Precision Medicine for Pediatric Glioma and NF1-Associated Tumors: The Role of Small Molecule Inhibitors
Pediatric gliomas encompass the most common brain tumor in children and are subdivided into pediatric low-grade gliomas (pLGGs) and pediatric high-grade gliomas (pHGGs). The era of molecular diagnosis has shifted the treatment paradigms and management of these patients. RAS/MAPK pathway alterations...
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| Language: | English |
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MDPI AG
2025-05-01
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| Series: | Current Oncology |
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| Online Access: | https://www.mdpi.com/1718-7729/32/5/280 |
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| author | Samuele Renzi Julie Bennett Nirav Thacker Chantel Cacciotti |
| author_facet | Samuele Renzi Julie Bennett Nirav Thacker Chantel Cacciotti |
| author_sort | Samuele Renzi |
| collection | DOAJ |
| description | Pediatric gliomas encompass the most common brain tumor in children and are subdivided into pediatric low-grade gliomas (pLGGs) and pediatric high-grade gliomas (pHGGs). The era of molecular diagnosis has shifted the treatment paradigms and management of these patients. RAS/MAPK pathway alterations serve as the driver in the majority of pLGGs, a subset of pHGG and NF1-related plexiform neurofibromas (PNs). The role of small molecule inhibitors in the treatment of these tumors has evolved in the past decade, facilitated through multiple clinical trials and moving into earlier stages of treatment. Although these developments hold promise, questions remain regarding targeted therapy, the long-term toxicities, the duration of treatment and the potential effects on the natural history of the tumor behavior. |
| format | Article |
| id | doaj-art-b5eb2001abff4d49aab1a359c7debc19 |
| institution | DOAJ |
| issn | 1198-0052 1718-7729 |
| language | English |
| publishDate | 2025-05-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Current Oncology |
| spelling | doaj-art-b5eb2001abff4d49aab1a359c7debc192025-08-20T03:14:39ZengMDPI AGCurrent Oncology1198-00521718-77292025-05-0132528010.3390/curroncol32050280Precision Medicine for Pediatric Glioma and NF1-Associated Tumors: The Role of Small Molecule InhibitorsSamuele Renzi0Julie Bennett1Nirav Thacker2Chantel Cacciotti3Division of Pediatric Hematology/Oncology, CHU de Québec-Université Laval, Québec City, QC G1V 0E8, CanadaDivision of Hematology/Oncology, The Hospital for Sick Children, Toronto, ON M5G 1E8, CanadaDivision of Hematology/Oncology, Children’s Hospital of Eastern Ontario (CHEO), Ottawa, ON K1H 8M8, CanadaDivision of Hematology/Oncology, Department of Pediatrics, London Health Sciences Centre & Western University, London, ON N6A 5W9, CanadaPediatric gliomas encompass the most common brain tumor in children and are subdivided into pediatric low-grade gliomas (pLGGs) and pediatric high-grade gliomas (pHGGs). The era of molecular diagnosis has shifted the treatment paradigms and management of these patients. RAS/MAPK pathway alterations serve as the driver in the majority of pLGGs, a subset of pHGG and NF1-related plexiform neurofibromas (PNs). The role of small molecule inhibitors in the treatment of these tumors has evolved in the past decade, facilitated through multiple clinical trials and moving into earlier stages of treatment. Although these developments hold promise, questions remain regarding targeted therapy, the long-term toxicities, the duration of treatment and the potential effects on the natural history of the tumor behavior.https://www.mdpi.com/1718-7729/32/5/280gliomaMEK inhibitorBRAF inhibitorPan-RAF inhibitorFGFR inhibitorTRK inhibitor |
| spellingShingle | Samuele Renzi Julie Bennett Nirav Thacker Chantel Cacciotti Precision Medicine for Pediatric Glioma and NF1-Associated Tumors: The Role of Small Molecule Inhibitors Current Oncology glioma MEK inhibitor BRAF inhibitor Pan-RAF inhibitor FGFR inhibitor TRK inhibitor |
| title | Precision Medicine for Pediatric Glioma and NF1-Associated Tumors: The Role of Small Molecule Inhibitors |
| title_full | Precision Medicine for Pediatric Glioma and NF1-Associated Tumors: The Role of Small Molecule Inhibitors |
| title_fullStr | Precision Medicine for Pediatric Glioma and NF1-Associated Tumors: The Role of Small Molecule Inhibitors |
| title_full_unstemmed | Precision Medicine for Pediatric Glioma and NF1-Associated Tumors: The Role of Small Molecule Inhibitors |
| title_short | Precision Medicine for Pediatric Glioma and NF1-Associated Tumors: The Role of Small Molecule Inhibitors |
| title_sort | precision medicine for pediatric glioma and nf1 associated tumors the role of small molecule inhibitors |
| topic | glioma MEK inhibitor BRAF inhibitor Pan-RAF inhibitor FGFR inhibitor TRK inhibitor |
| url | https://www.mdpi.com/1718-7729/32/5/280 |
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