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Targeted drugs are typically the first treatment for chronic myeloid leukemia (CML). These types of drugs focus on the changes inside cancer cells that help them grow.

Unlike standard chemotherapy drugs, which work by attacking rapidly growing cells in general (including cancer cells), these drugs target a specific protein in CML cells.

How targeted drugs work to treat CML

CML cells contain an abnormal protein known as BCR::ABL1 (or BCR-ABL), which causes CML cells to grow out of control. BCR::ABL1 is a type of protein known as a tyrosine kinase.

Drugs known as tyrosine kinase inhibitors (TKIs) that attach to BCR::ABL1 and stop it from working are the most common treatment for CML.

TKIs to treat CML

TKIs that can be used to treat CML include:

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

These drugs seem to work best when CML is in the chronic phase, but some can also help people with more advanced disease (accelerated or blast phases).

In most people, the TKIs don't seem to make the leukemia go away completely, so these drugs need to be taken indefinitely to keep it in remission. But for some people who have very good, long-lasting responses to treatment, it might be possible to stop taking these drugs, or to at least lower the dose.

(See Treating Chronic Myeloid Leukemia by Phase to learn more.)

Taking TKIs as directed

TKIs are taken daily by mouth as pills, capsules, or a liquid. To get the best outcomes, it's important to take them exactly as directed.

Follow your doctor’s instructions exactly

Skipping or adjusting doses can affect the way TKI treatment works. See Getting Oral Chemotherapy for more on how to best manage cancer treatments you take at home.

TKIs can interact with many other drugs

TKIs can have serious interactions with some other drugs, over-the-counter supplements, and even certain foods (such as grapefruit, star fruit, and pomegranate).

Give your cancer care team an up-to-date list of everything you're taking, including prescription and over-the-counter drugs, vitamins, and herbal supplements. Always check with your doctor before starting any new medicine or supplement, to be sure it's safe.

Tell your doctor if there is a chance you are pregnant

TKIs are not typically used during pregnancy. These drugs might harm a fetus if you take them when you are pregnant.

Imatinib

Imatinib (Gleevec) was the first drug to specifically target the BCR::ABL1 protein.  It is still sometimes the first drug used to treat CML, especially if it is chronic phase, low-risk CML. Most often, CML responds to treatment with imatinib, and these responses often last for many years.

Imatinib is taken once or twice a day, either as a pill (Gleevec or generic pills) or as an oral solution (Imkeldi).

Side effects of imatinib

Common side effects of imatinib can include:

• Diarrhea
• Nausea
• Muscle pain
• Fatigue
• Skin rash
• Abnormal liver function tests

These side effects are generally mild, and some might go away over time. Most can be treated if needed.

Fluid buildup

Another common side effect is fluid buildup around the eyes, feet, or abdomen (belly). In rare cases the fluid may collect in the lungs or around the heart, which can cause trouble breathing.

If you are taking this drug, tell your cancer care team right away if you notice sudden weight gain or fluid buildup anywhere in your body, or if you have trouble breathing.

Low blood cell counts

Your white blood cell and platelet counts might drop while you’re getting this drug. 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.

When blood counts get low at the start of treatment, it might be because the cells in your bone marrow that are making new blood cells are part of the leukemia process and are being affected by the drug.

Over time, normal blood-forming cells should take over, and your blood counts will begin to rise. But if your blood counts get too low, your doctor might need to lower your dose, or you might need to stop taking the drug for a time.

This can also happen later in treatment. Again, your doctor may lower your dose to see if your blood counts improve.

Dasatinib

Dasatinib (Sprycel) is a second-generation TKI that targets the BCR::ABL1 protein.

It can be used as the first treatment for CML. It can also be helpful for people who can’t take imatinib because of side effects or because it isn’t working.

This drug is taken as a pill, typically once a day.

Side effects of dasatinib

Many of the possible side effects of dasatinib are similar to those of imatinib (see above), including:

• Nausea
• Diarrhea
• Muscle pain
• Fatigue
• Skin rash
• Fluid buildup
• Liver problems
• Low blood cell counts (which can lead to increased risk of infections, bleeding or bruising easily, and feeling tired, weak, or short of breath)

Heart and blood vessel problems

This drug may cause heart or blood vessel problems, including an abnormal heart rate, a heart attack, or small strokes that last only a short time (known as transient ischemic attacks or TIAs).

It’s important to get medical help right away if you have symptoms such as shortness of breath, chest pain, a fast or irregular heartbeat, dizziness, or feeling faint.

Pleural and pericardial effusions

Fluid buildup around the lungs (pleural effusion) or heart (pericardial effusion) is a serious side effect that can happen with this drug. The fluid can be drained off with a needle, but it can build up again and the dose of dasatinib may need to be lowered.

Nilotinib

Nilotinib (Tasigna, Danziten) is another second-generation TKI that targets the BCR::ABL1 protein. This drug can be used as a first treatment for CML. It can also be used for people who can’t take imatinib or whose CML no longer responds to it.

It is taken as a pill or capsule, typically twice a day.

Side effects of nilotinib

Side effects of nilotinib can include:

• Nausea
• Diarrhea
• Muscle pain
• Fatigue
• Skin rash
• Fluid buildup
• Low blood cell counts (which can lead to increased risk of infections, bleeding or bruising easily, and feeling tired, weak, or short of breath)
• Decreased blood flow to the legs, heart, or brain
• Liver problems
• Blood chemical changes that may need to be treated (for instance, low potassium and magnesium levels)

It can also cause high blood sugar levels and pancreatitis (inflammation of the pancreas, which can lead to severe belly pain, nausea, and vomiting), but this is rare.

Prolonged QT syndrome

This drug can also affect your heart rhythm, causing a condition called prolonged QT syndrome. It usually doesn't cause any symptoms, but it can be serious or even fatal.

Because of this, it’s important to have an electrocardiogram (EKG) before starting nilotinib and again while being treated.

 Prolonged QT syndrome sometimes happens when nilotinib interacts with other drugs or supplements. It's especially important that your cancer care team knows about any medicines you take, including over-the-counter medicines, vitamins, herbs, and supplements.

Also check with your cancer care team before starting any new medicine, to be sure it's safe.

Bosutinib

Bosutinib (Bosulif) is another second-generation TKI that targets the BCR::ABL1 protein. It's an option as the first treatment for CML. It can also be used if another TKI is no longer working.

This drug is taken as a pill, typically once a day. It is often better tolerated if a person starts with a low dose and then increases to the full dose over several weeks.

Side effects of bosutinib

Common side effects of bosutinib can include:

• Nausea and vomiting
• Diarrhea
• Belly pain
• Fatigue
• Skin rash
• Fluid buildup
• Liver problems
• Low blood cell counts (which can lead to an increased risk of infections, bleeding or bruising easily, and feeling tired, weak, or short of breath)

Your cancer care team will check your blood test results regularly to watch for low blood counts and problems with your liver.

Less often, this drug can also cause heart problems, including congestive heart failure or heart attack.

Ponatinib

Ponatinib (Iclusig) is a TKI that binds to the BCR::ABL1 protein more tightly than some of the other TKIs.

This might make it more effective, but it can also lead to some serious side effects (see below). Because of this, ponatinib is typically only used if the other TKIs don’t work or if the leukemia cells have a gene change called a T315I mutation. Ponatinib works against CML cells that have this mutation.

This drug is taken as a pill, typically once a day.

Side effects of ponatinib

Side effects of ponatinib can include:

• Belly pain
• Headache
• Skin rash
• Joint pain
• Fluid buildup
• Fatigue
• Fever
• Nausea
• High blood pressure
• Nerve damage
• Eye problems
• Liver problems, which might be severe
• Pancreatitis (inflammation of the pancreas, which can lead to severe belly pain, nausea, and vomiting)
• Low blood cell counts (which can lead to an increased risk of infections, bleeding or bruising easily, and feeling tired, weak, or short of breath)

Blood clots

Ponatinib raises the risk of serious blood clots, which can lead to heart attacks and strokes or might block arteries and veins in the arms and legs. Surgery or another procedure may be needed to treat these blood clots.

The risk of serious blood clots is higher in older people; those with certain risk factors, such as high blood pressure, high cholesterol, or diabetes; and those who have already had a heart attack, stroke, or poor circulation. But they can happen to anyone taking this drug.

Heart problems

This drug may cause heart problems, including an abnormal heart rate, a heart attack, or a weakening of the heart muscle leading to a condition known as congestive heart failure (CHF).

Get medical help right away if you have symptoms such as shortness of breath, chest pain, a fast or irregular heartbeat, dizziness, or feeling faint.

Asciminib

Asciminib (Scemblix) targets a different part of the BCR::ABL1 protein than the other TKIs, so it might sometimes be helpful when other TKIs aren’t.

It can be used as the first treatment for chronic phase CML. It can also be used if other TKIs have already been tried or if the leukemia cells have a gene change called a T315I mutation. Asciminib works against CML cells that have this mutation.

This drug is taken as a pill, typically once a day or twice a day, depending on the dose.

Side effects of asciminib

Side effects of asciminib can include:

• Muscle, bone, or joint pain
• Skin rash
• Fatigue
• Nose, throat, or sinus infections
• Headache
• Belly pain
• Diarrhea
• High blood pressure
• Pancreatitis (inflammation of the pancreas, which can lead to severe belly pain, nausea, and vomiting)
• Low blood cell counts (which can lead to increased risk of infections, bleeding or bruising easily, and feeling tired, weak, or short of breath)
• Abnormal blood test results, including liver enzymes, cholesterol, and certain chemicals in the blood

Heart and blood vessel problems

Asciminib may cause heart or blood vessel problems, including an abnormal heart rate, heart attack, stroke, blood clots, and heart failure. Get medical help right away if you have symptoms such as shortness of breath, chest pain, a fast or irregular heartbeat, dizziness, or feeling faint.