Therapy Detail

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Drug Name Trade Name Synonyms Drug Classes Drug Description
Bortezomib Velcade Velcade (bortezomib) is a reversible proteasome inhibitor that inhibits survival of malignant cells and regulates bone remodeling (PMID: 26579531). Velcade (bortezomib) is FDA approved for the treatment of mantle cell lymphoma and multiple myeloma (FDA.gov).
Cytarabine Cytosar-U Ara-C Cytosar-U (cytarabine) is a cytidine analog that, when incorporated into DNA, inhibits DNA synthesis and repair (PMID: 32524309).
Etoposide Vepesid EPEG|Eposin|VP-16|VP-16-213 TOPO2 inhibitor 4 Vepesid (etoposide) binds to and inhibits DNA topoisomerase II, resulting in accumulated DNA damage, inhibition of replication, and cell death (NCI Drug Dictionary).
Midostaurin Rydapt PKC412|CGP 41251 CSF1R Inhibitor 23 FLT3 Inhibitor 54 KIT Inhibitor 50 PKC alpha Inhibitor 6 PKC beta Inhibitor 6 PKC Inhibitor (Pan) 10 VEGFR2 Inhibitor 35 Rydapt (midostaurin) is a multi-kinase inhibitor with activity against FLT3, KIT, PDGFRB, KDR (VEGFR2) and PKC, with higher selectivity for conventional PKC isoforms, which induces cell cycle arrest and apoptosis (PMID: 12124173, PMID: 23127174, PMID: 15914319). Rydapt (midostaurin) is FDA approved for FLT3-mutant AML in combination with chemotherapy, and is approved for use in aggressive systemic mastocytosis, systemic mastocytosis with associated hematological neoplasm, or mast cell leukemia as a single therapy (FDA.gov).
Mitoxantrone Novantrone CL 232315 Novantrone (Mitoxantrone) binds to and inhibits topoisomerase II, leading to DNA double strand breaks and decreased DNA repair (NCI Drug Dictionary).

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown acute myeloid leukemia not applicable Bortezomib + Cytarabine + Etoposide + Midostaurin + Mitoxantrone Phase I Actionable In a Phase I trial, Rydapt (midostaurin), in combination with Velcade (bortezomib) and mitoxantrone, Vepesid (etoposide), and Cytosar-U (cytarabine) (MEC), resulted in an overall response rate of 82.5% (19/23) in patients with relapsed or refractory acute myeloid leukemia receiving dose level 3 and above, with complete responses in 56.5% (13/23) of patients (PMID: 26784138). 26784138

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Clinical Trial Phase Therapies Title Recruitment Status


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