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chemotherapy-Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University-neuroblastoma
- Development stage
- Unknown
- Lead developer
- Sun Yat-Sen Memorial Hospital
- Modality
- Small Molecules
- Administration
- Intravenous, Oral
01
Overview
This combination chemotherapy regimen is being investigated by Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University for the treatment of stage 4/M neuroblastoma in pediatric patients. The regimen integrates arsenic trioxide (ATO), a small molecule that acts as a Hedgehog pathway inhibitor at the GLI level and inhibits the PI3K-AKT pathway, with trametinib, a selective MEK1/2 inhibitor targeting the MAPK signaling pathway. These targeted agents are added to a backbone of conventional induction chemotherapy consisting of alternating cycles of CAV (cyclophosphamide, pirarubicin, vincristine), PVP (cisplatin, etoposide), and CT (cyclophosphamide, topotecan). The combination is designed to achieve synergistic anti-tumor effects by counteracting compensatory pathway activations, such as ATO-induced MAPK activation and MAPK inhibitor-induced PI3K-AKT activation.
- Other names
- Arsenic Trioxide, MAPK Inhibitors, and Chemotherapy for Stage 4/M Neuroblastoma
02
Targets
TOP2A (DNA topoisomerase II)GLI1PI3K (Phosphoinositide-3-kinase regulatory subunit 6)AKT1 (Proto-oncogene serine/threonine-protein kinase Akt1)DNATUBB (Tubulin (alpha and beta subunits))TOP1 (DNA Topoisomerase I)MEK1 (Dual specificity mitogen-activated protein kinase kinase 1)
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