If you’ve been diagnosed with CML, it’s important to know what the best drug for you is. This is because the treatment you get will depend on your age, how advanced your disease is and whether you can have a stem cell transplant.
The best treatment for most people with chronic phase (CP) CML is a tyrosine kinase inhibitor (TKI). Medicines like imatinib, nilotinib, and dasatinib are used.
Imatinib
Imatinib, also known as Gleevec, is the most commonly used drug for CML. It works by stopping the BCR-ABL protein that causes CML from working so that it cannot grow and kill healthy cells.
Imatinib is given by mouth in tablets, usually once or twice a day, and is very effective in treating CML. Nearly all patients with chronic phase CML have their blood counts return to normal and their spleen shrinks after taking imatinib.
When your doctor prescribes this drug, you will be given a low dose of it and you may have blood tests to check how well it is working. You will be advised to take it with food and plenty of water to prevent gastrointestinal (GI) irritation.
Some people have side effects such as fluid retention and weight gain while on imatinib, which can be very serious. You may need to take drugs that make you pass more urine (diuretics) to help control these symptoms.
Another common side effect of imatinib is a rash, which can be mild or serious. You should tell your doctor if you have a rash, especially if it is red or itchy and gets worse.
Your doctor can give you advice on skin creams and lotions to use if you have a rash. You should not sunbathe or spend time outdoors if you have taken imatinib because your skin can become very sensitive to sunlight.
Nilotinib
If a patient has been diagnosed with CML and is able to take a TKI (see above), they are very likely to have their disease controlled for several years. It may even be possible to cure leukemia.
The best drug for CML is nilotinib, also known as Tasigna. It is a tyrosine kinase inhibitor (TKI), which blocks the BCR-ABL protein. This prevents leukemia cells with an abnormal chromosome called the Philadelphia chromosome from growing.
In a recent clinical trial of patients with newly diagnosed chronic phase CML, nilotinib showed similar efficacy to imatinib in the first year of treatment. Moreover, the majority of patients experienced a major response to the drug.
Nilotinib is a more selective inhibitor of BCR-ABL than other TKIs, and its concentration required to inhibit cell growth in vitro is manyfold lower. This makes nilotinib safer to use than other TKIs, which means it is less likely to cause serious side effects.
This is important because side effects can include low blood counts, rash, headache, nausea, and diarrhea. The most serious of these side effects can be heart and blood vessel problems.
This type of long-term treatment can be difficult for the patient. However, it is a necessary part of controlling leukemia and may help to ensure leukemia doesn’t come back. If this type of treatment is unable to control leukemia, it is often recommended that the patient have an allogeneic stem cell transplantation.
Dasatinib
CML is a blood cancer that starts in the bone marrow. Treatment usually starts with a drug called a tyrosine kinase inhibitor (TKI). These drugs target a protein called BCR-ABL and can help to keep cancer from coming back.
The first drug for CML was imatinib (Gleevec). It revolutionized the way that people with this condition were treated and changed the way that they lived their lives. However, it can sometimes be hard to cope with the side effects of imatinib. Fortunately, newer drugs called dasatinib and nilotinib have also been developed.
They work by targeting a specific protein that is found in the BCR-ABL fusion gene. When the protein is blocked, the cancer cells cannot grow.
Currently, dasatinib is used for chronic phase CML and for patients who are resistant to imatinib or who cannot tolerate the side effects of imatinib. It is taken daily and you will have regular blood tests to check how well it is working.
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Bosutinib
Bosutinib (Bosulif) is one of the drugs used to treat CML. It works by blocking a protein made by CML cells that have the Philadelphia chromosome. This stops them from growing.
It comes as tablets that you take once a day with food. It must be taken exactly as your doctor tells you to. It may cause some side effects, including abdominal (belly) pain and headache. You may also have high blood pressure or diarrhea.
This is usually mild and can be controlled by taking the right amount of the drug. If your blood pressure is very high, your doctor might reduce the dose until it is back to normal.
Other side effects that are usually mild include low blood counts, rashes, and fatigue. You may need to have regular blood tests during treatment so that your doctor can monitor your condition.
If you have very low blood counts, your doctor might reduce the dose of bosutinib until your blood count is higher. Your doctor may also give you a drip to help you have enough red and white blood cells.
The first goal of treatment is to achieve a complete cytogenetic response, which means that your blood has no evidence of CML cells with the Philadelphia chromosome. This can be done with a bone marrow test, or after 6 to 12 months of treatment.
Ponatinib
Ponatinib is a drug that treats chronic phase, accelerated phase, or blast phase CML and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in patients who have used at least 2 other treatments that didn’t work well. It’s also used to treat people who have a type of CML with a gene change called the T315I mutation, which makes them resistant to other drugs.
This medicine blocks a special protein in the body called BCR-ABL tyrosine kinase. It also helps prevent the cancer cells from multiplying and spreading.
The FDA approved this medicine in 2012 for use as an initial treatment for people with newly diagnosed or relapsed CML, and when other drugs don’t work. It’s a pill that’s taken once a day.
Doctors may adjust the dose of this medication or stop it temporarily if you have problems with your blood counts. This can happen if you have anemia, low levels of white blood cells or platelets called neutropenia and lung problems like fluid around your lung or pulmonary hypertension.
Another medicine that doctors sometimes use with this drug is a fusion protein that stops CML from forming in your bone marrow. It’s a type of drug called a cytotoxic tyrosine kinase inhibitor, or TKI.
TKIs block the BCR-ABL tyrosine-kinase enzyme so that the CML cells die quickly. They can also help prevent leukemia from recurring. They can be used with other drugs, such as chemotherapy, or in combination with a stem cell or bone marrow transplant.
Asciminib
CML can stay under control with a drug called a tyrosine kinase inhibitor (TKI). Treatment is usually given to people in the chronic phase of the disease, and it often works well for years.
The most common type of TKI used for CML is imatinib (Glivec). Some doctors will also try other TKIs, such as dasatinib (Sprycel) or nilotinib (Tasigna). If these drugs do not work, your doctor may recommend other drugs or treatments, such as a drug called omacetaxine mepesuccinate (Synribo) that stops the growth of cancer cells.
A newer drug, asciminib, is a more advanced treatment for people with chronic-phase CML who haven’t responded to other TKIs. It has been shown to work in both people with a T315I mutation and those without.
Asciminib is a small pill that you take twice daily for a few months. It works by blocking the tyrosine kinase activity of the BCR-ABL1 gene. It can help stop the CML from growing and improve the quality of life.
It is a good choice for patients who haven’t responded to other drugs or who can’t take a drug that they are allergic to. It can also improve the chances of a complete hematologic response or a major molecular response, which means that there are no active leukemic cells in your body.
It is a newer drug that binds to a myristoyl pocket in the ABL protein. It’s the first FDA-approved CML treatment that does this.
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