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Genomics

By: Haseltine, William A. | Brookings Review, Winter 2001 | Article details

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Genomics


Haseltine, William A., Brookings Review


The Path Ahead for Science, Medicine, and Society

We are living in a time of breathtaking advances in our understanding of how the human body works. We are learning what makes us function at the most fundamental level: that of genes and the proteins they produce. Automated analytical equipment--much of it based on the same powerful capabilities that have given us desktop supercomputers--now makes it possible for a research group to accomplish in a few weeks what would a few years ago have been unimaginably time-consuming.

Until recently, a veil of mystery obscured the myriad biochemical mechanisms that control the cells making up our bodies. Physicians and scientists fighting disease could observe only relatively superficial changes in response to their treatments. Today, however, we have the tools to tease out the fundamental mechanisms involved in disease and to put that knowledge to use. However uncomfortable it might make some people, human beings are in fact extremely complex machines that can be understood. The veil is starting to lift.

As we learn how our molecular components work, we are also learning how to manufacture and manipulate them. Clinical researchers are discovering how to use human molecules--genes, proteins, and antibodies--as drugs. Dozens of such drugs have already successfully run the gauntlet of clinical trials and been approved for use: clotting factors for hemophiliacs, Epogen for anemia, and Enbrel for rheumatoid arthritis, to name just three. These and other biopharmaceuticals have increased life span and quality of life for patients with diseases as diverse as cancer, multiple sclerosis, and diabetes.

Such natural human substances are destined to overtake the small-molecule drugs that dominated late 20th-century medicine, in terms both of therapeutic power and of market share. Indeed, as the traditional pharmaceutical industry faces a serious but little-recognized crisis of productivity, the science of genomics, a new discipline that has captured most human genes in a useful form, is entering its full flowering. While the rate of discovery of conventional drugs declines, genomics is identifying a rapidly increasing number of possible biopharmaceuticals.

Over the coming decade these two …

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