Daraxonrasib (RMC-6236) is an effective targeted therapy for RAS -mutant neuroblastoma - PubMed
5 hours ago
- #targeted therapy
- #neuroblastoma
- #RAS-mutant
- Daraxonrasib (RMC-6236) is a targeted therapy effective for RAS-mutant neuroblastoma (NB).
- Neuroblastoma is the most common extracranial solid tumor in children, with poor outcomes in relapsed or refractory high-risk cases.
- RAS-mutant NB shows activation in the MAPK pathway, but MEK and KRAS G12C inhibitors have failed as single-agent therapies.
- RMC-6236 demonstrates significant preclinical and early clinical activity in RAS-mutant cancers, including NB.
- In RAS-mutant NB models, RMC-6236 reduces cell viability, suppresses MAPK signaling, and increases cell death.
- In vivo studies show RMC-6236 reduces tumor growth and extends survival in RAS-mutant NB mouse models.
- RMC-6236 upregulates BIM and BCL-2, enhancing BIM:BCL-2 complexes, which can be targeted by venetoclax for improved efficacy.
- The study supports expanded clinical testing of RMC-6236 for RAS-mutant NB, a subset with poor outcomes.