| ID | Title | Body | Facebook Description | Facebook image | Facebook Title | Facebook Video | Twitter Description | Twitter Image | Twitter Title | Meta tags |
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| 13606 | 2018 CCIA Projects | A Platform Trial Design to Accelerate Translational Therapies in a Canine Disease Model of ALS Accelerate Cellular Immunotherapy Development for Treatment of Life-Threatening Childhood Disorders Effects of apoE-Enhancing Compounds on Alzheimer’s Disease Phenotypes In Vivo Harnessing Human Brain and Liver Microphysiological Systems for Testing Therapeutics for Metastatic Melanoma Harnessing the Power of CTSA-CDRN Data Networks: Using Social Determinants of Health, Frailty and Functional Status to Identify At-Risk Patients and Improve Risk Adjustment Impact of Breast Milk on Infant Gut Microbiome Increasing Access to Clinical Microbiome Specimens via a Living µBiome Bank Peer-based Retention Of people who Use Drugs in Rural Research (PROUD-R2) Precision Medicine in the Diagnosis of Genetic Disorders in Neonates TCR and BCR Deep Sequencing to Distinguish Autoimmune Recurrence from Allograft Rejection TEAMSS – Transforming Expanded Access to Maximize Support & Study Transforming Exercise Testing and Physical Activity Assessment in Children: New Approaches to Advance Clinical Translational Research in Child Health Repurposing Misoprostol to Prevent Recurrence of Clostridium difficile Infection A Platform Trial Design to Accelerate Translational Therapies in a Canine Disease Model of ALS University of Missouri Principal Investigator: Joan Ripley Coates, D.V.M., M.S., and Sarah A. Moore, D.V.M. Grant Number: 1-R21TR002277-01A1 Collaborating Institutions: The Ohio State University, Tufts University, North Carolina State University, Voyager Therapeutics Amyotrophic lateral sclerosis (ALS) is an incurable neurodegenerative disease in people. Despite best efforts, nearly all trials of new therapies have failed to improve outcomes. This project will leverage spontaneous degenerative myelopathy (DM) in dogs, a relevant model of human ALS, to interrogate and optimize a novel therapeutic approach, with the ultimate goal of markedly improving translational efficiency. Learn more about this project in the NIH RePORTER. Accelerate Cellular Immunotherapy Development for Treatment of Life-Threatening Childhood Disorders Seattle Children’s Hospital Principal Investigator: Julie R. Park, M.D., and Bonnie W. Ramsey, M.D. Grant Number: 1-U01TR002487-01 Collaborating Institutions: Children’s National Health System, UCSF Benioff Children’s Hospital, Children’s Hospital Los Angeles, University of Colorado Denver, Fred Hutchinson Cancer Research Center Cellular immunotherapies are emerging as a paradigm-shifting, personal and precise treatment for a range of fatal rare diseases in children. Early Phase I trials in refractory leukemia have provided proof of concept that this approach can be lifesaving. We propose to establish a consortium of pediatric hospitals affiliated with Clinical and Translational Science Awards (CTSA) Program hubs proposed in this application, called the Consortium for Pediatric Cellular Immunotherapy, to accelerate production and delivery of these novel lifesaving therapies to children. Learn more about this project in the NIH RePORTER. Effects of apoE-Enhancing Compounds on Alzheimer’s Disease Phenotypes In Vivo Columbia University Principal Investigators: Tae-Wan Kim, Ph.D., and John R. Cirrito, Ph.D. Grant Number: 1-R21TR002029-01A1 Collaborating Institution: Washington University in St. Louis Increasing brain levels of apolipoprotein E (apoE) is an attractive therapeutic strategy for treating Alzheimer’s disease (AD). The goal of this proposal is to determine whether pharmacological elevation of apoE levels can alleviate AD neuropathogenesis in mouse models of AD. Successful completion of our proposed studies will lead to the identification of compounds that are suitable for mechanistic study and drug discovery for AD. Learn more about this project in the NIH RePORTER. Harnessing Human Brain and Liver Microphysiological Systems for Testing Therapeutics for Metastatic Melanoma Vanderbilt University Principal Investigator: John Peter Wikswo, Ph.D., M.S., William L. Murphy, Ph.D., M.S., and D. Lansing Taylor, Ph.D. Grant Number: 1-U01TR002383-01 Collaborating Institutions: University of Wisconsin–Madison, University of Pittsburgh School of Medicine, AstraZeneca This clinical study is designed to compare the outcome from drugs chosen and not chosen by the treating physician (i.e., the patient’s own response to therapy can be measured against the drug response predicted by microphysiological system models). Successful translation of these models from the laboratory to the clinic would improve in vitro prediction of the response of patient-specific tumors. This would have a near-term impact in clinical trials of investigatory drugs and ultimately could guide the selection of optimal therapies for each patient. Learn more about this project in the NIH RePORTER. Harnessing the Power of CTSA-CDRN Data Networks: Using Social Determinants of Health, Frailty and Functional Status to Identify At-Risk Patients and Improve Risk Adjustment University of Texas Health Science Center at San Antonio Principal Investigator: Paula K. Shireman, M.D., M.S. Grant Number: 1-U01TR002393-01 Collaborating Institutions: University of Texas at Austin, University of Texas Health Science Center at Houston, University of Pittsburgh Social risk factors, including poverty, being a minority, lack of an education, and frailty, are all associated with having worse health outcomes and with more difficulties recovering after surgical procedures. Safety net hospitals take care of many people with social risk factors and are being judged on the quality of care provided without accounting for the challenges faced by poor people. We will study the effect of social risk factors and frailty on health outcomes after surgery to identify patients who would benefit from care pathways that help poor and/or frail people. Learn more about this project in the NIH RePORTER. Impact of Breast Milk on Infant Gut Microbiome University of Rochester Principal Investigator: Kirsi Järvinen-Seppo, M.D., Ph.D., and R. John Looney, M.D. Grant Number: 1-R21TR002516-01 Collaborating Institutions: University of California, San Diego, Icahn School of Medicine at Mount Sinai The gut microbiome plays a critical role in the development of the immune system, and breastfeeding is a major factor affecting infant gut microbiome composition. However, breast milk composition differs between mothers. This study will provide critical new knowledge about the impact of breast milk composition on the development of the infant gut microbiome. Research conducted in this project is used to establish maternal interventions to modify breast milk composition to favor protection against allergic and noncommunicable inflammatory diseases. Learn more about this project in the NIH RePORTER. Increasing Access to Clinical Microbiome Specimens via a Living µBiome Bank Medical University of South Carolina Principal Investigator: Alexander V. Alekseyenko, Ph.D. Grant Number: 1-R21TR002513-01 Collaborating Institutions: University of Iowa, The George Washington University The clinical enterprises generate volumes of specimens suitable for translational research, yet those are not used to their full potential. At the same time, traditional microbiology biobanking is expensive, limiting many health care organizations from having a viable biobanking solution. By leveraging widely adopted commercial electronic health record capabilities, the Living µBiome Biobank will redefine how recruitment and specimen collection is performed, by allowing for just-in-time capture of existing microbiome specimens and making them available for translational research across CTSA Program hubs. Learn more about this project in the NIH RePORTER. Peer-based Retention Of people who Use Drugs in Rural Research (PROUD-R2) Oregon Health & Science University Principal Investigator: Philip Todd Korthuis, M.D., M.P.H., and April Marie Young, Ph.D., M.P.H. Grant Number: 1-U01TR002631-01 Collaborating Institutions: University of Kentucky Research Foundation, Emory University, University of North Carolina at Chapel Hill, The Ohio State University Optimal strategies for recruiting and retaining people with opioid use disorder in clinical research in rural America remain unknown. The Peer-based Retention Of people who Use Drugs in Rural Research (PROUD-R2) study leverages the national rural opioid initiative infrastructure, with sites in rural Oregon and Appalachian Kentucky and Ohio, and partnerships with three Clinical and Translational Science Award sites and the National Institute on Drug Abuse (NIDA) Clinical Trials Network to test an innovative peer-based strategy to improve research participation and retention of people who use drugs in rural communities. Learn more about this project in the NIH RePORTER. Precision Medicine in the Diagnosis of Genetic Disorders in Neonates Tufts Medical Center Principal Investigator: Jonathan M. Davis, M.D., and Jill Lamanna Maron, M.D., M.P.H. Grant Number: 1-U01TR002271-01 Collaborating Institutions: Rady Children’s Institute for Genomic Medicine, University of North Carolina at Chapel Hill, Columbia University, Icahn School of Medicine at Mount Sinai, UPMC Children’s Hospital of Pittsburgh, University of Michigan Directed application of a rapid targeted next-generation sequencing (TNGS) technology to augment traditional clinical and diagnostic testing should improve our ability to detect underlying genetic causes and improve outcomes in our highest risk neonates. We will (1) perform a prospective study of the impact of TNGS on time to molecular diagnosis and the accuracy of this diagnosis in symptomatic neonates in the neonatal intensive care unit (NICU); (2) evaluate lengths of hospital stay and total hospital costs for high-risk neonates admitted to the NICU; (3) determine the time from initial diagnosis to initiation of an appropriate therapy, enrollment in a clinical trial or initiation of palliative care; and (4) integrate a user-friendly, electronic mechanism for accelerated TNGS result return and re-review. Learn more about this project in the NIH RePORTER. TCR and BCR Deep Sequencing to Distinguish Autoimmune Recurrence from Allograft Rejection Columbia University Health Sciences Principal Investigator: Megan Sykes, M.D. Grant Number: 1-R21TR002279-01 Collaborating Institution: University of Pennsylvania This project will attempt to distinguish rejection from recurrent autoimmune liver disease after liver transplantation by using a sequencing approach to identify and track the B- and T-cell clones that cause autoimmunity and rejection. We will thereby better understand the interplay between alloimmune and autoimmune responses causing liver transplants to fail and ultimately provide better treatment to patients needing transplants for these diseases. Success of this approach will be applicable to autoimmune diseases of other organs as well, including the kidneys, heart and lungs. Learn more about this project in the NIH RePORTER. TEAMSS – Transforming Expanded Access to Maximize Support & Study University of Michigan at Ann Arbor Principal Investigator: George Alexander Mashour, M.D., and Kevin J. Weatherwax Grant Number: 1-U01TR002488-01 Collaborating Institutions: University of Rochester, University of Texas Southwestern Medical Center, Duke University TEAMSS (Transforming Expanded Access to Maximize Support and Study) seeks to advance clinical care and translational research by improving patient access to experimental therapies through a federated, national consortium for expanded access interventions. Expanded access provides an opportunity for patients who either lack therapeutic options or are ineligible for clinical trials to potentially benefit from the clinical use of experimental drugs, biologics and medical devices. By developing this national consortium, we will build a positive impact by, for the first time, creating a foundation for an integrated, nationwide approach to expanded access that can improve care for the most vulnerable patients. Learn more about this project in the NIH RePORTER. Transforming Exercise Testing and Physical Activity Assessment in Children: New Approaches to Advance Clinical Translational Research in Child Health University of California, Irvine Principal Investigator: Dan M. Cooper, M.D., and Shlomit Radom-Aizik, Ph.D. Grant Number: 1-U01TR002004-01A1 Collaborating Institutions: Ann & Robert H. Lurie Children’s Hospital of Chicago, Children’s Hospital Los Angeles Despite the critical role played by physical activity in growth and disease prevention across the lifespan, exercise testing and physical activity assessments have failed to achieve their potential in clinical research or practice. In this project, three CTSA Program sites and two national organizations will work together to study novel approaches to laboratory exercise testing in healthy children and in challenging health conditions. The test results will be linked to metrics of habitual physical activity, inflammation and exercise-responsive gene signals. A toolkit of innovative metrics for exercise testing that can transform clinical research and practice focused on exercise and its impact on children will be made available. Learn more about this project in the NIH RePORTER. Repurposing Misoprostol to Prevent Recurrence of Clostridium difficile Infection Vanderbilt University Medical Center* Principal Investigator: David M. Aronoff, M.D. Grant Number: 5U01TR002398-02 Collaborating Institutions: Washington University in St. Louis; The University of North Carolina at Chapel Hill Clostridium difficile infection (CDI) is a leading nosocomial infection and the primary identifiable cause of antibiotic-associated diarrhea. Recurrent CDI (rCDI) complicates 20 to 30 percent of primary episodes of disease and is associated with an increased risk of death and poor quality of life. Evidence from epidemiological and animal studies suggests that the U.S. Food and Drug Administration–approved drug misoprostol (a prostaglandin analog) may prevent rCDI. This project will use a randomized, double-blind, placebo-controlled trial to assess the efficacy and safety of misoprostol in the prevention of first recurrence of CDI in adults age 50 and older. Three aims are proposed: Determine whether misoprostol modifies the rate and severity of rCDI during the first 8 weeks after standard vancomycin therapy. Determine the effect of misoprostol on the gut microbiome, diarrhea occurrence and severity, and the development of C. difficile antitoxin antibodies. Continue the clinical development of misoprostol to optimize its potential to impact clinical practice and rapidly affect human health. Learn more about this project in the NIH RePORTER. * Note: This U01 was funded by Drug Development Partnership Programs, Office of the Director, National Center for Advancing Translational Sciences. | In 2018, NCATS funded the third set of CCIA projects. | 2018 CCIA Projects | In 2018, NCATS funded the third set of CCIA projects. | 2018 CCIA Projects | ||||
| 13777 | Assay Guidance Workshop Agenda — February 2, 2019 | February 2, 2019 — 8:00 a.m.-5:00 p.m. ET Walter E. Washington Convention Center, 801 Mount Vernon Place, NW, Washington, DC 20001 8:00 a.m.-8:20 a.m.: Robust or Go Bust: An Introduction to the Assay Guidance Manual G. Sitta Sittampalam, Ph.D., NCATS, NIH 8:20 a.m.-9:05 a.m.: Key Concepts for Robust Biochemical & Cell-Based Assays Timothy L. Foley, Ph.D., Pfizer Inc. 9:10 a.m.-9:55 a.m.: Treating Cells as Reagents to Design Reproducible Screening Assays Terry Riss, Ph.D., Promega Corporation 10:00 a.m.-10:30 a.m.: Beverage Break 10:30 a.m.-11:15 a.m.: Assay Interpretation: Studies in Mechanisms and Methods in Assay Interferences Douglas Auld, Ph.D., Novartis Institutes for BioMedical Research 11:15 a.m.-12:00 p.m.: Biophysical Approaches to Small Molecule Discovery and Validation Michelle Arkin, Ph.D., University of California, San Francisco 12:00 p.m.-1:00 p.m.: Lunch 1:00 p.m.-1:45 p.m.: In Vitro Toxicological Testing Using a qHTS Platform Menghang Xia, Ph.D., NCATS, NIH 1:45 p.m.-2:30 p.m.: In Vitro Assessments of ADME Properties of Lead Compounds Xin Xu, Ph.D., NCATS, NIH 2:30 p.m.-3:00 p.m.: Beverage Break 3:00 p.m.-3:45 p.m.: Basic Assay Statistics, Data Analysis and Rules of Thumb Thomas D.Y. Chung, Ph.D., Mayo Clinic 3:50 p.m.-4:35 p.m.: System Suitability of In Vitro Screening Assays in Drug Discovery V. Devanarayan, Ph.D., Charles River Laboratories 4:40 p.m.-5:00 p.m.: Open Discussion | View the February AGM agenda | /sites/default/files/agm-cover-update_900x600.jpg | Assay Guidance Workshop Agenda — February 2, 2019 | View the February AGM agenda | /sites/default/files/agm-cover-update_900x600.jpg | Assay Guidance Workshop Agenda — February 2, 2019 | ||
| 13525 | Rare Diseases Are Not Rare! 2018 Challenge Winners | We launched our first Rare Diseases Are Not Rare! Challenge in 2018 seeking creative ways to help raise awareness about rare diseases and the need for patient support. NCATS selected 3 winners and 11 honorable mentions. View the first place entry.See details about the 2018 Challenge.First PlaceNancy Netherland “Unicorns and Super Heroes Are Rare — Rare Diseases Are Not” Second PlaceChristina Loccke, Lindsey Bergstrom, Sarah Theos “In the Land of Rare Disease”Third PlaceMatthew Beardall, Sami Assaf, Naveen Upender “Unicus”Honorable Mentions(in alphabetical order)Donna Appell “Dare to Be Rare”Richard Bauer for Complete HealthVizion “What Are the Chances?”Kelly Cullen “A ‘Not So Rare’ Instagram Story”Marissa Gaines “Rare Is Everywhere in a Genetic Garden”Karen Gunderson “Viral Rare Disease Educational Quiz”Naka King “Do You Know Rare?”Sierra Pellechio “Raising Awareness About Rare Disease Through Infographics”Ana Cristina Puhl Rubio “It Is Not Rare to Have a Rare Disease”Cassandra Sims “Rare Butterflies”Louise Mary Tiranoff, Lynne McVeigh “Rare Diseases? They Aren’t That Rare”Vanessa Vogel-Farley “I Have Some News…” | NCATS announces the winners of the Rare Diseases Are Not Rare! Challenge. | NCATS Rare Diseases Are Not Rare! 2018 Challenge Winners and Honorable | NCATS announces the winners of the Rare Diseases Are Not Rare! Challenge. | NCATS Rare Diseases Are Not Rare! 2018 Challenge Winners and Honorable | ||||
| 13546 | NCATS' Role in the NIH HEAL Initiative | #featured-stories > #secondary-image > .img-responsive { min-height: 312px; } As part of the Helping to End Addiction Long-termSM Initiative, or the NIH HEAL InitiativeSM, NCATS is applying breakthrough approaches of translational science to advance new treatments for opioid misuse and addiction and for pain. Learn more about NCATS’ role. | As part of the NIH HEAL Initiative, NCATS is applying breakthrough approaches of translational science to advance new treatments for opioid misuse and addiction and for pain. | /sites/default/files/nih-heal-graphic-horizontal_900x600_1.png | NCATS' Role in the NIH HEAL Initiative | As part of the NIH HEAL Initiative, NCATS is applying breakthrough approaches of translational science to advance new treatments for opioid misuse and addiction and for pain. | /sites/default/files/nih-heal-graphic-horizontal_900x600_1.png | NCATS' Role in the NIH HEAL Initiative | ||
| 13543 | Human Cell-Based Screening Platform Development | NCATS focuses on creating human cell-based models of opioid misuse and addiction and of pain. Through the following three initiatives, NCATS-supported researchers will develop models at the cell, tissue and integrated systems levels. The goal is to develop accurate research models that reflect the complexity of human biology and better predict how potential new drugs will affect humans. iPSC-Derived Neurons for Pain, Addiction and Overdose Scientists in NCATS’ Stem Cell Translation Laboratory (SCTL) are developing a renewable supply of human cell-based models, focusing on patient-derived induced pluripotent stem cell (iPSC) lines that give rise to specialized, functional nerve cells that are relevant for opioid misuse and addiction and for pain. NCATS offers robotic manufacturing of validated and quality-controlled human cells with the intent of making subsequent tests reproducible and reliable. These cells can be used in models to leverage diverse research projects that the Center conducts or supports. Through its Division of Preclinical Innovation, NCATS will provide access to iPSC lines, iPSC-derived cell types, protocols, cutting-edge technologies and multidisciplinary scientific expertise. 3-D Bioprinted Tissue Models of Pain, Opioid Use Disorder and Overdose for Drug Screening NCATS’ 3-D Tissue Bioprinting program experts will develop complex human, biologically relevant, 3-D tissue-in-a-well models of addiction, pain and the blood-brain barrier for high-throughput drug screening, using bioprinting techniques, iPSC-derived neurons and other cells supplied by the SCTL. Collaborators will have access to NCATS’ research resources. NCATS also supports collaborations with the external research community to develop and use 3-D biofabricated tissue models as novel drug screening platforms to advance preclinical discovery and development of non-addictive treatments for pain, opioid use disorder and overdose. Projects have two phases. During the initial phase, scientists will apply 3-D biofabrication technologies to develop novel multicellular tissue systems for drug screening, using human iPSC-derived cells representing sensory/pain neurons, brain regions, and other tissues involved in pain, addiction and overdose, including tissue models of the blood-brain barrier. In the second phase, researchers will test the new systems for drug screening. View RFA-TR-19-005 to learn more. Tissue Chips to Model Pain, Addiction and Overdose NCATS provides funding for investigators to create and test devices that can model the mechanisms or effects of pain-relevant signaling, addiction, or opioid use disorders, using human tissues in in vitro tissue- and organ-on-chip systems. Tissue chip systems are promising platforms because they can closely mimic human physiology and can model normal and diseased states. View RFA-TR-19-003 to learn more. | NCATS-supported researchers will develop human cell-based models of opioid misuse and addiction and of pain at the cell, tissue and integrated systems levels. | Human Cell-Based Screening Platform Development | NCATS-supported researchers will develop human cell-based models of opioid misuse and addiction and of pain at the cell, tissue and integrated systems levels. | Human Cell-Based Screening Platform Development | ||||
| 13540 | Accelerating the Translation of Novel Compounds Toward Investigational New Drugs (INDs) for Subsequent Clinical Testing | Using the center’s therapeutic development resources, we are working with the external research and development community and other scientists at NIH to identify and test existing and possible new drugs for preclinical development. The following three initiatives are focused on facilitating the testing needed to bring promising drug candidates to first-in-human studies. Creating Pharmacological Probes for Novel TargetsResearch experts in pain and opioid misuse and addiction have identified compounds that act on biological targets of opioids and pain and tested them in cell- and animal-based models. We provide access to resources and expertise to advance the preclinical development of these compounds and further develop them into drug-like compounds to position them for eventual testing in humans. Available collaborative resources include compound libraries, high-throughput screening, test validation, informatics tools and medicinal chemistry. The libraries will leverage repositories of natural products available through the National Cancer Institute and the Fogarty International Center. Developing Investigational Drugs Ready for Clinical TestingScientists working with NCATS’ Preclinical Therapeutic Development Branch, including those in the Therapeutics for Rare and Neglected Diseases and Bridging Interventional Development Gaps programs, are identifying and optimizing new drug candidates for opioid misuse and addiction and for pain and opioid use disorder. Researchers will determine which candidates can be further developed and tested to enable IND applications to the U.S. Food and Drug Administration and subsequent early-phase testing in humans. Available resources include medicinal chemistry, pharmacology, testing of the metabolic properties of compounds, compound safety profiles, optimal drug formulation for administration in humans, and manufacturing methods to produce sufficient quantities of potential drugs for preclinical and clinical evaluation. | NCATS scientists will work with the external research and development community, as well as with other NIH scientists, to identify and test potential new drugs for preclinical development. | Accelerating the Translation of Novel Compounds Toward INDs | NCATS scientists will work with the external research and development community, as well as with other NIH scientists, to identify and test potential new drugs for preclinical development. | Accelerating the Translation of Novel Compounds Toward INDs | ||||
| 13537 | NCATS-Supported NIH HEAL Projects | Through the NIH HEAL (Helping to End Addiction Long-termSM) Initiative, NCATS is applying breakthrough approaches of translational science to advance new treatments for the opioid crisis, the public health challenge of our era. NCATS HEAL-related projects include human cell-based testing platforms; novel drugs to treat pain, addiction, and overdose; and comparative effectiveness studies through the NIH HEAL Pain Management Effectiveness Network. Human Cell-Based Screening (HCBS) Platforms to Treat Pain, Addiction and Overdose (Daniel Soñé Photography) NCATS scientists will work with the broader scientific community to develop HCBS platforms to treat pain, addiction and overdose. Scientific teams will develop these new platforms using induced pluripotent stem cell–derived neurons, 3-D bioprinted tissue models, and tissue chip systems that model pain, addiction and overdose. Learn more about HCBS platform development. Accelerating the Translation of Novel Compounds Toward INDs for Subsequent Clinical Testing NCATS experts also will accelerate the translation of novel compounds toward investigational new drug (IND) applications needed for subsequent clinical testing. To do this, NCATS experts will work collaboratively with investigators to develop new chemical structures to modulate novel targets, create pharmacological probes for new targets, and make investigational drugs ready for clinical testing. Learn more about how NCATS is accelerating this process for subsequent clinical testing. The NIH HEAL Pain Management Effectiveness Research Network (NIH HEAL Pain Management ERN) Using NCATS’ Clinical and Translational Science Awards Trial Innovation Network, the NIH HEAL Pain Management ERN will support studies to compare the effectiveness of existing pain treatments and novel approaches to prevent and manage pain while reducing the risk of addiction. The goal is to provide clinicians with information about the effectiveness of treatments or management strategies that reduce opioid use and pain associated with many types of diseases or conditions. Learn more about how the NIH HEAL Pain Management ERN will use the Trial Innovation Network for these studies. | NCATS HEAL-related projects include human cell-based testing platforms; novel drugs to treat pain, addiction, and overdose; and comparative effectiveness studies. | NCATS-Supported NIH HEAL Projects | NCATS HEAL-related projects include human cell-based testing platforms; novel drugs to treat pain, addiction, and overdose; and comparative effectiveness studies. | NCATS-Supported NIH HEAL Projects | ||||
| 13534 | NIH HEAL Initiative Funding & Collaboration Opportunities Led by NCATS | As part of the National Institutes of Health (NIH) Helping to End Addiction Long-term® Initiative, or NIH HEAL Initiative®, we support and facilitate a variety of funding and collaboration opportunities designed to address the opioid crisis, including pain management.Current Funding OpportunitiesSee our funding page for open HEAL opportunities. (Daniel Soñé Photography)Translational Research Collaboration OpportunitiesHow do I:• Translate my animal-based research discovery into a human-based model for continued testing and development?• Advance my research on a potential therapeutic from plated cells into more complex, human cell–based models? Develop a small molecule “probe” compound that can test the therapeutic hypothesis in cell-based or animal model systems? Are these questions that you are wrestling with on your therapeutic development project? NCATS welcomes your NIH HEAL Initiative–related proposals to develop novel probes and human cell–based models in team-based research collaborations with NCATS’ intramural scientists in the Division of Preclinical Innovation. • As research collaborators, you bring a wealth of background knowledge and a starting point for a particular translational project.• NCATS scientists provide expertise and resources to transform those starting points into therapeutically useful tools, platforms or probe molecules.• Approved projects result in formation of joint project teams that will work together to design and follow milestone-driven project plans to achieve agreed-upon deliverables. Learn more about collaborating with NCATS scientists and how to submit a proposal. The NIH HEAL Pain Management Effectiveness Research Network (NIH HEAL Pain Management ERN) Using NCATS’ Clinical and Translational Science Awards Trial Innovation Network, the NIH HEAL Pain Management ERN will support studies to compare the effectiveness of existing pain treatments and novel approaches to prevent and manage pain while reducing the risk of addiction. The goal is to provide clinicians with information about the effectiveness of treatments or management strategies that reduce opioid use and pain associated with many types of diseases or conditions. Learn more about how the NIH HEAL Pain Management ERN will use the Trial Innovation Network for these studies. | NCATS supports and facilitates a variety of funding, prize and collaboration opportunities designed to address the opioid crisis, including pain management. | NIH HEAL Funding, Prize & Collaboration Information | NCATS supports and facilitates a variety of funding, prize and collaboration opportunities designed to address the opioid crisis, including pain management. | NIH HEAL Funding, Prize & Collaboration Information | ||||
| 13531 | About NCATS' Role in the NIH HEAL Initiative | NCATS is playing a major role in the National Institutes of Health (NIH) Helping to End Addiction Long-termSM Initiative, or NIH HEAL InitiativeSM, a trans-agency effort focused on improving prevention and treatment strategies for opioid misuse and addiction, and enhancing pain management. Launched in April 2018 with funding from Congress, the NIH HEAL Initiative, brings new hope for people, families, and communities affected by the national opioid public health crisis. Learn more about the NIH HEAL InitiativeSM. NCATS’ Role in the NIH HEAL Initiative With NIH HEAL Initiative support, NCATS is providing a suite of translational science resources and expertise to investigators working on opioid and pain research. With its expert collaborative infrastructure already in place, the Center is leading trans-NIH collaborative Initiative efforts that include: Developing new testing platforms that more closely model human biology than currently available cell and animal models, using induced pluripotent stem cells (iPSCs), tissue chips and 3-D tissue bioprinting; Identifying and de-risking potential therapies that work in novel ways through assays (tests) and high-throughput screening to select and develop compounds that show promise as potential drugs; Accelerating the identification of promising chemical structures and developing those into pharmacological or drug-like compounds; Advancing promising new drug candidates through rigorous preclinical efficacy and safety studies for first-in-human clinical trials as required by the U.S. Food and Drug Administration; and Facilitating opportunities to partner with other NIH Institutes and Centers as well as clinical research institutions nationwide to conduct studies to determine the effectiveness of existing pain management treatments. Through the NIH HEAL Initiative, NCATS is applying the breakthrough approaches of translational science to advance new treatments for the opioid crisis, the public health challenge of our era. HEAL Awards Issued by NCATS With NIH HEAL Initiative support, NCATS issued awards to investigators working on opioid and pain research. The programs and awards focus on ASPIRE Design Challenges to develop innovative and catalytic approaches towards solving the opioid crisis and tissue chips to model nociception, addiction and overdose. Read more about the program details and awards. HEAL New Innovator Awards Using Mouse Pain Scales to Discover Unusual Pain Sensitivity and New Pain Targets Genetic background plays an important role in how different people feel pain. To understand how genes affect pain, NCATS-funded researchers will study hundreds of mice from different genetic backgrounds. New artificial intelligence methods will be used to analyze the data gathered. The goals of this study are to find new genes involved in pain sensing and to shed light on why some people are more sensitive to pain. In the long term, this research will help in the development of non-addictive pain medications. Learn more about the project. Scale Up Single-Cell Technologies to Map Pain-Associated Genes and Cells Across the Lifespan Chronic pain can last for months or years and reduce quality of life. The causes of chronic pain are not well understood. NCATS has funded researchers to decode the genes and cells involved in chronic pain using single-cell -omics technology. Researchers will study these pathways across the life span of mice, including during pregnancy, to discover new biomarkers and treatment targets for chronic pain. Learn more about the project. Related Resources NIH HEAL Initiative Complete List of NIH HEAL Initiative Funding Opportunities Contact Steven T. Pittenger, Ph.D. | As part of the NIH HEAL Initiative, NCATS aims to facilitate and speed the process of developing and demonstrating new treatments for opioid misuse and addiction and for pain. | /sites/default/files/pain-HEAL_900x600.jpg | About NCATS’ Role in the NIH HEAL Initiative | As part of the NIH HEAL Initiative, NCATS aims to facilitate and speed the process of developing and demonstrating new treatments for opioid misuse and addiction and for pain. | /sites/default/files/pain-HEAL_900x600.jpg | About NCATS’ Role in the NIH HEAL Initiative | ||
| 13522 | Charting a New Path: NCATS Internship Helps Promising Student Soar | Translational Science Highlight NCATS mentors trainees through NIH-wide programs to help expand the pool of diverse translational scientists, including individuals from underrepresented groups and geographic areas. Training experiences at NCATS strengthen participants’ skills across research disciplines and help prepare trainees for successful scientific careers. Bryan Queme was having a bad day. The 23-year-old senior biochemistry major at Biola University in southern California had received emails from several institutions informing him that his summer internship applications had been rejected. He was disappointed, but undaunted, and began researching other opportunities. That’s when the note from NIH arrived. “I saw the email and figured that NIH had rejected me too,” Queme said. “But then I read it — and read it again. I was speechless: NIH was interested in me! I was in shock and very happy.” The email informed Queme that he had been accepted into the NIH College Summer Opportunities to Advance Research (C-SOAR) program in the Office of Intramural Training & Education. He would be matched with NCATS’ Assay Development and Screening Technology (ADST) laboratory under the mentorship of lab director James Inglese, Ph.D. Cracking the Scientific Code Patricia K. Dranchak, Ph.D., shows a lab technique to 2018 summer intern Bryan Queme. Launched in early 2018, NIH’s C-SOAR program is designed to engage a diverse group of individuals who are considering careers in the biomedical sciences. Specifically, C-SOAR seeks students who have not yet had the opportunity to participate in a research-intensive summer program, who lack opportunities to conduct research during the academic year, or who cannot explore their interests in biomedical research at their academic institutions. Erika L. Barr, Ph.D., is the director of the C-SOAR program. She said that Queme is precisely the type of scientist that C-SOAR is looking to attract. “Bryan’s an example of the importance of all students having access to opportunities,” Barr said. “He is a student who’s very focused, he’s passionate, and he’s smart and driven. He’s a high achiever.” “From the get-go, Bryan was deeply curious about science,” Inglese said. “He immediately started digging into the issues and problems that we were studying in the ADST lab. He was very tenacious to absorb as much as he could about the problems and help us find solutions.” Drawing on his strong biochemistry background and proficiency in computer science and coding, Queme designed and introduced new molecular modeling protocols to aid the NCATS lab’s scientists in understanding a new potential drug class mechanism being developed for a neglected tropical disease. “Bryan quickly saw that molecular modeling would be helpful,” Inglese explained. “He researched and downloaded the software on his own and figured out how to use it to solve the problem we were working on. He had an immediate impact on our project with his computational skills.” Reaching for the Top Queme’s journey to the NCATS lab in Rockville, Maryland, began in South Central Los Angeles, where he grew up in a single-parent household. “My mom emigrated from Guatemala when she was 20 years old and ended up in L.A.,” Queme said. “She strongly encouraged me to get good grades and go to college, but the academics at my high school weren’t really that good.” Queme’s life was forever changed in 2013 when he applied for and received a grant from the Gates Millennium Scholars (GMS) program. Funded by a grant from the Bill & Melinda Gates Foundation, GMS was established in 1999 to provide outstanding African American, American Indian/Alaska Native, Asian/Pacific Islander American and Hispanic American students with an opportunity to complete an undergraduate college education in any discipline. The grant was the only way that Queme could afford to go to college. Queme was accepted into the biochemistry program at the University of California, Los Angeles (UCLA), but his first year there presented two challenges. “I realized that UCLA wasn’t right for me,” he said. “And during that year, my mom was diagnosed with diabetes. Seeing how the doctors took care of my mother inspired me to want to help others, and so I went the pre-med route.” Queme transferred to Biola University, a small private school in nearby Orange County, where he thrived. In May 2018, Queme became the first member of his family to graduate from college. He also realized the many challenges faced by physicians in providing quality care to patients. “I thought, ‘How else can I make an impact?’ And that’s when I decided that I wanted to go into research,” he said. “I really hope that I can inspire other lower-income people and people of color to study science.” “Bryan’s experiences shaped and molded him and helped guide him on a career path,” Barr said. “He’s hungry for education, for accomplishing career goals, exploring and taking advantage of opportunities. I really respect that passion in him. He’s going to do great things.” Bryan Queme in front of a poster display that describes his summer research project with NCATS. Joining the NCATS Team Almost immediately after graduation, Queme landed on the East Coast for the first time to begin his NCATS internship. He acclimated quickly, both to the steamy summer weather of the Washington, D.C., area and to the NCATS lab, where he soon became an important member of the ADST team. “C-SOAR is an 8- to 10-week program. We know our interns will learn how to conduct rigorous science, but it is never clear how much of a contribution someone will be able to make in such a short period of time,” said Jessica M. Faupel-Badger, Ph.D., director of training and education in NCATS’ Division of Preclinical Innovation. “Bryan made a significant contribution. Everyone was delighted to have Bryan as part of the group. Everyone wanted to keep him longer.” At the conclusion of his internship, Queme headed straight to Boston to pursue a master’s degree in computer science. However, his desire for more research experience has led him back to NCATS for now. Beginning in January 2019, he will spend roughly a year working with Inglese’s team through the NIH Postbaccalaureate Intramural Research Training Award program. The opportunity will enable Queme to get further practical experience in a laboratory and continue the work in molecular modeling that he is passionate about. At the same time, he is applying to doctoral programs. “I really want to focus on computational science and on drug discovery and development,” he said. “At NCATS, I got to see real translational problems being tackled by intelligent people and saw how they approached them, how they tackled them.” Posted December 2018 | NCATS mentors students via NIH-wide training programs to strengthen their skills across research disciplines and help prepare them for scientific careers. Meet former NCATS intern Bryan Queme. | /sites/default/files/Queme-lab_1260x630.jpg | Internship Helps Student Soar | NCATS mentors students via NIH-wide training programs to strengthen their skills across research disciplines and help prepare them for scientific careers. Meet former NCATS intern Bryan Queme. | /sites/default/files/Queme-lab_1260x630.jpg | Internship Helps Student Soar |
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