Therapy Detail
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| Therapy Name | Midostaurin + Ruxolitinib |
| Synonyms | |
| Therapy Description | |
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| Drug Name | Trade Name | Synonyms | Drug Classes | Drug Description |
|---|---|---|---|---|
| Midostaurin | Rydapt | PKC412|CGP 41251 | CSF1R Inhibitor 28 FLT3 Inhibitor 69 KIT Inhibitor 57 PKC alpha Inhibitor 6 PKC beta Inhibitor 6 PKC Inhibitor (Pan) 11 VEGFR2 Inhibitor 37 | 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). |
| Ruxolitinib | Jakafi | INCB 18424|INCB018424|INCB18424 | JAK1 Inhibitor - ATP competitive 5 JAK2 Inhibitor - ATP competitive 15 | Jakafi (ruxolitinib) is an inhibitor of protein tyrosine kinases JAK1 and JAK2, thus resulting in reduced inflammation and reduced proliferation (PMID: 22474318). Jakafi (ruxolitinib) is FDA approved to treat bone marrow cancer, specifically intermediate or high-risk myelofibrosis (FDA.gov) |
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| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|
| FLT3 exon 14 ins JAK2 V617F | hematologic cancer | sensitive | Midostaurin + Ruxolitinib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Rydapt (midostaurin) and Jakafi (ruxolitinib) inhibited downstream signaling and cell growth in transformed hematologic cells expressing a FLT3-ITD mutation and JAK2 V617F in culture (PMID: 33149267). | 33149267 |
| FLT3 exon 14 ins JAK1 V658F | hematologic cancer | sensitive | Midostaurin + Ruxolitinib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Rydapt (midostaurin) and Jakafi (ruxolitinib) inhibited Flt3 and Stat5 phosphorylation and growth of transformed hematologic cells expressing a FLT3-ITD mutation and JAK1 V658F in culture (PMID: 33149267). | 33149267 |
| FLT3 exon 14 ins JAK1 S703I | hematologic cancer | sensitive | Midostaurin + Ruxolitinib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Rydapt (midostaurin) and Jakafi (ruxolitinib) inhibited downstream signaling and cell growth in transformed hematologic cells expressing a FLT3-ITD mutation and JAK1 S703I in culture (PMID: 33149267). | 33149267 |
| FLT3 exon 14 ins JAK3 V722I | hematologic cancer | sensitive | Midostaurin + Ruxolitinib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Rydapt (midostaurin) and Jakafi (ruxolitinib) inhibited downstream signaling and cell growth in transformed hematologic cells expressing aa FLT3-ITD mutation and JAK3 V722I in culture (PMID: 33149267). | 33149267 |
| FLT3 exon 14 ins JAK1 R724H JAK3 A573V | hematologic cancer | sensitive | Midostaurin + Ruxolitinib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Rydapt (midostaurin) and Jakafi (ruxolitinib) inhibited downstream signaling and cell growth in transformed hematologic cells expressing a FLT3-ITD mutation, JAK1 R724H, and JAK3 A573V in culture (PMID: 33149267). | 33149267 |
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| Clinical Trial | Phase | Therapies | Title | Recruitment Status | Covered Countries | Other Countries |
|---|
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