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decitabine + midostaurin
- Development stage
- Phase 2
- Lead developer
- Astex Pharmaceuticals
- Modality
- Small Molecules
- Administration
- Intravenous, Oral
01
Overview
Decitabine + midostaurin is a combination of two small molecule drugs used in investigational regimens for the treatment of acute myeloid leukemia (AML), particularly in older or unfit patients. Decitabine is a hypomethylating agent that incorporates into DNA and inhibits DNA methyltransferases, leading to global DNA hypomethylation, reactivation of tumor suppressor genes, and decreased neoplastic cell proliferation. Midostaurin is a multikinase inhibitor targeting FLT3, KIT (CD117), PDGFR, VEGFR2, and protein kinase C alpha; it induces apoptosis in leukemia cells expressing these kinases. While both agents are approved individually for hematologic malignancies—decitabine for myelodysplastic syndromes and AML, and midostaurin (Rydapt) for FLT3-mutated AML—their combination has been studied in clinical trials but has not demonstrated clear additional benefit over monotherapy with decitabine in unfit AML patients[4][5][8]. The combination remains investigational.
- Other names
- 5-aza-2'-deoxycytidinePKC412PKC-412PKC 412
02
Targets
DNMT1 (DNA (cytosine-5)-methyltransferase 1)DNMT3B (DNA (cytosine-5)-methyltransferase 3B)FLT3 (Fms related receptor tyrosine kinase 3)KIT (c-KIT proto-oncogene receptor tyrosine kinase)PDGFR (PDGFR family)PRKCA (Protein kinase C alpha)VEGFR2 (Vascular endothelial growth factor receptor 2)DNMT3A (DNA methyltransferase 3 alpha)HDAC (HDAC family)
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