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| 13273 | NCATS Announces NIH HEAL Initiative-Related Funding Opportunities | December 19, 2018NCATS is supporting three new funding opportunities and a prize challenge through the Helping to End Addiction Long-termSM Initiative, or NIH HEAL InitiativeSM. The opportunities are part of a collection that NIH released on Dec. 10, 2018, to solicit the best and brightest research ideas to bolster existing research in the areas of addiction, pain and opioid use disorders.Paige Derr creates a 3-D bioprinted tissue model in NCATS’ 3-D Tissue Bioprinting Lab. (Daniel Soñé Photography, LLC, Photo)As part of the NIH HEAL Initiative, NCATS is providing a suite of translational science resources and expertise to investigators working on opioid and pain research. The Center will lead trans-NIH collaborative Initiative efforts to:Develop new testing platforms that more closely model human biology;Find, de-risk, test and optimize promising compounds;Identify new chemical structures and develop them into drug-like compounds;Advance new drug candidates through rigorous preclinical efficacy and safety studies needed for first-in-human clinical trials; andFacilitate opportunities to partner with other NIH Institutes and Centers as well as clinical research institutions to conduct comparative-effectiveness studies of existing pain management treatments.Details about each of these four opportunities are as follows:3-D Bioprinted Tissue Models of Pain, Opioid Use Disorder and OverdoseLetters of Intent Due: Jan. 28, 2019Applications Due: Feb. 28, 2019Through RFA-TR-19-005, NCATS is supporting collaborations with the external research community to develop and use 3-D biofabricated tissue models as new drug screening platforms to advance preclinical discovery and development of non-addictive treatments for pain, opioid use disorder and overdose. NCATS intends to fund approximately two to three awards for a total of $1.5 million in fiscal year 2019.Tissue Chips to Model Pain, Addiction and OverdoseLetters of Intent Due: Jan. 28, 2019Applications Due: Feb. 28, 2019Selected applicants through RFA-TR-19-003 will receive funding to create and test devices that can model the mechanisms or effects of pain-relevant signaling, addiction or opioid use disorders using human tissues in tissue- and organ-on-chip systems. NCATS intends to commit approximately $5 million in fiscal year 2019 to fund four to six awards.ASPIRE Design ChallengesChallenges Open: Dec. 31, 2018Submission Deadline: May 31, 2019Winners Announced: August 2019NCATS has issued a series of challenge competitions for novel chemical compound concepts that have the potential to address pain, addiction and overdose. NCATS will issue up to 22 prizes for the creation of a database, an electronic laboratory knowledge portal, machine learning algorithms and biological assays. An additional challenge will focus on a combined solution for two or more areas. View NOT-TR-18-031 and the ASPIRE Design Challenges page to learn more. NIH HEAL Pain Management Effectiveness Research Network (ERN)Letters of Intent Due: Jan. 2, 2019January 2019 Update: There has been a change to the Key Dates in RFA-NS-19-021. Specifically, the first receipt date will change from February 1, 2019, to February 11, 2019. Please view NOT-NS-19-028 for additional details.Led by the National Institute of Neurological Disorders and Stroke, the NIH HEAL Pain Management ERN will support studies to compare the effectiveness of existing pain treatments and new approaches to prevent and manage pain while reducing the risk of addiction. The network will use the NCATS Clinical and Translational Science Awards (CTSA) Program hubs and the Trial Innovation Network to implement clinical trials and studies of interest to multiple NIH Institutes, Centers and Offices and support studies that provide evidence to inform practice-based guidelines. NIH intends to fund approximately five awards up to a total of $4 million in fiscal year 2019. View RFA-NS-19-021 for more information.View a complete list of current NIH HEAL Initiative funding opportunities, including more that involve NCATS. | NCATS Announces New NIH HEAL Funding Opportunities | NCATS Announces New NIH HEAL Funding Opportunities | ||||||
| 13288 | Tissue Chip Researchers Create a Working Model of a Heart Chamber | Scientists can take cells from the skin or blood of adults, turn them back into a type of stem cell called induced pluripotent stem cells (iPSCs) and direct these iPSCs to become heart cells that beat in a dish. But these cells do not have the 3-D structure of the heart, where they line four hollow chambers that contract to pump blood through the body. This 3-D model of a human left heart ventricle could be used to study diseases, test drugs and develop new treatments for heart conditions such as arrhythmia. (Harvard University Photo/Luke MacQueen and Michael Rosnach) Now, researchers supported by NCATS’ Tissue Chip for Drug Screening program have developed a 3-D working model of a human heart chamber. The advance, reported in Nature Biomedical Engineering, is a crucial step toward innovative approaches to study heart diseases, test potential drugs for toxicity or make patient-specific models to test new medications. The team from Harvard University built a scale model of a left ventricle, the chamber that pumps oxygen-rich blood out to the body. The key was engineering the 3-D ventricle chamber using rat heart cells or human heart cells derived from iPSCs in biodegradable scaffolds. Through ultrasound and other clinical tests, the researchers demonstrated that the model mimics the structure and contractile function of the natural heart. Using this engineered heart chamber, they developed a model for a type of arrhythmia (irregular heartbeat). This system may provide a way to study arrhythmia heart diseases to understand their causes, to screen drugs for their effects on the heart and to develop new drugs for heart diseases. Read more about the advance from Harvard University. Posted December 2018 | Researchers supported by NCATS’ Tissue Chip for Drug Screening program have developed a 3-D working model of a human heart chamber. | /sites/default/files/3d-heart_1260x630%202.jpg | Working Model of a Heart Chamber | Researchers supported by NCATS’ Tissue Chip for Drug Screening program have developed a 3-D working model of a human heart chamber. | /sites/default/files/3d-heart_1260x630%202.jpg | Working Model of a Heart Chamber | ||
| 13264 | A Model for Accelerating Translational Research in Real Time | Before a clinical trial can begin, an institutional review board (IRB) must determine that the study is ethical and that participants’ rights are protected. This oversight step is crucial, but it can take a long time and delay studies from starting, particularly those at multiple sites. Credit: Albert Einstein College of Medicine Recognizing this translational barrier, NIH created the single IRB policy for multisite trials, and NCATS has spearheaded initiatives to implement this policy. Although these efforts have removed redundancy in the IRB process, roadblocks can still delay single IRB reviews. Investigators of the Clinical & Translational Science Institute of Southeast Wisconsin (CTSI) at the Medical College of Wisconsin (MCW) recognized this problem at their NCATS Clinical and Translational Science Awards (CTSA) Program hub. The investigators devised and piloted an innovative approach called Real-Time IRB that reduced the time from study submission to final approval by 70 percent. It is common for IRB members to have questions or require small changes to a study before they can approve it. The back and forth between the IRB and researchers to resolve issues can take a lot of time. In fact, the MCW investigators found that studies that needed small changes took about 36 days longer to receive approval than studies that the IRB approved upon first review. The Real-Time IRB approach requires the study team to attend the IRB meeting and discuss their proposed study with the committee. The team leaves the room while the IRB discusses any issues or modifications needed. The study team can then update its documents in a nearby room in “real time” while the IRB discusses other studies on the agenda. Later during the same meeting, the study team shares its changes with the committee for approval that same day. Read more about this work in the October 2018 issue of the Journal of Empirical Research on Human Research Ethics. Posted December 2018 | CTSA Program investigators 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. | /sites/default/files/real-time%20irb_1260x630.jpg | A Model for Accelerating Translational Research in Real Time | CTSA Program investigators 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. | /sites/default/files/real-time%20irb_1260x630.jpg | A Model for Accelerating Translational Research in Real Time | ||
| 13240 | NCATS Collaborations Lead to Potential New Drug Combinations for Childhood Cancers | Translational Science HighlightNCATS scientists are bridging translational gaps in moving promising compounds and drugs closer to clinical trials for several childhood cancers that lack effective treatments. In addressing this unmet need, the NCATS team is building expertise in drug interactions that will help it continue to foster new collaborations to tackle translational challenges.In the past several decades, researchers have made great gains in treating children who have cancer, particularly those with blood cancers such as leukemia and lymphoma. But finding effective therapies for certain cancer types has been more difficult.To help address this challenge, a team of researchers in NCATS’ Chemistry Technology and Matrix Screening programs is rethinking treatment approaches for several difficult-to-treat childhood cancers. Using NCATS’ matrix combination screening capabilities, team leader Craig Thomas, Ph.D., and his colleagues are taking some of the guesswork out of drug discovery. The team uses this screening technique to rapidly test the effects of thousands of different drug combinations on key disease processes. Scientists can examine the most effective combinations, find the best doses of each drug and learn more about their effects on cells. The NCATS team and its collaborators focus on drug combinations that work in synergy and that can be tested in laboratory models and, ultimately, people.“The matrix screening technology is a perfect example of how translational science should work,” said Anton Simeonov, Ph.D., NCATS scientific director. “This is the nature of what NCATS does — finding answers and enabling others to develop therapies that can be tested through clinical trials to improve patients’ lives.”Collaborating at NIHNCATS’ chemistry technology lead Craig Thomas, Ph.D. (left), discusses the results of a drug screen with scientists Michele Ceribelli, Ph.D., and Scott Hoyt, Ph.D. (Daniel Soñé Photography)One disease under study is Ewing sarcoma, a rare bone and soft tissue cancer that mostly affects teenagers and young adults. Although usually initially treatable, like many cancers, it is more difficult to cure if it returns.Christine Heske, M.D., who treats children and young adults with Ewing sarcoma and studies the disease at NIH’s National Cancer Institute (NCI), recently teamed with the NCATS group to show that Ewing sarcoma cells were particularly sensitive to nicotinamide phosphoribosyltransferase (NAMPT inhibitors), a class of drugs that has been tested only in adult cancer types. These drugs block the activity of an enzyme that is important for the metabolic function of cells. Cancer cells seem to rely on this enzyme more heavily than normal cells do.Using matrix screening, NCATS researchers demonstrated how NAMPT inhibitors worked together with poly (ADP-ribose) polymerase (PARP) inhibitors, a class of drugs approved by the Food and Drug Administration to treat ovarian and breast cancers, in laboratory cell models of Ewing sarcoma. Meanwhile, Heske’s team at NCI showed the effectiveness of the drug combination in mouse models of the cancer. The researchers reported their results in the Dec. 1, 2017, issue of Clinical Cancer Research.“Seeing which compounds and drugs have enhanced anticancer activity together may allow us to lower doses of each in treating patients,” Heske noted. She is hopeful the work eventually will lead to testing in patients.In another collaborative effort, NCATS scientists, NCI colleagues and an international team of researchers examined rhabdomyosarcoma (RMS), a rare muscle cancer that often affects children and adolescents. There are two main forms of the disease in children, and both can have difficult treatment paths.One form of RMS is caused by a genetic mix-up in which DNA changes places on a chromosome, a structure that houses genetic material inside a cell. This error prompts cells to mistakenly grow out of control — a hallmark of cancer. The other major form of RMS works through a different biological route.The collaborators screened compounds and drugs for their activity against both types of RMS. They found combinations of compounds that were more active against each form of cancer.The first set of results, published in the August 2017 issue of Cancer Discovery, showed that a group of compounds called BRD4 inhibitors was more effective against the form of RMS caused by the genetic switch. The scientists further reported in the July 4, 2018, issue of Science Translational Medicine that the other form of RMS seemed more susceptible to a combination of compounds that worked against a completely different target.“These screens identified important vulnerabilities in each form of RMS because of each one’s unique sensitivities to certain classes of drugs,” said NCI’s Marielle Yohe, M.D., Ph.D., a co-author on both studies. “This insight can help us determine which drugs are most effective.”One Partnership Leads to AnotherTreatment with the drug trametinib can help immature muscle cells, which characterize the cancer rhabdomyosarcoma, develop into mature cells (shown in the bottom two panels in green). In the top two panels, immature cells (blue) are treated with a control drug, with no effect. (Reprinted with permission from Yohe, et al., Sci. Transl. Med. 10, eaan4470 (2018))For the past three years, NCATS has collaborated with Stanford University neuro-oncologist Michelle Monje, M.D., Ph.D., to study a rare childhood brain tumor called diffuse intrinsic pontine glioma (DIPG). These aggressive, hard-to-treat tumors are the leading cause of brain cancer death among U.S. children.Monje and her team had discovered that a drug called panobinostat showed activity against DIPG cells. Buoyed by some preliminary successes, Monje wanted to look at how a range of compounds and combinations affected the cancer.In 2015, NCI’s Katherine Warren, M.D., who was working with Monje, spearheaded a collaboration with NCATS. Since then, Monje, Thomas and their teams have used NCATS’ matrix screening technology to examine single compounds and combinations of compounds against DIPG patient cells. While they continue studies with panobinostat, they also are confronting another challenge: finding effective treatments that cross the brain’s protective blood-brain barrier.“We’re examining possible drug targets identified through the NCATS screens and are working to bring the most promising combinations to clinical trials,” Monje said. “The pieces are beginning to fall into place.”Honing Translational ExpertiseThese collaborative efforts with disease experts enable NCATS scientists to build their translational knowledge portfolio. By participating in dozens of such studies, NCATS is gaining vast knowledge about how drugs can interact, which is a complication associated with many diseases and disorders. NCATS scientists are discovering that some combinations nearly always work in tandem against certain types of cancers, especially those combinations that affect processes cancer cells need to survive.“We want to learn the broader trends and build institutional expertise,” Thomas said. “If we understand why some molecules and combinations are more or less effective and what variations in a person’s genome drive certain responses, we can make better decisions as we translate basic science discoveries to improve human health.”Posted December 2018 | To help address challenges in treating children with cancer, a team of NCATS researchers is rethinking treatment approaches for several difficult-to-treat childhood cancers. | /sites/default/files/craig-thomas-cancer_1260x630.jpg | Potential New Drug Combinations for Childhood Cancers | To help address challenges in treating children with cancer, a team of NCATS researchers is rethinking treatment approaches for several difficult-to-treat childhood cancers. | /sites/default/files/craig-thomas-cancer_1260x630.jpg | Potential New Drug Combinations for Childhood Cancers | ||
| 13246 | NCATS Makes Science Fun for Local Middle Schoolers | As part of the 10th annual Frontiers in Science and Medicine Day on Nov. 9, 2018, nearly 100 middle school students experienced a glimpse of what it is like to be a scientist at NCATS. Sponsored by Johns Hopkins University (JHU) on and near its Montgomery County, Maryland, campus, the annual event includes hands-on science activities and lab tours, with the goal of encouraging careers in related fields. A student shows the result of his chromatography experiment during Frontiers in Science and Medicine Day 2018. (Daniel Soñé Photography) Students explored several of NCATS’ high-throughput screening robotic labs, viewed images from high-powered electron microscopes and saw science experiments in action. Charles “Pepper” Bonney, who oversees and maintains NCATS’ laboratory equipment, conducted the tour and explained many of the different types of jobs in the lab. Several NCATS postdoctoral students conducted chromatography experiments with the middle schoolers. Chromatography is a laboratory technique commonly used to separate a mixture of chemical substances into its individual components, so that they can be thoroughly analyzed. The students drew designs on special paper used in lab experiments with both permanent and water-soluble markers to test the different effects that water and ethanol had on the designs. During the experiment, students were able to see how the marker ink traveled up the paper at different speeds in the two types of liquid. NCATS was one of many academic institutions, biotechnology and pharmaceutical companies, and government agencies that hosted, in total, more than 600 students from Briggs Chaney and Benjamin Banneker middle schools. View the Flickr album from the event. Posted December 2018 | As part of the 10th annual Frontiers in Science and Medicine Day, nearly 100 local middle school students got a glimpse of what it is like to be a scientist at NCATS. | /sites/default/files/frontiers2018_1260x630.jpg | NCATS Makes Science Fun for Local Middle Schoolers | As part of the 10th annual Frontiers in Science and Medicine Day, nearly 100 local middle school students got a glimpse of what it is like to be a scientist at NCATS. | /sites/default/files/frontiers2018_1260x630.jpg | NCATS Makes Science Fun for Local Middle Schoolers | ||
| 13150 | NCATS Director Statement: Translational Science to Address the Opioid Crisis | The toll of opioid addiction and overdose death continues to devastate individuals, families and communities, contributing to an unprecedented decrease in life expectancy in the United States over the past several years. New, safe interventions are urgently needed to combat opioid misuse and addiction, and to treat pain. Translational science is playing a crucial role in advancing knowledge about the underlying biology of addiction and pain, developing new interventions to treat them, and determining which interventions are most effective. With NIH HEAL (Helping to End Addiction Long-termSM) Initiative support, NCATS is providing a suite of translational science resources and expertise to investigators working on opioid and pain research. With its expert collaborative infrastructure already in place, the Center is leading trans-NIH collaborative HEAL efforts that include: Developing new testing platforms that more closely model human biology than currently available cell and animal models, using induced pluripotent stem cells (iPSCs), tissue chips and 3-D tissue bioprinting; Identifying and de-risking potential therapies that work in novel ways through assays (tests) and high-throughput screening to select and develop compounds that show promise as potential drugs; Accelerating the identification of promising chemical structures and developing those into pharmacological or drug-like compounds; Advancing promising new drug candidates through rigorous preclinical efficacy and safety studies for first-in-human clinical trials as required by the U.S. Food and Drug Administration; and Facilitating opportunities to partner with other NIH Institutes and Centers as well as clinical research institutions nationwide to conduct studies to determine the effectiveness of existing pain management treatments. Through the NIH HEAL InitiativeSM, NCATS is applying the breakthrough approaches of translational science to advance new treatments for the opioid crisis, the public health challenge of our era. Christopher P. Austin, M.D. Director National Center for Advancing Translational Sciences Posted December 2018 | With NIH HEAL Initiative support, NCATS is providing a suite of translational science resources and expertise to investigators working on opioid and pain research. | /sites/default/files/dr-austin-robot1260x630.jpg | Director Statement: Translational Science to Address the Opioid Crisis | With NIH HEAL Initiative support, NCATS is providing a suite of translational science resources and expertise to investigators working on opioid and pain research. | /sites/default/files/dr-austin-robot1260x630.jpg | Director Statement: Translational Science to Address the Opioid Crisis | ||
| 13120 | NCATS Researchers Develop New Approach to Identify Potential Cancer Drugs | Translational Science Highlight NCATS researchers overcame a translational roadblock by developing a series of assays (tests) to identify compounds that disrupt a driver of several cancers. The work, using NCATS’ high-throughput screening technology to test thousands of compounds at once, provides a template for other scientists to discover new compounds that could be useful as cancer therapies. For years, scientists have been searching for ways to find molecules that could block NSD2, an enzyme that affects gene activity and is thought to play an important role in the development of several types of cancer. But finding these molecules — called NSD2 inhibitors — has been difficult, mainly because researchers lacked useful laboratory assays (tests) to identify compounds that could block NSD2 activity. NCATS scientists, led by biology group leader Matthew Hall, Ph.D., and senior scientist Nathan Coussens, Ph.D., sought to bridge this gap by developing a new strategy to find NSD2 inhibitors. One reason NSD2 is challenging to study is that it is only active on a structure called a nucleosome, which is hard to reproduce in the lab. A nucleosome consists of genetic material — DNA — wrapped around proteins, like thread around a spool. NSD2 belongs to a family of proteins that can control gene activity by altering the nucleosome, which then makes genes more available for the cell to turn on and off. Many cancers are associated with higher-than-normal NSD2 activity. When NSD2-regulated gene activity is increased, this can include genes that drive cancer development. This illustration depicts a family of proteins — including the cancer target protein NSD2 (highlighted in yellow) — arranged around a structure called a nucleosome. NCATS researchers developed a new series of high-throughput screening assays to determine whether various compounds could affect NSD2’s ability to modify nucleosome proteins. (Kyle Brimacombe Photo) The NCATS team developed a new series of high-throughput screening assays to determine whether various compounds could affect NSD2’s ability to modify nucleosome proteins. With the help of scientists at the biotechnology company Reaction Biology Corp., which provided the enzyme and nucleosomes to study, these assays allowed researchers to test different doses of many compounds at the same time. Over four months, they examined more than 16,000 compounds for their effects in disrupting NSD2 function. After narrowing the search to only 174 candidate compounds, the NCATS team developed another series of assays that pinpointed five final compounds for further testing. NCATS and Reaction Biology researchers reported their work in the Journal of Biological Chemistry. The NCATS scientific team had to determine whether the compounds were suitable as starting points for possible drugs. A compound that inhibited an enzyme such as NSD2, for example, also could affect the activities of other related enzymes at the same time and turn out to be a poor drug candidate. Ultimately, none of the five compounds were considered suitable for further development as drugs. “In many cases, molecules we identified looked promising early on, but when we tested them further, they affected too many other enzymes, which ruled them out as viable drug candidates,” Coussens said. “We wanted to develop a strategy that others could use to build on with their own resources. We were able to provide a template for identifying molecules that inhibit NSD2.” The findings set the stage for future discovery of new compounds that can block NSD2 activity and potentially be useful against cancers, including a form of childhood leukemia, multiple myeloma, and lung and stomach cancers. Members of the NSD2 protein family also have been implicated as cancer drug targets. The researchers’ study design can be applied to these related proteins as well. Next, Coussens, Hall and their colleagues plan to work with medicinal chemists to further study NSD2 and improve the properties of some of the more promising compounds they uncovered to further develop them as potential drugs. “At NCATS, we try to tackle ‘tough’ targets, with the goal of creating tools that help scientists understand the biology and therapeutic relevance of those targets,” Hall said. “Although the NSD2 inhibitors we identified may not be ideal yet, we are planning to screen hundreds of thousands of molecules to find those that the research community might be able to make more useful.” Posted December 2018 | NCATS researchers develop a series of assays (tests) to identify compounds that disrupt a driver of several cancers. | /sites/default/files/hall-cancer_1260x630.jpg | New Approach to Identify Potential Cancer Drugs | NCATS researchers develop a series of assays (tests) to identify compounds that disrupt a driver of several cancers. | /sites/default/files/hall-cancer_1260x630.jpg | New Approach to Identify Potential Cancer Drugs | ||
| 13075 | NCATS Announces Joni Rutter as New Deputy Director | November 28, 2018NCATS Director Christopher P. Austin, M.D., announced the selection of Joni L. Rutter, Ph.D., as the Center’s new deputy director. Rutter is slated to join NCATS in early 2019.“I am thrilled that Dr. Rutter is joining the NCATS leadership team,” Austin said. “I look forward to working with her to advance the translational science that will get more treatments to more patients more quickly.”Joni Rutter, Ph.D. (NCATS)Rutter is currently serving as director of scientific programs at the NIH All of Us Research Program, which she helped to build successfully from the ground up. She leads All of Us scientific, programmatic development and implementation efforts to build a national research cohort of 1 million or more U.S. participants to advance precision medicine. All of Us aims to build the most diverse biomedical data resource of its kind to help researchers gain better insights into the biological, environmental and behavioral factors that influence health. As deputy director of NCATS, Rutter will be responsible for planning, executing and assessing the Center’s complex and multifaceted preclinical and clinical programs, and will be a key national spokesperson for translational science. She will also help guide NCATS’ Advisory Council and Cures Acceleration Network Review Board activities, and serve as the Center’s scientific liaison to All of Us, helping to establish more robust interactions with NCATS programs.Prior to joining All of Us, Rutter was the division director of neuroscience and behavior at the NIH National Institute on Drug Abuse (NIDA). There, she led integrated research on basic and clinical neuroscience, brain and behavioral development, genetics, epigenetics, computational neuroscience, bioinformatics and drug discovery. She also coordinated the NIDA Genetics Consortium and biospecimen repository.Rutter is internationally recognized for her work in basic and clinical research in human genetics and in the study of genetic and environmental risk factors focusing on the fields of cancer and addiction. She earned her Ph.D. from Dartmouth Medical School and completed a fellowship at the NIH National Cancer Institute.“I am excited to join Dr. Austin and the entire NCATS team,” Rutter said. “Translational science is vital for enhancing the diagnostics and treatment in all areas of health and disease, and I look forward to adding to NCATS’ deep experience and palpable passion for innovation throughout its intramural and national research programs.” | On Nov. 28, 2018, NCATS announced Joni Rutter, Ph.D., as its new deputy director. Rutter is slated to join NCATS in early 2019. | /sites/default/files/dd_rutter_1260x630.jpg | NCATS Announces Joni Rutter as New Deputy Director | On Nov. 28, 2018, NCATS announced Joni Rutter, Ph.D., as its new deputy director. Rutter is slated to join NCATS in early 2019. | /sites/default/files/dd_rutter_1260x630.jpg | NCATS Announces Joni Rutter as New Deputy Director | ||
| 13060 | Paul G. Yock, M.D. | Paul Yock is the Martha Meier Weiland Professor of Medicine and co-chair of Stanford University’s Department of Bioengineering, with courtesy appointments in the Graduate School of Business and the Department of Mechanical Engineering. Yock began his faculty career as an interventional cardiologist at the University of California, San Francisco (UCSF) and then moved to Stanford University in 1994. He served as acting chief of the Division of Cardiovascular Medicine from 1997 to 1998. After undergraduate and graduate study at Amherst College and Oxford University, respectively, Yock received his M.D. from Harvard Medical School, followed by internship and residency training at UCSF and a fellowship in cardiology at Stanford University. Yock founded the Stanford Center for Research in Cardiovascular Interventions (now the Stanford Center for Cardiovascular Technology) and has trained more than 25 fellows in intravascular ultrasound and interventional cardiology. In 1986, he founded Cardiovascular Imaging Systems, Inc., which was acquired by Boston Scientific in 1994. Yock also directs Stanford Biodesign, a unit of Stanford’s Bio-X initiative, whose the mission is to develop leaders in biomedical technology innovation. Twenty-five fellows have completed the Biodesign Innovation Fellowship since 2001. | /sites/default/files/paul_yock_1260x630_0.jpg | Paul G. Yock, M.D. | /sites/default/files/paul_yock_1260x630_0.jpg | Paul G. Yock, M.D. | ||||
| 13054 | NCATS Day Participants Share Perspectives to Advance Research Engagement | Translational Science Highlight NCATS Day is a cornerstone of the Center’s efforts to engage and collaborate with a variety of diverse stakeholders — particularly patients and their communities — to advance translational science. “I’m not too thrilled about injections,” one woman said. “I’m a big scaredy-cat with needles,” agreed the man next to her. Both were participants in a demonstration of an “engagement studio”— a way of gathering community input on a proposed research project — during NCATS Day 2018: Engaging Patients and Communities for Smarter Science. The event also featured diverse panelists discussing best practices for developing strong research relationships, the value of partner perspectives and the importance of sharing results. Aided by a facilitator, the engagement studio participants shared why they would or would not want to participate in a research study about a possible new injectable drug for arthritis pain. Their input provided investigator Michael Iadarola, Ph.D., of the NIH Clinical Center, with ideas about how to best reach community members who might want to enroll in his study. Making Engagement Real NCATS Day 2018 participants report on their findings from the breakout sessions. (Daniel Soñé Photography) The concept of engagement studios has been developed and refined throughout the past decade by Vanderbilt University collaborators, including Consuelo H. Wilkins, M.D., M.S.C.I., who is the co-principal investigator of the NCATS-funded Recruitment Innovation Center (RIC). “If you’re doing study recruitment well, you’re engaging people,” Wilkins said. “But if the only reason you’re interacting with people is to recruit them, you’re not engaging.” True engagement means that the community’s input leads to changes in the researchers’ plan for the study, she added. Cheryl Jernigan, CPA, FACHE, lead patient advocate at the University of Kansas Medical Center, said that in her experience, neither scientists nor patients fully realize why patient input is needed. “Scientists think they know what we want, but they don’t,” Jernigan said. “We think we can’t tell them things because we don’t know science.” For example, Iadarola had assumed that patients would love the fact that his arthritis drug is injected into the knee, because it meant the drug wouldn’t travel through the bloodstream to the whole body. “This, to me, had always been a real advantage,” he said. After the engagement studio, however, Iadarola realized that the injection might make some people reluctant to participate. Sharing Results with Participants One of the most important elements of engagement, Wilkins and Jernigan agreed, is telling participants about results of the research to which they contributed. “The foundation of respecting people as partners in research should be discussions about what is of value to them,” Wilkins said. ”Returning results is a key part of that.” Jernigan shared that she has often been involved in the planning stages of a study as a patient advocate, then never heard whether the study was funded. It is much worse for patients who volunteer their time, fight their discomfort with medical procedures, and sometimes even risk their lives to participate in a study, then never hear the results, she added: “It’s appalling to know that all of these people gave their time and didn’t know what happened.” This breakdown in communications may stem partly from the fact that many academics are uncomfortable communicating with non-academics, Wilkins said. She also noted that traditional ways of sharing results, such as through journal articles and presentations at scientific conferences, are good for researchers’ careers, but investigators do not have similar incentives to share their results with study participants or the general public. To help bridge this gap, RIC investigators have created templates that researchers can use to share results with study participants and community members. Part of a Recruitment and Retention Toolbox, the templates are easy to use and follow the format of a journal abstract, which is familiar to scientists. Advancing Science Through Collaboration NCATS Day 2018 working group members and panelists. (Daniel Soñé Photography) Kristi Graves, Ph.D., is a clinical psychology researcher at Georgetown University who specializes in issues related to cancer. She has worked for 10 years with Nueva Vida, an organization that supports Latino families in the Washington, D.C., area who are affected by cancer. She emphasized the importance of collaborating with patients and their advocacy groups in designing study questions. A few years into her collaboration with Nueva Vida, Graves received funding for a three-year project testing a series of workshops to help cancer patients and their caregivers improve their quality of life and coping skills. “It was a highlight of my career,” Graves said. The ideas for the workshops came from Nueva Vida staff, and they and other community partners were involved in designing the sessions. For example, at one meeting, a partner asked if the study included questions on the financial burden of cancer. Adding that question produced compelling data. The project came about in the first place because of Nueva Vida, Graves explained. Nueva Vida’s executive director called to tell her about a new organization that was funding research on community-focused science. Graves and her colleagues rushed to put together an ultimately successful proposal. Peter Grayson, M.D., M.Sc., head of the Vasculitis Translational Research Program at the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), said the Vasculitis Foundation, a patient advocacy group, was critical to his early career success. The foundation helped promote him to the research and patient communities. The resulting interactions have helped him define his study questions and better understand that scientists and patients often have very different perspectives on what is most important. “For example, keeping patients off dialysis is important, but more important to them is finding ways to deal with fatigue, because that’s their daily experience,” he said. NCATS Director Christopher P. Austin, M.D., carried this theme of collaboration through his concluding remarks for NCATS Day 2018, reflecting on the importance of partnership for scientific progress. “The history of science shows that insights from the community — the medical community as well as the patient community — have led to many scientific insights that have public health significance,” he said. “At NCATS, we feel strongly that the only way to be sure we know what we’re doing is to ask people and involve them in the work from the very beginning.” View photos from the event. Posted November 2018 | NCATS Day is a cornerstone of the Center’s efforts to engage and collaborate with a variety of diverse stakeholders, particularly patients and their communities, to advance translational science. | /sites/default/files/ncats-day-2018_1260x630.jpg | NCATS Day Participants Share Perspectives | NCATS Day is a cornerstone of the Center’s efforts to engage and collaborate with a variety of diverse stakeholders, particularly patients and their communities, to advance translational science. | /sites/default/files/ncats-day-2018_1260x630.jpg | NCATS Day Participants Share Perspectives |
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