Cancer Drug Fails, So
August 2, 2007
NEW YORK — Garo Armen froze as colleagues broke the bad news last year about a kidney-cancer vaccine he had championed for over 12 years at a cost of $300 million: The clinical trial had failed. Shares of his tiny biotechnology firm, Antigenics Inc., plunged. His ambition and patients’ hopes were dashed.
Now Mr. Armen is on the move — traveling the world on a quest to persuade regulators to approve the vaccine despite the initial results. His argument: The vaccine worked well in a subgroup of less-sick patients.
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Yet the U.S. Food and Drug Administration has never approved a drug based on efficacy in a subgroup of patients, defined after a trial. With results in hand, the agency believes, companies theoretically could examine many subgroups before finding one in which their drug appears to work. “It’s like shooting an arrow at a target and after the arrow hits the target, declaring that’s a bull’s eye,” says Robert T. O’Neill, director of biostatistics at a FDA center. “But you don’t look at the arrows on the ground that didn’t make it.”
At root is the question of how much proof is enough — and whether standards should be less rigid to allow quicker approval if medicines have few apparent side effects and would treat deadly diseases in people with limited or no treatment options.
Underlying Mr. Armen’s challenge is the broader frustration between the fast pace of biological breakthroughs in understanding cancer and the relative trickle of novel medicines coming to market to treat it. Critics argue that standards of proof should be relaxed in some cases. They say traditional ways of assessing efficacy could block or delay treatments that bring incremental benefit but may collectively contain cancer.
“We have to find creative ways of getting cancer drugs to patients even if we end up being wrong a few times,” says David Kessler, dean of the school of medicine at the University of California, San Francisco. He was FDA commissioner during the AIDS epidemic, when he amended the approval process to speed 12 new treatments to market.
[Garo Armen]
FDA officials defend the current approval standards, saying it is better to delay a new drug until there is definitive proof it works. “I don’t think you want the FDA to approve a drug saying there’s a chance this works. I think you need to know the therapy works,” says Robert Temple, director of the office of medical policy at the FDA.
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“If somebody uses a vaccine that doesn’t work, they will be forgoing something else,” Dr. Temple says. While he recognizes that some patients have “no options left,” he says the best route for them is to appeal to the FDA and to drug manufacturers for permission to use experimental medicines.
For years, companies have been hesitant to publicly tangle with the FDA, but some are now openly challenging its standards. Pharmacyclics Inc., a California biotech firm, refiled an application in April for approval of a cancer drug, despite the agency’s refusal to review it in February. Cell Therapeutics Inc., a Seattle biotech firm, argues its lung-cancer drug is being delayed by the FDA’s insistence it conduct another trial. Prostate-cancer patients and shareholders have protested the FDA’s refusal in May to approve a cancer vaccine from Dendreon Corp., another Seattle biotech firm, recently buying a half-page ad in the Washington Post, entitled, “Dysfunction at the FDA.”
“When we’re near death, and when there’s little in the medicine cabinet, don’t send these companies back to do another trial,” says Laurie Fenton Ambrose, president of the Washington, D.C.-based Lung Cancer Alliance. She says many lung-cancer patients “would be willing to take greater risk that a treatment doesn’t work.”
The FDA says it can’t comment on specific companies’ data. In 2004, the agency announced a plan to develop ways to approve drugs more quickly. It has held hearings and launched studies, but has yet to adopt significant new guidelines. Meantime, the FDA has been on the defensive on a different front, caught in the cross hairs of patient lawsuits against drug companies whose approved medicines — most notably the painkiller Vioxx — turned out to have serious side effects in rare cases.
Mr. Armen’s journey into drug development began when he was manager of a small health-care investment fund in 1993. Mr. Armen had been obsessed with cancer research since losing his mother to breast cancer soon after emigrating from Turkey 20 years earlier. The two shared a one-room Brooklyn apartment where Mr. Armen administered her morphine shots until she died.
A young researcher from Mount Sinai School of Medicine showed up in Mr. Armen’s office, pitching his research as the basis for a cancer vaccine. Unlike traditional vaccines that prepare the immune system to head off a disease, cancer vaccines aim to induce the body to fight a disease already attacking the body.
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