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A Brain Tumor/Organotypic Slice Co-culture System for Studying Tumor Microenvironment and Targeted Drug Therapies
Published on: November 7, 2015
Acid ceramidase is a novel drug target for pediatric brain tumors
Ninh B Doan1,2, Ha S Nguyen2, Andrew Montoure2
1Biotechnology and Bioengineering Center, Medical College of Wisconsin, Milwaukee, Wisconsin, 53226, USA.
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.
Insights
Acid ceramidase is highly expressed in pediatric brain tumors, particularly radioresistant types. Inhibitors targeting this enzyme show promise as a novel therapeutic strategy for these challenging childhood cancers.
Area of Science:
- Oncology
- Pediatric Medicine
- Biochemistry
Background:
- Pediatric brain tumors are the most common childhood solid tumors and a leading cause of cancer-related death.
- Current treatments for pediatric brain tumors are often challenging and limited in efficacy.
Purpose of the Study:
- To investigate the role of acid ceramidase in pediatric brain tumors.
- To evaluate the potential of acid ceramidase inhibitors as a therapeutic strategy for pediatric brain tumors.
Main Methods:
- Quantification of acid ceramidase expression in pediatric brain tumor samples (medulloblastoma, glioblastoma, atypical teratoid rhabdoid tumors).
- Assessment of the efficacy of acid ceramidase inhibitors using IC50 values.
- Evaluation of carmofur as a potential therapeutic agent.
Main Results:
- Significant expression of acid ceramidase was observed in medulloblastoma, pediatric glioblastoma, and atypical teratoid rhabdoid tumors.
- Acid ceramidase levels were highest in radioresistant tumors, suggesting a role in radioresistance.
- Acid ceramidase inhibitors demonstrated high efficacy with low IC50 values (4.6-50 μM) against these pediatric brain tumors.
- Carmofur, an existing acid ceramidase inhibitor, showed promise for further investigation.
Conclusions:
- Acid ceramidase is a potential novel drug target for adjuvant pediatric brain tumor therapies.
- Acid ceramidase inhibitors represent a promising therapeutic avenue for treating pediatric brain tumors.
- Carmofur warrants further preclinical and clinical investigation for pediatric brain tumor treatment.
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