Targeted Therapy for Acute Lymphocytic Leukemia (ALL)

(Note: This information is about treating acute lymphocytic leukemia (ALL) in adults. To learn about ALL in children, see Leukemia in Children.)

Targeted therapy drugs work by attacking specific parts of cancer cells. They are different from standard chemotherapy (chemo) drugs. They sometimes work when chemo doesn't, and they often have different side effects. Some of these drugs can be useful in certain cases of acute lymphocytic leukemia (ALL).

Targeted drugs for ALL with the Philadelphia chromosome (Ph+ ALL)

In about 1 out of 4 adult patients with ALL, the leukemia cells have the Philadelphia chromosome. This is an abnormal chromosome formed by the swapping of genetic material between chromosomes 9 and 22, which creates a new gene called BCR-ABL. Cells with the BCR-ABL gene make an abnormal protein that helps the cells grow.

Drugs called tyrosine kinase inhibitors (TKIs) have been developed to attack this protein. Examples include:

• Imatinib (Gleevec^®)
• Dasatinib (Sprycel^®)
• Nilotinib (Tasigna^®)
• Ponatinib (Iclusig^®)
• Bosutinib (Bosulif^®)

In patients with Ph+ ALL, adding a TKI to chemo helps increase the chance that the leukemia will go into remission. Continuing on one of these drugs can also help keep the leukemia from coming back. If one TKI doesn't work (or is no longer working), another one might be tried.

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 can get worse at higher than usual doses of the drug. Other, more serious side effects can occur as well, depending on which drug is used.

Immunotherapy drugs for ALL

Some of the immunotherapy drugs used to treat ALL might also be considered forms of targeted therapy, because they work by attaching to specific parts of leukemia cells. Examples include:

• Blinatumomab (Blincyto)
• Inotuzumab ozogamicin (Besponsa)

For more information on these drugs, see Immunotherapy for Acute Lymphocytic Leukemia (ALL).