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| 11362 | Repurposed Drug Approach May Thwart Spread of Cancer Cells | NCATS experts helped NIH-supported scientists use a new approach to find drugs that may help prevent the spread of prostate cancer. The top image shows antibodies (labeled with a red dye) that target CD63, a protein found on exosomes. The bottom image shows an advanced prostate cancer cell line that has been genetically engineered to turn on a protein that glows green and attaches to CD63. For cancer cells to spread to other places in the body, they need to communicate with each other. One way they do this is through chemical messages delivered in exosomes, which are biological sacs that carry information from cell to cell. Scientists have evidence, for example, that one type of genetic material called “extracellular” RNA (exRNA) contained in exosomes can play a role in prostate cancer growth. While scientists would like to better understand the role of exosomes in cancer, they also see a potential treatment opportunity. But no current drugs target exosomes in cancer cells. Now, researchers supported through NCATS’ Extracellular RNA (exRNA) Communication program, which is funded through the NIH Common Fund, are taking a novel approach to exosomes in cancer. Tulane University School of Medicine’s Asim B. Abdel-Mageed, D.V.M., Ph.D., in collaboration with an NCATS team of preclinical researchers led by Marc Ferrer, Ph.D., recently published new findings in Scientific Reports. The team found several different types of drugs — including antibiotics, antifungal medicines and anti-inflammatory agents — were effective in preventing advanced prostate tumor cells from releasing exosomes or in blocking exosome production. Much work remains to determine which individual drugs might be useful in patients. “These compounds and drugs alone aren’t going to shrink tumors but would likely reduce growth and prevent the cancer from spreading further when administered with standard therapies,” said Ferrer. “This study shows that cost-effective, repurposed drugs can potentially affect cancer treatment by targeting new mechanisms.” The scientists believe that further research into this approach could be useful in other advanced cancers, and in diseases such as Alzheimer’s disease. Posted June 2018 | NCATS preclinical staff helped NIH Extracellular RNA Communication program-supported scientists use a new approach to find drugs that may help prevent the spread of prostate cancer. | /sites/default/files/exrna_cancer_1260x630.jpg | Repurposed Drug Approach May Thwart Spread of Cancer Cells | NCATS preclinical staff helped NIH Extracellular RNA Communication program-supported scientists use a new approach to find drugs that may help prevent the spread of prostate cancer. | /sites/default/files/exrna_cancer_1260x630.jpg | Repurposed Drug Approach May Thwart Spread of Cancer Cells | ||
| 11364 | New Study Shows Medication-Based Treatment After Opioid Overdose Can Save Lives | Survivors of opioid overdose have a higher risk of death than individuals who have not experienced an overdose. Effective strategies to lower that risk are critically important to combatting the opioid epidemic in the United States. Medications to treat opioid use disorder (OUD) are one potential strategy, but research about the effect of medication use on survival after an overdose is limited. To address that gap, a team led by researchers from Boston University, an NCATS Clinical and Translational Science Awards (CTSA) Program hub, reviewed medical records for more than 17,500 adults who had survived an opioid overdose. The goal was to determine whether survivors who received medications for OUD were less likely to die from another overdose within the next 12 months, compared with survivors who did not receive treatment. CTSA Program funding supported statistical analysis for the study, which also included support from the National Institute on Drug Abuse. The study included all three medications currently approved by the Food and Drug Administration to treat OUD: methadone, buprenorphine and naltrexone. The results, published in the Annals of Internal Medicine, showed that survivors who received methadone were 59 percent less likely to die from another opioid overdose and that those who received buprenorphine were 38 percent less likely to die. Naltrexone treatment showed no significant effect on survival. Only 30 percent of the survivors in the study received medication-based treatment following overdose. The results demonstrate a strong link between the use of medications to treat OUD and a reduced risk of overdose death. These findings can help inform effective approaches to save more lives from opioid overdose. Learn more about this study. Posted June 2018 | NCATS-supported researchers at Boston University found a strong link between the use of medications to treat opioid use disorder and a reduced risk of overdose death. | /sites/default/files/opioids_nida_1260x630.jpg | New Study Shows Medication After Opioid Overdose Can Save Lives | NCATS-supported researchers at Boston University found a strong link between the use of medications to treat opioid use disorder and a reduced risk of overdose death. | /sites/default/files/opioids_nida_1260x630.jpg | New Study Shows Medication After Opioid Overdose Can Save Lives | ||
| 11366 | CTSA Program Researchers Advance Heart Condition Study Through Precision Medicine and Digital Health | Translational Science Highlight With NCATS support, researchers are advancing precision medicine and digital health to combat a dangerous heart condition through improved screening and diagnostic methods. This research is validating an approach that could be applied to many complex disorders. Atrial fibrillation (AFib), a type of irregular heartbeat, puts patients at increased risk for stroke. AFib can be treated with medications or various procedures, and the risk of stroke can be reduced with blood thinners. One issue, however, is that many people do not know they have AFib until after a life-threatening event such as stroke occurs. Health professionals need better ways to identify and evaluate individuals at risk. One solution is to use a person’s unique genetic characteristics to guide and tailor health screening, diagnosis and treatment decisions. This precision medicine approach for diagnosis and treatment is increasingly becoming a clinical reality. But progress has been limited for complex disorders like AFib that do not have a single genetic cause. Researchers at the Scripps Translational Science Institute in San Diego, part of NCATS’ Clinical and Translational Science Awards (CTSA) Program, are working to address this challenge for common heart conditions with complex causes. These efforts can ultimately help advance precision medicine approaches to preventing and diagnosing other types of complex diseases. Precision Medicine Meets Wearable Technology As a cardiologist at the Scripps Clinic, Evan Muse, M.D., Ph.D., observed that some patients were being diagnosed with AFib only after they had survived a stroke: “There had to be a better way to identify these patients before devastating complications like stroke occurred.” This is where precision medicine could help. Every person has a unique genome, or collection of genes. But some individuals have specific “genetic variants” in common that make them more likely to develop a disease or condition, such as AFib. Each variant can increase a person’s genetic risk for the disease, so researchers combine many known variants into an algorithm that calculates a person’s overall genetic risk score. Evan Muse, M.D., Ph.D., leads a discussion during a Journal Club meeting with trainees at the Scripps Translational Science Institute. (Scripps Translational Science Institute Photo) Previous research on how well the score could predict a person’s risk of developing AFib centered on patient data that already existed. Muse and his colleagues wanted a real-time approach that would begin with tracking patients who had not yet been diagnosed with AFib and follow them over time. This type of prospective study provides important evidence for the test’s validity, as well as information to help health professionals determine whether they should use the test with their patients. One challenge to conducting a prospective study was ensuring that Muse and his team did not miss anyone who had the condition. Typically, AFib is detected with a test in a doctor’s office or by having the person wear a portable device that monitors heart rhythm for 24 to 48 hours. But both approaches can miss people who only have brief episodes of AFib every now and then. Muse saw an opportunity to take a digital health approach by using a newer mobile heart monitor the size of a small bandage that stays on for up to two weeks, measuring and recording every heartbeat. With CTSA Program interests in digital health and precision medicine, Muse received support to launch a study to evaluate more than 900 people who were at risk for AFib and had symptoms of the condition but had not been diagnosed. The study team determined the genetic risk score of the participants and evaluated them for AFib using the new wearable monitor. Translating Risk into Empowerment The genetic risk scores for study participants accurately predicted who would be diagnosed with AFib via digital monitoring. Participants with the highest scores were more than three times as likely to have AFib as those with the lowest scores, even after considering traditional risk factors such as smoking and age. The results were published in the March 2018 issue of PLOS Medicine. Many people in the study would not have been diagnosed using the conventional approach to AFib screening. The genetic risk score could help doctors decide which patients would benefit most from more intense screening. Muse also sees the risk score as a way to make patients more active partners in their health outcomes. Commercially available wearables, such as smartwatches, can already detect irregular heartbeats, so people who know they are at high risk could monitor their health in addition to working with their doctor. “People shouldn’t feel doomed if they have a high risk score,” Muse said. “I want people to feel empowered. There are things you can do to reduce your risk.” For example, losing weight can help prevent or reduce the number of AFib episodes. Training the Next Generation of Leaders The AFib study was Muse’s first clinical trial as lead investigator. He credits his mentoring and training in the CTSA Clinical Research KL2 Scholar program for preparing him for the new role. The KL2 program provides project funding and guidance to early-stage clinical investigators to help them gain the skills to conduct multidisciplinary, translational and team-based research. This collaborative training helped Muse coordinate among 17 clinical sites for the AFib study — no small challenge for a first-time lead investigator. “It really demonstrates the strengths of the CTSA Program at Scripps and the excellent team of leaders, who helped guide me at every step,” Muse said. Muse said he will put his KL2-developed skills to further use as he continues to work to advance precision medicine and digital health. Currently, he is preparing to lead a pilot study in the NIH-led All of Us Research Program, an unprecedented precision medicine effort to gather data from more than 1 million U.S. participants to accelerate research and improve health. Posted June 2018 | Investigators from the Scripps Translational Science Institute advance precision medicine to combat a dangerous heart condition through improved screening and diagnostic approaches. | /sites/default/files/scripps_muse_1260x630.jpg | NCATS-Funded Researchers Apply Precision Medicine to Digital Health | Investigators from the Scripps Translational Science Institute advance precision medicine to combat a dangerous heart condition through improved screening and diagnostic approaches. | /sites/default/files/scripps_muse_1260x630.jpg | NCATS-Funded Researchers Apply Precision Medicine to Digital Health | ||
| 11447 | NIH-Supported Scientists Discover New Personalized Approach to Diagnosing and Treating Rare Food Allergy Disease | Human eosinophils, a type of white blood cell, are shown isolated from blood. In patients with eosinophilic esophagitis, these cells store and release packages of inflammatory proteins (red) that can damage the throat and esophagus. (Cincinnati Children’s Hospital Medical Center Photo/Julie Caldwell) Diagnosing and treating rare diseases can be difficult, in part due to widely dispersed and small populations of both patients and scientific experts. This lack of sufficient access to patients and expertise makes it challenging to understand the natural course of a rare disease and, in some cases, its different forms. Eosinophilic esophagitis (EoE) is a rare and potentially dangerous food allergy disease caused by an immune system reaction that results in damage to the throat and esophagus (which connects the throat and stomach) and problems with swallowing. To improve the understanding of EoE, scientists at Cincinnati Children’s Hospital Medical Center and the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR) examined — and studied tissue samples from — 185 child and adult patients with EoE from 10 sites associated with the consortium. The CEGIR is supported by NCATS' Rare Diseases Clinical Research Network and the National Institutes of Health’s National Institute of Allergy and Infectious Diseases and National Institute of Diabetes and Digestive and Kidney Diseases. The researchers assessed the patients by detailing their individual genetic and clinical features, as well as the appearance of their diseases through an endoscope — which enabled them to peer into the esophagus — and under a microscope. Through this four-pronged approach, they identified three distinct forms of EoE that were each associated with different clinical and genetic characteristics. This advance is reported in The Lancet Gastroenterology & Hepatology. “In the past, patients were diagnosed with EoE, or they weren’t,” said co-author Marc Rothenberg, M.D., Ph.D., at Cincinnati Children’s. “Now that we know there are three distinct forms of this disease, we’re developing a more personalized approach to helping these patients.” Posted June 2018 | With support from the Rare Diseases Clinical Research Network, scientists have developed a personalized approach to diagnosing and treating eosinophilic esophagitis. | /sites/default/files/rdcrn_eos_1260x630.jpg | Team Identifies Three Distinct Forms of Rare Food Allergy Disease | With support from the Rare Diseases Clinical Research Network, scientists have developed a personalized approach to diagnosing and treating eosinophilic esophagitis. | /sites/default/files/rdcrn_eos_1260x630.jpg | Team Identifies Three Distinct Forms of Rare Food Allergy Disease | ||
| 11372 | NIH-Supported Scientists Uncover Clues to Spinal Cord Development, Neurodegenerative Disease | Layers of spinal cord motor nerve cells (top, in blue) and blood vessel cells (bottom, in red) are shown interacting on a tissue chip, a 3-D platform that supports living tissue and cells. (Cedars-Sinai Board of Governors Regenerative Medicine Institute Photo). Cells in the developing human brain and spinal cord are constantly “talking,” sending chemical signals to help each other grow. But understanding such “crosstalk” isn’t always easy. While scientists have evidence that spinal cord nerve cells, or neurons, interact with blood vessel cells in the brain to help shape the developing spinal cord, there is much yet to be discovered about this process. To take a closer look, researchers at Cedars-Sinai Medical Center — supported by NCATS and the National Institute of Neurological Disorders and Stroke through the NCATS-led Tissue Chip for Drug Screening program — used stem cells and tissue chips to mimic conditions in the early human spinal cord. Tissue chips are 3-D tissue platforms that model the structure and function of human organs. The results, reported in Stem Cell Reports, could provide a better understanding of the development of some neurodegenerative diseases, such as amyotrophic lateral sclerosis (more commonly known as ALS), and lead to possible treatment strategies. In this study, investigators first converted adult human skin cells into stem cells, which have the potential to become any type of cell. Then the investigators made these stem cells into either early-stage spinal cord neurons or brain blood vessel cells and grew them together in a specially engineered tissue chip environment that supports living tissues and cells. The researchers found that blood vessels in the brain can trigger the growth and maturation of spinal cord neurons early in development. “The tissue chip environment provides a better representation of neuron development and how cell types work together than has been possible in the laboratory,” said Cedars-Sinai co-author Clive Svendsen, Ph.D. Posted May 2018 | NIH-supported researchers at Cedars-Sinai have used stem cells and 3-D tissue chips to mimic conditions in the development of the human spinal cord. | /sites/default/files/tissue-chip-spinal-cord_1260x630.jpg | Tissue Chips Help Uncover Clues to How Cells “Talk” to Each Other | NIH-supported researchers at Cedars-Sinai have used stem cells and 3-D tissue chips to mimic conditions in the development of the human spinal cord. | /sites/default/files/tissue-chip-spinal-cord_1260x630.jpg | Tissue Chips Help Uncover Clues to How Cells “Talk” to Each Other | ||
| 11406 | New Funding Opportunities Available for Collaborative Rare Diseases Research | A child with WAGR syndrome.June 11, 2018On June 11, 2018, NCATS announced two new Rare Diseases Clinical Research Network (RDCRN) program funding opportunities to support collaborative clinical research on rare diseases. The Center is accepting applications for Rare Diseases Clinical Research Consortia and a Data Management and Coordinating Center (DMCC); both funding opportunities have a deadline of Oct. 9, 2018.The RDCRN is intended to advance the diagnosis, management and treatment of rare diseases with a focus on clinical trial readiness. Each consortium will promote highly collaborative, multi-site, patient-centric, translational and clinical research with the intent of addressing unmet clinical trial readiness needs.Approximately 7,000 rare diseases affect an estimated 25 million — approximately one in 10 — Americans, but only a few hundred of these diseases have an approved treatment. Through the RDCRN, physicians, scientists, and their multidisciplinary teams work together with patients and patient advocacy groups to study more than 200 rare diseases at sites across the nation.“The RDCRN is uniquely positioned to foster important clinical research advances and bridge translational gaps in the understanding and treatment of rare diseases,” said Anne Pariser, M.D., director of the NCATS Office of Rare Diseases Research, which oversees the RDCRN.NIH launched the RDCRN program in 2003. RDCRN-supported research includes natural history studies that chart the normal progression of a disease over time, as well as studies focused on the effects of treatments or ways to detect and diagnose disease. Such information is necessary to conduct and evaluate clinical trials for new therapies.The RDCRN consortia have a long history of accomplishment. For example, in 2014, researchers studying rare immune system disorders through the RDCRN’s Primary Immune Deficiency Treatment Consortium published a report in the New England Journal of Medicine showing the importance of evaluating newborns and early treatment in improving the lives of individuals with a rare immune disease. More recently, in ongoing studies, clinicians and researchers with the RDCRN’s Lysosomal Disease Network have collaborated with scientists at a biotechnology company to examine the use of gene editing in rare disease patients.The DMCC manages the collection, storage and quality control of clinical research data for the network. The RDCRN will emphasize data standards, which will facilitate data sharing among its members and others outside the network also studying rare diseases. NCATS will provide “cloud” computing services through the DMCC, enabling greater data accessibility and use. The DMCC will provide expertise and consulting to research consortia in several areas, such as the development and management of research study protocols, biostatistics, and study design.The NIH Institutes, Centers and Offices that partner with NCATS on the RDCRN program include the National Cancer Institute, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the Office of Dietary Supplements, the National Institute of Allergy and Infectious Diseases, the National Institute of Diabetes and Digestive and Kidney Diseases, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Institute of Neurological Disorders and Stroke, the National Institute of Mental Health, the National Institute of Dental and Craniofacial Research, and the National Heart, Lung and Blood Institute.For more information, visit https://ncats.nih.gov/research/research-activities/RDCRN. | Two new funding opportunities will support collaborative clinical research on rare diseases. | /sites/default/files/grdr_boy_1260x630.jpg | New FOA Available for Collaborative Rare Diseases Research | Two new funding opportunities will support collaborative clinical research on rare diseases. | /sites/default/files/grdr_boy_1260x630.jpg | New FOA Available for Collaborative Rare Diseases Research | ||
| 11449 | RDCRN in Action | The RDCRN program is designed to advance medical research on rare diseases. It facilitates clinical research in rare diseases. Read the latest news about this program below.March 2023Collaboration Opens Door to Potential Therapies for Children With a Rare DiseaseClinicians historically have focused on treating the symptoms of a rare lung disease called primary ciliary dyskinesia (PCD). A recent study using medical images from children with PCD could help point to new therapeutic candidates for slowing damaging effects linked with the disease. It also can help diagnose this disease sooner. The study was a collaboration among researchers funded through the Genetic Disorders of Mucociliary Clearance Consortium within the Rare Diseases Clinical Research Network (RDCRN) and the Colorado Clinical and Translational Sciences Institute. February 2022 Osteogenesis Imperfecta Study Reveals Potential Therapy Approach RDCRN-funded researchers confirmed a common problem in the way the body forms bone in osteogenesis imperfecta (OI), a hereditary disorder that causes brittle bones and fractures. Bones in people with OI often produce too much of a protein called tumor growth factor beta (TGF-β). As reported in the Journal of Clinical Investigation, the researchers have shown that treating people with moderate OI using fresolimumab, an antibody drug that blocks TGF-β, could improve their bone mass. Fresolimumab’s maker, Sanofi, has launched a clinical trial to further advance this approach in patients with OI. May 2020 NIH-Supported Research Survey to Examine Impact of COVID-19 on Rare Diseases CommunityAn online survey launched by the RDCRN aims to find out how the COVID-19 pandemic is affecting individuals with rare diseases, their families and their caregivers. Results will help the rare disease research community shed light on the needs of people with rare diseases during the COVID-19 pandemic. March 2020 A Collaborative Approach to Diagnosing Alzheimer’s and Rare SyndromesNCATS, the National Institute on Aging and the National Institute of Neurological Disorders and Stroke collaborate on the development of a potential blood test that could make diagnosing Alzheimer’s and rare neurodegenerative syndromes easier and faster. January 2020 Grants Help Move Promising Rare Disease Interventions to Clinical TrialsTo close the gap between development of candidate interventions for rare diseases and clinical testing, NCATS and the Eunice Kennedy Shriver National Institute of Child Health and Human Development created rare-disease Clinical Trial Readiness grants to support projects that collect data needed to advance to clinical trials. December 2019 NCATS-Supported Consortium Plays Pivotal Role in New Porphyria DrugYears of NIH-supported studies contributed to the U.S. Food and Drug Administration approval of a new drug for acute intermittent porphyria—a rare, debilitating condition. The NCATS-supported Porphyrias Consortium played a pivotal role in its development. October 2019 NIH Awards Funding to 20 Rare Disease Research TeamsNIH has awarded approximately $31 million to 20 teams to study a wide range of rare diseases and an additional $7 million to support the research efforts. An important focus of the latest group of awards is clinical trial readiness and preparation. | Read the latest news about the RDCRN program. | /sites/default/files/rdcrn_1260x630.jpg | RDCRN in Action | Read the latest news about the RDCRN program. | /sites/default/files/rdcrn_1260x630.jpg | RDCRN in Action | ||
| 11370 | NIH-Supported Research Helps Advance a Potential Rare Disease Gene Editing Treatment into Clinical Trials | Translational Science HighlightNIH's Rare Diseases Clinical Research Network-supported research helps overcome a translational roadblock by charting how several rare, inherited disorders progress in patients over time. This information enabled a gene editing therapy for a group of rare diseases with few treatments to advance to clinical trials.Lysosomal diseases affect about one in 6,000 people worldwide. These disorders are characterized by an abnormal buildup of various toxic materials in the body’s cells — the result of enzyme deficiencies. A pair of rare, inherited lysosomal diseases, Hurler and Hunter syndromes, can cause enlarged facial features, heart and other organ damage, bone and joint problems, developmental delays, brain damage, and early death.In February 2013, scientists from Sangamo BioSciences, Inc. (now Sangamo Therapeutics, Inc.) asked University of Minnesota clinical biochemical geneticist Chester Whitley, M.D., Ph.D., to help them find out whether their novel gene editing technique could prevent or reverse the two syndromes. If the therapy proved safe and effective in animals, then clinical trials with patients could follow.With support from the National Institutes of Health (NIH), initially through its Eunice Kennedy Shriver National Institute of Child Health and Human Development, Whitley and colleague R. Scott McIvor, Ph.D., had developed extensive experience in using mouse models to study several types of lysosomal diseases — including Hurler and Hunter syndromes — and their effects on the brain, in addition to gene therapy approaches.Magnetic resonance imaging reveals the brain’s structure. In this image, enlarged spaces (arrows) around blood vessels in the brain of a patient with Hunter syndrome, a neurodegenerative disease, show a physical effect of the disorder. (University of Minnesota Photo/Igor Nestrasil, M.D., Ph.D. and Carol Nguyen)Subsequently, as part of the National Center for Advancing Translational Sciences (NCATS) Rare Diseases Clinical Research Network (RDCRN)-funded Lysosomal Disease Network (LDN) — which included support from the National Institute of Neurological Disorders and Stroke and the National Institute of Diabetes and Digestive and Kidney Diseases — Whitley and LDN scientists conducted clinical studies of affected children and adults. The research included natural history studies, which revealed how these diseases progress over time. Knowledge of a disease’s natural history is essential to establishing the scientific foundation for developing effective treatments. “NIH support has been crucial to understanding the underlying biology and natural progression of lysosomal diseases,” said Anne Pariser, M.D., director of NCATS’ Office of Rare Diseases Research. “This support also helps scientists establish collaborations among specialized clinical sites, setting the stage for innovative research.”Standard therapies for these disorders, such as injecting the missing enzyme directly into the body and bone marrow transplantation, have limited effectiveness and frequently have significant side effects.Working with Sangamo researchers, Whitley and LDN Fellow Li Ou, Ph.D., found that a gene editing technique could prevent or reverse Hurler syndrome in some tissues in mice with the disease. When the researchers evaluated the technique’s effects in a learning behavior test, the results in treated mice suggested that the therapy reached the brain. Along with similarly promising findings in mice with Hunter syndrome, the stage was set for the next step: testing in patients.In November 2017, Sangamo made headlines when scientists made the first attempt to edit a gene inside the human body in a patient with Hunter syndrome.RDCRN: Collaborative Approaches Address Roadblocks to Rare Diseases ResearchEstablishing the LDN was a watershed moment for many who study lysosomal diseases. RDCRN support enabled previously separate groups of lysosomal disease researchers from several institutions to pool patient data, experiences and expertise. With larger numbers of lysosomal disease patients available, the researchers could conduct the important natural history studies that examined how patients fared over time, with and without treatment.Such studies enabled scientists to develop cognitive tests and brain imaging tools. RDCRN support eventually led to the creation of a national network of 10 centers for brain testing and imaging for patients with lysosomal diseases.Patients and patient advocacy groups are part of the LDN, as both supporters and participants, and share their perspectives on living with lysosomal diseases. “We work with patients and families to figure out how best to design research studies that will help meet their needs,” Whitley said. “They let us know what we’re doing well and where we’re falling short.”Cyndi Frank is a patient with Gaucher disease, a related lysosomal disease. She has participated in several trials over the past 40 years, including an LDN study of a possible drug treatment in the brain, and said it was vital that researchers listen to patients. “Doctors aren’t patients, and they can miss things,” she said. “They are worrying about treating the underlying disease problems, some of which can be life threatening. But many aspects of living with a disease can get overlooked. It can be difficult to appreciate, for example, what it means to endure almost disabling fatigue.”The LDN’s Latest RoleWhitley and LDN researchers have worked with Sangamo to develop the current clinical trial and recruit participants. Some of the centers involved in the Sangamo trial are part of the LDN, including the UCSF Benioff Children’s Hospital Oakland and the University of Minnesota.Sangamo and LDN scientists will gauge the safety of the gene editing technique in patients and will also watch for signs of effectiveness by using LDN-developed tools, including brain imaging. In addition, LDN investigators will help study and evaluate the effects of the gene editing therapy on patients’ thinking and learning abilities. In the meantime, LDN researchers continue to examine how these diseases and treatments affect the brain over time.“These kinds of studies will help clinicians tailor treatments,” added LDN investigator Elsa Shapiro, Ph.D.. “There are many drug and biotechnology companies working in this field right now that are relying on this kind of information coming out of the LDN.”Posted May 2018 | RDCRN-supported researchers charted how several rare diseases progressed in patients over time, helping to enable a potential gene-editing therapy to advance to clinical trials. | /sites/default/files/rdcrn_brain_1260x630.jpg | Potential Gene-Editing Treatment Advances to Clinical Trials | RDCRN-supported researchers charted how several rare diseases progressed in patients over time, helping to enable a potential gene-editing therapy to advance to clinical trials. | /sites/default/files/rdcrn_brain_1260x630.jpg | Potential Gene-Editing Treatment Advances to Clinical Trials | ||
| 11368 | CTSA Program Hubs Collaborate to Assess Community Engagement | Community members are the ultimate beneficiaries of research. (Daniel Soñé Photography, LLC) Community engagement is a crucial feature of successful translational research. Community members are the ultimate beneficiaries of research results, and when they have a voice in the research process, outcomes are more relevant to their needs and more readily adopted. However, the effectiveness of community engagement efforts can be difficult to measure, and better evaluation methods and tools are needed. Recognizing these challenges, investigators at the University of Rochester, an NCATS Clinical and Translational Science Awards (CTSA) Program hub, launched a pilot study to develop and test a community engagement assessment process. The goal is to help academic health centers evaluate and enhance their engagement activities, which in turn will improve the translation of research results into real-world practice. Seven other CTSA Program hubs participated in the pilot study, and all eight institutions are using the results to improve their engagement efforts. Learn more about the assessment tool. Posted May 2018 | The University of Rochester conducted a pilot study with seven other CTSA Program hubs to develop and test a community engagement assessment process. | /sites/default/files/rochester_1260x630.jpg | CTSA Program Hubs Collaborate to Assess Community Engagement | The University of Rochester conducted a pilot study with seven other CTSA Program hubs to develop and test a community engagement assessment process. | /sites/default/files/rochester_1260x630.jpg | CTSA Program Hubs Collaborate to Assess Community Engagement | ||
| 10843 | Translational Science Training at Partner Institutions | NCATS supports the development of translational science training and career development programs at universities and research institutions across the country to cultivate an innovative translational science workforce. This is accomplished through investments in the NCATS Clinical and Translational Science Award (CTSA) Program and the Rare Diseases Clinical Research Network (RDCRN). Each year, these programs enable the educational and professional development of hundreds of scholars who become the next generation of leading translational scientists. See below for the descriptions of training programs offered by our partner institutions. Learn more about how the CTSA Program promotes activities and opportunities to support a clinical and translational science workforce in this fact sheet. NCATS CTSA Program: Research Training TL1 Program This institutional National Research Service Award supports the training of predoctoral and medical students and postdoctoral fellows seeking a practical introduction to clinical and translational research. The objective of the TL1 program is to increase the number of well-trained clinician-scientists who can lead the design and oversight of future clinical investigations critical to the overall mission of NCATS and NIH. Contact: Carol Merchant, M.D. NCATS CTSA Program: Mentored Career Development KL2 Program This institutional Mentored Career Development Award supports the training of early-career scholars seeking advanced research training in therapeutics, clinical interventions and behavioral modifications to improve health. KL2 Program appointees, referred to as Clinical Research KL2 Scholars, come from a variety of health-related fields and receive a rich career development experience in a multidisciplinary setting with protected research time. The objective of the program is to facilitate the development of clinician-scientists who will ultimately lead collaborative research efforts in translational science. Contact: Joan Nagel, M.D., M.P.H. NCATS CTSA Program: Eli Lilly Scholars Externship Program This is an opportunity for an externship in clinical and translational sciences at Eli Lilly and Company for scholars, trainees and investigators who are supported by the NCATS Clinical and Translational Science Award (CTSA) Program. The externship at Eli Lilly, a large pharmaceutical company, facilitates the training and education of participants in techniques and principles needed for translational research. Contact: Joan Nagel, M.D., M.P.H. NCATS CTSA Program: Research Supplements to Promote Re-Entry and Diversity This award supplement program enhances the diversity of the clinical and translational research workforce by recruiting and supporting students, postdoctorates and eligible investigators from groups that have been shown to be underrepresented in health-related research and those who are or have become disabled and need additional support to accommodate their disability to continue to work on a research project. This program also supports individuals with a high potential to re-enter an active research career after an interruption for family responsibilities or other qualifying circumstances. Contact: Erica Rosemond, Ph.D. NCATS CTSA Program: I-Corps™ Train-the-Trainer Program The Train-the-Trainer program fosters I-Corps™ leaders at NCATS CTSA Program hubs who in turn will provide entrepreneurship training to other translational scientists at their institution on how to translate their research into a business product. These trained individuals will help disseminate evidence-based practices in the commercialization of clinical and translational research activities and build entrepreneurial capacity locally and regionally across CTSA Program hubs. Contact: David Wilde, M.D., Ph.D. Training Component of NCATS-Led RDCRN Program The NCATS RDCRN Program offers training opportunities for new investigators in rare diseases clinical research within each consortium. The RDCRN provides a unique environment for clinical rare diseases research that can be used to prepare new scientists for careers in the field and provide the opportunity for established scientists to reorient their research careers toward rare diseases research through relevant courses, mentorship and participation in clinical research. Contact: Tiina Urv, Ph.D. NCATS also offers intramural training in translational research and translational science. Learn more about training at NCATS. | NCATS supports the development of translational science training and career development programs at universities and research institutions. | /sites/default/files/ncats_scientists_1260x630.jpg | Translational Science Training at Partner Institutions | NCATS supports the development of translational science training and career development programs at universities and research institutions. | /sites/default/files/ncats_scientists_1260x630.jpg | Translational Science Training at Partner Institutions |
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