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Overview

What Is Targeted Therapy?

Targeted therapy is a cancer treatment that uses information about a tumor’s genes, proteins, or other molecular features to guide drug selection. These medicines are designed to act on specific changes that help cancer cells grow, divide, or spread. Depending on the medicine and cancer, treatment may slow cancer growth, stop it, or destroy targeted cancer cells, often with a different pattern of effects than less selective treatments.

Which Conditions It Treats

Which Conditions Does Targeted Therapy Treat?

Targeted therapy can be used for selected lung, breast, colorectal, kidney, ovarian, blood, and other cancers when testing identifies a relevant treatment target. The cancer type describes where the cancer began, while biomarker status describes molecular features that may determine whether a particular drug is likely to help. For example, not every lung cancer is eligible for the same lung cancer therapy, and treatment decisions depend on the tumor’s test results.

  • EGFR or ALK alterations in some non-small cell lung cancers may be treated with medicines that block those signaling pathways.
  • HER2 overexpression in some breast or gastric cancers may make HER2-directed treatment an option.
  • BRAF changes in selected cancers may be treated with BRAF-directed medicines, sometimes combined with another pathway inhibitor.
  • BRCA-related tumors may be treated with PARP inhibitors that interfere with cancer-cell DNA repair.

Drugs Used

Drugs Used in Targeted Therapy

Targeted therapy drugs include small-molecule inhibitors and monoclonal antibodies. Small-molecule inhibitors can enter cells and block internal signaling or DNA-repair pathways, while monoclonal antibodies usually bind targets on the cell surface or in the surrounding tumor environment. Gene therapy for cancer is a separate treatment approach and should not be assumed to be interchangeable with all targeted medicines.

Drug categoryHow it actsTypical examplesCommon delivery pattern
Small-molecule inhibitorsBlock intracellular signaling or repair pathwaysEGFR, ALK, BRAF, or PARP inhibitorsOften oral, with drug-specific schedules
Monoclonal antibodiesBind targets on cancer cells or in the tumor environmentHER2- or VEGF-directed medicinesOften intravenous or subcutaneous administration

During the Procedure / What to Expect

What to Expect During Targeted Therapy

Before treatment, the oncology team confirms the treatment target and reviews your medicines, allergies, and health conditions. The prescribed drug may be taken by mouth or given by infusion, and monitoring can include symptom reviews, physical examinations, blood tests, and sometimes imaging. The schedule depends on the medicine and may continue over repeated treatment cycles.

How treatment is given

Oral medicines may be taken daily or at intermittent intervals at home, while infused medicines are commonly given in an outpatient infusion center or clinic. Premedication may be used when needed to reduce the chance of an infusion reaction. Treatment may involve one medicine or a combination, and following the prescribed schedule is important; do not change or stop treatment without speaking with the oncology team.

Side Effects & Risks

Side Effects and Risks

Side effects depend on the specific medicine, the molecular target, the dose, and other treatments being used. Although targeted therapies are designed to act more selectively than traditional chemotherapy, they can still affect healthy tissues and cause important complications. Your oncology team can explain which effects are most relevant to your cancer therapy and how they will be monitored.

  • Skin effects may include an acne-like rash, dry or itchy skin, redness, or changes around the nails.
  • Gastrointestinal effects may include diarrhea, nausea, appetite changes, or mouth sores.
  • General effects may include fatigue, weakness, and reduced appetite.
  • Some medicines can cause high blood pressure, liver problems, bleeding, or abnormal clotting.
  • Infused medicines may cause infusion or allergic reactions during or shortly after treatment.

Safety warning

Report serious symptoms promptly

Contact your oncology team promptly for trouble breathing, facial or throat swelling, severe diarrhea or dehydration, chest pain, unusual bleeding, a severe rash, yellowing of the skin or eyes, or fever during treatment. If symptoms are severe or rapidly worsening, seek emergency care.

Recovery

Recovery and Follow-Up

Targeted therapy usually involves repeated treatment and monitoring rather than one recovery period. Rest as needed, drink fluids unless your clinician has advised otherwise, eat nutritious foods as tolerated, and use recommended skin care when relevant. Keep follow-up appointments and take oral medicines exactly as prescribed, including on days when you feel well.

How response is monitored

Follow-up may include clinic visits, blood tests, imaging, and review of symptoms and daily functioning. These assessments help clinicians determine whether the cancer is responding and whether side effects are manageable. Based on the findings, the team may continue, adjust, pause, or change the cancer therapy.

When to Call a Doctor

When to Call a Doctor

Contact your care team

Warning signs during targeted therapy

Contact your oncology team promptly for a new or worsening rash, persistent diarrhea or vomiting, inability to drink, fever, unusual bleeding, severe weakness, yellow skin or eyes, or markedly reduced urination. Do not wait for the next scheduled visit if these symptoms develop.

Emergency symptoms

Get urgent medical help

Seek emergency care for trouble breathing, swelling of the face or throat, severe chest pain, sudden confusion, fainting, or signs of a severe allergic or cardiac reaction. If possible, tell emergency staff which targeted medicines you are taking.

Diseases & Conditions

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