Chronic myeloid leukemia is a blood cancer driven by a gene mutation that produces an abnormal protein called BCR-ABL. Tyrosine kinase inhibitors — pills that block this protein — have transformed CML from a fatal disease into one most people live with long-term. Current research focuses on stopping treatment safely, overcoming resistance, and eliminating residual cancer cells.
What's actually going on in research
Trials are testing newer tyrosine kinase inhibitors with fewer side effects, strategies for treatment-free remission after years on therapy, and drugs that target resistant mutations. Researchers are also studying combination approaches that might eliminate leukemia cells that persist despite treatment, and predictive markers that identify who can safely stop medication.
Treatment-free remission
Studies show about half of people who achieve deep remission on a tyrosine kinase inhibitor can stop treatment without relapse. Trials are refining the criteria for safe stopping and testing strategies to increase success rates.
Third-generation inhibitors
Asciminib and other newer drugs work differently than imatinib and second-generation inhibitors, potentially with fewer cardiovascular and metabolic side effects. They're being tested in people newly diagnosed and those who've developed resistance.
Eradicating leukemia stem cells
Even during remission, dormant CML cells persist and can cause relapse if treatment stops. Trials are testing drugs that target these stem-like cells, aiming for cure rather than lifelong treatment.
What to know before you search
Eligibility typically depends on disease phase (chronic, accelerated, or blast), previous tyrosine kinase inhibitor treatments, response level, and specific mutations if resistance has developed.
What types of trials are currently open
- First-line treatment trials — Comparing newer tyrosine kinase inhibitors to imatinib in newly diagnosed CML to see which achieves deeper remission faster.
- Resistance trials — Testing drugs for people whose CML no longer responds to their current tyrosine kinase inhibitor, often due to additional mutations.
- Treatment discontinuation trials — Carefully monitored studies where people in deep remission stop their medication to see if remission holds without treatment.
- Combination therapy trials — Adding a second drug to a tyrosine kinase inhibitor to try to eliminate residual leukemia cells and increase cure rates.
- Quality of life studies — Tracking side effects, fatigue, and daily functioning on different CML treatments to understand long-term tolerability.
Recently added Chronic Myeloid Leukemia trials
Receive antifungal medication to prevent serious infections during cancer treatment
This is a single-center, single-arm, observational clinical study evaluating the efficacy and safety of low-dose liposomal amphotericin B (50 mg/day, intravenous, once daily) for the prevention of invasive fungal infections in adult patients (aged 18-75 years) with hematological malignancies who develop prolonged neutropenia (absolute neutrophil count ≤0.5×10\^9/L, expected to last \>7 days) and are at high risk for invasive fungal disease. Participants are those who, per the treating physician's routine clinical decision, have been initiated on liposomal amphotericin B prophylaxis at 50 mg/day due to intolerance or toxicity to other antifungal agents. The primary outcome is the incidence of proven or probable invasive fungal disease. Secondary outcomes include incidence of pneumonia, persistent unexplained fever \>4 days, use of additional systemic antifungal therapy, and adverse events. A total of 30 participants will be enrolled. Data will be collected at baseline, during treatment, and within 7 days after treatment completion.
Receive a cell therapy infusion for chronic myeloid leukemia
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.
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