From the Acting Director
NCATS has made much progress since its establishment late last year. It has launched a number of major research initiatives, participated in innovative partnerships, and established a presence at NIH and throughout the biomedical research community — all while simultaneously standing up a brand new center. I thank our dedicated NCATS team members, who continue to accomplish milestone achievements.
For example, today, NCATS marked its first interagency collaborative initiative by announcing 17 new research projects for its Tissue Chip for Drug Screening program. Funded researchers will create 3-D human tissue chips that accurately model the structure and function of human organs to help better predict drug safety.
Yesterday, NCATS held a technical assistance webinar for several hundred potential applicants who wanted to learn more about applying for Institutional Clinical and Translational Science Awards. These grants provide critical infrastructure and support to academic research institutions to strengthen clinical and translational research.
In June, NIH released funding opportunity announcements for its Therapeutics Discovery initiative, which we featured in this newsletter in May. This collaborative program matches researchers with nearly 60 industry-provided compounds to test ideas for new therapeutic uses for potential drug candidates.
These are just a few of the many stories and updates featured in this issue of the NCATS e-newsletter. Looking ahead, save the date for our first NCATS Advisory Council meeting on September 14, 2012, on the NIH campus in Bethesda, Md. Watch our Events page for videocast information.
Thank you for your continued support and interest in our work to advance translational sciences!
Thomas R. Insel, M.D.
Acting Director
National Center for Advancing Translational Sciences
What's New at NCATS?
NIH Funds Tissue Chip Development to Help Predict Drug Safety
NCATS Announces CTSA Funding Opportunity, Holds Webinar
NIH Announces Funding Opportunities for Speeding Therapeutics Discovery
Senator Moran Tours NCATS Laboratories
University of Miami Joins CTSA Consortium
An Epigenetic Strategy to Kill Cancer Tumors
Collaboration May Help Uncover Treatments for Rare Neurologic Disease
NIH Funds Tissue Chip Development to Help Predict Drug Safety
This month, NIH awarded 17 grants for projects designed to create 3-D chips with living cells and tissues that accurately model the structure and function of human organs, such as the lung, liver and heart. When complete, these chips then will be tested with compounds known to be safe or toxic in people to find the most reliable drug safety signals. Results from this research could help predict the safety of potential drugs in a faster, more cost-effective way.
The grants are part of the NIH’s Tissue Chip for Drug Screening program ― first established by the NIH Common Fund and the National Institute of Neurological Disorders and Stroke, and now led by NCATS. This dynamic initiative is the result of collaborations with NIH, Defense Advanced Research Projects Agency (DARPA), and U.S. Food and Drug Administration. Over the next five years of the program, NIH plans to commit up to $70 million.
"Serious adverse effects and toxicity are major obstacles in the drug development process,” said Thomas R. Insel, M.D., NCATS acting director. “With innovative tools and methodologies, such as those developed by the tissue chip program, we may be able to accelerate the process by which we identify compounds likely to be safe in humans, saving time and money, and ultimately increasing the quality and number of therapies available for patients."
More than 30 percent of promising medications have failed in human clinical trials because they are determined to be toxic despite promising pre-clinical safety and toxicology studies in animal models. Tissue chips are a newer human cell-based approach that potentially can identify safe or toxic therapeutic candidates early in the development process. This may enable scientists to predict more accurately how effective a therapeutic candidate would be in clinical studies.
Visit the NCATS website to learn more about the 2012 project awards and funding for the program.
Wyss Institute for Biologically Inspired Engineering Photo
NCATS Announces CTSA Funding Opportunity, Holds Webinar
On July 6, 2012, NIH released a new funding opportunity announcement (FOA) for Institutional Clinical and Translational Science Awards (CTSAs). The CTSA program provides critical infrastructure and support needed to strengthen the entire spectrum of NIH-supported clinical and translational research. These integrated academic homes for clinical and translational science focus on enhancing the quality, safety, efficiency and cost-effectiveness of translational research as well as training the translational research workforce.
On July 23, 2012, NCATS held a technical assistance webinar for several hundred potential applicants. Materials from this webinar soon will be available on the NCATS website. Key submission dates for this funding announcement are below:
| Letter of Intent Due | December 10, 2012 |
| Application Due | January 8, 2013 |
| NIH Peer Review | February/March 2013 |
| NCATS Advisory Council Review | May 2013 |
| Earliest Award Start | July 1, 2013 |
For more information, read the FAQ and visit the CTSA funding page of the NCATS website. For those who have additional questions after reading the FOA and FAQ, please forward inquiries to CTSAFOAQuestions@mail.nih.gov.
Emory University Photo
NIH Announces Funding Opportunities for Speeding Therapeutics Discovery
In June, NIH released three new funding opportunities designed to help investigators research promising new treatments for patients. The Discovering New Therapeutic Uses for Existing Molecules program, led by NCATS, is an NIH-industry collaboration that matches researchers with nearly 60 compounds to test ideas for new therapeutic uses for potential drug candidates.
On June 12, 2012, NCATS issued a news release to unveil a library of 58 industry-provided therapeutic agents (more than doubling the number of compounds available at the program’s initial launch in May). The eight pharmaceutical companies participating in the program are:
- Abbott
- AstraZeneca
- Bristol-Myers Squibb Company
- Eli Lilly and Company
- GlaxoSmithKline
- Janssen Pharmaceutical Research & Development, L.L.C.
- Pfizer
- Sanofi
By broadly sharing compounds that already have cleared key steps in the development pipeline, the research community can have a head start in contributing their expertise to advance these resources for new indications.
“Each company participating in this innovative collaboration has made substantial research and development investments to advance these molecules to the point where they can be used in clinical studies,” said NCATS Acting Director Thomas R. Insel, M.D. “If researchers funded through this effort can demonstrate new uses for the molecules, it could significantly reduce the amount of time it takes to get a treatment to patients in need.”
NCATS will provide up to $20 million to fund six to eight two- to three-year staged, cooperative agreement research grants for the pilot phase of this program in Fiscal Year 2013. If specific milestones are met, funded researchers will conduct pre-clinical validation and clinical feasibility studies in the first stage, and proof-of-concept clinical trials in the second stage, to test whether one of the molecules may be effective against a previously unexplored disease target. The pilot also is intended to test the utility of the template agreements by reducing the time spent in negotiations.
On June 25, NCATS held a technical assistance webinar for those interested in participating in the therapeutics discovery program. View slides from this event.
Learn more about this program, including therapeutics discovery FAQ and funding.
Photo by Paul R. Kennedy
Senator Moran Tours NCATS Laboratories
Last month, U.S. Sen. Jerry Moran (R-Kan.) and several staff members visited the laboratories and offices of NCATS’ Division of Pre-Clinical Innovation (DPI) in Rockville, Md. NIH Director Francis Collins, M.D., Ph.D., joined the senator — a member of the Senate Appropriations subcommittee that sets NIH’s budget — for the briefing and tour led by DPI Director Christopher P. Austin, M.D. After providing an overview of the center’s role at NIH, Austin provided details on a collaborative research project that is developing new therapeutics for blood cancers. Called The Learning Collaborative, this innovative program joins partners from NCATS’ Therapeutics for Rare & Neglected Diseases (TRND) program, the University of Kansas and the Leukemia & Lymphoma Society, with the goal of targeting novel and repurposed drugs for the treatment of rare blood cancers and finding industry partners to develop them. Leaders from each of these institutions were on hand to share their successes. Sen. Moran was pleased to hear about the investments made by each partner and the project’s rapid progress from lab to clinic. To learn more about this project, see the archived FasterCures webinar on "Collaboration in Action."
Photo by Tom Bush
University of Miami Joins CTSA Consortium
Earlier this month, NCATS announced that the University of Miami received a Clinical and Translational Science Award (CTSA), becoming the newest member of the CTSA consortium. The University of Miami now joins approximately 60 other institutions dedicated to transforming the clinical and translational research process. The award, which is co-funded by NCATS and the National Institute on Minority Health and Health Disparities, will support the Miami Clinical and Translational Science Institute dedicated to making significant contributions in minority health and health disparities and in training underrepresented minorities in clinical and translational research. Learn more about this award.
Photo by John Zillioux
An Epigenetic Strategy to Kill Cancer Tumors
A team of NIH-funded scientists has developed a new method that changes the way genes are regulated to effectively cause cancer tumors to shrink and die in the laboratory. Supported in part by a Penn State University Clinical and Translational Science Institute pilot grant award and funding from the National Cancer Institute, Penn State professors Yanming Wang and Gong Chen recently published research in the Journal of Biological Chemistry suggesting that stopping the action of an enzyme called PAD4 (peptidylarginine deiminase 4) sets off a chain reaction of “molecular switches” in cancer cells. The effect is to switch the internal cell signals from growth to death.
Funded in part by NIH’s Clinical and Translational Science Awards (CTSA) program, this multidisciplinary team is hopeful that their discovery will aid in the development of anti-cancer drugs that only target cancerous tissue without damaging healthy cells and vital organs. Chemotherapy, a standard cancer treatment, damages both healthy and diseased cells. The studied anti-cancer drug has the potential to reduce or eliminate these side effects and improve chemotherapies for cancer patients. Read more about this new strategy.
Collaboration May Help Uncover Treatments for Rare Neurologic Disease
A research collaboration including scientists from NCATS and the University of Wisconsin–Madison, helped identify three promising molecular compounds from a collection of approved drugs to pursue as potential treatments for Charcot-Marie-Tooth disease (CMT), a genetic neurological disease for which there are no current treatments.
The nonprofit CMT Association initiated and supported the university research, and findings were reported on July 20, 2012, in the ACS Chemical Biology journal. The research team screened nearly 3,000 approved and investigational drugs from the NCATS Pharmaceutical Collection in a laboratory test, or assay, for CMT. Identifying an already approved drug that possibly is effective for another disease can have many advantages over developing a medicine from the start, including shortening the time it may take to apply for human clinical trials.
“These findings demonstrate what can be accomplished when a disease foundation and the academic researchers it funds work with the therapeutic development experts at NCATS to translate basic research findings into the first steps of developing a drug,” said Christopher P. Austin, director of the NCATS Division of Pre-Clinical Innovation. “At NCATS, we plan to form more successful collaborations like this one in the future.”
These findings may prove useful in assay development for other rare diseases. Further research is needed and CMT Association already is expanding efforts within this collaboration. Read the full feature.
Upcoming Events
July
CTSA Public-Private Partnerships Webinar Set for July 25
On July 25, 2012, from 2 to 3 p.m. ET, the Clinical and Translational Science Awards (CTSA) program is sponsoring a free webinar highlighting speakers from NIH and Prize4Life to discuss their public-private partnerships. Melanie Leitner from Prize4Life, Inc., and Barbara Mittleman of NIH’s program on public-private partnerships will speak on this topic. To participate, call 1-877-568-4109 and provide the participant code of 224-128-092. Register in advance or visit the Events page for more information.
August
Fifth Annual CTSA Conference Focuses on Evaluating Community Engagement Success
The Duke Translational Medicine Institute is sponsoring a two-day national Clinical and Translational Science Awards (CTSA) conference in August 2012 to design methods, metrics and outcomes useful in evaluating community-engaged research. Supported in part by NCATS, the “5th Annual National CTSA Community Engagement Conference” will be held August 23–24, 2012 at the Bethesda North Marriott Hotel in Bethesda, Md. Stakeholders from multiple organizations, including the Centers for Disease Control and Prevention and the Association of American Medical Colleges, participated in planning presentations.
The conference, which will include community leaders, researchers, practice-based clinicians and those interested in community-engaged research, will showcase:
- Methods and metrics for successful community-engaged research
- How to apply these methods to diverse local settings
- Tools for assessing and improving the effectiveness of community engagement in improving health outcomes
Registration is $175. Posters and abstracts are due July 29, 2012. Participants must register by August 19, 2012. Poster presentations as well as small group sessions also are featured. Visit the conference website for more information.
NIH Offers Translational Science Training Program Boot Camp in August
On August 30–31, 2012, NIH is offering a two-day translational science training “boot camp” designed for NIH postdoctoral fellows and graduate students to learn more about the bench-to-bedside process. The course, which includes a tour of NCATS pre-clinical laboratories and a presentation by NCATS Senior Scientist Sitta Sittampalam, will provide students with insight into the drug development process, enhance understanding of research areas outside of their fields of study, increase opportunities for collaboration and networking, and assist in professional skills development. To participate, students must submit a one-page research abstract that includes a proposal detailing how collaborating with NCATS’ Division of Pre-Clinical Innovation could advance their research project. Visit the NIH Office of Intramural Research's Office of Intramural Training and Education website for more information.
September
NCATS Advisory Council Meeting Planned for September 14
Save the date for the first NCATS Advisory Council meeting planned for September 14, 2012, on the NIH campus in Bethesda, Md. Details are forthcoming and will be available on the NCATS Advisory Council page.
NCATS in the News
NCATS and its programs are in the news frequently. Below are a few examples of recent media coverage:
- A Philadelphia Meeting Talks Treatment for a Rare Disease, The Philadelphia Inquirer, July 15
- NCATS Launches Drug Repurposing Program, Nature Biotechnology, July 10
- NCATS Opens Repurposing Project with 58 Compounds, 17 from Pfizer, BioPharma Today, July 9
- Drug Repurposing Programmes Get Lift Off, Nature, July 1
- U.S. Supreme Court Upholds Obama Health-Care Reform Law, Nature News Blog, June 28
- TRND Navigates the Fragmented Landscape of Rare Diseases, Chemistry & Biology, June 21
- How to Leverage the NCATS Template Across the Industry, Medical Progress Today.com, June 14
- Abandoned Drug Effort Picks Up Steam, Nature News Blog, June 12
- More Big-Name Pharma Firms Join NCATS Therapeutic Repurposing Effort, GenomeWeb Daily News, June 12
- Five More Companies Join NIH's Drug Reuse Program, ScienceInsider, June 12
- Five More Pharmaceutical Companies Join NIH Initiative to Speed Therapeutic Discovery, NIH, June 12
- Rare Disease Drug Development: A Thriving Business Model, Life Science Leader, June 1
Be sure to visit our News & Events page to learn more about these stories and other NCATS programs in the news.