Search

Simple Summary: Liquid biopsy is a technique that uses minimally invasive or noninvasive methods to detect disease biomarkers in biofluids, such as blood and urine. For childhood cancers, this approach is promising, especially when tissue biopsies are challenging. By analyzing tiny particles called extracellular vesicles, the small particles in biofluids which carry genetic and molecular information from cancer cells, this liquid biopsy technique can aid early diagnosis, treatment monitoring, and outcome prediction, potentially improving the outcomes of childhood cancers. This review article introduces the concept of extracellular vesicle liquid biopsy, summarizes the progress that is being made in the diagnosis of multiple types of pediatric malignancies, and provides prospects for using this extracellular vesicle method to guide the development of novel cancer therapeutics. Liquid biopsy involves the utilization of minimally invasive or noninvasive techniques to detect biomarkers in biofluids for disease diagnosis, monitoring, or guiding treatments. This approach is promising for the early diagnosis of childhood cancer, especially for brain tumors, where tissue biopsies are more challenging and cause late detection. Extracellular vesicles offer several characteristics that make them ideal resources for childhood cancer liquid biopsy. Extracellular vesicles are nanosized particles, primarily secreted by all cell types into body fluids such as blood and urine, and contain molecular cargos, i.e., lipids, proteins, and nucleic acids of original cells. Notably, the lipid bilayer-enclosed structure of extracellular vesicles protects their cargos from enzymatic degradation in the extracellular milieu. Proteins and nucleic acids of extracellular vesicles represent genetic alterations and molecular profiles of childhood cancer, thus serving as promising resources for precision medicine in cancer diagnosis, treatment monitoring, and prognosis prediction. This review evaluates the recent progress of extracellular vesicles as a liquid biopsy platform for various types of childhood cancer, discusses the mechanistic roles of molecular cargos in carcinogenesis and metastasis, and provides perspectives on extracellular vesicle-guided therapeutic intervention. Extracellular vesicle-based liquid biopsy for childhood cancer may ultimately contribute to improving patient outcomes. [ABSTRACT FROM AUTHOR]

Simple Summary: The detection of genomic aberrations in cancers has yielded a wealth of information to discover oncogenic drivers or pathogenic variants that are relevant for the development of precise treatment strategies. Recent studies have shown promising outcomes in adult cancer patients with well characterized cancer genetic biomarkers. However, the development of precise treatments for pediatric cancers is difficult due to the limited number of accessible samples and the fact that well-defined target genetic aberrations are limited. Here, we review the current landscape of pediatric precision oncology compared to adults and highlight the examples of single-arm and multiple-arm designs of pediatric precision treatments. Over the past decades, several study programs have conducted genetic testing in cancer patients to identify potential genetic targets for the development of precision therapeutic strategies. These biomarker-driven trials have demonstrated improved clinical outcomes and progression-free survival rates in various types of cancers, especially for adult malignancies. However, similar progress in pediatric cancers has been slow due to their distinguished mutation profiles compared to adults and the low frequency of recurrent genomic alterations. Recently, increased efforts to develop precision medicine for childhood malignancies have led to the identification of genomic alterations and transcriptomic profiles of pediatric patients which presents promising opportunities to study rare and difficult-to-access neoplasms. This review summarizes the current state of known and potential genetic markers for pediatric solid tumors and provides perspectives on precise therapeutic strategies that warrant further investigations. [ABSTRACT FROM AUTHOR]