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Some targeted therapy drugs can be useful in treating acute lymphocytic leukemia (ALL), also known as acute lymphoblastic leukemia.

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.

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

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

In about 1 out of 4 adults with ALL, the leukemia cells have the Philadelphia chromosome.

This abnormal chromosome forms when genetic material between chromosomes 9 and 22 swaps, creating a new gene called BCR::ABL1 (or BCR-ABL). Cells with the BCR::ABL1 gene make an abnormal protein that helps the cells grow.

Tyrosine kinase inhibitors (TKIs)

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

• Imatinib (Gleevec, Imkeldi)
• Dasatinib (Sprycel)
• Nilotinib (Tasigna)
• Ponatinib (Iclusig)
• Bosutinib (Bosulif)

In people with Ph+ ALL, using a TKI along with other treatments like chemo or an immunotherapy drug helps increase the chance the leukemia will go into remission. Continuing one of these drugs after the initial treatment can also help keep the leukemia from coming back. If one TKI doesn't work, or stops working, another one might be tried.

These drugs are taken daily as pills or capsules. Imatinib is also available as a liquid (under the name Imkeldi).

Side effects

Common side effects of TKIs include:

• Diarrhea
• Nausea
• Muscle pain
• Fatigue
• Skin rash
• Liver problems
• Fluid buildup
• Low blood cell counts

Low blood cell counts can lead to problems such as an increased risk of infections, bleeding or bruising easily, and feeling tired, weak, or short of breath.

Other, more serious side effects can also happen, depending on the TKI. If you take one of these drugs, ask your cancer care team what to look out for.

Targeted drugs for ALL with a KMT2A gene change

In a small number of people with ALL, the leukemia cells have a type of change known as a translocation in the KMT2A gene. This causes the cells to make an abnormal KMT2A protein, which can combine with another protein called menin to help the cells grow.

Revumenib (Revuforj)

Revumenib (Revuforj) is a menin inhibitor. It stops menin from combining with the KMT2A protein.

This drug can be used to treat ALL with a KMT2A translocation if the ALL is no longer responding to treatment or has come back (relapsed) after other treatments. Your blood or bone marrow can be tested to see if your leukemia cells have this gene change.

This drug is taken by mouth as pills, typically twice a day.

Side effects

Common side effects of this drug include:

• Nausea and vomiting
• Bleeding
• Diarrhea or constipation
• Fatigue
• Muscle pain
• Increased risk of infection
• Swelling in the arms and legs
• Loss of appetite

This drug may also cause serious heart rhythm problems.

These heart rhythm problems might lead to an irregular heartbeat, which can be life-threatening. Your cancer care team will check your blood mineral levels and get electrocardiograms (EKGs) to test your heart rhythm before and during treatment with this drug.

Another important possible side effect is differentiation syndrome.

This happens when the leukemia cells release certain chemicals into your blood. It most often occurs shortly after starting treatment, but it sometimes happens weeks or months later.

Symptoms include fever, coughing or breathing problems (from fluid buildup in the lungs and around the heart), dizziness or lightheadedness (from low blood pressure), urinating less often (from damage to the kidneys), and severe fluid buildup elsewhere in the body.

It can often be treated by stopping the drug for a while and taking other medicines, such as dexamethasone or hydroxyurea.

Immunotherapy drugs for ALL

Some 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:

• Rituximab (Rituxan and other names)
• Blinatumomab (Blincyto)
• Inotuzumab ozogamicin (Besponsa)

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