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Announcement: Access to the COVID-19 Data Analytics Platform Is Open |
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Latest NewsNational COVID Cohort Collaborative (N3C) Data EnclaveMore than 4.5 million COVID-19-positive patients and more than 14 billion rows of data are included in this enclave. Apply for N3C access today.The public-facing N3C Cohort Exploration Dashboard provides high-level information about the N3C cohort and N3C Data Enclave.More than 300 projects are underway using the enclave data to examine associations between COVID-19 patient outcomes and social determinants of health. View the current list of publications.Study shows a link between immune dysfunction with an increased risk for COVID-19 breakthrough infection.September 2, 2020A Resource Unlike Any OtherThe NCATS N3C maintains one of the largest collections of clinical data related to COVID-19 symptoms and patient outcomes in the United States. Having access to a centralized enclave of this magnitude allows research teams to study, probe and answer clinically important questions about COVID-19 that they could not have answered previously.With stewardship from NCATS, more than 70 institutions, including more than 40 Clinical and Translational Science Awards (CTSA) Program hubs, worked together to build this extensive database to help researchers study COVID-19 and identify potential treatments as the pandemic evolves.Harmonized dataThe N3C platform translates the different ways that contributing hospitals store patient data into a single, common format to enable combined “apples-to-apples” analyses.Robust in scale and scopeCurrently, 89 sites across the country have agreed to transfer diverse data from individuals tested for COVID-19, including demographics, symptoms, laboratory test results, procedures, medications, medical conditions, physical measurements and more.By marshalling the national reach of the CTSA Program network, N3C is ensuring that the data represent the diversity of the country so researchers can understand and address geographic and population disparities during the pandemic.Powerful analytics capabilitiesThe platform is built to enable machine-learning approaches and rigorous statistical analyses to identify connections and patterns more quickly than is possible through traditional methodologies. These advanced analytics approaches can lead to the simultaneous exploration of multiple questions — and to the revelation of likely answers — on a powerful scale.Centralized and secureThe data reside and remain in NCATS’ secure, cloud-based database, certified through the Federal Risk and Authorization Management Program, or FedRAMP, which provides standardized assessment, authorization and continuous monitoring of cloud products and services, thereby ensuring the validity of the data while protecting patient privacy.Three levels of protected data are included for analysis:Limited Data Set (LDS): Consists of patient data that retain the following protected health information —dates of servicepatient ZIP codeDe-identified Data Set: Consists of patient data from the LDS with the following changes —Dates of service are algorithmically shifted to protect patient privacy.Patient ZIP codes are truncated to the first three digits or removed entirely if the ZIP code represents fewer than 20,000 individuals.Synthetic Data Set: Consists of data that are computationally derived from the LDS that resemble patient information statistically but are not actual patient data.Learn more about N3C data including data stewardship and protections and the requirements for accessing different levels of data.Hongfang Liu, Ph.D., program director for informatics at Mayo Clinic, is experienced in using clinical data for translational science research and to improve health care delivery. Dr. Liu explains how collaborating with experts in other disciplines to build the N3C Data Enclave will advance the science behind COVID-19 to deliver health care interventions and treatments.View all I Am Translational Science videos.A Powerful Tool for ResearchersThere are more than 250 projects underway to explore a range of questions. Access the complete listing of projects that have been submitted through the Data Use Request (DUR) process and were approved by the N3C Data Access Committee.Researchers interested in accessing the data will need to register with N3C and submit a DUR for review by the N3C Data Access Committee.New Challenge The Biomedical Advanced Research and Development Authority, in partnership with NCATS and the Eunice Kennedy Shriver National Institute of Child Health and Human Development along with the Health Resources and Services Administration’s Maternal and Child Health Bureau are sponsoring a challenge competition that will leverage de-identified electronic health record data to develop, train and validate computational models that can predict severe COVID-19 complications in children. This will equip health care providers with the information and tools they need to identify pediatric patients at risk. Health record data will be provided through the N3C Data Enclave.The N3C is a partnership among the NCATS-supported CTSA Program hubs, the National Center for Data to Health (CD2H), and NIGMS-supported Institutional Development Award Networks for Clinical and Translational Research (IDeA-CTR), with overall stewardship by NCATS.More information:Learn more about the N3C and see a demonstration of the analytics platform.Read the latest FAQs.Find forms and other N3C resources.If you have questions about the N3C, please email NCATS_N3C@nih.gov. |
The National COVID Cohort Collaborative (N3C) built the N3C Data Enclave, a COVID-19 national data resource. |
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N3C Data Enclave |
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2019 ExRNA Projects: Single Extracellular Vesicle Sorting, Isolation and Analysis of Cargo |
NCATS is administering six NIH Extracellular RNA (exRNA) Communication program projects that are supported by the NIH Common Fund. These projects will establish new tools, technologies and methodologies to sort, isolate and analyze extracellular vesicles (EVs) and their cargo, including exRNA. These projects will clarify the role of EVs in exRNA transport and cellular communication and accelerate the development of EV-based disease diagnosis and tracking protocols.
Single Extracellular Vesicle Sorting and Analysis
Molecular Dissection and Imaging of Extracellular Vesicles to Define Their Origin and Targets
Integrative, Multi-parametric Characterization of the EV Surface Protein and Nucleic Acid Landscape by Nano-flow and Sorting Cytometry
Microfluidics Array-Based Sorting, Isolation and RNA Analysis in Single Extracellular Vesicles
Enabling Isolation and Characterization of Single Extracellular Vesicles and Their Molecular Contents Using Multi-marker Surface Signatures
Acoustofluidic Separation, Purification and Raman Spectral Fingerprinting of Single EVs: From Cell of Origin to Target Cell and Biofluids
Single Extracellular Vesicle Sorting and Analysis
Investigator: Daniel T Chiu, Ph.D., University of Washington, Seattle
Grant Number: UG3-TR002874
EVs are membrane-enclosed particles that are secreted from cells into the extracellular space. They are highly diverse and carry different kinds of cargo, including exRNA. Effectively studying the subtypes of EVs and their cargo requires isolating them with high precision and sensitivity. Current EV isolation methodologies are prone to contamination. Moreover, established methodologies work in bulk, collecting many different EVs and reporting on the average, which obscures differences that could provide insight into EV subpopulations. This study team will develop and apply new technologies to study EVs at the single-EV level. These technologies include methods that sort individual EVs with high sensitivity and throughput and use a digital imaging platform that will help to quantify the RNA contents in both single and grouped EVs. These new technologies will enable better sorting and analysis of individual EVs, which will provide a deeper understanding of their biological role and lead to better utilization of EVs in diagnostic and therapeutic applications.
Learn more about this project in the NIH RePORTER.
Molecular Dissection and Imaging of Extracellular Vesicles to Define Their Origin and Targets
Investigator: Saumya Das, M.D., Ph.D., Massachusetts General Hospital, Boston; Tijana Talisman, Ph.D., Beckman Research Institute at City of Hope, Duarte, California; and Kendall Van Keuren-Jensen, Ph.D., Translational Genomics Research Institute: An Affiliate of City of Hope, Phoenix
Grant Number: UG3-TR002878
EVs can contain RNA (EV-RNA) that can be used to diagnose or predict many different diseases. Isolating and characterizing EV-RNA with respect to specific tissues and disease states, however, is challenging, preventing the widespread use of EV-RNA as a diagnostic tool in the clinic. This project team will develop technology that enables the isolation and characterization of EVs from hematopoietic cells (cells in the bone marrow and surrounding blood that can develop into any kind of blood cell), the brain and the heart. The team also will identify differences in EVs from human subjects with acute diseases, such as a heart attack or stroke, and from subjects who are undergoing physiological stress processes, such as exercise. This work will provide insight into how different tissues contribute to EV-RNA in standard, disease and stress conditions. This understanding will facilitate the discovery and development of EV biomarkers and the identification of tissue-specific EV-RNA, which can sensitively and accurately provide insight into disease progression and regression.
Learn more about this project in the NIH RePORTER.
Integrative, Multi-parametric Characterization of the EV Surface Protein and Nucleic Acid Landscape by Nano-Flow and Sorting Cytometry
Investigator: Ionita Calin Ghiran, M.D., Beth Israel Deaconess Medical Center, Boston; Jennifer Jones, M.D., Ph.D., National Cancer Institute, Bethesda; and Aleksandar Milosavljevic, Ph.D., Baylor College of Medicine, Houston
Grant Number: UG3-TR002881
Cells use EVs to communicate with other cells. This cellular communication can change in response to varying conditions, because EVs differ greatly in their cargo and membrane proteins. EV-based communication can trigger differences in gene expression and alter cell function in a cell-specific manner. In diseases like cancer, abnormal EV-based communication can even alter the host immune response and synchronize the behavior of secondary tumor development and growth. By isolating and comparing EVs in both healthy and diseased states, the components of EVs that signal and exacerbate disease status can be identified and used as biomarkers for diagnosing and tracking disease. Using EVs as biomarkers, however, is restricted by the lack of effective EV sorting methods, thereby forcing bulk-analysis strategies that are biased against low-abundance EV subsets. This study team will focus on the reliable and reproducible characterization of EVs with single-EV resolution. Specifically, the team will develop new ways to sort EVs, better detect components of EV subpopulations, and establish protocols that will promote standardization across laboratories and clinics. These methods will enable the sorting and characterization of unique EVs based on tissue type and disease state, which will greatly facilitate the utilization of EVs as disease biomarkers.
Learn more about this project in the NIH RePORTER.
Microfluidics Array-Based Sorting, Isolation and RNA Analysis in Single Extracellular Vesicles
Investigator: Eduardo Reategui, Ph.D., The Ohio State University, Columbus, and Yon Son Betty Kim, M.D., Ph.D., The University of Texas MD Anderson Cancer Center, Houston
Grant Number: UG3-TR002884
EVs are present in all biological fluids and contain different biomolecules, including DNA, RNA, proteins and metabolites. These biomolecules enable EVs to be used in fine-tuned cell-to-cell communication. The role of EVs in cellular communication is biologically valuable, but EV communication is difficult to study, because isolating and characterizing EVs is still technically challenging. Much of the current EV-analysis technology requires that many different EVs be consolidated and broken down to their internal contents, which are subsequently tested as a whole. This obscures individual EV information, showing RNA, DNA, proteins and metabolite content only in aggregate. The specialized role of EVs in cell-to-cell communication, however, is made possible by their differences. Thus, EVs must be studied on an individual level to establish the effects of different EVs on cellular communication, including how they relate to disease states. This study team will analyze the molecular content of individual EVs using multilevel sorting methods to isolate EV subpopulations based on size and molecular composition. In addition, the team will study EV-based cell-to-cell communication to determine whether and how certain EV subpopulations are involved in immune regulation and disease states. This EV analysis will be done using clinical samples from patients with glioblastoma, a cancer that can occur in the brain or spinal cord, to identify one or more subpopulations of EVs that may be involved in immunosuppression or associated with worse clinical outcomes in glioblastoma patients.
Learn more about this project in the NIH RePORTER.
Enabling Isolation and Characterization of Single Extracellular Vesicles and Their Molecular Contents Using Multi-marker Surface Signatures
Investigator: David Aaron Routenberg, Meso Scale Diagnostics, LLC, Rockville
Grant Number: UG3-TR002886
EVs are important for cell-to-cell communication and serve different roles in normal physiologic function and disease states. To serve these different roles and transmit different signals between cells, the EVs themselves must be different. EVs carry different signaling cargo, and they differ greatly in the proteins on their surfaces. This study team will develop a new way to isolate specific types of EVs based on these differing surface proteins. The team will target two or more cell type-specific EV surface molecules to isolate EV subsets. Once highly purified populations of EVs have been isolated, their molecular contents will be measured. By determining the molecular contents and surface proteins specific to EVs from defined cell types, the team can develop a new, scalable approach to identify multi-marker surface signatures for cell types. EVs with these surface signatures can then be isolated with greater specificity, which will enable EV studies that are more targeted than those possible with current methods. These new methods can then be applied to high-throughput systems to automate single-EV characterization, thereby enabling high-resolution studies that would otherwise be too large or too time consuming to be cost-effective.
Learn more about this project in the NIH RePORTER.
Acoustofluidic Separation, Purification and Raman Spectral Fingerprinting of Single EVs: From Cell of Origin to Target Cell and Biofluids
Investigator: David T Wong, D.M.D., D.M.Sc., University of California, Los Angeles; Tony Jun Huang, Ph.D., Duke University, Durham; Sung Kim, M.D., Samsung Medical Center, Seoul; Yong Kim, Ph.D., Dental Research Institute, Los Angeles; and Ya-Hong Xie, Ph.D., University of California, Los Angeles, Samueli School of Engineering, Los Angeles
Grant Number: UG3-TR002978
Gastric, or stomach, cancer can be a particularly painful and devastating disease, and diagnosing the disease can be difficult and invasive. A prior project, however, discovered and validated exRNA markers that can be used to effectively detect gastric cancer. Biomolecule cargo, including exRNA, can be contained in different types of EVs. The study team for this project will develop technology to isolate single EVs and characterize their exRNA cargos so that these cargos can be associated with different cells of origin, target cells and biofluids. In particular, the investigators of this project seek to identify salivary exRNA markers in EVs that are indicative of the presence and progress of gastric cancer. The completion of this project will result in a set of novel technologies enabling rapid, high-yield, single-EV-level isolation that facilitates the detection and tracking of gastric cancer.
Learn more about this project in the NIH RePORTER.
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NCATS is administering six NIH Extracellular RNA (exRNA) Communication program projects focused on single extracellular vesicle sorting, isolation and analysis of cargo. |
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2019 ExRNA Projects: Single Extracellular Vesicle Sorting |
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NIH launches analytics platform to harness nationwide COVID-19 patient data to speed treatments |
The National COVID Cohort Collaborative aims to transform clinical information from across the country into knowledge urgently needed to study COVID-19, including health risk factors that indicate better or worse outcomes of the disease, and identify potentially effective treatments. Watch a demonstration of the N3C platform. (NCATS)June 15, 2020The National Institutes of Health has launched a centralized, secure enclave to store and study vast amounts of medical record data from people diagnosed with coronavirus disease across the country. It is part of an effort, called the National COVID Cohort Collaborative (N3C), to help scientists analyze these data to understand the disease and develop treatments. This effort aims to transform clinical information into knowledge urgently needed to study COVID-19, including health risk factors that indicate better or worse outcomes of the disease, and identify potentially effective treatments.The N3C is funded by the National Center for Advancing Translational Sciences (NCATS), part of NIH. The initiative will create an analytics platform to systematically collect clinical, laboratory and diagnostic data from health care provider organizations nationwide. It will then harmonize the aggregated information into a standard format and make it available rapidly for researchers and health care providers to accelerate COVID-19 research and provide information that may improve clinical care. A demonstration of the platform can be viewed at ncats.nih.gov//research/research-activities/n3c.Having access to a centralized enclave of this magnitude will help researchers and health care providers answer clinically important questions they previously could not, such as, “Can we predict who might need dialysis because of kidney failure?” or “Who might need to be on a ventilator because of lung failure?” and “Are there different patient responses to coronavirus infection that require distinct therapies?”“NCATS initially supported the development of this innovative collaborative technology platform to speed the process of understanding the course of diseases, and identifying interventions to effectively treat them,” said NCATS Director Christopher P. Austin, M.D. “This platform was deployed to stand up this important COVID-19 effort in a matter of weeks, and we anticipate that it will serve as the foundation for addressing future public health emergencies.”Data access will be open to all approved users, regardless of whether they contribute data. The data are being provided to NCATS as a Limited Data Set (LDS) that retains only two of 18 HIPAA-defined elements: patient zip code and dates of service.NCATS, which is serving as stewards of the data, is taking multiple security and privacy measures. For example, NCATS oversees the use of N3C through user registration, federated login, data use agreements with institutions and data use requests with users. The data reside and remain in NCATS’ secure, cloud-based database certified through the Federal Risk and Authorization Management Program, or FedRAMP, which provides standardized assessment, authorization, and continuous monitoring for cloud products and services ensuring the validity of the data while protecting patient privacy. Approved users must analyze data within the platform. In addition, the N3C data will be used only for COVID-19 research purposes, including clinical and translational research and public health surveillance.The information available via the N3C enclave will be rich in scope and scale. There currently are 35 collaborating sites across the country and the platform contains diverse data from individuals tested for COVID-19. A key component is the harmonization of data, which translates the different ways that contributing hospitals store patient data into a single, common format to enable combined ‘apples to apples’ analyses. Contributing sites add demographics, symptoms, medications, lab test results, and outcomes data regularly over a five-year period, enabling both the immediate and long-term study of the impact of COVID-19 on health outcomes.The platform is built to enable machine learning approaches and rigorous statistical analyses, identifying connections and patterns more quickly than can be done through traditional methodologies. These advanced analytics approaches require large, robust datasets to generate statistically valid results and can lead to the simultaneous exploration of multiple questions – and the revealing of likely answers – on a powerful scale.“The exciting transformation this platform represents is in providing an environment where data and the power of the analytics can be used by researchers and clinicians to quickly examine and answer new COVID-19 hypotheses,” said Warren A. Kibbe, Ph.D., chief of Translational Biomedical Informatics in the Department of Biostatistics and Bioinformatics and chief data officer for the Duke Cancer Institute.The N3C harnesses the extensive resources of the NCATS-funded Clinical and Translational Sciences Awards (CTSA) Program and its Center for Data to Health (CD2H). “By leveraging our collective data resources, unparalleled analytics expertise, and medical insights from expert clinicians, we can catalyze discoveries that address this pandemic that none of us could enable alone,” said Melissa Haendel, Ph.D., director of CD2H at the Oregon Health & Science University School of Medicine and Director of Translational Data Science at Oregon State University. To learn more about the N3C, including data transfer and access, visit covid.cd2h.org.In addition to NCATS, other NIH support for the N3C comes from the National Cancer Institute, the National Institute of Diabetes and Digestive and Kidney Diseases and the National Institute of General Medical Sciences.About 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 NCATS and its programs, visit https://ncats.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…Turning Discovery Into Health® |
The National COVID Cohort Collaborative (N3C) systematically collects and analyzes clinical, laboratory and diagnostic data. |
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NIH launches analytics platform to harness nationwide COVID-19 patient |
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COVID-19 OpenData Portal |
One potential strategy against SARS-CoV-2, the novel coronavirus behind the disease COVID-19 and a worldwide pandemic, is to use old drugs in new ways. This approach is called drug repurposing and can cut the time it takes to develop U.S. Food and Drug Administration-approved drugs from as long as 10 to 15 years to just 1 to 2 years. To further reduce this timeline, NCATS created a new resource for scientists, the OpenData Portal, to openly and quickly share COVID-19-related drug repurposing data and experiments for all approved drugs.
NCATS researchers developed the portal by using SARS-CoV-2-related assays to screen over 10,000 compounds, including the NCATS Pharmaceutical Collection of nearly 3,000 approved drugs, for their activity against the virus. The resource includes information on assays (tests), protocols for using the assays, drug targets, mechanisms of drug action and screening assay data. These data, which include positive and negative results, can be viewed, sorted, searched and exported from the portal website. Screening data are uploaded to the website as they become available. All data on the site come from NCATS-validated SARS-CoV-2 assays.
The scientific community can use the data for a variety of drug repurposing activities, allowing them to formulate and test hypotheses, prioritize research opportunities and speed the search for effective therapies against the virus and the disease it causes.
For questions about NCATS’ COVID-19 OpenData Portal, please email Matt Hall.
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NCATS’ OpenData portal has datasets, experiments and protocols for SARS-CoV-2-related assays against all approved drugs. |
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COVID-19 OpenData Portal |
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National COVID Cohort Collaborative (N3C) |
The N3C is a partnership among the NCATS-supported Clinical and Translational Science Awards (CTSA) Program hubs and the National Center for Data to Health (CD2H), with overall stewardship by NCATS. Collaborators will contribute and use COVID-19 clinical data to answer critical research questions to address the pandemic.
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National COVID Cohort Collaborative Funding |
Current Opportunities
PAR-21-121: Limited Competition: Small Grant Program for the NCATS Clinical and Translational Science Award (CTSA) Program (R03 Clinical Trial Optional)
NOT-TR-21-022: Notice of Special Interest (NOSI): Availability of Emergency Competitive Revisions for the Clinical and Translational Science Award (CTSA) Program to Address COVID-19 Public Health Needs
NOT-TR-21-017: Notice of Special Interest (NOSI): Administrative Supplements for the Clinical and Translational Science Award (CTSA) Program to Address COVID-19 Public Health Needs
Expired Announcements
This expired funding announcement includes details on the application process, eligibility and timelines for the program:
NOT-TR-21-011: Notice of Special Interest (NOSI): Availability of Emergency Administrative Supplements to Clinical and Translational Science Award (CTSA) Program Awards to Address 2019 Novel Coronavirus Disease (COVID-19) Public Health Needs
NOT-TR-20-028: Notice of Special Interest (NOSI): Availability of Emergency Competitive Revisions and Administrative Supplements to Clinical and Translational Science Award (CTSA) Program Awards to Address 2019 Novel Coronavirus Disease (COVID-19) Public Health Needs
Contact
For questions about executing a Data Transfer Agreement, contact NCATSPartnerships@mail.nih.gov.
For other questions about the N3C, contact NCATS_N3C@nih.gov.
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National COVID Cohorts Collaborative Funding |
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National COVID Cohort Collaborative Program Resources |
Learn More About the Program
Additional details about the program are available on the National Center for Data to Health’s N3C website.
For CTSA Program investigators: Watch the presentation NCATS Director Christopher P. Austin, M.D., gave to the CTSA Program consortium on May 8, 2020 or download the slides.
Download Program Documents
NCATS Data Transfer Agreement (DTA)
OMOP Version 5.3.1 Common Data Specifications
For questions about executing the DTA, contact NCATSPartnerships@mail.nih.gov.
Find COVID-19 Updates
Learn about other NCATS-supported research activities to address the novel coronavirus 2019 (SARS-CoV-2) and the disease it causes (COVID-19).
Stay on top of the latest announcements and research information from NIH.
Get public health updates and resources from the U.S. Centers for Disease Control and Prevention.
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National COVID Cohort Collaborative Program Resources |
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National COVID Cohort Collaborative (N3C) |
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The N3C offers one of the largest collections of secure and deidentified clinical data in the United States for COVID-19 research. Its ever-growing size and capabilities allow researchers and clinicians to study COVID-19 health outcomes. N3C represents a shared vision for turning real-world data into the knowledge needed to address COVID-19 as the pandemic evolves.
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NIH-Supported Research Survey to Examine Impact of COVID-19 on Rare Diseases Community |
The illustration depicts the shape and outer structure of a coronavirus. The virus uses the spike-shaped proteins, shown in red, on its outer surface to invade human cells. (U.S. Centers for Disease Control and Prevention)May 7, 2020For the millions of people living with a rare disease, the novel coronavirus disease COVID-19 presents challenges, from potential reduced access to needed medical care to possible heightened anxiety and stress. A new online survey launched by the National Institutes of Health-supported Rare Diseases Clinical Research Network (RDCRN) aims to find out how the COVID-19 pandemic is impacting individuals with rare diseases, their families and their caregivers. Results will help the rare disease research community shed light on the needs of people with rare diseases during the COVID-19 pandemic and other potential health crises, in addition to informing future research efforts.The RDCRN, led by NIH’s National Center for Advancing Translational Sciences (NCATS), in collaboration with nine other NIH Institutes and Centers, currently is made up of 20 recently funded clinical research consortia focused on better understanding how rare diseases progress and developing improved approaches for diagnosis and treatment. Scientists from different disciplines at hundreds of clinical sites around the world work together with about 140 patient advocacy groups to study more than 200 rare diseases, including immune system disorders, heart, lung and kidney disorders, brain development diseases and more.“As a leader in fostering innovative, collaborative clinical research to improve the lives of individuals with rare diseases, the RDCRN is uniquely positioned to carry out a survey like this,” said Anne Pariser, M.D., director of the NCATS Office of Rare Diseases Research, which oversees the RDCRN. “The network has the necessary infrastructure, disease expertise, and access to patients through patient organizations to find answers to important questions.”Though individually rare, affecting only a few hundred to several thousand people, rare diseases collectively affect an estimated 30 million people in the United States. Many rare diseases are life-threatening, and about half of those affected are children.The research survey, developed and led by the RDCRN Data Management and Coordinating Center at Cincinnati Children’s Hospital Medical Center, is one of the first efforts nationwide to quantify the impact of a health crisis on the rare disease community. It is seeking responses from at least 5,000 people with a rare disease or caring for someone who has a rare disease. The survey will be distributed online to participants. In addition, some RDCRN-funded scientists plan to incorporate survey results into natural history studies, which follow patients to chart the progression and course of a disease. The survey is open to anyone with a rare disease, along with family and caregivers, and is not limited to the diseases studied within the RDCRN.The impetus for the survey began through conversations among network researchers and patient advocacy organizations. Patients, families and caregivers were worried about how COVID-19 might affect them.“People affected by a rare disease, and families and caregivers, initially asked how to avoid the virus,” said RDCRN Program Director Tiina Urv, Ph.D. “Then they became concerned about access to medicines and maintaining medical care during the pandemic, and the status of clinical trials. They were concerned about meeting the medical challenges that they face every day. We were hearing enough anecdotally that we wanted to get a clearer picture of the problem.”As consortia scientists and clinicians engaged with patient groups and patients, sharing information and advice, a plan came together over several weeks to conduct a scientific research study to gauge the impact of COVID-19 on those in the rare disease community.Questions in the research survey focus on a range of topics, from a patient’s ability to get proper care for a rare disease or condition to mental and emotional health. The survey asks what their concerns are as a person with a rare disease, or as family members and caregivers. Groups of people with different rare diseases and the community will have different needs and concerns, whether it is how to get needed medications or physical therapy to navigating an emergency room in a medical crisis.“We hope the study questionnaire will help us better estimate the proportion of rare disease patients who have been diagnosed with COVID-19, and find out how they are affected whether or not they had COVID-19,” said project principal investigator Maurizio Macaluso, M.D., Dr.P.H. at Cincinnati Children’s. “This survey provides an opportunity for the rare disease community to get timely data on the challenges they face.”The researchers also think the survey data may help them tease out answers to many other questions. For example, do some subgroups of people with rare disease fare better or worse with the virus? Are certain individuals more prone to infection because of their underlying rare condition or disease?Ultimately, the researchers hope the survey will help determine how the RDCRN can respond to the rare disease community’s concerns by providing information and advice through its network of medical experts and patient advocacy groups.“This survey is a great example of how the consortia and patient groups are working together as a network to make a difference for the entire rare disease community,” Urv said.For more information on the RDCRN COVID-19 survey, including how to participate, go to https://www.rarediseasesnetwork.org/COVIDsurvey. To learn more about the RDCRN, see https://ncats.nih.gov/research/research-activities/rdcrn.In addition to NCATS, other NIH funding for the RDCRN comes from the National Institute of Allergy and Infectious Diseases, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Institute of Neurological Disorders and Stroke, the National Heart, Lung, and Blood Institute, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute of Diabetes and Digestive and Kidney Diseases, the National Institute of Dental and Craniofacial Research, the National Institute of Mental Health and the Office of Dietary Supplements.About 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 NCATS and its programs, visit https://ncats.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…Turning Discovery Into Health® |
A new online survey launched by the NIH-supported RDCRN aims to find out how the COVID-19 pandemic is impacting individuals with rare diseases, their families and their caregivers. |
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NIH-Supported Research Survey to Examine Impact of COVID-19 on Rare Di |
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16380 |
2020 New Therapeutic Uses for Experimental Assets Projects |
In 2020, NCATS issued funding for one cooperative agreement designed to match academic research groups with selected compounds from industry as part of the New Therapeutic Uses program. The goal is to use molecules that already have undergone significant research and development by the pharmaceutical industry to more quickly advance new treatments for patients.
Use of the Src Family Kinase Inhibitor Saracantinib in the Treatment of Pulmonary Fibrosis
National Jewish Health
Principal Investigators: Gregory Paul Downey, M.D. (National Jewish Health), Naftali Kaminski, M.D. (Yale University), Maria L. Padilla, M.D. (Mount Sinai School of Medicine)
Grant Number: 4-UH3TR002445-02
Scarring of the lung, termed pulmonary fibrosis (IPF), is a chronic, progressive, and usually fatal disorder. While two drugs have recently been approved for the treatment of pulmonary fibrosis, neither is curative and nearly 40% of patients stop taking the drug within a year because of side effects. We propose to study the use of saracatinib, a drug originally developed to treat certain types of cancers, in the treatment of pulmonary fibrosis in both preclinical models and in a Phase 1b/2a clinical trial.
Learn more about this project in the NIH RePORTER.
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2020 New Therapeutic Uses for Experimental Assets Projects |
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