Targeted Therapy

Targeted therapies are cancer treatments that stop or slow the spread of cancer by interfering with specific areas of cancer cells that are involved in the cancer cell’s growth processes. Targeted therapy is different than chemotherapy, which focuses on cells that divide and grow quickly. Chemotherapy cannot distinguish between fast-growing cancer cells and fast-growing healthy cells in the body, like cells in the digestive tract, hair, bone marrow, and blood, and can cause multiple side effects. Targeted therapy focuses on other specific features of cancer cells, in an effort to provide treatment for cancer that does less damage to normal cells and tends to cause fewer side effects.1,2

Targeted therapy is also different from chemotherapy in the way it affects cancer cells. Targeted therapies are generally cytostatic, meaning they block the growth or spread of cancer cells. Chemotherapy drugs are typically cytotoxic, meaning they kill cancer cells.2

Types of targeted therapy

There are different types of targeted therapy that are used to treat certain blood cancers, including monoclonal antibodies, kinase inhibitors, proteasome inhibitors, and histone deacetylase (HDAC) inhibitors, as well as other forms of targeted therapy.

Monoclonal antibodies

Monoclonal antibodies are created in the laboratory to identify and block cancer growth or kill cancer cells. Some monoclonal antibodies are referred to as conjugated monoclonal antibodies because they are attached to a chemotherapy drug or radioactive particle, and the monoclonal antibody delivers the chemotherapy drug or radioactive particle to cancer cells. Monoclonal antibodies used to treat certain blood cancers include:

  • Alemtuzumab (brand name: Campath®)
  • Belantamab mafodotin-blmf (brand name: Blenrep)
  • Blinatumomab (brand name: Blincyto®)
  • Brentuximab vedotin (brand name: Adcetris®)
  • Daratumumab (brand name: Darzalex®)
  • Daratumumab and hyaluronidase-fihj (brand name: Darzalex Faspro™)
  • Elotuzumab (brand name: Empliciti™)
  • Gemtuzumab ozogamicin (brand name: Mylotarg™)
  • Ibritumomab tiuxetan (brand name: Zevalin®)
  • Inotuzumab ozogamicin (brand name: Besponsa®)
  • Isatuximab-irfc (brand name: Sarclisa®)
  • Mogamulizumab-kpkc (brand name: Poteligeo®)
  • Moxetumomab pasudotox-tdfk (brand name: Lumoxiti™)
  • Nivolumab (brand name: Opdivo®)
  • Obinutuzumab (brand name: Gazyva®)
  • Ofatumumab (brand name: Arzerra®)
  • Pembrolizumab (brand name: Keytruda®)
  • Polatuzumab vedotin-piiq (brand name: Polivy™)
  • Rituximab (brand name: Rituxan®)
  • Rituximab and hyaluronidase human (brand name: Rituxan Hycela™)
  • Rituximab-abbs (brand name: Truxima®; biosimilar to rituximab)
  • Rituximab-arrx (brand name: Rituxan®; biosimilar to rituximab)
  • Rituximab-pvvr (brand name: Ruxience™; biosimilar to rituximab)
  • Tafasitamab-cxix (brand name: Monjuvi®)3,4,6

Kinase inhibitors

Kinase inhibitors target a specific protein that is found on certain cancer cells. Kinase inhibitors used to treat certain blood cancers include:

  • Acalabrutinib (brand name: Calquence®)
  • Bosutinib (brand name: Bosulif®)
  • C​opanlisib (​brand name: ​Aliqopa™)​​​
  • Crizotinib (brand name: Xalkori®)
  • Dasatinib (brand name: Sprycel®)
  • Duvelisib (​brand name: ​Copiktra™)​
  • Fedratinib (brand name: Inrebic®)
  • Gilteritinib (brand name: Xospata®)
  • Ibrutinib (brand name: Imbruvica®)
  • Idelalisib (​brand name: ​Zydelig®)​​
  • Imatinib mesylate (brand name: Gleevec®)
  • Midostaurin (brand name: Rydapt®)
  • Nilotinib (brand name: Tasigna®)
  • Ponatinib (brand name: Iclusig®)
  • Ruxolitinib (brand name: Jakafi®)
  • Umbralisib (brand name: Ukoniq™)
  • Vemurafenib (brand name: Zelboraf®)
  • Zanubrutinib (brand name: Brukinsa™)5,6

Proteasome inhibitors

Proteasome inhibitors block the action of proteasomes (substances which remove proteins inside cancer cells), which can help cause the proteins to increase in the cancer cell and can lead to the cancer cell’s death. Proteasome inhibitors used to treat certain blood cancers include:

  • Bortezomib (brand name: Velcade®)
  • Carfilzomib (brand name: Kyprolis®)
  • Ixazomib (brand name: Ninlaro®)3,6

Histone deacetylase inhibitors

Histone deacetylase (HDAC) inhibitors block enzymes that are important for cell division and may block the growth of cancer cells. HDAC inhibitors used to treat certain blood cancers include:

  • Belinostat (brand name: Beleodaq®)
  • Panobinostat (brand name: Farydak®)
  • Romidepsin (brand name: Istodax®)
  • Vorinostat (brand name: Zolinza®)3,6

IDH inhibitors

IDH inhibitors block IDH proteins made from mutated IDH1 or IDH2 genes found in some individuals with blood cancer. IDH inhibitors used to treat certain blood cancers include:

  • Enasidenib (​brand name: ​Idhifa®)​
  • Ivosidenib (​brand name: ​Tibsovo®)​6

BCL-2 inhibitors

BCL-2 inhibitors inhibit B-cell lymphoma 2 (BCL2) proteins, which are proteins that can prevent cancer cells from naturally dying, which is called apoptosis. BCL-2 inhibitors used to treat certain blood cancers include:

  • Venetoclax (Venclexta™)​6

Methyltransferase inhibitors

Methyltransferase inhibitors block a protein called EZH2. EZH2 plays a role in turning on and off certain genes, allowing cancer cells to grow and divide. Methyltransferase inhibitors used to treat certain blood cancers include:

  • Tazemetostat (brand name: Tazverik™)

Hedgehog pathway inhibitors

Hedgehog pathway inhibitors target proteins produced by an overactive cell signaling pathway called hedgehog. Hedgehog pathway inhibitors used to treat certain blood cancers include:

  • Glasdegib (​brand name: ​Daurismo™)​6


Retinoids are involved in normal cell growth, differentiation, and death and are related to vitamin A. Retinoids used to treat certain blood cancers include:

  • Bexarotene (​brand name: ​Targretin®)​
  • Tretinoin (​brand name: ​Vesanoid®)​6


Antimetabolites stop or slow enzymes that are necessary for DNA synthesis within cancer cells, ultimately causing cell death. Antimetabolites used to treat certain blood cancers include:

  • Pralatrexate (​brand name: ​Folotyn®)6

Nuclear export inhibitors

Selective inhibitors of nuclear export (SINE) prevent the cell from removing tumor suppressor, growth regulatory, and anti-inflammatory proteins from the nucleus. SINE used to treat certain blood cancers include:

  • Selinexor (brand name: Xpovio™)6

Anti-CD123 therapy

CD123-directed cytotoxins target cell that express CD123 proteins, ultimately causing cell death. Anti-CD123 therapies used to treat certain blood cancers include:

  • Tagraxofusp-erzs (brand name: Elzonris®)

Peptide-drug conjugates

Peptide-drug conjugates combine a drug molecule with a peptide, which helps to more precisely deliver the drug to cancerous cells. Peptide-drug conjugates used to treat certain blood cancers include:

  • Melphalan flufenamide (Pepaxto®)

Common side effects of targeted therapies

Targeted therapies can cause side effects. Side effects are dependent on the type, and dosage, of the drugs given. Some common side effects of certain targeted therapies include:

  • Diarrhea
  • Liver problems
  • Fatigue
  • Skin problems, including rashes and dry skin
  • Problems with blood clotting and wound healing
  • High blood pressure
  • Gastrointestinal perforation (a rare side effect that causes a hole in the intestines)2

Most side effects from targeted therapies are temporary and tend to go away after treatment is completed. Many of the side effects can be managed or prevented. Communication between patients and their health care team is critical, and any side effects experienced should be brought to the attention of a doctor.2

These are not all the possible side effects of targeted therapies. Patients should talk to their doctor about what to expect with treatment with targeted therapy.

How targeted therapy is given

Targeted therapies may be delivered in a pill form to take by mouth, or they may be injected into a vein. The frequency and length of treatment depends on the type of blood cancer a person has, and the type of targeted therapy being used. Some targeted therapies are taken daily, while others may be administered weekly or monthly.2

Patients should talk to their doctor if they have any questions, or if they have questions regarding their targeted therapy regimen. Before starting treatment with targeted therapy, patients should tell their doctor about all their health conditions, as well as any medications (prescription and over-the-counter), herbal supplements, and vitamins they are taking.

How to tell if targeted therapy is working

To determine the effectiveness of targeted therapy, doctors may order blood tests, bone marrow biopsies, or imaging tests to measure the amount of cancer cells in the blood or bone marrow.2

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Written by: Emily Downward | Last reviewed: October 2020