Drug intelligence / Profile preview

GHA + azacitidine

Development stage
Unknown
Lead developer
Bristol Myers Squibb
Modality
Cytokines & Interferons → Recombinant Proteins and Enzymes, Small Molecules
Administration
Subcutaneous, Intravenous
01

Overview

GHA + azacitidine is a combination therapy consisting of GHA (presumed to refer to granulocyte colony-stimulating factor, specifically granulocyte-macrophage colony-stimulating factor, GM-CSF) and azacitidine. Azacitidine is a pyrimidine nucleoside analogue with antineoplastic and hypomethylating activity, primarily used in the treatment of myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). It works by incorporating into RNA and DNA, disrupting their metabolism, inhibiting protein synthesis, and inhibiting DNA methyltransferase—leading to impaired DNA methylation[3][4][5]. GM-CSF (if GHA refers to this agent) is a cytokine that stimulates the production of white blood cells. The combination has been studied as post-transplant maintenance therapy in AML or MDS patients to prevent relapse after allogeneic bone marrow transplantation. In clinical studies, this regimen was generally well tolerated with rare severe hematologic toxicities[6].

Brand names
VidazaLeukine
Other names
5-azacytidineazacytidinegranulocyte-macrophage colony-stimulating factor
02

Targets

DNMT (DNA methyltransferase)

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