Drug intelligence / Profile preview

cytarabine + idarubicin + GM-CSF + tioguanine + lomustine + mitoguazone

Development stage
Unknown
Lead developer
French Innovative Leukemia Organisation
Modality
Recombinant Proteins and Enzymes, Small Molecules
Administration
Intravenous, Oral, Subcutaneous
01

Overview

This multi-drug chemotherapy regimen served as the control arm in the GOELAMS SA4 (French Innovative Leukemia Organisation) Phase 3 clinical trial, which evaluated the addition of fludarabine to standard intensive therapy in elderly patients (ages 60-75) with de novo acute myeloid leukemia (AML). The regimen consists of three distinct phases: an induction phase utilizing cytarabine, idarubicin, and GM-CSF (molgramostim); a consolidation phase with intermediate-dose cytarabine and idarubicin; and a one-year maintenance phase involving 6-thioguanine and cytarabine, interspersed with re-induction courses of lomustine (CCNU), mitoguazone, and cytarabine. The combination targets leukemic cells through multiple mechanisms, including DNA synthesis inhibition, DNA intercalation, and alkylation, while GM-CSF is included to potentially sensitize blasts or accelerate hematologic recovery.

Other names
no fludarabine-French Innovative Leukemia Organisation-acute myeloid leukemiaGOELAMS SA4 control regimen
02

Targets

POLA1 (DNA polymerase alpha)CSF2R (Granulocyte-macrophage colony-stimulating factor receptor)AMD1 (S-adenosylmethionine decarboxylase)TOP2A (DNA topoisomerase II)MSH2 (DNA mismatch repair protein MSH2)DNA

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