Seventh Annual Public Interest Organization Meeting

January 30-31, 2006 – Bethesda, Maryland


Dr. Elizabeth G. Nabel, NHLBI Director, welcomed everyone to the meeting. She noted that it coincides with her 1st anniversary as Director, NHLBI, and that she will look forward to meeting with the PIOs annually.

NIH: Overview and Roadmap Progress

Dr. Elias Zerhouni, NIH Director, described his vision for the NIH. He also noted several new NIH initiatives and responded to questions from the PIO representatives.

Dr. Zerhouni stated that NIH's success is measured by its ability to deliver research results and thereby improve health. More than 21,000 individuals from across the country serve on NIH peer review and advisory committees and provide advice to the NIH. The PIOs are essential for conveying the needs of patients and families.

Dr. Zerhouni explained that the NIH is embracing a new paradigm and adopting a vision that emphasizes "3Ps":
  • Prediction of pathogenesis
  • Personalized, precise medicine
  • Preemption of disease before it occurs.

Pursuit of this vision requires a major expansion of research, particularly in the area of genetics. The first step in uncovering the earliest stages of a disease is the identification of every molecular pathway related to it. To accelerate such discovery, the NIH has initiated support for genome-wide association studies.

Two key challenges that the NIH faces are the flattening of its budget and the need for new investigators. To address them, the NIH recently announced:

Dr. Zerhouni said that the NIH will continue to invest strategically in areas of science that developed during the doubling of its budget. In addition, the NIH will continue to invest in traditional and new research methods to advance progress toward the 3Ps.


The PIO representatives endorsed Dr. Zerhouni's vision of the 3Ps, particularly the possibility of personalized medicine. Dr. Zerhouni asked the attendees to consider how PIOs could support NIH efforts to transform medicine and research. He emphasized that improvements in health will come only through new discoveries and fundamental insights from NIH-supported research. Dr. Zerhouni urged the PIOs to view the NIH as an investment, not a cost. He noted that the NIH budget amounts to only $96 per U.S. resident per year and the NHLBI budget amounts to only $10 per U.S. resident per year.

Drs. Zerhouni and Nabel responded to questions on a variety of topics, as summarized below.

Allocation of the NIH Budget

The NIH sets research priorities based on scientific opportunities and on needs, which encompass more than just morbidity and mortality from a disease.

Dr. Nabel said that the NHLBI constantly looks for scientific opportunities that can fulfill research needs pertaining to heart, lung, blood, and sleep disorders and blood resources. The NHLBI formally obtains advice on scientific priorities from various groups, including the NHLBI Board of Extramural Advisors and the NHLBAC. The Institute is embarking on a strategic planning effort that will involve broad consultation with the scientific community.

Innovative Research and the 3Ps

The PIOs expressed concern about the ability of the NIH to accelerate innovative research on the 3Ps, given funding limitations. Dr. Zerhouni said that the NIH is identifying research gaps and opportunities through a "bottom-up," three-tiered approach, as each Institute/Center (IC) refines its planning process, the OPASI identifies opportunities that cross the ICs (e.g., establishment of molecular libraries) to be funded from a common pool of resources, and the ICs participate in trans-NIH initiatives in specific research areas (e.g., obesity).


Dr. Zerhouni noted that his message for all ICs is to"challenge the status quo"—that is, to look beyond existing theories and use new and creative research methodologies and tools.

Clinical Research

Dr. Zerhouni agreed that the General Clinical Research Centers (GCRC) program has been an effective NIH mechanism for functionally integrating clinical research across disciplines and that it should be continued in some form. Reforming the GCRC program and establishing Institutional Clinical and Translational Science Awards are aspects of the NIH Roadmap initiative to transform the research enterprise. The future growth of the GCRC program will depend on the NIH budget.

Long-term Oxygen Treatment

Dr. Nabel noted that the NHLBI has embarked on a clinical trial to evaluate long-term oxygen treatment in mildly hypoxemic COPD patients. The NHLBI is partnering with the Centers for Medicare and Medicaid Services to support the effort.

Stem Cell Research

Dr. Zerhouni noted the need for a balanced research approach in tissue engineering and regeneration that would include using stem cells to treat chronic loss-of-function diseases. The NIH spends more than $500 million annually for stem cell research. Approximately 50 percent of this research is on human stem cells, and much of the effort is in bone marrow transplantation. Dr. Zerhouni said that the NIH is fully committed to supporting the basic science that is needed to advance this field. He mentioned that applications of tissue engineering, such as the use of stem cells to build vessels, bone, and cartilage, are exciting opportunities that offer promise in the foreseeable future.

Gene-Environment Interactions

Dr. Zerhouni commented that research on gene-environment interactions is greatly expanding. He agreed that better measures of environmental triggers of diseases such as asthma are needed, and he encouraged the use of methods currently available to sequence the DNA of potential triggers.

Holistic Perspectives

Dr. Nabel noted that addressing patient quality of life (e.g., psychological, emotional, economic needs) in addition to medical needs is critically important. She said that the NHLBI is considering how to build psychological factors of health and disease into its portfolio of research, prevention, and education.

International Collaborations

Dr. Zerhouni remarked that completion of the haplotype map of the human genome stimulated increased international collaborations to identify genes associated with disease. He noted that autoimmune diseases (e.g., type 1 diabetes) constitute one of the most exciting fields of research and that the NIH's Collaborative Network for Clinical Research on Immune Tolerance will stimulate research on these diseases in human populations.

NIH Reauthorization

Dr. Zerhouni mentioned that several entities including the Institute of Medicine, the NIH, and Congress have addressed the need for NIH reauthorization. He noted that the NIH has not been reauthorized for 12 years and that the ongoing debate encompasses many difficult issues.

Last updated: June 15, 2006

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