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
Cohort of Patients With Chronic Myelomonocytic Leukemia
A prospective, non-interventional study involving the collection of biological samples with associated anonymised data. Biological samples will be collected at the time of diagnosis and/or as the disease progresses, with or without treatment, always in conjunction with tests scheduled as part of routine care or within the context of a clinical trial.
Real-world Study of Scemblix in the Treatment of Chronic Myeloid Leukemia in China
This is a multicenter, non-interventional real-world study designed to assess the efficacy and safety of asciminib in patients with newly diagnosed CML.The study uses a prospective data collection design to gather baseline, pre- and post-treatment, and long-term follow-up data, enabling a comprehensive assessment of asciminib's clinical benefits.
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