Research Papers:
Acid ceramidase is a novel drug target for pediatric brain tumors
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Abstract
Ninh B. Doan1,2, Ha S. Nguyen2, Andrew Montoure2, Mona M. Al-Gizawiy3, Wade M. Mueller2, Shekar Kurpad2, Scott D. Rand3, Jennifer M. Connelly4, Christopher R. Chitambar4, Kathleen M. Schmainda3,6, Shama P. Mirza1,7,8
1Biotechnology and Bioengineering Center, Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, USA
2Department of Neurosurgery, Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, USA
3Department of Radiology, Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, USA
4Department of Neurology, Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, USA
5Department of Medicine, Hematology/Oncology, Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, USA
6Department of Biophysics, Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, USA
7Obstetrics and Gynecology, Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, USA
8Department of Chemistry and Biochemistry, University of Wisconsin, Milwaukee, Wisconsin, 53211, USA
Correspondence to:
Ninh B. Doan, email: [email protected]
Keywords: pediatric glioblastoma, acid ceramidase inhibitors, carmofur, glioblastoma, medulloblastoma
Received: September 12, 2016 Accepted: February 15, 2017 Published: March 01, 2017
ABSTRACT
Pediatric brain tumors are the most common solid tumors in children and are also a leading culprit of cancer-related fatalities in children. Pediatric brain tumors remain hard to treat. In this study, we demonstrated that medulloblastoma, pediatric glioblastoma, and atypical teratoid rhabdoid tumors express significant levels of acid ceramidase, where levels are highest in the radioresistant tumors, suggesting that acid ceramidase may confer radioresistance. More importantly, we also showed that acid ceramidase inhibitors are highly effective at targeting these pediatric brain tumors with low IC50 values (4.6–50 μM). This data suggests acid ceramidase as a novel drug target for adjuvant pediatric brain tumor therapies. Of these acid ceramidase inhibitors, carmofur has seen clinical use in Japan since 1981 for colorectal cancers and is a promising drug to undergo further animal studies and subsequently a clinical trial as a treatment for pediatric patients with brain tumors.
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