Director's Message
On January 30, I attended a White House event where President Obama announced plans for an ambitious new Precision Medicine Initiative. This exciting effort aims to build on recent successes in developing treatments for certain cancers and other conditions, including rare diseases, based on genetic information. The study of rare disorders has frequently led to new approaches to diagnosis, prevention and treatment that are applicable to more common diseases, and I expect that the Precision Medicine Initiative will be no different.
Read more in the latest Director's Message.
Christopher P. Austin, M.D.
What's New at NCATS?
Study Demonstrates Success of NCATS' Rare Diseases Therapeutic Development Programs
CTSA Consortium Tackles Clinical Trial Recruitment Roadblocks
NCATS to Issue CTSA Innovation Funding Opportunities
ExRNA Scientists Search Saliva for Signs of Disease
Grant Funding for Small Businesses: Apply by April 6, 2015
Rare Disease Day at NIH to Be Webcast
CTSA Principal Investigators Convene on Evolving Initiatives
NIH Launches Phase II Trial for Rare Degenerative Muscle Disease Treatment
NCATS Announces New CTSA Steering Committee Members
NCATS Inventions Available for Licensing
Collaborate with NCATS Scientists
Study Demonstrates Success of NCATS' Rare Diseases Therapeutic Development Programs
A study published online in Science Translational Medicine on Feb. 25, 2015, found that two NCATS rare diseases programs have led to the reduced cost of developing new drugs and reduced financial risks. Read the full feature.
CTSA Consortium Tackles Clinical Trial Recruitment Roadblocks
NCATS' Clinical and Translational Science Awards (CTSA) program supports efforts to solve system-wide translational research problems in part by developing and implementing ways to improve the success of U.S. clinical trials. One initiative, CTSA Accrual to Clinical Trials, was launched recently to do just that by developing a nationwide network of sites that share electronic health record data to further multisite study feasibility and recruitment efforts. The initiative builds on existing platforms and operating models to create a "federated" network with common standards, data terminology and shared resources. Read the full feature.
NCATS to Issue CTSA Innovation Funding Opportunities
On Feb. 10, 2015, NCATS announced its intent to publish funding opportunities for Collaborative Innovation Awards through the Clinical and Translational Science Awards (CTSA) program. Read more at the following links:
- NOT-TR-15-005: Notice of Intent to Publish a Funding Opportunity Announcement for Pre-Applications for Collaborative Innovation Award, CTSA Program (X02)
- NOT-TR-15-006: Notice of Intent to Publish a Funding Opportunity Announcement for Collaborative Innovation Award, CTSA Program (U01)
ExRNA Scientists Search Saliva for Signs of Disease
What if clinicians could diagnose and treat a wide range of diseases, including stomach cancer, just by understanding the "conversations" between cells and then eavesdropping on the human body? Extracellular RNA (exRNA) — RNA that is outside the wall of a cell — could enable just that. ExRNA scientists at the University of California, Los Angeles, have found that saliva offers as many clues to a person's health as blood, and they are using this information to develop a noninvasive diagnostic test for stomach cancer. Read the full feature.
Grant Funding for Small Businesses: Apply by April 6, 2015
NCATS has four open funding opportunities through its Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) programs, which support small businesses developing innovative tools, technologies and intervention platforms. Applications are due by April 6, 2015. Find more details about each opportunity in the NIH Guide, or contact the NCATS Office of Strategic Alliances at NCATS-SBIRSTTR@mail.nih.gov.
Omnibus Solicitation
SBIR Direct to Phase II
- PAR-14-088 (R44)
Nucleic Acid Therapeutics
Rare Disease Day at NIH to Be Webcast
Want to participate in Rare Disease Day events at NIH on Feb. 27, 2015, but can't make it in person? Join in via the webcast. Sponsored by NCATS and the NIH Clinical Center, Rare Disease Day is designed to raise awareness about rare diseases, the challenges patients face and the importance of research collaborations. Learn more on the event website. View the full agenda. Watch live on February 27 at http://videocast.nih.gov.
CTSA Principal Investigators Convene on Evolving Initiatives
Leaders and other stakeholders in NCATS' Clinical and Translational Science Awards (CTSA) program met Feb. 3–4, 2015, in Bethesda, Maryland, to discuss and plan for evolving initiatives. NCATS Director Christopher P. Austin, M.D., opened the meeting with an overview of the NCATS mission and the "3Ds" (develop new approaches, technologies, resources and models; demonstrate their usefulness; and disseminate the data, analysis and methodologies to the community). Austin and NCATS Division of Clinical Innovation (DCI) Director Petra Kaufmann, M.D., M.Sc., also emphasized the five NCATS DCI strategic goals:
- Train, develop and cultivate future leaders in translational science.
- Innovate in translational science.
- Communicate effectively with internal and external audiences using clear, timely and consistent messages.
- Measure success of the CTSA program through a set of common metrics.
- Partner effectively with NIH and other stakeholders.
CTSA principal investigators then provided detailed presentations and thoughtful discussion about progress to date on five CTSA program initiatives. These include promoting Good Clinical Practice training for clinical trial personnel, using institutional review board reliance models for multisite studies, improving clinical trial recruitment, developing consensus recommendations for Scientific Review Committees, and reducing clinical trial contracting delays. In addition, Preston Campbell, M.D., of the Cystic Fibrosis Foundation, updated participants on an early example of precision medicine: He explained how a collaborative research model involving patients, industry, academia (including some CTSA hubs), and government helped enable the U.S. Food and Drug Administration's 2012 approval of ivacaftor (Kalydeco), the first drug to treat the underlying cause of cystic fibrosis.
Lead representatives of the five new CTSA Domain Task Forces (DTFs) also shared information. The DTFs are structured to address how to most effectively advance translational science through improvements in collaboration and engagement, informatics, integration of research across the lifespan, methods and processes, and workforce development. Watch for future NCATS news about the progress of these task forces.
NIH Launches Phase II Trial for Rare Degenerative Muscle Disease Treatment
NIH researchers have launched a phase II clinical trial to evaluate the effectiveness of the drug candidate DEX-M74 as a treatment for a rare degenerative muscle disease called GNE myopathy, also known as hereditary inclusion body myopathy. Symptoms of GNE myopathy, which currently has no available therapy, can emerge in adulthood and slowly lead to progressive muscle weakness. Scientists from NCATS and the National Human Genome Research Institute will conduct the clinical trial at the NIH Clinical Center. DEX-M74 was pre-clinically developed by NCATS researchers as part of the Therapeutics for Rare and Neglected Diseases (TRND) program. Read more about this TRND project.
NCATS Announces New CTSA Steering Committee Members
The NCATS Clinical and Translational Science Awards (CTSA) Steering Committee (NCSC) is responsible for providing strategic leadership as well as task force and consortium project oversight for the CTSA program. The NCSC fosters communication between CTSA principal investigators and NCATS, guides the task forces, and provides advice for NCATS to consider in its decision making.
Committee members include NIH and NCATS leaders, CTSA principal investigators, and other stakeholders.
In January 2015, NCATS announced the following new NCSC members:
- Josephine P. Briggs, M.D., National Center for Complementary and Integrative Health
- Carrie Byington, M.D., University of Utah
- Robert Califf, M.D., former director of the Duke Translational Medicine Institute and newly named deputy commissioner for Medical Products and Tobacco, Food and Drug Administration
- Robert H. Carter, M.D., National Institute of Arthritis and Musculoskeletal and Skin Diseases
- Robert A. Clark, M.D., MACP, University of Texas Health Science Center at San Antonio
- James H. Doroshow, M.D., FACP, Division of Cancer Treatment and Diagnosis, National Cancer Institute
- Rebecca D. Jackson, M.D., Ohio State University
- Sundeep Khosla, M.D., Mayo Clinic College of Medicine
- Amy Comstock Rick, J.D., Food and Drug Law Institute
- Harry Shamoon, M.D., Albert Einstein College of Medicine of Yeshiva University
- Julian Solway, M.D., University of Chicago
Other members of the committee are Alan I. Green, M.D., Dartmouth College (new co-chair); Petra Kaufmann, M.D., M.Sc., NCATS (co-chair); Christopher P. Austin, M.D., NCATS; Richard J. Barohn, M.D., University of Kansas; Walter J. Koroshetz, M.D., National Institute of Neurological Disorders and Stroke; Pamela M. McInnes, D.D.S., M.Sc.(Dent.), NCATS; Lee Nadler, M.D., Harvard University; Steven E. Reis, M.D., University of Pittsburgh; Harry P. Selker, M.D., M.S.P.H., Tufts University; and Thomas P. Shanley, M.D., University of Michigan.
NCATS Inventions Available for Licensing
NCATS researchers develop new inventions and technologies that contribute to the improvement of translational research. Recently, NCATS patented a novel method that increases the odds of identifying better drug candidates. This technology, called a coincidence reporter-gene system for high-throughput screening, will be further developed and commercialized by Promega. Read about how this technology is used.
NCATS Office of Strategic Alliances staff are responsible for the creation of unique collaborations and licenses to transfer such inventions to interested parties for further research, development and commercialization. View a list of NCATS patent applications available for licensing.
Upcoming Events
NIH will celebrate Rare Disease Day from 8:30 a.m. to 5:00 p.m. on Feb. 27, 2015. Sponsored by NCATS and the NIH Clinical Center, the event is free and open to the public. Register now, view the draft agenda or join the webcast.
April
On April 16–18, 2015, the Association for Clinical and Translational Science and the American Federation for Medical Research are sponsoring the Translational Science 2015 Annual Meeting to bring together all of the disciplines involved in clinical and translational research, for the shared benefits of networking and education. Presenters include Petra Kaufmann, M.D., M.Sc., director of NCATS' Division of Clinical Innovation, and Robert M. Califf, M.D., newly named deputy commissioner for Medical Products and Tobacco, Food and Drug Administration, and former principal investigator at the Duke Translational Medicine Institute, which is supported by the Clinical and Translational Science Awards program.
International Society for Extracellular Vesicles (ISEV) Annual Meeting
On April 23–26, 2015, ISEV will hold its annual meeting, which will feature cutting-edge research presentations and keynote speakers, including NIH Director Francis S. Collins, M.D., Ph.D., and Xandra O. Breakefield, Ph.D., extracellular vesicle pioneer and Harvard University professor of neurology. ISEV has scheduled a Joint Education Day on April 22 as a prelude to this event.
May
Second EATRIS Conference: Building Bridges in Translational Medicine
On May 27–28, 2015, the EATRIS (European Infrastructure for Translational Medicine) conference will bring together key international stakeholders in translational research, providing a unique opportunity to interact with a wide range of key stakeholders: patient organizations; research funders; and representatives from government, industry and regulatory agencies. On May 28, NCATS Director Christopher P. Austin, M.D., will serve as one of the keynote speakers during Session 4: Global Collaboration in Translational Medicine.
NCATS in the News
- Financing translation: Analysis of the NCATS rare-diseases portfolio • Science Translational Medicine • Feb. 25, 2015
- Study Suggests Alternative Approach to AIDS Vaccine • NIH Director's Blog • Feb. 24, 2015
- Researchers Identify Potential Ebola-Fighting Drugs • Icahn School of Medicine at Mount Sinai • Feb. 18, 2015
- Biodefense Aims to Simulate Human Bodies by Linking Mini "Organs on Chips" • Scientific American • Feb. 17, 2015
- Biodefence Researchers Seek 'Homo Chippiens' • Nature • Feb. 17, 2015
- Go Vegan? It Might Reduce Obese Children's Risk of Heart Disease • Norwalk Reflector • Feb. 12, 2015
- New Screening Tool Could Speed Development of Ovarian Cancer Drugs • Science Newsline • Feb. 10, 2015
- Breaking Dogma? • ASBMB Today • Feb. 6, 2015
- New Imaging Technique Significantly Improves Detection of Invasive Breast Cancers in Dense Breast Tissue • Oncology Nurse Advisor • Feb. 6, 2015
- Among New York Subway's Millions of Riders, a Study Finds Many Mystery Microbes • The New York Times • Feb. 5, 2015
- Obama Proposes 3% More for NIH; Higher User Fees for FDA • Genetic Engineering & Biotechnology News • Feb. 3, 2015
- Futuristic 'Tricorder' Medical Device to Be Tested at UCSD • Times of San Diego • Feb. 3, 2015
- Lazarus Drugs • The Scientist • Feb. 1, 2015
Collaborate with NCATS Scientists
NCATS researchers are seeking collaborators in the following areas:
NCATS Chemical Genomics Center (NCGC)
NCGC offers biomedical researchers access to large-scale screening capacity along with the medicinal chemistry and informatics expertise necessary to develop chemical probe molecules and to study the functions of genes, cells and biochemical pathways. For inquiries or to obtain NCGC probe molecules, contact Ajit Jadhav.
NCGC researchers also seek collaborators for assay development and high-throughput screening, chemistry and chemistry technology, automation, and informatics. Learn more.
NIH RNA Interference (RNAi) Initiative
The NIH RNAi initiative, administered by NCATS, provides state-of-the-art, high-throughput RNAi genome-wide screens for humans and mice. This resource is available only to NIH researchers. Scientists interested in performing high-throughput RNAi screens can contact Madhu Lal-Nag, Ph.D., for more information.
Toxicology in the 21st Century (Tox21) Program
The Tox21 program aims 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 an assay nomination form to Menghang Xia, Ph.D. Proposed assays must be compatible with the high-throughput screening guidelines as described in the assay guidance criteria.