PAC-1 is a new type of small molecular targeted drugs which is being developed for caspase-3 zymogen (procaspase-3) in recent years. Studies in vitro have shown that it can selectively inhibit tumor cell activity

by directly activating procaspase-3 to induce apoptosis. Its anti-tumor effect is clear in vivo. For the NCI-H226 subcutaneous transplanted mice model of lung cancer

the inhibition rate of 50 mg·kg-1 reached 53%

and inhibition rate of 100 mg·kg-1 reached 77% by intragastric administration. In this review we also discussed its analogues

pharmacodinanics

mechanism

toxicology and pharmcokinetics. These results demonstrate that PAC-1 has reliable antitumor activities in vivo and in vitro

but PAC-1 also have disadvantages of low bioavailability and neurotoxicity. In the future

its chemical structure can be modified to improve anti-tumor activities

bio-availability

and reduce the possible side-effects or toxicities.