Drug intelligence / Profile preview

isotretinoin + sargramostim

Development stage
Unknown
Lead developer
Partner Therapeutics
Modality
Recombinant Proteins and Enzymes, Small Molecules
Administration
Oral, Subcutaneous, Intravenous
01

Overview

**Isotretinoin + sargramostim** is a combination of two drugs commonly used in post-consolidation therapy for high-risk neuroblastoma. **Isotretinoin** is a retinoid (a form of vitamin A) that induces cytodifferentiation and apoptosis in neuroblastoma cells, acting as an antineoplastic agent. **Sargramostim** is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) that stimulates the production and activation of white blood cells, enhancing immune response and promoting recovery from bone marrow suppression. When used together, especially after stem cell transplant, this combination may improve survival and remission rates by both directly affecting tumor cells (isotretinoin) and supporting immune recovery/tumor cytotoxicity (sargramostim)[1][3][5][7].

02

Targets

RARA (Retinoic acid receptor alpha)RARG (Retinoic acid receptor gamma)RARB (Retinoic acid receptor beta)CSF2R (Granulocyte-macrophage colony-stimulating factor receptor)

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