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| 7349 | Small Business Award Spurs Innovation to Improve Data Use | Translational Science Highlight Through its Small Business Innovation Research program, NCATS supported the development of a technology to use electronic health records data to advance value-based and enhanced patient care. Electronic health records (EHRs) hold a wealth of information that can be used to look at how well health care providers are treating their patients and point to ways to improve care. The data can also help researchers conduct population health studies. But much of this useful clinical information is entered in the notes section of EHRs rather than into a specific field. Referred to as “unstructured data,” this information is hard to extract and use in health analyses. This bothered Dan Riskin, M.D., M.B.A., FACS, adjunct professor of surgery and biomedical informatics research at Stanford University, clinical informaticist and CEO of Vanguard Medical Technologies (VMT). He created a spin-off company from VMT, Health Fidelity, to develop technology to use this unstructured clinical data to analyze patient risk and quality from the EHR. In 2012, NCATS awarded Riskin and Health Fidelity a Small Business Innovation Research (SBIR) Phase 1 grant to develop and evaluate the technology and to help bring it to market. NCATS’ SBIR and Small Business Technology Transfer (STTR) programs speed innovation in translational science. Often, small startup companies lack the early-stage funding to carry an idea through for further investment and into commercial markets to benefit patients. Dan Riskin, M.D., M.B.A., FACS “NCATS helps bridge this translational gap by awarding grants, contracts and technical assistance to small businesses and research organizations focused on advancing translational research and technologies that will improve disease prevention, detection and treatment,” said Lili Portilla, M.P.A., director of NCATS’ SBIR and STTR programs. “The NCATS grant came at a defining time for a defining technology,” Riskin said. “Based on work from that grant, we were able to push the boundaries of unstructured data use, exploring automated assessment of cohorts, quality and risk.” Technology Can Make the Process More Cost-Effective The value-based care model provides rewards or penalties based on treatment outcomes, rather than just the volume of treatments used in the fee-for-service model. The goal is to improve the quality of care and the patient experience. But this requires analyzing patient risk and outcomes by using the right data in the right format. Manually getting the data — by having someone review each patient’s chart — is too time-consuming and cost-prohibitive. That’s where natural language processing comes in. It is a way for computers to extract meaning from human language and is used to do everything from automatically translating between languages to correcting grammar and summarizing blocks of text. Built in collaboration with Columbia University, Riskin’s technology was unique in that it was developed specifically for use with clinical language, which is highly specialized. Doctors use a lot of abbreviations and acronyms in their notes, which can make the meaning ambiguous, and this can make them especially difficult to automate. For example, “DM” in an EHR notes section could either mean “diabetes mellitus” or refer to someone’s initials. The new technology pulls out meaningful data based on the context, such as whether a medication used to treat diabetes is found elsewhere in the EHR. The software can also look for risk factors that directly affect outcomes, such as whether the patient is regularly taking their medication. Spurring High-Risk Early Development With the initial SBIR grant, Health Fidelity could program the software for natural language processing of physicians’ notes and then evaluate the precision of the platform. At the time, Health Fidelity already had undergone a first round of venture capital funding. “Still, we had quite a bit of research to do,” Riskin said. “Some of the high-risk basic science efforts funded by NCATS became foundational to the company’s first commercial product.” Riskin added that having validation from NIH’s peer review that the Health Fidelity team’s work was scientifically sound was invaluable and that value-based care is an important area of scientific research. Because of this funding, Health Fidelity was able to make great strides before many others in the field were even thinking about technologies to analyze narrative data for value-based care. Health Fidelity was therefore in a prime position when recent health care and related legislation helped accelerate the move to this model. In 2013, Health Fidelity established collaborations with University of Pittsburgh Medical Center (UPMC) and several other large institutions to pilot-test the technology. A year later, the company signed a deal with UPMC and raised $29 million in investment capital. The company has since flourished, generating revenue and giving hospitals and health systems a better way to look at patient risk and pave the way to improved care through value-based models. “The NCATS grant was crucial to the company’s success,” said Riskin. “And the work is now supporting value-based care for hundreds of thousands of patients.” Since bringing this technology to market, Riskin has handed off his leadership role at Health Fidelity and returned to VMT, where he is working to solve the next critical problem in health care through innovative technologies. “Dan’s story is just one example of how leveraging an SBIR award to obtain additional investment from the venture capital community can lead to further success,” Portilla said. “By providing early-stage funding for developing new translational bioinformatics tools, NCATS is improving health through smarter science.” Posted July 2017 | Through its small business program, NCATS supported the development of a technology to use electronic health records data to advance value-based and enhanced patient care. | /sites/default/files/riskin_900.jpg | Small Business Award Spurs Innovation to Improve Data Use | Through its small business program, NCATS supported the development of a technology to use electronic health records data to advance value-based and enhanced patient care. | /sites/default/files/riskin_900.jpg | Small Business Award Spurs Innovation to Improve Data Use | ||
| 7347 | News Brief: NCATS Announces Quarterly Update to Its Assay Guidance Manual | In July 2017, authors of NCATS’ Assay Guidance Manual (AGM) ― a free, best-practices online resource devoted to the successful development of robust, early-stage drug discovery assays ― added two new chapters and revised two others. Updated quarterly, the AGM provides step-by-step guidance for high-throughput screening, lead optimization and early phases of regulated drug development projects. Histone Acetyltransferase (HAT) Assays in Drug and Chemical Probe Discovery in the In Vitro Biochemical Assays section, describes essential experimental considerations for performing HAT assays along with practical strategies for assay optimization and validation. The content may benefit researchers performing primary, orthogonal and counter assays involving HATs in the context of drug discovery, chemical biology and molecular pharmacology. Assay Interference by Aggregation, in the Assay Artifacts and Interferences section, focuses on identifying and mitigating nonspecific bioactivity in assays due to compound aggregation. Accounting for potential bioactivity due to aggregation in assay design and readout interpretation can save significant time and resources when following up on bioactive test compounds. This chapter will benefit researchers engaged in bioassay development for drug and chemical probe discovery. Additionally, AGM authors have revised two chapters: Advanced Assay Development Guidelines for Image-Based High Content Screening and Analysis and HPLC-MS/MS for Hit Generation. The next update is scheduled for October 2017. Posted July 2017 | NCATS Announces Quarterly Update to Its Assay Guidance Manual | NCATS Announces Quarterly Update to Its Assay Guidance Manual | ||||||
| 7193 | Metarrestin for the Treatment of Pancreatic Cancer | Pancreatic cancer is the fourth leading cause of cancer-related deaths. As the disease progresses, cancer cells travel (metastasize) from the pancreas to other parts of the body. Despite substantial improvements in cancer patient survival, metastatic diseases remain ineffectively treated. The majority of pancreatic cancer patients are diagnosed only after the cancer has spread, and nearly all patients succumb to the metastatic burden. These researchers have identified both a cellular target that is prevalent in metastatic cancer cells and a compound that inhibits the metastatic process. The aim of this project is to continue to develop this therapy to prepare it for human trials. Scientific Synopsis The perinucleolar compartment (PNC) is a subcellular structure whose formation closely associates with metastatic potential of cancer cells. The PNC, which is located at the nucleolar periphery, is a dynamic subnuclear body enriched with RNA transcripts and RNA-binding proteins. It is highly prevalent in metastatic tumors, metastatically transformed cancer cell lines, and cancer stem cells. It is rarely found in normal cells, including human embryonic stem cells. A high PNC prevalence positively correlates with disease progression (stages and grades) in tested primary tumors, including breast, colorectal, and ovarian cancers, and inversely correlates with patient outcomes. Current evidence indicates that PNC prevalence reflects the metastatic capability of a given cellular population derived from solid tissue origins. Reduction of PNC prevalence was used as a phenotypic marker to screen for compounds that interfere with cellular mechanisms essential for the metastatic capability of cancer cells. This led to the discovery of a compound that was further optimized in a medicinal chemistry campaign to produce metarrestin, a compound that reduces PNC prevalence in multiple cell lines without significant impact on cell viability. Metarrestin shows anti-oncogenic properties in vitro, including inhibition of migration and invasion. It demonstrates desirable pharmacokinetic properties and bioavailability, and in an animal model of pancreatic metastasis, it significantly reduced metastatic burden in the lung and liver and extended survival. Lead Collaborator National Cancer Institute, Bethesda, MD Udo Rudloff, M.D., Ph.D. Public Health Impact An estimated 54,000 people will be diagnosed with pancreatic cancer in 2017, with an overall five-year survival rate of only 5 percent to 7 percent. Most cases are diagnosed after the cancer has progressed to an advanced stage, where the metastatic burden is high. However, even with early detection and treatment, there is near universal recurrence due to systemic metastasis. Therapy that can affect metastatic progression may improve the outcome for patients at all stages of disease. Outcomes BrIDGs program scientists completed formulation development, manufacture of Good Manufacturing Practice (GMP) drug product, and pharmacokinetic and IND-directed toxicology studies. As a result of BrIDGs support, an IND was cleared by the FDA, allowing the collaborator to initiate clinical trials. See ClinicalTrials.gov, NCT04222413. | In an animal model of pancreatic metastasis, metarrestin significantly reduced metastatic burden and extended survival. | Metarrestin for the Treatment of Pancreatic Cancer | IIn an animal model of pancreatic metastasis, metarrestin significantly reduced metastatic burden and extended survival. | Metarrestin for the Treatment of Pancreatic Cancer | ||||
| 7192 | Using the Preimplantation Factor (PIF) to Treat Graft-Versus-Host Disease | A number of diseases arise due to defects in bone marrow cells. Such conditions may be treated through bone marrow transplantation (BMT). Graft-versus-host disease (GVHD) is a major complication that can occur after BMT. The transplanted cells from the donor (graft) treat the cells of the patient (host) as “foreign.” Instead of helping the recipient, the immune cells from the donor graft attack the host’s cells, tissues and organs, as if fighting an infection. Acute GVHD soon after transplantation can be mild, moderate or severe; it can even fatal if not controlled. The disease may also present as a later-onset chronic condition. Therapy for chronic GVHD is associated with a lifetime of immune-suppressive drugs that can have long-term side effects and toxicities. The preimplantation factor (PIF), a peptide normally present during early stages of viable pregnancy, has been shown to protect against GVHD. The aim of this project was to continue development of PIF to prepare it for human trials in GVHD patients. Scientific Synopsis BMT is a well-established approach for treating malignant and non-malignant hematopoietic diseases, although it is considered a last resort. Even when matched donors are used, many patients develop GVHD, which, when not fatal, can progress to a chronic, lifelong condition. Symptoms of GVHD can involve a number of tissues and organs, including the skin, hair, lungs, liver and gastrointestinal tract. Current treatments include immunosuppressive agents and steroids, although these regimens do not work for all patients. Effective, non-toxic, long-term treatment for chronic GVHD is imperative. PIF is an evolutionarily conserved peptide that accompanies and supports viable embryo development, regulating inflammation, immunity and transplant acceptance. PIF has been shown to regulate several pro-inflammatory genes and proteins, block activated T-cell proliferation, and reduce oxidative stress. In this project, synthetic PIF (sPIF) activity in the GVHD context was assessed after BMT. Short-term, low-dose administration of sPIF in preclinical models was shown to promote and sustain engraftment of donor bone marrow cells, prevent the development of GVHD, preserve the beneficial graft-versus-leukemia effect and significantly reduce overall mortality. sPIF treatment led to protection against dermatitis, hepatitis and colon ulceration, the three classic hallmarks of GVHD. sPIF treatment also reduced mortality following allogeneic BMT from an unrelated donor and promoted long-term cellular engraftment in syngeneic transplant from a genetically identical donor. These findings suggest that PIF could be useful against the range of acute and chronic adverse effects that develop after BMT, possibly making the overall procedure more feasible rather than a last resort. Lead Collaborator BioIncept, LLC, Cherry Hill, NJ Eytan Barnea, M.D. Public Health Impact Each year, approximately 20,000 people undergo bone marrow transplantation in the United States. Most patients in need of transplantation do not have a matching donor in their family, increasing their likelihood of GVHD after receiving BMT from an unrelated source. sPIF has the potential to reduce the long-term, multi-organ complications of GVHD as an effective, non-toxic alternative to current therapies. Beyond GVHD, this project represents a “many diseases at a time” approach, in light of additional preclinical data generated by the collaborators. Leveraging the data developed by BrIDGs toward GVHD, the lead collaborators are moving into additional therapeutic indications, including mitigation of radiation-induced gastrointestinal damage and traumatic brain injury. Outcomes The BrIDGs team completed the IND-directed studies that will form the basis of a regulatory filing by the collaborators at BioIncept to begin clinical trials. Project Details Synthesis of Good Manufacturing Practice (GMP) material Investigational New Drug-directed toxicology | PIF has the potential to reduce the long-term, multi-organ complications of GVHD as an effective, non-toxic alternative to current bone marrow transplant therapies. | Using the PIF to Treat Graft-versus-Host Disease | PIF has the potential to reduce the long-term, multi-organ complications of GVHD as an effective, non-toxic alternative to current bone marrow transplant therapies. | Using the PIF to Treat Graft-versus-Host Disease | ||||
| 7181 | Assay Guidance Workshop Agenda — August 2017 | Monday, August 7, 2017 — 8:00 a.m.–5:40 p.m. ET William F. Bolger Center, 9600 Newbridge Drive, Potomac, MD 20854 8:00 – 8:15 a.m.: Welcome: An Introduction to the Assay Guidance Manual Christopher P. Austin, M.D., NCATS, NIH 8:15 – 9:05 a.m.: Strategies for Assay Selection and the Development of Robust Biochemical Assays Nathan P. Coussens, Ph.D., NCATS, NIH 9:05 – 9:55 a.m.: Treating Cells as Reagents to Design Reproducible Screening Assays Terry Riss, Ph.D., Promega Corporation 9:55 – 10:05 a.m.: Beverage Break 10:05 – 10:55 a.m.: Assay Development for High-Content Screening O. Joseph Trask, Jr., Ph.D., PerkinElmer, Inc. 10:55 – 11:45 a.m.: Assay Interpretation: Studies in Mechanisms and Methods in Assay Interferences Douglas Auld, Ph.D., Novartis Institutes for BioMedical Research 11:45 a.m. – 1:00 p.m.: Lunch 1:00 – 1:50 p.m.: Assay Interference by Chemical Reactivity Jayme L. Dahlin, M.D., Ph.D., Brigham and Women’s Hospital 1:50 – 2:40 p.m.: Basic Assay Statistics, Data Analysis and Rules of Thumb Thomas D.Y. Chung, Ph.D., Mayo Clinic 2:40 – 3:30 p.m.: Reproducibility and Differentiability of Compound Potency Results from Screening Assays in Drug Discovery V. Devanarayan, Ph.D., AbbVie, Inc. 3:30 – 3:40 p.m.: Beverage Break 3:40 – 4:30 p.m.: Avoiding Assay Artifacts and Interferences in Assay Operations G. Sitta Sittampalam, Ph.D., NCATS, NIH 4:30 – 5:20 p.m.: In Vitro Toxicological Testing Using a qHTS Platform Menghang Xia, Ph.D., NCATS, NIH 5:20 – 5:40 p.m.: Closing Remarks Anton Simeonov, Ph.D., NCATS, NIH | Assay Guidance Workshop Agenda - August 2017 | Assay Guidance Workshop Agenda - August 2017 | ||||||
| 7177 | News Brief: NCATS and Eli Lilly Collaborate to Bridge Cultural, Research Divide | The space between academia and industry research is often referred to as the great divide in translational science. Not only are there different laboratory environments and cultures, historically there has not been much opportunity for those in these separate sectors to interact with each other. Seeking to help scientists bridge the gap, NCATS is partnering with Eli Lilly and Company on the NCATS-Eli Lilly scholars externship program. The effort, which pairs scholars, trainees and investigators from NCATS’ Clinical and Translational Science Awards Program with an Eli Lilly project team for up to one year, is based at Eli Lilly’s headquarters in Indianapolis. NCATS and Eli Lilly designed the program to be a fully immersive experience that enhances collaboration between academic researchers and industry scientists and trains participants in the techniques and principles of translational research. Two inaugural scholars completed their externships last winter and found the experience invaluable. H. Clay Conner, Ph.D., a postdoctoral researcher at the University of Notre Dame, a member institution of the Indiana Clinical and Translational Science Institute, completed his externship in the Eli Lilly pharmacology department. He examined animal models used to test drugs for type 2 diabetes. Since the animals don’t naturally develop diabetes, each model represents a unique aspect of the human disease. He analyzed large amounts of data in animals and humans to determine which models were best for testing different classes of diabetes drugs. “In academia, after our research is published, we don’t always know who takes it from there and how it ends up in the clinic,” Conner said. “During the externship, I worked with industry scientists who spend all their time developing a drug, and I saw how translation from bench to bedside really happens.” Ju Youn “Valerie” Kim, Ph.D., a clinical research scholar in the Weill Cornell Clinical and Translational Science Center in New York, worked with regulatory affairs professionals at Eli Lilly. She studied the U.S. Food and Drug Administration (FDA) approval process for oncology drugs to learn more about issues including scientific data criteria needed to support changes in how drugs are administered. Kim also participated in mock FDA meetings and real-world discussions with the FDA on potential investigational new drug applications, which are needed to test a new drug in clinical trials. She enjoyed applying her analytical skills to scientific and business questions, as well as the team-oriented approach of the global regulatory affairs department. “Working collectively with other regulatory scientists was an incredible experience,” Kim said. “The environment in the company was collaborative and synergistic.” Both participants found their Eli Lilly experiences to be extremely helpful to their careers, and emerged with a deeper appreciation for how academic-industry collaborations can help translate scientific discoveries into patient benefit. The 2017 Eli Lilly participants will be selected soon and begin this summer. Find out more about the externship program here. Posted June 2017 | NCATS and Eli Lilly partnered on the NCATS-Eli Lilly scholars externship program, which pairs scholars, trainees and investigators from NCATS’ CTSA Program with an Eli Lilly project team for up to one | /sites/default/files/clay_conner_900.jpg | NCATS and Eli Lilly Collaborate to Bridge Cultural, Research Divide | NCATS and Eli Lilly partnered on the NCATS-Eli Lilly scholars externship program, which pairs scholars, trainees and investigators from NCATS’ CTSA Program with an Eli Lilly project team for up to one | /sites/default/files/clay_conner_900.jpg | NCATS and Eli Lilly Collaborate to Bridge Cultural, Research Divide | ||
| 7174 | News Brief: NCATS Supports Expanded Eye Screening for Underserved Diabetics | Through a community partnership between the Clinical and Translational Science Awards Program hub at the University of California, Los Angeles, and the Los Angeles County Department of Health Services, researchers discovered that offering diabetic retinopathy screening in primary care settings reduced examination waiting periods and increased the number of patients served. Implemented at 15 local primary care centers serving primarily low-income Latino communities, the screenings are maximizing access for this underserved population. Learn more about this program. Posted June 2017 | Through a CTSA Program-supported partnership researchers discovered that offering diabetic retinopathy screening in primary care settings reduced waiting periods and increase patients seen. | /sites/default/files/ucla-900x600.jpg | NCATS Supports Expanded Eye Screening for Underserved Diabetics | Through a CTSA Program-supported partnership researchers discovered that offering diabetic retinopathy screening in primary care settings reduced waiting periods and increase patients seen. | /sites/default/files/ucla-900x600.jpg | NCATS Supports Expanded Eye Screening for Underserved Diabetics | ||
| 7168 | CTSA Program Investigators Collaborate to Enhance Newborn Screening | March 2019 Update Translational Science Highlight An NCATS-supported multidisciplinary team is addressing translational research hurdles — including ethics review and informed consent processes — to add genetic tests to newborn screenings and improve early diagnosis. Don Bailey, Ph.D., director of the Center for Newborn Screening, Ethics, and Disability Studies at RTI International in Research Triangle Park, North Carolina, had been on a quest to collect evidence to support the inclusion of fragile X syndrome and other rare genetic diseases in standard newborn screenings. These efforts had stalled because adding a new screening test to the set of these screenings requires that an effective treatment be available for the specific disease. It’s a catch-22 situation and a translational bottleneck: Without evidence that a treatment is more effective when started earlier, a test for a genetic disease cannot be included in the standard newborn screening panel. But the evidence cannot be collected without a screening program. Rare disorders, like fragile X syndrome, pose an even greater challenge because of the tremendous difficulty identifying enough cases to conduct studies, especially before symptoms appear. Efforts are now underway to change that through Early Check, a research program in which up to 120,000 North Carolina families each year will be offered voluntary screening for fragile X syndrome and other genetic conditions yet to be determined. NCATS is supporting Early Check through a Clinical and Translational Science Awards (CTSA) Program Collaborative Innovation Award (CCIA). The NCATS support is enabling an interdisciplinary team from RTI and three CTSA Program hubs to collaborate on developing and implementing Early Check while tackling other translational hurdles inherent in conducting large-scale, multisite research. The team, which is led by Bailey — the principal investigator for Early Check — is developing innovative electronic formats for educational materials and the informed consent process. In addition, the study sites have agreed to rely on a single institutional review board (IRB) to streamline ethics reviews of the study. “NCATS’ CCIA served as a catalyst to bring together our synergistic areas of expertise to tackle the very difficult challenge of diagnosing and treating rare diseases,” said Bailey. “Our goal is to establish and prove the benefit of a foundation that could be used to evaluate screening tests for many other conditions.” Fragile X syndrome is caused by changes in part of the X chromosome. It is the most common cause of inherited intellectual disability in males and a significant cause of intellectual disability in females as well. Although there is no cure for fragile X syndrome, early intervention can help children achieve their full potential. However, these therapies cannot begin until a child is diagnosed, usually about age 3. The Early Check project, supported in part by The John Merck Fund, is designed to determine whether high-quality early intervention strategies, initiated well before they are usually provided, can have a significant influence on children’s development and family outcomes. “Often, parents first become concerned when their child is about 1 year old and appears to have language delays or difficulty learning to crawl or walk,” said Cynthia Powell, M.D., M.S., lead investigator for the team from the University of North Carolina (UNC) at Chapel Hill. “The families may consult pediatricians and a variety of specialists before genetic testing finally reveals the cause of the child’s developmental delays. By then, the window for early intervention is closed, and the parents might even have an additional child born with fragile X syndrome.” Through Early Check, receiving a diagnosis when a child is only a few months old could help families by reducing the typical three-year diagnostic delay, opening up possibilities for early treatment. As one of the CTSA Program hubs involved in the Early Check project, the North Carolina Translational and Clinical Sciences Institute at UNC will coordinate confirmatory testing following a positive screening test, clinical follow-up support for identified patients, and genetic testing for family members. The UNC bioinformatics group will support RTI experts to ensure the security and privacy of study data and participants’ health information. RTI will establish a research registry and initiate natural history studies for identified children. Key to the project’s success is a strong partnership with the newborn screening program at the North Carolina State Laboratory of Public Health. Two additional CTSA Program hubs have integral roles: The Wake Forest Clinical and Translational Science Institute in Winston-Salem will provide research bioethics expertise for Early Check, and the Duke Clinical and Translational Science Institute in Durham will provide guidance on linking families with clinical trials, depending on the particular condition being screened. How Screening Works The initial Early Check screening panel will test for fragile X syndrome and one or more additional conditions. “Our goal was to select conditions that were not approved for the newborn screening panel as established by the federal government,” Powell said. “We looked for rare genetic disorders that occur frequently enough in the general population that we would be able to enroll several patients with true positive results.” Early Check information will be available in obstetricians’ offices, birthing centers and hospitals. Educational and informed consent materials will be accessible mainly through electronic media. The researchers will arrange for Early Check testing on the blood samples already being collected from infants’ heels and allowed to dry on absorbent paper cards as part of the state’s standard newborn screening. The research team will inform the parents about the test results. Confirmatory testing will be done for any positive screening results. Powell anticipates the screening tests will be negative for most infants in Early Check, which may give some parents greater peace of mind. “Learning of a positive result, however, enables parents to explore options and increase their understanding about the different possibilities for their child,” she said. New Models for Education and Consent Families from anywhere in North Carolina will be invited to participate in Early Check. But a key translational hurdle for research conducted at multiple sites is ensuring that informed consent is administered in a consistent and effective way. In addition, informing and obtaining agreement to participate from up to 120,000 families per year would be nearly impossible if the families and researchers had to meet in person to go through the consent process. To address these challenges, the Early Check team is implementing communication strategies that include the use of electronically accessible materials. “The consent process will be critical to the success of Early Check,” said Nancy M.P. King, J.D., an expert in bioethics and informed consent at the Wake Forest School of Medicine. “Early Check is intended to be a prototype for investigating the feasibility of adding new genetic screening to newborn screening panels nationwide. We are making the education, outreach, and consent and permission processes available in electronic form, allowing many new parents to enroll using a smart phone.” Still, the ethical concerns are potentially complex, and testing can be confusing. Some new parents may not fully understand standard newborn screening, King noted, requiring researchers to spell out differences between the Early Check research program and standard testing. “There are concerns that adding Early Check into the hospital experience might cause people to refuse the standard newborn screening, but past experience shows that is unlikely,” she said. King thinks the Early Check model will help determine whether it is possible to surmount several translational challenges, such as facilitating newborn screening for new candidate conditions and simplifying efforts to recruit children into clinical trials early, before symptoms appear. If successful, the program could serve as a model for other states considering how to develop the evidence needed to add new conditions to their newborn screening programs. “Early Check’s streamlined design — relying on a single IRB and using electronic media for informed consent and participant education — could be a model for other large-scale studies of inherited disorders,” King said. “We want to establish a foundation for testing other candidate conditions so that avoidable delays in diagnosis can be eliminated and early treatment can be initiated if it is proven effective.” Posted June 2017 March 2019: Newborn Screening Launches Early Check launched in October 2018 and is screening for fragile X syndrome and spinal muscular atrophy. Spinal muscular atrophy is a genetic condition that causes progressive muscle weakness; in severe cases, it leads to death around age 2 or 3. Before launching, the program needed an affordable and validated screening test for fragile X syndrome, but such a test did not exist. The Early Check team worked with the biotechnology company Asuragen to develop a test and confirm its performance. Parents-to-be in North Carolina can use the online form to sign up for Early Check during pregnancy or up to four weeks after a child’s birth. Read more about the program in the January 2019 issue of Brain Sciences. Posted March 2019 | Fragile X syndrome is to be included in standard newborn screenings. | /sites/default/files/earlycheck-website_900x600.jpg | CTSA Program Investigators Collaborate to Enhance Newborn Screening | Fragile X syndrome is to be included in standard newborn screenings. | /sites/default/files/earlycheck-website_900x600.jpg | CTSA Program Investigators Collaborate to Enhance Newborn Screening | ||
| 7113 | Developing, Demonstrating and Disseminating Innovations That Turn Science into Medicine Faster | The CTSA Program is designed to strengthen and support the entire spectrum of translational research from scientific discovery to improved patient care. Read the latest in CTSA Program informatics news. September 2018 March 2018 July 2017 February 2017 December 2016 December 2015 March 2015 February 2015 September 2018 NCATS-Supported Research Shows Promise for Stretchable, Wearable Electronics Researchers at the Altman Clinical and Translational Research Institute, a CTSA Program hub at the University of California, San Diego, overcame technological hurdles and found a way to make stretchable electronics in 3-D. This advance could open up new diagnosis and treatment possibilities, such as measuring heart signals, tracking eye movements or controlling a robotic limb. March 2018 New CTSA Program Informatics Center to Create a Nationwide Data Ecosystem NCATS established the National Center for Data to Health to develop standardized approaches and best practices that address operational and institutional barriers to sharing data. July 2017 NCATS Supports Novel Methods to Improve Institutional Review Board Efficiencies NCATS’ Clinical and Translational Science Awards Program support helped a University of Wisconsin–Madison Ph.D. student discover new ways of identifying factors affecting institutional review board review times. February 2017 Improving Patient-Reported Outcome Data for Research through Seamless Integration of the PROMIS Toolkit and Computer-Adaptive Testing Modules into EHR Workflow A team of CTSA Program researchers are developing and evaluating a suite of software tools that will allow all CTSA Program sites to integrate Patient-Reported Outcomes Measurement Information System (PROMIS) tools directly into their electronic health records (EHRs). December 2016 NCATS-Supported Researchers Recruit Citizen Scientists to Help Mine Biomedical Literature A team of bioinformatics scientists from the Scripps Translational Science Institute invented a web-based technology platform to arrange biomedical literature into a format that is easier for computers to organize and analyze. December 2015 CTSA Program Domain Task Forces: Spotlight on Informatics The CTSA Program Informatics Domain Task Force convenes experts from all CTSA Program hubs, the Food and Drug Administration and broader research communities to focus on creating a sustainable data ecosystem to speed discoveries through the innovative use of informatics in precision medicine, education, patient recruitment, community engagement and dissemination of health information. March 2015 Improving Patient Recruitment: The Accrual to Clinical Trials Initiative During the annual CTSA Program principal investigators meeting in February 2015, the University of Pittsburgh’s Steven , M.D., provided an update on the Accrual to Clinical Trials initiative. The NCATS-supported effort, which was launched in September 2014 to overcome clinical trial roadblocks, aims to create a network of sites that share electronic health record data to further multisite study feasibility and recruitment efforts. February 2015 CTSA Program Consortium Tackling Clinical Trial Recruitment Roadblocks The CTSA Program’s Accrual to Clinical Trials initiative was launched to develop a nationwide network of sites that share electronic health record data to further multisite study feasibility and recruitment efforts. | Advance the Use of Cutting-Edge Informatics | /sites/default/files/ctsa-goal5.jpg | CTSA Program Goal 5 | Advance the Use of Cutting-Edge Informatics | /sites/default/files/ctsa-goal5.jpg | CTSA Program Goal 5 | ||
| 7112 | Providing a National Resource for The Rapid Response to Urgent Public Health Needs | The CTSA Program is designed to strengthen and support the entire spectrum of translational research from scientific discovery to improved patient care. Read the latest in CTSA Program news about improving the quality and efficiency of translational research, particularly multisite clinical trials. October 2019 August 2019 July 2019 June 2019 February 2019 December 2018 November 2018 August 2018 July 2018 June 2018 April 2018 December 2017 November 2017 October 2017 July 2017 April 2017 March 2017 February 2017 December 2016 October 2016 September 2016 June 2016 May 2016 February 2016 September 2015 May 2015 February 2015 May 2014 October 2013 January 2013 March 2012 October 2019 CTSA Program-supported Study of Ketogenic Diet Intervention in Alzheimer’s Disease Shows Promise CTSA Program-supported researchers tested the hypothesis that ketones could serve as a source of energy for the brain in people with Alzheimer’s disease (AD). The study found improved brain function in those with mild AD after a three-month ketogenic diet. August 2019 CTSA Program Supports Research on Rapid Diagnosis of Genetic Diseases in Seriously Ill Children NCATS-supported researchers have developed an automated approach to diagnosis of genetic diseases in seriously ill children to allow faster diagnosis and initiation of treatment, and, ultimately, better outcomes. The study appeared in the April 24, 2019, issue of Science Translational Medicine. July 2019 CTSA Program Researchers Aim to Improve Health Care from All Sides A research framework that enables the recruitment of hospitalized patients for a wide range of studies can help identify optimal care approaches during and after hospitalization. Undergraduate students play a valuable role in the studies at University of Chicago Medicine, where they recruit patients, conduct follow-up activities, and learn about clinical research. June 2019 CTSA Program Support Enables Development of Life-Saving Blood Loss Monitor NCATS-supported researchers used applied machine-learning to develop an innovative device that detects internal bleeding and monitors a patient’s response to blood loss. The monitor helps medical staff identify appropriate treatment before a patient goes into life-threatening shock. The device was approved by the U.S. Food and Drug Administration in 2018 and is in clinical use today. February 2019 CTSA Program Supports Emerging Research on Health Effects of Plastics An early-career investigator has developed techniques to study how chemicals used in medical devices affect the still-developing hearts of pediatric patients. Program support also enabled her to secure independent funding for larger studies. December 2018 A Model for Accelerating Translational Research in Real Time Investigators from the CTSA Program hub at the Medical College of Wisconsin have devised and piloted an innovative approach called Real-Time IRB that reduces the time between study submission to the institutional review board and final approval by 70 percent. November 2018 NCATS-Supported Research Shows Potential for Altering Body Fat CTSA Program-supported researchers at Columbia University have discovered a new way to take white fat from a mouse, turn it into brown fat and return it to the mouse. These findings could have implications for treating obesity. August 2018 CTSA Program Researchers Shed Light on Improved Melanoma Treatment Researchers at the University of California, Los Angeles, a CTSA Program hub, discovered four subtypes of melanoma — pointing to new treatment approaches. July 2018 NCATS-Supported Study Highlights Troubling Trend in Opioid Prescribing Researchers at the University of California, Los Angeles, supported in part by NCATS' CTSA Program, discovered a troubling opioid prescribing trend. Patients who took a type of anti-anxiety medicine were more than twice as likely as the general public to receive new opioid prescriptions despite a higher overdose risk. CTSA Program Support May Lead to Blood Test for Colon Cancer Screening University of Alabama at Birmingham researchers, supported in part by NCATS’ Clinical and Translational Science Awards (CTSA) Program, teamed up with scientists from the HudsonAlpha Institute for Biotechnology to identify markers in the blood that could be used to detect precancerous polyps in patients. June 2018 New Study Shows Medication-Based Treatment After Opioid Overdose Can Save Lives Researchers from Boston University, a CTSA Program hub, have found a strong link between the use of medications to treat opioid use disorder and a reduced risk of overdose death. CTSA Program funding supported statistical analysis for this study, which also included support from NIH’s National Institute on Drug Abuse. CTSA Program Researchers Advance Heart Condition Study Through Precision Medicine and Digital Health Researchers at the Scripps Translational Science Institute in San Diego, a CTSA Program hub, are working to address the challenge of diagnosing atrial fibrillation, a common heart condition with complex causes. The team’s efforts can ultimately help advance precision medicine approaches to preventing and diagnosing other types of complex diseases. April 2018 CTSA Program Support Enables Innovative Study of Brain Function A CTSA Program-funded researcher tests innovative training to help people with schizophrenia “correct” disordered brain waves. Promising results suggest the training could potentially help improve memory. December 2017 CTSA Program Supports Research on Translating Evidence into Practice CTSA Program-supported researchers explore the dissemination and clinical implementation of therapies, interventions and policies to ultimately improve human health. November 2017 CTSA Program-Supported Investigators Validate Biomarkers for NPC1 Treatment With support from the Sharing Partnership for Innovative Research in Translation (SPIRiT) consortium, Daniel Ory, M.D., of Washington University in St. Louis, and Charles Vite, D.V.M., Ph.D., of the University of Pennsylvania, collaborated to validate biomarkers intended to monitor the response to therapy for Niemann-Pick diseases type C1 (NPC1). October 2017 CTSA Program-Supported Researchers Test Low-Cost Treatment for Deadly Illness Virginia Commonwealth University researchers have discovered a low-cost and potentially lifesaving treatment for sepsis. Support from the CTSA Program helped streamline translation of this promising therapy to multisite clinical studies. July 2017 NCATS Supports Novel Methods to Improve Institutional Review Board Efficiencies NCATS’ Clinical and Translational Science Awards Program support helped a University of Wisconsin–Madison Ph.D. student discover new ways of identifying factors affecting institutional review board (IRB) review times. April 2017 NCATS Trial Innovation Network Investigators Tackling Clinical Trial Inefficiencies Through NCATS’ new Trial Innovation Network, CTSA Program-supported investigators are working collaboratively to tackle inefficiencies in clinical trials. March 2017 NCATS SMART IRB Platform Agreement All CTSA Program sites have signed on to the NCATS Streamlined, Multisite, Accelerated Resources for Trials (SMART) Institutional Review Board (IRB) Platform agreement, which will enable all participating study sites to rely on one IRB for each study. February 2017 North Carolina CTSA Program Hubs Create Regulatory Expertise Platform Three North Carolina CTSA Program hubs collaborated to create a platform for sharing regulatory expertise. Regulatory Guidance for Academic Research of Drugs and Devices features a best practices website to help researchers better navigate the complex regulatory environment in translational science. CTSA Program Good Clinical Practice Standards Published Investigators leading the CTSA Program good clinical practice (GCP) standards initiative published three papers in the Journal of Clinical and Translational Science. GCP incorporates established ethical and scientific quality standards for the design, conduct, recording and reporting of clinical research involving human subjects. Optimizing Translational Veterinary Trials to Advance Human Outcomes The CTSA One Health Alliance has established a primary mission of creating an organized network of veterinary academic centers that will conduct translational clinical trials in veterinary patients with spontaneous disease. SPARC: The Strategic Pharma-Academic Research The Strategic Pharma-Academic Research Consortium aims to spark innovative collaborations between CTSA Program hubs and the biopharmaceutical industry. The vision is to establish a public-private partnership for patient-focused discoveries that generate greater knowledge and better approaches to next-generation and targeted therapies. December 2016 NCATS Presents Early Plans for Trial Innovation Network, Solicits Project Proposals Efforts to align and harmonize three Trial Innovation Centers and the Recruitment Innovation Center are underway within the NCATS Trial Innovation Network, a new collaborative initiative made possible through the CTSA Program. October 2016 NCATS Launches Trial Innovation Network NCATS launches its Trial Innovation Network, which features a single institutional review board system, master contracting agreements, quality-by-design approaches, and a focus on evidence-based strategies for recruitment and patient engagement. NCATS Funds Collaborative Innovation Awards In fall 2016, NCATS funded the first set of CTSA Program Collaborative Innovation Awards, which are designed to stimulate team-based research across the program’s network. September 2016 CTSA Program Supports New Ultrasensitive Diagnostic Test Development Stanford University researchers have developed a new test for diagnosing diseases, including thyroid cancer, HIV and type 1 diabetes. The method appears to be many times more sensitive than some traditional diagnostic tests, meaning that it potentially can detect illnesses earlier, enabling clinicians to treat patients sooner and possibly slow disease progression. June 2016 CTSA Program Resources Help UCLA Scientists Visualize Key Enzyme in Cancer and Aging With support from NCATS’ CTSA Program, scientists at the University of California, Los Angeles (UCLA), recently produced the clearest-ever image of telomerase, an enzyme involved in cancer and aging. This knowledge could inform the development of anti-cancer and anti-aging therapies. May 2016 NCATS Introduces Plans for New Single IRB Reliance Platform On May 2, 2016, NCATS held a workshop for CTSA Program representatives and other innovators in clinical research management to educate them about using its new single institutional review board (IRB) reliance platform for multisite clinical studies. February 2016 CTSA Program Collaboration Provides Investigators with Access to High-Throughput Screening and Drug Discovery Expertise CTSA Program hub researchers at the University of New Mexico, the University of Kansas Medical Center, and the University of North Carolina, Chapel Hill, collaborated to establish the Drug Rescue, Repurposing and Repositioning Network to more rapidly test existing drugs and advance those showing promise into clinical trials. September 2015 Progress Toward a National CTSA Program Institutional Review Board Reliance Agreement CTSA Program investigators made progress toward creating IRBrely, a national institutional review board (IRB) reliance agreement to streamline the review and approval of multisite clinical trials. May 2015 Michigan CTSA Program Supports Development of Blood Analysis Device A Michigan research team funded in part by NCATS’ CTSA Program and the National Heart, Lung, and Blood Institute has developed a life-saving diagnostic device that can test extremely small blood samples. CTSA Program Supports Streamlined Clinical Trial Recruitment Researchers at the University of New Mexico Clinical and Translational Science Center recently established the Participant Recruitment Service. Supported through NCATS’ CTSA Program, the goal is to streamline and improve recruitment rates for clinical trials while maintaining patient privacy. February 2015 CTSA Initiative Yields Standard Agreement for Industry-Sponsored Clinical Trials A standard agreement intended to reduce the contract negotiation time for industry-sponsored multisite studies, including Phase IIB and Phase III trials, is now available to download. Creating Review Standards for Investigator-Initiated Research Studies The NCATS Scientific Review Standards initiative aims to develop a consensus CTSA Program scientific review process that can ensure feasibility and scientific validity for research projects before reviews by institutional review boards. Establishing Good Clinical Practices for Clinical Study Personnel At the February 2015 CTSA Program principal investigators meeting, Thomas Shanley, M.D., of the University of Michigan shared progress and next steps for the CTSA Program’s Good Clinical Practice initiative. May 2014 IRB Reliance: A New Model for Accelerating Translational Science Within the CTSA Program, several institutions are participating in institutional review board (IRB) reliance networks to streamline this complicated process. October 2013 Five CTSA Program Hubs Enable NIH-Funded Research on Innovative Allergy Therapy Finding a new way to treat and possibly prevent the severest food allergy reactions could improve the lives of millions. A consortium of scientists at five institutions that receive NIH funding, including through NCATS’ CTSA Program, took up this challenge. January 2013 Pitt Researchers Work to Restore Function in Paralysis Patients A team of researchers at the University of Pittsburgh and its medical center have published breakthrough brain-computer-interface research that provides hope to nearly 6 million paralyzed individuals and another 1.7 million amputees nationwide. March 2012 Converting Brain Signals into Action A multidisciplinary team of researchers at the University of Pittsburgh received critical help from CTSA Program regulatory experts to develop a micro-electrocorticography grid that may help paralyzed individuals move again. Designing Solutions to Improve Health for All A team in Stanford University’s Biodesign program designed the OneBreath ventilator for use in emergency pandemic situations and for patient care in resource-poor countries. | Innovate Processes to Increase the Quality and Efficiency of Translational Research, Particularly of Multisite Trials | CTSA Program Goal 4 | Innovate Processes to Increase the Quality and Efficiency of Translational Research, Particularly of Multisite Trials | CTSA Program Goal 4 |
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