Such scientific understanding is still a huge step away from having a drug in hand, though. The deCode researchers still needed to find a compound that could safely hit the target. But luck was on their side. During the 1980s and ’90s, pharmaceutical companies had developed drugs to inhibit precisely this same protein, which they believed to be involved in asthma and other inflammatory diseases. DeCode decided to license a compound made by German drugmaker Bayer, says Mark Gurney, deCode’s head of drug discovery. The drug had proved safe and had already made it to the last stage of human trials before Bayer shelved it because it wasn’t much more effective than existing asthma drugs. Since deCode didn’t have to make a new drug from scratch, it saved itself five to seven years of work, says Gurney.
So far, the company’s scientists have discovered 15 drug targets – proteins implicated in common diseases such as osteoporosis and schizophrenia. That’s more “than any other individual research group,” says James Weber, director of the Center for Medical Genetics at the nonprofit Marshfield Clinic Research Foundation in Marshfield, WI. “Most other labs in universities don’t have the scale to crank the genes out like deCode.”
Not only is the quantity of the drug targets they identify unusual, say deCode researchers, but so is their quality. Traditional methods of uncovering targets – studying lab animals or cells in petri dishes – often result in drug candidates that aren’t very effective, says Gurney. “DeCode has unique assets that allow one to do more powerful and vastly more successful genetics studies of common, complex diseases,” says Klaus Lindpaintner, head of the Roche Center for Medical Genomics. “This helps us feel more confident that the targets are more relevant to disease, and medicine derived against them will actually work.”
The result: while traditional pharmaceutical companies will pursue half a dozen or more different targets in parallel for one disease, deCode places its bets on just one or two. That means less work, time, and money devoted to research on targets that turn out to be irrelevant, says Gurney.
That efficiency is starting to pay off. In addition to the heart-attack-drug clinical trials already under way, deCode plans to begin human trials of three more drugs by next year. Two of those trials will involve drugs that, like the heart attack drug, were originally developed by other companies for other uses. But early next year, deCode will begin human tests of its first drug made from scratch: a treatment for peripheral arterial disease, a narrowing of the arteries in the limbs. DeCode is also working on six other drug targets in partnership with Roche and Merck. All in all, what eight years ago started out as a quirky, off-the-beaten-path gene-hunting startup is starting to look a lot like a real drug company.