Alice Ulreich had to face the reality that the cancer spotted in her colon a year ago had spread to her liver, and that chemotherapy–the most common treatment for the disease–was no longer effective for her.
Her options, doctors said, were to remove 80 percent or her liver–a surgery she probably would not survive–or to try an experimental drug, CPT-11, available only in France and Japan.
“I told them go get it. Get anything you can,” said Ulreich, 63, who for four months had not received any treatment.
“God put everyone here for a purpose,” she said. “If my purpose is to be a guinea pig, then let’s go for it.”
If faced with the same prognosis, many terminally ill patients would make the same choice, according to health professionals. When death is a reality, some patients are willing to try almost anything that offers hope.
A measure implemented last month by the U.S. Food and Drug Administration is designed to offer new hope to cancer patients who are literally counting the days they have left. The measure could cut in half the year it usually takes for FDA approval. That means cancer drugs, such as CPT-11 and as many as 100 others, could hit the U.S. market as early as six months after they are submitted to the FDA.
Access to such drugs could tack days onto the lives of many terminally ill patients and make their final days more enjoyable by reducing sickness.
Doctors warn, however, that just because a drug receives FDA approval doesn’t mean it will extend life.
“I certainly understand the frustration of patients with cancer. These are people who will take any treatment which holds hope for them,” said Dr. Ellen Gaynor, associate professor of medicine in the hematology/oncology division of Loyola University Medical Center. “But it could create some false hopes.”
Physicians also said that this potentially bright hope for the future of cancer patients also has a dim side with far-reaching repercussions.
“It’s a clear detriment to research when drugs get approved early,” said Dr. Raymond Woosley, chairman of pharmacology at Georgetown University. Woosley is working on legislation with FDA members to improve the use of experimental drugs.
Since the FDA implemented accelerated approval for some AIDS drugs in 1991, research for that disease has experienced slight setbacks, experts said. They fear such setbacks in cancer research could have far-reaching effects.
While current cancer patients would benefit most from the new measure, long-term research that could aid future cancer patients may suffer, said Dr. Michael Roizen, chairman of anesthesia and critical care at the University of Chicago Hospitals.
“(Patients) will opt out of the (research) system that shows what really works to instead get what they hope works,” he said. “That delays treatment for everyone coming after them.”
And there is always the risk that a drug not subjected to the level of testing currently in place to meet FDA standards could be harmful.
FDA officials acknowledge the potential problems, but they say the benefits outweigh any potential flaws.
“We’re never in a situation of having all the information available. Every single time a drug is used, more information is gathered,” said Dr. Randolph Wykoff, a spokesman for the FDA.
And most terminal patients, they say, would agree with this approach.
Ulreich was willing to try CPT-11, even though she knew nothing about its possible side effects and had no idea how it was administered.
In order to secure the drug, Ulreich’s doctors sent letters, faxes and placed international phone calls on her behalf to the producers, a Japanese company known for making yogurt products.
Two weeks ago, Ulreich won “compassionate use” approval, a process the FDA uses to dole out experimental drugs on a case-by-case basis. And on Friday, she received her first dosage of CPT-11.
Had the drug, which has been administered to American cancer patients in clinical studies for about a year, been already approved by the FDA, Ulreich could have received it with a simple doctor’s prescription.
Under the new measure, experimental drugs, normally subjected to three phases of testing, will skip the final level used to determine whether the drug actually extends life. Instead, if the drug is found just to shrink a tumor, it can be approved.
Loyola’s Gaynor acknowledges that this will provide greater availability of new drugs to patients. But, “Will we know as much about those drugs? No,” she said.
But Ulreich’s concern is simpler.
“I’m going to die anyhow,” said Ulreich. “(The drug) gives me something to hope for.”
