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Molecular Profile KMT2A - AFF1
Therapy I-BET151
Indication/Tumor Type acute lymphoblastic leukemia
Response Type sensitive

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
KMT2A - AFF1 acute lymphoblastic leukemia sensitive I-BET151 Preclinical - Pdx & cell culture Actionable In a preclinical study, treatment with I-BET151 in acute lymphoblastic leukemia cell lines harboring a KMT2A-AFF1 fusion resulted in growth inhibition, cell cycle arrest, increased apoptosis, and down-regulation of BRD4 targets CDK6, c-MYC, and BCL2 in culture, and resulted in reduced leukemic engraftment and decreased disease burden in a patient-derived xenograft (PDX) model and prolonged survival and reduced cellularity in a cell line xenograft model (PMID: 29748211). 29748211
PubMed Id Reference Title Details
(29748211) Antileukemic Efficacy of BET Inhibitor in a Preclinical Mouse Model of MLL-AF4+ Infant ALL. Full reference...