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| 19509 | NCATS Director Statement in Support of Ending Structural Racism and Health Disparities | NIH established the UNITE initiative to address structural racism and promote racial equity and inclusion at NIH and within the larger biomedical research enterprise. (NIH)March 1, 2021NCATS, and I as its director, stands alongside the rest of NIH in its commitment to end structural racism in biomedical research and is fully engaged in the UNITE initiative. At NCATS, we have committed ourselves — in words and in actions — to support diversity, equity and inclusion and to address the scientific, operational, organizational and cultural problems that have contributed to racial inequities across the biomedical research enterprise.NCATS has a culture of innovation and collaboration that is necessary for advancing translational science. This culture will aid us in developing strategies that holistically and effectively address this historically difficult problem.Diversity and inclusion — in basic, preclinical, and clinical research, personalized medicine and population-level research — are also essential to addressing health equity. The choices we make regarding diseases to study, research questions to address and priorities to set will contribute to or undermine our efforts to address health equity. Issues of health care access, utilization and delivery also present opportunities to apply translational science approaches to ensure that the research we do reaches everyone who can benefit, as rapidly as possible.In addition to our active role in UNITE, NCATS is examining our existing efforts as they relate to structural racism and health disparities. We recognize that some of our own practices and approaches may, in fact, contribute to the continued marginalization of groups and inequities in health-related research and health outcomes. We are identifying new ways to incorporate diversity, equity and inclusion into all our major activities, policies and operations.We each have a role to play in ending structural racism. One way you can help is by responding to the NIH Request for Information seeking input from the public and stakeholder organizations. The RFI is open through April 9, 2021, and responses to the RFI will be made publicly available. | NCATS stands with NIH in its commitment to end structural racism in biomedical research and supports the UNITE initiative. | /sites/default/files/UNITE_Web_Badge_URL_0.jpg | NCATS Director Statement in Support of UNITE Initiative | NCATS stands with NIH in its commitment to end structural racism in biomedical research and supports the UNITE initiative. | /sites/default/files/UNITE_Web_Badge_URL_1.jpg | NCATS Director Statement in Support of UNITE Initiative | ||
| 19491 | Drug Repurposing Toolbox | In December 2019, NCATS, the U.S. Food and Drug Administration (FDA), and the Reagan-Udall Foundation held a workshop to discuss research and regulatory challenges of off-patent drug repurposing and formulate actionable solutions to those challenges. Although finding a new therapeutic use for an existing drug seems like a simple way to get more treatments to more patients more quickly, repurposing off-patent drugs generally has little potential for return on the investment made, particularly if generic forms of the drug are already on the market. As a result, despite the potential for repurposed drugs to be brought to market relatively quickly, companies have little financial incentive to invest their resources in finding new therapeutic uses for existing drugs. One recommendation that emerged from the workshop was to provide a toolbox of information for drug repurposing in general, not just focused on off-patent drugs, as a central location for people who are interested in repurposing research. Workshop participants expressed concern that many researchers may be interested in repurposing research but not know where to start, and a central hub or toolbox could serve as critically needed starting point. In response to that recommendation, NCATS established the Drug Repurposing Toolbox to provide easy access to guidance, relevant publications, information about repurposing conferences and other resources, including funding and collaboration opportunity announcements. For questions about the Drug Repurposing Toolbox, please email NewTherapeuticUses@mail.nih.gov. | NCATS’ Drug Repurposing Toolbox has publications, funding opportunities and other resources related to drug repurposing. | Drug Repurposing Toolbox | NCATS’ Drug Repurposing Toolbox has publications, funding opportunities and other resources related to drug repurposing. | Drug Repurposing Toolbox | ||||
| 19458 | NIH Reddit “Ask Me Anything” on Rare Diseases | .ama-card { box-shadow: 0 4px 8px 0 rgba(0,0,0,0.2); border: solid 1px rgba(0,0,0,0.2); border-radius: 10px; padding: 15px; padding-bottom: 5px; margin-bottom: 15px; } h3 { margin-top: 0; } On Feb. 23, 2021, NIH Director Francis S. Collins, M.D., Ph.D., NCATS Director Christopher P. Austin, M.D., and NCATS Office of Rare Diseases Research Director, Anne R. Pariser, M.D., hosted a Reddit “Ask Me Anything” (AMA) on rare diseases research and gene-based approaches to developing therapies. NIH leaders answered 23 questions about topics, such as gene therapy and gene editing, COVID-19 and rare diseases, treatment development and research support, data sharing, and approaches for translating basic research into the clinic. Read some highlights from the AMA below or see the full conversation on Reddit. Francis Collins From Lovememychem Hi, thank you so much for doing this! What do you see as the biggest barriers to developing therapies for these diseases? As a researcher in the basic sciences, my experience has been that there seems to be a considerable amount of applicable research ongoing even for rare diseases in the academic/preclinical world, but that these have not been pursued for development as therapy. Is this a sentiment you would classify as more broadly true, and if so, what are some of the policy steps that you feel can be taken to improve the situation! Again, thanks so much for doing this! From NIHGov (Francis Collins) Much has been written about the so-called “Valley of Death.” Basic science discoveries can lead to fundamental understandings of the causes of disease, but translating that into clinical benefit is a long and difficult journey. For rare diseases where the commercial benefits of a successful therapy may be insufficient to inspire private sector interest, good ideas about therapy may simply not get pursued. NIH is intensely interested in developing ways to cross this valley. One way is for NIH-supported researchers to push the research agenda further along--essentially de-risking a project which may then be appealing to a private sector partner. This is a lot of what the National Center for Advancing Translational Sciences (NCATS) does. NIH can also work with Food and Drug Administration (FDA) to identify ways to facilitate clinical developments that can utilize a template which has already been approved, so that every project doesn’t have to start from square one. We are doing that right now for gene therapy. Read more about basic science research at NIH: https://www.nih.gov/news-events/basic-research-digital-media-kit From StringofLights How do biases and inequities in healthcare affect the rare disease community in particular? For example, there are issues around access to treatments, but there are also biases that may lead to a delayed or incorrect diagnosis. What has been done to address this? Does the NIH have a role in overcoming these issues? Thank you! From NIHGov (Francis Collins) It is unfortunately true that our healthcare system is not free of bias. Rare diseases are no exception. Clearly in the United States, there are health inequities that affect certain populations’ access to healthcare. In addition, rare diseases may encounter a version of bias from providers who are simply unfamiliar with the particular condition and are therefore unprepared to offer the optimal clinical recommendations. NIH seeks to make all of its information on rare diseases accessible to patients and providers. NIH also has a major program in health disparities that aims to identify factors that contribute to bias and to test interventions to try to address those inequities. Our most important partners in addressing these problems are patients and their families, so it is a really good thing that the rare disease community is so active in this space. Christopher Austin From Adventurous-Cat-8736 Thanks for doing this AMA! Given the problems inherent in translating results from one species to another and the ethical concerns with animal research, what is NIH doing to advance non-animal research into rare diseases? From NIHGov (Christopher Austin) Several NIH Institutes and other government agencies have been working to advance non-animal research and animal alternatives for many years; see for example https://www.niehs.nih.gov/research/atniehs/dntp/assoc/niceatm/index.cfm. NCATS has been at the forefront of this work, both in its Tox21 collaboration with NIEHS/NTP, EPA and FDA and in its Tissue Chip for Drug Screening program, which has developed many human cell-based microfluidic bioreactors to mimic human responses to drugs and toxicants, and to model rare diseases and responses to therapeutics. From Batcheffect Thank you for hosting this AMA! No field depends more on equitable data sharing than rare diseases, but neither academic researchers nor private institutions (companies) have much incentive to do so. In fact, the opposite is generally true, since keeping data access exclusive ensures a competitive advantage. What can NIH/government do to further promote (enforce?) data sharing by academic and private institutions? From NIHGov (Christopher Austin) Data sharing is critical to all science, and as such NIH has recently announced an important – and more demanding – policy on sharing of data from NIH-supported research. ClinicalTrials.gov is another very important required data-sharing program. Complete, open and prompt sharing of data in an interpretable fashion is particularly critical for NCATS, because translational science is a fundamentally integrative discipline, deriving general insights from the aggregation of many individual translational research efforts. But as with so many other issues in translational science, the methods, standards and operational best practices required to efficiently produce translationally useful new insights from the aggregated data that facile sharing allows have yet to be developed and demonstrated, and are major areas of NCATS innovation. Our open informatics work in drug development (e.g., OpenData Portal) and rare diseases (e.g., GARD), the NCATS-coordinated Rare Diseases Clinical Research Network (RDCRN) Data Management and Coordinating Center, and the unprecedented National COVID Cohort Collaborative program are all examples of NCATS data sharing and dissemination initiatives that are accelerating translational discovery. Watch for my February Director’s Message, which will be posted in the next few days, on just this topic of data sharing! Anne Pariser From StringofLights Thank you for doing an AMA! How are you all doing today? Has the internet changed how the rare disease community organizes and generates support? Do you think this has had any impact on the development of treatments? Thank you! From NIHGov (Anne Pariser) The internet has opened many doors for rare disease community organizations including: 1. Bringing people together from around the world - it can lessen the isolation that many individuals with rare diseases and their families experience; 2. It provides the ability to share vetted information and best practices; 3. It gives patients and families a voice - they are able to share their experiences with a broad audience, thereby educating people about the rare disease experience; 4. It gives the groups the opportunity to address inaccurate information; 5. It provides the ability to help bring patients together to assist in recruitment efforts for clinical trials. From Stoopkid4Ever Hi! First I want to say thank you for doing this! I am signed up for Rare Across America and am attending rare disease day at NIH, so this is a fun bonus! I have idiopathic hypersomnia and there are currently no FDA approved medications for it. What do you think the answer is to advancing clinical research to understand disease and help get them under control? What can we as patients do to help this along? I am part of the CoRDS registry and have participated in every clinical research trial that has come my way, but I'm interested to know if there is more I could be doing. Thank you again! From NIHGov (Anne Pariser) I am sorry to hear of your diagnosis with idiopathic hypersomnia (IH), which is a chronic disorder that results in daytime sleepiness, unrefreshing sleep and difficulty awakening, among other symptoms. A first step for many rare diseases is to better understand the disease course through natural history studies (NHS) and registries, as you are doing. For patient groups interested in starting and conducting good quality registries and NHS, additional resources are available through NCATS’ RaDaR program. Another option is to find a patient organization for your disorder, or start a foundation or patient group if one doesn’t exist. Patient advocacy groups (PAGs) or foundations can help you to find and work with other patients and advocates to fully understand the disease, and to work together toward research and care. The NCATS Toolkit for Patient-Focused Therapy Development (Toolkit) provides a resource that describes the process for starting a patient group. Joining together with other patients to start to develop a research agenda can help to develop a priority list for next steps in a disease. Some other suggestions: Explore the NCATS Toolkit for more information on the research process and how you can start or support research on your condition. Work with larger rare disease organizations to bring attention to rare diseases, and to take part in educational programs to empower patients. Meet with the researchers conducting clinical research trials. Ask the researchers how you can contribute to research, such as helping to inform the patient community about ongoing research and research needs, and meeting the research team to help them understand your disease, among others. Consider working with researchers and clinicians to hold a scientific meeting to help you develop or organize a scientific agenda. NCATS and other Institutes/Centers (ICs) at NIH help support scientific conferences through grants. Please see NCATS’ conference grants page for more information. The primary NIH IC that works on idiopathic hypersomnia is the National Institute of Neurological Disorders and Stroke (NINDS). Please see their information page for more resources and information | NIH leaders hosted a Reddit AMA on rare diseases research leading up to Rare Disease Day at NIH 2021. | /sites/default/files/Reddit%20AMA%20Graphic%20-%20Square.png | NIH Reddit “Ask Me Anything” on Rare Diseases | NIH leaders hosted a Reddit AMA on rare diseases research leading up to Rare Disease Day at NIH 2021. | /sites/default/files/Reddit%20AMA%20Graphic%20-%20Square_0.png | NIH Reddit “Ask Me Anything” on Rare Diseases | ||
| 19296 | Pre-Application Webinar for NCATS RFA-TR-21-008, Multi-disciplinary Machine-assisted, Genomic Analysis and Clinical Approaches to Shortening the Rare Diseases Diagnostic Odyssey (UG3/UH3 Clinical Trial Optional) | Date: March 12, 2021 Time: 3:00 a.m. to 4:30 p.m. EST View slides from the webinar presentation. Read Q&As about the FOA. Purpose NCATS staff involved in this Funding Opportunity Announcement (FOA) will provide orientation and technical assistance to potential applicants to the above-referenced FOA by explaining the goals and objectives for the FOA and answer questions from webinar attendees. Participation in this webinar, although encouraged for potential applicants, is optional and is not required for the submission of an application in response to RFA-TR-21-008. Note: Questions about the scope of the FOA will be addressed during the webinar; however, questions about investigators’ specific study aims will not be addressed in the webinar. For more information, see NOT-TR-21-021. | Information about NCATS’ pre-application webinar for funding opportunity RFA-TR-21-008 | Pre-Application Webinar for NCATS RFA-TR-21-008 | Information about NCATS’ pre-application webinar for funding opportunity RFA-TR-21-008 | Pre-Application Webinar for NCATS RFA-TR-21-008 | ||||
| 19056 | 2020 CCIA Projects | Testing the Effectiveness of the Customized Career Development Platform (CCDP): A Cluster Randomized Trial Training Promotoras/Community Health Workers Using Culturally and Linguistically Appropriate Research Best Practices Empowering the Participant Voice: Collaborative Infrastructure and Validated Tools for Collecting Participant Feedback to Improve the Clinical Research Enterprise Harnessing Clinical Genomic Characterization to Accelerate Translational Advances for Patients with Intellectual and Developmental Disabilities Collaborative Care Teams for Hospitalized Patients with Opioid Use Disorders: Translating Evidence into Practice SMART IACUC: A Path to Harmonized Veterinary Multi-Site Trial Review Boston University Center for Molecular Discovery Chemical Library Consortium: Fostering Collaborations Between Chemists and Biologists for Translational Discovery Feasibility and Safety of Interleukin-1 Blockade to Treat Cardiac Sarcoidosis Gut Microbiome and Steroid Hormones Gut Microbiota in the Modulation of Outcomes After Liver Transplant Determining the Acceptability and Feasibility of Mobile-Health Approaches to Gather Clinical Information from Patients at Home Following Hospital Discharge Translating Scientific Evidence into Practice Using Digital Medicine and Electronic Patient-Reported Outcomes Testing the Effectiveness of the Customized Career Development Platform (CCDP): A Cluster Randomized Trial University of Pittsburgh Principal Investigator: Doris M. Rubio, Ph.D. Grant Number: 1R21TR003094-01 Collaborating Institutions: University of Southern California; University of Pennsylvania; Indiana University The NIH requires that all research trainees supported by NIH funds use Individualized Development Plans (IDPs) to guide their progress and productivity. IDPs enable trainees to set goals and objectives and report milestones related to their research, training and career progression. The Customized Career Development Platform (CCDP) is an online IDP that enables goal setting, tracking of career milestones and mentor-mentee interactions, and oversight by research program administrators. The cluster randomized trial aims to evaluate the effectiveness of the CCDP among programs that support NIH-funded clinical and translational science trainees across the United States. Learn more about this project in the NIH RePORTER. Training Promotoras/Community Health Workers Using Culturally and Linguistically Appropriate Research Best Practices University of Michigan Principal Investigator: Susan L. Murphy, Ph.D. Grant Number: 1U01TR003409-01 Collaborating Institutions: University of California, Davis; University of Florida Community health workers (CHWs) are increasingly being incorporated into research teams for their ability to reduce barriers in translation, particularly in regard to research on health disparities. Training for CHWs in research best practices is variable, not tailored to the research role of CHWs and not designed to train individuals who work in culturally or linguistically diverse communities. This project will develop a culturally and linguistically sensitive online research best practices course to train CHW “Champions,” demonstrate training efficacy at community sites and disseminate the course throughout the CTSA consortium. The project has the following specific aims: Develop and evaluate a research best practices online course for CHWs. Demonstrate the effectiveness of research best practices training for CHWs at community sites. Disseminate the course throughout the CTSA consortium and beyond. Learn more about this project in the NIH RePORTER. Empowering the Participant Voice: Collaborative Infrastructure and Validated Tools for Collecting Participant Feedback to Improve the Clinical Research Enterprise The Rockefeller University Principal Investigator: Rhonda G. Kost, M.D. Grant Number: 1U01TR003206-01 Collaborating Institutions: Duke University; Wake Forest University; University of Rochester; Johns Hopkins University; Vanderbilt University Partnering with research participants to understand and improve their experiences in clinical research is a high priority for research investigators, institutions and their federal sponsors. However, researchers often lack the tools, expertise and technology to easily collect the participant feedback needed to tailor studies in participant-responsive ways. The goal of this project is to develop a new, streamlined infrastructure to enable routine collection of research participants’ feedback about their research experiences using the Research Participant Perception Survey. Six Clinical and Translational Science Award (CTSA) Hubs and other stakeholders will create the infrastructure and share it with the CTSA consortium and others through the REDCap Shared Library. Learn more about this project in the NIH RePORTER. Harnessing Clinical Genomic Characterization to Accelerate Translational Advances for Patients with Intellectual and Developmental Disabilities Washington University in St. Louis Principal Investigator: John N. Constantino, M.D. Grant Number: 1U01TR002764-01A1 Collaborating Institutions: Harvard Medical School; University of Wisconsin–Madison, Children’s Research Institute; The University of North Carolina at Chapel Hill; University of California, Los Angeles; Albert Einstein College of Medicine; Vanderbilt University Medical Center; University of California, Davis; Baylor College of Medicine; University of Pennsylvania; Johns Hopkins University; University of Washington, Unprecedented advances in understanding genetic susceptibility to intellectual and developmental disabilities (IDDs) have been made throughout the last decade. Rare copy number and sequence variants are now known to account for a major share of population-attributable risk for IDDs. Clinical identification of pathogenic variants has generated opportunities to accelerate discovery and improve clinical treatment, but serious knowledge gaps persist regarding how to estimate the pathogenicity of genetic abnormalities in individual patients. This project aims to achieve the following: Establish standards for feasible neurobehavioral characterization of IDD patients. Integrate phenotypic and clinical genomic characterization of patients to directly promote progress in IDD gene and variant curation. Establish an IDD patient registry as an extension of the NCATS Center for Data to Health Initiative. Learn more about this project in the NIH RePORTER. Collaborative Care Teams for Hospitalized Patients with Opioid Use Disorders: Translating Evidence into Practice Cedars-Sinai Medical Center (University of California, Los Angeles) Principal Investigator: Itai Danovitch, M.D., M.B.A. Grant Number: 1U01TR002756-01A1 Collaborating Institutions: The University of New Mexico; Tufts University; RAND Corporation (Tufts University); Baystate Medical Center/Baystate Health (University of Massachusetts Medical School) Patients with opioid use disorder (OUD) are frequently hospitalized, and although treatment is effective, it is dramatically underutilized, leaving patients at high risk of continued misuse, future overdose and readmission. Interdisciplinary collaborative care teams (CCTs) are a new approach to address translational roadblocks in OUD treatment delivery. CCTs offer expertise that most hospital-based physicians lack, create an organized system of care and address barriers to follow-up care. A mixed- methods, multisite, randomized pragmatic trial will compare OUD patients who receive inpatient care with and without CCTs to determine whether CCTs increase translational efficiency. Learn more about this project in the NIH RePORTER. SMART IACUC: A Path to Harmonized Veterinary Multi-Site Trial Review The Ohio State University Principal Investigator: Sarah A. Moore, D.V.M., M.S. Grant Number: 1R21TR003191-01 Collaborating Institutions: Tufts University; University of Missouri; Brigham and Women’s Hospital (Harvard University) Veterinary clinical trials using spontaneous animal models of human disease accelerate successful translation and can assist in rapidly identifying ineffective treatments before progression to human clinical trials. Institutional animal care and use committees (IACUCs) review multicenter veterinary clinical trials on a site-by-site basis. Site-specific protocols for approval and monitoring make harmonizing cross-institutional efforts challenging and create inefficiencies and inconsistencies that decrease rigor and reproducibility and increase time to trial completion. This proposal aims to design and implement SMART IACUC, a cross-network platform for single review of multicenter veterinary clinical trials. SMART IACUC will harmonize veterinary clinical trial review across three partner institutions, educating and training key stakeholders to ensure broad adoption and success of the developed platform and evaluating and refining the platform using a multi-institutional test case. Learn more about this project in the NIH RePORTER. Boston University Center for Molecular Discovery Chemical Library Consortium: Fostering Collaborations Between Chemists and Biologists for Translational Discovery Boston University Principal Investigator: John A. Porco, Ph.D. Grant Number: 1U01TR002625-01A1 Collaborating Institutions: The University of North Carolina at Chapel Hill; University of California, Los Angeles; The University of Chicago; New York University, Vanderbilt University; University of Notre Dame (Indiana University); University of Massachusetts; NCATS The successful translation of a bioactive small molecule into a safe, effective therapeutic involves collaboration between chemists and biologists. The Boston University Center for Molecular Discovery (BU-CMD) is a laboratory that connects chemists who make molecules with biologists who wish to test them in various diseases. The BU-CMD curates and openly distributes a small-molecule screening collection of diverse, structurally complex chemotypes through a consortium of biological screeners. This program will leverage existing BU-CMD resources across multiple CTSA hubs, establishing an expandable molecule distribution infrastructure for seeding and fostering collaborative research projects between chemists and translational scientists to advance curative research for challenging biological disease areas. Learn more about this project in the NIH RePORTER. Feasibility and Safety of Interleukin-1 Blockade to Treat Cardiac Sarcoidosis Virginia Commonwealth University Principal Investigator: Antonio Abbate, M.D., Ph.D. Grant Number: 1R21TR003103-01 Collaborating Institutions: University of Michigan; American Heart Association Sarcoidosis is an inflammatory disease that can affect any organ system. Rarely, sarcoidosis can affect the heart, leading to life-threatening heart rhythm problems, heart failure and death. Interleukin-1 (IL-1) may play a role in granuloma formation in sarcoidosis and inflammasome formation in cardiac sarcoidosis. Clinical trials have shown that IL-1 blockers can improve outcomes in ischemic heart disease and heart failure. This pilot study seeks to evaluate whether IL-1 blockade with the IL-1 receptor antagonist anakinra can safely modulate systemic inflammation in patients with active cardiac sarcoidosis. The results will serve as proof-of-concept data to conceive future phase III studies involving CTSA hubs across the country. A novel targeted treatment could bring about a much-needed paradigm shift in the treatment of cardiac sarcoidosis. Learn more about this project in the NIH RePORTER. Gut Microbiome and Steroid Hormones Rush University Medical Center Principal Investigator: Ece A. Mutlu, M.D., M.S., M.B.A. Grant Number: 1R21TR003105-01A1 Collaborating Institutions: Northwestern University The gastrointestinal tract (GIT) microbiome plays a significant role in the bioavailability and physiological effects of steroid hormones (e.g., estrogens, progestogens, androgens) that are extensively metabolized in the GI tract. Exposure to high estrogen levels is a risk factor for breast cancer (BC), but it is not known if BC patients have alterations in GIT bacterial taxa. This translational proposal aims to characterize fecal bacterial taxa and steroid hormone levels in BC patients and to identify bacterial taxa and their candidate genes that contribute to the metabolism of steroid hormones within the GIT. Understanding which bacterial taxa play a role in GIT steroid hormone metabolism and identification of bacterial taxa and genes that are involved in steroid metabolism can potentially be used to design individualized microbiome-based therapies directed at these organisms. Learn more about this project in the NIH RePORTER. Gut Microbiota in the Modulation of Outcomes After Liver Transplant Virginia Commonwealth University Principal Investigator: Jasmohan S. Bajaj, M.D., M.S. Grant Number: 1R21TR003095-01A1 Collaborating Institutions: Columbia University Cirrhosis is a major cause of morbidity and mortality, and the only reliable cure is liver transplant (LT). However, a sizable proportion of post-LT patients develop multidrug-resistant organisms (MDROs), cognitive impairment and metabolic syndrome, which can be life threatening and result in long-term disability or incomplete recovery of pre-LT function. Some evidence indicates that altered gut microbiota play a role in post-LT complications. This proposal represents the first step toward using pre-LT microbial modulation in preventing post-LT complications, with the central hypothesis that gut microbial composition and function before LT can successfully predict post-LT outcomes. The study aims to determine the role of pre-LT gut microbial composition and function in the prediction of post-LT infections through MDRO colonization, in the development of post-LT metabolic syndrome and in cognitive recovery after LT. Learn more about this project in the NIH RePORTER. Determining the Acceptability and Feasibility of Mobile-Health Approaches to Gather Clinical Information from Patients at Home Following Hospital Discharge The University of Iowa Principal Investigator: Philip M. Polgreen, M.D., M.P.H. Grant Number: 1R21TR003410-01 Collaborating Institutions: Washington University in St. Louis The emerging ubiquity of personal computing devices provides new opportunities to collect health-related data outside traditional clinical environments. The promise of collecting meaningful data increases if survey questions can be paired with “objective” information collected from sensors capable of collecting health-related data. The CTSA hub at The University of Iowa has designed, tested and deployed custom m-Health software to aggregate information from subjects using SMS, web-based tools and mobile apps. We also have integrated information directly from sensors (e.g., step counters, blood pressure cuffs, scales, thermometers) into our custom software so that we can remotely collect objective health-related data from research participants. In this proposal, we will use our m-Health platform to gather clinical data from patients in their homes following discharge from the hospital on outpatient parenteral antimicrobial therapy (OPAT). The study will achieve the following: Assess the feasibility of using a mobile health approach to gather clinical data from patients in their homes following discharge from the hospital on OPAT. Determine patient and researcher perspectives regarding the utility of our approach. Estimate the sample size necessary to power a study to determine the utility of remotely collected patient-reported data for predicting hospital readmissions among patients discharged on OPAT. Learn more about this project in the NIH RePORTER. Translating Scientific Evidence into Practice Using Digital Medicine and Electronic Patient-Reported Outcomes Icahn School of Medicine at Mount Sinai Principal Investigator: Ashish Atreja, M.D., M.P.H. Grant Number: 1U01TR002997-01A1 Collaborating Institutions: Northwestern University; Cleveland Clinic Lerner College of Medicine (Case Western Reserve University) Delivery of health care traditionally has been limited to in-person office visits or hospitalizations, although patients spend the majority of their time at home or work. Digital medicine (e.g., apps, remote monitoring, telemedicine, patient-reported outcomes) has the potential to bridge this gap, but it is unclear how to implement it as a mainstream clinical practice that can lead to high-level patient and provider adoption. Through the creation of a Digital Transformation Network for inflammatory bowel disease, we plan to reduce digital disparities and scientifically address the evidence gap of digital health interventions across populations and communities. We hope that this study will help us build an evidence-based approach to determine whether digital medicine can engage a diverse group of patients and improve outcomes and, if it can, how it can be reproduced and replicated across different settings to address the T3 and T4 translational gaps. Learn more about this project in the NIH RePORTER. | 2020 CCIA Projects | 2020 CCIA Projects | ||||||
| 18894 | National Network Accelerates COVID-19 Clinical Research | .main-content #body-content a{ font-weight:400; text-decoration-color: #809aca; } .main-content #body-content a.action, .main-content #body-content p.action{ font-weight: bold; color: #30787D; font-size: 14px; text-decoration-color:#30787D; } .main-content #body-content h2{ color: #30787D; font-size: 28px; } .intro-block { background-color: #E3EBED; padding: 20px; } .outro-block { background-color: #E3EBED; padding: 20px; margin-top: 20px; } .caption-right-sm { width:100%; margin: 2%; border: none; background-color: #E3EBED; border-bottom: 4px solid #30787D; } .caption-text { padding: 2% 2% 0 2%; } .main-content #body-content .caption-text p{ line-height: 1.2; } .image-left{ background-color: #E3EBED; margin: 20px 0 0 0; } .image-left .col-lg-5 { padding: 0; } .image-text{ padding: 4% 3%; } section{ width: 100%; clear: both; } .modal-image-button { margin-left: 20px; margin-bottom: 15px; width: 20%; float: right; } .modal-image-button > input { border: 4px solid #30787D; position: relative; width: 100%; } @media (max-width: 1199px) { .modal-image-button { margin-left: 15px; margin-bottom: 15px; width: 21%; } } @media (max-width: 992px) { .modal-image-button { margin-left: 15px; margin-bottom: 15px; width: 23%; } } @media (min-width: 992px) { .caption-right-sm { float: right; width: 30%; margin: 0% 0% 2% 3%; } .image-left{ border-right: 4px solid #30787D; } } @media (max-width: 750px) { .modal-image-button { margin-left: 15px; margin-bottom: 15px; width: 150px; } } CTIV-1 Clinical Trial FlyerCloseDownload PDFRunning multisite clinical trials is complicated, expensive and time consuming. We are making the process more efficient for two adaptive Phase 3 clinical trials by using the existing clinical research infrastructure and joint networks of its CTSA Program. Both trials are part of the Accelerating COVID 19 Therapeutic Interventions and Vaccines (ACTIV) public-private partnership. public-private partnership. They seek to evaluate the safety and efficacy of drugs to treat COVID 19 but focus on different approaches and levels of disease severity.• The ACTIV-1 clinical trial tested three immune modulator drugs that help reduce the effects of an overactive immune response to determine whether they could speed up recovery and reduce deaths in adults hospitalized with moderate to severe COVID-19. Researchers found that two of the three drugs, infliximab and abatacept, substantially improved clinical status and reduced deaths, although they did not significantly shorten time to recovery. The CTSA Program — with its extensive capacity and broad geographical reach — played a key role in adding U.S. study sites and enrolling patients. On July 10, 2023, JAMA published the study on abatacept and infliximab.Learn more about the preliminary results from the ACTIV-1 trial, which launched in October 2020.• The ACTIV-6 trial is testing several existing drugs — an approach called drug repurposing — to see whether they can provide safe, effective symptom relief and prevent hospitalization in people with mild-to-moderate COVID 19. Two CTSA Program hubs serve as coordinating centers and partner with the Patient-Centered Outcomes Research Institute to expedite enrollment. ACTIV-6 is testing the safety and effectiveness of several drugs, including ivermectin, fluvoxamine and fluticasone, in treating mild to moderate COVID-19 symptoms at home. Results of the ACTIV-6 clinical trial are available. Read the ivermectin 400, ivermectin 600 and fluvoxamine peer-reviewed publications and the preprint for fluticasone.Learn more about the ACTIV-6 trial, which was announced in May 2021.Innovative Clinical Trials Are Delivering Answers on Convalescent Plasma’s Effectiveness as a COVID-19 TherapyThe CTSA Program played a key role in clinical trials that aim to determine whether convalescent plasma is a viable therapy for COVID-19. CTSA Program institutions rapidly expanded enrollment and accelerated data analysis in two randomized, placebo-controlled trials, CONTAIN COVID-19 and PassItOn, evaluating convalescent plasma as a treatment for people hospitalized with COVID-19. These studies show a nimble and coordinated response to bring safe, effective COVID-19 therapies to patients sooner.Read more about the CONTAIN COVID-19 and PassItOn clinical trials, which launched in September 2020.• The CONTAIN COVID-19 trial showed that for people hospitalized with COVID-19, convalescent plasma taken from those who had recovered was not more effective than placebo in delivering clinical improvement 14 days and 28 days after treatment began. Read a statement about the results published in January 2022. • Results from PassItOn showed that among adults hospitalized with COVID-19, participants who received convalescent plasma had nearly identical clinical outcomes as participants who received the placebo at 28 days following treatment. Read the results of the trial published in June 2022. Leveraging Established Partnerships Accelerates Startup of Early COVID‑19 StudiesAs the COVID 19 pandemic rapidly emerged, our CTSA Program stepped up to ensure effective support and rapid implementation of clinical research studies aimed at treating and understanding various aspects of COVID 19. For example, in March 2020, a CTSA Program collaboration between the Indiana Clinical and Translational Sciences Institute (CTSI) and the South Carolina Clinical & Translational Research (SCTR) Institute resulted in a study to determine whether health care providers at a hospital have been exposed to the virus that causes COVID 19. The SCTR launched a fast-track approval process, refined the protocol and implemented the study within 15 days.Read more about the collaboration.Illuminating the Body’s Response to SARS-CoV-2 InfectionUnderstanding how the immune system responds to SARS-CoV-2, the virus that causes COVID 19, could allow clinicians to use the patient’s stage of infection to inform treatment selections and better tailor patient therapies. CTSA Program–funded scientists in Colorado mapped how the human immune system shifts its approach over the course of infection by SARS-CoV-2. Led by scientists at the University of Colorado Anschutz Medical Campus, Aurora, the research team examined the immune system’s molecular mechanisms in response to SARS-CoV-2 infection. In a study published in March 2021, they tracked the body’s response to infection in hospitalized COVID 19 patients. The researchers analyzed which immune system cells were more prevalent or less prevalent during each stage of infection. The resulting biosignature data could be used to help determine the optimal treatment approach for patients hospitalized with COVID 19.Read more about this research.Sharing Best Practices and Lessons Learned for Addressing COVID-19 in ResearchTThe Journal of Clinical and Translational Science published a special issue in June 2021 titled “Re-engineering the Clinical Research Enterprise in Response to COVID-19: The CTSA Experience,” which explores the many ways the CTSA Program pivoted to address COVID-19. The articles in this issue touch on many of the program focus areas and illuminate how the supported hubs enhance translation and catalyze innovation. COVID-19 created a new clinical and translational research landscape, and the efforts highlighted in this journal issue show how researchers can continue to address COVID-19 and future public health emergencies.Read more about this special issue. | NCATS collaborative networks are enabling the rapid launch of clinical studies for COVID-19 | /sites/default/files/virus_landing_900x600_2.jpg | NCATS’ collaborative networks accelerate COVID-19 clinical research | NCATS collaborative networks are enabling the rapid launch of clinical studies for COVID-19 | /sites/default/files/virus_landing_900x600_3.jpg | NCATS’ collaborative networks accelerate COVID-19 clinical research | ||
| 18897 | Translational Science Resources | .text-box { background-color: #E3EBED; padding: 20px; } .text-box a { font-weight: normal; } .text-box .action { font-weight: bold; color: #30787D; font-size: 15px; text-decoration-color:#30787D; } New treatments take far too long to develop, require an average of 10–15 years and fail 95 percent of the time. This infographic shows how translational science is improving the process to get more treatments to more patients more quickly. Click the image below to download. Credit: National Center for Advancing Translational Sciences Download the 508-compliant infographic (PDF - 105.8 KB) Learn more about translational science: Drug Discovery, Development and Deployment Maps Infographics Issues in Translation Translational Science Skills Translational Science Spectrum Transforming Translational Science (PDF - 620.6KB) Articles about translation: Opportunities and Challenges in Translational Science Translational Misconceptions Translating Translation | Translational science is working to improve the research process to get more treatments to more people more quickly. | /sites/default/files/NCATS_TS_Infographic_top_section_thumbnail_0.jpg | Translational Science Resources | Translational science is working to improve the research process to get more treatments to more people more quickly. | /sites/default/files/NCATS_TS_Infographic_top_section_thumbnail_1.jpg | Translational Science Resources | ||
| 18795 | NCATS ASPIRE Laboratory | @media only screen and (min-width: 768px) { .aspire-graphic-concept-desktop { width: 55%; float: right; padding: 15px 0 5px 15px; } .aspire-graphic-concept-mobile, .aspire-graphic-concept-mobile-p { display: none; } } @media only screen and (max-width: 767px) { .aspire-graphic-concept-desktop, .aspire-graphic-concept-desktop-p { display: none; } .aspire-graphic-concept-mobile { width: 100%; } } Vision for ASPIREThe overall goal of ASPIRE is to allow real-time translational science by combining expertise in chemistry, biology, pharmacology, automation and AI/ML to provide a rapid decision-making platform to advance lead molecules to the clinic. More information about the capabilities of the intramural laboratory will be added in the coming months.Learn more about ASPIRE capabilities in the following areas.BiologyThe goal of ASPIRE’s biology component is to form a biological activity profile library of molecules in near real time to identify novel, next-generation molecules with drug-like properties. Early profiling will include solubility, biochemical and cellular activity using pharmacologically relevant assay reagents and assay platforms. Additional testing will include target engagement, biophysical properties, in vitro PK/PD/toxicology and proven biological activities in human-induced pluripotent stem cell (hiPSC)–derived 3D printed tissues or organoids as necessary for molecular scaffolds of interest. A key goal of the biology component is the quick turnaround of test data and biological annotations.ChemistryThe chemical synthesis component of ASPIRE will focus on developing core expertise and technologies involved in reaction screening, which automatically will be translated directly into successful reaction batch production of synthetic targets. A key aim is to push the entire synthesis workflow from design to biological evaluation, with minimal human intervention, through use of strategic protocol standardization parameters. The goal is to begin innovative technologies that speed up researchers’ ability to advance chemical synthesis automatically and to allow researchers to focus entirely on higher-order intellectual activities that lead to key discoveries and hypothesis generation.InformaticsInformatics research in ASPIRE is focused on creating a “chemical intelligence” powered integrated computational platform to drive the autonomous design and synthesis of novel therapeutics and the exploration of unknown chemical space and knowledge. To accomplish this goal, the ASPIRE initiative is building a high-quality reaction knowledgebase via the integration of historical and high-throughput synthesis data. With the help of AI/ML methods — including network analysis, traditional machine learning and deep learning techniques — ASPIRE aims to develop novel computational methods for the design and synthesis of novel bioactive molecules of therapeutic potential. The methods will form an automated computational pipeline addressing molecular modeling, retrosynthesis planning, reaction execution, and optimization of reaction conditions and the properties of the target molecules.ASPIRE prioritizes the early dissemination of novel reaction informatics methods and potentially public data sets. In the future, ASPIRE will host a collection of source code repositories and computational tools for both NCATS and extramural researchers to use.CollaborationCollaborations are critical to the successful implementation of ASPIRE. The project intends to develop and share tools, technologies and standardized protocols in automation, chemistry, biology, pharmacology and informatics — both internally and in partnership with extramural researchers. These assets will be openly shared with appropriate protections for intellectual property rights for registered institutions that are willing to share their research.Partnerships can be managed with Confidentiality Agreements (CDA), Research Collaboration Agreements (RCA) and other instruments compatible with individual research institutions.AutomationOne of the aspects of automation that is most appreciated, particularly in the life sciences, is its ability to relieve researchers of routine tasks that can be readily developed into well-orchestrated processes run by robots. In addition to allowing scientists more time and opportunity to pursue innovative activities, automation can lead to better reproducibility, which, in turn, leads to more rapid and convincing discoveries. We have extensive experience in robotically executed biological assays and aims to use some of that expertise to improve its ability to synthesize molecules with minimal human involvement, which is a challenge when the goal is to integrate other key aspects of molecular synthesis as well, such as purification, characterization and plating for screening.InfrastructureWe have thought a lot about how to develop a proper infrastructure for ASPIRE. One key infrastructure objective is space, and we recently secured the renovation of a 4,400 ft2 open laboratory space to house ASPIRE and supporting technologies. We are designing the space with an eye toward the flexibility, portability and evolution of technology solutions to keep pace with the rapidly changing scientific discovery and drug development landscape. Another key infrastructure objective involves developing hardware and software frameworks that can be used as work process standards, which will lead to lower costs in the development, operation and duplication of the technology elsewhere. Our ultimate goal is to promote the development of platforms similar to ASPIRE to improve transdisciplinary partnership and speed up the discovery of innovative and effective treatments. | NCATS’ ASPIRE aims to enable real-time translational science by combining expertise in chemistry, biology, pharmacology, automation and AI/ML. | NCATS ASPIRE Laboratory | NCATS’ ASPIRE aims to enable real-time translational science by combining expertise in chemistry, biology, pharmacology, automation and AI/ML. | NCATS ASPIRE Laboratory | ||||
| 8088 | Center Overview | Discover more about NCATS, its director and staff, and job and training opportunities. | ||||||||
| 18678 | PaVe-GT: Collaborative NIH Effort Aimed at Creating a Gene Therapy Playbook, Making Rare Disease Treatments More Accessible | PaVe-GT is aimed at streamlining the development of gene therapies for rare diseases. Here, an adeno-associated virus carries a gene into a cell. (Darryl Leja, NHGRI)November 30, 2020New project will test whether using the same gene therapy delivery system and manufacturing methods can speed rare disease clinical trialsA new NIH initiative spearheaded by NCATS aims to make gene therapy development and clinical testing more streamlined and less expensive — and potentially more accessible to millions of people with rare diseases.Only about 5% of the approximately 7,000 rare diseases have a treatment approved by the U.S. Food and Drug Administration (FDA). Most of these disorders are caused by a defect in a single gene, meaning many potentially can be treated by gene therapy. Gene therapy entails replacing a malfunctioning gene with a working version.New technologies — including the use of a modified virus to deliver genes to cells that need properly functioning genes — are making gene therapy an increasingly attractive treatment option for individuals with rare diseases. Yet, thousands of disorders are so rare that companies may be reluctant or unable to invest the years of research and millions of dollars necessary to develop, test and bring a gene therapy for one disease to market.NCATS, in conjunction with NIH’s National Human Genome Research Institute (NHGRI), National Institute of Neurological Disorders and Stroke (NINDS), and Eunice Kennedy Shriver National Institute of Child Health and Human Development, recently launched the Platform Vector Gene Therapy (PaVe-GT) initiative to test whether it is feasible to use the same gene delivery system and manufacturing methods for multiple rare diseases in gene therapy clinical trials. Using a common delivery system and manufacturing method makes gene therapy for rare diseases more accessible because the costs for therapeutic development and testing are shared.“We’re pretty good at preventing, treating and even curing single-gene diseases in animal models, but very few candidate gene therapies get to clinical trials, often because of the cost and the complexity of developing a clinical trial for a single disease,” said P.J. Brooks, Ph.D., a program officer in the NCATS Office of Rare Diseases Research and one of the leaders of the initiative. “Most rare diseases are not commercially viable for companies to invest in, and patients have to find other resources.”For scientists to make a dent in treating thousands of rare diseases, Brooks said, “We have to take advantage of relative commonalities among diseases and adopt a many-diseases-at-a-time approach.”In a recent publication in Human Gene Therapy, Brooks and PaVe-GT colleagues described the make-up of the pilot project. PaVe-GT researchers will use a common gene delivery vehicle, adeno-associated virus (AAV), for gene therapy to treat four rare genetic diseases. They plan to use the same AAV delivery system to carry a therapeutic gene to the right place in the body but switch the cargo — the gene — for each disease.Each disease currently is being studied at the NIH Clinical Center. They include two inherited muscle weakness/neuromuscular junction disorders (Dok7 deficiency and Collagen Q deficiency) and two inherited metabolic diseases (propionic acidemia and isolated methylmalonic acidemia). The neuromuscular junction is the site where a nerve fiber and a muscle cell chemically communicate. “While we’re making progress against these disorders that we’ve been studying for years, we also hope we can improve the efficiency of the gene therapy clinical trial process and develop a blueprint for other similar rare disease gene therapy projects,” said NHGRI geneticist and study leader Charles Venditti, M.D., Ph.D.PaVe-GT researchers also suggest that many preclinical studies, such as those that examine the toxicity and distribution of an investigational therapy in the body, could prove useful for multiple disorders, further streamlining gene therapy development.Transparency mattersTo enable others to understand and learn from the scientific and procedural progress of PaVe-GT, scientists will share their data publicly. They will work closely with FDA officials to chart the progress on preclinical studies, including the safety, toxicity and distribution of an investigational therapy in the body. PaVe-GT researchers and their FDA colleagues will discuss such project aspects as gene manufacturing, gene delivery and clinical trial design. These conversations and subsequent FDA feedback — normally considered proprietary by companies — will be available to the public on the PaVe-GT website as well.“Some companies are already using similar approaches to study groups of diseases, but certainly not in such a systematic and transparent way,” said Carsten Bönnemann, M.D., an NINDS pediatric neurologist and neurogeneticist who leads two of the studies. “Our goal is to show the feasibility of and the potential roadblocks — as well as opportunities — in an approach to treating more than one disorder at a time. People can track the entire gene therapy development process, from the preclinical studies in the lab to a clinical trial.”“Most smaller companies would never have access to these types of studies and documentation that go into developing a gene therapy,” said Venditti. “We’re hoping this information is helpful to rare disease organizations as well, which in some cases are attempting to study a disease on their own. Perhaps our results will encourage these organizations and academic investigators to go after diseases that really need new treatments.”One such disease foundation is Cure Raghav, which is led by Sanath Ramesh, a software engineer in Seattle. His son, Raghav, has an ultra-rare, fatal disease. After working with various clinical and scientific experts and exploring treatment options for his son, Ramesh turned his attention to gene therapy as a long-term solution. “I quickly realized the financial and logistical hurdles, as well as the lack of time and resources, that make this so difficult,” he said. PaVe-GT, he noted, “is an important step to meeting the needs of the rare disease community.”“PaVe-GT is akin to creating a generic gene therapy,” said Donald Lo, Ph.D., an NCATS translational scientist and senior author on the Human Gene Therapy paper. “Rare disease groups and scientists will be able to use this playbook and adapt it to their needs, avoiding having to raise the many millions of dollars it currently takes to access proprietary gene therapy technologies. If it works, it potentially will be transformational for the field.” | The PaVe-GT initiative aims to make gene therapy and clinical testing more accessible to people with rare diseases. | /sites/default/files/PaVe-GT%20NHGRI%20NPC%20Gene_900x600px_0.jpg | PaVe-GT: Making Rare Disease Treatments More Accessible | The PaVe-GT initiative aims to make gene therapy and clinical testing more accessible to people with rare diseases. | /sites/default/files/PaVe-GT%20NHGRI%20NPC%20Gene_900x600px_1.jpg | PaVe-GT: Making Rare Disease Treatments More Accessible |
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