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Targeted therapy

As the name suggests, targeted therapy drugs are designed to attack specific targets, such as receptors or proteins, found on and in cancer cells.  The goal with targeted therapy is to stop or slow tumor growth by attacking genetic features on cells that regulate growth and division. These drugs may also help other cancer treatments, such as chemotherapy, work better.

While chemotherapy targets fast-growing cells throughout the body, whether they are cancerous or not, targeted therapy directs drugs to specific features on cancer cells that may be identified through advanced genomic testing or other laboratory tests or diagnostic procedures.

Types of targeted therapy

There are many different types of targeted therapy, and some may fit into more than one category. The most common types are:

Monoclonal antibodies

Monoclonal antibodies are a type of treatment strategy that uses engineered molecules designed to attack specific features found on cancer cells. Monoclonal antibodies may be used either alone to destroy cancer cells, or as carriers of other substances delivered either for treatment or diagnostic purposes. For example, chemotherapeutic agents or radioactive substances can be attached to monoclonal antibodies to deliver them directly to the tumor cells. Some more specific examples of targeted therapy, including small-molecule drugs and immunotherapy, may fit into this category.

Learn more about monoclonal antibodies

Small-molecule drugs

These drugs target molecules that have a low molecular weight and, because of their size, are better able to penetrate cells and find their intended target. Some EGFR inhibitors, angiogenesis inhibitors and apoptosis inducers are examples of small-molecule drugs.


Immunotherapy is a broad category of treatments designed to jump start the immune system to better recognize and attack cancer cells. Some immunotherapy treatments are designed to target communication between certain receptors either on cancer cells or immune cells. By disrupting this communication, immune cells may no longer be able to hide from the immune system, leaving them more vulnerable to an immune response. Checkpoint inhibitor drugs are an example of immunotherapy.

Learn more about immunotherapy

Hormone therapy

Some cancers need hormones to grow. Hormone therapies are designed to target a specific hormone, often estrogen, and reduce the amount of that hormone in the body, slow the production of new hormones or block them from feeding cancer cells.

Learn more about hormone therapy

Examples of targeted therapy

Angiogenesis inhibitors: Angiogenesis is the process by which cells create new blood vessels. When tumors reach a certain size, they use proteins called growth factor receptors to tap into the body's vascular system to create a steady flow of blood that may help fuel tumor growth. These drugs may be used to stop cancer cells from developing new blood supplies.

Apoptosis inducers: When cells become old or damaged, they are designed to kill themselves, a process called apoptosis. Some cancer cells resist apoptosis and continue to grow and produce more damaged cells. These drugs are intended to help restore the apoptosis functionality.

Checkpoint inhibitors: Cancer cells and immune cells use protein receptors to exchange signals at so-called checkpoints. These signals help immune cells differentiate healthy cells from cancer cells. These drugs are designed to disrupt those signals and expose the cancer cells for attack.

Learn more about checkpoint inhibitors

EGFR inhibitors: Epidermal growth factor receptor, a protein that helps regulate cell growth, may be found in high levels on cancer cells. These drugs are designed to block EGFR proteins and help slow tumor growth.

mTOR inhibitors: The mammalian target of rapamycin (mTOR) is a protein that helps regulate cell division and proliferation. These drugs target the mTOR signaling pathway and may help slow the division of cells in a tumor.

PARP inhibitors: Cells—whether they are healthy or cancerous—can monitor damage with a protein called poly ADP-ribose polymerase (PARP) and summon genes to repair the damage. PARP inhibitors are designed to stop the repair process in cancer cells, leaving the damaged cells to die.

Proteasome inhibitors: These drugs are designed to stop cancer cells from recycling old proteins into new ones, leading to a toxic overload of proteins that may kill the cell. Proteasomes inhibitors also are thought to increase levels of proteins that promote cell death.

Signal transduction inhibitors: Human cells are constantly sending information back and forth to help regulate cell behavior, including growth and division. Signal transduction inhibitors are designed to block those signals, slowing growth or killing the cells.

Tyrosine kinase inhibitors: Among the many kinase inhibitors, these drugs block signals, sent to a cell's nucleus, that may cause the cell to grow or multiply. Tyrosine kinase enzymes are often hyperactive in cancer cells and, when activated by a signaling molecule, may trigger uncontrolled growth or cell division.

Types of cancer treated by targeted therapy

The U.S. Food and Drug Administration (FDA) has approved targeted therapy drugs for many cancers, including the most common types, such as:

The FDA also has approved some targeted therapies to treat cancers found with specific genetic features, no matter where in the body they originated. So-called agnostic cancers, these include tumors with features such as:

How are targeted therapy drugs delivered?

How and where targeted drugs may be given depends on many factors, including clinical considerations and personal preferences. You may receive your targeted therapy in a clinic or outpatient care center, at a hospital or doctor’s office or at home.

Targeted therapy drugs may be administered:

Orally: In pill, capsule or liquid form, taken by mouth, usually at home

By injection: Delivered through a needle into a vein, muscle or under the skin

By infusion: Delivered into a vein through an intravenous drip, a process that may take several hours

Patients who may require frequent injections or infusions may opt to get a port or catheter through which drugs may be administered more efficiently and with less pain. How your targeted therapy drugs are administered depends on a variety of factors, and may also depend on the type of drug prescribed. For instance:

Targeted therapy drugs are powerful and may cause side effects in caregivers and family members who come in contact with them. If you’re taking a targeted therapy at home, make sure to be aware of special handling, storage and disposal requirements. Some drugs taken at home may cost more than comparable drugs given by injection or infusion. Talk to your care team and your insurance provider to determine costs and coverage for these drugs.

Targeted therapy drugs may be given in combination with other cancer treatments, such as surgery, chemotherapy or radiation therapy. At Cancer Treatment Centers of America® (CTCA), our oncologists will work closely with you and the rest of your care team to discuss targeted therapy and other treatment options based on your specific cancer type and individual needs.

What to expect

There are dozens of targeted therapy drugs designed to treat different cancers, and every targeted therapy plan is tailored to a patient’s specific cancer and treatment goals. Each drug has different dosage requirements and may produce different side effects. What to expect from targeted therapy may vary widely depending on many factors, including the type of drug prescribed, your overall health and whether your targeted therapy is part of a plan that includes other treatments, such as radiation therapy or surgery. If targeted therapy is part of your treatment plan, your care team will talk to you about your options and what to expect.

If you choose to administer your own targeted therapy drugs, talk to your doctor about safety protocols for handling drugs. If infusion or injection is the recommended option, your care team will tell you when and where to go and how long the treatments will take.

Before targeted therapy

Some targeted therapies, especially checkpoint inhibitors, must be given by infusion, which may take several hours. You may want to prepare for a long visit to the hospital, outpatient care center or wherever your infusion is taking place.

Make yourself comfortable: Wear loose-fitting clothing to allow access to your port or catheter. Consider bringing a blanket or pillow.

Bring a buddy: Consider bringing a friend or family member to keep you company and to drive you to and from your appointment.

Keep busy: Bring a tablet or book to read or watch a program during your session.

Bring a snack: Make sure to have some water and a light bite to eat during your session.

After targeted therapy

Expect to follow up with your medical oncologist or other members of your care team during and after your targeted therapy regimen.

Monitor side effects: Contact your care team if you’re concerned about developing side effects or if they become severe.

Stay hydrated: Make sure to eat a healthy diet and drink plenty of fluids while taking targeted therapy drugs.

If you have a medical emergency, dial 911.

Questions to ask

You may have many questions about your targeted therapy treatments. Don’t hesitate to ask your oncologist or other members of your care team questions about what to expect from your treatment. Some questions to consider include:

Protect yourself and others

Cancer drugs, such as those used in targeted therapy and chemotherapy, may be passed on to others through bodily fluids, including mucus, sweat, tears, semen, urine, vomit or stool. These drugs should be handled only by the patient taking them. Some must be kept in the bottle or box in which they came and cannot be transferred into a daily dosage container. Some drugs require the patient to wear gloves while handling.

Keep bathrooms clean and immediately wash towels or clothing that may have fluids on them. Caregivers should wash hands frequently and wear gloves when necessary.

Throughout your treatment, your medical oncologist will monitor the progress of your chemotherapy regimen and modify your treatment plan accordingly.

Managing side effects

Targeted therapy drugs are designed to bypass healthy cells, a process intended to produce fewer side effects. However, some targeted therapy drugs may influence cell function and produce side effects. Skin issues are among the most common side effects of targeted therapy. They include:

Other side effects of targeted therapy include:

Some side effects of targeted may fade quickly after treatment ends. Other side effects may linger for weeks. It’s important to tell your care team how the side effects are affecting you and which side effects may be improving or getting worse.

At CTCA®, our medical oncologists work closely with integrative care providers who offer services intended to help manage side effects and improve your quality of life. Supportive care services that may be helpful to targeted therapy patients may include:

Clinical trials

Clinical trials are a key testing ground for determining the effectiveness and safety of new treatments and drugs for many diseases, including cancer. Many targeted therapy drugs are in clinical trials, including brand-new drugs that have not yet been approved by the FDA, as well as approved drugs that are in trials to treat cancers for which they are not yet approved.

As part of our commitment to providing new and innovative treatments, our oncologists may recommend that cancer patients enroll in carefully selected clinical trials. If you meet specific criteria, clinical trials may offer cancer treatment options that would otherwise be unavailable to you.

Each patient is considered for a clinical trial on an individual basis and may qualify at any stage of disease or treatment. Talk to your doctor about whether a clinical trial is a good option for you and ask about the risks, benefits and various requirements involved.

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