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| 7703 | NOT-TR-17-023 | Application Process Step I: Challenge Prospective applicants must complete a challenge to initiate the application process to ensure that they have the requisite skills to develop a reasoning tool. The challenge tasks themselves are designed to provide important background and insight into the task of building a biomedical reasoning tool prototype for this program and thereby improve the quality of proposals received. The challenge is a multilevel computational exercise. The challenge may be completed as an individual, or as a team that will be part of the proposal, provided the team is using a common login. Upon successful completion of each level, additional sections of the funding opportunity will be revealed. To begin the challenge, go to https://ncats.io/challenge/start. Step II: Concept Letter The computational exercises control access to the instructions, so completion of the computational exercises will enable applicants to view instructions that are necessary to submit the required concept letter. Step III: Full Proposal After objective review of concept letters is completed; successful teams will receive written notification with instructions for submitting a full proposal and giving a virtual presentation to the review panel. Key events and dates are provided in the table below. Instructions for submitting the concept letter will be provided in the funding opportunity announcement upon completion of the challenge. Expertise and Skills Successful completion of the application process will require applicants to have specific skills related to translational research and software development. Applicants will need to demonstrate technical skills, including familiarity with web communication protocols, a variety of programming languages and software stack, and general algorithmic techniques in the areas of artificial intelligence, machine learning, and knowledge engineering, as well as problem solving skills, especially creativity and persistence. For applicants who are familiar with the specific languages and packages most useful for solving different tasks, the entire challenge process may take between 2 and 8 hours to complete; applicants without the necessary background in each of these areas may take substantially longer. Eligibility All U.S. and foreign organizations and U.S. citizens are eligible to apply. This funding opportunity is open to U.S. and foreign organizations, including academic institutions and commercial entities; subcontracts are allowed. U.S. citizens may also apply as individuals without an organizational affiliation and may be direct recipients of an award. Non-citizen individuals residing in the U.S. or foreign country who are not affiliated with either a U.S. or foreign organization are not eligible to be direct recipients of an award. Key Events and Dates Key Events Dates Action Needed by Applicants Challenge opens (Step I) Sept. 7, 2017 Solve challenge puzzle to complete Step I of the process. Concept letters due (Step II) Sept. 22, 2017 Submit concept letter following instructions provided through the challenge before 11 p.m. EDT* to complete Step II of the process. Objective review of concept letters completed Oct. 2, 2017 Receive written notification with instructions for submitting a full proposal and giving a virtual presentation to the review panel if the outcome of objective review is favorable. Biomedical Data Translator public meeting Oct. 25, 2017 Attend meeting in person (optional); Webex will be available. Written, full proposal for reasoning tool development that includes milestones due (Step III) Nov. 20, 2017 Submit by email the 10-month plan and milestones by 5 p.m. local time* [action taken by AOR for organizations or signing representative for individuals] to initiate Step III of the process. Virtual presentations of proof-of-concept software and 10-month project plan Nov. 28-29, 2017 Participate in the virtual meeting with the review panel to complete Step III of the process.** Awards announced January 2018 * Letters and proposals received after these times will not be accepted. ** Applicants should save-the-date to ensure availability for the virtual interview. | NOT-TR-17-023 | NOT-TR-17-023 | ||||||
| 7697 | Gene Therapy for Rare, Pediatric Condition Moves Closer to Reaching U.S. Patients | Credit: NIH National Human Genome Research Institute PhotoA collaboration between researchers from NCATS’ Therapeutics for Rare and Neglected Diseases (TRND) program and Agilis Biotherapeutics, Inc., of Cambridge, Massachusetts, has resulted in a gene therapy for the rare pediatric condition aromatic L-amino acid decarboxylase (AADC) deficiency moving closer to market years ahead of schedule. Following a recent meeting with the U.S. Food and Drug Administration (FDA), Agilis will begin the process of seeking final approval to treat U.S. patients with this life-threatening disorder (PDF - 67KB).The AADC enzyme is necessary to produce important chemical messengers in the brain and other parts of the central nervous system. Children with AADC deficiency commonly experience severe developmental delays, weak muscle tone and involuntary movement of the limbs. There is no approved treatment for AADC deficiency, and patients with severe forms of the disorder usually die in the first decade of life.The gene therapy, called AGIL-AADC, restores AADC enzyme production in the brain. Agilis licensed the gene therapy from National Taiwan University, where investigators had conducted clinical studies on patients with severe AADC deficiency. While these studies showed drastic and sustained improvements following a single dose of AGIL-AADC, the investigators lacked preclinical data required by the FDA. The small market for this ultra-rare disease and the need to conduct additional studies threatened to stall the project.In 2016, through a cooperative research and development agreement, Agilis partnered with NCATS scientists in the TRND program, which is designed to speed the development of new treatments for diseases with significant unmet medical needs. TRND scientists supported the development of AGIL-AADC in several ways, including conducting preclinical safety studies and producing AGIL-AADC in a way that met FDA requirements.In just over a year, these efforts led to a meeting with the FDA to review the preclinical, clinical and manufacturing data. In an unusual step, FDA reviewers determined that Agilis did not have to repeat clinical trials in the United States, clearing the path for the company to file a Biologics Licensing Application (BLA), which, if approved, would allow the company to market AGIL-AADC to patients.“The successful partnership between the Agilis and TRND teams helped shorten the timeline by years for this potentially lifesaving therapy to reach patients,” said Nora Yang, Ph.D., director of portfolio management and strategic operations in NCATS’ Division of Preclinical Innovation.As Agilis prepares to file a BLA, the TRND and Agilis team is conducting an epidemiological study to better define the prevalence of AADC and identify patients who might gain access to this potentially lifesaving gene therapy.AGIL-AADC is poised to be among the first FDA-approved gene therapies for treating a central nervous system disorder. Moreover, it would be the first therapy supported by the TRND program to receive marketing approval from the FDA and become available to patients.“This significant regulatory milestone demonstrates how the TRND program plays a catalytic role in enabling and accelerating progress on rare disease therapies through public-private partnerships,” Yang said.Posted September 2017 | An NCATS collaboration has resulted in moving a gene therapy for the rare pediatric condition aromatic L-amino acid decarboxylase (AADC) deficiency closer to market years ahead of schedule. | /sites/default/files/double-helix-900px.jpg | Gene therapy for rare, pediatric condition closer to reaching patients | An NCATS collaboration has resulted in moving a gene therapy for the rare pediatric condition aromatic L-amino acid decarboxylase (AADC) deficiency closer to market years ahead of schedule. | /sites/default/files/double-helix-900px.jpg | Gene therapy for rare, pediatric condition closer to reaching patients | ||
| 7652 | Assay Guidance Workshop Agenda — October 23, 2017 | Monday, October 23, 2017 — 8:00 a.m. - 6:30 p.m. ET University of North Carolina at Chapel Hill, North Carolina, 27599, USA 8:00 - 8:15 a.m.: Robust or Go Bust: An Introduction to the Assay Guidance Manual G. Sitta Sittampalam, Ph.D., NCATS, NIH 8:20 - 9:00 a.m.: Statistical Design of Experiments for Assay Development Steven D. Kahl, Eli Lilly and Company 9:10 - 9:50 a.m.: Treating Cells as Reagents to Design Reproducible Screening Assays Terry Riss, Ph.D., Promega Corporation 10:00 - 10:15 a.m.: Beverage Break 10:15 - 10:55 a.m.: Assay Development for High Content Screening O. Joseph Trask, Jr., Ph.D., PerkinElmer 11:05 - 11:45 a.m.: Label-Free HTS Assays using Mass Spectrometry James McGee, Ph.D., Eli Lilly and Company 11:55 a.m. - 12:45 p.m.: Lunch 12:45 - 1:25 p.m.: Assay Interpretation: Studies in Mechanisms and Methods in Assay Interferences Douglas Auld, Ph.D., Novartis Institutes for BioMedical Research 1:35 - 2:15 p.m.: Assay Interference by Chemical Reactivity Jayme L. Dahlin, M.D., Ph.D., Brigham and Women's Hospital 2:15 - 2:30 p.m.: Beverage Break 2:30 - 3:10 p.m.: Basic Assay Statistics, Data Analysis and Rules of Thumb Thomas D.Y. Chung, Ph.D., Mayo Clinic 3:20 - 4:00 p.m.: Reproducibility and Differentiability of Compound Potency Results from Screening Assays in Drug Discovery V. Devanarayan, Ph.D., AbbVie, Inc. 4:10 - 4:50 p.m.: Assay Operations: Keeping your Assays Robust and Reproducible Jeffrey R. Weidner, Ph.D., QualSci Consulting, LLC 4:50 - 5:00 p.m.: Beverage Break 5:00 - 5:40 p.m.: In Vitro Toxicological Testing using a qHTS Platform Menghang Xia, Ph.D., NCATS, NIH 5:50 - 6:30 p.m.: In Vitro Assessments of ADME Properties of Lead Compounds Xin Xu, Ph.D., NCATS, NIH 6:30 - 7:30 p.m.: Reception | Assay Guidance Workshop Agenda — October 2017 | Assay Guidance Workshop Agenda — October 2017 | ||||||
| 7616 | NCATS Furthers Efforts to Create a Data Ecosystem to Explore Disease Connections | Translational Science Highlight A computational tool that improves connections among the mounting levels of disparate biomedical data types would transform translational science by enabling connections to diverse and comprehensive disease and biology information. Could more effective targets and treatments for common and rare diseases already exist, but have eluded researchers because disparate pieces of information were not connected? “Enabling comprehensive discovery of commonalities among diseases would turbo-charge the translational process,” said NCATS Director Christopher P. Austin, M.D. “This is the goal for our ambitious Biomedical Data Translator program.” Powerful new technologies are reshaping the biomedical research landscape, enabling scientists to map and decipher the 3 billion chemical letters that make up the human genome and unravel the molecular mysteries of all kinds of diseases. It is becoming possible to identify the hundreds of environmental stimuli and chemicals people are exposed to each day. Electronic medical records contain warehouses of patient information and clinical databases house details on genomic and environmental variants that can affect disease susceptibility. While these technologies produce massive amounts of data, the pace of data generation has largely outstripped researchers’ ability to make sense of the results. Ideally, scientists could easily mine data from different sources to gain new insights into disease causes and biology as well as the relationship between disease biology and clinical signs and symptoms. However, disconnected data sources and lack of understanding of how disparate data types — such as genomic, cellular and patient — relate to each other has hindered the pace of progress. Through its Translator program, NCATS is supporting research to develop a computational platform that enables connections among conventionally siloed data types. Translator aims to bring these together in an ecosystem that will reveal complex relationships that help scientists better understand disease and generate hypotheses and treatment options. “If successful, Translator will help scientists use data more effectively,” said Noel Southall, Ph.D., a program leader in the NCATS Division of Preclinical Innovation. “This tool will bring into focus relationships among data, enabling the researcher to develop hypotheses based on connections that otherwise were not apparent.” An inability to see connections among data can be frustrating, especially in translational science. For example, researchers might find, through a drug screening, that a drug known to treat one disease is useful against another disease. When scientists dig deeper into the literature, they often find the connection between that drug and that disease was already there. “Researchers spend a lot of time rediscovering things we already knew because the connection between knowledge sources doesn’t exist,” Southall said. “We’re frequently unable to see the relationships among different data types that can affect clinical outcomes.” Feasibility Testing When NCATS awarded its initial Translator funding in fall 2016, 11 teams were charged with working together to develop “demonstration” projects aimed at assessing whether a biomedical data translator could be created. There are now 12 teams determining the feasibility of building Translator by focusing their efforts on data integration and analysis. Killer T cells surround a cancer cell. Alex Ritter, Jennifer Lippincott Schwartz and Gillian Griffiths/National Institutes of Health Photo “We need these projects to give us a sense of what is possible and how to move forward,” Southall said. For each demonstration project, awardees are identifying data sources that would be needed for a comprehensive Translator and designing ways to combine the data to address a specific biomedical problem. The projects are wide-ranging. While some researchers examine the impact of environmental exposures on the onset or worsening of disease, others evaluate the ability to help patients for whom existing approaches have failed to identify the origin of disease symptoms. Still other scientists focus on understanding relationships between common and rare diseases. The demonstration projects are especially collaborative. “A single group cannot build the Translator,” said Christine M. Colvis, Ph.D., NCATS Drug Development Partnership Programs director. “Scientifically and technically, the research teams have realized that to make the data interoperable and gain understanding from them, they must work closely and take advantage of complementary expertise.” In one project, for example, three groups of researchers with different areas of expertise have combined their resources to study Fanconi anemia, a rare, inherited disease that affects the bone marrow and can cause developmental problems and cancer. Data analytics experts at the University of California, San Diego and scientists at the Renaissance Computing Institute and the University of North Carolina at Chapel Hill, who have experience working with environmental data, are working with clinicians and scientists from Oregon Health & Science University in Portland to test a computational strategy to study variations in genes and environmental exposures and how they might play roles in the disease. Transcending the Languages of Biology and Disease “Speaking different languages sets up translational roadblocks to seeing relationships among data,” Colvis said. “If we could redefine diseases based on the data we bring together in Translator, we might see connections among illnesses not based on what organ systems are involved but rather the biology underlying those conditions. Translator has the potential to get a basic scientist to begin to think about downstream clinical connections in a different way.” Translator queries could run from the more general to the very specific. The system might be asked, for example, to identify all diseases with a particular symptom that affect a particular cell type. Or a query could request all molecular pathways that, when disrupted, lead to malfunctions of cellular structures in a particular organ in people with specific genomic characteristics. Translator might ultimately change how scientists and clinicians think about disease and treatment. Physicians and biologists tend to think of disease in different ways and speak different languages. Physicians diagnose and treat disease based on signs and symptoms affecting specific organs, while biomedical researchers often consider disease in terms of molecular changes in specific proteins, pathways or cell types. Supporting High-Risk, High-Reward Research NCATS funds Translator through its Cures Acceleration Network (CAN), which includes Other Transaction authority (OTA). With CAN and OTA, NCATS has more funding flexibility to support high-risk, high-reward programs such as Translator. In one case, a team realized after six months that its demonstration project idea wouldn’t work. But the “failure” turned out to be helpful. “The idea was very innovative and made us think differently about how we consider data and how to build Translator,” Southall said. “We are assessing feasibility, and it’s okay if some of the things we try fail as long as we learn from the experience, including which directions work and which don’t.” “We’re still trying to figure out what’s possible,” he continued. “We originally thought about Translator as a ‘big database lookup.’ If you put all the facts and databases in one place, eventually you could keep asking questions and stitch together answers.” Now, less than one year later, Southall and his colleagues are exploring a somewhat different model that would also enable researchers to infer data that are not even available yet. Posted August 2017 | NCATS Biomedical Data Translator scientists aim to create a computational tool to improve connections among biomedical data types and provide insights into disease biology and treatments. | /sites/default/files/translator-feature_900px_.jpg | NCATS Furthers Efforts to Create a Data Ecosystem to Explore Disease | NCATS Biomedical Data Translator scientists aim to create a computational tool to improve connections among biomedical data types and provide insights into disease biology and treatments. | /sites/default/files/translator-feature_900px_.jpg | NCATS Furthers Efforts to Create a Data Ecosystem to Explore Disease | ||
| 7606 | NCATS Enables Scientists, Community Clinicians to Collaborate on Health Initiatives | Translational Science Highlight NCATS’ Clinical and Translational Science Awards Program supports public health improvements by enabling innovative research partnerships between university scientists and community clinicians. Rhonda G. Kost, M.D., and Jonathan N. Tobin, Ph.D., who co-direct the Community Engaged Research Core at The Rockefeller University’s Center for Clinical and Translational Science (CCTS), regularly work together to facilitate conversations among three groups of people: basic scientists in the laboratory, clinicians in the New York City area and health care providers at community health centers. A clear channel of communication across these groups is a necessity, and for NCATS, this work is helping to overcome a key translational research hurdle. “Basic scientists and community physicians tend to speak different languages and have different priorities,” said Kost, the academic navigator and clinical research officer at Rockefeller’s CCTS, an NCATS Clinical and Translational Science Awards (CTSA) Program hub. “Our goal is for them to understand each other and where their interests align, and find ways to more quickly move science discoveries out of the lab and into interventions to improve people’s lives.” With this goal in mind, Rockefeller’s CCTS leadership and investigators partnered with Clinical Directors Network (CDN), a practice-based research network of clinicians working in underserved communities, with sites in the New York City area and across the country, to create a program that fosters interdisciplinary research partnerships. This collaborative effort, called the community-engaged research navigation (CEnR-Nav) program, has resulted in more than 40 research projects, including more than 20 clinical research studies. Rockefeller CCTS participants frequently include clinical scholar trainees and early career physician-scientists, in addition to faculty and graduate students. Community Acquired MRSA Project research team meeting with members from The Rockefeller University Center for Clinical and Translational Science, Clinical Directors Network and community health centers. The Rockefeller University and Clinical Directors Network, Inc. Photo “We want to engage community and research participants in all phases of research that we do, including designing studies and thinking about how the information can best be converted into programs and policies that have an impact on public health,” added Rockefeller CCTS Director Barry Coller, M.D. Tobin, who also is CDN president/CEO, works closely with Kost to navigate and foster community engagement opportunities. Together, they have wide-ranging responsibilities, from coordinating meetings and matchmaking among potential collaborators to helping participants brainstorm research questions of mutual interest and developing study goals and methodological approaches. Although community health concerns and needs often drive research projects, ideas can come from many sources, including a critical laboratory discovery; conversations among patients, clinicians and investigators; or a grant proposal opportunity. Understanding Bacterial Resistance For example, Rockefeller scientists parlayed a faculty member’s expertise in studying drug-resistant bacteria outbreaks in hospitals on three continents and New York City into a collaboration with New York City-area community health centers to monitor drug resistance in households. Rockefeller microbiologist Alexander Tomasz, Ph.D., studied the causes of drug resistance in outbreaks of methicillin-resistant Staphylococcus aureus (MRSA), a potentially life-threatening bacterial infection, in hospitals in England, Japan and Brazil. In earlier work, he had discovered a change in a gene that makes bacteria antibiotic resistant. Tomasz was also studying patterns of hospital-acquired MRSA infection in New York City hospitals and had organized a surveillance network that sent MRSA samples to his lab for detailed examination. Coller brought Tomasz, Tobin and Kost together to study the distribution, causes, treatments and outcomes of community-acquired MRSA. They partnered with community clinicians to establish a community-based surveillance system to determine whether MRSA resistance was a problem in health centers and households and, if so, identify the molecular underpinning of the resistance. CTSA Program support then enabled the research team to collect preliminary prospective data and identify research questions of interest to investigators and clinicians. Additional NCATS funding supported the enrollment of 150 patients across CDN sites in New York and Texas to collect biological specimens and clinical information to help understand the dynamics of the infection in the community. The research has resulted in a comparative effectiveness study, created with patient and community clinician perspectives in mind and funded by the Patient-Centered Outcomes Research Institute, to examine approaches for preventing recurrence of community-acquired MRSA. The scientists, clinicians and community partners are studying patients, household members and home environments as potential reservoirs of infections and are characterizing the bacteria living on the surfaces of homes and in household members. The goal is to determine whether evidence-based hospital infection prevention procedures can be implemented in homes by community health workers and whether these procedures are effective in reducing reinfection and household transmission. Affecting Birth Outcomes Tobin previously also led an NIH National Institute of Mental Health-funded study that involved 14 New York City hospitals and health centers. For this project, clinicians enrolled over 1,200 pregnant teens who were randomized to receive either usual individual prenatal care or prenatal care in a group setting. The researchers wanted to see which model was more effective in reducing the incidence of low-birthweight babies. They found that teenagers receiving prenatal care in the group setting were less likely to have smaller babies for their gestational ages and had better birth outcomes, including babies with more normal birth weights and fewer days in the neonatal intensive care unit. With support from Rockefeller’s CCTS and the Sackler Foundation, Tobin and his community colleagues reconvened this partnership and built a research data collection system based on extracting routine clinical care observations from the electronic health records of primary care practices. With Rockefeller investigators Jan Breslow, M.D., and Peter Holt, M.D., the research team is currently examining diet, behavioral and genetic influences, and other factors that can affect birth outcomes and cardiometabolic health in this teenaged population affected by health disparities. The Rockefeller CCTS CEnR-Nav program approach aligns with the CTSA Program goal of including the community perspective in research. “It’s important for us to get a good sense of what the community is concerned about,” Coller said. “The CEnR-Nav community engagement model brings together community members, patients, scientists and clinicians to help guide hypothesis-driven science that can lead to improved community health.” Posted August 2017 | NCATS’ CTSA Program supports public health improvements by enabling innovative research partnerships between university scientists and community clinicians. | NCATS Support Enables Collaboration on Public Health Initiatives | NCATS’ CTSA Program supports public health improvements by enabling innovative research partnerships between university scientists and community clinicians. | NCATS Support Enables Collaboration on Public Health Initiatives | ||||
| 7594 | News Brief: NCATS-Supported Study Shows Lipoic Acid May Benefit Multiple Sclerosis Patients | Rebecca Spain, M.D., M.S.P.H., a Clinical and Translational Science Awards Program scholar at Oregon Health & Science University (OHSU), found evidence suggesting that an over-the-counter antioxidant, lipoic acid, can help multiple sclerosis (MS) patients. Her study revealed a 68 percent improvement over placebo in slowing the rate of whole brain atrophy in patients with secondary progressive MS. Read the July 3, 2017, OHSU news release. Posted August 2017 | NCATS-Supported Study Shows Lipoic Acid May Benefit Multiple Sclerosis | NCATS-Supported Study Shows Lipoic Acid May Benefit Multiple Sclerosis | ||||||
| 7593 | News Brief: NYU CTSA Program Hub Hosts Science Café | The New York University (NYU) Clinical and Translational Science Institute ― an NCATS Clinical and Translational Science Awards (CTSA) Program hub ― hosted a “science café” featuring informal, open dialogue about research needs and collaboration opportunities. Nearly 100 attendees from the NYU research community and beyond participated to learn about CTSA Program-supported resources. Posted August 2017 | NYU CTSA Program Hub Hosts Science Café | NYU CTSA Program Hub Hosts Science Café | ||||||
| 7592 | News Brief: CTSA Program Hub Expands Successful Radio Show with Podcast | Since 2013, the Clinical & Translational Science Institute of Southeast Wisconsin at the Medical College of Wisconsin has aired a monthly radio segment, Discovery Radio, to inform the local community about translational research discoveries relevant to their health. Topics include biomedical research and the community, caring for children through translational science, and the NIH All of Us Research Program. In May 2017, program officials expanded Discovery Radio’s audience by launching a podcast that already has been downloaded more than 1,100 times. Learn more about Discovery Radio. Posted August 2017 | CTSA Program Hub Expands Successful Radio Show with Podcast | CTSA Program Hub Expands Successful Radio Show with Podcast | ||||||
| 7542 | NCATS Day Poster: NCATS Therapeutic Development Branch: TRND & BrIDGs | Download the poster here. | NCATS Therapeutic Development Branch: TRND & BrIDGs | NCATS Therapeutic Development Branch: TRND & BrIDGs | ||||||
| 7541 | NCATS Day Poster: Biomedical Data Translator | Download the poster here. | Biomedical Data Translator | Biomedical Data Translator |
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