Chronic myeloid leukemia (CML) research continues to lead to new treatment options that may improve the outlook for people living with the disease.

New treatments may help some people with blood cancers live longer, healthier lives. Scientists are developing new medications and improving existing ones to help make treatment plans easier to follow and more effective.

This article covers four facts about the latest CML research, including new treatment options and what may be coming next. To learn more about what’s new in the CML field, talk to your cancer care team.

What We Know About Chronic Myeloid Leukemia

CML, also called chronic myelogenous leukemia, is a cancer that affects myeloid cells. These are young cells in the bone marrow that make blood.

Under normal conditions, myeloid cells develop into healthy blood cells, such as:

• Red blood cells, which carry oxygen throughout the body
• White blood cells, which help fight infections
• Platelets, which help in blood clotting to prevent severe bleeding

In CML, cells develop a mutation, or a change in a gene, that creates an abnormal gene called BCR-ABL.

This change is also called the Philadelphia (Ph) chromosome. It causes young myeloid cells to grow out of control and not work correctly.

These leukemia cells grow and divide in a disordered way. Eventually, cancer cells crowd out healthy cells in your bone marrow and get into your bloodstream.

TKIs have changed how doctors treat CML, and researchers continue to study better ways to use these medications.

Most CML treatments focus on blocking abnormal genes and proteins to slow the cancer’s growth and spread. Chemotherapy and bone marrow transplants are also options for some people with CML.

Many targeted therapies work well, but they may stop working for some people over time. That’s why researchers continue to study new and improved medications.

Research on CML has led to new treatments that may help some people live longer and feel better. Here are four facts about recent research related to this type of cancer.

1. The FDA Has Approved New Drugs for CML

Drugs called tyrosine kinase inhibitors (TKIs) are a key part of many cancer treatment plans. These targeted therapies block proteins that help cancer cells grow and divide.

TKIs previously approved to treat CML include imatinib (Gleevec) and nilotinib (Tasigna).

Recently, the U.S. Food and Drug Administration (FDA) approved a new TKI called asciminib (Scemblix) and expanded the approval of bosutinib (Bosulif) for use in infants and children with CML.

Asciminib

The FDA approved asciminib in October 2024. This medication treats people with newly diagnosed CML in the chronic phase, when the cancer is usually growing more slowly. Your leukemia cells must have the Ph chromosome in order for you to qualify for treatment.

Studies showed that asciminib worked for 68 percent of people. In comparison, other TKIs worked for 49 percent of people.

Asciminib is typically taken orally (by mouth) twice daily. The most common side effects seen in clinical trials included:

• Thrombocytopenia (low platelet count)
• Neutropenia (low white blood cell count)
• COVID-19 infection
• Diarrhea
• Fatigue (tiredness that does not improve with rest)
• Muscle pain

Expanded Approval of Bosutinib

The FDA also approved bosutinib in September 2023 for treating infants and children ages 1 and older with CML. To take bosutinib, children must have chronic phase CML that’s positive for the Ph chromosome.

To receive this treatment, a child must have newly diagnosed CML, or other treatments must have stopped working.

The most common side effects of bosutinib in infants and children include:

• Abdominal pain
• Diarrhea or constipation
• Skin rashes
• Nausea and loss of appetite
• Liver function problems
• Fever
• Headache

Most TKI drugs must be taken on an empty stomach. A newly approved form of nilotinib can be taken at any time, regardless of when you last ate.

Bosutinib was first approved in 2012 for adults with Ph-positive CML that no longer responded to other therapies. It may be used in different phases of CML, including:

• Chronic phase
• Accelerated phase
• Blast phase

2. A New Formulation of Nilotinib No Longer Requires Fasting

If you’ve ever taken nilotinib (Tasigna), you know the medication needs to be taken on an empty stomach. This is because food can increase how much of the drug your body absorbs, leading to potentially dangerous effects on the heart. As a result, doctors strictly advise fasting before and after taking Tasigna.

In November 2024, the FDA approved a new formulation of nilotinib (Danziten), which can be taken with or without food. Researchers hope that removing the mealtime restriction will help people stick with their treatment.

Danziten is approved for:

• Newly diagnosed, chronic phase Ph-positive CML
• Chronic phase or accelerated phase Ph-positive CML that is resistant to other treatments

Common side effects of Danziten include:

• Nausea
• Headache
• Fatigue
• Vomiting and diarrhea
• Fevers
• Night sweats

Read more about how fasting affects people taking CML treatment.

3. New CML Treatments Are Currently in Clinical Trials

Over time, some people with CML develop new gene changes. These changes can make leukemia harder to treat. One mutation called T315 can stop most TKIs from working against leukemia cells.

Fortunately, researchers continue looking for new ways to target and destroy these cancer cells. Some approved TKIs, such as asciminib and ponatinib (Iclusig), may help treat certain types of CML, including CML that has stopped improving with other treatments.

Meanwhile, new drugs are in clinical trials to see if they’re safe and effective against CML that no longer responds to current treatments.

Studies are looking into whether it’s safe to stop TKIs at some point, and if so, when and for whom.

Cancer vaccines are also a new area in CML research. Traditional vaccines teach the immune system to recognize bacteria and viruses so the body can fight off infections.

A cancer vaccine would work similarly, but instead of preventing disease, it could train your immune system to recognize and attack leukemia cells. Researchers are currently investigating whether cancer vaccines can help treat CML.

Immunotherapies also help your immune system recognize and destroy cancer cells. These medications block certain proteins to activate immune cells against leukemia cells.

The American Cancer Society notes that researchers are studying a combination of immunotherapy with TKIs. The goal is to find a drug combination that will be more effective than TKIs alone.

4. Studies Are Testing Whether TKI Therapy Can Be Stopped

A new area of CML research looks at whether some people can safely stop treatment after they’ve been in remission, meaning their cancer is under control and signs of CML are very low or cannot be found.

If you’re in remission for a few years, you may be able to stop taking your TKI. Studies show that stopping treatment can help you avoid unwanted side effects.

Stopping treatment can also improve your overall quality of life. However, these potential benefits of stopping treatment need to be weighed against the potential risks of CML becoming active again.

The studies on stopping CML treatment are only a few years old. There are still ongoing clinical trials to learn what may happen over time. For example, researchers still need to learn how to treat any CML that comes back.

It’s also important to learn how to tell who may be able to stop treatment and who should stay on therapy.

CML treatment decisions are best made with your oncologist (cancer specialist). Never stop taking your medications without checking with your doctor.

Ask Your Cancer Care Team About Recent Advances

After your CML diagnosis, it’s key to keep in close contact with your cancer care team. Oncologists stay up to date on the latest research and new treatments.

Your team may have suggestions for newer therapies that could work better for you or be easier to keep taking.

If you’re eligible, you may also want to join a clinical trial to try an investigational treatment — one that’s still being studied.

Check with your oncologist to see if you’re able to join a study. Not everyone qualifies, but there are always new trials looking for participants.

Join the Conversation

On MyLeukemiaTeam, people share their experiences with leukemia, get advice, and find support from others who understand.

Have you talked with your oncology team about new CML treatments? Would you try one? Let others know in the comments below.