Chronic Myeloid Leukemia

FTO inhibitors (e.g., CS1, FB23-2) appreciably impair the growth of resistant cells either alone or in combination with nilotinib...These findings offer new insights into cancer drug resistance and advance the application of RNA nanotechnology for treating leukemia. The research provides a foundation for developing novel, targeted therapies for resistant leukemia.

P2, N=45, Not yet recruiting, University Health Network, Toronto

P=N/A, N=600, Recruiting, Novartis Pharmaceuticals | N=100 --> 600 | Trial completion date: Jul 2025 --> Jun 2030 | Trial primary completion date: Jul 2025 --> Jun 2030

Overall, this work applies bioisosteric replacement to generate a new chemotype and uncovers a mechanistically divergent lead. The distinct, ABL1-independent mechanism of compound 2a establishes a solid foundation for future optimization and highlights its potential as a starting point for developing novel antimyeloproliferative agents with a different therapeutic profile.

The initial treatment approach emphasized cytoreduction therapy with hydroxyurea, intravenous fluid administration, and preventive medication with allopurinol to protect against the risk of tumor lysis syndrome. After the patient became stabilized, imatinib, a first-line tyrosine kinase inhibitor, was started...As highlighted by this case, the importance of prompt diagnosis, the initiation of cytoreduction therapy, and the use of molecular therapy in treating CML in children cannot be neglected. CML in children is an uncommon but curable form of leukemia.

Single-cell DNA sequencing suggested that the SF3B1 mutation likely preceded BCR::ABL1 and blunted the expected granulopoiesis, thereby explaining the myelodysplastic syndrome phenotype without leukocytosis. This case illustrates how single-cell analysis can reveal meaningful clonal interactions that would not be evident with traditional bulk sequencing.

P4, N=85, Completed, Novartis Pharmaceuticals | Active, not recruiting --> Completed | Trial completion date: Nov 2026 --> Mar 2026 | Trial primary completion date: Nov 2026 --> Mar 2026

P1, N=50, Not yet recruiting, Stanford University | Initiation date: Feb 2026 --> Jun 2026

In the appropriate clinical context, strictly defined Harlequin cells on routine peripheral blood smears may serve as a sensitive and highly specific morphologic clue for CML. Recognition of this readily accessible feature may facilitate prompt BCR::ABL1 confirmatory testing, reduce diagnostic ambiguity, and help avoid unnecessary ancillary studies.

Subsequently, the CML burden declined as the AML clone regrew. This case highlights the importance of accurately assessing clonal changes using genetic analysis when implementing molecular targeted therapy for hematologic malignancies.

P=N/A, N=600, Recruiting, Pfizer | Active, not recruiting --> Recruiting

P=N/A, N=2000, Recruiting, Wake Forest University Health Sciences | Trial completion date: Jun 2026 --> Dec 2026 | Trial primary completion date: Jun 2026 --> Dec 2026