Paxalisib (GDC-0084) is a brain-penetrant PI3K/mTOR inhibitor that potently inhibits AKT phosphorylation and reduces orthotopic tumour growth in vivo.285,286 Paxalisib is currently in phase I/II clinical trials for both newly diagnosed and progressive/recurrent glioblastoma in adults (NCT03522298, NCT01547546) and children (NCT05009992) with encouraging preliminary results.
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