Leukemia medications kill cancer cells and prevent cancer cells from dividing and multiplying. Leukemia medications may include chemotherapy, targeted therapy, or immunotherapy drugs.

Leukemia medications work in different ways to destroy cancer cells or prevent them from growing.

This article examines the medications doctors may use to treat leukemia and their common side effects.

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Leukemia medications work in different ways to kill cancer cells. Doctors consider several factors before recommending a treatment approach, including:

  • the type of leukemia
  • the stage of the cancer
  • a person’s age
  • whether people have had previous treatments and how the cancer responded

For some people, one drug therapy may effectively treat leukemia, while others may need a combination of medications.

Using a combination of drugs may reduce the likelihood of cancer cells becoming drug-resistant and increase the chance of curing the cancer or achieving long-term remission.

Learn more about common cancer medications.

Types of drug therapy for leukemia may include the following:


Cancer cells rely on the building blocks of DNA and RNA to grow and survive. Antimetabolites mimic these natural building blocks, so the cancer cells attach to the drug instead of DNA or RNA. This means the cells cannot copy the genetic material to divide and multiply and cannot survive.

Antimetabolites include:

  • cladribine (Leustatin)
  • fludarabine (Fludara)
  • pentostatin (Nipent)

Learn more about pentostatin and other antimetabolites.


Antimitotics prevent mitosis, which is the term for cell division. Antimitotics damage the cancer cells, preventing them from dividing and multiplying.

Antimitotics include:

  • vincristine (Oncovin)
  • vinblastine (Velban)

Antitumor antibiotics

Antitumor antibiotics attach to the DNA in cancer cells to kill the cells and prevent them from dividing and multiplying.

Antitumor antibiotics include:

  • daunorubicin (Cerubidine)
  • doxorubicin (Adriamycin or Doxil)
  • idarubicin (Idamycinv)
  • mitoxantrone (Novantrone)

Learn more about these antineoplastic drugs.

Asparagine-specific enzymes

Asparagine-specific enzymes derive from Escherichia coli (E. coli) bacteria. Some cancer cells require an amino acid called asparagine to grow. Asparagine-specific enzymes break down asparagine to prevent cancer cells from growing.

Examples include:

  • asparaginase erwinia chrysanthemi
  • L-asparaginase (Elspar)

Learn more about bacteria and what they do.


Biosimilars have a similar structure and result as biologics but are more affordable. Biosimilars include:

  • rituximab-arrx (Riabni)
  • rituximab-abbs (Truxima)

Learn the difference between biologics and biosimilars.


Bisphosphonates do not directly target cancer cells, but they can treat the high calcium levels that some leukemias cause. Bisphosphonates can help reduce bone breakdown, pain, and the risk of fractures.

Bisphosphonates include:

  • zoledronic acid (Zometa or Reclast)
  • pamidronate (Aredia)

Learn about bisphosphonates for osteoporosis.


Chemotherapy can damage or kill cancer cells and prevent them from growing and multiplying. Chemotherapy includes antimetabolites, antitumor antibiotics, and drugs that damage DNA.

Learn about R-CHOP therapy here.

DNA-damaging agents (antineoplastics) and alkylating agents

DNA-damaging agents, antineoplastics, and alkylating agents alter DNA to prevent cancer cells from growing.

DNA-damaging agents include:

  • cyclophosphamide (Cytoxan)
  • carboplatin (Paraplatin)

Alkylating agents include:

Learn all about the side effects of Leukeran.

DNA-repair enzyme inhibitors

DNA-repair enzyme inhibitors attack enzymes in cancer cells that repair DNA damage. Without proper DNA repair, the cells cannot grow.

Examples include:

  • etoposide (VePesid)
  • topotecan (Hycamtin)

Learn about oral chemotherapy drugs.

Histone deacetylase inhibitors

Histone deacetylase inhibitors target proteins that affect DNA to attack cancer cells. Examples include:

  • vorinostat (Zolinza)
  • romidepsin (Istodax)


Corticosteroids are synthetic hormones that can kill cancerous immune cells or lymphocytes. Examples include:

Learn more about corticosteroids here.

Hypomethylating or demethylating agents

Hypomethylating or demethylating agents slow down or reverse methylation in cancer cells — an important process in how cells grow and replicate. Examples include:

  • decitabine
  • azacitidine

Learn more about cancer that has grown past the cells where it developed.


Immunomodulators suppress or stimulate the immune system to attack cancer cells. Immunomodulators include:

  • interferon alfa-2a (Roferon-A)
  • interferon alfa-2b (Intron A)

Learn about the side effects of interferon.

Janus-associated kinase (JAK) inhibitors

JAK inhibitors interfere with signaling pathways that can lead to inflammatory and immune responses in leukemia. JAK inhibitors include:

Learn more about the immune system here.

Monoclonal antibodies

Monoclonal antibodies target specific antigens on cancer cells to destroy the cancer cell. Monoclonal antibodies include:

Learn more about monoclonal antibody therapy.

Phosphoinositide 3-kinase inhibitors (PI3K inhibitors)

PI3K inhibitors block PI3K enzymes in cancer cells, which may prevent cancer cells from growing. PI3K inhibitors include:

  • idelalisib (Zydelig)
  • duvelisib (Copiktra)

Learn more about chronic lymphocytic leukemia (CLL).

Proteasome inhibitors

Proteasome inhibitors work by blocking the action of proteins called proteasomes, helping to kill cancer cells in your body. Proteasome inhibitors include:

Learn more about targeted therapy for CLL.

Selective inhibitors of nuclear export (SINE)

SINE drugs block proteins that support tumor growth, causing cancer cells to die. SINEs include:

Learn more about the different types of tumors.

Tyrosine kinase inhibitors

Tyrosine kinase inhibitors attach themselves to proteins called tyrosine kinase, which prevents them from signaling abnormal white blood cells to grow. Tyrosine kinase inhibitors include:

Learn more about how tyrosine kinase inhibitors can help treat chronic myeloid leukemia.

Side effects may vary with each type of drug, but common side effects of leukemia medications include the following:

Find resources and information about leukemia here.

Leukemia medications aim to destroy cancer cells or prevent them from growing. Medications may include chemotherapy, targeted therapy, or immunotherapy drugs.

Doctors will decide the best course of treatment based on certain factors, such as a person’s age and the stage and type of leukemia.