Abstract
BACKGROUND: Convection-enhanced delivery (CED) is a technique that delivers therapeutic agents directly and effectively into the brain parenchyma. Glioma affecting brainstem is one of the important candidates that could be targeted with CED. In this report, we describe our recent effort to develop novel CED based therapeutic approach for gliomas affecting brainstem. METHODS: Direct local convection-enhanced delivery of nimustine hydrochloride (ACNU) monitored with real-time MRI is our strategy. We are now in clinical trial against recurrent malignant glioma cases affecting brainstem. Pilot feasibility study recruited three patients. Subsequently, until today, we have recruited 7 patients as Phase I trial. On the other hand, we have developed an ultrasound assisted infusion device for brainstem infusion. RESULTS: In the pilot study, CED of ACNU demonstrated an efficacy against a patient suffering recurrent glioblastoma at brainstem. Succeeding phase I study is still ongoing, but so far, CED of ACNU is safe and feasible even against brainstem disease. Although the new device we developed is still in pre-clinical development, improved distribution was achieved using this device. CONCLUSIONS: CED of ACNU can be safely performed with real-time MRI monitoring. This strategy can be a novel therapeutic approach for gliomas affecting brainstem. New ultrasound assisted device achieved improved distribution in animal brain. Clinical application is warranted. SECONDARY CATEGORY: Pediatrics.