Keeping Current with New and Experimental Treatments
The following article is excerpted from Chapter
6 of Advanced Breast Cancer: A Guide to Living with Metastatic Disease, 2nd
Edition, by Musa Mayer, copyright 1998, published by O'Reilly & Associates,
Inc. For book orders/information, call (800) 998-9938. Permission is granted
to print and distribute this excerpt for noncommercial use as long as
the above source is included. The information in this article is
meant to educate and should not be used as an alternative for
professional medical care.
In the ongoing struggle for control of the disease, new treatments are
constantly being introduced into clinical trials. In the last several years,
breast cancer research has been burgeoning, spurred in part by the influx of
government research funding that advocates from the National Breast Cancer
Coalition and other organizations have fought so hard to win since 1992.
Basic cancer research begun a quarter century ago has accelerated understanding
of the mechanism of cancers on a cellular level. Many innovative treatment
methods are just beginning to flow from these new developments, as well as
further refinements of conventional drugs. Now that more energy is being
expended in genetic and molecular biology research, there is every reason to
believe that within the next few years, sophisticated new methods that are
targeted (only to cancer cells) rather than systemic (affecting normal body
cells)--and thus less toxic--will be found to further prolong life for those
with metastatic breast cancer. Some of the more exciting new developments are
touched upon in the section, "What's new?" later in the chapter.
Because this field is rapidly evolving, it makes good sense, when you discover
your breast cancer has metastasized, or when the disease progresses further, to
devote some real time and effort to researching the state of what is currently
known about treatment for your specific situation. As we've pointed out
elsewhere, having a spouse, friend or family member do the research for you can
work just as well. There may well be new clinical trials for which you are
eligible. There may be innovative treatments unpublished as yet in the
literature, or not yet widely known, that your doctor may not even be aware of.
Those who do participate in clinical trials perform an invaluable service,
advancing and refining knowledge in the field of oncology that will eventually
help other patients. The National Alliance of Breast Cancer Organizations
(NABCO), in collaboration with the National Cancer Institute, estimates the
rate of breast cancer patients receiving treatment in clinical trials at about
5 percent. This very low level of participation is thought to have slowed the
rate of progress in treating the disease successfully. Why more women do not
take part has to do with limitations on research funding, as well as with
public and physician attitudes and barriers erected by the health care system.
NABCO speculates that certain misunderstandings perpetuate the low
By law, all clinical trials must include safeguards to protect patients. Trials
must be reviewed by both the sponsoring institutions and the medical community
for patient safety and scientific merit. Trials sponsored by the National
Cancer Institute (NCI) are required to meet extensive ethical standards of the
National Institutes of Health (NIH).
In a clinical trial, drugs are first tested for dosage and efficacy, and then
the best available treatment is compared with one or more experimental
treatments that have already been tested in the laboratory and on patients to
determine levels of toxicity. Phase III clinical trials are often done at
multiple treatment sites so that enough patients can be enrolled to make the
findings valid. There are usually strict requirements for acceptance, and extra
testing and record keeping is done for evaluating progress. The treatment
protocols, both standard and experimental, are designed by experts in the
field, based on the latest findings. Patients are closely monitored for
symptoms and side effects, so medical care is generally excellent. If possible,
the study is "blind," so that raised expectations of the experimental treatment
don't confound the findings. So that the results can be scientifically valid,
patients are typically randomly assigned by a computer to the different study
groups, or "arms." This is the best way to rule out differences in outcome that
may be due to other factors. Sometimes, in smaller studies, patients are
matched or "case controlled" instead, so that very similar case histories can
Participants in a clinical trial must give their informed consent based on
extensive, understandable information and explanation, and may withdraw from a
trial at any time. Patient information is kept strictly confidential.
Most health plans do not cover experimental treatments or the associated doctor
and hospital costs. However, TRICARE/CHAMPUS, the Department of Defense health
program, has agreed to reimburse members who participate in NCI clinical
trials. The NCI along with the American Society of Clinical Oncology (ASCO) and
advocates are working with government and private health providers to insure
coverage of patient participation in high quality peer-reviewed clinical
Ask your oncologist to suggest clinical trials that may be relevant to your
case, the National Cancer Institute suggests, since he or she is likely to have
access to trial information. But be prepared to take a lot of the initiative
yourself in investigating and researching possibilities. While most doctors are
happy to do so, some may hesitate about losing their patients to a clinical
trial team. Others may be biased toward research at their own institutions.
Also, oncologists treating patients with many different kinds of cancer may not
have the time or inclination to research all available trials, and may be aware
only of the higher-profile trials.
Since clinical trials often take years to enroll enough patients and
demonstrate results, sometimes ethical problems result when it begins to look
as if an experimental drug will be more or less beneficial, or sometimes even
harmful, than the standard treatment with which it is being compared. If the
protocol being tested involves currently available substances, women will often
elect not to join a clinical trial, in which they run a fifty-fifty chance
(sometimes less, if there is more than one experimental arm of the study) of
being randomized not to receive the drug. Instead, if the drug is already
approved for other uses, they may try to find a doctor who will treat them
independently. This has happened in recent years with the proliferation of
off-trial use of high-dose chemotherapy with stem cell transplant, discussed
later in this chapter.
Clinical trials advance medical knowledge, and they also benefit patients.
Women who participate in clinical trials do so in part because they know that
the knowledge gained will help others, ultimately, and also because this is one
way of getting state-of-the-art treatment, observation and care. Most
importantly, they choose to participate because they hope to gain access to
experimental treatments at least as good as the best standard care for their
stage of disease. In her book The Activist Cancer Patient, Beverly Zakarian
advises a commonsense approach. Forget the popular myth of trial participants
as "guinea pigs," she says:
There is only one important question: Are YOUR best interests served by
participating in a clinical trial? Because a clinical trial is, practically
speaking, just another treatment option, you should evaluate the information
and make treatment choices the same way you would if it were not a
As stated earlier, the NCI CancerNet Service on the Internet is a good place to
look for experimental treatments currently in clinical trials, as is the
Centerwatch Clinical Trials Listing Service. Both web sites explain the nature
of clinical trials.3
- Joining a Clinical Trial, from the National Alliance of Breast
Cancer Organizations (NABCO) website.
- Zakarian, Beverly, The Activist Cancer Patient: How to Take Charge of
Your Treatment (New York: John Wiley and Sons, 1996), p. 120.
- NCI CancerNet Clinical Trials information and
Trials Listing Service.
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