Precision medicine in pediatric solid cancers

• Verfasst von: Blattner-Johnson, Mirjam [VerfasserIn]
• Verfasst von: Jones, David T. W. [VerfasserIn]
• Verfasst von: Pfaff, Elke [VerfasserIn]
• Titel: Precision medicine in pediatric solid cancers
• Verf.angabe: Mirjam Blattner-Johnson, David T.W. Jones, Elke Pfaff
• E-Jahr: 2022
• Jahr: September 2022
• Umfang: 14 S.
• Fussnoten: Gesehen am 11.11.2022
• Titel Quelle: Enthalten in: Seminars in cancer biology
• Ort Quelle: London : Academic Press, 1995
• Jahr Quelle: 2022
• Band/Heft Quelle: 84(2022), Seite 214-227
• ISSN Quelle: 1096-3650
• DOI: doi:10.1016/j.semcancer.2021.06.008
• Datenträger: Online-Ressource
• Sprache: eng
• Sach-SW: central-nervous-system
• Sach-SW: childrens oncology group
• Sach-SW: european consortium care
• Sach-SW: ewing sarcoma
• Sach-SW: Genomics
• Sach-SW: high-grade
• Sach-SW: large-cell lymphoma
• Sach-SW: long-term
• Sach-SW: pax3-foxo1 fusion gene
• Sach-SW: Pediatric cancer
• Sach-SW: phase-i trials
• Sach-SW: Precision medicine
• Sach-SW: soft-tissue tumors
• Sach-SW: Targeted therapy
• K10plus-PPN: 1822055059

Abstract

Despite huge advances in the diagnosis and treatment of pediatric cancers over the past several decades, it re-mains one of the leading causes of death during childhood in developed countries. The development of new targeted treatments for these diseases has been hampered by two major factors. First, the extremely heteroge-neous nature of the types of tumors encountered in this age group, and their fundamental differences from common adult carcinomas, has made it hard to truly get a handle on the complexities of the underlying biology driving tumor growth. Second, a reluctance of the pharmaceutical industry to develop products or trials for this population due to the relatively small size of the 'market', and a too-easy mechanism of obtaining waivers for pediatric development of adult oncology drugs based on disease type rather than mechanism of action, led to significant difficulties in getting access to new drugs. Thankfully, the field has now started to change, both scientifically and from a regulatory perspective, in order to address some of these challenges. In this review, we will examine some of the recent insights into molecular features which make pediatric tumors so unique and how these might represent therapeutic targets; highlight ongoing international initiatives for providing comprehen-sive, personalized genomic profiling of childhood tumors in a clinically-relevant timeframe, and look briefly at where the field of pediatric precision oncology may be heading in future.