From the Director | What's New at NCATS? | Research Opportunities Volume 01 • Issue 01 • March 21, 2012

Director's Message

Thomas Insel

I'm pleased to share with you the inaugural e-newsletter for the NIH National Center for Advancing Translational Sciences (NCATS). As the Center continues to take shape, this newsletter will provide regular updates to keep our stakeholders informed about our work and new ways we can collaborate to speed scientific progress. We hope you'll take a moment to let us know what you think.

Much has happened since December 23, 2011, when NCATS became the newest center at NIH. Last month, we introduced ourselves to about 1,000 representatives from the academic, pharmaceutical, biotechnology, nonprofit, advocacy and patient communities as part of a Virtual Town Hall. On February 29, we celebrated Rare Disease Day, established to raise awareness about the challenges faced by those affected by rare diseases and the importance of research to develop new diagnostics and treatments. Last week, we announced an innovative partnership to profile the effects of thousands of approved and investigational medicines and make these data publicly available. In addition, we have established charters for the NCATS Advisory Council and the Cures Acceleration Network Board.

Earlier this week, an NIH research team was able to make some surprising discoveries about antioxidants using high-throughput screening systems supported by NCATS. As reported in the Proceedings of the National Academy of Sciences on March 19, the investigators found that some antioxidants can damage DNA and kill cells instead of protecting them. The findings may be useful in developing cancer treatments. And today, we are partnering with FasterCures on a webinar to highlight innovative approaches to disease research.

All of us at NCATS are excited about the opportunities on the horizon to advance translational sciences. Read on to learn more about our progress to date!

Thomas R. Insel, M.D.
Acting Director
National Center for Advancing Translational Sciences

What's New at NCATS?

NIH and Lilly Collaborate to Profile Effects of Approved and Investigational Medicines

NCATS Is Focus of Congressional Hearing

Webinar Highlights Innovative Approaches to Disease Research

NCATS Scientist Named NIH Federal Engineer of the Year

FasterCures Hosts Town Hall with NCATS Leadership

Molecular structure

NIH and Lilly Collaborate to Profile Effects of Approved and Investigational Medicines

NIH and Eli Lilly and Company announced on March 13 that they will generate a publicly available resource to profile the effects of thousands of approved and investigational medicines in a variety of sophisticated disease-relevant testing systems. Comprehensive knowledge of the biological profiles of these molecules may enable biomedical researchers to better predict treatment outcomes, improve drug development, and lead to more specific and effective approaches.

Through the collaboration, NCATS will have its pharmaceutical collection of 3,800 approved and investigational medicines screened using Lilly's state-of-the-art Phenotypic Drug Discovery (PD2) panel. This panel features assays, or tests, that are designed to reveal novel mechanisms or pathways of potential medicines and, as part of this collaboration, approved medicines as well. As such, the panel may provide new insights for drug discovery.

US Capitol

NCATS Is Focus of Congressional Hearing

"Despite phenomenal progress in basic science, we still lack effective treatments for far too many diseases, and this translational pipeline to get there is long, 14 years on the average, and it's leaky," said NIH Director Dr. Francis Collins during congressional testimony on March 20. Both Collins and NCATS Acting Director Dr. Thomas Insel testified on the President's FY 2013 budget request before the U.S. House of Representatives Appropriations Subcommittee on Labor, Health and Human Services, Education, and Related Agencies, chaired by Rep. Denny Rehberg (R-MT).

The hearing focused on both the NIH and NCATS budgets and featured panel discussions about the many opportunities that exist for NCATS to work in synergy with private industry to improve the translation process. Collins also restated NIH's commitment to fund basic biomedical research as its highest priority. Insel showcased NIH's Clinical and Translational Science Awards program, saying "80 percent of [the NCATS] budget is the CTSA program" and that in the next five years, the goal is to increase "engagement of communities, not only as a source of patient volunteers, or research volunteers, but increasingly to get them in at the front end to help define what the research problems need to be, and to bring them in as a full partner." Insel emphasized, too, that fixing the pipeline is "what NCATS is all about … figuring new ways to develop compounds and new ways to develop diagnostics."

With a budget of approximately $575 million, some individuals, including Dr. Roy Vagelos, a former Merck CEO and chair of Regeneron Pharmaceuticals, were skeptical that NCATS would be able to tackle a problem that industry has been unable to solve with nearly $50 billion. However, Todd Sherer, CEO of the Michael J. Fox Foundation for Parkinson's Research had a different perspective: "From the work of our foundation, we have about $50 million of research each year, and we feel we're making a significant impact because there are clear areas that the industry does not focus on." He added that NCATS could help make the "$50 billion that industry is spending more efficient and have a greater chance of actually leading to more therapies … this is exactly the catalytic function NCATS will serve."

Scott Koenig, president and CEO of MacroGenics, Inc., echoed this sentiment and said NCATS could provide "a unique opportunity" by focusing on one of the biggest challenges in the drug development process: understanding how a drug will work in every way, shape or form. "And so, if we're able to reduce this in a laboratory test, to identify something that will give a response that ultimately years down the line will produce a clinical benefit, we are of an ability to shorten that whole process of drug development and get drugs to patients much earlier."

On March 28, 2012, Collins will testify again on the NIH budget before the Senate Appropriations Subcommittee on Labor, Health and Human Services, Education, and Related Agencies led by Sen. Tom Harkin (D-IA). He will be accompanied by Insel; Dr. Harold Varmus, director, National Cancer Institute; Dr. Anthony S. Fauci, director, National Institute of Allergy and Infectious Diseases; Dr. Griffin Rodgers, director, National Institute of Diabetes and Digestive and Kidney Diseases; Dr. Richard Hodes, director, National Institute on Aging. A third hearing before the House for public witnesses is scheduled for March 29, 2012.

Webinar Highlights Innovative Approaches to Disease Research

March 21, 2012, 1:00 p.m. ET

Today, the director of the NCATS Division of Pre-Clinical Innovation will participate in a free webinar designed to showcase innovative approaches to disease research. "Collaboration in Action," sponsored by FasterCures, will highlight an innovative partnership involving the NCATS Chemical Genomics Center, the University of Kansas Cancer Center and the Leukemia & Lymphoma Society. This collaboration, known as The Learning Collaborative, was formed in June 2010 with the goal of targeting repurposed drugs as well as new drugs for the treatment of rare blood cancers. These collaborators have a shared commitment to accelerate drug therapies to human and/or clinical proof-of-concept by identifying and employing industry best practices on high-impact projects. Learn more about this model and hear about opportunities for future collaborations.

Presenters include:

Register for the webinar today. For more information, visit FasterCures.

Sam Michael

NCATS Scientist Named NIH Federal Engineer of the Year

Sam Michael, a scientist within NCATS Division of Pre-Clinical Innovation, was recently named NIH's Federal Engineer of the Year. He received the honor during a ceremony on February 23, 2012, at the National Press Club in Washington, D.C. The award, sponsored by the National Society of Professional Engineers, was in recognition of his leadership of the establishment of a world-class chemical compound screening facility at NIH.

As director of automation and compound management at NCATS, Michael and his team create, operate and maintain a suite of automated high-throughput robotic screening systems. These systems enable the development of hundreds of chemical probes that investigators worldwide use to validate new drug targets and chemical leads for development of new drugs for dozens of currently untreatable diseases. Read the NIH Record article (third story).

FasterCures Hosts Town Hall with NCATS Leadership

On February 27, 2012, approximately 1,000 people joined NCATS Acting Director Dr. Thomas Insel for a virtual town hall meeting about the new NIH Center. Participants from the academic, pharmaceutical, biotechnology, nonprofit, advocacy and patient communities had opportunities to ask questions about NCATS. Hosted and moderated by FasterCures Executive Director Margaret Anderson, the forum focused on the role of the new center and how it would collaborate with these groups to advance translational science.

View the archived webinarpresentation materials and FasterCures' related blog for the full story. Visit FasterCures to learn about future webinars.

Research Opportunities and Announcements

Visit the NCATS Open Opportunities page for a complete list of funding and program announcements.

Coming Soon

In the next few weeks, NCATS will launch a new website. Visit us often at ncats.nih.gov to learn about the Center's latest news and events, innovative research initiatives and programs, and current opportunities for collaboration.

We Want to Hear from You

We welcome your feedback to ensure that we are meeting the needs of all of our stakeholders. Please e-mail us directly at info@ncats.nih.gov, follow us on Facebook and Twitter, view our YouTube channel, and join the NCATS e-mail list for other Center announcements.

For language access assistance, contact the NCATS Public Information Officer.