Targeted Therapy for Acute Lymphocytic Leukemia

In recent years, new drugs that target specific parts of cancer cells have been developed. These drugs work differently than standard chemotherapy (chemo) drugs. They often have different (and less severe) side effects. These drugs are often referred to as targeted therapy. Some of these drugs can be useful in certain cases of acute lymphocytic leukemia (ALL).

About 1 out of 4 adult patients with ALL have leukemia cells with the Philadelphia chromosome. This is an abnormal chromosome formed by the swapping of material between chromosomes 9 and 22. This forms a new gene called BCR-ABL. The Philadelphia chromosome and BCR-ABL gene are also found in the cells of a different leukemia – chronic myeloid leukemia (CML). Cells with the BCR-ABL gene make an abnormal protein that helps the cells grow. Drugs have been developed to attack this protein. These drugs are called tyrosine kinase inhibitors (or TKIs), and include imatinib (Gleevec®), dasatinib (Sprycel®), nilotinib (Tasigna®), bosutinib (Bosulif®), and ponatinib (Iclusig®). Although these drugs were originally aimed at treating CML, some of them have been found to be helpful in treating patients with ALL that has the Philadelphia chromosome.

In studies of patients whose ALL cells contain the Philadelphia chromosome, adding one of these drugs to chemo helps more patients go into remission after treatment. Continuing on these drugs can also help keep the leukemia from coming back.

These drugs are taken daily as pills. Common side effects include diarrhea, nausea, muscle pain, fatigue, and skin rashes. These are generally mild. A common side effect is swelling around the eyes or in the hands or feet. Other possible side effects include lower red blood cell and platelet counts at the start of treatment. All of these side effects get worse at higher than usual doses of the drug. Other more serious side effects can occur, as well, which differ depending on which drug is used.

More information about side effects of targeted therapy drugs can be found in Targeted Cancer Therapy.

The American Cancer Society medical and editorial content team
Our team is made up of doctors and master's-prepared nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

Last Medical Review: December 2, 2014 Last Revised: February 18, 2016

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