The Drug That Could Have Been
When a young company called ImClone said it had a hot new cancer drug, its stock leapt on the bet that it would get an OK from the FDA. Now the stock has tanked, ImClone's charismatic CEO is indicted, and, Martha Stewart is in trouble, too. But the big losers are cancer victims. Here's the real story behind ImClone and why it failed to win the FDA's approval
No one listens to Sam anymore. Used to be, he would stroll into a New York society party and heads would turn. When he told tales of his plans to bring an important new cancer drug to suffering patients, friends listened raptly. "How soon would the new drug be approved?" they asked. Should they invest? Martha was one of the true believers. "Sam set up a situation," says a friend who jumped in, "where he personally got involved in people putting money into the company."
Sam, of course, would be Sam Waksal. Last week's indictment of the founder and CEO of ImClone on 13 counts of securities fraud and other charges added yet another chapter to the evolving summer scandal of seemingly boundless greed in America's corporate suites. That Stewart, the popular doyen of domesticity, has been implicated in the mess adds a nice frisson. Never mind that ordinary investors are out millions. Yet what has been lost in all the anger over corporate misfeasance is the fact that thousands of cancer sufferers for whom Waksal's drug promised a respite, and maybe even a cure, are left high and dry. When the Food and Drug Administration decided not to approve ImClone's new drug, Erbitux, it put a hole below the waterline of ImClone, and it also delayed the arrival of a potentially powerful weapon in the war on cancer, probably by at least several years. How that happened is an illuminating and cautionary tale, a story not just of fortunes lost, but of life and death.
It is also a story, a U.S. News investigation shows, of colossal failure--several, in fact. The FDA's system for bringing lifesaving drugs to market, a "fast track" process, utterly collapsed. The biotech division of the FDA that handled the Erbitux application from ImClone was manifestly not up to the task. The communication that should have taken place between the FDA and the drug maker, especially the guidance necessary to bring forth a new and useful drug, simply didn't happen. ImClone, eager for the many millions Erbitux approval would mean, tried to jump through regulatory hoops too quickly, ultimately failing to provide enough basic science to support its FDA application. Bristol-Myers Squibb, anxious to add a new cancer drug to its pipeline, failed to heed concerns raised by its own in-house advisers. In the end, in an irony that borders on the truly tragic, a study that showed that Erbitux might well be a highly effective aide in combating cancer ultimately sealed its doom.
Bright beginnings
The story of Erbitux might be said to have begun at the spring meeting of the American Society of Clinical Oncology in New Orleans in May 2000. Sam Waksal, 54, brash and charming as ever, and his more serious brother, Harlan, 49, were working the room. And they were hearing some very good news. Erbitux was being tested in clinical trials for several kinds of cancer at prestigious medical centers. One such trial was known as Study 9923. In that trial, Erbitux was combined with a proven chemotherapy drug called irinotecan being given to patients with colorectal cancer--a disease that kills 56,600 Americans annually. "Physicians involved in the study kept coming to our booth and telling us, `Your drug really works,' " says Harlan Waksal, who replaced his brother as CEO of ImClone in May. "We didn't know to what extent, but they were telling us that some patients were responding dramatically."
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