Effective targeting of PDGFRA-altered high-grade glioma with avapritinib
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F65269705%3A_____%2F25%3A00081972" target="_blank" >RIV/65269705:_____/25:00081972 - isvavai.cz</a>
Alternative codes found
RIV/00216224:14110/25:00141295
Result on the web
<a href="https://www.sciencedirect.com/science/article/pii/S1535610825000704" target="_blank" >https://www.sciencedirect.com/science/article/pii/S1535610825000704</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1016/j.ccell.2025.02.018" target="_blank" >10.1016/j.ccell.2025.02.018</a>
Alternative languages
Result language
angličtina
Original language name
Effective targeting of PDGFRA-altered high-grade glioma with avapritinib
Original language description
PDGFRA is crucial to tumorigenesis and frequently genomically altered in high-grade glioma (HGG). In a comprehensive dataset of pediatric HGG (n = 261), we detect PDGFRA mutations and/or amplifications in 15% of cases, suggesting PDGFRA as a therapeutic target. We reveal that the PDGFRA/KIT inhibitor avapritinib shows (1) selectivity for PDGFRA inhibition, (2) distinct patterns of subcellular effects, (3) in vitro and in vivo activity in patient-derived HGG models, and (4) effective blood-brain barrier penetration in mice and humans. Furthermore, we report preliminary clinical real-world experience using avapritinib in pediatric and young adult patients with predominantly recurrent/refractory PDGFRA-altered HGG (n = 8). Our early data demonstrate that avapritinib is well tolerated and results in radiographic response in 3/7 cases, suggesting a potential role for avapritinib in the treatment of HGG with specific PDGFRA alterations. Overall, these translational results underscore the therapeutic potential of PDGFRA inhibition with avapritinib in HGG.
Czech name
—
Czech description
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Classification
Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
—
OECD FORD branch
30204 - Oncology
Result continuities
Project
<a href="/en/project/NU20-03-00240" target="_blank" >NU20-03-00240: Whole exome, low-coverage genome and transcriptome sequencing as tools for precision oncology in paediatric patients with high-risk and relapsed solid tumors</a><br>
Continuities
P - Projekt vyzkumu a vyvoje financovany z verejnych zdroju (s odkazem do CEP)
Others
Publication year
2025
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
Cancer Cell
ISSN
1535-6108
e-ISSN
1878-3686
Volume of the periodical
43
Issue of the periodical within the volume
4
Country of publishing house
US - UNITED STATES
Number of pages
26
Pages from-to
740-756
UT code for WoS article
001473767500001
EID of the result in the Scopus database
2-s2.0-86000767953