简介内容：ACT001 is an orally active PAI-1 inhibitor by inhibiting the phosphorylation of PI3K and AKT. ACT001 inhibits the phosphorylation of STAT3 and PD-L1 expression by directly binding to STAT3. ACT001, a fumarate salt form of DMAMCL (a prodrug of Micheliolide), can cross the blood-brain barrier. ACT001 has a synergistic effect with cisplatin through the inhibition of the PAI-1/PI3K/AKT pathway. ACT001 has potent anti-glioblastoma (GBM) activity and immunomodulatory effects [1] [2].
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