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All Drugs Need Not Be Smart Bombs
Matthew Herper, 02.13.04, 7:31 AM ET

Instead of clobbering every cell in the body with chemotherapy in the hopes that the malignant ones die first, such drugs are aimed straight into the chinks of a tumor's armor. The payoff, in theory, is better cancer drugs with fewer side effects. And that theory is starting to bring real benefits. Erbitux, the ImClone Systems (nasdaq: IMCL - news - people ) colon cancer treatment approved yesterday by the U.S. Food and Drug Administration, is one such treatment.  Avastin, a colon cancer drug made by Genentech, is expected to be approved soon.

But such smart bombs aren't the only source of advancement for cancer treatment. Chemotherapy can also be made safer and more effective. For example, look at Alimta, an Eli Lilly  drug approved by the FDA on February 5. In combination with another chemotherapy drug, it is the first treatment proven to extend the lives of patients who suffer from the type of lung cancer caused by exposure to asbestos. Adding Alimta adds an average three months to patients' lives.

Only 2,000 Americans are diagnosed with the asbestos-related lung cancer each year. But Alimta also seems to be effective against non-small cell lung cancer with fewer side effects than Taxotere, the current standard of care made by Aventis. More than 130,000 Americans develop non-small cell lung cancer each year. "Alimta is an extremely promising chemotherapy," says Robert Hazlett, an analyst at SunTrust Robinson Humphrey. "I think it's being under appreciated by investors." He forecasts that sales could eventually surpass $1 billion a year.

Like much-hyped biotech drugs like Erbitux and Avastin, scientists developed by being keenly aware of what proteins in cancer cells made promising drug targets. The drug grew out of a collaboration between Edward C. Taylor, a professor at Princeton, and scientists at Lilly. Taylor developed a chemical that seemed like a particularly potent form of a well-known class of chemotherapies called anti-folates. It was particularly good at inhibiting the production of certain chemicals used as rungs on the DNA ladder--which is one way to kill a cancer cell. With some modifications, Lilly scientists came up with a molecule that also inhibited two other cancer drug targets. Says Homer Pearce, a Lilly scientist who shepherded Alimta through development: "It was a triple whammy."

Pearce and his colleagues called the drug a "multi-targeted anti-folate". That's what the "mta" in Alimta stands for. What about the rest of the name? "Some marketing person came up with that," Pearce says.

Hitting lots of cancer targets at once could be the next big thing in cancer research, says Nicholas Vogelzang, the oncologist who headed up the clinical trials that led to Alimta's approval. He is currently director of the Nevada Cancer Institute. Instead of trying to be targeted, he says, drugs might be better off being promiscuous and hitting lots of targets at once. New targeted drugs being developed by Pfizer and Novartis try this strategy from the ground up. At the end of the day, it's how much a medicine can help a patient that matters.

"It's a drug that frankly is among the easiest chemotherapy drugs to give," Vogelzang says of Alimta. It still comes with some harsh side effects, including nausea, loss of appetite, low white blood cell counts, anemia, and rash. But chemotherapy can be far worse. It is not associated with hair loss as other drugs are. "People can actually get chemotherapy without wearing the scarlet A," Vogelzang says.