Gene Blues.

• Author: THOMPSON, NICHOLAS
• Position: United States Patent and Trademark Office overloaded - Industry Overview

Is the Patent Office prepared to deal with the genomic revolution?

IT'S EASY TO TRIP ON A YELLOW-AND-GREEN binder on the 12th floor of the United States Patent and Trademark Office's (PTO) biotechnology building. They're falling off the shelves, piled in corners, crowding every desk. Each represents a patent application and a claim to something new and special. Some come from wild-eyed, street-corner scientists; others come from more reputable sources. Grab a dusty file from the PTO's archives and you might just find a submission by a Thomas Edison. Increasingly, though, the shiny new binders arrive from large companies, packed with almost endless chains of the letters A, C, G, and T--sequences of the chemicals that make up our genes and control our bodies, from the thickness of our bones to our susceptibility to Tay-Sachs disease.

When these binders arrive loaded with genetic information, young men and women, often recent chemistry or biology PhDs, examine them against the PTO's requirements. The examiners initially reject most submissions but subsequently accept about half after the applicant makes suggested revisions. If approved, the submitting company gets 17 to 20 years of monopoly rights to that gene, depending on how long the application process took.

The consequences of this administrative procedure should inspire awe. Consider that at least four binders have come in, or are on the way, from rival pharmaceutical companies trying to patent the gene that creates beta-secretase, the enzyme that scientists believe causes Alzheimer's disease. Financially, the company that wins the patent battle stands to make enough money to buy Versailles. Ethically, it means a chemical structure that exists in all of us, and has evolved since life began to form in the oceans, gets entrusted to a company that probably didn't exist when Clinton was first elected president.

The winner might share the patent with the world, recouping research costs and taking a good profit. If so, a broad choice of affordable Alzheimer's drugs could come on the market. But the patent winner could also decide to shut down all research on beta-secretase by rival companies, universities, and public agencies. Then it could raise prices through the roof, making the drug inaccessible to most of the world, but maximizing its take from rich people willing to sell the family silver for a bottle of the pills. Public money funded much of the research that led to the discovery of beta-secretase, but neither the patent office nor any other branch of government seems to have any control over whether the patent is hoarded or shared.

Unfortunately, if prior work on Alzheimer's is any indication, we may be in trouble. In 1999, for example, Athena Diagnostics prevented laboratories from freely using a key gene at the core of an Alzheimer's diagnostic test and mandated that all tests be routed through Athena's own laboratories for a fee because the company controlled the patent. Subsequently, critics suggest, overall research on the gene may have slowed since many laboratories research the same genes they test for. Athena is now owned by another drug company, Elan, currently involved in a legal struggle with the nonprofit Mayo Clinic over the clinic's Alzheimer's research using mice containing a human gene patented by the company.

Elan is hard at work researching beta-secretase and its prior actions suggest it might well take a hard line if it earns a patent. According to author David Shenk, who is currently writing a book on Alzheimer's: "Elan is doing some spectacular research. But ethically, there seems to be a disconnect between its lab work and its legal agenda" Other companies are split the same way and, no matter which one gets the beta-secretase patent, restricted access will mean a lot of people staying sick longer than they should.

We are on the cusp of a medicinal revolution, fueled by a marvelously innovative pharmaceutical industry dominated by the United States. Most drugs work either by activating or disabling specific proteins. By figuring out the genes that create our natural proteins, we move one giant step closer to designing proteins that make us live longer and expunging those that make us fall ill. Pharmaceutical companies have only developed a few gene-based drugs, and there are several deeper layers of biological complexity to mine beyond the genome. But we are learning how to play with the building blocks of life and hurtling toward the day when we have genetic tests to determine which diseases we are likely to get, as well as drugs to cure all the ones we do.

In the midst of the revolution, the only government agency helping to define the market is the PTO, but the organization's mandate consists of little more than giving the thumbs up or thumbs down to applications. If an applicant can show that her invention meets four criteria--it's novel, useful, non-obvious, and fully-disclosed--she gets a patent, regardless of...