Proof of concept for the clinical utility of a convection enhanced delivery platform for immuno-oncology drugs against brain tumors

Here we present a post-hoc analysis of the long-term follow-up data for the Phase IIB study NCT00431561 regarding the clinical activity of the TGFbeta2-specific RNA therapeutic OT101/Trabedersen in recurrent/refractory WHO Grade 3 anaplastic astrocytoma (R/R AA) patients. OT101 was administered intratumorally by continuous infusion via CEDOT for 5 consecutive months to 27 recurrent/refractory WHO Grade 3 anaplastic astrocytoma (R/R AA) patients in two dose cohorts (2.5 mg/cycle and 19.8 mg/cycle). 14 of the 27 patients (51.9%) had a sustained partial response (PR) or complete response (CR). The median overall survival (OS) time of 1136 days was significantly better than the 590 days median OS (Log Rank χ2=6.5, P-value=0.011) of the temozolomide (TMZ)-treated control patient population (N=11). *Correspondence to: Fatih M. Uckun, Immuno-Oncology Program, Oncotelic Inc, Agoura Hills, CA; Ares Pharmaceuticals, St. Paul, MN, USA, E-mail: fatihuckun53@gmail.com