Nucleic acid therapy in pediatric cancer. (October 2022)

• Record Type: Journal Article
• Authors: Li, Yongshu; Huang, Bihui; Xue, Zhichao; Gao, Yunhua; Zhuo, Zhenjian
• Is Part Of: Pharmacological research. Volume 184(2022)
• Publication Date: 2022-10
• Subjects: siRNA short interfering RNA -- ASO antisense oligonucleotides -- mRNA messenger RNA -- CNS central nervous system -- NB neuroblastoma -- ALL acute myeloid leukemia -- WT Wilms tumors -- RB retinoblastoma -- OS osteosarcoma -- FDA United States Food and Drug Administration -- EMA European Medicines Agency -- GalNAc N-acetylgalactosamine -- LNPs lipid nanoparticles -- HTT gene Huntingtin gene -- LDL-C low-density lipoprotein cholesterol -- FH Familial hypercholesterolemia -- ALT alanine aminotransferase -- DMD Duchenne muscular dystrophy -- SMN survival of motor neuron -- SMA spinal muscular atrophy -- hATTR hereditary transthyretin mediated amyloidosis -- 2′-MOE 2'-O-methoxyethyl -- FCS familial chylomicronemia syndrome -- HTG hypertriglyceridemia -- TEAE treatment-emergent adverse event -- ALAS1 5'-Aminolevulinate Synthase 1 -- PH1 primary hyperoxaluria type 1 -- AGT glyoxylate aminotransferase -- LSM least-squares means -- PCSK9 proprotein convertase subtilsin/kexin type 9 -- modRNA modified messenger RNA -- LGG low-grade glioma -- HGG high-grade glioma -- DNET dysembryoplastic neuroepithelial tumor -- PA pilocytic astrocytoma -- GG ganglioglioma -- PXA pleomorphic xanthoastrocytoma -- SEGA subependymal giant cell astrocytoma -- DA diffuse astrocytoma -- OSR overall survival rate -- CMV cytomegalovirus -- DCs dendritic cells -- GM-CSF Granulocyte Macrophage-Colony Stimulating Factor -- PBMCs lysosomal associated membrane protein -- GBM Glioblastoma -- Lnc-RNA long non-coding RNA -- circRNAs Circular RNAs -- AE Adverse Events -- SAE Serious Adverse Events; Nucleic acid therapy -- Pediatric cancer -- Drug -- Treatment; Pharmacology -- Periodicals; Pharmacology -- Periodicals; Research -- Periodicals; Médicaments -- Recherche -- Périodiques; Pharmacologie -- Périodiques; 615.105
• DOI: 10.1016/j.phrs.2022.106441
• Languages: English
• ISSNs: 1043-6618

Abstract

Abstract: The overall survival, progress free survival, and life quality of cancer patients have improved due to the advance in minimally invasive surgery, precision radiotherapy, and various combined chemotherapy in the last decade. Furthermore, the discovery of new types of therapeutics, such as immune checkpoint inhibitors and immune cell therapies have facilitated both patients and doctors to fight with cancers. Moreover, in the context of the development in biocompatible and cell type targeting nano-carriers as well as nucleic acid-based drugs for initiating and enhancing the anti-tumor response have come to the age. The treatment paradigms utilization of nucleic acids, including short interfering RNA (siRNA), antisense oligonucleotides (ASO), and messenger RNA (mRNA), can target specific protein expression to achieve the therapeutic effects. Over ten nucleic acid therapeutics have been approved by the FDA and EMA in rare diseases and genetic diseases as well as dozens of registered clinical trails for varies cancers. Though generally less dangerous of pediatric cancers than adult cancers was observed during the past decades, yet pediatric cancers accounted for a significant proportion of child deaths which hurt those family very deeply. Therefore, it is necessary to pay more attention for improving the treatment of pediatric cancer and discovering new nucleic acid therapeutics which may help to improve the therapeutic effect and prognoses in turns to ameliorate the survival period and quality of life for children patient. In this review, we focus on the nucleic acid therapy in pediatric cancers. Graphical Abstract: ga1