Phase I Trial of DNX-2401 for Diffuse Intrinsic Pontine Glioma Newly Diagnosed in Pediatric Patients. Issue 5 (27th October 2017)

• Record Type: Journal Article
• Authors: Tejada, Sonia; Alonso, Marta; Patiño, Ana; Fueyo, Juan; Gomez-Manzano, Candelaria; Diez-Valle, Ricardo
• Is Part Of: Neurosurgery. Volume 83:Issue 5(2018)
• Page Start: 1050
• Page End: 1056
• Publication Date: 2017-10-27
• Subjects: Brain tumors -- Diffuse glioma -- Oncolytic adenovirus -- Pediatric tumors; Nervous system -- Surgery -- Periodicals; 617.48005
• DOI: 10.1093/neuros/nyx507
• Languages: English
• ISSNs: 0148-396X

Abstract

Abstract: BACKGROUND: There are no effective treatments for diffuse intrinsic pontine gliomas (DIPGs); these tumors cannot be surgical resected, and diagnosis is based on magnetic resonance imaging. As a result, tumor tissues for molecular studies and pathologic diagnosis are infrequent. New clinical trials are investigating novel medications and therapeutic techniques in an effort to improve treatment of patients with DIPGs. OBJECTIVE: To determine the safety, tolerability, and toxicity of an oncolytic adenovirus, DNX-2401, injected into the cerebellar peduncle in pediatric subjects with DIPG and to collect tumor samples of this type of tumor. METHODS: Phase I, single-center, uncontrolled trial. A tumor biopsy will be performed through the cerebellar peduncle, and DNX-2401 will be injected immediately after the biopsy. Standard therapy consisting of radiotherapy and chemotherapy will follow in 2 to 6 wk. EXPECTED OUTCOMES: Improvement of overall survival and quality of life in patients with DIPG and collection of tumor specimens to study the molecular profiling of these tumors. DISCUSSION: The aims of this trial are to contribute to the sample collection of DIPG and to offer treatment during the tumor tissue biopsy using the virus. If this virus works as expected, it could kill the tumor cells with no damage to healthy tissue, functioning as a targeted therapy. It is important to note that edema has not been observed with this virus in all trials performed to date. The information obtained through this and other similar studies may be useful for developing or improving new therapies in the battle against DIPG.