Perifosine: Interim Phase I data
Interim data from 24 patients in an open-label, dose-escalation, U.S. Phase I trial showed that perifosine produced a 12-month progression-free survival (PFS) rate of 50%, including 1 complete remission based on iodine-131-meta-iodobenzylguanidine (MIBG) scan. Additionally, 3 patients had improved MIBG scan and normalized bone marrow histology at >37 months follow-up. Perifosine was well tolerated with no grade 3 adverse events. The trial is scheduled to enroll 36 patients with chemo- and radiotherapy-resistant neuroblastoma to receive a 100-200 mg/m 2 loading dose of perifosine on day 1 followed by 50-75 mg/m 2 maintenance doses until disease progression or dose-limiting toxicities (DLTs). Data were presented at the Advances in Neuroblastoma meeting in Toronto. ...