Therapies that target intracellular pathways are mostly oral therapies because

Whereas chemotherapy induces apoptosis by interfering with cell division of both cancerous and healthy cells, targeted therapy exploits targets, proteins, enzymes, or genes specific to malignant cells through a variety of mechanisms of action, which helps prevent drug resistance. The agents work by either inhibiting angiogenesis, blocking chemical signals that tell cells to divide or carry out normal function, or delivering toxic substances to a cell.

Extracellular Versus Intracellular

Some targeted agents capitalize on extracellular components of target receptors, and others work within the intracellular environment target to interfere with processes inside a malignant cell. 

Extracellular targeted therapies: Tumor-associated antigens act as a beacon on the outer surface of cancer cells, calling attention to malignant cells for better specificity. Such activity has been likened to a “lock and key effect.” For a tumor-associated antigen to be most susceptible to targeted therapy, the antigen should be specific to cancer cells only, located on the surface of tumor cells, present in large numbers, and critical to tumor cell survival. Some monoclonal antibodies are classified as extracellular targeted therapies.  

Intracellular targeted therapies: Other ways to alter and interfere with processes in malignant cells involve signal transduction or blood vessel formation that are directly involved in cancer cell proliferation and tumor growth. Examples of agents against these targets include EGFR inhibitors (e.g., cetuximab, erlotinib), HER2 inhibitors (e.g., trastuzumab, pertuzumab), and tyrosine kinase inhibitors (e.g., imatinib, dasatinib).

Side-Effect Profiles and Management

As with other pharmacologic therapies, expected side effects are related to the mechanism of action. Understanding how an agent works, the target it exploits, or the cellular process it interrupts speaks volumes about what we can expect our patients to experience and strategies to prevent complications. 

For monoclonal antibodies—agents that end in “mab”—the nomenclature provides clinicians with indicators as to which side effects to anticipate. The middle of the generic name is indicative of its effects, lending insight into where to expect side effects. With pertuzumab or rituximab, the “tu” indicates the effect is right at the tumor and patients may experience pain at the tumor site. With bevacizumab, the “ci” indicates that the cardiovascular system is affected and patients can expect circulation disruption resulting in hypertension or impaired wound. See Figure 1 for more information about drug nomenclature.

Depending on the agent used, expected side effects can be an indicator that the drug is working and the therapy does not need to be altered. For example, with erlotinib, acute dermatologic reactions are expected and serve as an indicator that therapy is effective. A collaborative approach to interprofessional management and supportive care can ensure symptoms are controlled while allowing therapy to continue. 

Oral Adherence

Many targeted agents, especially those working on intracellular activities, are given orally. ONS provides various resources to help clinicians promote oral adherence in their practice, including patient education videos and the Oral Adherence Toolkit.

Research suggests that using targeted therapies in combination with chemotherapy, radiation, and immunotherapy is most effective, and advancements in genetic profiling and molecular testing have allowed for better patient selection for targeted therapies. As we understand more about somatic genetic mutations and next-generation sequencing, we are better able to predict whether adding targeted therapies to treatment regimens will improve outcomes. 

Nurses are becoming increasingly involved with educating patients and caregivers about the benefits and intricacies of targeted therapies, the role of genetic and tumor testing in determining a treatment plan, and the survivorship issues related to the agents.

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What is targeted drug therapy?

Targeted drug therapy is the use of medicines that target or are directed at proteins on cancer cells that help them grow, spread, and live longer. Targeted drug therapy can be used to treat oral cavity and oropharyngeal cancers by destroying cancer cells or slowing their growth. Their side effects are different from chemotherapy (sometimes less severe) and many are taken as a pill.

Some targeted drugs, for example, monoclonal antibodies, work in more than one way to control cancer cells and may also be considered immunotherapy because they boost the immune system.

Drug that targets cancer cells with EGFR changes

Epidermal growth factor receptor (EGFR) is a protein that helps cancer cells grow. Drugs that target EGFR can be used to treat some oral cavity or oropharyngeal cancers.

Cetuximab for oral cavity or oropharyngeal cancers

Cetuximab (Erbitux) is a monoclonal antibody, which is a man-made version of an immune system protein. It targets the epidermal growth factor receptor (EGFR) protein on the surface of certain cells that helps cells grow and divide. Oral cavity and oropharyngeal cancer cells often have higher than normal amounts of EGFR. By blocking EGFR, cetuximab can help slow or stop cancer cell growth.

Cetuximab can be combined with radiation therapy for some earlier stage cancers. For more advanced cancers, it may be combined with standard chemo drugs such as cisplatin, or it may be used by itself.

Cetuximab is given by infusion into a vein (IV), either once a week or every other week.

Possible side effects of targeted drug therapy

Many people develop skin problems such as an acne-like rash on the face and chest during treatment, which in some cases can lead to infections. Cetuximab can make your skin very sensitive to the sun, so you'll need to protect your skin while getting treatment and for at least months after treatment. Other side effects may include headache, tiredness, fever, and diarrhea.

A rare but serious side effect of cetuximab is an allergic reaction during the first infusion, which could cause problems with breathing and low blood pressure. You may be given medicine before treatment to help prevent this.

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  • References

Leeman JE, Katabi N, Wong RJ, Lee NY and Romesser PB. Ch. 65 - Cancer of the Head and Neck. In: Niederhuber JE, Armitage JO, Doroshow JH, Kastan MB, Tepper JE, eds. Abeloff’sClinical Oncology. 6th ed. Philadelphia, Pa. Elsevier; 2020.

Mendenhall WM, Dziegielewski PT, and Pfister DG. Chapter 45- Cancer of the Head and Neck. In: DeVita VT, Lawrence TS, Rosenberg SA, eds. DeVita, Hellman, and Rosenberg’s Cancer: Principles and Practice of Oncology. 11th ed. Philadelphia, Pa: Lippincott Williams & Wilkins; 2019. 

National Comprehensive Cancer Network (NCCN). NCCN Clinical Practice Guidelines in Oncology. Head and Neck Cancers. Version 2.2020 -- June 09, 2020. Accessed at www.nccn.org/professionals/physician_gls/pdf/head-and-neck.pdf on September 21, 2020.

Last Revised: March 23, 2021

American Cancer Society medical information is copyrighted material. For reprint requests, please see our Content Usage Policy.

Treating Oral Cavity and Oropharyngeal Cancer

  • Surgery for Oral Cavity and Oropharyngeal Cancer
  • Radiation Therapy for Oral Cavity and Oropharyngeal Cancer
  • Chemotherapy for Oral Cavity and Oropharyngeal Cancer
  • Targeted Therapy for Oral Cavity and Oropharyngeal Cancer
  • Immunotherapy for Oral Cavity and Oropharyngeal Cancer
  • Palliative Treatment for Oral Cavity and Oropharyngeal Cancer
  • Treatment Options for Oral Cavity Cancer by Stage
  • Treatment Options for Oropharyngeal Cancer by Stage

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What is oral targeted therapy?

Targeted drug therapy is the use of medicines that target or are directed at proteins on cancer cells that help them grow, spread, and live longer. Targeted drug therapy can be used to treat oral cavity and oropharyngeal cancers by destroying cancer cells or slowing their growth.
Help the immune system destroy cancer cells. Certain targeted therapies can mark cancer cells so it is easier for the immune system to find and destroy them. Other targeted therapies help boost your immune system to work better against cancer.

What are the two most common types of targeted therapy for cancer?

The two main groups of targeted therapy drugs are monoclonal antibodies and small molecule inhibitors.

How is targeted therapy different from chemotherapy?

Targeted drugs often work by blocking cancer cells from copying themselves. This means they can help stop a cancer cell from dividing and making new cancer cells. Traditional chemotherapy, however, kills cancer cells that have already been made.