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| 15307 | Ketogenic Diet May Offer a New Approach to Treating Alzheimer’s Disease | Along with blood vessels (red) and nerve cells (green), this mouse brain shows abnormal protein clumps known as plaques (blue). These plaques multiply in the brains of people with Alzheimer’s disease and are associated with the memory impairment characteristic of the disease.Credit: Alvin Gogineni, GenentechMarch 22, 2022The brain relies on glucose as its primary source of energy, but in people diagnosed with Alzheimer’s disease (AD), the brain is less able to use glucose for fuel. Researchers at the University of Kansas, an NCATS Clinical and Translational Science Awards (CTSA) Program hub, recently explored the potential of another source of energy—ketones—to improve brain function in people with AD. Ketones are produced when glucose is in short supply and the body turns to fat as its main energy source. Although the brain’s ability to use glucose declines in AD, its ability to use ketones does not.The research team, led by Russell Swerdlow, M.D., and Debra Sullivan, Ph.D., conducted a pilot study—the Ketogenic Diet Retention and Feasibility Trial (KDRAFT)—to assess the feasibility and cognitive effects of a ketogenic diet in individuals with AD. Ten participants with mild AD followed the ketogenic diet for three months, followed by a return to a normal diet for one month. Standard cognitive tests administered during the study showed that cognitive scores significantly improved after the three-month intervention. Test scores returned to baseline after the one-month washout period.Participants with more advanced dementia dropped out of the study, suggesting that those with mild AD are better able to adhere to the diet.The predominant theory about the cause of AD suggests that the build-up of the protein beta-amyloid in the brain disrupts communication between neurons and ultimately kills brain cells. Dr. Swerdlow’s research suggests that amyloid plaques are a result, rather than a cause, of AD and that defects in brain energy metabolism may be the underlying cause. The results of the KDRAFT study appear to support this hypothesis, although larger studies are needed. The research team, however, seems to have established proof of principle that fuel other than glucose can help restore energy metabolism in the brain. | Ketogenic Diet May Offer a New Approach | Ketogenic Diet May Offer a New Approach | ||||||
| 15262 | Fiscal Year 2019 Funded Projects and Prizes | With The Helping to End Addiction Long-termSM Initiative, or NIH HEAL InitiativeSM support, NCATS is providing a suite of translational science resources and expertise to investigators working on opioid and pain research. Learn more about NCATS-supported Initiatives and the funded projects and prizes. ASPIRE Design Challenges The goal of the 2018 NCATS ASPIRE Design Challenges is to generate innovative and catalytic approaches toward solving the opioid crisis through the development of next-generation addiction-free analgesics with new chemistries, data-mining, and analytical tools and technologies, as well as biological assays that can revolutionize discovery, development, and preclinical testing of new and safer treatments for pain, opioid use disorder (OUD), and overdose. Read more about the 2018 NCATS ASPIRE Design Challenge Winners and 2020 NCATS ASPIRE Reduction-to-Practice Challenge. Tissue Chips to Model Nociception, Addiction, and Overdose This initiative will use human tissue to create microphysiological systems (MPS), also known as tissue chips or “organs-on-chips,” to understand the mechanisms underlying nociception (the nervous system’s response to painful stimuli), pain, addiction, or OUD-like pathophysiologies. The MPS models will then be used to test new therapies to improve pain management and treat opioid addiction. Learn more about the funded projects. NCATS Trial Innovation Network (TIN) Pain Management Effectiveness Research Network, a multisite research cooperative program, aims to improve pain care by evaluating the effectiveness of a broad range of therapies to guide clinical practice in real-world settings. The network will leverage the infrastructure of the NCATS’ Trial Innovation Network to support clinical trials that compare the effectiveness of existing non-addictive therapies or of existing or novel approaches for prevention and management of pain. Learn more about the program and view details about the funded projects. | Fiscal Year 2019 Funded Projects and Prizes | Fiscal Year 2019 Funded Projects and Prizes | ||||||
| 15214 | NIH Funding Bolsters Rare Diseases Research Collaborations | Researchers from the RDCRN-supported Myasthenia Gravis Rare Disease Network are studying rare, chronic autoimmune diseases that affect how well nerves and muscles communicate with each other. Neuromuscular junctions, where motor nerves (green) send signals to muscles (red), are shown here. (Xiangyang Zhang, Ph.D., The George Washington University School of Medicine & Health Sciences)October 3, 2019New grants aimed at better understanding diseases, moving potential treatments closer to the clinicOf an estimated 6,500 to 7,000 known rare diseases, only a fraction—maybe 5%—have U.S. Food and Drug Administration-approved treatments. To increase that percentage, the National Institutes of Health has awarded approximately $31 million in grants in fiscal year 2019 to 20 teams—including five new groups—of scientists, clinicians, patients, families and patient advocates to study a wide range of rare diseases. An additional $7 million has been awarded to a separate data coordinating center to support these research efforts.The grants, which support consortia that together form the Rare Diseases Clinical Research Network (RDCRN), are aimed at fostering collaborative research among scientists to better understand how rare diseases progress and to develop improved approaches for diagnosis and treatment. This is the fourth five-year funding cycle for the RDCRN, which is supported by multiple NIH Institutes and Centers and led by NIH’s National Center for Advancing Translational Sciences (NCATS) and the NCATS Office of Rare Diseases Research.Individually, most rare diseases affect only a few hundred to several thousand people; collectively, rare diseases affect more than 25 million Americans. Many rare diseases are life-threatening and about half of those affected are children.Because rare diseases affect a small number of people, they can be extremely challenging to study. Scientists often lack basic information about a rare disease’s symptoms and biology, and the ways a disease can affect people over time. Research funding can be scarce.“Over the years, RDCRN scientists have partnered with patients and advocates to develop new insights into the causes and progression of—and potential therapies for—rare diseases that were simply not receiving the attention they deserved,” said NCATS Director Christopher Austin, M.D. “Their pioneering work in discerning underlying clinical differences and commonalities in hundreds of rare conditions has already changed the rare disease landscape in immeasurable ways.”Established by Congress under the Rare Diseases Act in 2002, the RDCRN has included more than 350 sites in the United States and more than 50 in 22 other countries. To date, they have encompassed 237 research protocols and included more than 56,000 participants in studies ranging from immune system disorders and rare cancers to heart and lung disorders, brain development diseases and more.Each RDCRN member is a consortium of clinical and scientific experts and patient groups who study a group of rare diseases. Each consortium must study three or more diseases, partner with rare disease patient advocacy groups, provide rare disease research training to investigators and perform natural history studies that chart the course and progression of diseases. The primary focus of the RDCRN is clinical research, and the network does not generally support clinical care outside of research activities.A key component of the RDCRN is the Data Management and Coordinating Center (DMCC), which was awarded to the Cincinnati Children’s Hospital Medical Center. The DMCC manages shared resources and data from the RDCRN research studies. The DMCC emphasizes the standardization of data, increased data sharing and broad dissemination of research findings.The RDCRN consortia have a rich history of accomplishment. For example, Lysosomal Disease Network scientists led crucial natural history studies and gene editing research that provided a foundation for first-in-human genome editing clinical studies for a rare metabolic disease. Primary Immune Deficiency Treatment Consortium members showed the advantage of early stem cell transplants for patients with a rare immune system disorder, severe combined immunodeficiency, and the group’s work contributed to advances in gene therapy-based treatments for the disease.New groups, new emphasisThe five new consortia are:The Global Leukodystrophy Initiative Clinical Trials Network. Lead: Children’s Hospital of PhiladelphiaCongenital and Perinatal Infections Rare Diseases Clinical Research Consortium. Lead: The University of Alabama at BirminghamFrontiers in Congenital Disorders of Glycosylation. Lead: Mayo Clinic, Rochester, MinnesotaHyperphenylalaninemia Disorders Consortium. Lead: Oregon Health & Science University, PortlandMyasthenia Gravis Rare Disease Network. Lead: The George Washington University, Washington, D.C.According to ORDR director Anne Pariser, M.D., an important focus of the latest group of awards is on clinical trial readiness.“Some of the RDCRN research groups have been working together for 10 or 15 years and have gathered important data and developed a good understanding of the diseases they study, in addition to new potential therapies. We’re emphasizing the need to be prepared to conduct clinical trials,” Pariser said.“We’re trying to get the drug candidates closer to be ready for clinical testing and de-risk the processes that lead to a successful clinical trial,” said RDCRN program officer Tiina Urv, Ph.D. “To get funding to conduct trials, they need to have strong natural history studies that show how the disease progresses, ways to measure outcomes of treatments and biomarker studies that provide indicators of how a drug is working in patients.”Collaboration is key. Consortia can involve numerous partner research teams from different sites, along with rare disease patients and advocacy groups. Scientists from different institutions come together to pool patients, data, experience and resources.“Scientists can’t work alone. They wouldn’t have enough patients, and they wouldn’t have adequate resources and information about the diseases,” Urv said. “Patients and families help scientists decide what is important to study, test and treat.”To read more about the five new consortia, 15 continuing consortia and the DMCC, see: https://ncats.nih.gov//research/research-activities/rdcrn/consortia.In addition to NCATS, other NIH funding support comes from the National Institute of Allergy and Infectious Diseases, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Institute of Neurological Disorders and Stroke, the National Heart, Lung, and Blood Institute, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute of Diabetes and Digestive and Kidney Diseases, the National Institute of Dental and Craniofacial Research, the National Institute of Mental Health and the Office of Dietary Supplements.About the National Center for Advancing Translational Sciences (NCATS): NCATS conducts and supports research on the science and operation of translation—the process by which interventions to improve health are developed and implemented—to allow more treatments to get to more patients more quickly. For more information about how NCATS is improving health through smarter science, visit https://ncats.nih.gov.About the National Institutes of Health (NIH): NIH, the nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit https://www.nih.gov. | NIH funding bolsters rare diseases research collaborations | NIH funding bolsters rare diseases research collaborations | ||||||
| 15181 | NIH Awards $89 Million for Additional Projects to Advance Genome Editing | An illustration depicting an editing tool repairing a DNA strand. Some gene-editing tools use enzymes to cut the disease-causing DNA and bind the unchanged sequences back together. (XVIVO)September 30, 2019Funding aimed at developing new technologies, novel research approaches in the Somatic Cell Genome Editing ProgramGenome editing is a promising technology that could offer new treatments or cures for diseases, but challenges remain. To help address them, the National Institutes of Health (NIH) has awarded 24 additional grants to researchers across the United States and Canada through the Somatic Cell Genome Editing (SCGE) Program. This group of awards from the SCGE Program totals approximately $89 million over four years. This brings the total number of projects supported to 45 and total funding to approximately $190 million over six years, pending available funds.The human genome contains thousands of genes, each of which contains the information that cells use to make proteins. In genetic diseases, there is a problem with a gene such that a cell may not make a protein, or it may make a protein incorrectly. Genome editing aims to change the DNA sequence so that cells can make the right proteins again. NIH created the SCGE Program in January 2018 to improve genome-editing technologies and make genome-editing therapies more widely available. All the projects focus on somatic (nonreproductive) cells in the body, which do not pass on DNA changes to future generations. As a result, the changes made through somatic cell genome editing — which can carry both risks and benefits — cannot be inherited. The program is supported by the NIH Common Fund and led by NCATS. NCATS announced the first group of SCGE grants last October.“Genome editing has extraordinary potential to alter the treatment landscape for common and rare diseases,” said Christopher P. Austin, M.D., NCATS director and SCGE Program Working Group chair. “The field is still in its infancy, and these newly funded projects promise to improve strategies to address a number of challenges, such as how best to deliver the right genes to the correct places in the genome efficiently and effectively. Together, the projects will help advance the translation of genome-editing technologies into patient care.”The initial group of SCGE grantees is beginning to show research results. For instance, University of Wisconsin scientists recently reported the development of a tiny “nanocapsule” to deliver gene-editing tools to targeted cells more accurately and effectively than before. Information about SCGE approaches, technologies and tools will be disseminated in a toolkit through the SCGE dissemination and coordinating center.“NIH Common Fund programs are meant to have exceptional impact across a broad area of science,” said Elizabeth Wilder, Ph.D., director of NIH’s Office of Strategic Coordination, which oversees the Common Fund. “As a Common Fund program, SCGE aims to transform the field of genome editing by developing new tools and moving them toward clinical use.”An illustration depicting tailored delivery vehicles that are packaged with gene-editing tools. These delivery techniques must be specific to enter only the cells with the disease-causing DNA and avoid the unaffected ones. (XVIVO)New AwardsThe newly funded projects expand the number of target cells and tissues, as well as the types of genome-editing technologies under investigation. One project, for example, will assess the ability of nanoparticles to deliver editing tools to up to 30 different types of target tissues. This will be the first SCGE project to use zinc finger nuclease technology as an editing tool. This technology, which differs from the better known CRISPR-Cas9 technique, already has been used in patients in a clinical trial to treat a rare metabolic disease.A different initiative will develop tools and technologies that will enable monitoring and tracking of genome-edited cells in humans to better assess the safety and efficacy of genome-editing therapies.Another new award supports the development of an epigenome editor, which can regulate how genes are expressed in target cells. And whereas most genome-editing tools use enzymes as molecular scissors to make specific cuts in DNA, two projects, including one that is newly funded, use small pieces of modified DNA to trigger the cell’s natural abilities to repair damaged DNA and edit the genome without cutting the DNA.“The DNA repair approach is very different from better-known genome-editing methods, such as CRISPR-Cas9,” said P. J. Brooks, Ph.D., program director for the NCATS Office of Rare Diseases Research and SCGE program coordinator. “When we developed the program, we felt that it was important to include a diversity of technologies and approaches.”Brooks also emphasized the inclusion of independent testing centers within the SCGE to test and validate the new delivery technologies developed in the program. He noted that such independent validation will accelerate translation of these technologies into clinical use.In addition to NCATS, other NIH Institutes and Centers managing these awards include the National Heart, Lung, and Blood Institute; National Institute of Allergy and Infectious Diseases; National Institute of Neurological Disorders and Stroke; National Institute of Arthritis and Musculoskeletal and Skin Diseases; National Institute of Biomedical Imaging and Bioengineering; National Human Genome Research Institute; and the Office of Research Infrastructure Programs.For more information about somatic cell genome editing, visit ncats.nih.gov/research/research-activities/scge. For a list of program grantees and information about their awards, see commonfund.nih.gov/editing/fundedresearch. | NIH Awards $89 Million for Additional Projects | NIH Awards $89 Million for Additional Projects | ||||||
| 15199 | ASPIRE in Action | 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.Credit: National Center for Advancing Translational SciencesApril 20212020 NCATS ASPIRE Reduction-to-Practice Challenge WinnersThe 2020 ASPIRE Reduction-to-Practice Challenge aimed to spur the development of a comprehensive integrated platform for translational innovation in pain, opioid use disorder and overdose.September 20192018 NCATS ASPIRE Design Challenges WinnersThrough the ASPIRE Design Challenges, NCATS is using the ASPIRE platform to support the Helping to End Addiction Long-termSM Initiative, or NIH HEAL InitiativeSM. The 2018 NCATS ASPIRE Design Challenges were launched to encourage innovative and catalytic approaches toward solving the opioid crisis. | ASPIRE in Action | ASPIRE in Action | ||||||
| 15124 | Translator Award Policy Guide | NCATS Other Transaction Award Policy Guide for the Biomedical Data Translator Program (PDF, 255KB). This document describes flexible policies for implementing Other Transaction (OT) awards to support the Biomedical Data Translator program. NCATS will use OT awards, as authorized through the Cures Acceleration Network, to aggregate the necessary scientific and technological expertise for its Biomedical Data Translator program. OT awards will enable NCATS to nimbly add or subtract specific expertise, tools, technologies, and approaches. | Translator Award Policy Guide | Translator Award Policy Guide | ||||||
| 15205 | 2018 NCATS ASPIRE Design Challenges Winners* | Through the ASPIRE Design Challenges, NCATS is using the ASPIRE platform to support the Helping to End Addiction Long-termSM Initiative, or NIH HEAL InitiativeSM, a transagency effort focused on improving prevention and treatment strategies for opioid misuse and addiction and enhancing pain management. The goal of the 2018 NCATS ASPIRE Design Challenges is to generate innovative and catalytic approaches toward solving the opioid crisis through the development of next-generation addiction-free analgesics with new chemistries, data-mining and analytical tools and technologies, as well as biological assays that will revolutionize discovery, development and preclinical testing of new and safer treatments for pain, opioid use disorder (OUD) and overdose. An event was held to recognize the ASPIRE Design Challenges Winners on October 28, 2019. Winners were recognized for innovative approaches towards solving the opioid crisis and revolutionizing the discovery, development and preclinical testing of next-generation, safer and non-addictive analgesics to treat pain, as well as new treatments for opioid use disorder (OUD) and overdose. Each awardee gave a 10-minute presentation about their proposed design. Photos from the event are accessible through the NCATS Flickr album. *According to the rules for these ASPIRE Design Challenges, non-U.S. citizens and non-permanent residents were not eligible to win a monetary prize (in whole or in part). Their participation as part of a winning team was recognized below. NCATS ASPIRE Challenge 1: Integrated Chemistry Database for Translational Innovation in Pain, Opioid Use Disorder and Overdose NCATS ASPIRE Challenge 2: Electronic Synthetic Chemistry Portal for Translational Innovation in Pain, Opioid Use Disorder and Overdose NCATS ASPIRE Challenge 3: Predictive Algorithms for Translational Innovation in Pain, Opioid Use Disorder and Overdose NCATS ASPIRE Challenge 4: Biological Assays for Translational Innovation in Pain, Opioid Use Disorder and Overdose NCATS ASPIRE Challenge 5: Integrated Solution for Translational Innovation in Pain, Opioid Use Disorder and Overdose NCATS ASPIRE Challenge 1: Integrated Chemistry Database for Translational Innovation in Pain, Opioid Use Disorder and Overdose Collaborative Drug Discovery (CDD), Inc. Integrated Preclinical, Clinical, and Post-Clinical Database Dr. Barry Bunin Dr. Krishna Dole Dr. Jacob Bloom Dr. Samantha Jeschonek Dr. Lawrence Callahan Dr. Christopher Lipinski This proposal will provide datasets linking target and off-target information about drugs with their clinical outcomes, including adverse effects and abuse rates. The resulting database will be unique in its integrated content, standardization and metadata about experimental and study design. ToxTrack Inc. Integrated Chemistry Database Dr. Thomas Luechtefeld Daniel Marsh This proposal describes an Integrated Chemistry Database (ICD), which utilizes novel technologies to further translational sciences and research. Data from many sources can be easily connected, versioned and visualized. Indiana University School of Informatics, Computing and Engineering CIPHER Knowledgebase for Pain, Opioid Abuse and Overdose Dr. David J. Wild Dr. Gaurav Chopra The project team will develop an integrated chemistry database that encodes structure, properties, simulation and experimental data for compounds useful for translational innovation in pain, opioid abuse disorder and overdose. The solution extends beyond typical existing databases to provide integration with machine learning methods. Stanford University Real-Time Text Mining to Empower Opioid Researchers Dr. Russ Altman Dr. Jake Lever This proposal will provide a living and learning database that, through advanced text mining approaches, would reflect the current knowledge available in the literature in a structured and high-quality manner. Instead of prescribing the knowledge required for addiction experts, the project team would provide them the tools to ask questions of the literature and extract meaningful data about current therapeutics. NCATS ASPIRE Challenge 2: Electronic Synthetic Chemistry Portal for Translational Innovation in Pain, Opioid Use Disorder and Overdose University of Michigan A Simple Electronic Notebook for the Future of Chemistry Dr. Tim Cernak By modelling the electronic Synthetic Chemistry Portal (eSCP) on a traditional simple and inviting actual paper notebook, and providing a free and open source software, the project team aspires to engage a majority of chemistry users. Labs around the world will be encouraged to publish apps along with their manuscripts, to aid in the reproducibility of their science. Features of the electronic notebook will be the ability to import reaction templates, and the ability to export reaction data in a machine-readable format. Institute of Organic Chemistry and Institute of Toxicology and Genetics, KIT, Germany An eSCP as infrastructure of distributed eLNs Dr. Nicole Jung Dr. Stefan Brase Dr. Pierre Tremouilhac Felix Bach Dr. Ravindra Peravali The electronic Synthetic Chemistry Portal (eSCP) is an information system for synthetic chemists but also for medicinal chemists and biologists to store, collect, share, search and analyze data. The portal will support innovation in Pain, Opioid Use Disorder and Overdose with respect to implemented tools, algorithms and connected information but the described concept may be used for other specific projects or a generic approach. NCATS ASPIRE Challenge 3: Predictive Algorithms for Translational Innovation in Pain, Opioid Use Disorder and Overdose University of Pittsburgh Deep and Generative Structure-Based Models for Drug Discovery Dr. David Koes Dr. Matthew Ragoza Jocelyn Sunseri Paul Francoeur Jonathan King The project team proposes to make open source tools and resources available to researchers that enable the rapid identification and optimization of small molecule therapeutics for structure-enabled molecular targets for pain and opioid abuse. Georgia Institute of Technology Predictive Algorithms for Pain, Opioid Abuse Disorder and Overdose Dr. Jeffrey Skolnick Dr. Mu Gao Dr. Hongyi Zhao To accelerate the discovery of ideal small molecules, the project team will design novel predictive machine learning (ML) modules. The goal is to recognize, in a diverse collection of ligands, the essential structural and functional properties for the desired mode of action. Allchemy Predictive Algorithms for Translational Innovation in Pain, Opioid Use Disorder and Overdose Dr. Bartosz Grzybowski Dr. Sara Szymkuc Dr. Wiktor Beker Dr. Rafal Roszak Allchemy proposes developing a software platform that will facilitate discovery of new, selective, and non-addictive analgesics. Overall, the proposed technology is unique in that it considers synthesizability of the virtual candidates with accurate AI evaluation of their biological activity. Jacobs School of Medicine and Biomedical Sciences, State University of New York (SUNY), Buffalo Optimum Analgesic Discovery by Multiscale Interactomic Profiling Dr. Ram Samudrala Dr. Gaurav Chopra The project team developed the Computational Analysis of Novel Drug Opportunities (CANDO) platform for shot-gun repurposing: to screen every drug against every disease. Here, the project team will use the multiscale interactomic profiling approach implemented in CANDO to analyze the compounds in the library, as well as virtually design new molecules that will lead to the discovery of optimal nonaddictive analgesics. ToxTrack Inc. Deep and Active Learning for Analgesics Dr. Thomas Luechtefeld Daniel Marsh This proposal describes Deep and Active Learning for Analgesics (DALA)—a platform to accelerate research of analgesics and opioid-related addition and treatments. DALA is able combine large datasets to target multiple endpoints within the same neural network, and its ability to predict for new endpoints will increase with each new endpoint. NCATS ASPIRE Challenge 4: Biological Assays for Translational Innovation in Pain, Opioid Use Disorder and Overdose AnaBios Ex Vivo Human Model of Pain for Translational Drug Discovery Dr. Andre Ghetti Dr. Anh-Tuan Ton Dr. Tim Indersmitten Yannick Miron, M.Sc. The project team proposes a novel preclinical discovery strategy, which explores the physiology and pharmacology of ex vivo human sensory neurons isolated from dorsal root ganglia of organ donors. The new strategy of testing novel non-addictive analgesics in sensory neurons of patients with chronic pain will result in a clear translational advantage over canonical cell lines and animal models. Columbia University Patient-on-a-Chip Exosome Bioassay for Opioid Abuse Disorders Dr. Gordana Vunjak-Novakovic Dr. Jorienne de Nooij Dr. Kacey Ronaldson-Bouchard Naveed Tavakol The project team will establish a “patient on a chip” platform consisting of sensory neuron, liver and heart tissues, connected by vascular perfusion. The “patient on a chip” will be validated against clinical data, to develop a reproducible testing system with strong impact on the NIH Helping to End Addiction Long-termSM Initiative, or the NIH HEAL InitiativeSM. Montana Molecular Physiologically Relevant, Phenotypic Assay for Pain Dr. Thomas Hughes Anne Marie Quinn, M.P.H. Dr. Frances Lefcort To create a phenotypic assay in which physiologically relevant, human nociceptors in culture are stimulated with known pain stimuli that produce fluorescence changes in genetically encoded fluorescent biosensors for second messengers, the project team envisions development of a new viral vector that replicates in iPSC cells such that it is maintained, episomally, in dividing cells and avoids silencing. Symmetric Computing Spectral Assay for Screening MOR Allosteric Modulators Richard Anderson Dr. Kimberly Stieglitz The project team proposes to develop a novel high throughput assay that will be useful for the rapid verification of the binding of small molecules to an allosteric site on the mu-opioid receptor protein as well as other proteins targeted for drug discovery. CiBots Biological Assay for Next Generation Analgesics (BANGA) Dr. Babak Esmaeli-Azad Dr. James Zapf Dr. Evan Snyder Dr. Mark H. Ellismann Dr. Shaochen Chen To produce “ex-vivo” neuronal tissues that replicate multicellular interactions seen in authentic human tissues, the project team will create a potentially game-changing process by knitting together a number of extant cutting-edge technologies. The Biological Assay for Next Generation Analgesics (BANGA) approach is a dramatic shift from the commonly used recombinant lines or animal cells and could revolutionize the discovery of analgesics. NCATS ASPIRE Challenge 5: Integrated Solution for Translational Innovation in Pain, Opioid Use Disorder and Overdose Arizona State University Integrated Discovery Chemputer Toward Addiction Free Opiates Dr. Sara Walker Dr. Leroy Cronin Dr. Hessam Mehr Dr. Laia Nadal Mathew Craven Dr. Phil Kitson Dr. Gerado Aragon-Camarasa The project team will develop an integrated solution that leverages our world leading Chemputer invention for the design, encoding, discovery and synthesis of new candidate molecular entities that will be explored as addiction-free-opiates (AFOs). The outcomes will be an unprecedented set of new compound libraries, based upon either the known opioid structures, biochemical properties, or physical properties as well as assessing bioavailability and pharmokinetics. Contact Dobrila D. Rudnicki, Ph.D. | ASPIRE in Action | ASPIRE in Action | ||||||
| 15154 | Discovering Science Yet Driven by the Heart | Anandi Krishnan, Ph.D. Instructor, Department of Pathology, Stanford University profiles.stanford.edu/anandi-krishnan Anyone making their way in the world has had several influences, positive and negative, that shape their present. For me, those contributions were the wealth of experiences that an ever-striving middle class home in India affords—quiet conviction, grit and a certain sanctity around education, scholarship and giving back to society. But beyond the home, it was really love that led me into a scientific career. Joining hands with my husband right after earning my bachelor’s degree in engineering, I left India to do doctoral work in the United States at what turned out to be a most fortunate and influential choice: the laboratory of Dr. Erwin Vogler and the study of blood protein/surface interactions and mechanisms of thrombosis. Dr. Vogler was a committed mentor who shared his time and wisdom generously, and methodically fostered my growth as a translational scientist well before such a term was the trend. He had an uncompromising commitment to curiosity-driven basic research, and in our laboratory, we often found ourselves enthusiastically tinkering with various biological and physical systems. I would say that it is really here that I developed my deep love for science, and that spark is what has kept me pursuing science through various life hurdles. In securing a postdoctoral fellowship grant with the American Heart Association Bugher Foundation (a multi-university research network bringing together basic and clinical research trainees), I became completely immersed within the laboratories of Richard Becker and Bruce Sullenger, as well as the Duke Clinical and Translational Science Institute. However, despite substantial early research progress, my research career came to a halt at the crucial postdoc-to-faculty transition because of multiple family medical circumstances. Although I did not immediately see a path forward, I had previous and current mentors to guide me and help me prepare for the NCATS research re-entry grant opportunity. The nearly two years of the re-entry research that ensued opened a new research direction for me. The focused, protected time; a dedicated mentorship team; and wide access to cutting-edge university infrastructure immediately facilitated the next transition—my current career development award. As a woman in science who has navigated uncommon pathways to sustainable research, I sincerely hope my story will send a message to young women everywhere to persist in following their passion and to believe firmly that supportive doors do come open for them. I would only add that I am still learning the skills necessary for a successful career in translational science and work every day to overcome my own fears and doubts as I move forward. I would like to thank the National Institutes of Health—specifically, the NCATS—for the remarkable research reentry opportunity, as well as the National Human Genome Research Institute, for my current mentored career development award that has facilitated continued research. I hope my story will serve to inspire others to pursue science despite overwhelming life challenges. Current Research Platelets are anucleate cells that circulate in the blood and play critical roles in multiple processes and diseases, from their traditional function in hemostasis and wound healing to inflammation, immunity, cancer metastasis, and angiogenesis. Evidence is evolving that the molecular signature of platelets may be changed in disease conditions where these processes are altered. Therefore, the study of the molecular mechanisms that control the activation and aggregation of platelets will lead to the design of diagnostic tests and therapeutic strategies to treat platelet deficiencies. My research uses next-generation RNA sequencing and advanced bioinformatics analyses to develop signatures of disease and therapy in a model cancer patient group—myeloproliferative neoplasms. To the best of my knowledge, my current work is the first comprehensive RNA-sequencing study of the platelet transcriptome in blood malignancies and will be the first to demonstrate integrated metadimensional analysis of patient genetic, transcriptomic and clinical data in these cohorts. Importantly, although blood diseases are a natural place to begin using these approaches given the ease of access and measurement, results will be generalizable to other types of genetic and rare diseases. The innovative data and the systems genomics approaches, built directly on the strengths of my mentorship team, will generate new knowledge about human disease and biology. | Discovering Science Yet Driven by the Heart | Discovering Science Yet Driven by the Heart | ||||||
| 15151 | Working to Reduce Cancer Disparities and Fulfilling Childhood Dreams | Jennifer Cunningham Erves, Ph.D., M.P.H., M.A.Ed., M.S., CHES Assistant Professor of Research, Department of Internal Medicine, Meharry Medical College medicine.mc.vanderbilt.edu/person/Jennifer-cunningham-erves-phd-mph-med-ms-ches Improving cancer health disparities has always been a childhood dream of mine. As a doctoral student, I conducted a mixed-methods study with African American (AA) mothers on their intentions to vaccinate their daughters for human papillomavirus (HPV). I proceeded to do a postdoctoral fellowship in community engagement, in which I did a survey of parental willingness to allow their child to participate in HPV vaccine clinical trials. During this time, I transitioned to assistant professor at Meharry Medical College and set a career goal to become an independent researcher who makes a significant contribution to the field of public health oncology, focusing on cancer prevention behaviors and the reduction of cancer disparities. In 2015, I identified research mentors and applied for and received an NCATS diversity supplement under the Vanderbilt Institute for Clinical and Translational Research Grant. This award allowed me to gain clinical and translational science training to increase the reach and relevance of my work. I was immersed in didactic training to learn more about foundational concepts related to clinical research. I also took coursework at Vanderbilt University (e.g., clinical trials, grant writing, statistics), worked on my mentors’ projects and networked at conferences to gain practical clinical research experience. This entire experience allowed me to view the research process with a new perspective. While I engaged in didactic training, I also was involved in practical research to promote my understanding of clinical and translational science. My research identified immunization stakeholder views (AA families, providers, pharmacists, and school and health department staff) about alternative settings—pharmacies, health departments and schools—to increase HPV vaccine rates. This work provided preliminary data to extend this research as a Vanderbilt Patient-Centered Outcomes Research (PCOR) Career Knowledge, Education, and Training (K12) Scholar. My research elicited the opinions of AA families on the needs and preferences in decision-making regarding HPV vaccination. Findings from both studies indicated AA families prefer the medical home to alternative settings to receive the HPV vaccine, and the vaccine-hesitant families differ in educational needs and channels for HPV, cancer and vaccine education. During this time, I was selected as a Leading Emerging and Diverse Scientists to Success Fellow (2016–2017) and an Expanding National Capacity in PCOR through Training Fellow (2017–2018) to gain the skills to launch my research career. More recently, I applied for a National Institutes of Health (NIH) K01 grant, with continued support from my mentors, using the data from the supplement and my internal K12 award. This award would allow me to learn more about behavioral intervention development for cancer prevention using health communication and designing clinical trials to assess the efficacy of interventions to promote HPV vaccination for cancer prevention. My initial application did not get a priority score for funding; however, my resubmission received a significantly higher score. Overall, the diversity supplement provided the foundation for me to secure my non-tenure-track faculty position, obtain my internal K12 award and extend my work to apply for and receive favorable reviews for my K01 application. All minority investigators should pursue these opportunities with a committed mentoring team to serve as “stepping stones” for career development. Current Research My research experiences have yielded an extensive knowledge base and skillset in biology, public health and community engagement. Through my current research agenda, I seek to develop and implement a tailored health communication intervention to increase HPV vaccination among children of vaccine-hesitant parents. I am using a community-engaged research approach to understand health communication strategies and their effect on vaccine-hesitant parental decision-making for HPV vaccination. This work promotes translational research by developing a behavioral intervention that potentially could be identified as an evidence-based practice to promote HPV vaccination among vaccine-hesitant parents. | Working to Reduce Cancer Disparities and Fulfilling Childhood Dreams | Working to Reduce Cancer Disparities and Fulfilling Childhood Dreams | ||||||
| 15163 | CTSA Program Diversity, Re-entry and Reintegration Supplement Awardee Profiles | .cardbox { box-shadow: 0 2px 2px 0 rgba(0,0,0,0.14), 0 3px 1px -2px rgba(0,0,0,0.12), 0 1px 4px 0 rgba(0,0,0,0.2); min-width: 40%; border-radius: 5px; /* margin-left: 30px; width: 85%; */ background-color: #eff6f7; border-left: 9px solid #662e6b; min-height: 150px; margin-bottom: 30px; display: flex; } .cardimg { vertical-align: center; max-width: 100%; height: auto; padding-top: 20px; padding-left: 10px; display: table-cell; } .cardtext{ display: table-cell; padding-left: 10px; vertical-align: top; } (function ($) { $( ".border" ).after( "" ); })(jQuery); The CTSA Program is committed to improving the diversity of the workforce and to supporting re-entry into active research careers for those individuals that have taken an eligible hiatus from research. Below we highlight outstanding CTSA Program diversity and re-entry research supplement awardees who have used the supplement to advance their careers and fulfill their research objectives. Learn more about the CTSA Program Diversity, Re-entry and Reintegration Supplements. Ewan K. Cobran, Ph.D. Assistant Professor, Department of Clinical and Administrative Pharmacy, University of Georgia Decreasing Cancer Disparities by Increasing Patient Understanding of Prognostic Genetics Profile Jennifer Cunningham Erves, Ph.D., M.P.H., M.A.Ed., M.S., CHES Assistant Professor of Research, Department of Internal Medicine, Meharry Medical College Working to Reduce Cancer Disparities and Fulfilling Childhood Dreams Profile Anandi Krishnan, Ph.D. Instructor, Department of Pathology, Stanford University Discovering Science Yet Driven by the Heart Profile Aisha Langford, Ph.D, M.P.H. Assistant Professor, Department of Population Health, New York University School of Medicine Celebrating Wins Both Big and Small! Profile Roland A. Matsouaka, Ph.D. Assistant Professor, Department of Biostatistics and Bioinformatics & Duke Clinical Research Institute, Duke University School of Medicine Using Abstract Math to Solve Real-World Crises Profile Juan Vasquez, M.D. Assistant Professor, Department of Pediatrics (Hematology/Oncology), Yale School of Medicine Harnessing the Immune System to Help Treat Cancer Profile | CTSA Program supplement awardees have used the supplement to advance their careers and fulfill their research objectives. | /sites/default/files/diversity_reentry_reintegration_landing_social-image_400x400_1.jpg | CTSA Program Diversity, Re-entry and Reintegration Profiles | CTSA Program supplement awardees have used the supplement to advance their careers and fulfill their research objectives. | /sites/default/files/diversity_reentry_reintegration_landing_social-image_400x400_2.jpg | CTSA Program Diversity, Re-entry and Reintegration Profiles |
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