Overview of Targeted Therapies for Cancer

Targeted cancer therapies are drugs designed to interfere with specific molecules necessary for tumor growth and progression. Traditional cytotoxic chemotherapies usually kill rapidly dividing cells in the body by interfering with cell division. A primary goal of targeted therapies is to fight cancer cells with more precision and potentially fewer side effects.

Classification and naming

Targeted cancer agents are broadly classified as either monoclonal antibodies or small molecules.

Therapeutic monoclonal antibodies target specific antigens found on the cell surface, such as transmembrane receptors or extracellular growth factors. In some cases, monoclonal antibodies are conjugated to radio-isotopes or toxins to allow specific delivery of these cytotoxic agents to the intended cancer cell target.

Small molecules can penetrate the cell membrane to interact with targets inside a cell. Small molecules are usually designed to interfere with the enzymatic activity of the target protein.

As with any drug, targeted cancer therapies typically have several different names. One or more names is use to designate the chemical compound during development; if successful, the drug receives a generic name and then a brand name used by the pharmaceutical company for marketing. For example, the small molecule STI-571 became known as imatinib (generic name) and is marketed by Novartis under the brand name Gleevec™.

The name of a targeted agent provides clues to the type of agent and its cellular target. 

Monoclonal antibodies end with the stem "-mab" (monoclonal antibody). Small molecules end with the stem "-ib" (indicating that the agent has protein inhibitory properties). Monoclonal antibodies have an additional substem designating the source of the compound, e.g., "-ximab" for chimeric human-mouse antibodies, "-zumab" for humanized mouse antibodies, and "-mumab" for fully human antibodies. Both monoclonal antibodies and small molecules contain an additional stem in the middle of the name describing the molecule’s target; examples for monoclonal antibodies include "-ci-" for a circulatory system target and "-tu-" for a tumor target, while examples for small molecules include "-tin-" for tyrosine kinase inhibitors and "-zom-" for proteasome inhibitors. At the beginning of the generic name is a prefix that is unique for each agent. Some examples include:

Monoclonal antibodies
bevacizumab humanized monoclonal antibody with a circulatory system target (VEGF-A)
cetuximab chimeric monoclonal antibody with a tumor target (EGFR)
ipilimumab fully human antibody with an immune system target (CTLA-4)
Small molecules
bortezomib small molecule proteasome inhibitor
imatinib small molecule tyrosine kinase inhibitor
seliciclib small molecule cyclin-dependent kinase inhibitor

The FDA has approved multiple targeted drug cancer therapies, and many more are being studied in clinical trials either alone or in combination with other treatments. A partial list of currently approved targeted therapies for solid malignancies and their molecular targets is provided below.

Agent Target(s) FDA-approved indication(s)
Ado-trastuzumab emtansine (Kadcyla) HER2 (ERBB2/neu)
  • Breast cancer (HER2+)
Afatinib (Gilotrif) EGFR (HER1/ERBB1), HER2 (ERBB2/neu)
  • Non-small cell lung cancer (with EGFR exon 19 deletions or exon 21 substitution (L858R) mutations)
Aldesleukin (Proleukin)
  • Renal cell carcinoma
  • Melanoma
Alemtuzumab (Campath) CD52
  • B-cell chronic lymphocytic leukemia
Axitinib (Inlyta) KIT, PDGFRβ, VEGFR1/2/3
  • Renal cell carcinoma
Belimumab (Benlysta) BAFF
  • Lupus erythematosus
Bevacizumab (Avastin) VEGF ligand
  • Colorectal cancer
  • Glioblastoma
  • Non-small cell lung cancer
  • Renal cell carcinoma
Bortezomib (Velcade) Proteasome
  • Multiple myeloma
  • Mantle cell lymphoma
Bosutinib (Bosulif) ABL
  • Chronic myelogenous leukemia (Philadelphia chromosome positive)
Brentuximab vedotin (Adcetris) CD30
  • Hodgkin lymphoma
  • Anaplastic large cell lymphoma
Cabozantinib (Cometriq) FLT3, KIT, MET, RET, VEGFR2
  • Medullary thyroid cancer
Canakinumab (Ilaris) IL-1β
  • Juvenile idiopathic arthritis
  • Cryopyrin-associated periodic syndromes
Carfilzomib (Kyprolis) Proteasome
  • Multiple myeloma
Cetuximab (Erbitux) EGFR (HER1/ERBB1)
  • Colorectal cancer (KRAS wild type)
  • Squamous cell cancer of the head and neck
Crizotinib (Xalkori) ALK, MET
  • Non-small cell lung cancer (with ALK fusion)
Dabrafenib (Tafinlar) BRAF
  • Melanoma (with BRAF V600 mutation)
Dasatinib (Sprycel) ABL
  • Chronic myelogenous leukemia (Philadelphia chromosome positive)
  • Acute lymphoblastic leukemia (Philadelphia chromosome positive)
Denosumab (Xgeva) RANKL
  • Giant cell tumor of the bone
Erlotinib (Tarceva) EGFR (HER1/ERBB1)
  • Non-small cell lung cancer
  • Pancreatic cancer
Everolimus (Afinitor) mTOR
  • Pancreatic neuroendocrine tumor
  • Renal cell carcinoma
  • Nonresectable subependymal giant cell astrocytoma associated with tuberous sclerosis
  • Breast cancer (HR+, HER2-)
Gefitinib (Iressa) EGFR (HER1/ERBB1)
  • Non-small cell lung cancer with known prior benefit from gefitinib (limited approval)
Ibritumomab tiuxetan (Zevalin) CD20
  • Non-Hodgkin's lymphoma
Ibrutinib (Imbruvica) BTK
  • Mantle cell lymphoma
  • Chronic lymphocytic leukemia
Imatinib (Gleevec) KIT, PDGFR, ABL
  • GI stromal tumor (KIT+)
  • Dermatofibrosarcoma protuberans
  • Multiple hematologic malignancies including Philadelphia chromosome-positive ALL and CML
Ipilimumab (Yervoy) CTLA-4
  • Melanoma
Lapatinib (Tykerb) HER2 (ERBB2/neu), EGFR (HER1/ERBB1)
  • Breast cancer (HER2+)
Nilotinib (Tasigna) ABL
  • Chronic myelogenous leukemia (Philadelphia chromosome positive)​
Obinutuzumab (Gazyva) CD20
  • Chronic lymphocytic leukemia
Ofatumumab (Arzerra, HuMax-CD20) CD20
  • Chronic lymphocytic leukemia
Panitumumab (Vectibix) EGFR (HER1/ERBB1)
  • Colorectal cancer (KRAS wild type)
Pazopanib (Votrient) VEGFR, PDGFR, KIT
  • Renal cell carcinoma
Pertuzumab (Perjeta) HER2 (ERBB2/neu)
  • Breast cancer (HER2+)
Ponatinib (Iclusig) ABL, FGFR1-3, FLT3, VEGFR2
  • Chronic myelogenous leukemia
  • Acute lymphoblastic leukemia (Philadelphia chromosome positive)
Regorafenib (Stivarga) KIT, PDGFRβ, RAF, RET, VEGFR1/2/3
  • Colorectal cancer
  • Gastrointestinal stromal tumors
Rituximab (Rituxan, Mabthera) CD20
  • Non-Hodgkin’s lymphoma
  • Chronic lymphocytic leukemia
  • Rheumatoid arthritis
  • Granulomatosis with polyangiitis
Romidepsin (Istodax) HDAC
  • Cutaneous T-cell lymphoma
  • Peripheral T-cell lymphoma
Ruxolitinib (Jakafi) JAK1/2
  • ​Myelofibrosis
Sipuleucel-T (Provenge)
  • Prostate cancer
Sorafenib (Nexavar) VEGFR, PDGFR, KIT, RAF
  • Hepatocellular carcinoma
  • Renal cell carcinoma
  • Thyroid carcinoma
Temsirolimus (Torisel) mTOR
  • Renal cell carcinoma
Tocilizumab (Actemra) IL-6R
  • Rheumatoid arthritis
  • Juvenile idiopathic arthritis
Tofacitinib (Xeljanz) JAK3
  • Rheumatoid arthritis
Tositumomab (Bexxar) CD20
  • Non-Hodgkin's lymphoma
Trametinib (Mekinist) MEK
  • Melanoma (with BRAF V600 mutation)
Trastuzumab (Herceptin) HER2 (ERBB2/neu)
  • Breast cancer (HER2+)
  • Gastric cancer (HER2+)
Vandetanib (Caprelsa) EGFR (HER1/ERBB1), RET, VEGFR2
  • Medullary thyroid cancer
Vemurafenib (Zelboraf) BRAF
  • Melanoma (with BRAF V600 mutation)
Vismodegib (Erivedge) PTCH, Smoothened
  • Basal cell carcinoma
Vorinostat (Zolinza) HDAC
  • Cutaneous T-cell lymphoma
Ziv-aflibercept (Zaltrap) PIGF, VEGFA/B
  • Colorectal cancer

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Contributors: Richard G. Abramson, M.D.

Last Updated: March 3, 2014

Disclaimer: The information presented at MyCancerGenome.org is compiled from sources believed to be reliable. Extensive efforts have been made to make this information as accurate and as up-to-date as possible. However, the accuracy and completeness of this information cannot be guaranteed. Despite our best efforts, this information may contain typographical errors and omissions. The contents are to be used only as a guide, and health care providers should employ sound clinical judgment in interpreting this information for individual patient care.