Chemotherapy for Chronic Myelogenous Leukemia

At NYU Langone’s Perlmutter Cancer Center, most people with chronic myelogenous leukemia (CML) are treated with targeted chemotherapy drugs. They are designed to block signals sent by a protein known as BCR-ABL, which produces an excessive amount of another protein called tyrosine kinase, causing leukemia cells to grow and multiply uncontrollably.

Although it isn’t possible to eradicate all CML cells, these medications can help people achieve remission, meaning cancer cells are no longer detected in the blood. For most people with CML, these drugs are the only necessary treatment. They are taken daily by mouth for the rest of a person’s life.

Currently, five targeted medications for CML are approved by the Food and Drug Administration (FDA). Gleevec® was the first medication of its kind available to people with this type of leukemia. It is frequently the first treatment prescribed for people with CML, though other types of targeted chemotherapy drugs may be prescribed instead.

Most people respond to Gleevec®. Their white blood cell levels drop considerably, and they continue to take the medication for years.

In rare situations, however, the cancer doesn’t respond to treatment. If blood cell levels don’t change significantly after three months, or if you experience significant side effects, your doctor may prescribe one of the second- or third-generation forms of this type of medication.

Side effects can include diarrhea, drowsiness, fatigue, nausea, and swelling of the legs and around the eyes.

Doctors monitor your blood for signs of BCR-ABL transcription, in which genetic information causes certain proteins to form, to determine if the number of CML cells is increasing. This can be a sign of a relapse. If targeted treatments aren’t working, or if the side effects are affecting quality of life, a person may need a stem cell transplant to treat this leukemia.