Chronic myeloid leukemia (CML) is driven almost entirely by a single genetic change — the BCR-ABL fusion gene — that tyrosine kinase inhibitors (TKIs) can control so effectively that most patients now have near-normal life expectancy. Research has shifted toward achieving treatment-free remission and managing the rare patients who resist all TKIs.
What's actually going on in research
Third-generation TKIs like asciminib specifically target the BCR-ABL myristoyl pocket rather than the ATP-binding site, overcoming resistance to earlier drugs including ponatinib. Treatment-free remission — stopping TKIs and maintaining deep molecular response — is now achievable for many patients and is being optimized in trials. Emerging approaches using immune therapies to eradicate residual CML stem cells that hide from TKIs are in early trials.
Asciminib (STAMP inhibitor)
Asciminib targets BCR-ABL through a completely different binding site than prior TKIs, producing responses in patients resistant or intolerant to two or more earlier therapies.
Treatment-free remission
Trials are defining who can safely stop TKI therapy after achieving deep molecular remission and what predicts durable response-free status.
CML stem cell eradication
CML stem cells persist even with deep molecular remission. Trials are testing immunotherapy and combination approaches that may eliminate this reservoir to achieve genuine cure.
What to know before you search
Eligibility depends on CML phase (chronic, accelerated, blast), specific BCR-ABL kinase domain mutations, prior TKI history, and molecular response depth.
What types of trials are currently open
- TKI optimization trials — Comparing different TKIs as initial therapy and testing newer generation drugs in resistant or intolerant patients.
- Treatment-free remission trials — Testing criteria and monitoring strategies for safely stopping TKI therapy.
- Resistant CML trials — Evaluating options for T315I and compound mutations resistant to available TKIs.
- Stem cell eradication trials — Testing immunotherapy combinations to eliminate residual CML stem cells and achieve deeper remissions.
- Observational studies — Tracking long-term cardiovascular effects of TKIs and quality of life on treatment.
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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