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| 8670 | CTSA Program-Supported Investigators Validate Biomarkers for NPC1 Treatment | With support from the Sharing Partnership for Innovative Research in Translation (SPIRiT) consortium, Daniel Ory, M.D., of Washington University in St. Louis, and Charles Vite, D.V.M., Ph.D., of the University of Pennsylvania, collaborated to validate biomarkers intended to monitor the response to therapy for Niemann-Pick disease type C1 (NPC1). SPIRiT, a collaboration among six Clinical and Translational Science Awards (CTSA) Program hubs, was critical in advancing an NCATS Therapeutics for Rare and Neglected Diseases (TRND) project to test a cyclodextrin therapy for NPC1 in clinical studies. Read more about the cyclodextrin study. At left, fibroblasts homozygous for mutations in NPC1 demonstrate an increased accumulation of red Lysotracker staining indicative of the storage disease. At right, addition of cyclodextrin rescues this lysosomal storage defect. (NCATS Photo) Posted November 2017 | CTSA Program-Supported Investigators Validate Biomarkers for NPC1 | CTSA Program-Supported Investigators Validate Biomarkers for NPC1 | ||||||
| 8669 | NCATS Explores Science with Middle School Students | Local middle school students tour NCATS’ laboratories during the Frontiers in Science and Medicine event on Oct. 27, 2017. NCATS staff engaged nearly 60 local middle school students in science experiments and led tours of the Center’s laboratories during the ninth annual Frontiers in Science and Medicine event on Oct. 27, 2017. Sponsored by the Johns Hopkins University Montgomery County Campus, the event is designed to expose students to science and medicine and to encourage them to pursue careers in these fields. NCATS senior scientists and postdoctoral students helped students from Kingsview Middle School in Germantown, Maryland, and Poole Middle School in Poolesville, Maryland, participate in a chromatography experiment. Chromatography is a laboratory technique for the separation of a mixture. Using permanent and non-permanent markers, the students tested the different effects that water and ethanol (used as solvents) had on special chromatography paper used in laboratory experiments. During the experiment, students could watch the components of marker ink travel up the paper at different speeds, causing them to separate, in the two types of liquid. The students also learned how Center staff use cutting-edge equipment for scientific research. Charles “Pepper” Bonney, NCATS’ laboratory automation specialist, explained how assay plates are used to test different combinations of potential medicines. He also demonstrated how high-throughput screening robots test the different combinations of chemical compounds in the plates and showed the students different imaging devices that enable researchers to see the results of the assays. View event photos and videos on Flickr. Learn more about the event. Posted November 2017 | NCATS staff engaged nearly 60 middle school students in science experiments and led tours of the Center’s laboratories during the 9th annual Frontiers in Science and Medicine event on Oct. 27, 2017. | /sites/default/files/frontiers-in-sciencex900.jpg | NCATS Explores Science with Middle School Students | NCATS staff engaged nearly 60 middle school students in science experiments and led tours of the Center’s laboratories during the 9th annual Frontiers in Science and Medicine event on Oct. 27, 2017. | /sites/default/files/frontiers-in-sciencex900.jpg | NCATS Explores Science with Middle School Students | ||
| 8668 | NCATS Human Subjects Research Prior Approval Frequently Asked Questions | Why do awardees need prior approval for pilot studies and KL2 projects involving human subjects? Which studies require prior approval? How do I submit a prior approval request? What information and documents should be included in a prior approval request? Is IRB approval always required for NCATS prior approval? Who should submit prior approval request documents and to whom? Do research studies involving human cell lines or tissue repositories need prior approval? If an investigator has not received a response to a prior approval request, may the investigator proceed with the pilot study or K-scholar project? In general, how long does the prior approval process take? How will investigators be notified of decisions regarding prior approval requests? How can an investigator request additional information about the prior approval process and/or requirements? What can investigators do to maximize the efficiency of the review process? When NCATS requests clarification on a prior approval request, should the institution resend only the questioned/corrected documents? Is prior approval required for studies that have been determined by the IRB to fall under Human Subjects Research Exemption 4 (45 CFR 46.101[4])? Is prior approval for delayed onset human subjects research required for TL1 awardees? Why do awardees need prior approval for pilot studies and KL2 projects involving human subjects? Consistent with the HHS regulations (45 CFR 46.120) and NIH policies on human subjects protections, NCATS awardees must seek approval from NCATS to conduct research involving human subjects that was not described in the original, peer-reviewed grant application. To ensure appropriate protections for human subjects in activities proposed under the pilot award or KL2-scholar programs, awardees proposing human subjects research activities that were not part of the original peer-reviewed application must follow NCATS procedures for approval of new protocols. For more information, please refer to NOT-OD-15-129: Prior NIH Approval of Human Subjects Research in Active Awards Initially Submitted without Definitive Plans for Human Subjects Involvement. Which studies require prior approval? Prior approval is required for all pilot studies and KL2-scholar projects involving human subjects supported by NCATS, including full funding support, partial funding support or voluntary committed cost share. How do I submit a prior approval request? Human Subjects Research Prior Approval requests must be submitted via the eRA Human Subjects System (HSS). What information and documents should be included in a prior approval request? Please see NCATS Human Subjects Research Prior Approval (HSRP) Requests. Is IRB approval always required for NCATS prior approval? Yes. Who should submit prior approval request documents and to whom? The Institutional Signing Official (SO) should submit prior approval requests via the eRA Human Subjects System (HSS), including the completed NCATS Human Subjects Research Prior Approval Addendum. Do research studies involving human cell lines or tissue repositories need prior approval? Research with human cell lines or tissue repositories that meet the definition of human subjects research requires prior approval. The NIH Glossary defines human subjects as “A living individual about whom an investigator (whether professional or student) conducting research obtains data through intervention or interaction with the individual or obtains identifiable private information. Regulations governing the use of human subjects in research extend to use of human organs, tissues and body fluids from identifiable living individuals and to graphic, written or recorded information derived from such individuals.” If an investigator has not received a response to a prior approval request, may the investigator proceed with the pilot study or K-scholar project? No. The investigator must wait for NCATS Office of Grants Management (OGM) staff to provide a decision in writing to the grantee institution’s SO. In general, how long does the prior approval process take? Generally, NCATS will respond within 30 days of receiving a complete request. Please note that if a request is returned for any reason, the 30-day turnaround time resets. How will investigators be notified of decisions regarding prior approval requests? NCATS OGM staff will email the Institutional SO with a copy to the CTSA Program PI, to notify the grantee institution of prior approval request decisions. How can an investigator request additional information about the prior approval process and/or requirements? The investigator may contact NCATS via the following email address: NCATSDOPAinquiry@mail.nih.gov. What can investigators do to maximize the efficiency of the review process? It is NCATS’ goal to complete the prior approval review process efficiently, so it is important for investigators to submit all necessary documents and to promptly respond to inquiries. When NCATS requests clarification on a prior approval request, should the institution resend only the questioned/corrected documents? Yes. Only the pertinent documents or attachments should be submitted in response to the request via the eRA HSS module. Is prior approval required for studies that have been determined by the IRB to fall under Human Subjects Research Exemption 4 (45 CFR 46.101[4])? For human subjects research studies that meet the criteria for Exemption 4 under 45 CFR part 46.101(b), the investigator must submit the request via the eRA HSS with the Exemption 4 determination. The completed NCATS Human Subjects Research Prior Approval Addendum, as an attachment in HSS Section 5.1, is required. Is prior approval for delayed onset human subjects research required for TL1 awardees? Most TL1 awardees are working on their mentors’ funded studies, and prior approval is not required. If a postdoctoral trainee is funded directly through the NCATS CTSA Program hub to conduct an independent research study involving human subjects, please notify NCATS via the following email address: NCATSDOPAinquiry@mail.nih.gov. | Prior Approval of Delayed Onset Research Involving Human Subjects FAQs | Prior Approval of Delayed Onset Research Involving Human Subjects FAQs | ||||||
| 8667 | Michael G. Kurilla Joins NCATS as Clinical Innovation Director | Michael Gregory Kurilla, M.D., Ph.D., will direct the NCATS Division of Clinical Innovation beginning Dec. 10, 2017. Kurilla’s new role will include leading the Clinical and Translational Science Awards (CTSA) Program, which supports innovative solutions to advance the efficiency, quality and impact of translational science with the ultimate goal of getting more treatments to more patients more quickly. As the largest NIH initiative, the CTSA Program supports a national network of medical research institutions that work together to tackle system-wide scientific and operational problems in clinical and translational research that no one team can overcome. Kurilla currently serves as director of the Office of BioDefense, Research Resources and Translational Research at NIH’s National Institute of Allergy and Infectious Diseases (NIAID), where he also has been involved with biodefense product development. Prior to joining NIAID in 2003, Kurilla was an associate director for infectious diseases at Wyeth. He also worked at Dupont in antimicrobials, and on molecular pathology at the University of Virginia Health Sciences Center. “Mike has a wealth of experience in the management of complex and complicated translational science initiatives,” said NCATS Director Christopher P. Austin, M.D. “His record of expertise and accomplishment across the translational sciences — including efforts to combat some of the most concerning scourges of our time such as Ebola and Zika virus — makes him ideally suited to lead NCATS’ clinical innovation efforts.” Kurilla has led several World Health Organization global research efforts and testified before Congress about public health emergencies including biological threats. He is an esteemed author of numerous scientific publications on infectious diseases and has received many prestigious awards — including a Department of Health and Human Services Secretary’s Award for Distinguished Service — for his expertise and research advances. In addition, he previously served on the CTSA Program Steering Committee. Kurilla holds a Ph.D. in microbiology and immunology as well as an M.D. from Duke University, was a postdoctoral research fellow at Harvard Medical School, and completed a residency in pathology at Brigham and Women’s Hospital. His B.S. degree is from the California Institute of Technology. “The NCATS CTSA Program encapsulates the overall mission of NIH, taking fundamental scientific biomedical discovery and translating that knowledge into better health and health care for all Americans as well as the world,” Kurilla said. “The opportunity is challenging but also humbling, and I look forward to working with all elements of the program to advance its goals.” Posted November 2017 | Michael Gregory Kurilla, M.D., Ph.D., will direct the NCATS Division of Clinical Innovation beginning Dec. 10, 2017. | /sites/default/files/new-dci-kurilla_1260x630.jpg | Michael G. Kurilla Joins NCATS as Clinical Innovation Director | Michael Gregory Kurilla, M.D., Ph.D., will direct the NCATS Division of Clinical Innovation beginning Dec. 10, 2017. | /sites/default/files/new-dci-kurilla_1260x630.jpg | Michael G. Kurilla Joins NCATS as Clinical Innovation Director | ||
| 8666 | Time Sensitive Information: For CTSA Program Hub Applications to Meet FOA Due Dates on or after Feb. 2, 2018 | CTSA Program Funding Opportunity Through its new CTSA Program funding opportunity announcement (FOA), PAR-18-464, NCATS is updating the forms and instructions for application submission, and incorporated modified review criteria that align with the changes in application content. On Dec. 7, 2017, NCATS issued a notice related to PAR-18-464. There are no changes to NCATS’ plans for the CTSA Program. New Forms and Instructions NIH regularly updates the form set used for grant applications. As announced in NOT-OD-17-062, all applicants, including CTSA Program applicants, will transition to FORMS-E for due dates on or after Feb. 2, 2018. Notably, the new form set will standardize the way applicants provide information pertinent to human subjects research and clinical trials. The majority of form changes introduced in FORMS-E packages relate to the consolidation of human subjects and inclusion enrollment report information, previously collected across multiple forms, into a new Public Health Service (PHS) Human Subjects and Clinical Trials Information form. This new form also expands clinical trial data collection to ensure the appropriate level of information for review and to improve oversight. This new form also: Consolidates research participant, inclusion enrollment and clinical trial information; Collects information on research participants and clinical trials at the study level; Expands the use of discrete form fields to capture clinical trial information, which will provide the level of detail needed for peer review; Leads applicants through clinical trial information collection requirements; Presents key information to reviewers and agency staff in a consistent format; and Aligns with ClinicalTrials.gov(where possible) and positions NIH for future data exchange with ClinicalTrials.gov. Additional changes relate to incorporation of recent Grants.gov changes to Research & Related (R&R) Budget and Small Business Innovation Research and Small Business Technology Transfer (SBIR/STTR) information forms. The Forms E application guide is available on the How to Apply - Application Guide website. To comply with NIH best practices related to eliminating or reducing redundancy in FOAs, some application instructions that are provided in the SF424 (R&R) Application Guide have been removed from the FOA. The absence of such instructions in the body of the FOA does not reflect any change in the relative importance of the information. Modified Review Criteria As announced in NOT-OD-17-118, applications that include clinical trials will be evaluated using additional scored review criteria questions added to Significance, Investigator(s), Innovation, Approach, and Environment. In addition, a new stand-alone criterion, Study Timeline, has been added. FOAs that do not accept clinical trials but permit clinical trial research experience will include additional scored review criteria questions added to existing criteria as follows: Individual Career Development (K) Awards: Research Plan and Mentor(s), Co-Mentor(s), Consultant(s), Collaborator(s) Training (T) Awards: Preceptors/Mentors | Through its new CTSA Program Funding Opportunity Announcement, NCATS has updated the forms and instructions for application submission. | Time Sensitive Information: For CTSA Program Hub Applications | Through its new CTSA Program Funding Opportunity Announcement, NCATS has updated the forms and instructions for application submission. | Time Sensitive Information: For CTSA Program Hub Applications | ||||
| 8665 | CTSA Program Supports Research on Translating Evidence into Practice | Translational Science Highlight An NCATS-supported program fosters research on the dissemination and clinical implementation of therapies, interventions and policies to ultimately improve human health. Researchers around the country are developing and testing innovative therapies and interventions to help patients. But those efforts, no matter how promising, are not always easily adopted into the clinic. To address this clinical implementation challenge across the translational science spectrum, the Washington University in St. Louis Institute of Clinical and Translational Sciences (ICTS) — a Clinical and Translational Science Awards (CTSA) Program-supported hub — launched the Dissemination and Implementation Research Core (DIRC). In the following Q&A, DIRC Director Enola Proctor, Ph.D., breaks down the science of dissemination and implementation and discusses how the unique services at the DIRC are making a difference. What is implementation and dissemination research? Dissemination research is the study of how we can effectively convey evidence and information. Implementation research addresses the challenge of how we can move effective programs, guidelines, interventions and policies into real-world settings so that their potential health benefit for patients and the public at large can be realized. Enola Proctor, Ph.D., Shanti K. Khindka Distinguished Professor at the Brown School at Washington University in St. Louis. Can you provide examples of what researchers in these fields study? An example of dissemination research might be the study of how we can best explain the benefits of a new vaccine to the general public or to a targeted group such as parents. For example, would narratives and stories work better than data? In implementation research, we may ask how we can help health care providers change habitual care and turn a new procedure into routine practice. Why do we need dissemination and implementation research? I’ve heard many people say, “If there is a new treatment, why don’t people just deliver it?” If it were that easy, we wouldn’t have such frequent reports of a lower quality of care than we desire. There was a landmark study recently on the quality of health care in America. The authors showed that we are delivering the recommended medical treatment about 40 percent of the time. For some indicators, such as appropriate use of antibiotics, we’re stuck at 50 percent. There are many areas where real-world practice lags behind the evidence for the most appropriate care. That sounds pretty daunting. Is there an example of a DIRC project that resulted in an improved clinical practice? One example was a project with colleagues at St. Louis Children’s Hospital. We know that babies and small children in intensive care units often become anemic because too much blood is drawn for tests. And yet there are safe and effective guidelines for minimal blood draw. So we asked, “How do we change practice to reduce the rates of blood overdraw?” We were successful by using a multipronged approach that included provider training, using smaller vials to draw blood, and changing habits, such as bundling several orders for blood measurements so they can be accomplished with one draw. How did DIRC come about, and what kinds of services do you provide? The DIRC started 10 years ago with CTSA Program pilot funding. Implementation research is a newer field that requires a distinct methodology and set of research tools. Our CTSA Program hub recognized this and established the DIRC as a research methods core. Our focus is on providing guidance and consultation to investigators affiliated with our hub who are preparing to conduct dissemination or implementation research. You can read more about it in a recent paper we published. For example, we meet one-on-one with investigators to discuss the research design, measures and overarching framework for their studies. Our team, which includes doctoral students and postdocs, helps investigators find literature and funding announcements. And we review and provide critical feedback for grant proposals. We have also developed toolkits, such as how to write aims for this research field, measure implementation outcomes and choose what implementation strategies to test. What kind of impact have these services had on the CTSA Program hub and university research community? We’ve grown in more than a decade from just a handful of people involved in this field at the university to 60 to 70 people who now are doing some kind of implementation science through actively funded grants. And our demand is steadily increasing. We’ve really built our core of implementation researchers to become a solid part of the CTSA Program hub. The DIRC is building the community and also strengthening their grant proposals so they can secure funding to conduct this important research, with the ultimate goal of improving health care. Implementation science is really a team science, so we have worked to forge collaborations. We have regular meetings of our implementation science community where people come together from all parts of our campus that are involved in our CTSA Program hub. That’s been really rewarding because we’ve seen terrific collaborations form and contribute to our success in developing rigorous proposals. It’s a beautiful fit with the CTSA Program goals, and I’m thrilled that the response has been so positive. You have championed the idea of “design for dissemination.” Can you explain what that means? Our DIRC co-director Ross Brownson, Ph.D., pioneered this concept. There is a gap between the “care that could be” and the “care that is,” and we know one reason for that is that interventions can be burdensome if they are developed in the lab without an eye to how they will be delivered and used in practice. We can interact with clinical investigators during the process of treatment discovery and development. For example, a really important question for people who are developing treatments in the lab could be, “How would this particular drug be delivered to infants?” Asking those kinds of questions early on may mean that our treatment discoveries won’t be so cumbersome and will be more readily adopted. Given the DIRC’s success, do you think more CTSA Program hubs will adopt a similar approach? I think this is a great model for all CTSA Program hubs and beyond. There is an appetite among investigators for this kind of work because it’s high-impact work. It’s also creative, challenging and feasible because NIH is soliciting proposals. We haven’t really completed our translational challenge until each and every CTSA Program hub has also made progress in the later stages of the translational pipeline. Posted December 2017 | CTSA Program-supported researchers explore the dissemination and clinical implementation of therapies, interventions and policies to ultimately improve human health. | /sites/default/files/proctor_1260x630.jpg | CTSA Program Supports Research on Translating Evidence into Practice | CTSA Program-supported researchers explore the dissemination and clinical implementation of therapies, interventions and policies to ultimately improve human health. | /sites/default/files/proctor_1260x630.jpg | CTSA Program Supports Research on Translating Evidence into Practice | ||
| 8481 | 2017 Therapeutic/Indication Pairing Projects | In summer and fall 2017, NCATS issued 10 Therapeutic/Indication Pairing projects. The funded preclinical projects are serving as “use cases” to demonstrate the utility of an independent crowdsourcing effort or a computational algorithm to predict new therapeutic uses of an existing drug or biologic. View the projects below: Computational Repurposing of Chemotherapies for Pulmonary Hypertension Preclinical Evaluation of Vorinostat in Alopecia Areata Preclinical Testing of a Novel Therapeutic for Nonalcoholic Steatohepatitis Repurposing Pyronaridine as a Treatment for Chagas Disease Single-Cell-Driven Drug Repositioning Approaches to Target Inflammation in Atherosclerosis Impact of SAR152954 on Prenatal Alcohol Exposure-Induced Neurobehavioral Deficits An Endoplasmic Reticulum Calcium Stabilizer for the Treatment of Wolfram Syndrome Utilization of Phenotypic Precision Medicine to Identify Optimal Drug Combinations for the Treatment of Hepatocellular Carcinoma Targeting Glucose Metabolism for the Treatment of Hepatocellular Carcinoma Application of a Repurposed FDA Approved Drug as a Local Osteogenic Agent Computational Repurposing of Chemotherapies for Pulmonary Hypertension University of Pittsburgh at Pittsburgh Principal Investigator: Stephen Y. Chan, M.D., Ph.D., FAHA Grant Number: 1-UH2-TR-002073-01 Project investigators will test a new mathematical algorithm to determine if specific combinations of chemotherapies ― drugs that have been tested for treatment of cancer ― are effective in treating pulmonary hypertension (PH). There are few effective medications for PH, a disease that affects the blood vessels in the lungs. If successful, this work could accelerate tests for two chemotherapies for individuals with PH and accelerate the development of new molecular inhibitors. Learn more about this project in the NIH RePORTER. Preclinical Evaluation of Vorinostat in Alopecia Areata Columbia University Health Sciences Principal Investigator: Angela M. Christiano, Ph.D. Grant Number: 1-UH2-TR-002090-01 Alopecia areata (AA) is a common autoimmune form of hair loss that affects approximately six million people in the United States. The project team has performed genetic and gene expression studies in AA and identified a drug candidate to repurpose. They will evaluate the drug for preclinical efficacy in AA. Learn more about this project in the NIH RePORTER. Preclinical Testing of a Novel Therapeutic for Nonalcoholic Steatohepatitis Icahn School of Medicine at Mount Sinai Principal Investigator: Joel Thomas Dudley, Ph.D. Grant Number: 1-UH2-TR-002077-01 Nonalcoholic steatohepatitis, or NASH, is a serious and growing health threat in the United States, affecting nearly 5 percent of the population. The goal of this project is to see if a drug with established safety information can be repurposed as a potential new treatment for NASH. If successful, the work could accelerate efforts to bring new therapies to the clinic for treatment of NASH. Learn more about this project in the NIH RePORTER. Repurposing Pyronaridine as a Treatment for Chagas Disease Collaboration Pharmaceuticals, Inc. Principal Investigators: Sean Ekins, Ph.D., D.Sc., Collaboration Pharmaceuticals, Inc.; Jair Lage de Siqueira Neto, Ph.D., University of California, San Diego; and Ester Cerdeira Sabino, M.D., Ph.D., University of Sao Paulo Grant Number: 1-UH2-TR-002084-01 Approximately 6 to 7 million people in Latin America are infected with the eukaryotic parasite Trypanosoma cruzi, the cause of the deadly Chagas disease. Investigators on this project identified an antimalarial drug using computational Bayesian repurposing methods. The project team will test for the minimum effective dose to treat the acute model of this disease, while assessing the potential for combination therapy. Learn more about this project in the NIH RePORTER. Single-Cell-Driven Drug Repositioning Approaches to Target Inflammation in Atherosclerosis Icahn School of Medicine at Mount Sinai Principal Investigator: Chiara Giannarelli, M.D., Ph.D. Grant Number: 1-UH2-TR-002067-01 Atherosclerotic cardiovascular disease (CVD) is the leading cause of mortality and disability worldwide. Even in patients treated with the best standard-of-care programs, residual illness and death remain high. New strategies that directly target atherosclerosis—the main cause of CVD—are needed. This project may provide proof-of-concept evidence for the use of network-driven computational approaches to identify new therapies for atherosclerosis. Using quantitative non-invasive imaging as a translational platform for measuring drug efficacy in a preclinical setting could facilitate a smooth and fast translation of new therapeutics into clinical trials. Learn more about this project in the NIH RePORTER. Impact of SAR152954 on Prenatal Alcohol Exposure-Induced Neurobehavioral Deficits University of New Mexico Health Sciences Center Principal Investigator: Daniel D. Savage, Ph.D. Grant Number: 1-UH2-TR-002082-01 At least 2 to 4 percent of children born in the United States each year are at risk for having prenatal alcohol-associated brain damage, which can cause cognitive disabilities. This research project team will examine the effectiveness of a novel class of drugs for improving neurophysiologic and learning deficits in a well-established animal model of Fetal Alcohol Spectrum Disorder. Learn more about this project in the NIH RePORTER. An Endoplasmic Reticulum Calcium Stabilizer for the Treatment of Wolfram Syndrome Washington University in St. Louis Principal Investigator: Fumihiko Urano, M.D., Ph.D. Grant Number: 1-UH2-TR-002065-01 Endoplasmic reticulum (ER) is an emerging target for human chronic diseases, especially in diabetes and neurodegeneration. Wolfram syndrome is a rare autosomal recessive genetic disorder characterized by juvenile-onset diabetes and neurodegeneration; it is considered a prototype of ER disorder. This project may lead to a breakthrough for treatments of such common diseases as diabetes, neurodegeneration and blindness. Learn more about this project in the NIH RePORTER. Utilization of Phenotypic Precision Medicine to Identify Optimal Drug Combinations for the Treatment of Hepatocellular Carcinoma University of Florida Principal Investigators: Ali Zarrinpar, M.D., Ph.D., University of Florida; and Dean Ho, M.S., Ph.D., University of California, Los Angeles Grant Number: 1-UH2-TR-002087-01 This project applies a computational platform (Phenotypic Precision Medicine) to optimize combination therapy for the treatment of liver cancer. Project researchers will demonstrate the ability to devise novel combination therapies and then plan a clinical trial to test the feasibility and efficacy of this approach to treat liver cancer. Learn more about this project in the NIH RePORTER. Targeting Glucose Metabolism for the Treatment of Hepatocellular Carcinoma University of California, San Francisco Principal Investigator: Bin Chen, Ph.D. Grant Number: 1-R21-TR-001743-01 Hepatocellular carcinoma is the third leading cause of cancer-related death globally, and effective treatments are limited. This project will use the recent public molecular datasets to predict drugs for hepatocellular carcinoma and evaluate them using thorough preclinical models. Learn more about this project in the NIH RePORTER. Application of a Repurposed FDA Approved Drug as a Local Osteogenic Agent Emory University Principal Investigators: Nick J. Willett, Ph.D., and Sreedhara Sangadala, Ph.D. Grant Number: 1-R21-TR-001751-01 This project will investigate the potential of a family of Food and Drug Administration-approved small molecules to be repurposed as a therapeutic strategy to induce bone formation for spine fusion. Learn more about this project in the NIH RePORTER. | In the summer and fall of 2017, NCATS issued 10 bench-to-clinic awards. | 2017 Bench-to-Clinic Projects | In the summer and fall of 2017, NCATS issued 10 bench-to-clinic awards. | 2017 Bench-to-Clinic Projects | ||||
| 8478 | NCATS Unveils Patient-Focused Therapy Development Toolkit | Translational Science Highlight NCATS partnered with patient organizations to create a web-based platform of patient-focused rare diseases translational science resources, many of which also are applicable to other diseases. NCATS is committed to engaging patients and their support organizations as essential partners in the therapeutics research and development process. To bolster these efforts, the Center partnered with members of the rare diseases community to create the NCATS Toolkit for Patient-Focused Therapy Development. The Toolkit is a platform of online resources to support patient groups seeking information about the process of therapy development and provides them with the tools they need to advance their research. Leaders of the Sept. 8, 2017, Demonstration and Dissemination meeting (from left to right): Christopher P. Austin, M.D., Anne R. Pariser, M.D., Ronald J. Bartek, Petra Kaufmann, M.D., M.Sc., and Annie Kennedy. On Sept. 8, 2017, more than 230 patients, caregivers and other community members participated in the official launch of the Toolkit on the NIH campus in Bethesda, Maryland, and via videocast. In opening remarks, NCATS Director Christopher P. Austin, M.D., called the Toolkit the “crowning achievement” of NCATS’ commitment to involving patients in everything the Center does. “NCATS recognized there were many effective, patient-focused resources on therapy development available and wanted to support the patient community by combining them into one accessible and user-friendly online resource,” said Petra Kaufmann, M.D., M.Sc., director of the NCATS Office of Rare Diseases Research, as she unveiled the live portal at the meeting. “It’s my sincere hope that our new Toolkit will help accelerate the path from discoveries to better treatments for our patients.” Ronald J. Bartek, who co-founded the Friedreich’s Ataxia Research Alliance (FARA) after his son was diagnosed with the rare neurodegenerative condition, helped lead the Toolkit’s development with Annie Kennedy from Parent Project Muscular Dystrophy (PPMD). For nearly two years, they collaborated for with NCATS staff and a diverse range of other patient groups to identify and evaluate existing resources. The tools range from general guidance to educational videos to key scientific papers. “This Toolkit initiative is for us, by us,” Bartek said. Accelerating Early Discovery One challenge in rare diseases research is the relatively small number of patients with the disease, who are often geographically dispersed. Patient registries help patients and families find one another, bring rare disease communities together and help them to connect with research opportunities. Through registries and natural history studies, researchers can follow a group of people with a specific disorder over time to learn more about a condition. Registries can also involve patients in the clinical studies and therapeutic development process. Megan O’Boyle, who has a child with Phelan-McDermid syndrome (PMS), is the principal investigator for the PMS Data Network. She shared her experiences with launching the PMS International Registry for this rare genetic disorder that causes developmental delays, admitting that she didn’t know what a patient registry was when another parent first called to ask for help in establishing one. That request began a long journey that included contacting more than 30 other rare disease groups for advice and guidance. Today, the PMS registry is helping researchers and clinicians discover important insights about this condition. Megan O’Boyle presents a case study on setting up a patient registry for Phelan-McDermid syndrome. “If I were starting from scratch, frankly, I probably would have hung up on the woman that called me,” O’Boyle admitted. “But today, I would go to this fabulous Toolkit,” she added while highlighting the “Starting a Patient Registry” resources on the Toolkit Discovery page. Preparing for Clinical Trials Another challenge in rare diseases research is that some pharmaceutical companies may be hesitant to take on the risk of developing a potential therapy for a condition that affects a relatively small number of people. NCATS and many patient organizations work to make therapeutics development less risky for industry and help develop the foundational science needed to prepare for clinical trials. Jennifer Farmer, executive director of FARA, described how the alliance was well-poised to help when Reata Pharmaceuticals, Inc., approached them with a promising new therapy for Friedreich’s ataxia in 2014. FARA connected Reata with expert clinicians in its research network and facilitated early discussions. The alliance stayed involved in many aspects of the clinical trials for the therapy, such as determining which outcomes to measure in the trial and conveying why those endpoints were chosen to the U.S. Food and Drug Administration (FDA). The Toolkit’s Preparing for Clinical Trials page has information on many of these activities, including how FARA led the development of an international network of clinical research centers. Speeding Clinical Trials and FDA Review Kenneth Hobby, president of Cure SMA — spinal muscular atrophy — discussed the essential role patients played in getting the first treatment for SMA approved by the FDA in 2016. SMA, which leads to progressive muscle weakness, affects a wide range of patients from infancy to adulthood and leads to a wide range of severity in symptoms. Placebo-controlled clinical trials to evaluate a group of similar patients may, in some cases, be the best way to test whether a drug is effective. But it can be difficult to recruit and keep patients in these kinds of studies, since some of those accepted are randomly assigned to not receive the investigational therapy. Cure SMA’s close-knit community, which emphasizes care and support as well as research, maintained support from parents for placebo-controlled clinical trials that only evaluated a narrow age range. While this was a tremendous sacrifice for some patients and their parents, the resulting strong data helped get the therapy approved more quickly. In turn, Cure SMA worked with the involved pharmaceutical company and the FDA to make sure the therapy was approved for use in all patients with SMA — from infants to adults — using additional data from other studies in the development program. Kenneth Hobby speaks about the clinical trials and regulatory review process for a new treatment for spinal muscular atrophy. Information about participating in FDA meetings and recruiting participants for trials can be found on the Toolkit Clinical Trials and FDA Review page. Improving Access and Affordability after FDA Approval Although FDA approval of a treatment is often seen as the end goal, Kennedy emphasized that there is still a lot that patient groups can do once a therapy is on the market. She discussed PPMD’s efforts to ensure patients with Duchenne muscular dystrophy have access to and can afford the first two recently approved therapies. For example, PPMD used data from its registry to conduct a burden-of-disease study. This type of study establishes how much it costs to live with a condition over a lifetime, including lost wages for caregivers and costs to modify a home environment. PPMD used these cost estimates when discussing with insurance companies how much coverage they will provide for the new treatments. Kennedy said these estimates are especially important to justify the high upfront cost of some treatments, such as gene therapies. The After FDA Approval section of the Toolkit has information on conducting burden-of-disease studies, working with payers for reimbursement, creating therapy and care guidelines, and developing programs to speed diagnosis. Strengthening the Patient Community “Do not get overwhelmed: Get started!” was a major theme throughout the meeting. Participants agreed the Toolkit will help make taking those first steps easier and will energize them to make further progress. That was certainly the case for Beth McGinn, whose daughter has a rare leukodystrophy commonly referred to as LBSL — leukoencephalopathy with brain stem and spinal cord involvement and lactate elevation. “The Toolkit is exactly what I need,” said McGinn, whose foundation, A Cure for Ellie, is working with other families, researchers and clinicians to develop a mouse model that will be used to test drug therapies already being investigated in similar conditions. “We are nearing a stage where we will need to form a registry, so to have all that information in one place is super useful.” Partnering with others in the community was another lesson of the day. For example, rare disease organizations with extremely small numbers of patients can partner with groups with similar conditions to pool their resources to make progress. The Toolkit can facilitate these connections and enable patient groups to learn from others’ successes — as well as missteps — to work faster and more cost-effectively. Sarah Hogate Bacon asks panel members a question at the Toolkit Demonstration and Dissemination meeting. “You have this cycle where newly diagnosed patients or their families reinvent the wheel over and over again,” said Sarah Hogate Bacon, a writer and patient advocate living with lymphangioleiomyomatosis. “To have a roadmap of all the steps that they can turn to is a real salvation.” The Toolkit: An Evolving Site NCATS and the patient community emphasized that the work was not over, and there was significant discussion about how the Toolkit could be improved or expanded. Attendees had plenty of suggestions for “Toolkit version 2.0,” such as adding information on repurposing existing drugs and how to ensure clinical trials include diverse populations. Still, suggestions for evolving the site did not detract from the consensus that the Toolkit initiative marked an important milestone for patient-focused research. Matthew Toth, Ph.D., from the Barth Syndrome Foundation, summarized it well: “I’ve never seen anything like this before, and I think it has changed the way we look at how drugs are discovered, how they are going to be used and how they are approved in the future.” Posted October 2017 | NCATS showcases web-based platform of patient-focused rare diseases resources at NIH event on Sept. 8, 2017. | /sites/default/files/toolkit-900_0.jpg | NCATS Unveils Patient-Focused Therapy Development Toolkit | NCATS showcases web-based platform of patient-focused rare diseases resources at NIH event on Sept. 8, 2017. | /sites/default/files/toolkit-900_1.jpg | NCATS Unveils Patient-Focused Therapy Development Toolkit | ||
| 8477 | CTSA Program-Supported Researchers Test Low-Cost Treatment for Deadly Illness | Alpha “Berry” Fowler III, M.D. Virginia Commonwealth University. Virginia Commonwealth University (VCU) researchers have discovered a low-cost and potentially lifesaving treatment for sepsis, a life-threatening condition in which the body’s immune system produces an extreme response to an infection. Support from NCATS’ Clinical and Translational Science Awards (CTSA) Program helped streamline translation of this promising therapy to multisite clinical studies. The VCU researchers demonstrated in preclinical studies that high-dose vitamin C improved survival after sepsis in mice. Vitamin C treatment also reduced inflammation and led to better lung function. The results from a small Phase I study in patients were also positive, but larger studies were necessary to test the effectiveness of high doses of intravenous vitamin C. Alpha (Berry) Fowler III, M.D., professor of internal medicine at the VCU School of Medicine and principal investigator of the study, credits CTSA Program hub resources and tools with helping move the therapy into a large, multicenter Phase II trial. This support included grant writing and biostatistics analysis expertise from the NCATS-funded VCU Wright Center for Clinical and Translational Research. The sepsis trial will conclude in early 2018. If the treatment is successful, it could save countless lives around the world. Posted October 2017 | VCU researchers have discovered a low-cost and potentially lifesaving treatment for sepsis. Support from the CTSA Program helped streamline translation of this therapy to multisite clinical studies. | /sites/default/files/berry-fowler_900x600_0.jpg | CTSA Program-Supported Researchers Test Low-Cost Treatment for Illness | VCU researchers have discovered a low-cost and potentially lifesaving treatment for sepsis. Support from the CTSA Program helped streamline translation of this therapy to multisite clinical studies. | /sites/default/files/berry-fowler_900x600_1.jpg | CTSA Program-Supported Researchers Test Low-Cost Treatment for Illness | ||
| 8476 | 2016 Rare Disease Day at NIH Photos | The 2016 Rare Disease Day at NIH was held on one of the rarest of days ― February 29 ― in order to raise awareness about these diseases, the people they affect, and research collaborations to address rare diseases challenges. With a slogan of “Patients & Researchers — Partners for Life,” the event brought together hundreds of rare disease patients, advocates, caregivers and researchers to discuss the latest in rare disease research advances. Sponsored by NCATS and the NIH Clinical Center, the event featured powerful presentations, remarks by congressional and NIH leadership, posters and exhibits, tours of the Clinical Center and more. Event partners included the Food and Drug Administration (FDA) Office of Orphan Products Development and the FDA Center for Drug Evaluation and Research’s Rare Diseases Program, the National Organization for Rare Disorders, Genetic Alliance, Global Genes, the EveryLife Foundation for Rare Diseases, and Uplifting Athletes. View some of the photos from the 2016 Rare Disease Day at NIH: Petra Kaufmann, M.D., M.Sc., director of NCATS’ Office of Rare Diseases Research and Division of Clinical Innovation, speaks at the 2016 Rare Disease Day at NIH. (Daniel Soñé Photography) Attendees collect information from the NCATS Office of Rare Diseases Research exhibit booth. (Daniel Soñé Photography) Attendees discuss the importance of rare diseases research. (Daniel Soñé Photography) Exhibitors share rare diseases resources with participants. (Daniel Soñé Photography) Beatrice Bowie, a sickle cell support group facilitator with Adventist Healthcare and an NIH Clinical Center Patient Advisory Group board member, shares what it is like to live with sickle cell disease. (Daniel Soñé Photography) Dianna Campbell, a rare disease patient, is one of many participants who poses questions to speakers. (Daniel Soñé Photography) Patricia Weltin of the Rare Disease United Foundation speaks with NIH Clinical Center Director John I. Gallin, M.D. (Daniel Soñé Photography) Poster presenters discuss research advances with participants during the event. (Daniel Soñé Photography) Sharon Terry, M.A., president and CEO of Genetic Alliance, discusses the importance of patient advocacy. (Daniel Soñé Photography) The Abbott family, whose son, Aidan, has ectodermal dysplasia, stands in front of a painting of him that is part of the “Beyond the Diagnosis Art Exhibit,” an effort led by the Rare Disease United Foundation. (NCATS photo) | 2016 Rare Disease Day at NIH Photos | 2016 Rare Disease Day at NIH Photos |
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