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| 6872 | CTSA Program Support Helps Talented Investigators Re-Establish Their Careers | Anandi Krishnan, Ph.D., is a blood/thrombosis researcher returning to science following an NCATS (2016-18) re-entry award. Krishnan’s current work is in identifying platelet transcriptomic signatures in myeloproliferative neoplasms. Investigators who take a break from research for personal reasons — such as to care for a family member — can have a difficult time returning to academia. To address this challenge and cultivate the best minds to tackle problems in translational science, NCATS funds career re-entry supplements through its Clinical and Translational Science Awards (CTSA) Program. One recipient is Anandi Krishnan, Ph.D., who was completing a successful postdoctoral fellowship before needing time off to care for her newborn child in 2011. One year later, Krishnan was ready to return to work, but despite having a stellar publication record, she was unable to obtain a faculty researcher position. Instead, she accepted an administrative position within Spectrum, the Stanford Center for Clinical and Translational Research, a CTSA Program hub. Still, she missed being a hands-on researcher. When Krishnan learned of NCATS’ career re-entry awards, she collaborated with faculty at Spectrum to successfully apply for a supplement to study myeloproliferative neoplasms (MPNs), a group of rare blood cancers. These diseases carry changes in blood cells’ genetic code that may contribute to complications such as blood clotting. Thanks to the funding, Krishnan is now examining platelets of MPN patients to identify genetic signatures, and she is compiling data to provide a better understanding of the diseases. Her work could ultimately help clinicians detect these diseases earlier and tailor treatments. And how does it feel to be actively engaged in research again? “Spectacular!” Krishnan said. “I am thankful for the support provided through the CTSA Program, and for my outstanding mentors, our dedicated research support staff, collaborators and, above all, patients who offer their samples. It really does take a village to do research.” Posted April 2017 | CTSA Program support helped Anandi Krishnan, Ph.D., get back into a research career after taking a leave of absence to care for her child. | CTSA Program Support Helps Investigators Re-Establish Careers | CTSA Program support helped Anandi Krishnan, Ph.D., get back into a research career after taking a leave of absence to care for her child. | CTSA Program Support Helps Investigators Re-Establish Careers | ||||
| 6739 | Eli Lilly Externship Opportunity in Clinical and Translational Sciences | Program Overview This is an opportunity for an externship in clinical and translational sciences at Eli Lilly and Company for scholars, trainees and investigators who are supported by the NCATS, Clinical and Translational Science Award (CTSA) Program. The externship at Eli Lilly, a large pharmaceutical company, will support the training and education of participants in techniques and principles needed for translational research. Education and training will be team oriented, and in areas of mutual interest for the trainee/scholar and Eli Lilly. Scientific, theoretical and practical knowledge will include an appreciation of translational and commercialization challenges in developing and bringing a biotechnology or pharmaceutical product to patients. The key objective of this NCATS externship program is to enhance a translational scientists’ skill sets and competencies in clinical trial design, clinical pharmacology, toxicology, regulatory affairs and other areas critical to the drug development process. It is expected that at the end of this externship that participants (coming from academia) will understand the work, “language” and culture of another part of the translational ecosystem (industry) and contribute to the growth of an interdisciplinary translational workforce that can promote cooperation and collaboration among academic and industry stakeholders. Externship Experience Watch a video featuring Josephine Taverna, M.D., an assistant professor at the University of Texas Health Science Center at San Antonio, who discusses her externship experience learning to mathematically model Alzheimer’s disease. The externship is an agreement made between Eli Lilly and the participant. The externship is a fully immersive experience and requires a commitment of six months at Eli Lilly’s main headquarters in Indianapolis, Indiana. Each participant will be matched with a mentor and integrated into the scientific project teams at Eli Lilly. Participants will work on intensive, hands-on projects that will expose them to the translational sciences processes and approaches that are relevant to identifying and developing novel diagnostics and therapeutics. It is expected that this intensive experience will help participants understand the mechanisms of a drug’s action, identify strategies in developing compounds, and evaluate PK/PD studies that are used in preclinical and clinical studies as well as the challenges associated with navigating the drug approval process from designing clinical trials to submitting a new Investigational New Drug (IND) application to the FDA. Participants will have the opportunity to interact with each other as a cohort in seminars/workshops to foster cross-fertilization of ideas and promote interdisciplinary learning. If there is interest, there will also be an opportunity for the participant to visit NIH/NCATS at the end. Eligibility This externship opportunity is open to CTSA Program trainees, scholars or investigators supported by CTSA Program hub award* funds who: Have a clinical doctoral degree (M.D. or Ph.D.) or its equivalent in biological or biomedical sciences, bioengineering or a related field; Have a record of accomplishments in clinical and translational science research; Have a keen interest in learning more about drug development and commercialization processes; and Are U.S. citizens or permanent residents of the United States. *Typically, candidates should fit into one of the following three categories: CTSA investigators supported under the UL1 CTSA Program TL1 Trainee, postdoctoral level CTSA Program KL2 scholar (if built in upfront in career development plan) Provisions Successful applicants will be provided with: Projects in the areas of PK/PD and clinical pharmacology as well as opportunities to participate in meetings/seminars (as appropriate), interact with other Lilly staff and also program staff at NIH/NCATS. To gain novel training, applicants are encouraged to select projects that are distinct from their academic research projects at their institutions. Lodging by Eli Lilly in Indiana. Travel costs associated with two roundtrips for participants between their institution and Eli Lilly in Indiana. Alternatively, one of the two roundtrip travels (airfare OR mileage reimbursement if driving) could be to NIH/NCATS to meet with program leadership. Please note that the participants’ stipend or salary must be provided from the CTSA Program Hub award as no new additional funds for salary or stipend are provided with this externship. A letter of commitment from the PI of the CTSA Program hub must accompany an application ensuring the participants’ stipend/salary will be provided for the term of the externship (see Application Procedure). Requirements Selected participants are required to: Agree to and sign a non-disclosure agreement with Eli Lilly. To attend an orientation session at Eli Lilly at the beginning of their experience. Showcase their work at the end of the externship at a research symposium at Eli Lilly. Upon completion of the externship, participants will be required to complete an exit interview with NCATS staff, a post-rotation evaluation and will receive a statement of completion. Application Procedure Discuss your interests with your CTSA Program hub PI and your mentor. Discuss your interests with the NCATS point of contact prior to applying. Apply: Please submit the following application package in PDF format: A cover letter signed by the applicant and the CTSA Program Hub PI (one page) A statement of research interests, your major achievements as a scientist, and a statement as to how this externship will benefit your research program and career development (no more than three pages) Indicate your top two project areas, if you were to be selected Your NIH biosketch Contact information for three references. One of the references must be the PI of the CTSA Program hub. All application materials should be submitted by the Authorized Organization Representative (AOR) via email to ncatselilillyexternship@mail.nih.gov by April 16, 2019, for consideration. Selection Process A review committee comprised of NCATS staff and Eli Lilly mentors and senior staff will review the applications, and a joint meeting will be held to review and select the participants. Announcements will be made by early June with a target start date of July 9, 2019. Please direct all inquiries to: Joan Nagel, M.D., M.P.H., NCATS 301-827-2512 | Eli Lilly Externship Opportunity in Clinical and Translational Science | Eli Lilly Externship Opportunity in Clinical and Translational Science | ||||||
| 6695 | Federal and State Support for Awardees | Small businesses may have access to state and federal support after receiving an NCATS SBIR/STTR award. Following are some current funding and technical assistance resources available from other federal agencies and state organizations. Federal Resources for Small Businesses U.S. Food & Drug Administration: Small Business & Industry Assistance (SBIA) The FDA’s Center for Drug Evaluation and Research (CDER) Small Business & Industry Assistance (SBIA) provides information about: Drug development New Drug Applications/Biologic License Applications Generic drug review Over-the-counter drug review Biosimilars, rare disease and orphan drugs Drug safety Post-marketing activities Compliance and other activities CDER also provides webinars, podcasts, learning tutorials and CDER SBIA Learn, which offers multimedia resources with information for small pharmaceutical businesses. United States Patent and Trademark Office: Inventor and Entrepreneur Resources The United States Patent and Trademark Office provides resources — including webinars — for inventors and entrepreneurs on patents, trademarks, scam prevention, pro bono help and events around the country. Its Inventors Assistance Center provides patent information and services to the public. Centers for Medicare & Medicaid Services: Small Entity Compliance Guides The Centers for Medicare & Medicaid Services (CMS) provides Small Entity Compliance Guides and other information throughout its website, as well as brochures and web-based training classes to educate providers and suppliers. State Resources for Small Businesses Some state-based organizations offer matching grant programs for SBIR and STTR awardees to help them reach the commercialization stage: Florida: The Florida High Tech Corridor Council offers matching grants for Phase II SBIR/STTR awardees. The award sizes range from $10,000 to as high as $150,000 and require the company to provide $3 to every $1 of the council award. Hawaii: The Hawaii SBIR/STTR Matching Grant program offers Hawaii companies up to 50 percent of Phase I and up to $500,000 for Phase II SBIR/STTR awards. Iowa: The Iowa Innovation Corporation offers up to $25,000 for SBIR/STTR Phase I proposals. Kentucky: The Kentucky SBIR/STTR Matching Funds Program provides matching funds up to $150,000 for Phase I and up to $500,000 for Phase II SBIR/STTR awardees. Maine: Maine provides SBIR Phase I and Phase II awardees with matching funds up to $50,000. Massachusetts (life-sciences): MassRamp provides supplemental funding grants in the range of $75,000 to $300,000 to Phase I SBIR/STTR awardees. The funds may be used to support activities not covered by a federal grant, additional funding if federal funding is not sufficient to complete the project, or to expand the scope if it is using the same technology. Applications are due Tuesday, March 28, 2017 at 12:00 p.m. (noon) EDT. Michigan: The Michigan ETF will match Phase I SBIR/STTR awards up to $25,000, and Phase II SBIR/STTR awards up to $125,000. Montana: The Montana SBIR/STTR Matching Funds Program provides supplemental funding for SBIR/STTR awardees, up to $60,000 per application. Nebraska: Nebraska offers Phase I and Phase II matching grants to awardees up to $100,000 or 65 percent of the federal SBIR/STTR grant, whichever is the lower amount. North Carolina: One North Carolina Small Business Program’s Phase I Matching Funds Program provides matching funds for Phase I SBIR/ STTR awardees up to 50 percent of the SBIR/STTR award, not to exceed $65,000. The current submission period ends June 30, 2017. Rhode Island: The Innovate Rhode Island Small Business Fund provides matching grants of up to $45,000 to Phase I SBIR/STTR awardees and up to $100,000 to Phase II SBIR/STTR awardees. South Carolina: The South Carolina Research Authority SBIR/STTR Phase I Matching Grant Program offers matching funds to South Carolina-based Phase I SBIR/STTR awardees up to 100 percent of the federal SBIR/STTR program award amount, not to exceed $50,000. The submission deadlines for FY 2019 are Aug. 8, 2018 and Feb. 7, 2019. Tennessee: The SBIR/STTR Matching Fund program is a strategic initiative to advance the commercialization of innovations across the state. Developed and administered by LaunchTN, the program is funded for FY 2020 at $3 million (no more than $150,000 per program year) through the Tennessee Department of Economic and Community Development. Virginia: The Center for Innovative Technology of the Commonwealth of Virginia has a matching funds program for recent SBIR/r STTR Phase I or II awardees. Applicants may seek up to $50,000 to be used to fill gaps not supported by the federal award. Submission deadline for 2017 was January 19. Wisconsin: The Wisconsin Economic Development Council offers matching grants for Phase I SBIR/STTR awardees up to $75,000 or 50 percent of Phase I SBIR/STTR awards. It also offers Phase II matching grants up to $75,000 per year for up to two years for Phase II SBIR/STTR awardees. Disclaimer: Information may change throughout the year and may not be reflected in this list. Please check the full program descriptions linked above for comprehensive and current information about eligibility and deadlines. | Small businesses may have access to state and federal support after receiving an NCATS SBIR/STTR award. See current funding and technical assistance resources from other federal and state agencies | NCATS SBIR/STTR Federal and State Support for Awardees | Small businesses may have access to state and federal support after receiving an NCATS SBIR/STTR award. See current funding and technical assistance resources from other federal and state agencies | NCATS SBIR/STTR Federal and State Support for Awardees | ||||
| 6627 | From ER Physician to Translational Scientist via CTSA Program Support | Translational Science Highlight A cornerstone of NCATS' CTSA Program is to cultivate the translational science workforce. Many hospital clinicians are interested in pursuing research, but they often do not have sufficient training in this area or the time to devote to such endeavors. But through NCATS’ Clinical and Translational Science Awards (CTSA) Program support, some talented clinicians interested in pursuing research opportunities are finding a way forward. For example, Faheem Guirgis, M.D., an emergency department physician at the Jacksonville campus of the University of Florida (UF), became fascinated with sepsis — an illness resulting from bloodstream infections — during his residency. He was particularly interested in the science underlying the causes of the illness and its physiologic effects. But with little research training, limited time and mentors, and a lack of access to biomedical research facilities, it was difficult for him to make progress. Enter Thomas A. Pearson, M.D., Ph.D., a cardiologist and cardiovascular epidemiologist, UF Health executive vice president for research and education, and the director of UF’s Mentored Career Development Award KL2 training grants. Pearson works from Gainesville at the university’s CTSA Program hub, which is funded by NCATS to cultivate the translational science workforce and speed discoveries to the patient bedside. KL2 awards provide support for early career development of multidisciplinary clinical and translational scientists. University of Florida Assistant Professor of Emergency Medicine Faheem Guirgis and Scientific Research Manager Ricardo Ungaro work with samples in the UF Sepsis and Critical Illness Research Center. “We established the ‘K College’ for early-career investigators to provide training in research design, biostatistics and grant application writing,” Pearson said. “The goal is to connect basic scientists with the clinicians who have the insights into potential applications, and Guirgis was the first recipient of a KL2 award at the UF College of Medicine at Jacksonville.” Pearson connected Guirgis with mentors among the faculty at the UF Sepsis and Critical Illness Research Center in Gainesville, more than 70 miles from Jacksonville, where Guirgis is based. The KL2 grant and resources provided through UF’s CTSA Program training hub enabled Guirgis to take advantage of distance-learning opportunities covering topics such as research methods and laboratory techniques. On the Gainesville campus, Guirgis worked with research mentors who are experts in oxidative stress and inflammation biology. “Once you have authorized and dedicated time to conduct research, you can overcome the distance barrier,” Guirgis said. Thanks to NCATS’ support and the university’s additional NIH research funding for its departments of surgery and aging, Guirgis was able to access samples, generate pilot data and tap into his mentors’ expertise to develop a sophisticated — and successful — application for an NIH Mentored Patient-Oriented Research Career Development award. With that support, Guirgis now has enough time to carry out a research project while continuing to take courses in clinical and translational research design and methods. For his research project, Guirgis is studying the question of why sepsis patients’ cholesterol levels drop precipitously, because those whose blood lipids are extremely low tend to fare very poorly. “With the help of NCATS’ and broader NIH support, I plan to enroll 160 sepsis patients at two university sites to identify lipid-based therapies for sepsis,” he said. In bridging the gap between clinician and clinician-scientist, UF is increasing its research capacity at a satellite campus with a large and underserved population. Creating opportunities for clinicians to gain practical research experience by working with interdisciplinary teams guided by experienced mentors is a key goal of the CTSA Program. This team science approach helps prepare clinician-scientists to better address today’s complex research challenges. “Being involved with research has helped me study the clinical trajectory of sepsis and develop a better clinical sense,” Guirgis said. “What’s more, my training in translational medicine has made me a better physician.” Posted March 2017 | CTSA Program hub (funded by NCATS) at UF helps physician pursue research project in sepsis patients. | From ER Physician to Translational Scientist via CTSA Program Support | CTSA Program hub (funded by NCATS) at UF helps physician pursue research project in sepsis patients. | From ER Physician to Translational Scientist via CTSA Program Support | ||||
| 6614 | New Chapters Added to the Assay Guidance Manual | In March 2017, NCATS’ Assay Guidance Manual (AGM) editors added two new chapters. The AGM is a public resource offering best practices and guidelines for biologically, physiologically and pharmacologically relevant assays for early-stage drug discovery. A diverse editorial team of nearly 30 members from industry, academia and government update the manual regularly; experts from Eli Lilly and Company developed the new chapters, which are as follows: The In Vivo Receptor Occupancy in Rodents by Liquid Chromatography-Mass Spectrometry/Mass Spectrometry chapter, in the In Vivo Assay Guidelines section of the manual, may assist investigators in determining the relationship between in vivo target engagement and pharmacological efficacy or toxicology assessments during the drug discovery process. The Automated Electrophysiology Assays chapter was developed to complement the Ion Channel Screening chapter in the In Vitro Cell Based Assays section. The authors provide guidance for assay development, instrument selection and cell-line choice for electrophysiology assays, along with detailed protocols. The next AGM update is planned for May 2017. | In March 2017, NCATS’ Assay Guidance Manual (AGM) editors added two new chapters. | New Chapters Added to the Assay Guidance Manual | In March 2017, NCATS’ Assay Guidance Manual (AGM) editors added two new chapters. | New Chapters Added to the Assay Guidance Manual | ||||
| 6611 | NIH, Academia and Patient Advocate Collaboration Speeds Niemann-Pick Type C1 Research | Translational Science Highlight Patient-focused translational science collaboration among government and academic scientists, patients, and patients’ advocates advances a potential treatment for the rare disease Niemann-Pick type C1. In early 2007, a group of academic scientists and an advocate for patients suffering with the rare, fatal neurological disease Niemann-Pick type C1 (NPC1) traveled internationally to advocate for NPC1 research support. The experience helped crystallize a growing idea: What if they could work in tandem to help translate the mounting scientific insights into the molecular biology of NPC1 into a singularly focused effort to develop a therapeutic? NIH and several parent-led foundations had supported earlier research leading to the discovery of the gene defect that causes the disease and how it progresses and behaves. Yet there still were no effective therapies, and they thought they could do more. Later that same year, the group helped launch an innovative research collaborative called Support of Accelerated Research for NPC (SOAR-NPC). As described in the Orphanet Journal of Rare Diseases, SOAR-NPC is a network of parents and other patient advocates, academic scientists, and government researchers. The goal was to develop a cocktail of drugs against NPC1 for testing in patients within three to five years. Two parent-led foundations established initial funding for SOAR-NPC, and other foundations followed with additional support. Patient advocates and scientists began meeting regularly to discuss priorities, approaches and progress. The researchers promised to openly share any resulting data among themselves, an approach that has led to an investigational NPC1 drug currently being tested in patients. "Since its advent, SOAR-NPC has represented a different kind of collaborative effort that is therapy-driven and distinguished by participation of patients and advocates on the research team," said NCATS Director Christopher P. Austin, M.D., one of the article's co-authors. "SOAR-NPC has the same team approach to translational science as NCATS, while demonstrating the power of an approach complementary to the usual solely scientist-driven paradigm, combining the knowledge, resources and determination of scientists, physicians, parents and other advocates." NPC1 occurs when a faulty gene fails to remove cholesterol and other lipids from cells. The lipids accumulate in the spleen, liver and brain, impairing movement and leading to slurred speech, seizures and dementia. People with NPC1 typically die in their teens, though a late-onset form of the disease affects young adults. There are two types of NPC disease: NPC1 and Niemann-Pick type C2 (NPC2). While both cause similar effects in patients, NPC1 is much more common. Members of the collaborative Niemann-Pick type C1 project team. (Credit: NCATS) "Developing a new therapy is a lengthy and complicated process that relies on understanding the basic science and mechanism of the disease as the first step," said co-author and patient advocate Jonathan Jacoby, chair of the Hide & Seek Foundation for Lysosomal Disease Research in Long Beach, California. "There was a sense among patient advocates and scientists that the way in which science functions in an academic environment isn't always the best or only way to do science and to advance drug therapies." Sharing ideas among scientists, parents and other advocates and working together are keys to the SOAR-NPC approach. Researchers discuss their unpublished research with each other and patient advocates. Scientists and advocates bring expertise in different fields, from pharmacology and cell biology to engineering and law, all of which can help solve a range of problems. Parents of patients bring a sense of urgency and can offer different insights into living with the disease. In addition, scientists and advocates disseminate NPC1 research information through webinars and meetings and organize events at universities to bring patients, families and researchers together. "Parents wanted to establish a platform to generate ideas and decide together with scientists what research approaches to pursue," said NCATS senior project manager and paper co-author Elizabeth A. Ottinger, Ph.D. "As a result, parents and other advocates are regularly briefed on science, and decisions about priorities and research directions are made through discussions with scientists." NIH's Therapeutics for Rare and Neglected Diseases (TRND) program — now overseen by NCATS — played a pivotal role in SOAR-NPC’s efforts to develop a new NPC1 therapy, helping to move a candidate drug along a translational path to clinical testing. In 2007, when SOAR-NPC was taking shape, there was a turning point in NPC1 research. Two scientists, including SOAR-NPC scientist Steven U. Walkley, D.V.M, Ph.D., independently found evidence that 2-hydroxypropyl-beta-cyclodextrin (HP-β-CD), a modified version of cyclodextrin, was effective against an NPC1 mouse model of the disease. The drug’s development subsequently was chosen as an early TRND pilot project. Through TRND, NCATS provides expertise and drug development resources for rare diseases research by collaborations with NIH, academic scientists, foundations and pharmaceutical companies. TRND staff accepted 2-hydroxypropyl-beta-cyclodextrin as one of the first drug candidates to move through preclinical development and into a clinical trial. For SOAR-NPC scientists and advocates, the TRND support was a game changer. TRND scientists mapped out a game plan for the types of studies required by the U.S. Food and Drug Administration (FDA), identifying the translational gaps in the drug development path. The scientists created collaborative project teams, which included NIH and SOAR-NPC investigators, bringing together expertise in chemistry, pharmacology, toxicology and other fields for drug development, to carry out the work needed to show safety and efficacy in laboratory and animal studies. The TRND-supported preclinical work led to a successful investigational new drug (IND) application to the FDA, a step that is required before studies in people can begin. NPC-SOAR is more than a decade old, and as the NPC1 drug development project has progressed from basic discovery into clinical studies, the team working on NPC translation has also changed. “Such changes are typical of the many-stage ‘relay race’ that is the translational process,” Austin said. New collaborators joined the project team when unanticipated roadblocks were encountered. For example, cyclodextrin looked promising in cells but had difficulty getting into the brain, since it was unable to cross the blood-brain barrier, a network of blood vessels that protects the brain. The NPC team met with the FDA and others, ultimately resulting in an alternate drug delivery route to reach the brain in clinical testing. Co-author Charles H. Vite, D.V.M., Ph.D., a SOAR-NPC researcher at the University of Pennsylvania in Philadelphia, collaborated with TRND scientists on a large animal model that enabled testing of the drug delivery path and its distribution in the brain. The TRND team also worked with co-author and SOAR-NPC researcher Daniel S. Ory, M.D., at Washington University School of Medicine in St. Louis, to develop a laboratory testing system for determining the concentration and effect of the drug in the brain. When potential hearing-associated effects from the drug were identified, collaborators from the National Institute on Deafness and Other Communication Disorders (NIDCD) stepped in to help address the problem. Specialists from NIDCD have monitored and cared for patients in the NPC1 clinical trial, including support through the NIDCD’s audiology unit. NCATS’ role in these types of therapeutic development efforts is to progress a project to the point at which it is “de-risked” — that is, sufficiently developed so that it can be handed off to another organization, typically a biotechnology or pharmaceutical company, with the resources and expertise to complete its development and marketing. In January 2015, Vtesse, Inc., a Gaithersburg, Maryland, biotechnology company, adopted the cyclodextrin-NPC project and moved a specific formulation of the drug, now called VTS-270, into clinical testing. SOAR-NPC scientist and co-author Forbes D. Porter, M.D., Ph.D., at the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), had already begun an early-stage clinical trial of cyclodextrin at the NIH Clinical Center in 2013. Vtesse subsequently began a Phase IIB/III trial in 2015 to study VTS-270 safety and effectiveness. While VTS-270 isn’t a cure, it may delay or reduce neurological symptoms, with the potential to provide a better quality of life for NPC1 patients. Every NCATS TRND project seeks to not only advance the development of a particular therapeutic but also provide translational science insights that can be applied to other projects. SOAR-NPC scientists continue to study NPC1 disease biology and to find new molecules as potential therapeutics that NCATS will consider adopting for development. In addition, NCATS and Vtesse are collaborating in laboratory studies to determine how cyclodextrin works in NPC cells and investigate whether cyclodextrin and a form of vitamin E may have the potential to treat other diseases similar to NPC. Researchers are still in the early stages of studies of a form of vitamin E that also appears to have potential as a treatment for NPC1. "SOAR-NPC started with an idea, a seed that NIH helped grow and turn into a collaborative focused on the drug development process," Ottinger said. In the past few years, SOAR-NPC has ramped up its translational science knowhow and its skills in project management. It is working with a drug development consultant, and its members better understand the complexities of the regulatory and approval process. "Translation is a team sport, and at every step in the quest for an effective NPC1 treatment, multiple patient organizations and many public- and private-sector researchers played essential roles,” Austin said. "Together, we are making advances that offer hope for both NPC1 patients and for those with similar disorders." Posted March 2017 | Patient-focused collaboration among government and academic scientists, patients, and their advocates advances a potential treatment for the rare disease Niemann-Pick type C1. | NIH, Academia and Patient Advocate Collaboration Speeds NPC Research | Patient-focused collaboration among government and academic scientists, patients, and their advocates advances a potential treatment for the rare disease Niemann-Pick type C1. | NIH, Academia and Patient Advocate Collaboration Speeds NPC Research | ||||
| 6573 | CD2H-CC Frequently Asked Questions | Budget and Funding What is the allowable budget that can be requested each year? Application Information Is it the expectation that CD2H-CC will promote the standardization of open-source licenses? Is it the expectation that CD2H-CC will promote the sharing of data-use agreements? One of the research objectives of the CD2H-CC is to promote software development standards for interoperability. Does the CD2H-CC have to create a process for the CTSA Program to come to a consensus on software development standards? Will the CD2H-CC promote the use of a specific type of software development standard? The CD2H-CC is tasked with creating a process for the assessment of merit-based projects. Will the CD2H-CC run a submission and selection process for these merit-based projects? Will this process include the issuance of subcontracts or awards? Will the CD2H-CC be facilitating a process for the development of new standards for software development? One of the research objectives of the CD2H-CC is to stimulate the use of cutting-edge biomedical research informatics and provide data science education for CTSA Program researchers. Are applicants expected to host or store high-quality educational resources and materials? How will the CD2H-CC engage the CTSA Program hubs? Are websites and website URLs allowed in the narrative sections of an application to RFA-TR-17-006? Can PD(s)/PI(s) that are key personnel from more than one CTSA Program hub submit an application? How much effort does each PD(s)/PI(s) have to contribute to the grant? How is CD2H-CC different from the CTSA Program Coordinating Center? How will the CD2H-CC interact with the larger CSTA Program Consortium? For More Information What if I have additional questions? Scientific/Research Contact(s) Peer Review Contact(s) Financial/Grants Management Contact(s) Budget and Funding What is the allowable budget that can be requested each year? The RFA indicates that NCATS intends to commit up to a total of $5 million per year for up to five years for one award. The award budget is limited to $3.5 million per year in direct costs. Consortium F&A costs are NOT included as part of the direct cost base limitation. Application Information Is it the expectation that CD2H-CC will promote the standardization of open-source licenses? Yes. CD2H-CC is expected to design a project that develops a sustainable model for the informatics products produced and used. This may include developing a public-private partnership model that would allow investigators to develop and commercialize their tools and would encourage entrepreneurship such that derivative versions of a product can be used for commercialization. Is it the expectation that CD2H-CC will promote the sharing of data-use agreements? Yes. The CD2H-CC is expected to design a project that provides a plan to promote data re-usability with relevant attributes, data-usage license, and provenance (integrity and validity). Applicants should include a Data-Sharing Plan: Data-Sharing Plan: All applications, regardless of the amount of direct costs requested for any one year, should address a Data-Sharing Plan. Consistent with collaborative nature of the CTSA Program, it is expected that the clinical and research datasets (patient, genotypic, molecular, phenotypic, behavioral, environmental, covariate and other relevant data) will be widely shared with the scientific community for research, as much as possible. At the same time, data privacy is particularly important, and as such, applicants should carefully observe standards of patient privacy, confidentiality and management of health information. Applicants should provide a description of how they will ensure data privacy. One of the research objectives of the CD2H-CC is to promote software development standards for interoperability. Does the CD2H-CC have to create a process for the CTSA Program to come to a consensus on software development standards? The expectation is the CD2H-CC will facilitate an agreed upon software development process that ensures quality, scalability, and the interoperability of the products that will be produced. The goal is to build tools that CTSA Program members will want to use and that those tools are interoperable with each other. Will the CD2H-CC promote the use of a specific type of software development standard? The CD2H-CC goal is to promote a reproducible process for software interoperability that facilitates a successful scalable, usable, professional product. The CD2H-CC is tasked with creating a process for the assessment of merit-based projects. Will the CD2H-CC run a submission and selection process for these merit-based projects? No; The CD2H-CC will not run a submission process. The expectation is that the CD2H-CC will create an open and transparent review process for applications submitted to the CTSA Program Will this process include the issuance of subcontracts or awards? No. The funding of the development of projects is outside the scope of this RFA. Projects will be funded by the CTSA Program Will the CD2H-CC be facilitating a process for the development of new standards for software development? No. The purpose of the CD2H-CC is not to support the invention of new standards or novel technology but the implementation of existing standards, and the dissemination of better processing methods that promote collaboration, and interoperability among existing knowledge sources to facilitate new discoveries. One of the research objectives of the CD2H-CC is to stimulate the use of cutting-edge biomedical research informatics and provide data science education for CTSA Program researchers. Are applicants expected to host or store high-quality educational resources and materials? The RFA does not provide guidance on how to address the research objectives; however, dissemination of high-quality educational resources and materials could be accomplished in several ways. Additionally, the review criteria ask reviewers to evaluate how well these proposed activities will stimulate the use of cutting-edge biomedical research informatics and data science education for CTSA Program researchers. How will the CD2H-CC engage the CTSA Program hubs? The CTSA Program is evolving into an innovative national consortium of academic medical centers – called “hubs” — that work together to support translational science and improve the research process to get more treatments to more patients more quickly. The CD2H-CC will be expected to work closely with the CTSA Program hubs, CTSA Program Coordinating Center (RFA-TR-16-021), Trial Innovation Centers (TICs) (RFA-TR-15-002), Recruitment Innovation Center (RIC) (RFA-TR-15-004), other innovative collaborative programs (for example, PAR-15-172), external partners, stakeholders, NCATS and NIH staff to accomplish the goals described in the funding opportunity. However, the RFA does not provide guidance on how this will happen, but a natural connection would be through the informatics domain task force member that is appointed to represent their CTSA Program hub. Are websites and website URLs allowed in the narrative sections of an application to RFA-TR-17-006? For the U24 activity code, the page limit for the research strategy is 12 pages and one page for the specific aims. Per the PHS SF424 guidance, “Unless otherwise specified in an NIH solicitation, internet website addresses (URLs) may not be used to provide information necessary to the review because reviewers are under no obligation to view the internet sites. Moreover, reviewers are cautioned that they should not directly access an internet site as it could compromise their anonymity.” In addition, the appendix cannot be used to circumvent page limits. The recent notice (NOT-OD-17-035) provides a reminder of the new appendix policy for NIH/AHRQ/NIOSH applications submitted for due dates on or after January 25, 2017. However, screen shots/images of or from websites are allowable within the narrative sections of an application. Can PD(s)/PI(s) that are key personnel from more than one CTSA Program hub submit an application? RFA-TR-16-006 is not a limited competition granting mechanism, meaning an applicant institution is not required to be awarded a CTSA Program hub. Applications involving multiple PD(s)/PI(s) are allowed. For institutions/organizations proposing multiple PDs/PIs, visit the Multiple Program Director/Principal Investigator Policy and submission details in the Senior/Key Person Profile (Expanded) Component of the SF424 (R&R) Application Guide. Only one application per institution (normally identified by having a unique DUNS number or NIH IPF number) is allowed. How much effort does each PD(s)/PI(s) have to contribute to the grant? The PD(s)/PI(s) are each expected to commit at least two person months effort per year and preferably three to six person months per year effort to the award. How is CD2H-CC different from the CTSA Program Coordinating Center? The CD2H-CC has distinct goals apart from the CTSA Program Coordinating Center. It is not expected that the CD2H-CC and the CTSA Program Coordinating Center will have any direct interaction. It is expected that the CD2H-CC will: 1) support and enhance a collaborative informatics community for the CTSA Program; 2) develop Good Data Practice (GDP) for information stewardship; 3) promote software standards for interoperability; 4) foster collaborative innovation in the area of informatics tools, methods and processes; 5) stimulate the use of cutting-edge biomedical research informatics and data science education for CTSA Program researchers; and 6) evaluate the impact of CD2H-CC activities to enhance health through the use of informatics resources. In contrast, the CTSA Program Coordinating Center will be responsible for providing an environment of excellence, developing and applying innovative approaches to collection, analysis, use and sharing of various types of data for strategic management of the CTSA Program, and creating an effective collaboration, coordination and communication infrastructure to support scientific, training, governance, workgroup and other types of consortium activities within the CTSA Program. How will the CD2H-CC interact with the larger CSTA Program Consortium? As described in the Cooperative Agreement Terms and Conditions of the Award, awardee(s) will agree to the governance of the CTSA Program Consortium; present semi-annual reports to the NCATS CTSA Program Steering Committee on the CD2H-CC current and planned activities; and provide and facilitate support for communication among key stakeholders, including the CTSA Program Informatics domain task force and the External Scientific Consultants, to accomplish the activities outlined in Section I of RFA-TR-17-006 For More Information What if I have additional questions? Send your questions via email to the following agency contacts: Scientific/Research Contact(s) Erica K. Rosemond, Ph.D. National Center for Advancing Translational Sciences (NCATS) Telephone: 301-594-8927 Email: rosemonde@mail.nih.gov Peer Review Contact(s) Carol Lambert, Ph.D. Telephone: 301-435-0814 Fax: 301-480-3660 Email: Lambert@mail.nih.gov Financial/Grants Management Contact(s) Dawn E. Walker National Center for Advancing Translational Sciences (NCATS) Telephone: 301-435-0833 Email: dawn.walker@nih.gov Answers in response to emailed questions may be added to this page. | Answers to Frequently Asked Questions for CD2H-CC | CD2H-CC Frequently Asked Questions | Answers to Frequently Asked Questions for CD2H-CC | Frequently Asked Questions About CD2H-CC | ||||
| 6571 | General Program Contacts, Division of Rare Diseases Research Innovation | Contact the staff listed below for more information about the NCATS Division of Rare Diseases Research Innovation. General contact information: (301) 435-0916 or NCATS Division of Rare Diseases Research Innovation. Stay connected with the Division of Rare Diseases Research Innovation on Facebook and Twitter. Area of Expertise Contact Phone Number Clinical Trial Readiness Grants Program Director: Alice Chen Grady, M.D. (301) 827-2015 Genetic and Rare Diseases (GARD) Information Center Program Director: Eric Sid, M.D., M.H.A. Program Administrator: Jim Dickens (301) 827-3073 (301) 827-9507 Genetic Therapies, General Program Director: Philip John (P.J.) Brooks, Ph.D. Program Administrator: Deanna Portero (301) 443-0513 (301) 451-9968 GRDR GUID (Global Unique Identifier) Program Director: Eric Sid, M.D., M.H.A. (301) 827-3073 NCATS Scientific Conference Grants Program Director: Alice Chen Grady, M.D. (301) 827-2015 NCATS Toolkit for Patient-Focused Therapy Development (Toolkit) Program Director: Eric Sid, M.D., M.H.A. Program Administrator: Jim Dickens (301) 827-3073 (301) 827-9507 Non-Viral Technologies for in vivo Delivery of Genome Editors Program Director: Philip John (P.J.) Brooks, Ph.D. (301) 443-0513 Rare Diseases Clinical Research Network (RDCRN) Program Director: Tiina Urv, Ph.D. Program Administrator: Joanne Lumsden, Ph.D. (301) 827-2746 (301) 443-3676 RDCRN Coalition of Patient Advocacy Groups (CPAG) Program Director: Tiina Urv, Ph.D. Program Administrator: Joanne Lumsden, Ph.D. (301) 827-2746 (301) 443-3676 Rare Disease Day at NIH Program Director: Alice Chen Grady, M.D. Program Administrator: Meera Shah, M.P.H. (301) 827-2015 (301) 827-6222 Rare Diseases Registry Program (RaDaR) Program Director: Eric Sid, M.D., M.H.A. Program Administrator: Jim Dickens (301) 827-3073 (301) 827-9507 Somatic Cell Genome Editing Program Program Director: Philip John (P.J.) Brooks, Ph.D. Program Administrator: Deanna Portero (301) 443-0513 (301) 451-9968 Trans-NIH Rare Diseases Working Group Program Director: Tiina Urv, Ph.D. Program Administrator: Meera Shah, M.P.H. (301) 827-2746 (301) 827-6222 All Other Inquiries Program Administrator: Tierra Clarke (301) 435-0916 Visit our rare disease research programs: The Genetic and Rare Diseases (GARD) Information Center provides up-to-date health information about numerous rare and genetic diseases. The Rare Diseases Registry Program (RaDaR), formerly known as the Global Rare Diseases Registry Data Repository program, aims to define best practices for patient registries. RaDaR also strives to identify and adopt standards to support high-quality registries for rare diseases therapeutics development. The Rare Diseases Clinical Research Network (RDCRN) is designed to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing. The NCATS Toolkit for Patient-Focused Therapy Development is a collection of online resources that can help patient groups advance through the process of therapy development. The Clinical Trial Readiness for Rare Diseases, Disorders and Syndromes (CTR) grants program supports projects focused on collecting the data needed to move promising rare disease therapies and diagnostics to clinical trial. The Platform Vector Gene Therapy (PaVe-GT) pilot project will test whether it is possible to increase the efficiency of gene therapy clinical trial startup by using the same gene delivery system and manufacturing methods for multiple gene therapies. | DRDRI Contacts | Program Contacts, Division of Rare Diseases Research Innovation | DRDRI Contacts | Program Contacts, Division of Rare Diseases Research Innovation | ||||
| 6542 | Therapeutic Areas of Interest | For the most recent call for proposals, Pfizer is interested in research projects that fall within the following therapeutic areas: Disease Area Focus Cancer — Investigators should select solid tumors, such as colorectal, breast, lung (small cell and non-small cell), pancreatic and ovarian. Autoimmunity/Inflammation — Areas of interest include irritable bowel syndrome, nonalcoholic fatty liver disease (NAFLD)/nonalcoholic steatohepatitis (NASH), atopic dermatology and psoriasis. Metabolic Diseases — Pfizer is specifically interested in type 2 diabetes mellitus and cachexia. Rare Diseases — Areas of interest include amyotrophic lateral sclerosis (ALS), Huntington’s disease, Duchenne or Becker muscular dystrophy, and non-malignant hematological disorders. Targets/Pathways Focus Immune activators/enhancers (e.g., nucleic acid sensing, toll-like receptors, etc.) DNA damage recognition & repair (e.g., replication stress or repair enzymes) Tissue-resident immune modulation (e.g., adaptive & innate, tumor microenvironment, organ-specific, such as skin or gut) Modulation of tumor cell heterogeneity, senescence and plasticity (including drug resistance) Regulation of epithelial biology (i.e., restoring barrier health/integrity) Modulation of fibrosis pathways [either metabolism/stress-induced or inflammation-induced (possibly tumor-driven)] Regulation of antigen-specific tolerance induction and/or modulation of T regulatory cells Treatments for repeat expansion diseases (including Huntington’s disease, ALS/ frontotemporal dementia and myotonic dystrophy) Interventions to address skeletal and cardiac muscle diseases (including Duchenne or Becker muscular dystrophies) Learn more about each of these therapeutic areas of interest. | Therapeutic areas of interest: Oncology, inflammation and immune disorders, cardiovascular and metabolic disease, neuroscience, rare monogenic diseases. | /sites/default/files/cancer-900px.jpg | Therapeutic Areas of Interest | Therapeutic areas of interest: Oncology, inflammation and immune disorders, cardiovascular and metabolic disease, neuroscience, rare monogenic diseases. | /sites/default/files/cancer-900px.jpg | Therapeutic Areas of Interest | ||
| 6541 | How CTI Works | The CTI collaboration enables NIH and Pfizer scientists to identify biologic compounds with activity in a pathway or target of interest and move these compounds through laboratory testing and into clinical evaluation. NIH intramural researchers selected for CTI projects will have identified disease-related pathways or mechanisms as potential therapeutic targets that culminate in Phase I clinical trials to demonstrate proof-of-mechanism. A cooperative research and development agreement (CRADA) collaboration with CTI includes, among other things, access to Pfizer's drug development expertise and publishing rights. Pfizer has the first right of refusal to exclusively license compounds and other intellectual property developed under the NIH-Pfizer CTI CRADA. However, research rights are not compromised by this collaboration. Should Pfizer decide not to move forward with a project, NIH still owns all the background intellectual property that it brought to the research program, and the investigator may continue his or her research independent of Pfizer’s CTI. A joint NIH-Pfizer steering committee governs the collaboration and is responsible for selecting research projects and making decisions about the progress of those projects. NIH’s intramural scientists can respond to CTI calls for proposals two times each year. Learn more about the current call for proposals and therapeutic areas of interest, or visit our frequently asked questions for more information. | The CTI collaboration will enable scientists to id biologic compounds with activity in a pathway or target of interest and move these compounds through laboratory testing and into clinical evaluation. | /sites/default/files/researcher-at-computer-900px.jpg | How CTI Works | The CTI collaboration will enable scientists to id biologic compounds with activity in a pathway or target of interest and move these compounds through laboratory testing and into clinical evaluation. | /sites/default/files/researcher-at-computer-900px.jpg | How CTI Works |
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