| ID | Title | Body | Facebook Description | Facebook image | Facebook Title | Facebook Video | Twitter Description | Twitter Image | Twitter Title | Meta tags |
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| 9778 | Matrix Screening Projects | NCATS experts are using a technology called matrix combination screening, which quickly narrows down a long list of potential drug combinations to find those with the most potential to help patients. The matrix screening approach uses NCATS’ high-throughput robotic screening platform to quickly conduct millions of tests to assess the effects of a combination of therapeutic compounds on cellular, molecular or biochemical processes that are relevant to a disease of interest. Explore selected matrix screening projects below: Development of Novel Chemical Genomic Platforms for Pathogenic Protozoa High-Throughput Combinatorial Screening Identifies Drugs that Cooperate with Ibrutinib to Kill Activated B-Cell-Like Diffuse Large B-Cell Lymphoma Cells High-Throughput Drug Screening Identifies Novel Therapeutic Options for BPDCN Identification of a Synergistic Combination Between PARP Inhibitors and NAMPT Inhibitors in Ewing Sarcoma mQC: A Quality Control Metric for High-Throughput Combination Screening PAX3-FOXO1 Establishes Myogenic Super Enhancers and Confers BET Bromodomain Vulnerability | Matrix Screening Projects | Matrix Screening Projects | ||||||
| 9675 | Through Externship, CTSA Program Scholar Learns New Approaches to Drive Her Research Forward | Taverna presented on her externship project to NCATS staff and leadership in February 2018. From left to right: Christopher P. Austin, M.D., director of NCATS, Joan Davis Nagel, M.D., M.P.H., medical officer in the Division of Clinical Innovation (DCI), Danise Subramaniam, Ph.D., senior director of global regulatory affairs at Eli Lilly, Josephine Taverna, M.D., and Michael G. Kurilla, M.D., Ph.D., director of NCATS’ DCI. In July 2017, Josephine Taverna, M.D., had just accepted a position as assistant professor of hematology and oncology at the University of Texas Health Science Center at San Antonio and was about to start a clinical research study on breast cancer. Supported in part by a Clinical and Translational Science Awards (CTSA) Program Mentored Clinical Research Scholar (KL2) award from the university’s Institute for Integration of Medicine and Science, her career as a clinical investigator was right on track. So she had a moment’s pause when she applied for and was offered an NCATS-Eli Lilly and Company externship. The externship program pairs CTSA Program scholars and trainees with a mentor at the pharmaceutical company, where they are fully embedded in a project team to enhance academic translational science researchers’ skill sets in drug development. It was a great opportunity for Taverna, but it also meant putting her KL2 research on hold and moving her family to Lilly’s headquarters in Indianapolis for six months. “I just jumped,” Taverna said, recalling her decision. “I have no regrets. It was a phenomenal experience working with and learning from talented researchers across the translational science spectrum at Lilly.” Now in its third year, the NCATS-Lilly externship program is intended to enhance translational scientists’ skills in areas critical to the drug development process, from clinical trial design and disease modeling to regulatory affairs. For Taverna, there was another moment’s pause when her mentors at Lilly encouraged her to take on a computer modeling project on Alzheimer’s disease, both of which were outside of her formal training. Taverna with her Eli Lilly mentoring team. From left to right: Evan Wang, Ph.D., Nieves Velez de Mendizabal, Ph.D., Brian A. Willis, Ph.D., Josephine Taverna, M.D., and Mansuo L. Hayashi, Ph.D. Over the course of the training, Taverna reviewed published research and worked with her team to develop a computer model simulating how tau, a key protein in the brain, behaves — and misbehaves — in people with Alzheimer’s disease. The technique, called quantitative systems pharmacology (QSP), uses large amounts of data from cell and animal studies and from patients to build a mathematical model of a disease system. Once researchers complete a computer model, they can use it to show the effects of using a drug to target a specific part of the disease system. The goal is to find an effective therapy for Alzheimer’s disease, a condition for which there has been little progress, despite decades of traditional drug discovery approaches. Taverna presented her work to NCATS leadership and staff in February 2018. “Working with Dr. Taverna was a great experience,” said Brian Willis, Ph.D., principal research scientist at Lilly. “She quickly came up to speed in the area of research and was able to make a real impact on our project.” Now, as Taverna returns to her patients and research in San Antonio, she hopes to use QSP to improve clinical trials for cancer. She thinks modeling will be especially useful for identifying which patients are most likely to respond to a given therapy and in supporting researchers’ efforts to predict the best drug dose to start with in early clinical trials. She will need to collaborate with people from many different fields to model complex disease systems. “My experience at Lilly emboldened me to make the connections I need to drive the science forward,” she said, adding that she is well positioned as a scholar in the CTSA Program to form such a multidisciplinary team. Taverna believes that seizing the opportunity was the right decision for her career and that it exposed her to new ideas and approaches that she hopes will help get new therapies to her patients faster. Watch a video of Taverna discussing her research and her externship experience at Lilly, supported through the CTSA Program: Posted March 2018 | Through the NCATS-Eli Lilly externship, CTSA Program Scholar Josephine Taverna, M.D., learns new approaches to drive her research forward. | /sites/default/files/eli_lily_extern1_1260x630.jpg | CTSA Program Scholar Learns New Research Approaches Through Externship | Through the NCATS-Eli Lilly externship, CTSA Program Scholar Josephine Taverna, M.D., learns new approaches to drive her research forward. | /sites/default/files/eli_lily_extern1_1260x630.jpg | CTSA Program Scholar Learns New Research Approaches Through Externship | ||
| 9659 | Novel Treatment for Hermansky-Pudlak Syndrome Pulmonary Fibrosis | Hermansky-Pudlak syndrome (HPS) is a rare genetic disorder that can result in a wide range of symptoms, depending upon which genetic mutation a patient inherits. In three of the 10 forms of HPS, patients develop pulmonary fibrosis (PF). Tissues deep in the lungs become scarred, interfering with the ability to breathe. PF is a leading cause of premature death in adults with HPS, and there are no treatments approved by the Food and Drug Administration (FDA) for HPS-PF. Scientists from the National Institute on Alcohol Abuse and Alcoholism (NIAAA) and the National Human Genome Research Institute (NHGRI) jointly identified that increased activity of two biological pathways contributes to the development of lung fibrosis in HPS-PF. The lead collaborators discovered that these two pathways can be blocked by a single drug molecule, offering protection against lung fibrosis in animal models of HPS. This project further developed this drug candidate for clinical testing in patients. Scientific Synopsis Hermansky-Pudlak syndrome is a rare genetic disorder primarily of lysosome-related organelle biogenesis. There are 10 different types of HPS, depending on which genetic mutation is inherited. Patients with HPS-1, HPS-2 or HPS-4 develop pulmonary fibrosis. In the absence of a Food and Drug Administration (FDA)-approved therapy for HPS-PF, there is an urgent need to identify new therapeutic targets and treatment strategies. It has been shown that the cannabinoid-1 receptor (CB1R) is overactivated in fibrotic lung tissue of mice and humans with HPS. In previous studies, rimonabant, a CB1R antagonist, demonstrated a modest ability to mitigate fibrosis in animal models. However, the neuropsychiatric side effects of CB1R inhibitors, through the blockade of receptors expressed in the central nervous system (CNS), led to rimonabant being withdrawn from all clinical use. In addition to CB1R, the activity of inducible nitric oxide synthase (iNOS) is also increased in PF, promoting lung inflammation and progression of fibrosis. Because the pathogenesis of HPS-PF is complex, targeting multiple pathways has been recommended as an approach to improve therapeutic efficacy. To target these independent drivers of PF simultaneously in HPS, the novel drug candidate MRI-1867 was designed as a dual inhibitor of both CB1R and iNOS. To avoid the CNS side effects of the first-generation CB1R antagonists, MRI-1867 was designed to be restricted to the peripheral tissues and specifically excluded from the brain. MRI-1867 is orally bioavailable, and in chronic treatment in animal models of PF it provided increased antifibrotic efficacy compared with targeting either CB1R or iNOS alone. Lead Collaborator National Institute on Alcohol Abuse and Alcoholism, Bethesda, MD Resat Cinar, Pharm., Ph.D. George Kunos, M.D., Ph.D. Malliga R. Iyer, Ph.D. Public Health Impact Fibrosis is a condition characterized by the thickening and scarring of connective tissue. It can occur as a result of tissue repair after an injury or in response to a continuous reactive process, such as chronic inflammation. MRI-1867 represents a potential platform treatment, including conditions of unknown causes in which fibrosis is the primary driver of morbidity or mortality. Outcomes The TRND project team developed a comprehensive project plan to advance MRI-1867 toward clinical testing. TRND scientists developed a synthetic process and a suitable formulation for manufacturing MRI-1867; developed and validated the analytical and bioanalytical assays necessary to evaluate the drug product; and conducted preclinical pharmacokinetics, safety and toxicology studies. After completion of these initial studies, the lead collaborator was able to take full control of the further development of MRI-1867 through a licensing agreement with Inversago Pharma. A Canadian CTA was cleared, enabling MRI-1867 (as INV-101) to begin Phase 1 studies. | Scientists from the NIAAA and the NHGRI work together on this project to further develop a drug candidate for clinical testing in patients. | Novel Treatment for Hermansky-Pudlak Syndrome Pulmonary Fibrosis | Scientists from the NIAAA and the NHGRI work together on this project to further develop a drug candidate for clinical testing in patients. | Novel Treatment for Hermansky-Pudlak Syndrome Pulmonary Fibrosis | ||||
| 9592 | Biomedical Research and Public Health Collaborations with NASA | NCATS is the home for collaborations on biomedical research and public health with the National Aeronautics and Space Administration (NASA). NCATS coordinates, fosters and develops research relationships that advance the goals of NIH, the broader U.S. Department of Health and Human Services (HHS) and NASA for the broad benefit of human health.For more information, email Danilo A. Tagle, Ph.D., NIH-NASA liaison point of contact.Read about why we would consider doing space-based biomedical research from former NCATS Director Christopher P. Austin.NIH-NASA CollaborationNIH and NASA have an ongoing collaboration to explore how space and Earth-based biomedical research can benefit human health here on Earth as well as address the challenges of health in low Earth orbit (LEO) and during deep space exploration missions. The collaboration encourages space-related biomedical research through the exchange of expertise, data, and scientific and technical information with NASA’s Division of Biological and Physical Sciences (BPS).In 2017, former NIH Director Francis S. Collins, M.D., Ph.D., and former NASA Deputy Administrator Dava J. Newman, Ph.D., signed a second NIH-NASA Memorandum of Understanding (MOU) (PDF - 410KB). The MOU enabled NIH and NASA to develop processes by which NIH grantees could access the International Space Station U.S. National Laboratory, as well as NASA facilities, for biomedical research projects designed to improve human health on Earth. As outlined in the MOU, NIH and NASA efforts include establishing a framework of cooperation to encourage interaction between NIH and NASA research communities and integrating results from that research into improved understanding of human physiology and health.In early 2018, Collins appointed NCATS’ director, Christopher P. Austin, M.D., as the new NIH liaison to NASA. This role was previously held by the directors of NIH’s National Institute of Arthritis and Musculoskeletal and Skin Diseases and NIH’s National Institute of Biomedical Imaging and Bioengineering.An integral part of this NIH-NASA collaboration includes the representation from over 20 of the 27 NIH institutes and centers on the NIH-NASA Scientific Potential/Actual Collaborative Efforts (SPACE) group.In December 2018, Deputy Secretary of HHS, Eric. D. Hargan appointed Austin to lead the advancement of human health research between HHS agencies and NASA. An interagency agreement (PDF - 273KB) was signed with the objectives to:• Share innovative ideas for addressing science and tech challenges• Promote collaboration and cooperation in research and development related to human and public health• Leverage shared resources including expertise, access to facilities and scientific resourcesThe HHS-NASA team has engaged with a number of HHS components to help build collaborative efforts. In January 2021, HHS appointed staff members to centralize the partnership and ensure continuity between administrations. NCATS remains a leader in these efforts, working alongside NASA and HHS staff to coordinate activities.NIH and NASA work together to coordinate funding and collaboration opportunities, workshops and conferences, special interest groups and other activities across agencies. For more information, see a list of activities as well as NCATS’ collaborative workspace. | Through an ongoing collaboration, NIH and NASA are exploring how biomedical research can address the challenges of deep space exploration and benefit human health in space and on Earth. | Biomedical Research and Public Health Collaborations with NASA | Through an ongoing collaboration, NIH and NASA are exploring how biomedical research can address the challenges of deep space exploration and benefit human health in space and on Earth. | Biomedical Research and Public Health Collaborations with NASA | ||||
| 9519 | New CTSA Program Informatics Center to Create a Nationwide Data Ecosystem | Melissa Haendel, Ph.D., program director for CD2H at Oregon Health & Science University in Portland Biomedical tools and technologies are evolving rapidly, enabling scientists to both generate and analyze more research data than ever before. But what happens when datasets become too large to share or when the data from various sources are so dissimilar that they cannot be combined easily with another related but different dataset? These types of data roadblocks slow or prevent the translation of scientific research into medical knowledge and, ultimately, health benefits. By studying how data are created, used, and shared, informatics experts address challenges that result from the ever-increasing volume and complexity of data generation. By making broad, high-level connections across problems and projects, these efforts can help ensure that new knowledge and approaches can be disseminated widely for the greatest potential in getting new treatments to more patients more quickly. Making data more meaningful, open and accessible is a key goal in NCATS’ efforts to improve translational science. In the fall of 2017, through its Clinical and Translational Science Awards (CTSA) Program, NCATS established the National Center for Data to Health (CD2H). Through the CD2H, informatics experts are developing standardized approaches and best practices, including algorithms and other specialized tools, to address operational and institutional barriers to sharing data. CD2H investigators are currently taking an inventory of the existing CTSA Program informatics and data-sharing resources that they can build upon. “Some of the most valuable assets CTSA Program hubs have are data, but their potential can only be realized when they are used more comprehensively with the help of various informatics approaches,” said Melissa Haendel, Ph.D., program director for CD2H at Oregon Health & Science University in Portland. “By working with others nationwide who are supported through the CTSA Program, we will identify problems and implement solutions to speed translation and provide better health for patients.” Posted March 2018 | NCATS established the National Center for Data to Health to develop standardized approaches and best practices that address operational and institutional barriers to sharing data. | /sites/default/files/cd2h_1260x630.jpg | New CTSA Program Informatics Center | NCATS established the National Center for Data to Health to develop standardized approaches and best practices that address operational and institutional barriers to sharing data. | /sites/default/files/cd2h_1260x630.jpg | New CTSA Program Informatics Center | ||
| 9525 | Valerie Montgomery Rice, M.D. (2020) | Valerie Montgomery Rice, M.D., is president and dean of the Morehouse School of Medicine in Atlanta. Rice also is a professor of obstetrics and gynecology and reproductive endocrinology and infertility. She joined Morehouse in 2011, after serving as professor and chair of the Department of Obstetrics and Gynecology, then dean and senior vice president, at Meharry Medical College. Her clinical and research interests are in reproductive medicine, ovarian cancer and menopause. She has an impressive record of research funding and publication. Montgomery Rice serves multiple professional organizations, including serving as chair of the American Medical Association’s OB/GYN Section. She holds numerous teaching and research awards and is recognized for her professional and community service, notably for support of minority and professional women. Her activities also reflect her interests in women’s health issues, minority health care and health disparities. Montgomery Rice received her degree from Harvard Medical School in 1987 and completed her internship and residency in gynecology and obstetrics at Emory University, followed by a fellowship in endocrinology and infertility at Hutzel Women’s Hospital in Detroit. | Dr. Montgomery Rice is president and dean of the Morehouse School of Medicine in Atlanta. | /sites/default/files/Montgomery%20Rice_900x600.jpg | Valerie Montgomery Rice, M.D. (2020) | Dr. Montgomery Rice is president and dean of the Morehouse School of Medicine in Atlanta. | /sites/default/files/Montgomery%20Rice_900x600.jpg | Valerie Montgomery Rice, M.D. (2020) | ||
| 9526 | Stephen P. Spielberg, M.D., Ph.D. (2020) | Stephen P. Spielberg, M.D., Ph.D., is editor-in-chief of Therapeutic Innovation and Regulatory Science. Spielberg has served as a clinical associate in pediatrics at the Eunice Kennedy Shriver National Institute of Child Health and Human Development and director of clinical pharmacology and toxicology at The Hospital for Sick Children in Toronto. He also has served as vice president for pediatric drug development for Johnson & Johnson. His expertise in pediatric pharmacology, toxicology and therapeutic development is evidenced by his extensive publication record, which includes recent commentaries on therapeutic innovation and regulatory science, pediatric drug development and personalized medicine. His publications reflect his interest in therapeutics for child and maternal health worldwide. Spielberg has published more than 140 scientific papers and received numerous awards for his work advancing pediatric therapeutics. He received his undergraduate degree in biology from Princeton and his M.D.-Ph.D. in pharmacology from the University of Chicago, completing an internship and his residency at Children’s Hospital Medical Center in Boston and a clinical fellowship in pediatrics at Harvard Medical School. | Dr. Spielberg is editor-in-chief of Therapeutic Innovation and Regulatory Science. | /sites/default/files/Stephen%20Speilberg_900x600.jpg | Stephen P. Spielberg, M.D., Ph.D. (2020) | Dr. Spielberg is editor-in-chief of Therapeutic Innovation and Regulatory Science. | /sites/default/files/Stephen%20Speilberg_900x600.jpg | Stephen P. Spielberg, M.D., Ph.D. (2020) | ||
| 9527 | Sharon F. Terry, M.A. (2019) | Sharon F. Terry, M.A., is the president and chief executive officer (CEO) of Genetic Alliance, a network of more than 10,000 organizations, 1,200 of which are disease advocacy organizations. Genetic Alliance engages individuals, families and communities to transform health. She is also the founding CEO of PXE International, a research advocacy organization for the genetic condition pseudoxanthoma elasticum (PXE), which affects Terry’s two adult children. As co-discoverer of the gene associated with PXE, she holds the patent for ABCC6 to act as its steward and has assigned her rights to the foundation. She developed a diagnostic test for the gene and conducts clinical trials. Terry is the author of 150 peer-reviewed papers, 30 of which are PXE clinical studies. She also is a co-founder of the Genetic Alliance Registry and BioBank. In her position at the forefront of consumer participation in genetics research, services and policy, Terry serves in a leadership role on many major national and international organizations, including the Precision Medicine Initiative Cohort Advisory Panel and the Accelerating Medicines Partnership. | Terry is the president and chief executive officer (CEO) of Genetic Alliance, a network of more than 10,000 organizations, 1,200 of which are disease advocacy organizations. | /sites/default/files/Sharon_Terry_900x1000px.jpg | Sharon F. Terry, M.A. (2019) | Terry is the president and chief executive officer (CEO) of Genetic Alliance, a network of more than 10,000 organizations, 1,200 of which are disease advocacy organizations. | /sites/default/files/Sharon_Terry_900x1000px.jpg | Sharon F. Terry, M.A. (2019) | ||
| 9517 | Ronald J. Bartek, M.A. (2020) | Ronald J. Bartek, M.A., is the co-founder and founding president of the Friedreich’s Ataxia Research Alliance, a nonprofit organization dedicated to educational and research efforts aimed at developing treatments and cures for Friedreich’s ataxia. He also serves on the board of directors of the National Organization for Rare Disorders and on the Data and Safety Monitoring Board of the NCATS-led Rare Diseases Clinical Research Network. Recently, Bartek served as a member of the National Advisory Neurological Disorders and Stroke Council and sat on a panel of patient advocates assembled by the director of the National Institute of Neurological Disorders and Stroke to help develop the Institute’s strategic plan and enhance two-way communication between the patient and scientific communities. His past experience includes serving as the partner and president of a business and technology development, consulting and government affairs firm (Mehl, Griffin & Bartek) and directing the American Friends of the Czech Republic. Bartek’s career also includes 20 years of federal executive branch and legislative branch service in defense, foreign policy and intelligence. Bartek graduated from the United States Military Academy at West Point and holds a master’s degree in Russian area studies from Georgetown University. | Mr. Bartek is the co-founder and founding president of the Friedreich’s Ataxia Research Alliance. | /sites/default/files/Ron_Bartek_900x1000px.jpg | Ronald J. Bartek, M.A. (2020) | Mr. Bartek is the co-founder and founding president of the Friedreich’s Ataxia Research Alliance. | /sites/default/files/Ron_Bartek_900x1000px.jpg | Ronald J. Bartek, M.A. (2020) | ||
| 9521 | Katharine Ku, M.S. (2020) | Katharine Ku is the executive director of the Stanford University Office of Technology Licensing, which is responsible for the licensing of various state-of-the-art university technologies and industry-sponsored research agreements, material transfer agreements and collaborations. In addition to leading Stanford’s pre-award Sponsored Projects Office, Ku has held various positions at Stanford and served as the vice president of business development at Protein Design Labs, Inc., in Mountain View, California. She is a member of the NIH Advisory Committee to the Deputy Director of Intramural Research and of the Biomedical Engineering Society’s Industry Advisory Board. Ku holds a B.S. in chemical engineering from Cornell University and an M.S. in chemical engineering from Washington University, and she is a registered patent agent. | Katharine Ku is the executive director of the Stanford University Office of Technology Licensing. | /sites/default/files/Katharine_Ku_900x1000px.jpg | Katharine Ku, M.S. (2020) | Katharine Ku is the executive director of the Stanford University Office of Technology Licensing, | /sites/default/files/Katharine_Ku_900x1000px.jpg | Katharine Ku, M.S. (2020) |
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