PQA 03 - PQA 03 Gynecological Cancer, Pediatric Cancer, and Professional Development Poster Q&A
3588 - Overall Experience of Two Clinical Trials for FUS-Mediated Blood Brain Barrier-Opening for Patients with DIPG/DMG Delivered in Radiation Oncology
Virginia Tech Carilion School of Medicine New York, NY, United States
L. Szalontay1, J. Fino2, X. Berg3, J. Lipina2, M. Gallitto4, G. De los Santos4, N. Yoh5, R. Gartrell1,6, P. Jovana2, J. Garvin7, A. Lignelli-Dipple8, W. Gomes8, N. Feldstein5, E. E. Konofagou9, S. Zacharoulis2, and C. C. Wu4,10; 1Department of Pediatrics Oncology, Columbia University Irving Medical Center, New York, NY, 2Department of Pediatrics, Columbia University Irving Medical Center, New York, NY, 3Columbia University Irving Medical Center, New York, NY, 4Department of Radiation Oncology, Columbia University Irving Medical Center, New York, NY, 5Department of Neurological Surgery, Columbia University Irving Medical Center, New York, NY, 6Johns Hopkins University, Baltimore, MD, 7Department of Pediatric Oncology, Columbia University Medical Center, New York, NY, 8Department of Radiology, Columbia University Irving Medical Center, New York, NY, 9Department of Biomedical Engineering, Columbia University, New York, NY, 10Herbert Irving Comprehensive Cancer Center, Columbia University Irving Medical Center, New York, NY
Purpose/Objective(s):DIPG/DMG is a fatal brain tumor commonly found in the brainstem with a median overall survival of 1 year. The blood-brain barrier (BBB) poses a major challenge to the delivery of therapeutic agents. Over the past 4-years we opened two Phase I clinical trials using Delsona focused ultrasound (FUS) device developed at Columbia to treat pediatric and AYA patients with relapsed DMG. The first study, we delivered FUS with panobinostatand second study we gave concurrent etoposide. Here we present the experience of delivering neuronavigation based FUS for pediatric patients with progressive DMG from the two studies.Materials/
Methods: Pediatric and AYA patients with biopsy proven DIPG/DMG at disease relapsed were enrolled onto clinicaltrial.org identified NCT04804709 (FUS with oral panobinostat) or clinicaltrial.org identified NCT05762419 (FUS with oral etoposide). Patients were allowed to have prior therapies, including radiation and reirradiation.FUS was delivered over the course of up to six or four cycles respectively. MRI was performed to validate BBB-opening and closure. Results: For the FUS panobinostat study, we enrolled 5 patients and accrued 3. The study was closed prematurely when panobinostat was taken off the market. For the FUS etoposide study, we enrolled 3 patients and accrued 2. This study is currently open. For the two trials combined, we delivered over twenty-five FUS treatments. Four of five patients accrued had achieved BBB-opening. 3 of 5 patients had reirradiation prior to enrollment. All FUS delivery were performed as an outpatient procedure in the Department of Radiation Oncology. The average time for BBB-delivery defined as patient entering the treatment room to completion of treatment was approximately 47 minutes. Side effects from FUS was limited to one grade 1 dermatological toxicity. Conclusion: FUS-mediated BBB-opening delivered as an outpatient procedure is preliminarilyfeasible in children with relapsed DIPG/DMG. Further studies are needed to assess for efficacy.
