Phase 2 study of aldoxorubicin in relapsed glioblastoma.

2027Background: Glioblastoma (GBM) is the most common primary brain tumor with a median survival of ~14 months after initial therapy. Relapsed patients usually receive bevacizumab with an ORR of~20%, and OS of ~31 weeks. Recently Prakash et al demonstrated in an orthotopic mouse model of GBM that IV aldoxorubicin (A) but not doxorubicin (D) significantly delayed tumor growth and prolonged survival by over 100%. D does not pnetrate the blood-brain barrier. A is a prodrug of D that is attached to a pH sensitive linker that binds covalently to albumin and is released preferentially within tumors. We assessed the preliminary efficacy and safety of A administered to GBM patients who progressed after first line therapy. Methods: Patients > 18 yrs with 1st relapse GBM received either 250 mg/m2 (21) or 350 mg/m2(7) every 21 days until tumor progression, unacceptable toxicity or withdrawal. Tumors were assessed every 6 weeks by MRI and in some instances Dynamic Susceptibility Contrast (DSC) MRI. Safety monitoring ...