^
    21h
    IL-1 signaling and inflammasomes in acute myeloid leukemia: mechanisms and therapeutic opportunities. (PubMed, Cell Mol Life Sci)
    Beyond IL-1 release, NLRP3 may interface with cellular stress responses and pyroptosis, thereby influencing both AML cells and their microenvironment through multiple mechanisms. Inflammasome signaling may act as a driver of therapy resistance while also representing a promising therapeutic target.
    Review • Journal
    |
    IL1B (Interleukin 1, beta) • NLRP3 (NLR Family Pyrin Domain Containing 3)
    21h
    RPL5 deficiency-induced ribosomal stress targets a select subset of proteins and inhibits the PI3K-Akt-mTOR signaling pathway to eradicate leukemia stem cells. (PubMed, Cell Death Dis)
    Ile and Val are glycogenic branched-chain amino acids (BCAAs) that regulate fundamental cell processes by affecting mTOR activation through BCAA metabolism. In conclusion, RPL5 depletion-induced ribosomal stress disrupted stemness maintenance by affecting BCAA metabolism in AML, specifically inhibiting the PI3K-Akt-mTOR signaling pathway, which resulted in LSC eradication.
    Journal
    |
    TP53 (Tumor protein P53) • MYC (V-myc avian myelocytomatosis viral oncogene homolog) • RPL5 (Ribosomal Protein L5)
    |
    TP53 mutation
    21h
    Clonal architecture of FLT3-ITD and acquired 13q uniparental disomy define prognostic heterogeneity and therapeutic vulnerabilities in acute myeloid leukemia. (PubMed, Neoplasia)
    In conclusion, FLT3-ITD clonal origin demonstrated significant impact on the outcome of AML. Acquired 13q UPD drives clonal evolution and disease progression in the DNMT3A/NPM1-origin subgroup, highlighting its potential as a therapeutic target.
    Journal
    |
    FLT3 (Fms-related tyrosine kinase 3) • NPM1 (Nucleophosmin 1) • HRD (Homologous Recombination Deficiency) • DNMT3A (DNA methyltransferase 1)
    |
    FLT3-ITD mutation • NPM1 mutation
    23h
    EVICTION: First-in-Human Study of ICT01 in Patients With Advanced Cancer (clinicaltrials.gov)
    P1/2, N=292, Active, not recruiting, ImCheck Therapeutics | Trial completion date: Dec 2025 --> Oct 2026 | Trial primary completion date: Sep 2025 --> Oct 2026
    Trial completion date • Trial primary completion date • Checkpoint inhibition • First-in-human
    |
    Keytruda (pembrolizumab) • ICT01
    1d
    Discovery of a dual-target CRBN-mediated degrader for IKZF1/3 and GSPT1 proteins. (PubMed, Bioorg Chem)
    Immunomodulatory drugs (IMiDs) like lenalidomide and pomalidomide degrade IKZF1/3 and are combined with other therapies to treat hematologic malignancies, including multiple myeloma and non-Hodgkin lymphoma. Molecular docking studies suggested that DIX-01 may form stable ternary complexes with CRBN-IKZF1 and CRBN-GSPT1, providing a structural basis for its dual-target degradation activity. Furthermore, DIX-01 significantly inhibited tumor growth in a zebrafish xenograft model transplanted with human acute myeloid leukemia cells (MV4-11), supporting its potential as a therapeutic agent for hematologic malignancies.
    Journal
    |
    IKZF1 (IKAROS Family Zinc Finger 1) • CRBN (Cereblon) • GSPT1 (G1 To S Phase Transition 1)
    |
    lenalidomide • pomalidomide
    1d
    The future of BRD9 inhibitors: a patent perspective (2019-present). (PubMed, Expert Opin Ther Pat)
    The recent patent data show how selective BRD9 degraders represent a significant step forward in terms of efficacy and selectivity, with promising results in preclinical models of acute myeloid leukemia (AML), synovial sarcoma (SS), and Huntington's disease (HD). Despite several critical issues, the selective degradation of this epigenetic target shows great potential to be an innovative therapeutic strategy.
    Review • Journal
    |
    SMARCA4 (SWI/SNF related, matrix associated, actin dependent regulator of chromatin, subfamily A, member 4)
    1d
    Liposomal Cytarabine and Daunorubicin (CPX-351) and Quizartinib for the Treatment of Acute Myeloid Leukemia and High Risk Myelodysplastic Syndrome (clinicaltrials.gov)
    P1/2, N=52, Recruiting, M.D. Anderson Cancer Center | Trial completion date: Dec 2025 --> Dec 2027 | Trial primary completion date: Dec 2025 --> Dec 2027
    Trial completion date • Trial primary completion date
    |
    Vanflyta (quizartinib) • Vyxeos (cytarabine/daunorubicin liposomal formulation)
    1d
    INCB 84344-102: Safety and Efficacy of Ponatinib for Treatment of Pediatric Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors (clinicaltrials.gov)
    P1/2, N=70, Recruiting, Incyte Biosciences International Sàrl | Trial completion date: Jan 2026 --> Feb 2028 | Trial primary completion date: Jan 2026 --> Feb 2028
    Trial completion date • Trial primary completion date
    |
    FLT3 (Fms-related tyrosine kinase 3) • ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase) • RET (Ret Proto-Oncogene) • FGFR (Fibroblast Growth Factor Receptor) • BLM (BLM RecQ Like Helicase)
    |
    EGFR mutation • KIT mutation • FGFR mutation • RET mutation
    |
    Iclusig (ponatinib)
    1d
    Cladribine, Idarubicin, Cytarabine, and Quizartinib in Treating Patients With Newly Diagnosed, Relapsed, or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome (clinicaltrials.gov)
    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
    Trial completion date • Trial primary completion date
    |
    cytarabine • Vanflyta (quizartinib) • idarubicin hydrochloride • cladribine • Starasid (cytarabine ocfosfate)
    1d
    Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant (clinicaltrials.gov)
    P1/2, N=16, Active, not recruiting, M.D. Anderson Cancer Center | Trial completion date: Dec 2025 --> Dec 2027 | Trial primary completion date: Dec 2025 --> Dec 2027
    Trial completion date • Trial primary completion date
    |
    cyclophosphamide • fludarabine IV
    2d
    High- Fiber/ Low-fat Diet for Prevention of Recurrent Clostridioides Difficile Infection in Oncology (clinicaltrials.gov)
    P=N/A, N=124, Active, not recruiting, University of Colorado, Denver | Recruiting --> Active, not recruiting | Trial completion date: Dec 2025 --> Mar 2026
    Enrollment closed • Trial completion date
    2d
    NCI-2019-08946: CPX-351 for the Treatment of Secondary Acute Myeloid Leukemia in Patients Younger Than 60 Years Old (clinicaltrials.gov)
    P2, N=21, Active, not recruiting, Roswell Park Cancer Institute | Suspended --> Active, not recruiting | N=46 --> 21 | Trial primary completion date: Jan 2027 --> Jun 2025
    Enrollment closed • Enrollment change • Trial primary completion date
    |
    Vyxeos (cytarabine/daunorubicin liposomal formulation)