CANCER CLINICAL TRIALS: TO JOIN OR NOT?

After Virginia Garner, 56, was diagnosed with chronic myelogenous leukemia (CML) five years ago, her doctor prescribed interferon, a standard treatment at the time. But it made Garner so weak she had trouble completing the simplest everyday tasks. "I'd have to stop and rest in the middle of blow drying my hair," says Garner, a high school teacher in California. Just as her physician was about to stop the treatment, she found out about a new drug that was designed to attack only the cancerous cells and make the hellish side effects suffered while on interferon a thing of the past. The drug was in clinical trials.

Garner jumped at the chance to be part of the clinical trial. It was, she felt, her last hope. She had to wait seven months to join, until her white blood cell count had increased enough so she met the requirements of the trial. Finally, in April of 1999, she got on the new medicine. It worked wonderfully. Recently, she heard the good news: leukemia cells have nearly disappeared from her body.

Garner's story is dramatic, but illustrates just how valuable being part of a cancer clinical trial can be. Below, an introduction to clinical trials and how to decide if they're right for you.

The Basics of Cancer Clinical Trials
Most people know that a clinical trial for cancer is a research study in which doctors attempt to find ways to improve cancer care and health. When people talk about cancer clinical trials, they are usually referring to a treatment trial. But there are actually four different types of clinical trials in all:

• Treatment trials, such as the one Garner joined, test a new cancer drug, a new combination, or a new method of cancer treatment. Treatment trials are further broken down into phases. Phase I includes a small number of patients, perhaps only a dozen, and evaluates how a treatment should be given (such as oral or intravenously), how often and how much. Phase II studies the effectiveness of the treatment. Phase III compares the new treatment to standard treatment.
• Quality of life cancer trials focus on how to improve comfort and quality of life for cancer patients.
• Prevention cancer trials test ways to avoid cancer, such as vitamins, medicine or other approaches.
• Screening trials for cancer focus on the best ways to find cancer in its earliest stages.

But consider the outlook of Lee Rosen, M.D., a researcher who directs cancer clinical trials at the University of California Los Angeles' Jonsson Cancer Center. "We need to learn something from each cancer patient," he says, describing the ideal big-picture scenario for furthering cancer treatment and prevention. Perhaps there is not a clinical trial that offers a brand-new treatment approach for every cancer, but there may be another trial that the patient or a family member might join to learn more about the specific cancer or cancer in general, he suggests.

Finding Clinical Trials
Virginia Garner's husband, Van, found the clinical treatment trial she entered by chance as he surfed the internet for information on her condition. But there are many other avenues to find clinical trials. Your own physician might be involved in one, or may point you to several. Or, check out these resources:

• The National Cancer Institute posts its clinical trials database, PDQ, on its site, www.cancer.gov.
• The Food and Drug Administration posts its Cancer Clinical Trials Directory at www.clinicaltrials.gov.
• The Coalition of National Cancer Cooperative Groups, Inc., posts a trial list at www.cancertrialshelp.org.
• The Pharmaceutical Research Manufacturers of America (PhRMA) posts a list of new cancer drugs under development at its site, www.phrma.org. (Click New Medicines; then pick Cancer from the pull down menu.)

Making the Decision
Before deciding whether to join a cancer treatment trial, weigh the pros and cons with your physician and family and friends. The advantages: Health care is provided by leading physicians. You will have access to new treatments before others do. During the trial, your health will be monitored very closely. You may find a better treatment than anything else available. Last but not least, you are contributing valuable input to cancer research and treatment. There are potential downsides, of course. The drug or approach might not work--or it might not work for you. There may be side effects or risks not known to the researcher until treatment is underway.

Patients' Perspectives
The decision to join or not to join also depends on your medical status and how you are doing on the current treatment. "It depends on how much you have to lose and how much you have to gain," says Garner. "In my case I had everything to gain. For me, this is a miracle. I am supposed to be dead by now." Instead, it's been nearly five years since she heard the diagnosis and she's happy, working and fulfilled. If your decision is not so clear cut, Garner suggests talking with the investigator or the coordinator of the trial. Get as much information as you can, and try to develop rapport with the leaders so they will give you their honest opinion about whether you should join.

Among the questions to ask:

• What results do you expect?
• What do you anticipate the side effects to be? (They may not honestly be able to answer these questions thoroughly, especially for Phase I studies.)

Common Questions, Misperceptions
One of the most common concerns voiced by callers to the National Coalition for Cancer Survivorship (www.canceradvocacy.org), an advocacy group, is the fear that they will receive no treatment at all--a placebo, says Steven Friedman, director of policy for the coalition. But in cancer treatment trials, placebos are rarely used, according to the National Cancer Institute. One instance in which a placebo might be used is in the case of a rare cancer in which no standard treatment exists. But normally, treatment trials involve comparing the standard treatment to the new treatment, so cancer patients rarely have to worry that they will receive no treatment.

You also have the right to leave a clinical trial at any time you wish, even after signing the informed consent form. Once a trial is over, what should you expect? Amy Applebaum expected closer follow up than she received. "When the trials are over, they don't continue to get information from you, and that would seem to be easy to do," she says. But she acknowledges that the relentless pace of cancer research may make that not as easy as it might seem.

Checking in with your Insurance
Before deciding to join a clinical trial, read over your health insurance policy or contract, says John Metz, chairman of the board of the California Consumer Health Care Council (www.cchcc.org), an advocacy organization. "It may not mention clinical trials," he says. In that case, call member services and ask some questions about your participation. The drugs provided in clinical trials are free of charge to subjects, but you may need help getting to the trial if it is out of the area, for instance. Some patients have difficulty with their insurance plans when they want to join clinical trials, says Friedman of the National Coalition for Cancer Survivorship, although the situation seems to be improving. A typical problem, he says, is a patient who wants to travel outside her HMO network or service area to get care in a clinical trial in another state.

After the Clinical Trial
Although the decision to join a cancer clinical trial can be a difficult one, patients like Amy Applebaum who have been there, done that and were successfully treated have only appreciation for their experience. "At this time," she says, "I am told I am cancer-free. There is no sign of cancer on my scans."