Administered at a dose of 40 mg/kg via intraperitoneal injection three days per week, GANT-61 inhibits CSC tumor growth in NOD/SCID IL2Rγ null mice^[2].

Furthermore, GANT61 (50 mg/kg, per oral) enhances the effects of chemotherapeutic drugs used in neuroblastoma treatment in an additive or synergistic manner. It also reduces the growth of established neuroblastoma xenografts in nude mice^[3].

GANT61 (20 μM) induces more significant cell death compared to targeting Smo (cyclopamine). It also inhibits the clonogenic survival of human colon carcinoma cell lines (0, 5, 10, 20 μM). Moreover, GANT61 (20 μM, 0-72 hr) downregulates Gli1 and Gli2 expression in HT29 cells. Additionally, GANT61 (0, 10 μM, or 20 μM) differentially regulates genes involved in the balance between cell death and survival^[1].

GANT-61 inhibits cell viability and induces apoptosis in pancreatic CSCs. It also suppresses the expression of downstream targets of the Shh pathway, decreases Gli-DNA interaction, Gli transcriptional activity, and Gli nuclear translocation in pancreatic CSCs. Furthermore, GANT-61 modulates genes associated with cell survival, cell death, and pluripotency, while also inhibiting the motility, invasion, and migration of CSCs^[2].

The sensitivity of GANT61 correlates positively with GLI1 and negatively with MYCN expression in the tested neuroblastoma cell lines. GANT61 downregulates the expression of GLI1, c-MYC, MYCN, and Cyclin D1, and induces apoptosis in neuroblastoma cells^[3].