Medical researchers must accelerate the pace of discoveries, make better use of all types of data, and collaborate more across disciplines, Dr Elias Zerhouni, director of the US National Institutes of Health (NIH), has urged.
Zerhouni told delegates at the Drug Discovery Technology conference in Boston that medicine is increasingly focused on treating patients before they show symptoms of diseases for which they are at risk.
To start, scientists should accept that relatively little is known about complex biological systems, and that much more remains to be discovered and understood, Zerhouni said in the conference's keynote speech. Major advances of the past few decades aside, it is still the case that scientists understand only a small percentage of how biological systems work and interact.
"We need to be humble," he said. "Something is missing in the equation, and that something is knowledge."
Filling in the missing pieces will lead to better drug discovery, for instance, with fewer failures late in the process of discovery and development, he said. When failures do occur, scientists need to make better use of information about why, because all data is potentially valuable, Zerhouni said, offering details, but no particular news regarding the NIH road map for future medical research.
The NIH serves as the federal steward of medical and behavioural research and consists of 27 institutes and centres. Operating as part of the Department of Health and Human Services, the NIH funds research and oversees federal medical policy.
The NIH road map established the initiatives the agency will focus on during the next decade, which scientists widely agree is a crucial time, with the need to pull together advances and discoveries of recent years into a more coherent, comprehensive approach, both to future research and to clinical practice.
Biomarkers are central to that approach, Zerhouni said. Biological markers include measurable and quantifiable biochemical features of individuals and population groups. They include enzyme concentrations, specific hormone concentrations that can be measured through blood tests and the distribution of a gene phenotype in a particular population. Biomarkers can indicate disease risk, psychiatric disorders and metabolic processes, among other things. Biomarkers are an important aspect of the emerging area of personalised healthcare to determine, for example, who will respond to a particular biopharmaceutical and who will not, based on the individual's genome.
But biomarkers are one of the areas where much more remains to be discovered and understood, Zerhouni said. Meanwhile, it is important to stave off skyrocketing healthcare costs and the rise in chronic conditions and diseases with an aging population. The NIH road map, therefore, emphasises the need to fund research, but also pushes the idea of helping patients head off more serious health problems.
For instance, the potential health risks of being overweight should be explained to patients who carry just a few extra pounds rather than waiting until people gain so much weight that their health is at risk, Zerhouni said. It's also much less expensive to work with a patient to get them to shed a few pounds through diet and exercise than to deal with someone who has much more weight to lose, or who develops diabetes, high blood pressure or other conditions related to being overweight.
High blood pressure is a general case in point, he said. The importance of controlling high blood pressure to reduce the risk of heart disease and stroke is well established. Yet patients whose blood pressure is in the high end of normal are likely not told they need to do what they can to control their blood pressure through exercise and diet.
When people with a tendency toward high blood pressure or being overweight reach middle age their risks of complications begin to rise. The time to do something is when their blood pressure and weight are at the high end of normal, Zerhouni said. That takes beating down resistance because people often don't see the need for intervention at that point. Yet, if their health isn't kept in check at that point, the costs to their bodies and to the healthcare system will escalate as symptoms do begin.
"In the 21st century we need to intervene before the symptoms appear, and we need to preserve normal functioning as long as possible," he said.
The NIH road map is online at http://nihroadmap.nih.gov
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