The essence of biotechnology is exploiting genes. Already, biotech companies have homed in on a few that make insulin or human growth hormone and have quickly produced new drugs. Scientific sleuths have also identified and deciphered the flaws in DNA that cause such inherited diseases as cystic fibrosis and muscular dystrophy. Their discoveries have yielded diagnostic tests and may eventually point the way to new therapies.
Yet these advances involve a mere fraction of the 50,000 to 100,000 genes in every human being. Within the spiraling strands of human DNA is a treasure trove of information. Who is likely to get heart disease or cancer--and how can those be prevented? How does the aging process work? Finding the answers is a tedious and expensive job: It cost $150 million just to identify the gene that causes cystic fibrosis. Such projects would move faster and cost less if there were a map that showed where bits of DNA are located.
`DRUGS GALORE.' So, two years ago, scientists conceived the Human Genome Project, a 15-year, $3 billion federal program to decipher humanity's genetic heritage. Billed as one of the great adventures of science--and as a key to keeping U.S. biotech companies ahead of such rivals as Japan--it will lay the foundation for a new leap in biology and biotechnology. "The objective is a new infrastructure for biology," says molecular biologist Leroy E. Hood of the University of Washington. Eventually, says Charles DeLisi, dean of engineering at Boston University and a founder of the genome effort, "this project will produce drugs galore."
Funded by the National Institutes of Health and the Energy Dept., the agencies that oversee the work, scores of scientists have started the mapping by trying to locate genetic signposts spaced along the 23 pairs of human chromosomes. At the same time, researchers are drawing up similar guides to the genes of roundworms, fruit flies, and mice. This effort is critical, since species share many genes--and the best way to determine what these genes do is to conduct animal experiments. Long-term, with men and mice alike, the goal is to "read" each piece of DNA between the genetic signposts, a task called sequencing. That process will identify thousands of new genes, plus the pieces of DNA between them that control the body's genetic machinery. Once genes are found researchers expect a quick commercial payoff in diagnostic tests for such conditions as a predisposition to heart disease. New drugs are further away.
Indeed, it's too early to point to important accomplishments, says David J. Galas, who heads the Energy Dept.'s part of the effort. Still, there are tantalizing clues to how powerful the new knowledge will be. The genetic map of the roundworm has helped California Institute of Technology biologist Paul Sternberg discover that a human cancer-causing gene called ras plays a key role in the division and function of cells during normal development.
A STORM. The promise of genome project has already raised the issue of commercialization. Last year, the NIH touched off a storm by applying for patents on 347 pieces of human brain genes sequenced by one of its biochemists, J. Craig Venter. On Feb. 12, the agency applied for 2,375 more patents. No one knows what most of these genes do, or where they are located on the chromosomes. And some experts fear that the NIH's moves could prompt universities and companies to patent blindly every gene in sight, simply to lock up rights to them. "We who are in charge of the genome project think this is a disaster," says Stanford biochemist and Nobel laureate Paul Berg, chairman of the NIH's genome advisory committee.
The NIH itself argues that merely publishing its findings might keep them from being followed up, since once they are public it may be difficult to win a patent. That would limit profit potential. Some patent lawyers think the NIH filings won't survive, since bits of unknown genes may not be inventions worthy of protection. If they are, the NIH promises to smooth commercialization.
So one way or another, whatever the Human Genome Project discovers will find its way to biotech companies. For an industry whose job is unraveling the unknown, that can only be a big plus.