cytarabine

P1/2, N=80, Recruiting, M.D. Anderson Cancer Center | Trial completion date: Dec 2025 --> Dec 2027 | Trial primary completion date: Dec 2025 --> Dec 2027

Proteomic profiling showed that NAMPT inhibition with KPT-9274 induced adaptive upregulation of BCL2, an anti-apoptotic protein, highlighting a survival mechanism...Additionally, NAMPT inhibition reduced PARP activity and impaired DNA repair pathways, sensitizing AML cells to cytarabine and hypomethylating agents. Together, these results demonstrate that NAMPT inhibition both potentiates venetoclax activity and enhances the cytotoxic effects of standard chemotherapies by targeting metabolic and DNA repair vulnerabilities. These findings provide strong preclinical support for evaluating NAMPT and BCL2 dual inhibition strategies in future AML clinical trials.

Cytarabine (ara-C) and fludarabine (F-ara-A) are key drugs in leukaemia treatment. Mechanistically, IMPDHi depleted allosteric SAMHD1 activators GTP and dGTP, thereby increasing active triphosphate metabolites in SAMHD1-proficient, but not SAMHD1-deficient, cells. Our findings suggest that the addition of IMPDHi to ara-C and F-ara-A may have therapeutic benefits in some AML cases.

For relapsed and refractory APL, relevant drug resistance gene monitoring should be carried out. Some relapsed and refractory APL patients who do not respond to conventional treatment are at risk of death. We report a successful case, the regimen of VEN targeted therapy combined with chemotherapy still holds promise for the treatment of future relapsed/refractory APL.

P2, N=162, Not yet recruiting, National Cancer Institute (NCI) | Trial completion date: Jul 2028 --> Nov 2027 | Initiation date: Nov 2025 --> Feb 2026 | Trial primary completion date: Jul 2028 --> Nov 2027

Additionally, the study on an Fc-silenced mutant (GZ1 LALAPG) indicated that Fc-mediated functions played a critical role in the enhanced therapeutic effects. These findings highlighted the potential of anti-CD36 mAb in combination with Ara-C as a novel treatment strategy for overcoming drug resistance in AML.

To investigate this question, we used a knock-in mouse that constitutively expresses a Calr frameshift allele and introduced conditional Ezh2 deletion triggered by tamoxifen...Short-term colony assays showed that inhibition of PPARγ modestly increased the anti-proliferative effect of cytarabine on AML-derived stem and progenitor cells, suggesting a possible reliance on FAO...At a non-critical stage, peripheral counts remain near-normal while bone marrow HSPC compartments are already distorted. AML-like, but not sMF-like, Flk2- CD48+ LSK cells transmit leukemia and display enhanced fatty-acid-oxidation signatures, suggesting a distinct, potentially targetable metabolic bias.

Idarubicin, cytarabine, etoposide (IA ± E) chemotherapy yielded superior survival, while azacitidine+venetoclax (AZA+VEN) regimens underperformed. The study was registered on the Chinese clinical trial registry (ChiCTR) platform (No. ChiCTR2500096484).

dexamethasone, ara-C, cisplatin (DHAP) was the most common salvage regimen (47.8%)...BCNU, etoposide, ara-C, melphalan (BEAM) was the most common conditioning regimen 91.9%...t-MN significantly contributes to NRM post-auto-HCT. Age at transplant is the primary risk factor, highlighting the need for vigilant monitoring and risk mitigation strategies.

Infiltrative mediastinal/retroperitoneal MS may present with esophageal obstruction and mimic lymphoma or carcinoma. High-index suspicion, targeted biopsy with high-power morphology, and a focused immunohistochemical panel are critical for timely diagnosis and treatment initiation.

P2, N=70, Suspended, St. Jude Children's Research Hospital | Recruiting --> Suspended

P3, N=468, Recruiting, Syndax Pharmaceuticals | Not yet recruiting --> Recruiting