Disclaimer: Not an advise. Informational and Educational use only.
Standard of care: ACNS0423 Study
Introduction: https://pmc.ncbi.nlm.nih.gov/articles/PMC5035517/
Reports outcomes (EFS/OS) and toxicity profile, including the central conclusion that this regimen had acceptable but meaningful hematologic toxicity and only modest improvement vs historical expectations.
Disclaimer: Not advocating for any specific drugs. Informational and educational purposes only for may be used to apply for SPIND only. May add more drugs.
Brain Tumor IND: ACT001 : How it works
Mechanism in glioblastoma models: targets STAT3 → lowers PD-L1 transcription / immunosuppression (preclinical + mechanistic). PMC
Additional glioma mechanism work: targets glioma stem-like cells and anti-tumor effects in GBM models (mechanistic preclinical). PMC
Brain Tumor IND: ACT001 : Safety:
- First-in-human Phase I (advanced glioma): reported safe / well tolerated with PK and tolerability focus (ASCO abstract / JCO supplement). ASCOPubs+1
- Pediatric DMG/DIPG Phase 1 dose escalation: reported well tolerated with preliminary anti-tumor activity at higher doses (conference abstract on Neuro-Oncology / OUP). OUP Academic+1
Brain Tumor IND: ACT001: Efficacy (signals, early):
- Phase I glioma: clinical responses noted in a subset (pathologic response mentioned in abstract). ASCOPubs+1
- Pediatric DMG/DIPG Phase 1: preliminary evidence of anti-tumor activity at highest dose levels (early signal, not definitive). PMC+1
- Recurrent GBM combo program (ACT001 + anti-PD-1): designed to evaluate both safety and efficacy in surgically accessible recurrent GBM. ClinicalTrials
Brain Tumor IND: ACT001: Existing Trials:
NCT05053880 — ACT001 + anti-PD-1 in surgically accessible recurrent GBM.
NCT06838676 — Phase II ACT001 in children/adolescents with DIPG and H3K27-altered HGG (CONNECT2110). ClinicalTrials+1
Other existing IND trials and hospital informations: