BETHESDA, MD—New National Institutes of Health Director Francis Collins, MD, PhD announced plans to leverage a recent increase in funding to strengthen the Institute’s position as a leader of scientific and medical research, and to become a leading proponent of health care nationally and worldwide upon taking his place at the helm of the agency last month. Dr Collins was sworn in August 17 as the 16th director of NIH. At a town hall meeting of NIH scientists, he laid out some broad themes that he believes the agency should focus its resources on in upcoming years. These include: improving and expanding medical knowledge, using that knowledge to benefit more people, and leveraging scientific data to contribute toward the current efforts to reform the nation’s health care system.

Technology and Translation

The answers that scientists receive through their research have always been limited by the scope of the questions they ask. New technology is allowing researchers to conduct studies with a broader scope and more ambitious goals, Dr Collins explained. Fields such as nanotechnology, chemistry, genomics, computer science and biomedicine provide scientists with opportunities to examine medical mysteries in areas where they used to be limited in what they could accomplish.

“We need to make those questions more comprehensive. We need to ask questions that have the word ‘all’ in them,” Dr Collins declared. “What are all the transcripts in a cell? What are all the protein interactions? What are all the genetic variations that play a risk in disease? Those kinds of questions are now approachable. Especially if we do the right job of making powerful databases publicly accessible to all who need them [and] empower investigators in small labs, as well as big labs, to plunge in to that kind of mindset.”

The field of cancer research is particularly primed for this kind of ambitious enterprise, Collins said. “The expansion of the Cancer Genome Atlas [an effort to accelerate understanding of the molecular basis of cancer] is now poised to go beyond the pilot project [and] it’s going to be a wonderful ride, and will teach us things about all of the reasons a cell goes bad and becomes malignant that we desperately need to know,” Dr Collins said. Other diseases such as autism would benefit greatly from new technology, allowing scientists to look at all of the possible causes of the diseases, both environmental and genetic.

In addition to its foundation of basic science, NIH needs to take advantage of new discoveries in the causes of diseases to push forward a bench-to-bedside agenda, Dr Collins explained. “We need to try to develop diagnostics and preventive strategies and therapeutics for the diseases we treat poorly and oftentimes can’t even diagnose.”

One such effort newly funded by Congress is theTREND (Therapeutics for Rare and Neglected Diseases) program. While there are more than 6,000 rare diseases, currently, therapies exist for only about 200 of them. Because of the small size of the patient population in each disease, they are not seen as attractive commercial investments, though collectively 20 million people are affected.

“Many of those diseases are having their causes identified by basic scientists, which positions the whole system to tackle therapeutic opportunities in new ways,” Dr Collins said. “Again, for that to be successful, we have to come up with a public-private partnership model where the front end of the therapeutic development is perhaps taken on in a more vigorous way by academic investigators funded by NIH than has necessarily been the case in the past. [Scientists should be] able to take an observation about the fundamental defect in the disease, figure out how to turn that into an assay that can be subjected to high throughput screening, identify promising compounds, and take that into the so-called ‘Valley of Death,’ where you try to take a promising compound and try to tweak it with appropriate medicinal chemistry to find something that has the right qualities to try in a clinical trial.”

This process is already happening in some areas of NIH, including the TREND program, Dr Collins said. The result is good science that also taps into the desire of researchers to use their work to affect a swift public benefit.

National and Global Health

NIH has always touted itself as the pinnacle of scientific objectivity—an agency separate from the partisan politics of Washington, DC, which lies just a few minutes down the road—and the debate over health care reform in the United States is one of the most partisan topics being discussed today. But that shouldn’t keep the agency from contributing to that discussion in any way it’s able, Dr Collins argued.

“We are being called upon increasingly at NIH to produce the data that’s necessary in order to make wise decisions about health care, and I don’t think we should be reluctant to respond,” he said. It is an opportunity for NIH to contribute to the discussion by helping legislators make decisions based on evidence and not prejudice. “NIH should be a major player to the solution to that ever-growing health care cost curve,” Dr Collin said.

The agency should also be a major player in the global health arena, helping to reposition the United States away from its perceived role of aggressor to something more benevolent. “The ability of NIH to play a role in U.S. soft power seems like an opportunity we should not pass up,” Dr Collins said. “The world has seen us for the past few years as the soldier to the world. Might we not do better, both in terms of our benevolent attitude and our diplomacy, by being more of a doctor to the world?” This should include expanding U.S. efforts at disease research and prevention beyond those areas already focused on, including AIDS, tuberculosis and malaria, to other conditions that are of concern to developing nations.

“Many of them are infectious diseases, but some of them are not communicable diseases that are increasingly common in the developing world,” Dr Collins said. “And we should focus not just on doing research on those countries, but with them and helping them to develop their own research capacity in the longer term.”

All of the agency’s efforts to expand and strengthen its position at the center of the medical research community are for naught if there is not a reinvigoration and empowerment of biomedical research, Dr Collins declared. “[We can do this] through stable and predictable funding increases; through high quality training programs; through a focus on encouraging young investigators; making sure our peer review system is rewarding risky and innovative approaches; emphasizing diversity in the workforces; and using the NIH common fund creatively to support projects that fall outside of the mandate of any single [institute],” he explained.

However, a lot of this will depend on the kind of financial support that NIH receives in upcoming years. It is a question that, Dr Collins admits, wakes him up on the middle of the night. He worries, “What is going to happen to the resource support of this amazing organization when the stimulus money runs out?” he asked. “Where will we be at that point? And will we find ourselves in one of those terrible experiences of falling off a cliff, which happened in 2003 and, as you know, made life difficult for the next 5 years.”

He noted that FY2011 is not that far away and that science does not operate on 2-year cycles. “Science, to make real progress, needs more support than that,” Dr Collins asserted. “Young investigators need to be confident that they have a funding trajectory that’s going to be stable enough that there’s a career for them, that if they keep coming with good ideas there will be support for them. The feast or famine scenario is incredibly destructive to the enterprise. It places our country at a disadvantage over some other countries that are on very strong growth trajectories.”

The battle for dollars in FY2011 will be a tough one, he said, and NIH will need to make the case that its functions help the country and the American people at multiple levels. He pointed out that, for every dollar spent by NIH on research, about $2.25 was returned within one year in terms of economic growth. There is also, he said, a strong case for job creation, though most government agencies can make the same argument. “We have to make that case that what NIH is doing is going to have a benefit to the public as quickly as possible,” he said. “Making this case has to be job one.”

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