Targeted Therapy

Reviewed by: HU Medical Review Board | Last reviewed: May 2023

Targeted therapies are cancer treatments that stop or slow the growth and spread of cancer. They do this by interfering with specific areas of cancer cells that are involved in cell growth, or by focusing on specific features that are unique to cancer cells.1,2

How does targeted therapy work?

Targeted therapy is different than chemotherapy, which focuses on cells that divide and grow quickly. Chemotherapy cannot tell the difference between fast-growing cancer cells and fast-growing healthy cells in the body, like those in the digestive tract, hair, bone marrow, and blood. This can cause many side effects. Targeted therapy focuses on other specific features of cancer cells in an effort to treat cancer without doing damage to normal cells. This causes fewer side effects.1,2

Targeted therapies (immunotherapies) also boost the immune system’s response to cancer cells. Some targeted therapies do kill cells directly; for example, by inhibiting anti-apoptotic proteins.

Targeted therapy is also different from chemotherapy in the way it affects cancer cells. Targeted therapies usually block the growth or spread of cancer cells. Chemotherapy drugs typically kill cancer cells.2

Examples of targeted therapy

Different types of targeted therapy are used to treat certain blood cancers.

Monoclonal antibodies

Monoclonal antibodies are created in a lab to identify and block cancer growth or kill cancer cells. Some monoclonal antibodies are called conjugated monoclonal antibodies because they are attached to a chemotherapy drug or radioactive particle. These antibodies deliver chemotherapy drugs or radioactive particles to cancer cells. Monoclonal antibodies used to treat certain blood cancers include:3,4,6

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

Kinase inhibitors

Kinase inhibitors target a specific protein (kinases) found on or in some cancer cells. Kinase inhibitors used to treat certain blood cancers include:5,6

  • Acalabrutinib (Calquence®)
  • Asciminib (Scemblix®)
  • Avapritinib (Ayvakit™)
  • Bosutinib (Bosulif®)
  • C​opanlisib (​Aliqopa™)​​​
  • Crizotinib (Xalkori®)
  • Dasatinib (Sprycel®)
  • Duvelisib (​Copiktra™)​
  • Fedratinib (Inrebic®)
  • Gilteritinib (Xospata®)
  • Ibrutinib (Imbruvica®)
  • Idelalisib (​Zydelig®)​​
  • Imatinib mesylate (Gleevec®)
  • Midostaurin (Rydapt®)
  • Nilotinib (Tasigna®)
  • Pacritinib (Vonjo™)
  • Ponatinib (Iclusig®)
  • Ruxolitinib (Jakafi®)
  • Umbralisib (Ukoniq™)
  • Vemurafenib (Zelboraf®)
  • Zanubrutinib (Brukinsa™)

Proteasome inhibitors

Proteasome inhibitors block the action of proteasomes, substances that remove proteins inside cancer cells. This can increase proteins in cancer cells and cause them to die. Proteasome inhibitors used to treat certain blood cancers include:3,6

  • Bortezomib (Velcade®)
  • Carfilzomib (Kyprolis®)
  • Ixazomib (Ninlaro®)

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:3,6

  • Belinostat (Beleodaq®)
  • Panobinostat (Farydak®)
  • Romidepsin (Istodax®)
  • Vorinostat (Zolinza®)

IDH inhibitors

IDH inhibitors block IDH proteins made from mutated IDH1 or IDH2 genes found in some people with blood cancer. IDH inhibitors used to treat certain blood cancers include:6

  • Enasidenib (​Idhifa®)​
  • Ivosidenib (​Tibsovo®)​
  • Olutasidenib (Rezlidhia®)

BCL-2 inhibitors

BCL-2 inhibitors inhibit B-cell lymphoma 2 (BCL2) proteins, which are proteins that can prevent cancer cells from naturally dying. This process is called apoptosis. BCL-2 inhibitors used to treat certain blood cancers include:6

  • Venetoclax (Venclexta™)​

EZH2 inhibitors

EZH2 inhibitors block a protein called enhancer of zeste homolog 2 (EZH2). EZH2 plays a role in turning on and off certain genes, allowing cancer cells to grow and divide. EZH2 inhibitors used to treat certain blood cancers include:6

  • Tazemetostat (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:6

  • Glasdegib (Daurismo™)​


Retinoids are involved in normal cell growth, differentiation, and death. They are related to vitamin A. Retinoids used to treat certain blood cancers include:6

  • Bexarotene (​Targretin®)​
  • Tretinoin (​​Vesanoid®)​
  • ATRA (all-trans-retinoic acid)

Nuclear export inhibitors

Selective inhibitors of nuclear export (SINE) prevent cells from removing tumor suppressor, growth regulatory, and anti-inflammatory proteins from the nucleus. SINE used to treat certain blood cancers include:6

  • Selinexor (Xpovio™)

Anti-CD123 therapy

CD123-directed cytotoxins target cells that express CD123 proteins. This causes cell death. Anti-CD123 therapies used to treat certain blood cancers include:6

  • Tagraxofusp-erzs (Elzonris®)

What are the possible side effects?

Side effects can vary depending on the specific drug you are taking. Common side effects of certain targeted therapies include: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 you and your doctor is key, and any side effects you are experiencing should be brought to the attention of your doctor.2

These are not all the possible side effects of targeted therapy. Talk to your doctor about what to expect or if you experience any changes that concern you during treatment with targeted therapy.

Other things to know

Targeted therapies may be delivered:2

  • Under the skin (subcutaneously)
  • In pill form to take by mouth
  • By injection into a vein

The frequency and length of treatment depend on the type of blood cancer you have and the type of targeted therapy being used. Some targeted therapies are taken daily, while others may be taken weekly or monthly.2

To determine the effectiveness of targeted therapy and measure the number of cancer cells in your blood or bone marrow, your doctor may order:2

Talk to your doctor if you have questions about your targeted therapy regimen. Before beginning treatment for blood cancer, tell your doctor about all your health conditions and any other drugs, vitamins, or supplements you are taking. This includes over-the-counter drugs.

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