Drugs By Design

Thanks to genetics, the pharmaceutical industry is exploding with new ideas

By Christine Gorman Monday, Jan. 11, 1999

It is the year 2025, and some things haven’t changed. The sky is still blue. The Dow is poised to set another record. And Jose Rodriguez (Michigan State, class of ’04) has just learned that he has colon cancer. But he’s not too concerned. Thanks to the genetic revolution that swept over the pharmaceutical industry 30 years earlier, scientists have developed a variety of anticancer drugs that work far better, and with fewer side effects, than the old poison-and-burn treatments of the late 20th century.

The oncologist takes a few cells from Jose’s tumor and places them on a microchip. Within minutes, the chip identifies five mutant genes that, like some kind of diabolical cheerleading squad, have pushed Jose’s cancer to grow, grow, grow. Someday, perhaps soon, doctors will be able to fix the wayward genes themselves. Until then, they will have to rely on the next best thing: drugs developed by pharmaceutical firms that block the destructive messages generated by the errant genes. Jose’s physician selects a combination of treatments that matches the tumor’s genetic profile. Six months later, no trace of Jose’s cancerous growth can be found.

That scenario is not as farfetched as it sounds. Talk to anyone in the pharmaceutical industry, and you’ll soon discover that genetics is the biggest thing to hit drug research since a penicillium mold floated into Alexander Fleming’s petri dish. Sure, scientists have long known genes play a role in almost every ailment from Alzheimer’s to yellow fever. But it is only in the past few years that they’ve learned how to use that information to identify a multitude of new targets and pathways for drug design. Let’s count the ways.


Geneticists estimate that there are 2,000 to 5,000 genes that either cause, or predispose humans to, various diseases. In practical terms, that means there will be many, many more potential avenues of research than the entire pharmaceutical industry could possibly hope to investigate over the next 20 years. Each company has a different strategy for exploiting that bonanza, and most are more than happy to tell you what’s wrong with the other guy’s approach. But they all agree on a few key points:

–Drugs will be safer, more powerful and much more selective than ever before.

–Doctors will be able to consult your genetic profile to determine ahead of time whether you are more likely to respond to one type of medication or another.

–Computers and other digital technologies are going to play a much bigger role in evaluating new research and determining how patients should be treated.


To understand how this promising future might come to pass, it pays to review a little history. Back in the old days–which is to say just a few decades ago–the process of discovering a new drug was a lot like shooting a quiver of arrows into the air and then running around to see what they hit. Occasionally scientists would get lucky, as Fleming did in 1928, but most of their efforts were wasted.


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