NIH Roadmap for Medical Research – One Year Later
A Briefing by the National Institutes of Health Director
and Senior Staff
Thursday, October 14th, 2004
One year after launching the NIH Roadmap for Medical Research, the National Institutes of Health (NIH) has made significant progress toward accelerating the pace of discovery. Newly funded projects aim to ramp up the efficiency of the medical research enterprise by orders of magnitude. Among these are innovative programs to train clinical researchers and to fund highly creative thinkers, a nationwide interconnected network of biocomputing centers, and projects supporting the development of a diverse array of small molecules and imaging probes freely available to all researchers.
The scientific community’s response to NIH Roadmap solicitations has been robust, yielding many more new grant applications than expected. In establishing the Roadmap, NIH purposefully intended to usher new researchers and new fields into the fold.
“ 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,” said NIH Director Elias A. Zerhouni, M.D. “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. We will continue to assess opportunities and see if we can do business differently. Trying to convince Congress to make it easier for us to collaborate, we banked on intellectual freedom. With the new Pioneer Award program, NIH has provided five years of funding to each of nine exceptionally creative people, encouraging these scientists to pursue their highly innovative ideas with unprecedented intellectual freedom. Tomorrow’s breakthroughs hinge on letting creative minds take chances. Through the Pioneer Award program, NIH is experimenting with how to alter scientific review practices to accommodate very creative projects that may have no proven track record for success, but whose outcome could well change medical practice in dramatic ways. Implementing the Roadmap embraces teamwork. Several aspects must be preserved. We want to be very careful.
Zerhouni went on to say that money for a venture capital might be put at risk. However, given the need for a healthier community of science, the roadmap has gotten a positive reaction from patient groups and members of Congress.
“ There are new pathways to discovery,” explained Zerhouni. “Science is a continuum of ‘bench-to-bedside.’ We need to encourage that continuum.”
As the scientific landscape evolves, researchers must have the ability to accurately measure biological processes in real time, in living samples. Several Roadmap projects within this theme were designed to develop a 21st-century toolkit that will enable scientists to better understand the workings of biological systems. A nationwide consortium of molecular screening centers, the first of which opened this summer on the NIH campus, will share a common database of literature and experimental data through PubChem (http://pubchem.ncbi.nlm.nih.gov), which is freely available to scientists in the public and private sectors. This network will be unprecedented in its broad utility to basic and clinical researchers.
“ Biomedical research has developed extraordinarily powerful data generation methods, such as imaging and DNA sequencing. The wide availability of equally capable analysis toolkits will be crucial to capitalize on this information,” said National Institute of General Medical Sciences Director Jeremy M. Berg, Ph.D. “The capacity to screen molecules is powerful. It involves assay development, robotics and instrumentation, and tools to analyze behavior in compounds. Proposals are in for six extramural screening centers.”
Molecular imaging is an emerging research area aimed at imaging specific molecular pathways in living tissues and cells, particularly those that are key targets in disease processes. Unlike anatomical imaging, molecular imaging displays the biochemical and physiological abnormalities that underlie disease, rather than simply the consequences of these abnormalities. Thus far, however, these methods have been limited by the poor sensitivity and specificity of currently used molecular probes. The molecular imaging initiative will encourage the development of new probes that will achieve an improvement of one-to-two orders of magnitude in the ability to detect and image specific molecular events in vivo.
“ We are also developing high-resolution probes for cellular imaging,” said Berg. “Nine P20 center grants are being funded through the roadmap. The goal of this initiative is to create molecular probes and imaging systems that are sensitive enough to detect and image individual molecules within living cells. The improved technology that will result from these efforts will increase the resolution of the images within living cells by 10-100-fold and will constitute an important step forward in understanding cell biology.”
Berg then went on to discuss the Building Blocks, Biological Pathways, and Networks Initiative. This involves complex elements working together in a feat of biological teamwork to promote normal development and sustain health. These systems work because of intricate and interconnected pathways that enable communication among genes, molecules, and cells. Scientists are still working to discover all of these pathways and to determine how disturbances in them may lead to disease. Two centers were funded to conduct this research in FY 2004 at $7.4 million. FY 2005 roadmap funds are devoted to membrane-bound protein structure.
The National Centers for Biomedical Computing Initiative will create a networked computational infrastructure for the nation’s biomedical computing needs. The centers will also play a major role in educating and training researchers to engage in biomedical computing. These four centers were funded in FY 2004 at $12 million through a U54 grant. Applications are due on January 24th.
A long-term goal of the NIH Roadmap is to create materials and devices at the level of molecules and atoms to cure disease or repair damaged tissues, such as bone, muscle, or nerve. Nanomedicine is an offshoot of nanotechnology. A nanometer is one billionth of a meter, too small to be seen with a conventional lab microscope. Nano-medicine centers will focus on developing methods to precisely define the physical characteristics of structures inside cells at the molecular level. Teams of scientists from an array of disciplines, including cell biology, biochemistry, mathematics, physics and engineering, will work together to develop new technologies so they can better understand the molecular interactions within living cells and the physical and chemical properties of molecular structures at the nanoscale. Awards made in FY 2004 will support planning activities for the development centers that will be funded in FY 2005 and FY 2006.
Patricia A. Grady, Ph.D., R.N., F.A.A.N., Director of the National Institute of Nursing Research, discussed research teams of the future. “This initiative is meant to encourage multi- and inter-disciplinary teams and promote investigators to take creative approaches,” said Grady. “We will be looking for evidence of scientific innovation and creativity. It will be a testimony of intrinsic motivation, enthusiasm, and intellectual energy.”
Planning grants will be awarded to begin interdisciplinary research programs that will address significant and complex biomedical problems, particularly those that have been resistant to more traditional approaches. One inventive program, entitled Training for a New Interdisciplinary Workforce, applies an entirely new funding mechanism toward supporting interdisciplinary work at all levels, from undergraduate students through postdoctoral researchers. Interdisciplinary research will involve P20 centers and innovative training programs. P20 exploratory centers for interdisciplinary research will increase the complexity of questions and require both multidisciplinary research and interdisciplinary research.
“ Behavioral epidemiology integrates theoretical, methodological, and analytical problems,” said Grady. “It also integrates new disciplines to address antimicrobial resistance. New funding mechanisms support interdisciplinary work from undergraduate students. It will include short-term and long-term training. Undergraduate engineers will engage in clinical research. With harmonization among public and private partnerships, we will try to identify and take advantage of opportunities.
Robert Star, M.D., Senior Advisor for Clinical Research at the Office of Science Policy in the Office of the Director at NIH, discussed the need to speed testing results. “We can re-energize the clinical research workforce,” he said. “This would involve enhancing training for clinical research and supporting translational research. We doubled funding for the NIH clinical center in FY 2004 through the Pre-doctoral Clinical Research Training Program (Request For Application in FY 2005). We set up a contract to look at the feasibility of this. There will be an apprenticeship in mentored multi-disciplinary team centers. Regional Translational Research Centers offer clinical services, core technologies provided nationally, and combined centers. An RFA is available for planning grants in FY 2005 and 2006.”
Star also discussed the Patient-Reported Outcomes Measurement Information System. The goal is to develop item banks. It will integrate clinical research networks and informatics and link existing networks so clinical studies and trials can be conducted more effectively. This system will also coordinate and improve policies and requirements for clinical research.
When asked how much money has been spent on the Roadmap and how much will be spent in fiscal year 2009, Dushanka Kleinman, D.D.S., M.Sc.D, Assistant Director for Roadmap Coordination at the NIH Office of the Director, said $129 million was spent in FY 2004 and $507 million by FY 2009 – 1% of the NIH budget each year.
Another question had to do with the basic structure of the Roadmap and getting bench to bedside results. Someone from the audience asked if the NIH Roadmap will provide for scientific citing discoveries.
“ If you look at the initiatives that were mentioned, integration is a part of every component of the roadmap,” responded Zerhouni. “The roadmap cannot, however, cure every disease. This is like building highways the way we did in the fifties. We need to accelerate predictability before spending money on something and putting forth all of our effort.”
-Prepared by N.L. Cavarocchi
