Targeted molecular therapies, sometimes called "designer drugs," are moving onto the frontline of cancer treatments because they target cancer cells specifically.
Targeted molecular therapy is emerging as an important new path in the chemical treatment of many forms of cancer. The idea behind it is simple. So-called “designer drugs” are used to kill cancer cells, or at least significantly slow their growth, without killing healthy cells in the process.
How targeted molecular therapy works
The key to targeted molecular therapy is identifying the specific genes or proteins in a cell that prompt the cell to mutate, or change. Once those cell components are known, it then becomes possible to find ways to eliminate them without the collateral damage of killing healthy cells at the same time.
Some scientists refer to the protein “flags” in cells that targeted molecular therapy drugs are designed to counteract or stimulate as the “Achilles’ heel” of cancer. Many see these designer drugs of holding the potential to prove effective in the treatment of even advanced cancers.
The central problem is in the enormous variety of cancer types and cancer cells, which makes the identification of the harmful genes more complicated. Once identified, however, targeted therapy treatment makes it possible for a drug to kill the cancer cell, limit its life span, or turn off the signal that tells the cell to grow.
Targeted therapies work in a number of ways, with different goals and different forms of administration. Monoclonial antibodies, which are usually given intravenously, target the outside of cancer cells or the area around a cancer tumor. Sometimes these antibodies actually kill cancer cells; in other cases, they help direct chemotherapy or radiation therapy to more specifically target the harmful cells.
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The targeted therapy drugs
Small-molecule drugs, usually taken in pill form, block the cancer cell’s means of growing and spreading. Some do so by preventing the tissue around a tumor from making the blood vessels it needs to carry essential nutrients to the tumor.
Some of these drugs change the proteins in cancer cells, which kills the cells. Others bring toxins to kill cancer cells but not healthy ones. Still others trigger the immune system to kill cancerous cells.
Targeted therapies are now being used in the treatment of, among other cancer types, breast cancer, lung cancer, colon cancer, and a number of melanomas. Sometimes they are used alone, but they are usually administered in combination with other chemotherapy drugs, as well as with radiation therapy and surgery.
Targeted therapy downsides and side effects
Like other chemotherapy drugs, “designer” targeted therapy drugs may have side effects, and many do—although they tend to be problems with skin, nails and eyes, rather than the more common side effects of standard chemotherapy drugs. Also, the science involved in developing them generally makes them more expensive.
Two factors that complicate targeted therapies are that they only work in tumors in which a target can be identified. Even when a target is identified, it is not a given that the tumor will respond to the specific drug.
As usual, testing is critical. Your doctor will need to know as much as possible about the specific genes and proteins in your tumor, as well as about some of its other qualities.
No one disputes the importance of targeted therapies, and many doctors and researchers call them the next “revolution” in cancer treatment. Speaking of the drug glivec (generic name imatinib) in the treatment of some forms of leukemia (although it is also used in the treatment of other cancers), Paul A. Bunn Jr., of the American Society of Clinical Oncology, commented, “This is as important as it gets. A cancer-specific target, a drug specifically designed for the target, the most effective agent ever. Read my lips. This is real, not mice.”
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