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Table 1 Compounds shown pre-clinically to target telomere maintenance mechanisms, to be prioritised for evaluation in neuroblastoma

From: Novel therapeutic strategies targeting telomere maintenance mechanisms in high-risk neuroblastoma

Drug Target Pre-clinical Data Clinical Trials
6-thio-dG Telomerase Pre-clinical efficacy in TERT activated neuroblastoma models No
Tetra-Pt (bpy) ALT In-vitro and in-vivo activity in U2OS ALT osteosarcoma model No
CX-5461 ALT In-vitro activity in U2OS and SAOS2 ALT osteosarcoma cell lines On-going adult phase I clinical trial (NCT0271997)
Pifithrin-α ALT In-vitro and in-vivo activity in U2OS ALT osteosarcoma model No
Trabectedin ALT In-vitro activity in a panel of ALT cell lines (sarcoma, breast cancer and melanoma) -FDA approval for certain soft tissue sarcomas [68]
-Paediatric sarcoma phase II data [69]
-On-going paediatric clinical trials (NCT04067115)