期刊
NANOMEDICINE-NANOTECHNOLOGY BIOLOGY AND MEDICINE
卷 23, 期 -, 页码 -出版社
ELSEVIER
DOI: 10.1016/j.nano.2019.102115
关键词
Pediatrics; Cancer; Medulloblastoma; Atypical teratoid/rhabdoid tumor; Poly(beta-amino ester) nanoparticle
资金
- NIH [R01CA228133]
- Johns Hopkins University Deans Research Fund
- NSF [DGE-1232825, DGE-0707427]
- Samsung
- Bloombergsimilar toKimmel Institute for Cancer Immunotherapy
- [EY001765]
Together, medulloblastoma (MB) and atypical teratoid/rhabdoid tumors (AT/RT) represent two of the most prevalent pediatric brain malignancies. Current treatment involves radiation, which has high risks of developmental sequelae for patients under the age of three. New safer and more effective treatment modalities are needed. Cancer gene therapy is a promising alternative, but there are challenges with using viruses in pediatric patients. We developed a library of poly(beta-amino ester) (PBAE) nanoparticles and evaluated their efficacy for plasmid delivery of a suicide gene therapy to pediatric brain cancer models-specifically herpes simplex virus type I thymidine kinase (HSVtk), which results in controlled apoptosis of transfected cells. In vivo, PBAE-HSVtk treated groups had a greater median overall survival in mice implanted with AT/RT (P = 0.0083 vs. control) and MB (P < 0.0001 vs. control). Our data provide proof of principle for using biodegradable PBAE nanoparticles as a safe and effective nanomedicine for treating pediatric CNS malignancies. (c) 2019 Elsevier Inc. All rights reserved.
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