Effective targeting of PDGFRA-altered high-grade glioma with avapritinib
Abstract
Details
- Title: Subtitle
- Effective targeting of PDGFRA-altered high-grade glioma with avapritinib
- Creators
- Lisa Mayr - Comprehensive Cancer Center ViennaSina Neyazi - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterKallen Schwark - University of MichiganMaria Trissal - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterAlexander Beck - Ludwig-Maximilians-Universität MünchenJenna Labelle - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterSebastian K. Eder - St Anna Children's HospitalLiesa Weiler-Wichtl - Comprehensive Cancer Center ViennaJoana G. Marques - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterCarlos A.O. de Biagi-Junior - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterCostanza Lo Cascio - Harvard Medical SchoolOwen Chapman - Sanford Burnham Prebys Medical Discovery InstituteSunita Sridhar - Sanford Burnham Prebys Medical Discovery InstituteRishaan Kenkre - Sanford Burnham Prebys Medical Discovery InstituteAditi Dutta - Sanford Burnham Prebys Medical Discovery InstituteShanqing Wang - Sanford Burnham Prebys Medical Discovery InstituteJessica Wang - Sanford Burnham Prebys Medical Discovery InstituteOlivia Hack - Harvard UniversityAndrezza Nascimento - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterCuong M. Nguyen - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterSophia Castellani - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterJacob S. Rozowsky - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterAndrew Groves - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterEshini Panditharatna - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterGustavo Alencastro Veiga Cruzeiro - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterRebecca D. Haase - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterKuscha Tabatabai - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterSibylle Madlener - Comprehensive Cancer Center ViennaJack Wadden - University of MichiganTiffany Adam - University of MichiganSeongbae Kong - University of MichiganMadeline Miclea - University of MichiganTirth Patel - University of MichiganKatharina Bruckner - Comprehensive Cancer Center ViennaDaniel Senfter - Comprehensive Cancer Center ViennaAnna Lämmerer - Comprehensive Cancer Center ViennaJeffrey Supko - Massachusetts General HospitalArmin S. Guntner - Johannes Kepler University of LinzHana Palova - Central European Institute of TechnologyJakub Neradil - University Hospital BrnoNatalia Stepien - Comprehensive Cancer Center ViennaDaniela Lötsch-Gojo - Medical University of ViennaWalter Berger - Comprehensive Cancer Center ViennaUlrike Leiss - Comprehensive Cancer Center ViennaVerena Rosenmayr - Medical University of ViennaChristian Dorfer - Comprehensive Cancer Center ViennaKarin Dieckmann - Medical University of ViennaAndreas Peyrl - Comprehensive Cancer Center ViennaAmedeo A. Azizi - Comprehensive Cancer Center ViennaAlicia Baumgartner - Comprehensive Cancer Center ViennaOndrej Slaby - Central European Institute of Technology – Masaryk UniversityPetra Pokorna - Central European Institute of TechnologyLouise M. Clark - Dana-Farber Cancer InstituteAmy Cameron - Dana-Farber Cancer InstituteQuang-De Nguyen - Dana-Farber Cancer InstituteHiroaki Wakimoto - Massachusetts General HospitalFrank Dubois - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterNoah F. Greenwald - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterPratiti Bandopadhayay - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterRameen Beroukhim - Broad InstituteKeith Ligon - Dana-Farber/Boston Children's Cancer and Blood Disorders CenterChristof Kramm - Universitätsmedizin GöttingenAnnika Bronsema - Universität HamburgSimon Bailey - Newcastle UniversityAna Guerreiro Stucklin - University Children's Hospital ZurichSabine Mueller - University of California, San FranciscoMary Skrypek - Children’s Minnesota - St. Paul HospitalNina Martinez - Thomas Jefferson UniversityDaniel C. Bowers - The University of Texas Southwestern Medical CenterDavid T.W. Jones - Hopp Children's Cancer Center HeidelbergChris Jones - Institute of Cancer ResearchNatalie Jäger - German Cancer Research CenterJaroslav Sterba - Masaryk UniversityLeonhard Müllauer - Medical University of ViennaChristine Haberler - Medical University of ViennaChandan Kumar-Sinha - University of MichiganArul Chinnaiyan - University of MichiganRajen Mody - University of MichiganLukas Chavez - Sanford Burnham Prebys Medical Discovery InstituteJulia Furtner - Danube Private UniversityCarl Koschmann - University of MichiganJohannes Gojo - Comprehensive Cancer Center ViennaMariella G. Filbin - Dana-Farber/Boston Children's Cancer and Blood Disorders Center
- Resource Type
- Journal article
- Publication Details
- Cancer cell, Vol.43(4), pp.740-756.e8
- DOI
- 10.1016/j.ccell.2025.02.018
- PMID
- 40086436
- PMCID
- PMC12121847
- NLM abbreviation
- Cancer Cell
- ISSN
- 1535-6108
- eISSN
- 1878-3686
- Publisher
- Elsevier Inc
- Grant note
- Sajni Chakrabarti Fund for Pediatric Brain Cancer ResearchCaroline Mortimer FundDMG Precision Medicine CollaborativeClaudia Adams Barr ProgramCuming Family Fund for Pediatric Brain Tumor ResearchAnita, SophiaAthena Fund to Advance DIPGMaleiaStrong FoundationMorris FamilyDIPG All-In InitiativeNIH Director's New Innovator Award: DP2NS127705 Burroughs Wellcome FundSontag FoundationHelen Gurley Brown Foundation Helen Gurley Brown Presidential Initiative at Dana-Farber Cancer InstituteAlex's Lemonade Stand FoundationNational Cancer Institute SPORE grant: P50CA165962 National Cancer Institute U54 grant: CA274516 National Institutes of Health: R01-NS119231, R01-NS124607 DOD: CA201129P1 University of Michigan Chad Carr Pediatric Brain Tumor Center, Evans FamilyChadTough Defeat DIPG FoundationPediatric Brain Tumor FoundationMichael Miller Memorial FoundationForschungsgesellschaft fuer cerebrale Tumore and VereinMedical University of ViennaMedical-scientific fund of the Mayor of the federal capital Vienna: 19086 Austrian Science Fund: T906-B28, P30105 Ministry of Health of the Czech Republic: NU20-03-00240 Cancer Research UKCRIS Cancer Foundation and Brain Tumour Research
We thank Leonie Mikael for the helpful review of the manuscript. We acknowledge the support of Antony Hsieh, Mikael L. Rinne, Nina Perez, Sasa Dimitrijevic, and Sean Kim from Blueprint Medicines Corporation for their involvement in human avapritinib pharmacokinetic data generation and their review of this manuscript. We further thank Georg Schrockenfuchs, Dominik Kirchhofer, Carola Jaunecker, and Johannes Hainfellner for their support of this study. Several figures were created with BioRender.com . This work was supported by Sajni Chakrabarti Fund for Pediatric Brain Cancer Research (M.G.F.) , Caroline Mortimer Fund (M.G.F.) , DMG Precision Medicine Collaborative (M.G.F.) , Claudia Adams Barr Program (M.G.F.) , Cuming Family Fund for Pediatric Brain Tumor Research (M.G.F.) , Anita, Sophia, and Athena Fund to Advance DIPG Research and Care (M.G.F.) , Liv Like A Unicorn (M.G.F.) , We love you Connie Fund (M.G.F.) , MaleiaStrong Foundation (M.G.F.) , The Morris Family (M.G.F.) and the DIPG All-In Initiative (M.G.F.) . M.G.F. holds a NIH Director's New Innovator Award (DP2NS127705) , a Career Award for Medical Scientist from the Burroughs Wellcome Fund, the Distinguished Scientist Award from the Sontag Foundation, the Helen Gurley Brown Foundation Helen Gurley Brown Presidential Initiative at Dana-Farber Cancer Institute, and the A-Award from the Alex's Lemonade Stand Foundation. M.G.F. was also supported by National Cancer Institute SPORE grant P50CA165962 and by National Cancer Institute U54 grant CA274516. C.K. was supported by National Institutes of Health Grant R01-NS119231 and R01-NS124607, DOD Grant CA201129P1, University of Michigan Chad Carr Pediatric Brain Tumor Center, Evans Family, ChadTough Defeat DIPG Foundation, Catching Up with Jack, Pediatric Brain Tumor Foundation, Michael Miller Memorial Foundation, Morgan Behen Golf Classic, and Yuvaan Tiwari Foundation. J.G. was supported by Forschungsgesellschaft fuer cerebrale Tumore and Verein unser_kind. Other funding sources were Physician Researcher Pathway Scholarship of the Medical University of Vienna (L.M.) , the Medical-scientific fund of the Mayor of the federal capital Vienna (project #19086, L.M.) , the Austrian Science Fund (project T906-B28 to D.L.G. and P30105 to W.B.) , Ministry of Health of the Czech Republic (Grant number NU20-03-00240; H.P., O.S., J.S.) , and Walter Benjamin Program of the German Research Foundation (S.N.) . C.J. was supported by Cancer Research UK, CRIS Cancer Foundation and Brain Tumour Research.
- Language
- English
- Electronic publication date
- 03/13/2025
- Date published
- 04/14/2025
- Academic Unit
- Stead Family Department of Pediatrics; Hematology/Oncology
- Record Identifier
- 9984801668502771