Genomics

By Haseltine, William A. | Brookings Review, Winter 2001 | Go to article overview

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 intersecting trends promise a sea change in the practice of medicine that portends proportionate change in the pharmaceutical industry. Drugs will become more effective, and some key players in the biotechnology sector will become industrial powerhouses. Medicines that cure disease and greatly reduce the cost of care while providing many years of productive, active, healthy life will be developed, provided government policy, both in the United States and in other developed nations, supplies the necessary guarantees for protection of intellectual property and profit.

Regenerative Medicine and the Power of Biology

The reason biological molecules have such promise as medicine is that they can exert more subtle and proactive effects in the body than can conventional drugs. Whereas traditional drugs mostly work by inhibiting some cell constituent, a biological molecule can reprogram a cell, changing its activity and how it develops. By revealing the full range of natural biological molecules available for use as drugs, genomics has unlocked the potential tentatively demonstrated in the first phase of biotechnology more than a decade ago.

Last summer's triumphant declaration of victory by the projects aiming to sequence the human genome has drawn much popular interest, even though the job is not yet truly finished. And even when it is, this scientific achievement will be largely a distraction as far as the business community is concerned. A large segment of the pharmaceutical industry has had almost all human genes to work with for about the past five years, through collaborations with my company and others that have made headway in identifying genes. …

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