Chronic myeloid leukemia is a blood cancer driven by a genetic abnormality called the Philadelphia chromosome. Most people now take a daily pill called a tyrosine kinase inhibitor, which keeps the disease under control for years. The question has shifted from survival to whether some people can stop treatment safely.
What's actually going on in research
Trials are testing newer tyrosine kinase inhibitors that may work when earlier ones fail, combinations that might allow treatment-free remission, and drugs targeting leukemia stem cells that persist even when the disease is undetectable. Researchers are also studying which patients can safely stop their pills after years of deep response.
Treatment-free remission
Studies show that some people who achieve deep, sustained responses can stop their tyrosine kinase inhibitor and remain in remission. Trials are testing which patients are candidates and how to predict who will relapse.
Leukemia stem cells
Even when CML is undetectable, a small number of leukemia stem cells remain dormant. New drugs aim to eliminate these cells, which could allow more people to stop treatment permanently.
Resistance mechanisms
Trials are testing fourth-generation inhibitors and combination approaches for people whose disease develops resistance to current drugs. Understanding resistance patterns helps match people to the right next treatment.
What to know before you search
Eligibility typically depends on disease phase (chronic, accelerated, or blast), prior tyrosine kinase inhibitors tried, molecular response level, and whether specific resistance mutations are present.
What types of trials are currently open
- TKI comparison trials — Testing newer tyrosine kinase inhibitors against older ones to see if they control CML more quickly or with fewer side effects.
- Treatment discontinuation trials — Studies of stopping tyrosine kinase inhibitors after achieving deep molecular response, with careful monitoring for relapse.
- Combination trials — Testing a tyrosine kinase inhibitor plus another drug to deepen responses or target resistant disease.
- Stem cell targeting trials — Studies of drugs that aim to eliminate the dormant leukemia stem cells that persist during remission.
- Resistance trials — Testing treatments for CML that no longer responds to standard tyrosine kinase inhibitors, including newer-generation drugs and alternate approaches.
Recently added Chronic Myeloid Leukemia trials
Phase I Trial of Vididencel in CML-CP Patients With MRD Under TKI Treatment
The main purpose of the trial is to evaluate the safety and potential side effects of cell therapy vididencel in Chronic Myeloid Leukemia (CML) participants with measurable residual disease (MRD) unable to stop treatment with tyrosine kinase inhibitor (TKI). Patients may not receive any direct medical benefit from participating. Participants in the study should have been treated with TKI for at least 24 months prior to enrolment. This TKI must be of the same type throughout the 24 months. Participation in the active period of the study will take about 5 months after which patients will be followed with regular checks for a total of 3 years from the study start. The cell therapy is administered as 2 low-volume intradermal injections.The first 4 treatments will be performed once every two weeks over a period of 6 weeks. Further treatments are given 14 and 18 weeks after the start of participation as 1 low-volume intradermal injection.The belief is that addition of the study treatment (vididencel) to tyrosine kinase inhibitor therapy may potentially strengthen the immune defence so that enough leukemic cells are killed that the TKI treatment can eventually be decreased in dose or even stopped permanently, without the CML progressing again.
Impact of Targeted Therapy on Cancer-Related Cognitive Impairment
This study examines whether there are differences in brain health or well-being in patients receiving TKI therapy for leukemia compared to individuals who do not receive TKI therapy.
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