Assay
Cell line
IC
50
(nM)
Shh-induced
Gli-1 mRNA
Human HEPM
13
Shh-induced
Gli-1 Luciferase
Mouse TM3
7
3000
Tumour volume (mm
3
)
Gli, glioma-associated oncogene homolog zinc finger protein; Shh, Sonic Hedgehog
Gli-1 mRNA (human)
Luciferase (mouse)
Gli-1 promoter
LDE225
5mg/kg
10 mg/kg
20 mg/kg
Vehicle
8 10 12 14 16 18 20 22
0
500
1000
1500
2000
2500
3000
Days post-implantation
Tumour volume (mm
3
)
mean
±
SEM
Ptch +/-, p53 -/- MB model
LDE225: Preclinical Summary
LDE225 is a novel oral inhibitor of
Smo that potently inhibits Smo-
dependent proliferation
in vivo
in
preclinical studies
LD
E22