BBI Washington Editor

BETHESDA, Maryland – A little more than a year after launching its Roadmap for Medical Research program, the National Institutes of Health (NIH; Bethesda, Maryland) reports making significant progress toward accelerating the pace of medical discovery. During an October briefing by NIH Director Elias Zerhouni, MD, and other senior NIH staff, the agency outlined this progress since launch of the program in September 2003.

"We know that today's scientific landscape demands new ways of thinking, and we know we need to introduce a new paradigm for the conduct of medical research," Zerhouni said. "That's what the Roadmap is all about – creating a supportive environment for scientists and their ideas to come together in ways we've never seen before."

NIH defines its Roadmap as a series of initiatives designed to transform the country's medical research capabilities and speed the movement of scientific discoveries from the lab to practical applications in medicine.

"It's time to take a breather and look at where we've been and where we're going," Zerhouni said. "We are celebrating an anniversary."

He also admitted to having "a lot of trepidation" when the Roadmap was first proposed. But, he added: "Many people predicted that by the time we finished the planning after three years, we would be ready. They were right, and we have made a lot of progress."

The NIH said that some of the newly funded projects aim to "ramp up" the efficiency of medical research. According to Zerhouni, the scientific community's response to program solicitations during the past year has been robust, yielding many more new grant applications than expected.

He said NIH needed to establish a common framework and intellectual leadership in science beyond the mission of any one NIH institute.

"There are core themes of science that are converging across institutes," Zerhouni explained. "We weren't looking for more programs. We were looking for true transforming events that truly put the NIH on the path where at least a portion of its portfolio would explore new areas of unknown science. This was the driver behind the Roadmap."

One of the criticisms of NIH, he said, is that it has been slow to respond to emerging opportunities. "Different fields of science have become interdependent," noted. "It is important now to do business differently, without impeding quality research, banking on intellectual creativity and freedom to drive innovation."

Zerhouni compared the Roadmap experiment to venture capital for a start-up company. "You don't plan for a breakthrough – that's why it is a breakthrough," he said. "But you can create an atmosphere that fosters innovation and encourage a culture of change and teamwork."

Team science, as Zerhouni explained, is the underlying current of the entire NIH Roadmap effort. New approaches to research call for increased flexibility and innovative modes of scientific collaboration, he said. Offering scientists from different fields equal status as funded investigators on joint projects is one way NIH is attempting to create more productive teamwork. Another method has been to modify NIH grant application instructions to eliminate fiscal disincentives of establishing consortia.

Another new program, the NIH Director's Pioneer Award, provides five years of funding to each of nine researchers, encouraging these scientists "to pursue highly innovative ideas with unprecedented intellectual freedom."

Future initiatives include establishment of regional centers for translational research and specialized nanomedicine.

In FY04, NIH awarded $64 million to projects within the New Pathways to Discovery theme, $27 million to Research Teams of the Future projects and $38 million to projects within the Re-Engineering the Clinical Research Enterprise theme. The Roadmap represents less than 1% of NIH funding, Zerhouni said, "but it is an important 1%."

Highlights of recently funded projects within each theme:

Under the NIH's New Pathways to Discovery theme, scientists focus on new links between chemistry and biology. New programs are looking for ways to yield novel ways to generate and study small molecules, including exquisitely sensitive imaging probes. NIH hopes these resources will help unravel the functions of genes, cells, pathways and whole organisms, advancing scientists' ability to understand disease at its earliest stages. A nationwide consortium of molecular screening centers, the first of which opened this summer on the NIH campus, will share a database of literature and experimental data through PubChem, freely available to scientists in both the public and private sectors.

The National Technology Centers for Networks and Pathways are expected to yield more sensitive tools to study protein dynamics inside healthy and diseased cells. The centers will work together to develop novel technologies in the field of proteomics – the study of the proteins in a cell, tissue or organism.

Under the Research Teams of the Future program, the Training for a New Interdisciplinary Workforce, effort applies a new funding mechanism toward supporting interdisciplinary work at all levels, from undergraduate students through postdoctoral researchers.

The Re-Engineering the Clinical Research Enterprise theme is the most complex and difficult aspect of the Roadmap, according to NIH. Its goal is to accelerate and strengthen the nation's clinical research enterprise, resulting in bringing research results to clinical settings far more quickly than is currently possible.

The newly funded,computer-based Patient-Reported Outcomes Measurement Information System will attempt to document and analyze patient-reported symptoms, enhancing knowledge of a wide range of chronic disorders. Several initiatives within this theme, currently in development, are designed to grow a skilled clinical research workforce. For example, the recently funded Multidisciplinary Clinical Research Career Development program will train health professionals from a broad spectrum of disciplines and specialties in doing clinical research.

The NIH Clinical Research Scholars is a program intended to yield a new group of clinical researchers, trained in conducting clinical research in multidisciplinary, collaborative settings.

NIH also is funding 21 awards for exploratory centers for interdisciplinary research that will focus on obesity, insect-borne diseases, diabetes, vaccines, stroke rehabilitation and other health-related issues. These centers are designed to lower barriers to research and solve persistent biomedical problems.

Additionally, NIH said it is working to identify clinical research best practices to expand the scope of research activities, increase participation and facilitate research collaboration and information sharing in clinical settings.

Twelve newly funded feasibility studies are examining how to better integrate existing clinical research networks spanning a broad health spectrum, including cardiovascular disease, depression, cancer, tuberculosis, HIV and acute respiratory distress syndrome. Key areas of focus include the use of informatics and technology to streamline and expand clinical research networks.