July 2, 2012
Aeterna Zentaris reported results from a phase I trial of perifosine for the treatment of chemo-resistant and radio-resistant neuroblastoma. The open-label, dose-escalating study enrolled 24 patients who had been afflicted by neuroblastoma for 2.5 to 8.0 years. Subjects received 100-200mg/m2 perifosine on day one, followed by daily maintenance doses of 50-75mg/m2 perifosine as monotherapy. Anti-neuroblastoma activity was evident by a 50% progression-free survival rate at 12 months (standard error ±11%) and included one complete remission based on a normalized MIBG scan and three patients with improved MIBG scan and normalized bone marrow histology over prolonged follow-up (up to 37 months). The drug was well tolerated and safe, with no significant toxicity seen. Based on these data, Aeterna Zentaris will be pursuing perifosine as combination therapy.