What Is Molecular Targeted Therapy?

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  • Written By: Mary McMahon
  • Edited By: Shereen Skola
  • Last Modified Date: 08 October 2019
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Molecular targeted therapy is a cancer treatment that attacks particular molecules. This highly specific treatment interrupts the process of cell division to stop the proliferation of cancerous cells inside a patient. Numerous cancer drugs utilize this technique and pharmaceutical companies constantly have more in development to expand the arsenal of treatment options. Research and development of a given therapy can take over a decade in some cases.

The first step in the creation of molecular targeted therapy is the identification of a specific molecule to target. These molecules may be inside or outside the cells. Researchers can use tools like complex chemical analysis, DNA sequencing, and research on cells in culture to find molecules attached to particular cancers. They seek out unique molecules not found elsewhere in the body so they can focus on these structures in treatment and avoid collateral damage to healthy tissues.

Once researchers have a target, they can develop medications that will attach to the given molecule. This can include drugs that operate on the molecular level inside the cell as well as monoclonal antibodies that can attack molecules found on the cell surface. In this phase of development, researchers working on molecular targeted therapy need to be able to reliably pinpoint the desired molecules without danging other tissues. If a chemical compound appears promising, they can start testing to see how it performs in living organisms.


Many medications fail along the way because they don't work, cause unacceptable side effects, or perform unexpectedly. Once a molecular targeted therapy is cleared for use, it can be prescribed for cancer patients as part of a treatment plan. A doctor may recommend surgery in an effort to remove as many of the cancerous cells as possible, usually followed by chemotherapy to combat the remaining cancer. The molecular targeted therapy limits the chance of relapse by halting uncontrolled cell growth and preventing the recurrence of the cancer.

These drugs can be highly specific. Molecular targeted therapy starts with research on particular types of cancers to find rogue molecules that may make good targets, which means the drugs are not useful for broad spectrum chemotherapy. A drug developed to treat a brain cancer, for instance, may have no effect on cancers in the lungs, because the molecular structure of the disease is different. Researchers work with a variety of cancers and tend to focus on those which are most common in the process of developing new treatments.


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