Abstract
Simple SummaryDespite the remarkable effort of researchers to find more effective treatments for pediatric brain tumors, the prognosis continues to be poor. Forty percent of pediatric patients develop treatment resistance and relapse, and the morbidities and long-term side effects of systemic therapy remain concerning. Significant advances have been made by next-generation genomic profiling. Novel oncogenic drivers have been identified as potential targets for the treatment of pediatric brain tumors. Aptamers, which are synthetic single-strand oligonucleotides, specifically target, bind with high affinity, internalize, and deliver a wide range of therapeutic moieties inside the cells. Although several aptamers have been tested in preclinical and clinical studies for adult glioblastoma, the use of aptamers in pediatric neuro-oncology remains unexplored. Increased knowledge regarding the molecular mechanisms of the pathogenesis of pediatric brain tumors, as well as selection of novel aptamers and/or adaptation of aptamers currently used in adult glioblastoma, might open a novel research field.A relatively new paradigm in cancer therapeutics is the use of cancer cell–specific aptamers, both as therapeutic agents and for targeted delivery of anticancer drugs. After the first therapeutic aptamer was described nearly 25 years ago, and the subsequent first aptamer drug approved, many efforts have been made to translate preclinical research into clinical oncology settings. Studies of aptamer-based technology have unveiled the vast potential of aptamers in therapeutic and diagnostic applications. Among pediatric solid cancers, brain tumors are the leading cause of death. Although a few aptamer-related translational studies have been performed in adult glioblastoma, the use of aptamers in pediatric neuro-oncology remains unexplored. This review will discuss the biology of aptamers, including mechanisms of targeting cell surface proteins, various modifications of aptamer structure to enhance therapeutic efficacy, the current state and challenges of aptamer use in neuro-oncology, and the potential therapeutic role of aptamers in pediatric brain tumors.
Highlights
These results suggest that aptamer-based technology is a simple yet powerful tool that holds potential in neuro-oncology
Using in vivo fluorescence imaging, the authors demonstrated that particles covered with bifunctional aptamers more efficiently penetrated the blood–brain barrier (BBB) and accumulated in the tumor compared with the particle alone in U251-CD133+ glioma tumor-bearing nude mice [75]
Increasing evidence indicates that aptamers are promising anticancer drugs owing to their unique characteristics
Summary
Nucleic acids have a multifunctional nature. Synthetic single-strand nucleic acid aptamers are able to form complex shapes acting as a scaffold for molecular interactions, with high specificity and affinity. Aptamers are synthetic single-strand oligonucleotides that can bind and regulate the activity of proteins based on their tertiary structural interactions They have the potential to target, bind with high affinity, internalize, and deliver a wide range of therapeutic moieties inside the cells. Antibodies are a potent therapeutic tool, their use for the treatment of brain diseases, including neurodegenerative diseases and brain cancers, has been limited, due to the blood–brain barrier (BBB), which makes brain tissue very difficult to access by traditional antibody strategies Along with overcoming these limitations, features such as low immunogenicity, high stability, easy tissue penetration, and accessibility to chemical modifications make aptamers preferred over conventional therapeutic antibodies [8,11]. We discuss the current state and challenges of aptamer use in neuro-oncology with a focus on pediatric neuro-oncology, a field in which aptamers have not been explored
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