BTSA1

BTSA1 is a potent, high affinity and orally active BAX activator with an IC₅₀ of 250 nM and an EC₅₀ of 144 nM. BTSA1 binds with high affinity and specificity to the N-terminal activation site and induces conformational changes to BAX leading to BAX-mediated apoptosis^[1]. IC50 & Target: IC50: 250 nM ( BAX)^[1]
; EC50: 144 nM ( BAX)^[1] In Vitro: BTSA1 (5 μM; 6-24 hours; human AML cell lines) treatment reduced viability of all AML cell lines and displays substantial cell death activity within 6 hours^[1].
BTSA1 (2.5-10 μM; 6 hours; NB4 cells) treatment induces BAX translocation coincided with the release of cytochrome c from the mitochondria to the cytosol. Significant BAX mitochondrial translocation is induced in a BTSA1 dose-dependent manner^[1].
BTSA1 (0.15625-10 μM; 4-24 hours; OCI-AML3 cells) treatment induces dose-dependent caspase-3/7 activation in OCI-AML3 cells. Caspase-3/7 activation is monitored within 4-24 hours and maximal caspase-3/7 activation is detected in 4 hours^[1]. In Vivo: BTSA1 (10 mg/kg; intraperitoneal injection; every two days; NOD-SCID IL2Rγ null (NSG) mice) treatment significantly increases survival when compared to vehicle-treated mice. BTSA1 treatment induces significant suppression of leukemia growth^[1].