Paxalisib

Gliomas are the most frequent malignant tumors of the central nervous system, with varied outcomes and an unmet need of highly efficacious treatments. Paxalisib (GDC-0084) is a novel, orally administered, brain-penetrant inhibitor of phosphoinositide 3-kinase/mammalian target of rapamycin (PI3K/mTOR) that has been reported to significantly inhibit tumor growth in vitro and in vivo. A first-in-human multicenter phase I study in participants with recurrent high-grade glioma indicated that paxalisib penetrated the blood-brain barrier and had an acceptable safety profile. Despite limited clinical data, paxalisib may be a drug that is efficacious in glioma patients with genetic alterations in the PI3K/mTOR pathway. In this review, we summarize preclinical and clinical data for paxalisib in the treatment of gliomas.