From the Director | What's New at NCATS? | Research Opportunities Volume 01 • Issue 02 • May 09, 2012

From the Acting Director

Much has happened since our March issue of this e-newsletter. Just last week, NCATS unveiled its Discovering New Therapeutic Uses for Existing Molecules initiative, which will match researchers with more than 20 molecular compounds from Pfizer Inc., AstraZeneca, and Eli Lilly and Company to help scientists explore new treatments for patients. This innovative and collaborative initiative is open to all interested companies and could serve as a model for similar collaborations among government, biomedical research organizations and industry.

NCATS also has launched its brand-new website. Modeled after the NIH website, the new NCATS site features a wealth of information about NCATS-supported research, programs, current funding and collaboration opportunities, and recent translational science news and events.

In addition, NCATS and Eli Lilly developed a manual to help researchers navigate part of the translational science process. This online Assay Guidance Manual now is available as a free e-book from the National Library of Medicine.

In the coming weeks, NCATS will sponsor many informative scientific conferences. Take a look at several we have highlighted below, including one next week on "Natural History Studies of Rare Diseases: Meeting the Needs of Drug Development and Research." Supported in part by NCATS' Office of Rare Diseases Research and the Therapeutics for Rare and Neglected Diseases program, the NIH workshop will serve as a forum for experts in the design, conduct and evaluation of NIH studies to discuss the role of these studies in developing therapeutic candidates.

All of us at NCATS continue to appreciate your support for and interest in our work 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 Launches Industry-Research Partnership to Spur Therapeutic Development

NCATS Launches New Website

NCATS, Eli Lilly Create Assay Guidance Manual

NIH Promotes Workshop on Natural History Studies

Cures Acceleration Network Focus of June Institute of Medicine Event

NCATS Supports Yale Conference on Clinical Research Management

NCATS in the News

HHS Sec. Kathleen Sebelius, NIH Director Francis Collins and patient advocate Michael Manganiello talk with journalists at The National Press Club about the new NIH-NCATS Therapeutic Discoveries initiative. Photo HHS Sec. Kathleen Sebelius, NIH Director Francis Collins and patient advocate Michael Manganiello talk with journalists at The National Press Club about the new NIH-NCATS Therapeutic Discoveries initiative.

NIH Launches Industry-Research Partnership to Spur Therapeutic Development

Last week, Secretary of Health and Human Services Kathleen Sebelius and NIH Director Francis Collins unveiled a collaborative program that will match researchers with a selection of pharmaceutical industry compounds to help scientists explore new treatments for patients. In just four months, NCATS has partnered with Pfizer Inc., AstraZeneca, and Eli Lilly and Company, which have agreed to make more than 20 of their compounds available for this initiative.

"This initiative is an investment not only in our researchers, but also in our nation. When American scientists have the tools and resources to pursue the next great discovery, we all benefit. This makes our nation stronger, healthier and more competitive," Sebelius said during a press conference at The National Press Club in Washington, D.C.

In recent years, researchers have identified the causes of more than 4,500 diseases. But turning such knowledge into new therapies has proven difficult; effective treatments exist for only about 250 of these conditions. Working with industry will give NIH a greater chance of advancing the science necessary to provide new treatments for people who need them.

"Clearly, we need to speed the pace at which we are turning discoveries into better health outcomes," said Collins. "NIH looks forward to working with our partners in industry and academia to tackle an urgent need that is beyond the scope of any one organization or sector."

Through innovative programs, such as Discovering New Therapeutic Uses for Existing Molecules, NIH can address challenges in medicine development.

"Such visionary thinking is imperative if we want to take bold action to speed up drug development," Collins said.

Therapeutic development is a costly, complex and time-consuming process. Some compounds do not prove effective for the specific use for which they were developed. However, with additional research, some of these existing compounds may succeed for a different therapeutic use. Because these compounds already have undergone significant research and safety testing in humans, they are strong starting points for scientists.

By providing selected researchers with unprecedented access to pharmaceutical industry compounds, NIH and its partners aim to stimulate the development of therapies for diseases that lack treatments. To learn more about this innovative program, view a fact sheet and review the current program notice and request for information.

Photo by Ernie Branson

Screenshot of the new NCATS website

NCATS Launches New Website

Last month, NCATS launched its brand-new website. Modeled after the NIH website, the NCATS site features a wealth of information about the research and programs NCATS supports, current NCATS funding and collaboration opportunities, and news and events in translational science. Visitors to the site can explore three broad areas organized around clinical and translational science, rare disease research and therapeutics, and how NCATS is re-engineering the research process. A rotating slide set on the home page, updated regularly, highlights what is new at the Center.

This month, NCATS showcases several of its innovative initiatives and recent collaborations with industry partners. Researchers coming to the site also can view the latest advances in translational science and see a variety of novel tools that support scientists in this complex process.

Be sure to browse through our new site and explore all that it has to offer. As always, we welcome your feedback.

Cover of the Assay Guidance Manual

NCATS, Eli Lilly Create Assay Guidance Manual

On May 2, NCATS and Eli Lilly and Company jointly released an online Assay Guidance Manual designed to provide researchers with step-by-step guidance through the complex process of turning a basic research finding into an assay to start the process of discovering pharmacological tools and drugs. Assays are laboratory tests that enable researchers to examine thousands of compounds using state-of-the-art, high-throughput screening systems critical to drug discovery. Results from assays, known as chemical probes, can help scientists study protein and cell function as well as biological processes relevant to physiology and disease. This knowledge enables scientists to optimize these probes as potential candidates in the drug development pipeline.

The Assay Guidance Manual highlights best practices and features topics such as developing optimal assay reagents, protocols, data standards and performance validation tools. It also provides clear guidelines for scientists in academia, nonprofit, industry and government who want to develop potential assay formats compatible with high-throughput screening and structure activity relationship measurements of new and known molecular compounds.

More than 100 authors from around the world contributed content to this free tool, which is housed by the National Library of Medicine. NCATS plans to continually update and expand the content with contributions by scientists working in various disciplines of translation, drug discovery and drug development. Learn more about this new tool.

Workshop on Natural History Studies of Rare Diseases: Meeting the Needs of Drug Development and Research, May 16-17, 2012, NIH Campus, Bethesda, MD

NIH Promotes Workshop on Natural History Studies

On May 16–17, 2012, NIH and the U.S. Food and Drug Administration will hold a workshop on "Natural History Studies of Rare Diseases: Meeting the Needs of Drug Development and Research" on the NIH campus in Bethesda, Md. Supported in part by NCATS' Office of Rare Diseases Research and Therapeutics for Rare and Neglected Diseases program, the event will serve as a forum for experts in the design, conduct and evaluation of NIH studies to discuss the role of these studies in developing therapeutic candidates.

Natural history (NH) studies are an important tool for understanding the etiology, range of manifestations and progression of rare diseases. Well-conducted NH studies can yield information on biomarkers and other correlates of clinical outcome. Obtaining maximum value to support drug development programs depends on conducting these studies early, often long before potential therapeutic agents are identified for development. Comprehensive, good-quality NH studies designed with an eye toward supporting drug development programs can prevent some of the common problems that lead to stalled, slow or inefficient drug development for rare diseases.

Visit the workshop website to learn more, view the agenda, or register to attend this free event.

Cures Acceleration Network Focus of June Institute of Medicine Event

New collaborative approaches within federal agencies, academia and industry are focusing on the advancement of the drug development enterprise. Among these initiatives is the Cures Acceleration Network. CAN will be the focus of a public workshop by the Institute of Medicine (IOM) on June 4–5, 2012, at the Keck Center in Washington, D.C.

"Maximizing the Goals of the Cures Acceleration Network to Accelerate the Development of New Drugs and Diagnostics: A Workshop" aims to:

CAN was established to advance the development of high-need cures and reduce significant barriers between research discovery and clinical trials. The outcomes from this workshop are intended to help inform and guide NCATS leadership and the CAN Review Board in their efforts to implement CAN and advance translational science.

Visit the IOM website for registration information.

Yale Center for Clinical Innovation

NCATS Supports Yale Conference on Clinical Research Management

On June 4–5, 2012, the Yale Center for Clinical Investigation will host the 5th Annual Clinical Research Management Workshop: "Learning by Doing: Applying Evidence-based Tools to Re-engineer Clinical Research Management" at the Yale School of Medicine in New Haven, Conn. This annual event examines systematic issues that affect clinical research management, sharing both challenges and successes at every stage of process improvement. This year's event will:

The workshop features a keynote presentation; plenary, breakout and poster sessions; and networking opportunities. The registration fee is $60. Visit the event website to view the agenda and register for the workshop.

Photo by Robert Lisak

NCATS in the News

NCATS and its programs are in the news frequently. Below are a few examples of recent media coverage:

Be sure to visit our News & Events page to learn more about these stories and other NCATS programs in the news.

Research Opportunities and Announcements

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

Notice of Intent to Publish a Request for Pre-Applications for the NIH-Industry Pilot Program: Discovering New Therapeutic Uses for Existing Molecules (X02, UH2/UH3) • NOT-TR-12-001

Request for Information: Input on the NIH-Industry Program, Discovering New Therapeutic Uses for Existing Molecules • NOT-TR-12-002

Limited Competition: Revision Applications to Advance Evidence-Based Research Related to Protections for Human Subjects (UL1) • PAS-12-167

Limited Competition: Revision Applications to Advance Evidence-Based Research Related to Protections for Human Subjects (U54) • PAS-12-169

Limited Competition: Revision Applications to Advance Evidence-Based Research Related to Protections for Human Subjects (U24) • PAS-12-165

Limited Competition: Revision Applications to Advance Evidence-Based Research Related to Protections for Human Subjects (U10) • PAS-12-168

Research Supplements to Promote Re-Entry into Biomedical and Behavioral Research Careers (Admin Supp) • PA-12-150

Research Supplements to Promote Diversity in Health-Related Research (Admin Supp) • PA-12-149

We Want to Hear from You

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