Reporter
Miracle Workers
In the history of medicine, there hasn't been anything quite like M.D. Anderson, where curing cancer is a righteous mission.
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Scientists and the public alike had always been drawn to the economy of the notion—if cancer was a genetic disease, then why not treat the genes?—but it had become controversial. For one thing, the preferred means of importing the healthy genes to the tumor was the shell of the common cold virus. Even though the virus was stripped of its viral DNA, some scientists couldn't get past the idea of willfully injecting someone with an invasive virus. And when a patient in a Pennsylvania clinical trial involving the technique died a few years back, the subsequent bad publicity seriously tainted its reputation.
But Roth's experiments with lab animals and early trials with human patients had been successful. Gene replacement wasn't a magic bullet, but it could cause some tumors to reverse course and at least lengthen the lives of some patients with advanced malignancies. In Fleming's case, it took eighteen injections of p53 over four years to stabilize her lung tumor. Even Roth admits that she is not cured. "She represents a new paradigm for cancer," he says, "that is, patients who can live well for longer periods with their disease under control."
She represents another paradigm as well: Fleming wasn't the sort of patient you would expect to receive cutting-edge treatment. Neither wealthy nor indigent, she is a member of the broad middle class (like my dad) for whom the idea of getting the best treatment in the world seemed beyond reach. That she was able to prolong her life by finding such treatment at the Anderson via the Internet and afford it through her own Medicare coverage and the grant funding that supported Roth's study was, in its way, as great an accomplishment for the Anderson as the success of Roth's gene therapy. After all, the problem with the American health care system is not the quality of our bioscience but rather how to get that science to patients at a cost that doesn't make the hospital, the patient, or the insurance company go broke.
IN SOME WAYS, M. D. ANDERSON'S innovations in the business of cancer care have been as impressive as the gee-whiz science produced in its labs. Less than a decade ago, the hospital was headed for a financial meltdown. Many managed-care companies had deemed it too expensive and were directing their patients to cheaper treatment at general hospitals. At the same time, the cost of state-mandated care for the indigent had doubled. M. D. Anderson was out tens of millions of dollars it could have spent elsewhere. Worst of all, a state law prohibiting Texans from being admitted to the hospital without a doctor referral—to preempt charges that the state-supported institution was unfairly competing with private physicians—kept the center from seeing many paying patients. The crisis became severe enough to force a $90 million cut in the budget, including layoffs. "Patients couldn't get into the hospital; patients who did couldn't pay," recalls Anderson's former vice president for strategic planning, Martin Raber, himself a cancer survivor.
M.D. Anderson lobbied the Legislature and schmoozed the Texas Medical Association to get the prohibition against self-referral removed in 1995. In return, the center promised to find more budget cuts. But that was the easy part. The Anderson also had to persuade managed-care companies to let their patients come. When Mendelsohn arrived, in 1996, he told the HMOs that M.D. Anderson would play ball by managed-care rules.
"We had to convince them that it might be cheaper in the long run if we took care of the patient, even if we were more expensive to begin with," he says. "We have a thousand doctors, and about two thirds are clinicians who treat nothing but cancer, and one third are Ph.D.'s who study nothing but cancer. We have a very good chance of doing the right thing the first time."
The business acumen displayed by Mendelsohn, who is now 66, was unexpected. He had been a dark-horse candidate for the center's presidency and had attained the post largely because of his international reputation as a cancer researcher, not his skills as a manager or an entrepreneur. At Harvard, he studied under James Watson, the Nobel laureate who co-discovered the structure of DNA. He had taught and done research at the University of California at San Diego, then served as the chairman of medicine at Memorial Sloan-Kettering before taking the top post at the Anderson.
No one anticipated that it would be research that would get him in trouble. While at UC-San Diego in the early eighties, Mendelsohn developed a cancer treatment based on preventing a cell-growth signal from binding with cancerous cells, thus halting cell proliferation without damaging normal cells, as most chemotherapies do. The therapy, called C225, was promising, but he had trouble finding a corporate home for the development and marketing of the drug. In the early nineties he finally settled on a small New York-based biotech company called ImClone, which had been started by two brothers, Sam and Harlan Waksal. As it happened, the Waksals had been casting about for a "marquee medication" to put their company on the map. By the time Mendelsohn arrived at M.D. Anderson, C225 had entered clinical trials and soon after began to show promise.
Six years later, C225—since renamed Erbitux—has not only failed to win FDA approval but has become the focus of a scandal. In December 2001 the FDA shocked the world of cancer research by rejecting ImClone's application for accelerated review of Erbitux, citing the poor design of the clinical trials and incomplete data. That spring, news surfaced that, according to Securities and Exchange Commission investigators, ImClone CEO Sam Waksal had had insider knowledge of the FDA's impending rejection of the drug days before it was made public (and ImClone's stock price started to collapse). He had allegedly tried to dump some of his own holdings and advised family members and at least one friend—Martha Stewart—to do the same. Waksal has been indicted for bank fraud, forgery, and other charges in the insider-trading scandal, Stewart is under investigation, and Mendelsohn has been left to try to explain how he, a member of ImClone's board of directors and the inventor of its primary product, could have been outside the loop.
I had met Mendelsohn back in the spring of 2001, when I first began poking about on a story about the Anderson. White-haired, spry, and boyishly enthusiastic, he was erudite, animated, witty, and opinionated, and he uttered the single pithiest statement about the war on cancer I've ever heard: "Our bodies are so complex that we're lucky we don't get cancer every day. In fact, the five-year survival rate has doubled in my lifetime, so maybe we are winning the war." By the time I got back to him this summer, the ImClone controversy had exploded into a tangle of questions about what Mendelsohn knew and when he knew it. Three issues continue to dog him. The first involves whether his cashing out about $6.4 million in ImClone stock (options granted to him years ago as a scientific collaborator and consultant on his invention) during the year before the FDA's rejection of Erbitux was because of insider knowledge. The second is his role, if any, in the research that the FDA rejected. Finally, in late June, the Washington Post raised questions about why Mendelsohn had neglected to include his financial interest in Erbitux in patient-consent forms used in a trial of the drug at the Anderson (though not managed by him).
His response to my question about insider knowledge was a flat no. "In fact, I sold twenty percent of my shares because Bristol-Myers Squibb was buying twenty percent of ImClone and all stockholders were invited to tender their shares." A review of SEC filings reveals that Mendelsohn sold his shares in two lots—in April 2001 and then again in October—well before the late-December window during which the insider trading allegedly took place. While any large liquidation of ImClone stock within, say, a year before the Erbitux rejection might be viewed with suspicion, investigators appear to be focusing on transactions that occurred during the month before the FDA announcement. Mendelsohn's name has not surfaced.
As for his responsibility for the research that did not pass FDA muster, Mendelsohn explained that the clinical trial the FDA rejected had not taken place at the Anderson, let alone under his control. He added that when he took over the Anderson, he promulgated a policy prohibiting staff scientists with a financial interest in a particular therapy from either managing a clinical trial or directly administering it to patients. "I've never given any patient my drug," he told me. The Anderson did conduct trials of Erbitux during Mendelsohn's tenure, which led to the Washington Post story that he did not reveal his stake in the drug in patient-consent forms.
Even as Mendelsohn found himself explaining the ImClone mess, the Anderson was rescued by a U.S. News and World Report survey that named it the best cancer hospital in the nation for the second time in three years. The juxtaposition of the two events indicates the degree to which M.D. Anderson, and all of modern medicine, finds itself under ever-increasing scrutiny. Having watched the cancer war for a number of years now from several perspectives—medical journalist, bereaved son, and concerned potential victim—I understand that Big Science equals Big Business equals Big Politics. Still, as Houston scandals go, we need to keep this one in perspective. Ultimately, we can survive without Enron. Many of us can't survive without M. D. Anderson.
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