ABL1 (ABL proto-oncogene 1)

P1/2, N=20, Recruiting, Princess Maxima Center For Pediatric Oncology

By integrating multiple -omics modalities, we identify not only features of interest but also begin to unravel the regulatory interactions driving subtype-specific mechanisms of leukemogenesis. This integrated analytic approach paves the way for enhanced precision medicine for precise subtyping and treatment selection for pediatric leukemia patients.

Shared identity with PCPs-but not oncologists-was associated with improved adherence to molecular monitoring guidelines in CML. In chronic cancer care within integrated health care systems, PCPs are well positioned to reinforce oncology-driven testing plans. These findings highlight the importance of fostering patient-centered relationships and promoting workforce diversity in primary care, particularly given that historically disadvantaged patients experienced the greatest gains in adherence.

The introduction of imatinib in the early 2000s revolutionized CML treatment, transforming a fatal disease into a chronic condition with near-normal life expectancy for most patients...Emerging therapies such as vamotinib, KF1601, and combination regimens are being explored. Furthermore, new insights into non-kinase functions of BCR::ABL1 and the role of microRNAs in resistance open additional therapeutic avenues. This review provides a concise overview of CML from a historical and molecular perspective, highlighting diagnostic advances, evolving response criteria, and future directions in treatment.

Overall, close cooperation between human genetics and internal medicine can substantially contribute to an improvement in treatment success. A better understanding of molecular disease mechanisms enables an increasingly more precise, individualized and effective treatment.

Routine TDM was most frequently used for classical agents (methotrexate, cyclosporin A), antifungals, and antibiotics, but substantial interest was reported for targeted therapies, including BCL-2 inhibitors, BCR-ABL tyrosine kinase inhibitors, FLT3 inhibitors, and Bruton tyrosine kinase inhibitors. While firmly established for classical agents and anti-infectives, clinicians express growing interest in extending TDM to targeted therapies. Optimizing turnaround times, expanding assay availability, and integrating pharmacokinetics-informed dosing into clinical trials may further clarify the role of TDM within precision medicine approaches in hematology.

Adoption remains constrained by RNAseq availability, expertise and lesion-centric regulation. Prospective studies are needed to establish the clinical utility of newly described phenocopies, extending the advances made in subclassification and targeted treatment of BCR::ABL1-like ALL.

P1, N=30, Recruiting, Andrew E. Place, MD | Active, not recruiting --> Recruiting | N=13 --> 30 | Trial completion date: Jul 2028 --> Jul 2030 | Trial primary completion date: Jul 2026 --> Jul 2028

Although alternative treatment options exist, side effects may persist, or new ones occur after a therapy switch. Using a case-based approach, this review examines the incidence of non-hematologic and hematologic TEAEs with specific therapies, provides guidance on AE management, and describes the impact of therapy dose reduction on efficacy and tolerability.

P3, N=406, Active, not recruiting, Novartis Pharmaceuticals | Trial completion date: Jan 2028 --> Jan 2031

P2, N=60, Not yet recruiting, M.D. Anderson Cancer Center | Trial completion date: Sep 2031 --> Sep 2033 | Initiation date: Feb 2026 --> Aug 2026 | Trial primary completion date: Sep 2026 --> Sep 2031

The 6X-His-SUMO-tag of purified ABL1 was cleaved by ULP1 protease. The recombinant ABL-1 was subsequently used in crystallization trials to enlighten structural features of ABL-1 that could guide the development of novel therapeutics and drug screening platforms targeting ABL-1 in CML.