Researchers are exploring new treatment options for chronic myeloid leukemia (CML). Many treatments that have recently been studied or are currently under investigation are targeted therapy drugs.

CML is a slow-growing cancer that starts in bone marrow cells that produce blood cells. Because CML typically progresses slowly, the survival rate tends to be better than the rates for many other types of cancer.

In the United States from 2014 through 2020, the 5-year relative survival rate for CML was 70%. This means that, according to the available data, people with CML lived for at least 5 years 70% as often as people without CML.

Let’s examine the latest treatments approved to treat CML and some drugs that are currently being studied for treating this cancer.

Where’s the research at now for CML?

Researchers are currently examining how specific gene changes in CML cells can help determine how best to treat the cancer.

Targeted therapy drugs called tyrosine kinase inhibitors (TKIs) that target the BCR-ABL protein seem to work very well for many people, and several of these drugs have recently been approved by the Food and Drug Administration (FDA).

Researchers are examining new treatment options for CML that does not respond to these drugs. They’re also studying the potential effectiveness of several cancer vaccines that help your immune system react to cancer cells.

The Food and Drug Administration (FDA) granted accelerated approval to asciminib for newly diagnosed Philadelphia chromosome-positive CML in October 2024. Asciminib is a tyrosine kinase inhibitor (TKI) targeted therapy drug sold under the brand name Scemblix. Philadelphia chromosome-positive CML is a subtype of CML that makes up the majority of cases.

In September 2023, the FDA granted approval to bosutinib for use in children ages 1 year and older with chronic phase Philadelphia chromosome-positive CML. Bosutinib is another TKI targeted therapy drug.

The FDA approved the TKI targeted therapy drug nilotinib for use in children with newly diagnosed or resistant/intolerant Philadelphia-positive CML in the chronic phase in 2018. The chronic phase of CML is the mildest and earliest stage.

Also, the FDA approved dasatinib for use in children with Philadelphia chromosome-positive CML in 2017. Dasatinib is another TKI targeted therapy drug.

Clinical trials are studies comparing new treatments to current standard treatments. If new treatments are more effective, they become the new standard treatments.

Here’s some information about recently completed clinical trials for CML:

  • In a clinical trial completed in June 2024, researchers evaluated asciminib by itself for chronic phase CML with and without the T315I mutation, a mutation that’s found in most people with CML.
  • In a clinical trial completed in 2022, researchers evaluated the effectiveness of azacitidine with or without glasdegib in people with previously untreated acute myeloid leukemia, myelodysplastic syndrome, or chronic myelomonocytic leukemia.
  • In a clinical trial completed in 2020, researchers compared the effectiveness of bosutinib and imatinib for treating chronic phase CML.
  • A clinical trial completed in 2022 examined the effectiveness of cyclophosphamide/tacrolimus/mycophenolate mofetil vs. tacrolimus/methotrexate for preventing graft-versus-host disease after stem cell transplant.
  • A clinical trial completed in 2021 examined the effectiveness of dasatinib as a first-line treatment for newly diagnosed chronic phase CML.

Here are some of the clinical trials for CML that are in progress, as of January 2025:

  • A phase 3 clinical trial is examining the effectiveness of olverembatinib in people with chronic phase CML.
  • Another phase 3 clinical trial is examining the effectiveness of asiminib with imatinib compared to imatinib alone to treat chronic phase CML.
  • A third phase 3 clinical trial is comparing the effectiveness of asciminib to the effectiveness of bosutinib in treating chronic phase CML in people who were previously treated with at least two other TKIs.
  • A phase 4 clinical trial is examining the effectiveness of flumatinib vs. imatinib for newly diagnosed chronic phase CML.

Clinical trials often offer new treatments that may be more effective than current treatments. However, there’s no guarantee that you’ll receive the new drug. It depends on the design of the study and whether you’re placed in a control group.

When deciding whether to participate in a clinical trial, it’s important to talk with your doctor about the potential benefits and drawbacks. Clinical trials may be a good option for some people, such as those with cancer that is unlikely to respond to standard treatments.

Your doctor may be able to alert you about any clinical trials in your area that might be an option for you. Other places you can find clinical trials include:

There are six standard treatments currently used for CML:

Can you live 20 years with CML?

CML is generally a slow-growing cancer that can be controlled for many years. Some people with CML live for 20 years or longer after their diagnosis. About 85% of people with the most common type of CML live for at least 10 years.

Can CML go into remission?

In some people, CML goes into remission for many years. Remission means you don’t have any signs or symptoms of cancer on your tests.

Is there a cure for CML?

Allogenic stem cell transplant is currently the only potentially curative treatment for CML. This procedure involves transplanting donated stem cells and is usually offered only to younger people with good overall health.

Many TKI targeted therapy drugs have received FDA approval to treat CML in recent years. Researchers are continuing to examine treatments for people who don’t respond to these drugs.

Your doctor can help you determine the best treatment options for you. They can also help you find any clinical trials you may be eligible for in your area.