-NL.WHEN: The launch is scheduled for no sooner than July 14, 2022, at 8:44 p.m. EDT; monitor twitter.com/Space_Station for schedule changes.WHERE: Cape Canaveral, FloridaNIH SPOKESPEOPLE:Joni L. Rutter, Ph.D., Acting Director, NCATSDanilo A. Tagle, Ph.D., Director, NCATS Office of Special InitiativesTo schedule interviews with NCATS staff, please email ncatsinfo@mail.nih.gov.PROJECT DETAILS:Researchers at the University of California, San Francisco (UCSF) are using tissue chip technology to better understand how a person’s aging immune system can affect the body’s ability to heal injured tissue. Scientists know that immune cells can work less efficiently as people age, dimming the immune response (termed immunosenescence). They think that certain immune cells are tied to the ability of stem cells to regenerate tissue and heal injuries and wounds. The UCSF team is examining the effects of culturing immune cells and stem cells in space. Their initial space experiments revealed that immune cells showed accelerated aging, which hindered stem cells’ abilities to repair damaged tissue. When the current flight’s cells return to Earth, the scientists will analyze the changes to the cells’ biology and how these changes reverse in time on the ground. They hope this information will provide insights to improve the function of aging immune cells.Researchers at the University of Florida are using tissue chips to examine the loss of muscle strength and mass in space, which is similar to what happens in people as they age (a condition called sarcopenia). They will compare muscle cells derived from athletic, young volunteers and from sedentary, older ones, including the cells’ response to electrical stimulation on Earth and in space. When the project returns to Earth, the team will study genetic changes in the cells. Insights from the project could lead to new therapies to treat and even prevent muscle-wasting diseases. On a subsequent trip to the ISS, expected in the fall, the team will use the tissue chips to examine the effectiveness of a candidate drug in preventing muscle loss.MORE INFORMATION:For more information on NCATS and its tissue chip program, see:Tissue Chips in SpaceTissue Chip for Drug ScreeningTissue Chip ImagesAbout the National Center for Advancing Translational Sciences (NCATS): NCATS conducts and supports research on the science and operation of translation — the process by which interventions to improve health are developed and implemented — to allow more treatments to get to more patients more quickly. For more information about how NCATS helps shorten the journey from scientific observation to clinical intervention, visit https://ncats.nih.gov .About the National Institute of Biomedical Imaging and Bioengineering (NIBIB): NIBIB’s mission is to improve health by leading the development and accelerating the application of biomedical technologies. The Institute is committed to integrating engineering and physical sciences with biology and medicine to advance our understanding of disease and its prevention, detection, diagnosis and treatment. NIBIB supports emerging technology research and development within its internal laboratories and through grants, collaborations and training. More information is available at the NIBIB website: https://www.nibib.nih.gov/.About the National Institutes of Health (NIH): NIH, the nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical and translational medical research, and is investigating the causes, treatments and cures for both common and rare diseases. For more information about NIH and its programs, visit https://www.nih.gov. | NIH-supported tissue chip projects can help researchers study how aging-related conditions affect muscle and immune function. | /sites/default/files/Tissue_Chips_Take_Flight_900x600_0.png | To Improve Health on Earth, NIH-Funded Tissue Chips Head Into Space | NIH-supported tissue chip projects can help researchers study how aging-related conditions affect muscle and immune function. | /sites/default/files/Tissue_Chips_Take_Flight_900x600_1.png | To Improve Health on Earth, NIH-Funded Tissue Chips Head Into Space |
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