Mariella G. Filbin

Publications

1. Paediatric strategy forum for medicinal product development in diffuse midline gliomas in children and adolescents ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration. Eur J Cancer. 2025 Jan 13; 217:115230. View Abstract
2. Multi-omics approaches reveal that diffuse midline gliomas present altered DNA replication and are susceptible to replication stress therapy. Genome Biol. 2024 Dec 20; 25(1):319. View Abstract
3. Applying single-cell and single-nucleus genomics to studies of cellular heterogeneity and cell fate transitions in the nervous system. Nat Neurosci. 2024 Dec; 27(12):2278-2291. View Abstract
4. Neuroimmune-competent human brain organoid model of Diffuse Midline Glioma. Neuro Oncol. 2024 Nov 19. View Abstract
5. GABAergic neuronal lineage development determines clinically actionable targets in diffuse hemispheric glioma, H3G34-mutant. Cancer Cell. 2024 Aug 27. View Abstract
6. Diffuse midline glioma invasion and metastasis rely on cell-autonomous signaling. Neuro Oncol. 2024 03 04; 26(3):553-568. View Abstract
7. Clinically relevant molecular hallmarks of PFA ependymomas display intratumoral heterogeneity and correlate with tumor morphology. Acta Neuropathol. 2024 01 24; 147(1):23. View Abstract
8. An ERK5-PFKFB3 axis regulates glycolysis and represents a therapeutic vulnerability in pediatric diffuse midline glioma. Cell Rep. 2024 01 23; 43(1):113557. View Abstract
9. A road map for the treatment of pediatric diffuse midline glioma. Cancer Cell. 2024 01 08; 42(1):1-5. View Abstract
10. Clinical Efficacy of ONC201 in H3K27M-Mutant Diffuse Midline Gliomas Is Driven by Disruption of Integrated Metabolic and Epigenetic Pathways. Cancer Discov. 2023 11 01; 13(11):2370-2393. View Abstract
11. TIM-3 blockade in diffuse intrinsic pontine glioma models promotes tumor regression and antitumor immune memory. Cancer Cell. 2023 11 13; 41(11):1911-1926.e8. View Abstract
12. Mapping pediatric brain tumors to their origins in the developing cerebellum. Neuro Oncol. 2023 10 03; 25(10):1895-1909. View Abstract
13. A comprehensive genomic study of 390 H3F3A-mutant pediatric and adult diffuse high-grade gliomas, CNS WHO grade 4. Acta Neuropathol. 2023 09; 146(3):515-525. View Abstract
14. Why haven't we solved intracranial pediatric ependymoma? Current questions and barriers to treatment advances. Neoplasia. 2023 05; 39:100895. View Abstract
15. Characterizing the biology of primary brain tumors and their microenvironment via single-cell profiling methods. Neuro Oncol. 2023 02 14; 25(2):234-247. View Abstract
16. Common molecular features of H3K27M DMGs and PFA ependymomas map to hindbrain developmental pathways. Acta Neuropathol Commun. 2023 02 09; 11(1):25. View Abstract
17. Neuronal-Activity Dependent Mechanisms of Small Cell Lung Cancer Progression. bioRxiv. 2023 Jan 20. View Abstract
18. Erratum to: Characterizing the biology of primary brain tumors and their microenvironment via single-cell profiling methods. Neuro Oncol. 2023 Jan 05; 25(1):211. View Abstract
19. Therapeutic targeting of prenatal pontine ID1 signaling in diffuse midline glioma. Neuro Oncol. 2023 01 05; 25(1):54-67. View Abstract
20. Activation of Hedgehog signaling by the oncogenic RELA fusion reveals a primary cilia-dependent vulnerability in supratentorial ependymoma. Neuro Oncol. 2023 01 05; 25(1):185-198. View Abstract
21. The landscape of tumor cell states and spatial organization in H3-K27M mutant diffuse midline glioma across age and location. Nat Genet. 2022 12; 54(12):1881-1894. View Abstract
22. BAF Complex Maintains Glioma Stem Cells in Pediatric H3K27M Glioma. Cancer Discov. 2022 12 02; 12(12):2880-2905. View Abstract
23. VRK1 as a synthetic lethal target in VRK2 promoter-methylated cancers of the nervous system. JCI Insight. 2022 10 10; 7(19). View Abstract
24. Immunotherapy approaches for the treatment of diffuse midline gliomas. Oncoimmunology. 2022; 11(1):2124058. View Abstract
25. Imipridones affect tumor bioenergetics and promote cell lineage differentiation in diffuse midline gliomas. Neuro Oncol. 2022 09 01; 24(9):1438-1451. View Abstract
26. A druggable addiction to de novo pyrimidine biosynthesis in diffuse midline glioma. Cancer Cell. 2022 09 12; 40(9):957-972.e10. View Abstract
27. An affinity for brainstem microglia in pediatric high-grade gliomas of brainstem origin. Neurooncol Adv. 2022 Jan-Dec; 4(1):vdac117. View Abstract
28. Single-cell epigenetic analysis reveals principles of chromatin states in H3.3-K27M gliomas. Mol Cell. 2022 07 21; 82(14):2696-2713.e9. View Abstract
29. Bromodomain and Extra-Terminal Protein Inhibitors: Biologic Insights and Therapeutic Potential in Pediatric Brain Tumors. Pharmaceuticals (Basel). 2022 May 26; 15(6). View Abstract
30. GD2-CAR T cell therapy for H3K27M-mutated diffuse midline gliomas. Nature. 2022 03; 603(7903):934-941. View Abstract
31. Targeting integrated epigenetic and metabolic pathways in lethal childhood PFA ependymomas. Sci Transl Med. 2021 Oct 06; 13(614):eabc0497. View Abstract
32. Targeting fibroblast growth factor receptors to combat aggressive ependymoma. Acta Neuropathol. 2021 08; 142(2):339-360. View Abstract
33. The imitation game: How glioblastoma outmaneuvers immune attack. Cell. 2021 04 29; 184(9):2278-2281. View Abstract
34. Outcomes after first relapse of childhood intracranial ependymoma. Pediatr Blood Cancer. 2021 08; 68(8):e28930. View Abstract
35. Understanding the epigenetic landscape and cellular architecture of childhood brain tumors. Neurochem Int. 2021 03; 144:104940. View Abstract
36. Histone H3.3G34-Mutant Interneuron Progenitors Co-opt PDGFRA for Gliomagenesis. Cell. 2020 12 10; 183(6):1617-1633.e22. View Abstract
37. Infiltrative gliomas of the thalamus in children: the role of surgery in the era of H3 K27M mutant midline gliomas. Acta Neurochir (Wien). 2021 07; 163(7):2025-2035. View Abstract
38. Trametinib for the treatment of recurrent/progressive pediatric low-grade glioma. J Neurooncol. 2020 Sep; 149(2):253-262. View Abstract
39. Into Thin Air: Hypoxia Drives Metabolic and Epigenomic Deregulation of Lethal Pediatric Ependymoma. Dev Cell. 2020 07 20; 54(2):134-136. View Abstract
40. Molecular and clinicopathologic features of gliomas harboring NTRK fusions. Acta Neuropathol Commun. 2020 07 14; 8(1):107. View Abstract
41. Single-Cell RNA-Seq Reveals Cellular Hierarchies and Impaired Developmental Trajectories in Pediatric Ependymoma. Cancer Cell. 2020 07 13; 38(1):44-59.e9. View Abstract
42. The growing role of epigenetics in childhood cancers. Curr Opin Pediatr. 2020 02; 32(1):67-75. View Abstract
43. Stalled developmental programs at the root of pediatric brain tumors. Nat Genet. 2019 12; 51(12):1702-1713. View Abstract
44. Re-programing Chromatin with a Bifunctional LSD1/HDAC Inhibitor Induces Therapeutic Differentiation in DIPG. Cancer Cell. 2019 11 11; 36(5):528-544.e10. View Abstract
45. Histone Variant and Cell Context Determine H3K27M Reprogramming of the Enhancer Landscape and Oncogenic State. Mol Cell. 2019 12 19; 76(6):965-980.e12. View Abstract
46. Increasing value of autopsies in patients with brain tumors in the molecular era. J Neurooncol. 2019 Nov; 145(2):349-355. View Abstract
47. Rapid Generation of Somatic Mouse Mosaics with Locus-Specific, Stably Integrated Transgenic Elements. Cell. 2019 09 19; 179(1):251-267.e24. View Abstract
48. Mitogenic and progenitor gene programmes in single pilocytic astrocytoma cells. Nat Commun. 2019 08 19; 10(1):3731. View Abstract
49. Resolving medulloblastoma cellular architecture by single-cell genomics. Nature. 2019 08; 572(7767):74-79. View Abstract
50. An Integrative Model of Cellular States, Plasticity, and Genetics for Glioblastoma. Cell. 2019 08 08; 178(4):835-849.e21. View Abstract
51. Developmental origins and emerging therapeutic opportunities for childhood cancer. Nat Med. 2019 03; 25(3):367-376. View Abstract
52. Non-inflammatory tumor microenvironment of diffuse intrinsic pontine glioma. Acta Neuropathol Commun. 2018 06 28; 6(1):51. View Abstract
53. Developmental and oncogenic programs in H3K27M gliomas dissected by single-cell RNA-seq. Science. 2018 04 20; 360(6386):331-335. View Abstract
54. Gliomas in Children. Semin Neurol. 2018 02; 38(1):121-130. View Abstract
55. Molecular pathogenesis and therapeutic implications in pediatric high-grade gliomas. Pharmacol Ther. 2018 02; 182:70-79. View Abstract
56. Clinical targeted exome-based sequencing in combination with genome-wide copy number profiling: precision medicine analysis of 203 pediatric brain tumors. Neuro Oncol. 2017 Jul 01; 19(7):986-996. View Abstract
57. Decoupling genetics, lineages, and microenvironment in IDH-mutant gliomas by single-cell RNA-seq. Science. 2017 03 31; 355(6332). View Abstract
58. Single-cell RNA-seq supports a developmental hierarchy in human oligodendroglioma. Nature. 2016 11 10; 539(7628):309-313. View Abstract
59. Gliomas Genomics and Epigenomics: Arriving at the Start and Knowing It for the First Time. Annu Rev Pathol. 2016 05 23; 11:497-521. View Abstract
60. A Brain Tumor/Organotypic Slice Co-culture System for Studying Tumor Microenvironment and Targeted Drug Therapies. J Vis Exp. 2015 Nov 07; (105):e53304. View Abstract
61. Of Brains and Blood: Developmental Origins of Glioma Diversity? Cancer Cell. 2015 Oct 12; 28(4):403-404. View Abstract
62. How neuronal activity regulates glioma cell proliferation. Neuro Oncol. 2015 Dec; 17(12):1543-4. View Abstract
63. Metallothionein-I/II Promotes Axonal Regeneration in the Central Nervous System. J Biol Chem. 2015 Jun 26; 290(26):16343-56. View Abstract
64. Soluble adenylyl cyclase is necessary and sufficient to overcome the block of axonal growth by myelin-associated factors. J Neurosci. 2014 Jul 09; 34(28):9281-9. View Abstract
65. PTEN inhibition enhances neurite outgrowth in human embryonic stem cell-derived neuronal progenitor cells. J Comp Neurol. 2014 Aug 15; 522(12):2741-55. View Abstract
66. Coordinate activation of Shh and PI3K signaling in PTEN-deficient glioblastoma: new therapeutic opportunities. Nat Med. 2013 Nov; 19(11):1518-23. View Abstract
67. Secretory leukocyte protease inhibitor reverses inhibition by CNS myelin, promotes regeneration in the optic nerve, and suppresses expression of the transforming growth factor-ß signaling protein Smad2. J Neurosci. 2013 Mar 20; 33(12):5138-51. View Abstract
68. Rolipram promotes functional recovery after contusive thoracic spinal cord injury in rats. Behav Brain Res. 2013 Apr 15; 243:66-73. View Abstract
69. A novel role for PTEN in the inhibition of neurite outgrowth by myelin-associated glycoprotein in cortical neurons. Mol Cell Neurosci. 2011 Jan; 46(1):235-44. View Abstract
70. Feasibility and tolerability of bevacizumab in children with primary CNS tumors. Pediatr Blood Cancer. 2010 May; 54(5):681-6. View Abstract
71. A large-scale chemical screen for regulators of the arginase 1 promoter identifies the soy isoflavone daidzeinas a clinically approved small molecule that can promote neuronal protection or regeneration via a cAMP-independent pathway. J Neurosci. 2010 Jan 13; 30(2):739-48. View Abstract
72. Combined intrinsic and extrinsic neuronal mechanisms facilitate bridging axonal regeneration one year after spinal cord injury. Neuron. 2009 Oct 29; 64(2):165-72. View Abstract
73. Live or let die: CCM2 provides the link. Neuron. 2009 Sep 10; 63(5):559-60. View Abstract
74. Increased synthesis of spermidine as a result of upregulation of arginase I promotes axonal regeneration in culture and in vivo. J Neurosci. 2009 Jul 29; 29(30):9545-52. View Abstract
75. Tumor stabilization under treatment with imatinib in progressive hypothalamic-chiasmatic glioma. Pediatr Blood Cancer. 2009 Apr; 52(4):476-80. View Abstract
76. Neurotrophin 3/TrkC-regulated proteins in the human medulloblastoma cell line DAOY. Electrophoresis. 2009 Feb; 30(3):540-9. View Abstract
77. Pharmacokinetics and safety of intrathecal liposomal cytarabine in children aged <3 years. Clin Pharmacokinet. 2009; 48(4):265-71. View Abstract
78. PirB, a second receptor for the myelin inhibitors of axonal regeneration Nogo66, MAG, and OMgp: implications for regeneration in vivo. Neuron. 2008 Dec 10; 60(5):740-2. View Abstract
79. Mitosis-dependent protein expression in neuroblastoma cell line N1E-115. J Proteome Res. 2008 Aug; 7(8):3412-22. View Abstract
80. Synthesis, chaperoning, and metabolism of proteins are regulated by NT-3/TrkC signaling in the medulloblastoma cell line DAOY. J Proteome Res. 2008 May; 7(5):1932-44. View Abstract
81. BDNF activates CaMKIV and PKA in parallel to block MAG-mediated inhibition of neurite outgrowth. Mol Cell Neurosci. 2008 May; 38(1):110-6. View Abstract
82. The role of cyclic AMP signaling in promoting axonal regeneration after spinal cord injury. Exp Neurol. 2008 Feb; 209(2):321-32. View Abstract
83. The inhibition site on myelin-associated glycoprotein is within Ig-domain 5 and is distinct from the sialic acid binding site. J Neurosci. 2007 Aug 22; 27(34):9146-54. View Abstract
84. Therapeutic approaches to promoting axonal regeneration in the adult mammalian spinal cord. Int Rev Neurobiol. 2007; 77:57-105. View Abstract
85. Myelin-associated inhibitory signaling and strategies to overcome inhibition. J Cereb Blood Flow Metab. 2007 Jun; 27(6):1096-107. View Abstract
86. Recapitulate development to promote axonal regeneration: good or bad approach? Philos Trans R Soc Lond B Biol Sci. 2006 Sep 29; 361(1473):1565-74. View Abstract
87. How inflammation promotes regeneration. Nat Neurosci. 2006 Jun; 9(6):715-7. View Abstract
88. Nectin-like molecule 1 is a high abundance protein in cerebellar neurons. Amino Acids. 2006 Jun; 30(4):409-15. View Abstract
89. The cytokine interleukin-6 is sufficient but not necessary to mimic the peripheral conditioning lesion effect on axonal growth. J Neurosci. 2006 May 17; 26(20):5565-73. View Abstract
90. The medulloblastoma cell line DAOY but not eleven other tumor cell lines expresses minichromosome maintenance protein 4. Cancer Lett. 2006 Jul 08; 238(1):76-84. View Abstract
91. MAG induces regulated intramembrane proteolysis of the p75 neurotrophin receptor to inhibit neurite outgrowth. Neuron. 2005 Jun 16; 46(6):849-55. View Abstract
92. Overcoming inhibitors in myelin to promote axonal regeneration. J Neurol Sci. 2005 Jun 15; 233(1-2):43-7. View Abstract
93. Activated CREB is sufficient to overcome inhibitors in myelin and promote spinal axon regeneration in vivo. Neuron. 2004 Nov 18; 44(4):609-21. View Abstract
94. Expression of proteasomal proteins in ten different tumor cell lines. Amino Acids. 2004 Oct; 27(2):129-40. View Abstract
95. Combinatorial therapy with neurotrophins and cAMP promotes axonal regeneration beyond sites of spinal cord injury. J Neurosci. 2004 Jul 14; 24(28):6402-9. View Abstract
96. The phosphodiesterase inhibitor rolipram delivered after a spinal cord lesion promotes axonal regeneration and functional recovery. Proc Natl Acad Sci U S A. 2004 Jun 08; 101(23):8786-90. View Abstract
97. cAMP and Schwann cells promote axonal growth and functional recovery after spinal cord injury. Nat Med. 2004 Jun; 10(6):610-6. View Abstract
98. A role for cAMP in regeneration of the adult mammalian CNS. J Anat. 2004 Jan; 204(1):49-55. View Abstract
99. Neurotrophins elevate cAMP to reach a threshold required to overcome inhibition by MAG through extracellular signal-regulated kinase-dependent inhibition of phosphodiesterase. J Neurosci. 2003 Dec 17; 23(37):11770-7. View Abstract
100. Myelin-associated inhibitors of axonal regeneration in the adult mammalian CNS. Nat Rev Neurosci. 2003 Sep; 4(9):703-13. View Abstract
101. New roles for old proteins in adult CNS axonal regeneration. Curr Opin Neurobiol. 2003 Feb; 13(1):133-9. View Abstract
102. Arginase I and polyamines act downstream from cyclic AMP in overcoming inhibition of axonal growth MAG and myelin in vitro. Neuron. 2002 Aug 15; 35(4):711-9. View Abstract
103. Myelin-associated glycoprotein interacts with the Nogo66 receptor to inhibit neurite outgrowth. Neuron. 2002 Jul 18; 35(2):283-90. View Abstract
104. Spinal axon regeneration induced by elevation of cyclic AMP. Neuron. 2002 Jun 13; 34(6):895-903. View Abstract
105. A role for cAMP in regeneration during development and after injury. Prog Brain Res. 2002; 137:381-7. View Abstract
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