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| 7374 | First NCATS Day Demonstrates Commitment to Patients Through Smarter Science | Translational Science Highlight NCATS hosted an inaugural event to identify patients’ needs and brainstorm ways to foster engagement at every step of the translational science spectrum. To gain more insight about patients’ needs and discuss opportunities to integrate the patient perspective into translational research, NCATS convened the first NCATS Day on June 30, 2017, on the NIH campus in Bethesda, Maryland. More than 150 people attended, including patients, their families and other caregivers, and representatives of more than 75 patient and disease advocacy groups. With a theme of “Partnering with Patients for Smarter Science,” the all-day event enabled participants to learn more about NCATS and research supported by the Center. It also served as a forum for NCATS staff and researchers to hear directly from patients about their needs, establish new communication channels or strengthen existing ones and identify ways to enhance patient participation in research. The Power of Partnering with Patients The process of developing new treatments takes too long and is too costly. The situation, especially for rare diseases, has been bleak. “Incremental change is not going to do it,” said NCATS Director Christopher P. Austin, M.D., during his opening remarks. “We need a quantum-level change in our efficiency and effectiveness.” Austin noted that biomedical researchers are passionate about learning about diseases and finding new and better treatments but often fail to ask the people who are supposed to benefit from that work about what they want from the products under development. Although many NCATS programs place a high value on patient engagement, it is not always obvious how patients and their support organizations can be involved in research. This event was designed to begin a dialogue between NCATS and the public about how the patient perspective can be included in translational science. James Hendrix, Ph.D., director of global science initiatives for the Medical and Scientific Relations Division of the Alzheimer’s Association, thought the time might be right for disruptive research. “The time is coming to be heretical — even crazy — to transform the current paradigm for developing new treatments,” he said. “What seems to be the most logical path from basic science to new treatments may be too slow. The current situation demands approaches that are higher in risk but potentially higher in reward.” Daniel Soñé Photography. “Too frequently, at the end of the drug development process, the treatment misses the mark in terms of patients’ needs,” Austin said. “At NCATS, we are committed to getting input from the people living with diseases to make sure that research aligns with their priorities.” Dr. Christopher Austin. Daniel Soñé Photography. That is why NCATS strives to find ways to involve patients in all stages of research, ranging from preclinical studies using cell lines and animal models to human trials for evaluating investigational drugs and to dissemination and implementation research focused on identifying best practices. The second goal in NCATS’ Strategic Plan embodies this concept: As the eventual recipients of these research endeavors, patients’ participation as members of the research team provides insight and meaning to the creation, testing and clinical implementation of new medical interventions. About a third of NCATS Advisory Council members are disease advocates, patients or caregivers. In addition, institutions funded through NCATS’ Clinical and Translational Science Awards (CTSA) Program have community advisory boards to help promote patient engagement and provide feedback to researchers. CTSA Program hubs support training on patient and community engagement, and many employ patient engagement specialists. In addition, CTSA Program researchers have access to the Recruitment Innovation Center, which is transforming recruitment methods in order to enroll participants in clinical trials more quickly, thereby overcoming a major barrier in the testing of potential new treatments. A Promising Example of Collaboration with Advocates Many different paths are available to patient groups interested in accelerating translational science. For example, advocacy organizations can directly support research and scientists studying the disease of interest. This approach was highlighted in a case study presented during NCATS Day by Michael Iannotti, Ph.D., a postdoctoral fellow in the NCATS Division of Preclinical Innovation, and Jean-Marc Quach, M.B.A., the executive director of The Alpha-1 Project at the Alpha-1 Foundation. They demonstrated how the foundation is working with NCATS to find new treatments for patients who have a defect in an enzyme called alpha-1 antitrypsin, causing serious liver and lung disease. The Alpha-1 Project supports a research fellowship for Iannotti at NCATS, where he designs new assays (tests) and conducts research into promising drug candidates that could help correct the enzyme’s function. Michael Iannotti, Ph.D. (left), listened as Jean-Marc Quach, M.B.A. (right), explained, “This partnership is succeeding because it builds on the strengths of the Alpha-1 Foundation and NCATS. NCATS is sharing its technical expertise, technologies and libraries of compounds. The foundation contributes its deep knowledge of patients’ needs and the resources supporting Iannotti’s research. We are assembling tools and materials that bring us several steps closer to finding a cure for alpha-1 antitrypsin disease.” Daniel Soñé Photography. “Working with a disease advocacy group has given me opportunities to meet and talk with patients and their families,” Iannotti said. “These interactions would normally not be available to me, since my work is performed in a research lab, not in a clinic. Spending time with the patient community has strengthened my commitment to discovering therapies for alpha-1 antitrypsin deficiency.” NCATS Day participants found the case study enlightening and suggested that future communications include more of these examples presented not only by scientists but also by patients, caregivers and other advocates. Showcasing NCATS Programs and Initiatives A series of presentations and poster sessions highlighted a wide range of exciting work taking place at NCATS or with Center support. The idea was to ignite interest in translational research and help patients and advocates identify research programs that might fit with their interests and needs. For example, Mary Dollear, vice president of the Lupus Society of Illinois, said of NCATS’ Tissue Chip for Drug Screening program, “I am really excited to learn about this technology. People with lupus are often reluctant to participate in clinical trials, because they worry about their disease flaring up. But the tissue chips might be a way to test treatments on tissues from patients to see what might work for them — sort of like a clinical trial for each person.” Kathryn Devanny, M.A., M.P.H., director of advocacy for PatientWorthy, said, “I was interested in this event because I wanted to know more about how rare disease patient advocacy groups, both formal and informal, could interact with NCATS’ programs. Advocacy groups are a critical source of patient and disease information, spanning natural history to DNA, and their goals dovetail with those of NCATS. How would such organizations take the first step to working with NCATS? What is the best way for them to engage the scientists, researchers and technology experts at NCATS?” Daniel Soñé Photography. The Big Picture NCATS Day wrapped up with a group discussion focused on the key ideas advanced during earlier breakout sessions. Several themes emerged, including the importance of two-way communication between scientists and patients. The scientific community should discuss research in easily understood language. Efforts to boost science literacy could help communities understand the research process and scientific evidence, as well as roles that they can take on. Camille Hart, M.P.H., program manager for community engagement at the CTSA Program hub at the University of Arkansas for Medical Sciences, was on a quest for information about NCATS programs that would be of interest to community partners — both organizations and individuals — back in Arkansas. “Last month, we ran a Patient Scientist Academy to help patients learn about how they can get involved in research as advisors, grant reviewers or study participants,” Hart said. “I am always on the lookout for new ways to engage people in the research process.” Daniel Soñé Photography. For example, Jessica Nagro, federal government and health policy manager with the National Psoriasis Foundation, shared an idea: “Our foundation set up a program called Citizen Pscientist that links people with psoriatic disease in the first-ever global online research network dedicated to this condition. The Citizen Pscientists answer questions prepared by researchers, and they can conduct their own analyses and discuss the information.” Other comments touched on the need for training scientists to engage patients in research in meaningful ways, especially in early-stage studies. Event participants also underscored the importance of recruiting diverse and underserved populations for clinical trials. Sue Hargrave of Itching to Help!, LLC, NPC, an advocacy group helping people with chronic urticaria (hives), discussed a poster with Madhu Lal-Nag, Ph.D., lead of the trans-NIH RNAi facility in NCATS’ Division of Preclinical Innovation. Hargrave identified scientific literacy as a patient need and said she was at NCATS Day to “lurk and learn” from Center staff and representatives of other organizations. Communication tools might help patients sort fact from fiction. “Too often, patients focus on ‘treatments’ they read about on the Internet — things that are unproven,” she said. “How can we help people distinguish treatments based on real scientific evidence from anecdotes on social media?” Daniel Soñé Photography. Several participants expressed enthusiasm about engaging with NCATS and were interested in “on-ramps” or a “matchmaking” system to find the best fit between patient/disease organizations and the Center. Building on this idea, another suggestion was to create a “decision tree” for moving through translational science, including working with the U.S. Food and Drug Administration and the Centers for Medicare & Medicaid Services. Several people envisioned opportunities for advocacy groups to come together to develop research platforms and share resources to address collective needs. Stay Tuned — There’s More to Come NCATS Day was the first of many events being planned to strengthen ties between the Center and the patient community. On Sept. 8, 2017, NCATS will host rare diseases and other patient community members at its Toolkit for Patient-Focused Therapy Development: Demonstration and Dissemination Meeting. Participants will have an opportunity to learn how the toolkit can help with therapeutic development activities and to provide feedback to expand available content. “Translational science is a team sport,” Austin said. “We need contributions from all the team members, especially patients, their family members and advocacy organizations. By working together, we can get treatments to patients sooner and ensure that treatments meet patients’ needs.” And that’s what smarter science is all about. Posted July 2017 | NCATS hosted an inaugural event to identify patients’ needs and brainstorm ways to foster engagement at every step of the translational science spectrum. | /sites/default/files/ncats-day-900.jpg | First NCATS Day Demonstrates Commitment to Patients Through Science | NCATS hosted an inaugural event to identify patients’ needs and brainstorm ways to foster engagement at every step of the translational science spectrum. | /sites/default/files/ncats-day-900.jpg | First NCATS Day Demonstrates Commitment to Patients Through Science | ||
| 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 |
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