Treatments for Leukemia

Leukemia is a blood cancer. Because it can’t be removed from the body through surgery, chemotherapy and other medications are the first course of treatment. If you are diagnosed with a chronic leukemia, your physicians might advise waiting to begin treatment until the cancer progresses.

Clinical trials

Many leukemia patients at Siteman receive treatment through clinical trials. These studies evaluate the newest drugs and therapies, allowing patients to access treatments that have not yet been released on the market. Clinical trials can be a game-changer for patients who have not found success with other forms of treatment.

Siteman Cancer Center houses more clinical trials than any other facility in the region. The Washington University oncologists on your care team lead some of these trials themselves. They will know which trials might be a good fit for you and will be able to enroll you if necessary.

Your team will be sure to discuss all of your options with you. Don’t hesitate to approach them if you have any questions.

Patients with leukemia generally receive one or more of the following treatments.


Chemotherapies are drugs that destroy cancer cells. They are usually administered through an IV infusion, although some chemotherapies are now available as pills or injections.

Learn more about chemotherapy for leukemia at Siteman Cancer Center.


Immunotherapies are a new class of drug that helps the immune system recognize and destroy cancer cells.

Read about how the immune system can fight cancer.

Targeted therapy

Cancer cells use many different tactics to multiply and spread around the body. Targeted therapies are drugs that disable these tactics, rather than eliminating the cells as a whole. They try to prevent cancer cells from growing while protecting the normal cells around them. Targeted therapy is administered intravenously or as a pill, depending on the medication.

Stem cell transplantation

Leukemia arises when the stem cells that make new blood cells stop working properly. A stem cell transplant is a procedure that replaces the body’s blood-forming stem cells with new stem cells.

The procedure is also referred to as a bone marrow transplant because the stem cells are located in the bone marrow.

Patients may undergo a stem cell transplant if other forms of treatment are not sufficient to eliminate their disease. Transplants have cured many leukemia patients and helped others attain lengthy remissions.

Siteman Cancer Center specializes in performing safe, effective stem cell transplants.

CAR T-cell therapy

CAR T-cell therapy is a new form of treatment that modifies the patient’s immune system cells so they can attack cancer cells. It is licensed for use in pediatric patients with ALL. Adult leukemia patients with ALL may be able to access CAR T-cell therapy through clinical trials.

Read more about how CAR T-cell therapy helps your body fight cancer.

Treatment for acute lymphoblastic leukemia (ALL)

Patients with acute lymphoblastic leukemia typically begin their course of treatment with chemotherapy.  In many cases, they receive chemotherapy in the hospital so their care team can protect them from infection and manage their side effects.

Depending on the nature of their cancer, patients may also be treated with immunotherapy and targeted therapy drugs. Approximately 25% of ALL patients have a genetic mutation called the Philadelphia chromosome, which can be targeted with medications.

If patients need further treatment, their physicians may recommend a stem cell transplant. ALL patients usually receive stem cells from a donor. The donor will either be a member of the patient’s family or an unrelated individual who has “matched” to them.

Patients with ALL who are under 25 years old are eligible to receive CAR T-cell therapy. Older patients with ALL may also be able to undergo CAR T-cell therapy through a clinical trial.

Treatment for acute myeloid leukemia (AML)

An aggressive, fast-growing disease, AML is first treated with chemotherapy. Patients often undergo chemotherapy in the hospital because the drugs will leave them vulnerable to infection. At the hospital, their physicians and nurses can work to keep them safe and help them cope with side effects.

In addition to chemotherapy, AML patients may also receive targeted therapy drugs. These can be paired with chemotherapy or used on their own.

Stem cell transplantation can be an effective option for AML patients who are struggling with other forms of treatment. They will likely receive new stem cells from a donor.

Treatment for chronic lymphocytic leukemia (CLL)

Many cases of chronic lymphocytic leukemia are slow-growing. Physicians often wait to treat CLL until the patient begins to experience symptoms. Depending on the patient’s age at diagnosis, and the profile of the disease, some patients may not need to be treated at all.

Unlike ALL, CLL cannot be cured. If treatment becomes necessary, the goal is to control the disease and prevent it from advancing. Treatments can include chemotherapy, immunotherapy, targeted therapy, and stem cell transplantation.

Treatment for chronic myeloid leukemia

Chronic myeloid leukemia is characterized by a genetic defect known as the Philadelphia chromosome. Patients receive targeted therapy drugs that attack this chromosome and keep the leukemia in check. These targeted therapies are taken by mouth. Though CML can’t be cured, many patients see good results on targeted therapies alone and are able to lead normal lives.

If targeted therapies are not sufficient, CML can also be treated with chemotherapy and stem cell transplantation.

Hairy cell leukemia

This rare type of leukemia is usually managed by observation or a single-agent outpatient chemotherapy treatment for long-term management. Rarely, it may require investigational agents or small-molecule treatment.