Molecular Profile Detail

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Profile Name KMT2A - AFF1
Gene Variant Detail

KMT2A - AFF1 (gain of function)

Relevant Treatment Approaches

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Molecular Profile Indication/Tumor Type Response Type Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
KMT2A - AFF1 acute myeloid leukemia sensitive MI-503 Preclinical - Cell line xenograft Actionable In a preclinical study, MI-503 reduced expression of KMT2A (MLL) fusion target genes, inhibited growth of an acute myeloid leukemia (AML) cell lines harboring KMT2A-AFF1 (MLL-AF4) in culture, and inhibited tumor growth and progression in an AML cell line xenograft model harboring KMT2A-AFF1 (MLL-AF4) (PMID: 25817203). 25817203
KMT2A - AFF1 acute lymphoblastic leukemia predicted - sensitive AZD5153 Preclinical - Cell line xenograft Actionable In a preclinical study, AZD5153 inhibited proliferation of acute lymphocytic leukemia cells harboring KMT2A-AFF1 (MLL-AF4) in culture, resulted in tumor growth inhibition in cell line xenograft models (PMID: 27573426). 27573426
KMT2A - AFF1 leukemia sensitive I-BET151 + SGC0946 Preclinical - Cell culture Actionable In a preclinical study, I-BET151 and SGC0946 synergistically inhibited proliferation of leukemia cells harboring KMT2A-AFF1 in culture (PMID: 27294782). 27294782
KMT2A - AFF1 acute myeloid leukemia predicted - sensitive AZD5153 Preclinical - Cell line xenograft Actionable In a preclinical study, AZD5153 inhibited proliferation of acute myeloid leukemia cells harboring KMT2A-AFF1 (MLL-AF4) in culture, resulted in tumor growth inhibition in cell line xenograft models (PMID: 27573426). 27573426
KMT2A - AFF1 acute myeloid leukemia sensitive Dinaciclib Preclinical Actionable In a preclinical study, treatment with Dinaciclib resulted in induced cell death of acute myeloid leukemia cells harboring KMT2A-AFF1 in culture (PMID: 26627013). 26627013
KMT2A - AFF1 leukemia sensitive IRAK1/4 Preclinical - Cell culture Actionable In a preclinical study, leukemia cells harboring KMT2A-AFF1 demonstrated sensitivity to IRAK1/4 in culture, resulting in decreased cell proliferation (PMID: 28065413). 28065413
Clinical Trial Phase Therapies Title Recruitment Status
NCT02727803 Phase II anti-thymocyte globulin + Fludarabine + haNK cells + Melphalan anti-thymocyte globulin + Cyclophosphamide + Fludarabine + haNK cells + Rituximab anti-thymocyte globulin + Busulfan + Clofarabine + Fludarabine + haNK cells Personalized NK Cell Therapy After Chemotherapy and Cord Blood Transplant in Treating Patients With Myelodysplastic Syndrome, Leukemia, Lymphoma or Multiple Myeloma Recruiting