Research into CML

All cancer treatments have to be fully researched before they can be used for everyone. This is so we can be sure that:

  • they work
  • they work better than the treatments already available 
  • they're known to be safe

Research has helped to improve survival for CML, particularly over the last 20 years with the development of targeted cancer drugs called tyrosine kinase inhibitors (TKIs), such as imatinib. Researchers continue to learn about CML and are developing new treatments or combinations of treatments to further improve survival and quality of life.

Cancer Research UK has information about UK trials looking into CML.

Genetics of leukaemia cells

Scientists continue to study the changes that happen in the genes (sections of DNA) of leukaemia cells to learn more about how they develop, and why they can become resistant to treatment. Understanding this helps researchers develop new drugs.

Another area of research is to identify markers that can help predict response to treatment at the time of diagnosis.

Targeted cancer drugs

Targeted cancer drugs change the way that cells work, for example, they can block signals that tell cells to grow. There are different types of targeted cancer drugs. Some are described as cancer growth blockers. Cancer growth blockers called tyrosine kinase inhibitors (TKIs) are the main type of treatment for most people with CML.

Doctors currently use imatinib, nilotinib, dasatinib, bosutinib and ponatinib to treat CML. Researchers continue to look into these drugs to improve treatment results. Trials are looking at;

  • trying different doses of TKIs
  • giving a TKI with other treatments for CML
  • finding out if one of the TKIs is better than the others as a first treatment for CML
  • trying these TKIs in children and young adults
  • learning more about the side effects

Researchers around the world are looking at other targeted cancer drugs for CML including asciminib, axitinib and ruxolitinib.

Stopping TKIs

Until recently, it was thought that people with CML would need to stay on TKI treatment for life. Recent studies have shown that TKIs can be stopped in certain people who have had a good response to treatment for a number of years. They need frequent monitoring to check for it coming back. But more research and guidelines are needed to show when it is safe to stop treatment, how best to monitor patients, and what treatment to give if the CML comes back.

Stem cell transplants

High dose chemotherapy followed by donor stem cells (transplant) can cure some people with CML. This is very intensive treatment and some people do not survive the complications.

Your specialist will take into account your general fitness and age before doing a transplant. 

Half matched transplants

Doctors can't find a stem cell match for about 1 in 3 people who need a transplant. So for these people an option might be to have a half matched or haploidentical stem cell transplant. This transplant uses donor stem cells from a family member who is at least a 50% match. Researchers are looking at a better way to give this treatment and reduce the side effects.

Last reviewed: 
28 May 2019
  • Chronic Myeloid Leukaemia: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow up
    A. Hochhaus and others
    Annals of Oncology, 2017. Volume 28, Supplement 4, Pages 41-51

  • Chronic myeloid leukaemia: First line drug of choice
    E Jabbour
    American Journal of Hematology, 2016. Volume 91, Issue 1, Pages 59-66

  • Discontinuation of dasatinib or nilotinib in chronic myeloid leukemia: interim analysis of the STOP 2G-TKI study
    D Rea and others
    Blood, 2017. Volume 129, Issue 7, Pages 846-854

  • When to Stop Tyrosine Kinase Inhibitors for the Treatment of Chronic Myeloid Leukemia
    P Laneuville
    Current Treatment Options in Oncology, 2018. Volume 19, Issue 3

Related links