A wide variety of treatment options are used to fight leukemia. Which treatments your doctor will recommend depends largely on which type of leukemia you have. Some treatments may be combined in a treatment plan, while others may be used alone as a single treatment, also known as monotherapy. The goals of leukemia treatment can also differ. While there are treatments that can provide a cure for many cases of acute leukemia, the goal in treating chronic leukemia may be to achieve remission and prevent relapses or simply to slow cancer growth.
This article will cover the major categories of treatments used in leukemia and the basics of how they work to fight cancer. Each treatment category will have examples of situations when the leukemia treatment may be recommended.
More than 50 different drugs are used to treat blood cancers, which include leukemia, lymphoma, myeloma, and myeloproliferative neoplasms (MPNs). With so many different types of leukemia treatments available, what leads your doctor to recommend one option over another? Many factors influence which treatments are considered good options for you. Here are some of the most important factors.
The four main types of leukemia are:
Each of these types has many subtypes, and some are also classified by stage. ALL and AML are acute forms of leukemia, which grow rapidly and require immediate treatment. CLL/SLL and CML are chronic forms of leukemia, which in most cases are indolent (slow-growing) and may not require treatment right away.
Types of leukemia are categorized by which kind of white blood cell is involved. Genetic details about your cancer cells may also be important for treatment decisions. Not every treatment works well for every type of leukemia.
Read more about types of leukemia.
Your age and your general health can both be risk factors in treatment for leukemia. Some treatments have possible side effects that may be considered too risky for older adults or people living with other health conditions.
Your oncologist will also consider:
For instance, is it more important to you to live as long as possible or to maintain a high quality of life with few side effects from treatment? Is it your top priority to use the most effective treatment or to avoid the risk for serious side effects? Do you plan to have children in the future? These and other considerations should be weighed in discussion with your doctor about your cancer care.
The best treatment plan for you will be the one you and your doctor agree together is your best option.
Treatment for leukemia may be given at different points for different purposes. These phases of leukemia treatment may include induction therapy, consolidation therapy, and maintenance therapy.
In acute forms of leukemia (ALL and AML), chemotherapy is often used as an initial treatment to kill as many cancer cells as possible and wipe out all signs of disease, resulting in remission.
The goal of consolidation therapy, also known as post-remission therapy, is to destroy any remaining cancer cells and make sure leukemia will not relapse. Consolidation may include chemotherapy or a stem cell transplant.
In some cases, doctors may recommend taking one or more medications long-term — for years — after the induction and consolidation phases of leukemia treatment are complete. The goal of maintenance therapy is to prolong remission and prevent a relapse of leukemia.
Depending on your type of leukemia and other factors, you may use several types of treatment — either alone or in combination — at different times over the course of your cancer treatment.
Leukemia is caused by the rapid, uncontrolled growth of abnormal white blood cells that have undergone genetic mutations. Chemotherapy works by directly killing or damaging rapidly dividing cells, including cancer cells, in your body. These potent drugs must be toxic to kill cancer cells, but they can also damage healthy cells.
Depending on which drugs are involved, chemotherapy may be administered intravenously (by IV), injected into the skin or muscle, or taken orally as pills or capsules. Some chemotherapy may be taken at home. Other types may require travel to a cancer treatment center to receive each dose or a hospital stay for the duration of a treatment cycle, which may last weeks. Chemotherapy is prescribed before stem cell transplantation and may be used in combination with radiation therapy or targeted therapy.
There are many classes of chemotherapy drugs, including alkylating agents, antitumor antibiotics, and antimetabolites. Most chemotherapy drugs work by interfering with the DNA of cancer cells, preventing them from growing or causing them to die. Chemotherapy drugs are often combined into regimens. Here are a few of the most common chemotherapy regimens used in the four major types of leukemia.
A wide variety of chemotherapy combinations are used to treat specific subtypes of ALL. A typical chemotherapy regimen for ALL includes:
Depending on the specific details of an individual’s ALL, chemotherapy may also include drugs such as cyclophosphamide (Cytoxan), L-asparaginase or pegaspargase (Oncaspar), methotrexate, or cytarabine.
Targeted therapies may also be included along with chemotherapy for ALL.
A typical chemotherapy regimen for most types of AML includes:
Targeted therapies may also be included along with chemotherapy for AML.
In cases of CLL/SLL, a chemotherapy drug is often combined with a monoclonal antibody, or immune system protein, that can more precisely target cancer cells. This combination is known as chemoimmunotherapy.
Over the past few years, targeted therapy has come to the forefront of treatment of CLL/SLL. A few of the most common regimens for CLL/SLL include:
Chemotherapy is no longer a common treatment for CML. Instead, therapies called tyrosine kinase inhibitors (TKIs) that target BCR-ABL, the Philadelphia chromosome, are the mainstay of treatment.
Targeted therapy works by interfering with specific genes or proteins cancer cells need to grow and survive. There are many classes of targeted therapy drugs that operate in different ways, making them effective for different types of leukemia. Targeted therapies only work on cancer cells that carry their specific target. Major classes of targeted therapies for leukemia are described below.
TKIs target a protein called the BCR-ABL tyrosine kinase enzyme, which is necessary for leukemia cells in some types of leukemia to survive. TKIs used to treat CML and ALL with the Philadelphia chromosome include:
Bruton’s tyrosine kinase (BTK) inhibitors block the activity of a certain type of tyrosine kinase that cancer cells need to grow and stay alive. BTK inhibitors are used to treat CLL/SLL and some types of non-Hodgkin lymphoma, a type of blood cancer closely related to leukemia. The two BTK inhibitors currently used are ibrutinib (Imbruvica) and acalabrutinib (Calquence).
Venetoclax (Venclexta) is used to treat some cases of AML and CLL/SLL. Venclexta’s target is BCL-2, a protein that enables the survival of leukemic cells.
PI3K-delta is a kinase protein cancer cells need to signal and control growth. PI3K inhibitors work by blocking this protein in cases of CLL/SLL. PI3K inhibitors include idelalisib (Zydelig) and duvelisib (Copiktra).
In some cases of AML, cancer cells have a mutation in a gene called FLT3. FLT3 inhibitors used to treat AML with the FLT3 mutation include midostaurin (Rydapt) and gilteritinib (Xospata).
In AML that involves genetic mutations of the IDH genes (IDH1 or IDH2), IDH inhibitors can work by making sure white blood cells mature normally into healthy cells rather than cancer cells. IDH inhibitors include ivosidenib (Tibsovo) and enasidenib (Idhifa).
Immunotherapy is an approach to treating leukemia that modifies your immune system to be better at targeting and destroying cancer cells. There are different types of immunotherapy that work with different aspects of the immune system.
Some sources consider monoclonal antibodies a type of targeted therapy, while others describe these biologic drugs as a type of immunotherapy. Antibodies are proteins made by the immune system that can target genes and proteins found only on the type of white blood cell involved in leukemia.
Examples of monoclonal antibodies used to treat leukemia include:
Monoclonal antibodies can also be attached to another drug to create an antibody-drug conjugate. Antibody-drug conjugates use the antibody to deliver the drug directly to its target on cancer cells. Antibody-drug conjugates used in leukemia include gemtuzumab ozogamicin (Mylotarg) for AML and inotuzumab ozogamicin (Besponsa) for ALL.
Chimeric antigen receptor (CAR) T cell therapy works by modifying your own T cells to seek out and destroy the type of cells involved in leukemia. First, your T cells are harvested in a process similar to donating blood. Next, your T cells are shipped to a lab, where they are genetically modified to recognize and attack a certain receptor found on your cancer cells. Finally, the modified T cells are infused back into your body.
CAR-T cell therapies approved by the U.S. Food and Drug Administration (FDA) include:
Interferon is a substance produced by the immune system that fights leukemia by reducing the number of white blood cells. Interferon is sold under many brand names, including Alferon, Infergen, Intron A, and Roferon-A. Interferon is used to treat some cases of CML, although targeted therapies are used more commonly.
Stem cell transplants (formerly called bone marrow transplantation) are sometimes used to treat people with leukemia. Stem cell transplants can help compensate for higher doses of chemotherapy that will destroy bone marrow. The transplanted stem cells will form healthy bone marrow, replacing the destroyed cells after chemotherapy (and sometimes radiation therapy) is complete. In some people, stem cell transplantation can help cure leukemia. Not everyone with leukemia is eligible to receive stem cell therapy.
In allogeneic stem cell transplant, stem cells are harvested from the blood of a donor who may be a sibling or other close blood relative of the recipient or an unrelated person who is a good genetic match. Less commonly, an autologous stem cell transplant may be considered. In an autologous stem cell transplant, the stem cells are harvested from the person’s own blood.
Allogeneic stem cell transplants are used to treat ALL in adults, but only considered as a treatment for children after a relapse or when chemotherapy is ineffective. Allogeneic stem cell transplants are also used in cases of AML and CML.
Autologous stem cell transplants (ASCT) are sometimes used to treat ALL and AML. ASCT may be considered in cases where other treatments are ineffective and the person with leukemia is not a good candidate for allogeneic stem cell transplant, which can cause life-threatening side effects. ASCT may be used when a closely matched donor for allogeneic stem cell transplant is not available.
Radiation therapy (also known as radiotherapy) is not a common treatment in most cases of leukemia. However, radiation therapy may be used in certain situations in some cases of ALL, AML, CLL/SLL, and CML. Radiation may be used with chemotherapy to destroy bone marrow before receiving a stem cell transplant.
Radiation interferes with cell division. Since cancer cells divide much more rapidly than normal cells, they are more vulnerable to radiation. Radiation kills cancer cells, but the normal, healthy cells of your body are better able to survive and heal.
Some people with leukemia participate in clinical trials. Clinical trials contribute to cancer research and may provide access to new treatments for leukemia not yet available to everyone. It is important to know that participants in clinical trials are protected by the process of informed consent, which ensures that all potential risks and benefits are transparent before you decide to join a trial. Participants in clinical trials can withdraw at any time.
Some chronic forms of leukemia, including many cases of CLL/SLL, may not require treatment right away. Instead, doctors may recommend a “watch and wait” approach, also known as watchful waiting. During watch and wait (also called watchful waiting), you will be closely monitored by your doctor with regular appointments and blood tests. Watchful waiting may last for years until there are signs or symptoms that indicate cancer is beginning to grow more rapidly.
Whether or not you are actively treating your leukemia, making changes to improve your overall health can help you have a better quality of life and may even increase your treatment options for leukemia. Good nutrition and regular exercise can improve your energy and mood and help you recover faster from treatment or illness. If you smoke cigarettes, quitting smoking may help you live longer, reduce side effects and complications from cancer treatment, and help you recover faster from the effects of treatment.
More effective treatments for leukemia have increased survival rates and rates of cure and remission over the past 10 years. Whether a case of leukemia is curable or not depends on many factors. In some cases, chemotherapy (and sometimes radiation) followed by stem cell transplantation can provide a cure.
Leukemia Condition Guide