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Chronic myeloid leukemia (CML) is being studied in labs and in clinical trials around the world. Each year, researchers learn more about what causes the disease, how to test for it, and how to better treat it.

Research into the genetics of CML

Scientists have made great progress in understanding how changes in the DNA (genes) inside normal bone marrow cells can cause them to develop into CML cells. This has provided insight into why these cells grow too quickly, live too long, and fail to develop into normal blood cells.

Perhaps most importantly, this explosion of knowledge has been used to develop many new, effective drugs to treat CML (see below).

Researching new medicines for CML

The treatment of CML has been transformed over the last few decades with the development of drugs called tyrosine kinase inhibitors (TKIs) that specifically target the abnormal BCR::ABL1 protein, which drives the growth of CML cells.

Choosing the best targeted drug in each situation

TKIs such as imatinib, dasatinib, nilotinib, bosutinib, and others have proven to work very well for most people with CML. But they may not work for everyone, and sometimes they might stop working over time. Doctors are learning more about why this happens.

Each TKI is slightly different, and some people’s CML cells might have (or develop) certain gene changes that make the cells less likely to respond to one or more of these drugs. Doctors can now test the cells for many of these changes, which can help determine which TKIs are likely to be helpful.

This might be especially useful in people whose CML is still growing while getting a TKI, to determine which one should be tried next.

Doctors are also looking at combining TKIs with other types of drugs to see if this might be more effective than a TKI alone.

Looking at whether TKI treatment can be stopped

CML can often be treated effectively with TKIs, but it’s been hard to tell if people can truly be cured with these medicines. Most people getting TKIs need to take them every day, and like many drugs used to treat cancer, these drugs can have bothersome side effects.

An active area of research is looking at whether TKI treatment can be stopped in some people, without the CML coming back.

Studies have shown that trying to stop TKI treatment is most likely OK for people who meet certain criteria, such as having had a good response to a TKI for at least 3 years.

Doctors continue to deepen their understanding of:

• Which people might be the best candidates for stopping treatment
• How (and how often) they should be watched for possible signs of the CML coming back
• What should be done if that happens

Testing newer drugs for CML

Because CML can become resistant to TKIs over time, scientists continue to look for new drugs to treat CML, especially CML that no longer responds to TKIs.

Many kinds of drugs are being tested in clinical trials, including immunotherapy drugs. These might also be given along with TKIs in hopes of getting a better response than is seen with TKIs alone.