Chemotherapy: Snake-Oil Remedy? : It Has Its Purposes, but Use Is Dubious in Some Cancers

Martin F. Shapiro is an internist and an associate professor of medicine at UCLA

Recent revelations about the apparent ineffectiveness of the experimental cancer drug interleukin-2 are but the tip of an iceberg of misrepresentation and misunderstanding about cancer treatments. Cancer researchers, medical journals and the popular media all have contributed to a situation in which many people with common malignancies are being treated with drugs not known to be effective.

Chemotherapy is a serious undertaking. It often causes hair loss, severe nausea, vomiting, bone-marrow suppression with associated hemorrhaging, infections and death. The only reason for chemotherapy should be to cure cancer, prolong life or relieve symptoms. For some cancers (such as leukemia, lymphomas, breast and testicular cancer), chemotherapy accomplishes one or more of these objectives.

Unfortunately, for four of the most common kinds of cancer (colon and rectum, pancreas, stomach and most kinds of lung cancer) there is no convincing evidence that chemotherapy offers any benefit whatsoever. Yet many people with these types of cancers are being treated with chemotherapy, and are not aware that they are subjecting themselves to considerable risks, discomfort and expense for no perceptible benefit. (It should be noted that a small proportion of these cancers can be cured by surgical removal of the tumors, and patients may obtain some relief of symptoms with radiation therapy.)

One problem is the quality of available information. The course of any illness is uncertain. The best way to assess the value of a treatment is to compare the outcomes of persons chosen at random to receive treatment or not to receive it. Such "randomized clinical trials" showed the effectiveness of chemotherapy for leukemia, drug treatment for hypertension and Laetrile's ineffectiveness in advanced cancer.

Yet cancer literature is full of studies that take inappropriate statistical shortcuts. When they find no evidence that treated patients do better than untreated ones, many authors subdivide treated patients into a group who "responded" to treatment and another group who did not. If they find that so-called responders lived longer than non-responders, they conclude that the treatment is effective for some.

But is this true? Some cancer patients inevitably would live longer than others even if they started with similar stages of illness. While many factors influence the course of the illness, one certainly is the rate of tumor growth. A rapidly growing tumor may rebound more rapidly after the assault of anti-cancer drugs. Thus "responders" may experience tumor shrinkage for a while because their tumors were growing more slowly to start with. This does not necessarily mean that they gain any additional time from the treatment.

Some oncologists inform their patients of the lack of evidence that treatments work. Others may well be misled by scientific papers that express unwarranted optimism about chemotherapy. Still others respond to an economic incentive. Physicians can earn much more money running active chemotherapy practices than they can providing solace and relief from suffering to dying patients and their families.

The National Cancer Institute does not help matters. It claims that survival is improving for all kinds of cancer, in part due to chemotherapy. This obscures the fact that for some of the most common tumors any minimal advance in five-year survival more likely is due to earlier diagnosis than to any effect of chemotherapy.

Of course, the search for effective treatments must continue. Patients who do not want to resign themselves to dying might well choose to participate in controlled trials of experimental chemotherapeutic regimens. This would contribute to knowledge and might lead to answers. Very few cancer patients being treated for colon and rectum, pancreas, stomach and most kinds of lung cancer are in such trials today.

In the meantime the public needs to be skeptical of purveyors of allegedly effective treatments for these cancers. Medical journals should not publish reports that fail to demonstrate either more cures, improved survival or amelioration of symptoms, in comparison with untreated patients. The media also should stop proclaiming every report on a new cancer drug as the breakthrough that could emancipate humankind from malignancy.

Such unduly optimistic publicity helps to create a climate in which patients resign themselves to chemotherapy for conditions in which it does not work. One patient comes to mind--a man in his 30s who was dying of lung cancer. He had failed to respond to three different chemotherapeutic regimens. When he came in to start a fourth one, I asked him if it was what he really wanted to do. "Do I have any other choice?" he asked.

There is a choice. The patient, family and friends can work together with health-care providers to maximize comfort and function, to sort through the maze of emotional and social problems evoked by the illness and even to grieve together over the impending death. Some patients will want to try chemotherapy even if they know that there is no evidence that it works. But it also is no disgrace for patients to forgo chemotherapy. If they were more accurately informed, many more might select this option.

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