| ID | Title | Body | Facebook Description | Facebook image | Facebook Title | Facebook Video | Twitter Description | Twitter Image | Twitter Title | Meta tags |
|---|---|---|---|---|---|---|---|---|---|---|
| 11764 | CTSA Program Researchers Shed Light on Improved Melanoma Treatment | Thomas Graeber, Ph.D., lead researcher for the team at University of California, Los Angeles. (University of California, Los Angeles Photo) Melanoma is a deadly form of skin cancer. The disease starts in melanocytes, the cells of the body that make skin pigment. As this cancer progresses, some melanoma cells can revert back to an earlier stage of development. This process, called de-differentiation, can make the cells more resistant to existing treatments. Researchers at the University of California, Los Angeles, an NCATS Clinical and Translational Science Awards (CTSA) Program hub, wondered whether melanoma cells that de-differentiate might be vulnerable to other approaches to treatment. The team began by looking at the characteristics of 53 different melanomas. The cancer cells fell into four groups based on their stages of differentiation. The researchers found that two kinds of cancer treatment, targeted therapy and immunotherapy, actually encouraged melanoma cells to de-differentiate and become resistant to the treatment. And as the team had hoped, de-differentiated cells appear to be vulnerable to a new line of attack, with drugs known as ferroptosis inducers. Using these findings, doctors might be able to fight some types of melanoma with a combination of existing and ferroptosis-based treatments. Modern treatments lead to cell de-differentiation in cancers other than melanoma, so this work could potentially help find better treatments for other cancers as well. Learn more about this advance. Posted August 2018 | CTSA Program researchers at the University of California, Los Angeles, discovered four subtypes of melanoma, pointing to new treatment approaches. | /sites/default/files/ucla-melanoma_1260x630.jpg | CTSA Program Researchers Shed Light on Improved Melanoma Treatment | CTSA Program researchers at the University of California, Los Angeles, discovered four subtypes of melanoma, pointing to new treatment approaches. | /sites/default/files/ucla-melanoma_1260x630.jpg | CTSA Program Researchers Shed Light on Improved Melanoma Treatment | ||
| 11669 | A Specialized Platform for Innovative Research Exploration (ASPIRE) | The scientific field of chemistry has changed little over the past century. Most chemical production, or synthesis, is an artisanal, non-mechanized process. Research on automating chemistry is typically focused on manufacturing existing drugs and other chemicals, not on new discoveries. The process of new bioactive chemical discovery is still based on artisanal intuition and experimentation, making it slow and inefficient. NCATS proposes to transform chemistry from an individualized craft to a modern, information-based science through A Specialized Platform for Innovative Research Exploration* (ASPIRE). By addressing long-standing challenges in the field of chemistry, including lack of standardization, low reproducibility and an inability to predict how new chemicals will behave, ASPIRE is designed to bring novel, safe and effective treatments to more patients more quickly at lower cost. After more than a century of synthetic organic chemistry and pharmacology, scientists have no shared understanding of how the complete set of all possible chemicals overlaps with the world of biology. Translation requires the discovery and production of new chemicals that can modify biological targets. New biologically active chemicals can help researchers understand biological functions or become new drugs for treatment of diseases. Yet more than 99.9 percent of the possible chemicals that could be biologically relevant remain unexplored due to an antiquated way of approaching new chemistries, and it is currently impossible to predict the chemicals that will affect a particular biological target. ASPIRE builds on the power of recent and emerging technological innovations. Chemical laboratory automation has advanced, as has microfluidic flow chemistry, which involves tiny amounts of reagents that move continuously through a system. High-throughput screening allows many experiments to run simultaneously in parallel. Machine learning enables significant computational improvement based on collective successful and failed experiments. This convergence of technologies provides a new opportunity to break translational bottlenecks in chemistry and benefit science and health. This initiative promotes multidisciplinary collaborations among government, academic and pharmaceutical researchers; funders; professional societies; scientific publishers; and other stakeholders. ASPIRE supports NCATS’ work to develop, demonstrate and disseminate innovative technologies that will bring diagnosis and treatments to patients and will deliver on NIH’s efforts to increase reproducibility and scientific rigor. * Formerly named the Automated Synthesis Platform for Innovative Research and Execution. ASPIRE Past Activities Mapping Biologically Active Chemical Space to Accelerate Drug Discovery 2018 NCATS ASPIRE Design Challenges ASPIRE CAN Review Board Concept Clearance, Sept. 7, 2017 (PDF - 55KB) | NCATS proposes to transform chemistry from an individualized craft to a modern, information-based science through A Specialized Platform for Innovative Research Exploration* (ASPIRE). | /sites/default/files/aspire4_900x600.jpg | A Specialized Platform for Innovative Research Exploration (ASPIRE) | NCATS proposes to transform chemistry from an individualized craft to a modern, information-based science through A Specialized Platform for Innovative Research Exploration* (ASPIRE). | /sites/default/files/aspire4_900x600.jpg | A Specialized Platform for Innovative Research Exploration (ASPIRE) | ||
| 11534 | NCATS' Role in the NIH HEAL Initiative<sup>SM</sup> | Launched in April 2018, the NIH HEAL (Helping to End Addiction Long-termSM) Initiative is a trans-NIH effort to advance national priorities in addressing the opioid crisis through science. As part of the initiative, NIH developed a research plan to bolster research to enhance pain management strategies and improve treatments for opioid misuse and addiction. Overview More than 2 million Americans have an opioid use disorder, and even more misuse these medicines by taking them longer or in higher doses than prescribed. To address the opioid epidemic, new, safe interventions are needed to combat opioid misuse and addiction and to treat pain. Not only are more accurate research models needed to understand how potential new drugs will affect humans, but scientists also must identify existing drugs or develop new therapies that have potential as effective treatments for opioid misuse and addiction and for pain. NCATS' Role in NIH HEAL As part of the NIH HEAL Initiative, NCATS will facilitate and accelerate the process of developing and demonstrating new treatments for opioid misuse and addiction and for pain. Because NCATS focuses on translational science — through which new interventions are developed, demonstrated and disseminated to improve human health — the Center is poised to address the public health crisis of opioid use. NCATS is leading trans-NIH collaborative NIH HEAL efforts to: Develop 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; Identify and de-risk potential therapies that work in novel ways through development of assays (tests), high-throughput screening and optimization of promising compounds; Accelerate the identification of promising chemical structures and develop those into pharmacological or drug-like compounds; Advance promising new drug candidates through rigorous preclinical efficacy and safety studies for first-in-human clinical trials as required by the Food and Drug Administration (FDA); and Facilitate 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. NCATS is providing a suite of translational science resources, including high-throughput screening, and expertise to the research community working on opioid and pain research. With this expert collaborative infrastructure already in place, NCATS will accelerate the process of getting new treatments for pain and for opioid misuse and addiction to patients faster. NCATS-Supported NIH HEAL Projects NCATS supports several NIH HEAL-related projects. Human Cell-Based Screening (HCBS) Platforms and Novel Drugs to Treat Pain, Addiction and Overdose The HCBS Platforms and Novel Drugs to Treat Pain, Addiction and Overdose project has two components: HCBS platform development and Accelerating the translation of novel compounds toward Investigational New Drugs (INDs) for subsequent clinical testing. HCBS Platform Development NCATS will focus 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) will develop a renewable supply of human cell-based models, focusing on patient-derived iPSC lines that give rise to specialized, functional nerve cells that are relevant for opioid misuse and addiction and for pain. NCATS will offer 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. NCATS’ Division of Preclinical Innovation 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 Experts in NCATS’ 3-D Tissue Bioprinting program 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 is supporting 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 will 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 (BBB). 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 will provide funding for investigators to create and test devices that can model the mechanisms or effects of pain-relevant signaling, addiction, or opioid use disorders (OUDs), 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. Accelerating the Translation of Novel Compounds Toward INDs for Subsequent Clinical Testing Using the Center’s therapeutic development resources, NCATS scientists will collaborate with the external research and development community as well as other scientists at NIH to identify and test existing and potential 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. Developing New Chemical Structures to Modulate Targets of Pain, Addiction and Overdose Through this initiative, researchers will design and build new chemistries, biological assays, data-mining tools and other analysis technologies directed toward altering a cell’s pain and reward pathway. NCATS is asking the research community for concepts to design novel chemicals that have the potential for addressing pain, addiction and overdose. Promising ideas for the new chemical structures will be carried forward and tested through NCATS’ A Specialized Platform for Innovative Research Exploration (ASPIRE), an automated synthetic chemistry (ASC) modular platform. Using this platform, scientists will apply new ASC techniques and testing capabilities to accelerate the development of new therapeutics. ASPIRE Challenges NCATS has issued challenge prize competitions for an innovative translational science platform resulting from the integration of various components, such as design of a chemistry database, an electronic laboratory knowledge portal, machine learning algorithms and biological assays focused in the areas of pain, addiction and overdose. The goal is to revolutionize the discovery of novel compounds for the treatment of pain, opioid use disorder and overdose. The Center anticipates that this integrated platform ultimately can be generalized to address many of the roadblocks in automated chemistry. Creating Pharmacological Probes for Novel Targets Research experts in opioid misuse and addiction, and in pain, have identified compounds that act on biological targets of opioids and pain and tested them in cell- and animal-based models. NCATS will 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 also will leverage repositories of natural products available through the National Cancer Institute and the Fogarty International Center. Developing Investigational Drugs Ready for Clinical Testing Scientists working with NCATS’ Preclinical Therapeutics Development Branch, including those in the Therapeutics for Rare and Neglected Diseases and Bridging Interventional Development Gaps programs, will identify and optimize new drug candidates for opioid misuse and addiction and for pain. Researchers will determine which candidates can be further developed and tested to enable IND applications to the FDA 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. In addition to NCATS resources, the National Institute of Neurological Disorders and Stroke (NINDS) has published a funding opportunity announcement on “Optimization of Non-Addictive Therapies to Treat Pain (UG3/UH3 Clinical Trials Not Allowed).” The goal is to speed the preclinical translational development necessary to advance small molecules and biologics to clinical testing for treatment of pain. NIH HEAL Pain Management Effectiveness Research Network (NIH HEAL Pain Management ERN) Evidence for optimal pain management of some pain conditions is insufficient, such as long-term opioid use to manage chronic pain. The NIH HEAL Pain Management ERN will conduct 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. The network will use the NCATS Clinical and Translational Science Awards (CTSA) Program’s Trial Innovation Network and the CTSA hubs to implement clinical trials/studies of interest to multiple NIH Institutes, Centers and Offices and support studies that provide evidence to inform practice-based guidelines. View RFA-NS-19-021 to learn more. | With HEAL support, NCATS will accelerate the process of developing new treatments for opioid misuse and addiction and for pain. | NCATS' Role in the NIH HEAL Initiative | With HEAL support, NCATS will accelerate the process of developing new treatments for opioid misuse and addiction and for pain. | NCATS' Role in the NIH HEAL Initiative | ||||
| 11517 | Finding What’s Common Among Diseases, Seeing the Bigger Picture | Translational Science HighlightNCATS scientists are identifying biological commonalities among diseases and applying this knowledge of shared processes and pathways to the understanding of multiple human diseases and conditions.To find cures for diseases, scientists traditionally study one disorder at a time. While this approach has produced a wealth of information about the characteristics of individual diseases and potential treatments, it rarely results in transformational insights that improve research efficiency and effectiveness.Scientists at NCATS take a different approach. By working to address translational research obstacles through projects across many diseases, NCATS teams aim to learn more about what different diseases have in common. This knowledge of the bigger picture can help scientists advance the overall translational science process.Collaborators from across the globe have engaged the scientists in NCATS’ Division of Preclinical Innovation for help tackling translational roadblocks in their research. NCATS does not initiate this research; the investigators come to NCATS for help to make their translational research more efficient and effective. For example, NCATS scientists can create probes to explore the activity of molecules and conduct high-throughput screening to find new drug candidates against a disease. Through individual projects, NCATS researchers have made connections across seemingly unrelated diseases.Access to Specialized ExpertiseAn NCATS scientist prepares samples for high-throughput screening. (Daniel Soñé Photography, LLC, Photo)NCATS offers expertise in the use of small molecules to study and advance treatments for many different types of disease. A small molecule can be used as a probe to explore the role of a protein in a disease, or the small molecule could even become a drug. Molecules from NCATS’ compound libraries are checked against a target protein to see which, if any, molecules change the protein’s activity. This process involves thousands of experiments, which are conducted by robots. When promising small molecules emerge, NCATS scientists can pursue more detailed tests to learn about their activity.Researchers needing access to NCATS’ unique expertise and resources to address preclinical issues in their research have approached NCATS scientists to develop solutions and collect needed data. Several of these collaborations have involved projects studying neurodegenerative diseases, including Alzheimer’s, Huntington’s and Parkinson’s diseases.“We just happened to be approached by investigators who had these neurodegenerative diseases as their focus and had these novel therapeutic hypotheses in mind,” said NCATS Scientific Director Anton Simeonov, Ph.D.The NCATS Approach in ActionAs knowledge of individual diseases grew, so did the possibility of identifying commonalities among them. In many of these disorders, nerve cells in the brain, called neurons, have trouble getting rid of their waste. As the waste builds up, the cells gradually stop working. The results can be devastating and often fatal.“I’d been involved in a number of those projects where we thought we had the perfect molecule, but it didn’t go anywhere,” said Juan Marugan, Ph.D., group leader in the NCATS Chemical Genomics Center, who joined NIH after working in the pharmaceutical industry. “What we are looking for at NCATS is to fundamentally change the way we approach disease.”Marugan’s work on neurodegeneration began about a decade ago, on a project with researchers interested in the enzyme glucocerebrosidase (GCase). An enzyme is a protein that drives chemical reactions in cells; for GCase, that means breaking down a specific kind of fat. But in people with a rare disorder called Gaucher disease, cells produce a form of GCase that does not fold properly. The misfolded enzyme cannot remove the waste fat, which builds up in the brain and other organs and causes damage that can be life-threatening.For the GCase project, Marugan’s team screened a huge library of small molecules for ones that would make the enzyme fold correctly. Eventually, the researchers identified the small molecule NCGC607, which increases the activity of GCase in cells. This work could eventually lead to a drug to treat Gaucher disease that would be cheaper and easier for patients than the current invasive treatment of injections of the missing enzyme.People with Gaucher disease also have a high risk of developing Parkinson’s disease, which affects millions of people worldwide. Increasing the activity of GCase might help fight the abnormal proteins that build up in the brains of people with Parkinson’s disease, even if they don’t have Gaucher disease.A Broader PerspectiveNCATS’ robotic platform enables high-throughput screening of small molecules.Small molecule expertise was also the basis of a more recent neurodegeneration project on Huntington’s disease that involved other NIH and academic collaborators. Researchers at NCATS tested thousands of compounds and found that a small molecule called NCT-504 lowered the level of a key protein in diseased cells, making them more likely to survive.The project led to a broad insight as well: The molecule did not interact with the toxic protein directly. Instead, it worked by turning up the cell’s “recycling” system. Normally, cells use enzymes to break down large molecules like proteins and fats that the cells no longer need and use the components to make new molecules. But sometimes cells lose the ability to carry out their normal recycling processes, and the waste products pile up. Since waste products build up in many neurodegenerative disorders, the findings could lead to new treatments for conditions beyond Huntington’s disease.NCATS researchers have also found a small molecule that improves muscle function in the rare muscle disease Duchenne muscular dystrophy and studied fat buildup in the rare disease Niemann-Pick type C1 (NPC1). A small molecule involved in NPC1 has been licensed to a pharmaceutical company for further development and is being tested in humans now, showing promising results.In addition to projects on neurodegenerative diseases, NCATS scientists have projects focusing on the functioning of mitochondria, which are the structures in cells that make energy, and on how cancer cells make energy. Each area could be relevant to dozens of diseases.“If you can understand the basic principles of how a particular molecular problem leads to a particular disease, you can inform translation in ways that can lead to treatments for multiple diseases,” Simeonov said.Posted July 2018 | NCATS scientists are identifying biological commonalities among diseases and applying this knowledge of shared processes and pathways to the understanding of multiple human diseases and conditions. | /sites/default/files/neurodegeneration1_1260x630.jpg | Finding What’s Common Among Diseases, Seeing the Bigger Picture | NCATS scientists are identifying biological commonalities among diseases and applying this knowledge of shared processes and pathways to the understanding of multiple human diseases and conditions. | /sites/default/files/neurodegeneration1_1260x630.jpg | Finding What’s Common Among Diseases, Seeing the Bigger Picture | ||
| 11497 | NCATS-Supported Study Highlights Troubling Trend in Opioid Prescribing | (Flickr Photo/Cindy Shebley) Medications known as benzodiazepines are often prescribed to treat anxiety, panic attacks and other mental health issues. However, when benzodiazepines are prescribed along with opioids, a patient’s risk of opioid overdose can be as much as seven times higher than for a patient taking an opioid alone. Supported in part through NCATS’ Clinical and Translational Science Awards (CTSA) Program, University of California, Los Angeles, researchers analyzed 13,146 reported medical visits from 2005 to 2015 that included a new opioid prescription. The results were published in the April 12, 2018, issue of JAMA Psychiatry and show that overall, first-time prescriptions for opioids have dropped significantly since 2010. But a troubling trend persists: Despite the higher risk of overdose, patients already taking benzodiazepines were more than twice as likely to receive a new prescription for opioids in 2015 than the general population. Learn more about this research. Posted July 2018 | CTSA Program-supported scientists found that patients who took a type of anti-anxiety medicine were more likely to receive new opioid prescriptions despite a higher overdose risk. | /sites/default/files/ucla_opioids_1200x630.jpg | NCATS-Supported Study Highlights Troubling Trend in Opioid Prescribing | CTSA Program-supported scientists found that patients who took a type of anti-anxiety medicine were more likely to receive new opioid prescriptions despite a higher overdose risk. | /sites/default/files/ucla_opioids_1200x630.jpg | NCATS-Supported Study Highlights Troubling Trend in Opioid Prescribing | ||
| 11457 | Past NCATS Day Events | 2017 Agenda Presentations Posters Photos | Past NCATS Day Events | Past NCATS Day Events | ||||||
| 11435 | NCATS Ignites Translational Science Spark for Young Investigators | Translational Science HighlightNCATS partners with universities to train the next generation of translational scientists and help them benefit from international collaborations.Each day, scientists in the NCATS Division of Preclinical Innovation are hard at work to uncover discoveries that have the potential to become tomorrow’s treatments. In addition to doing exemplary research, many of these individuals are also training and cultivating the next generation of translational scientists.NCATS sponsors talented Ph.D. students, including several profiled in 2016, through the NIH Graduate Partnerships Program. These exceptional students split their time between an NIH Institute or Center and a host university, receiving mentorship and guidance from researchers at both institutions. NCATS works with each trainee to customize their experiences in ways that will best meet their interests and career goals.“The Graduate Partnerships Program enables some of the best and brightest young investigators to come to NCATS, and it helps us form new collaborations with the host university,” said Anton Simeonov, Ph.D., NCATS scientific director. “In exchange, we provide these promising scientists — each of whom brings a unique background and fresh perspective — with training in translational research and access to some of the best high-throughput screening facilities and compound libraries in the world.”Targeting TumorsDavid Morse, M.Phil., a graduate student in the NIH Oxford-Cambridge Scholars Program. (University of Cambridge/Department of Chemistry)For his master’s research at Cambridge University, David Morse, M.Phil., was trying to apply nanotechnology to tumor treatment, using tiny molecular vehicles to deliver anticancer drugs to tumors while leaving healthy cells alone. But Morse realized that researchers had an incomplete understanding of tumors that was limiting to his research.To help bridge that knowledge gap, Morse is working under the mentorship of Craig Thomas, Ph.D., who leads NCATS’ Chemistry Technology program, and Tuomas Knowles, Ph.D., at Cambridge, as part of the NIH Oxford-Cambridge (OxCam) Scholars Program.Now in his second year in the Ph.D. program, Morse is developing new techniques to study what genes are turned on in individual tumor cells. Much of what we know about tumors is based on studies that look at the cells in a tumor as a mixture, treating all cells as the same. This approach fails to capture the differences within tumors. Using the technique he helped develop, Morse found that tumor cells in the center of an ovarian tumor are very different from those on the tumor’s surface.Because the surface cells are more accessible to potential treatments, these results could help researchers design better drugs or ways to deliver existing drugs to ovarian tumors. Morse is now working to improve the new technique to track each cell and pinpoint precisely where each cell was in the tumor at the time of data collection.So far, Morse has worked not only with teams at NCATS and Cambridge, but also with collaborating laboratories at the Broad Institute and Harvard University.“Each institution brings their unique strengths to the project, with NCATS really excelling at scaling things up so that we can use this technique on tumors from many different patients,” Morse said.Advancing Drug AssaysDorian Cheff is a graduate student in a collaborative doctoral program between NCATS and the Karolinska Institutet in Sweden. (Karolinska Institutet/Qing Cheng)A desire to explore a career in research brought Dorian Cheff to the NIH post-baccalaureate program after her undergraduate studies at University of Michigan. A chance encounter with Matthew D. Hall, Ph.D., who leads the NCATS Chemical Genomics Center’s biology group, brought her to NCATS.“I just fell in love with the translational research process and NCATS as a whole,” Cheff said. “The people are great to work with and incredibly passionate about accelerating the way we do science.”After more than two years of mentorship with Hall, Cheff decided to continue her training as a Ph.D. student through the NIH Graduate Partnerships Program. Cheff and Hall reached out to Elias Arnér, M.D., Ph.D., at the Karolinska Institutet in Sweden, about collaborating on an understudied family of enzymes. One enzyme in the family, glutathione peroxidase, helps cancers survive during stress, such as drug or radiation treatment. Cheff chose the enzyme because it is particularly important in glioblastoma, an aggressive form of brain cancer. Her project is designed to find molecules that block the enzyme and make the cancer more sensitive to existing drugs. Cheff has been in Sweden since August 2017, working to make glutathione peroxidase in the laboratory and to create an assay (test) to identify molecules that stop the enzyme from working. Once she has refined the assay, she will bring it back to NCATS for testing with the Center’s high-throughput screening robots and equipment. Then she will screen NCATS’ collections of chemicals and molecules to find leads for a potential new drug against glioblastoma.“A great asset for my project is the collaboration between the medicinal chemists, biologists and informatics specialists at NCATS,” Cheff explained. “We’re all working together to figure out how to make promising molecules work better.”Combatting MalariaKimberly Breglio, D.Phil., a graduate of the NIH Oxford-Cambridge Scholars Program. (Penn State College of Medicine) Kimberly Breglio, D.Phil., was nearing the end of her medical school training when she decided to take a pause to conduct research. The NIH OxCam Scholars Program was ideal, because she did not have to take more classes or try out different labs before diving into her doctoral research.Working with Thomas at NCATS and Katja Simon, Ph.D., and David Roberts, D.Phil., at the University of Oxford, Breglio focused on possible ways the malaria parasite becomes resistant to drugs. She found a gene, ATG18, that seemed to be altered in many resistant parasites. Breglio used gene-editing tools to create malaria parasites that had this altered gene so she could study them in the lab. She screened the parasites against NCATS’ compound collections to try to understand why the altered gene seems to be beneficial to the parasite. Researchers at NCATS helped her sift through all the resulting data, which she plans to publish by the end of 2018. Her work will help researchers understand how malaria parasites survive stress, such as exposure to a drug.“NCATS has been great,” Breglio said. “There is so much cutting-edge research going on, and the multidisciplinary Chemistry Technology group provided many insights into my project.”Breglio is now completing her medical degree and plans to use her research skills as a clinical investigator to study how tropical diseases affect the skin. Like her fellow trainees working with NCATS mentors through the NIH Graduate Partnerships Program, she is now better equipped for a collaborative and multidisciplinary research career in tackling translational science problems.Through this and other training programs, NCATS is fostering tomorrow’s translational workforce. Learn more about Translational Science Training at NCATS.Posted July 2018 | Meet three graduate students working collaboratively on research projects with NCATS and university scientists from England and Sweden. | /sites/default/files/dpischolars_doriancheff_1260x630.jpg | NCATS Ignites Translational Science Spark for Young Investigators | Meet three graduate students working collaboratively on research projects with NCATS and university scientists from England and Sweden. | /sites/default/files/dpischolars_doriancheff_1260x630.jpg | NCATS Ignites Translational Science Spark for Young Investigators | ||
| 11459 | Assay Guidance Workshop Agenda — September 10-11, 2018 | September 10, 2018 — 7:30 a.m. - 5:00 p.m. ET William F. Bolger Center, 9600 Newbridge Drive, Potomac, Maryland 20854 7:30 a.m.: Registration 8:00 a.m.: Robust Assays Define Success in Preclinical Research Nathan P. Coussens, Ph.D., NCATS, NIH 8:15 a.m.: Target Validation in Physiologically Relevant Drug Discovery Models Madhu Lal-Nag, Ph.D., NCATS, NIH 9:00 a.m.: Treating Cells as Reagents to Design Reproducible Screening Assays Terry Riss, Ph.D., Promega Corporation 9:45 a.m.: Beverage Break 10:00 a.m.: Concepts in the Development and Validation of Robust Cell-Based and Biochemical Assays Timothy L. Foley, Ph.D., Pfizer Inc. 10:45 a.m.: Statistical Design of Experiments for Assay Development Steven D. Kahl, Eli Lilly and Company 11:30 a.m.: Lunch 1:00 p.m.: Introduction to Mass Spectrometry for Drug Screening and Lead Optimization Kenneth D. Roth, Ph.D., Eli Lilly and Company 1:45 p.m.: Integration of Biophysical Techniques for the Screening of Fragment Libraries and Lead Optimization Jose M. M. Caaveiro, Ph.D., Graduate School of Pharmaceutical Sciences, Kyushu University 2:30 p.m.: Basic Assay Statistics, Data Analysis and Rules of Thumb Thomas D. Y. Chung, Ph.D., Sanford Burnham Prebys Medical Discovery Institute 3:15 p.m.: Beverage Break 3:30 p.m.: Reproducibility and Differentiability of Compound Potency Results from Screening Assays in Drug Discovery Viswanath Devanarayan, Ph.D., Charles River Laboratories 4:15 p.m.: Assay Operations: Keeping Your Assays Robust and Reproducible Jeffrey R. Weidner, Ph.D., QualSci Consulting, LLC 5:00 p.m.: Adjourn September 11, 2018 — 7:30 a.m. - 5:30 p.m. ET NCATS, 9800 Medical Center Drive, Rockville, Maryland 20850 7:30 a.m.: Registration 8:00 a.m.: Bioassay Interference by Aggregation and Chemical Reactivity Jayme L. Dahlin, M.D., Ph.D., Brigham and Women’s Hospital 8:45 a.m.: In Vitro Toxicological Testing Using a qHTS Platform Menghang Xia, Ph.D., NCATS, NIH 9:30 a.m.: In Vitro Assessments of ADME Properties of Lead Compounds Xin Xu, Ph.D., NCATS, NIH 10:15 a.m.: Beverage Break 10:30 a.m.: Lead Selection and Optimization by Medicinal Chemistry Samarjit Patnaik, Ph.D., NCATS, NIH 11:15 a.m.: Assay Development for High-Content Screening O. Joseph Trask, Jr., PerkinElmer, Inc. 12:00 p.m.: Lunch 1:15 p.m.: Machine Learning in Cellular Assays: Getting Simple Answers from Complex Data Steve Haney, Ph.D. 2:00 p.m.: Hands-On Data Analysis Sessions (Participants will need to bring Laptop Computers) Data Analysis for High-Content Screening Jonny Sexton, Ph.D., Michigan Institute for Clinical and Health Research David A. Egan, Ph.D., Core Life Analytics Tools for Assay Operations and Validation Jeffrey R. Weidner, Ph.D., QualSci Consulting, LLC 5:30 p.m.: Depart | View the September AGM agenda | /sites/default/files/agm-cover-update_900x600.jpg | Assay Guidance Workshop Agenda — September 10-11, 2018 | View the September AGM agenda | /sites/default/files/agm-cover-update_900x600.jpg | Assay Guidance Workshop Agenda — September 10-11, 2018 | ||
| 11360 | CTSA Program Support May Lead to Blood Test for Colon Cancer Screening | The HudsonAlpha Institute for Biotechnology in Huntsville, Alabama. (HudsonAlpha Institute Photo). A colonoscopy is the most reliable way to find polyps, small clumps of cells that can form in the large intestine and lead to colon cancer. But colonoscopies are generally unpleasant and relatively expensive, so too many at-risk patients may avoid getting the procedure. A simple blood test would be a much less invasive way to detect potentially cancerous polyps. With that goal in mind, researchers at the University of Alabama at Birmingham (UAB) — an NCATS Clinical and Translational Science Awards (CTSA) Program hub — teamed up with scientists from the HudsonAlpha Institute for Biotechnology to identify markers in the blood that could be used to detect precancerous polyps. To find the markers, the researchers gathered blood samples from people undergoing routine colonoscopies at the UAB Medical Center and used a common lab technique to measure quantities of molecules called small RNAs. The study team found a set of small RNAs that were present in people who had colon polyps that were identified through colonoscopy. If larger studies result in similar findings, this set of small RNAs could become the basis of a blood test for colon polyps and, potentially, for colon cancer. This approach also could be applied to the early detection of other types of cancer. Learn more about this advance. Posted July 2018 | A research team, including scientists supported by the CTSA Program, identified markers in the blood that could be used to detect precancerous polyps in patients. | CTSA Program Support May Lead to Blood Test for Colon Cancer Screening | A research team, including scientists supported by the CTSA Program, identified markers in the blood that could be used to detect precancerous polyps in patients. | CTSA Program Support May Lead to Blood Test for Colon Cancer Screening | ||||
| 11451 | ExRNA in Action | ExRNA communication is a recently discovered cell-to-cell signaling process that holds enormous promise for improving our understanding of a wide variety of diseases. Read about ExRNA in action below. June 2018 Repurposed Drug Approach May Thwart Spread of Cancer Cells NCATS preclinical researchers helped NIH Extracellular RNA Communication program-supported scientists use a new approach to find drugs that may be effective in preventing cancer cell progression. The team found that antibiotics, antifungal medicines and anti-inflammatory agents were effective in preventing advanced prostate tumor cells from releasing exosomes or in blocking exosome production. September 2015 Researchers Working to Advance ExRNA Biomarkers and Therapies NCATS has announced it will spearhead the second phase of several NIH ExRNA Communication program projects to test and validate exRNA molecules for their potential as disease biomarkers and treatments. The first phase of these NCATS projects(link is external) focused on discovery and feasibility. The NCATS projects are funded by the NIH Common Fund. | Learn about exrna in action | ExRNA in Action | Learn about exrna in action | ExRNA in Action |
Last updated on