This past January, President Obama announced his intention to launch a Precision Medicine Initiative that would enable clinicians to tailor disease prevention and treatment recommendations to each individual based on their particular genetics, environment and lifestyle.
Christopher P. Austin, M.D.
On Sept. 3, 2015, NCATS Director Christopher P. Austin, M.D., named Petra Kaufmann, M.D., M.Sc., as director of the Center's Office of Rare Diseases Research. She also retains her position as director of the NCATS Division of Clinical Innovation. Read the full announcement.
NCATS has announced it will spearhead the second phase of several NIH ExRNA Communication program projects to test and validate exRNA molecules for their potential as disease biomarkers and treatments. Read the full announcement.
Select investigators in NCATS' Clinical and Translational Science Awards (CTSA) Program have made progress toward creating IRBrely, a national institutional review board (IRB) reliance agreement that builds upon the expertise of existing regional IRB models.
CTSA Program principal investigator Alan Green, M.D., of Dartmouth College, who leads the project along with a team of other CTSA Program investigators, is set to launch a pilot that will test components of IRBrely. The Childhood Arthritis & Rheumatology Research Alliance Registry, led by Laura E. Schanberg, M.D., of Duke University, will be used to test the effectiveness of the IRBrely pilot. Duke will serve as the reviewing IRB, and the Duke Clinical Research Institute will act as the lead study team. The pilot will test forms, workflows and standard operating procedures (SOPs) that were developed in support of IRBrely.
In addition, IRBrely project leadership currently are seeking opportunities to harmonize SOPs with existing networks that use reliance agreements to facilitate single IRB review for multisite clinical studies, such as through NCATS' Rare Diseases Clinical Research Network (RDCRN). Already, the IRBrely group and RDCRN leadership are working on a plan to merge processes and SOPs, and several RDCRN consortia are considering participation. Learn more about IRBrely.
The NIH/NCATS Global Rare Diseases Patient Registry Data Repository/GRDR® program is designed to advance research on rare diseases and, through application of scientific insights gained, to further research on common diseases as well. The goal is to develop a Web-based resource that integrates, secures and stores many different types of de-identified patient information from a wide range of sources — registries, electronic health records, research datasets, biospecimen repositories and more —all in one place.
The ability to retrieve data from such varied sources will enable researchers to conduct comparative and cross-disease analyses. Combining data from many disorders and facilitating its use in state-of-the-art collaborative research ultimately may lead to new understanding of these illnesses as well as the development of new drugs and therapeutics for the millions of patients with rare diseases.
In August 2015, NCATS awarded a supplemental grant to the Harvard Medical School (HMS) Department of Biomedical Informatics (DBMI) to further develop the GRDR. Led by Isaac Kohane, M.D., Ph.D., and Paul Avillach, M.D., Ph.D., the DBMI efforts will build in part on NIH's Big Data to Knowledge and related initiatives.
This NCATS-HMS collaboration will enable work to integrate different patient registries into the GRDR program and provide secure, encrypted access to these data to authorized users. Using this approach, data providers can more easily share their data with the wider research community and ultimately help get more treatments to more patients more quickly. Learn more about NCATS' GRDR program and Harvard's DBMI.
The Clinical and Translational Science Awards (CTSA) Program Workforce Development Domain Task Force (WD DTF) is making progress in developing the educational tools, resources and best practices for an improved translational science workforce. The WD DTF is led by Rebecca Jackson, M.D., Ohio State University, and Richard J. Barohn, M.D., Kansas University Medical Center, along with guidance from NCATS' Joan Davis Nagel, M.D., M.P.H.
WD DTF members decided first to focus on how to develop an online education platform that would enable all members of the translational science workforce to acquire knowledge in the field and allow users to rate and provide feedback on the resources. The group also is looking at ways to sustain the careers of early-stage investigators who are transitioning from mentored research career awards to independence, standardizing training in good clinical practices, and addressing best practices and strategies for training junior faculty in translational science research.
The WD DTF includes representatives from industry and the Food and Drug Administration to ensure that proposed projects address regulatory science issues and that translational science trainees are exposed to all career pathways, including academia, government and industry.
NCATS research associate and Kelly Services employee Srilatha Sakamuru recently received one of five Kelly Scientific Resources Scholarship Awards. The award will help support her pursuit of a doctorate in bioinformatics and computational biology at George Mason University in Fairfax, Virginia. Sakamuru currently works as a biologist for the Toxicology in the 21st Century program. She is interested in applying her degree to develop computational models to predict the potential of chemicals to disrupt biological pathways that may result in toxic responses.
On Sept. 3, 2015, NCATS held a joint meeting of the NCATS Advisory Council and Cures Acceleration Network (CAN) Review Board at the National Institutes of Health in Bethesda, Maryland. The meeting featured presentations by NCATS leaders and several others, followed by open discussions among Advisory Council and CAN Review Board members.
NCATS Director Christopher P. Austin, M.D., announced several staffing updates, including:
- Petra Kaufmann, M.D., M.Sc., as director of the Office of Rare Diseases Research, overseeing NCATS' Rare Diseases Clinical Research Network, the Genetic and Rare Diseases Information Center, and the NIH/NCATS Global Rare Diseases Patient Registry Data Repository/GRDR® program.
- Keith R. Lamirande, M.B.A., as associate director for administration and executive officer.
- Anton Simeonov, Ph.D., as scientific director overseeing the Division of Pre-Clinical Innovation.
Theresa K. Woodruff, Ph.D., director of the Women's Health Research Institute at Northwestern University, described the development of EVATAR™, a model of the female reproductive tract funded through NCATS' Tissue Chip for Drug Screening program.
Next, NCATS staff members presented three concept clearances:
- Christine M. Colvis, Ph.D., drug development partnership programs director, on a drug repurposing program.
- Michelle A. Culp, M.P.H., Office of Clinical Trials Operations and Management director, on the acquisition of a contract to provide scientific and technical support for NCATS.
- Elaine Collier, M.D., senior advisor in the Office of the Director, on providing NCATS support for investigator-initiated research on ethical issues in the biomedical, social and behavioral sciences.
Dorit Zuk, Ph.D., director of NCATS' Office of Policy, Communications and Strategic Alliances, spoke about NCATS' strategic planning process, discussed the timeline and requested feedback from Council members on proposed themes. Earlier in the meeting, NIH Principal Deputy Director Lawrence Tabak, D.D.S., Ph.D., presented on the progress of the NIH Strategic Plan.
Kaufmann, the final speaker of the public portion of the meeting, provided an update on the Clinical and Translational Science Awards (CTSA) Program. Her presentation included updates on innovating in multisite clinical research, integrating electronic health records and research data, and developing metrics to assist with strategic management.
Through the Accelerated Clinical Trial Agreement (ACTA) initiative, working group members supported in part through NCATS' Clinical and Translational Science Awards (CTSA) Program have developed two new standard agreements to expand clinicians' toolboxes: a Contract Research Organization (CRO) ACTA and a CRO Accelerated Confidential Disclosure Agreement. These standardized documents can greatly reduce contracting delays associated with CRO-managed, industry-sponsored, multisite study initiation.
These agreements are intended to shorten the contract negotiation time for industry or federally sponsored multisite studies and are the result of the ACTA initiative, which seeks to reduce clinical trial contracting delays by developing efficient contract models for multisite trials. The documents address issues such as recruitment, record retention, patient safety, the Health Insurance Portability and Accountability Act and confidentiality, patient billing, limit of liability, data use, and intellectual property.
Since the debut of the ACTA initiative in February 2015, 56 sites have expressed interest in using the agreements, and five clinical trials already are using them. Next, the ACTA group will develop a subcontracting template for use in federally sponsored clinical trials. In addition, efforts are underway to partner with others developing multisite clinical trial contracting templates, such as the Patient-Centered Outcomes Research Institute. Learn more about the ACTA initiative.
David J. Eckstein, Ph.D., is a health scientist administrator in the NCATS Office of Rare Diseases Research; Philip John (P.J.) Brooks, Ph.D., is a program director in the NCATS Division of Clinical Innovation. Learn more about Eckstein and Brooks — and many other NCATS staff members — via NCATS' Staff Profiles. Check back often to meet more of our team!
On October 21-22, the National Organization for Rare Disorders will hold its Rare Diseases and Orphan Products Breakthrough Summit in Arlington, Virginia. This event convenes top leaders from the Food and Drug Administration, NIH, patient organizations and the pharmaceutical/biotechnology industry. On October 21, NCATS Director Christopher P. Austin, M.D., will deliver the afternoon keynote address on the viability of drug repurposing with NIH. For more information and to register, visit the online event page.
- Government Funding for Assay Research and High-Throughput Screening • GEN News • Sept. 16, 2015
- Project Aims to Create Somali Educational Materials on Diabetes Control • KSTP • Sept. 9, 2015
- OHSU Team Gets Go-Ahead on Early Alzheimer's Detection Work • Portland Business Journal • Sept. 9, 2015
- Drug for Fungal Infections in Lung Transplant Recipients Increases Risk for Skin Cancer, Even Death • HealthCanal • Sept. 4, 2015
- Doctors Use State-of-the-Art Technology to Help Woman Who Hears Her Body's Internal Sounds • Medical Daily • Sept. 3, 2015
- Abandoning Linear Thinking (subscription required) • BioCentury Innovations • Aug. 27, 2015
- Closer Collaboration among Researchers, Execs Needed • Modern Healthcare • Aug. 22, 2015
NCATS researchers are seeking collaborators in the following areas:
Bridging Interventional Development Gaps (BrIDGs)
Through its BrIDGs program, NCATS assists researchers in advancing promising therapeutic agents through late-stage pre-clinical development toward an Investigational New Drug application and clinical testing. The 2015 project solicitation is coming soon; stay tuned to NCATS for more information.
NCATS Chemical Genomics Center (NCGC)
NCATS' NCGC offers biomedical researchers access to large-scale screening capacity and medicinal chemistry and informatics expertise to develop chemical probe molecules. These resources can help scientists study the functions of genes, cells and biochemical pathways. For inquiries or to obtain NCGC probe molecules, contact Ajit Jadhav. NCATS researchers also seek collaborators for assay development and high-throughput screening, chemistry and chemistry technology, automation, and informatics.
NIH RNA Interference (RNAi) Initiative
Through the NIH RNAi initiative, NCATS provides state-of-the-art, high-throughput RNAi genome-wide screens for humans and mice to NIH intramural researchers. For more information, contact Madhu Lal-Nag, Ph.D.
Pfizer's Centers for Therapeutic Innovation (CTI) for NIH Researchers
NCATS is facilitating Pfizer's CTI program at NIH, which pairs NIH intramural researchers and clinicians with Pfizer resources to pursue scientific and medical advances through joint therapeutic development of biologic compounds. To apply, submit a completed pre-proposal brief to your NIH Institute or Center's technology transfer office by Oct. 16, 2015. Download the pre-proposal brief template (Word - 37KB) and view pre-proposal submission instructions.
Therapeutics for Rare and Neglected Diseases (TRND)
The TRND program provides collaborators with access to significant in-kind resources and expertise to develop new therapeutics for rare and neglected diseases. NCATS soon will be accepting proposals through TRND for collaborative projects that focus on pre-clinical and early clinical development of new drugs. The 2015 project solicitation is coming soon; stay tuned to NCATS for more information.
Toxicology in the 21st Century (Tox21)
The goal of the Tox21 program is to test 10,000 chemicals and evaluate their potential to cause health problems. Any investigator may propose the development of biological assays for high-throughput screening. To suggest an assay, submit a nomination form (PDF - 44KB) to Menghang Xia, Ph.D. Proposed assays must be compatible with the high-throughput screening guidelines described in the assay guidance criteria.