cytarabine

P2, N=90, Not yet recruiting, SWOG Cancer Research Network

P1, N=16, Active, not recruiting, Children's Oncology Group | Trial completion date: Mar 2026 --> Dec 2026

This case highlights the diagnostic complexity of composite indolent B-cell lymphomas and underscores the importance of integrated clinical, morphologic, immunophenotypic, and molecular assessment to inform management and surveillance.

P1/2, N=67, Completed, Fondazione Gimema Franco Mandelli Onlus | Active, not recruiting --> Completed

Functional analyses demonstrated that altered ABCB6 expression did not significantly affect cell proliferation or apoptosis; however, targeted knockdown of ABCB6 markedly restored the sensitivity of K562/A02 cells to adriamycin (ADR) and cytosine arabinoside (Ara-C). Mechanistic studies revealed that ABCB6 contributes to ADR efflux from leukemia cells, thereby reducing intracellular drug accumulation and weakening its cytotoxic activity. Together, these findings establish ABCB6 as a previously unrecognized efflux transporter that contributes to MDR in leukemia and highlight ABCB6 as a potential therapeutic target for overcoming MDR.

Concurrently, it suppresses Polo-like kinase 1 (PLK1) expression, thereby inducing G2/M cell cycle arrest and activating apoptotic pathways. Collectively, our findings identify NTZ as a potent anti-tumor agent and chemosensitizer that restores Ara-C responsiveness, offering a promising combinatorial strategy for the treatment of refractory AML.

Traditional IT agents such as methotrexate or cytarabine were generally associated with modest survival outcomes, whereas more recent studies evaluating IT pemetrexed and molecularly guided regimens reported longer survival in selected cohorts, particularly in EGFR-mutant NSCLC-LM. Ommaya reservoir-based delivery may offer practical advantages for repeated treatment and CSF monitoring in appropriately selected patients, with acceptable toxicity and manageable device-related risks. Emerging data on pemetrexed-based intrathecal regimens and other molecularly informed approaches suggest potential benefit in selected subgroups, but prospective, multicenter, mutation-stratified studies are needed to refine patient selection, optimize dosing strategies, and define the comparative role of different intrathecal delivery routes.

These findings identify BMP and ERK signaling as context-dependent regulators of Ara-C response in AML and demonstrate that stromal interactions evolve during resistance acquisition. Targeting BMP and ERK pathways, together with modulators of DNA repair or drug efflux, may provide therapeutic strategies to reduce relapse and improve AML outcomes.

P3, N=556, Recruiting, Children's Oncology Group | Not yet recruiting --> Recruiting

P2, N=77, Recruiting, Tongji Hospital | N=110 --> 77

P1, N=8, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: May 2026 --> May 2027

P1/2, N=53, Active, not recruiting, University of Southern California | Trial completion date: Dec 2026 --> Dec 2027 | Trial primary completion date: Jun 2026 --> Dec 2026