Targeted cancer therapies are drugs or other substances that block the growth and
spread of cancer by interfering with specific molecules involved in tumor growth
and progression. Because scientists often call these molecules “molecular targets,”
targeted cancer therapies are sometimes called “molecularly targeted drugs,” “molecularly
targeted therapies,” or other similar names. By focusing on molecular and cellular
changes that are specific to cancer, targeted cancer therapies may be more effective
than other types of treatment, including chemotherapy and radiotherapy, and less
harmful to normal cells.
Many targeted cancer therapies have been approved by the U.S. Food and Drug Administration
(FDA) for the treatment of specific types of cancer. Others are being studied in
clinical trials (research studies with people), and many more are in preclinical
testing (research studies with animals).
Targeted cancer therapies are being studied for use alone, in combination with other
targeted therapies, and in combination with other cancer treatments, such as chemotherapy.
Targeted cancer therapies interfere with cancer cell division (proliferation) and
spread in different ways. Many of these therapies focus on proteins that are involved
in cell signaling pathways, which form a complex communication system that governs
basic cellular functions and activities, such as cell division, cell movement, how
a cell responds to specific external stimuli, and even cell death. By blocking signals
that tell cancer cells to grow and divide uncontrollably, targeted cancer therapies
can help stop cancer progression and may induce cancer cell death through a process
known as apoptosis. Other targeted therapies can cause cancer cell death directly,
by specifically inducing apoptosis, or indirectly, by stimulating the immune system
to recognize and destroy cancer cells and/or by delivering toxic substances to them.
The development of targeted therapies, therefore, requires the identification of
good targets—that is, targets that are known to play a key role in cancer cell growth
and survival. (It is for this reason that targeted therapies are often referred
to as the product of “rational drug design
Targeted cancer therapies have been developed that interfere with a variety of other
cellular processes. There are currently several FDA-approved targeted therapies
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Adapted from the National Cancer Institute's PDQ Database: http://www.cancer.gov/cancertopics/factsheet/Therapy/targeted.
(Accessed August 2013)