NCCN Guidelines for Patients
: Caring for Adolescents and Young Adults
Part 5: Understanding your treatment options
If you had been diagnosed with cancer a decade ago,
your treatment options would have been more limited
than they are today. Thanks to technical improvements
and research on how and why cancer develops, doctors
can now prescribe many therapies that target cancer with
greater accuracy than ever before. Understanding these
therapies can help you work with your treatment team
and make informed decisions about your treatment plan.
A clinical trial is a research study that tests a new
treatment to see if it is safe and effective. Treatments that
have been approved as a result of clinical trials are said
to be “evidence-based”—meaning that they have been
shown to have an effect on cancer and that any risks are
outweighed by the treatment’s beneficial effects.
Clinical trials are often the best option for cancer
treatment, particularly for AYA patients. For one thing,
you are certain to receive excellent care. All clinical trials
are designed to compare the new treatment against the
current standard of care, so even patients who aren’t
receiving the experimental drug will get the most cutting-
edge treatment available. Second, you will be treated by
experts. Third, the results of your treatment—both good
and bad—will be carefully tracked. Fourth, you may help
other patients with cancer.
Clinical trials may have risks too. Like any test or
treatment, there may be side effects. Also, there is
always a possibility that the new treatment might not
help. Another downside may be that paperwork or more
trips to the hospital are needed.
Phases of clinical trials
New cancer treatments aren’t offered to the public as
soon as they’re created. They need to go through a
series of trials to make sure they’re safe and work.
Clinical trials have four phases. They are labeled with
Roman numerals I–IV.
trials test a drug’s safety and determine the
best dose of a new drug with the fewest side effects.
The drug has already been tested in lab and animal
studies, but now it needs to be tested in patients.
Doctors start by giving very low doses of the drug to
the first group of patients. Higher doses are given
to the next groups until side effects appear or the
desired effect is seen. If a drug is found to be safe
enough, it can be tested in a phase II clinical trial.
Phase I trials are usually offered to patients only if
standard therapies have not worked, or if there is
no known effective therapy available to treat that
trials assess if a drug works for a specific
type of cancer. They are performed in larger groups
of patients for whom standard treatments aren’t
working. Often, new combinations of drugs are
tested. Patients are closely watched to see if the
drug works. The treatment team also looks for side
effects. If a drug is found to work, it can be tested in
a phase III clinical trial.