Chronic myeloid leukemia (CML) research is constantly advancing, leading to new and improved treatments that help improve the prognosis (outlook) of people living with the disease. New treatments now help people with blood cancers live longer, healthier lives. Scientists are not only developing new medications but also refining existing ones to make treatment plans more manageable and effective.

This article will explore four facts about the latest research on CML. We’ll cover newly available treatments and discuss what’s on the horizon. To learn more about what’s new in the CML field, talk to your cancer care team.

What We Currently Know About CML

CML, also known as chronic myelogenous leukemia, is a cancer that affects your myeloid cells — immature blood-forming cells in the bone marrow. Under normal conditions, myeloid cells develop into functional blood cells like:

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

In CML, cells develop a mutation (change in a gene) that forms an abnormal gene called BCR-ABL. Also known as the Philadelphia (Ph) chromosome, it makes immature myeloid cells that don’t work correctly. These leukemia cells grow and divide in a disordered way. Eventually, cancer cells crowd out healthy cells in your bone marrow and enter your bloodstream.

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

While many targeted therapies work well, some people become resistant over time. This means we need new and improved medications. Advances in CML research have now led to new treatments that enhance outcomes and quality of life. Here are four facts about the latest discoveries related to this cancer.

1. The FDA Has Approved New Drugs for CML

Drugs called tyrosine kinase inhibitors (TKIs) are the cornerstone of many cancer treatment plans. These targeted therapies specifically block proteins that help cancer cells grow and divide. TKIs previously approved to treat CML include imatinib (Gleevec) and nilotinib (Tasigna).

There are more TKI medications available to treat CML than ever before.

Recently, the U.S. Food and Drug Administration (FDA) approved a new TKI called asciminib (Scemblix) and expanded the use of the bosutinib (Bosulif) to include 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. Your leukemia cells must have the Ph chromosome in order for you to qualify for treatment. Studies showed that 68 percent of people responded to asciminib treatment. On the other hand, only 49 percent responded to other TKIs.

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 cells 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. Children must have chronic phase CML that’s positive for the Ph chromosome. To be eligible for treatment, the CML needs to be newly diagnosed or no longer responsive to other treatments.

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

Bosutinib was first approved in 2012 for adults with certain types of CML that no longer responded to other therapies, including:

• Chronic phase Ph-positive CML
• Accelerated phase Ph-positive CML
• Blast phase Ph-positive CML

2. A New Formulation of Nilotinib No Longer Requires Fasting

If you’ve ever taken the formulation of nilotinib (Tasigna), you’ll 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.

The FDA in November 2024 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.

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

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

3. New CML Treatments Are Currently in Clinical Trials

Unfortunately, some people with CML develop new genetic mutations over time. These can make their leukemia resistant to treatment. One mutation called T315I makes leukemia cells resistant to most TKIs.

Fortunately, researchers continue looking for new ways to target and destroy these cancer cells. Some TKIs, such as asciminib and ponatinib (Iclusig), may help improve treatment outcomes. Meanwhile, new drugs are currently in clinical trials to see if they’re safe and effective against CML that no longer responds to existing treatments.

Cancer vaccines are also a new and exciting 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 and exciting area of research in CML is whether people can safely stop treatment after a certain point after achieving remission.

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. It can also improve your overall quality of life. However, these potential benefits of stopping treatment must be weighed against 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 find out whether some people are good candidates for stopping treatment, while others should stay on therapy.

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

CML treatment decisions are best made in partnership with your oncologist (cancer specialist). Never stop taking your medications without consulting 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 investigative treatment. Check with your oncologist to see if you’re able to become part of a study. Not everyone qualifies, but there are always new trials looking for participants.

Find Your Team

On MyLeukemiaTeam, the social network for people living with leukemia and their loved ones, more than 20,000 members come together to ask questions, give advice, and share their stories with others who understand life with leukemia.

Have you asked your oncology team about recent advances in chronic myeloid leukemia research? Would you be comfortable trying a newly approved treatment? Share your questions and experiences in a comment below, start a conversation on your Activities page, or connect with like-minded members in Groups.