Medical School

  • Medical University of Graz , Graz , Austria


  • Boston Children's Hospital , Boston , MA


Pediatric Hematology/Oncology
  • Dana-Farber/Boston Children's Cancer and Blood Disorders Center , Boston , MA


Dr. Mariella Filbin earned her MD and PhD in biochemistry and molecular biology from the Medical University of Graz, Austria. After a pediatric residency at Boston Children’s Hospital, she completed a fellowship in pediatric hematology/oncology at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. She joined the faculty at Dana-Farber/Boston Children's as a pediatric neuro-oncologist in July 2017. In addition to her role in the treatment of patients with brain tumors, her research focuses on establishing cellular networks of tumor dependencies in pediatric brain tumors with the goal of finding new targeted therapies. Current areas of emphasis are: (1) utilizing single-cell transcriptomics to determine the tumor architecture and heterogeneity in pediatric brain tumors and (2) exploring the downstream effects of epigenetic driver mutations and their potential therapeutic applications in DIPG and high-grade gliomas.


  • American Board of Pediatrics, General Pediatrics
  • American Board of Pediatrics, Pediatric Hematology and Oncology


Publications powered by Harvard Catalyst Profiles

  1. Targeting integrated epigenetic and metabolic pathways in lethal childhood PFA ependymomas. Sci Transl Med. 2021 10 06; 13(614):eabc0497. View abstract
  2. Targeting fibroblast growth factor receptors to combat aggressive ependymoma. Acta Neuropathol. 2021 08; 142(2):339-360. View abstract
  3. The imitation game: How glioblastoma outmaneuvers immune attack. Cell. 2021 04 29; 184(9):2278-2281. View abstract
  4. Outcomes after first relapse of childhood intracranial ependymoma. Pediatr Blood Cancer. 2021 08; 68(8):e28930. View abstract
  5. Understanding the epigenetic landscape and cellular architecture of childhood brain tumors. Neurochem Int. 2021 03; 144:104940. View abstract
  6. Histone H3.3G34-Mutant Interneuron Progenitors Co-opt PDGFRA for Gliomagenesis. Cell. 2020 12 10; 183(6):1617-1633.e22. View abstract
  7. 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
  8. Trametinib for the treatment of recurrent/progressive pediatric low-grade glioma. J Neurooncol. 2020 Sep; 149(2):253-262. View abstract
  9. Into Thin Air: Hypoxia Drives Metabolic and Epigenomic Deregulation of Lethal Pediatric Ependymoma. Dev Cell. 2020 07 20; 54(2):134-136. View abstract
  10. Molecular and clinicopathologic features of gliomas harboring NTRK fusions. Acta Neuropathol Commun. 2020 07 14; 8(1):107. View abstract
  11. 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
  12. The growing role of epigenetics in childhood cancers. Curr Opin Pediatr. 2020 02; 32(1):67-75. View abstract
  13. Stalled developmental programs at the root of pediatric brain tumors. Nat Genet. 2019 12; 51(12):1702-1713. View abstract
  14. 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
  15. 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
  16. Increasing value of autopsies in patients with brain tumors in the molecular era. J Neurooncol. 2019 Nov; 145(2):349-355. View abstract
  17. Rapid Generation of Somatic Mouse Mosaics with Locus-Specific, Stably Integrated Transgenic Elements. Cell. 2019 09 19; 179(1):251-267.e24. View abstract
  18. Mitogenic and progenitor gene programmes in single pilocytic astrocytoma cells. Nat Commun. 2019 08 19; 10(1):3731. View abstract
  19. Resolving medulloblastoma cellular architecture by single-cell genomics. Nature. 2019 08; 572(7767):74-79. View abstract
  20. An Integrative Model of Cellular States, Plasticity, and Genetics for Glioblastoma. Cell. 2019 08 08; 178(4):835-849.e21. View abstract
  21. Developmental origins and emerging therapeutic opportunities for childhood cancer. Nat Med. 2019 03; 25(3):367-376. View abstract
  22. Non-inflammatory tumor microenvironment of diffuse intrinsic pontine glioma. Acta Neuropathol Commun. 2018 06 28; 6(1):51. View abstract
  23. Developmental and oncogenic programs in H3K27M gliomas dissected by single-cell RNA-seq. Science. 2018 04 20; 360(6386):331-335. View abstract
  24. Gliomas in Children. Semin Neurol. 2018 02; 38(1):121-130. View abstract
  25. Molecular pathogenesis and therapeutic implications in pediatric high-grade gliomas. Pharmacol Ther. 2018 02; 182:70-79. View abstract
  26. 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
  27. Decoupling genetics, lineages, and microenvironment in IDH-mutant gliomas by single-cell RNA-seq. Science. 2017 03 31; 355(6332). View abstract
  28. Single-cell RNA-seq supports a developmental hierarchy in human oligodendroglioma. Nature. 2016 11 10; 539(7628):309-313. View abstract
  29. 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
  30. 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
  31. Of Brains and Blood: Developmental Origins of Glioma Diversity? Cancer Cell. 2015 Oct 12; 28(4):403-404. View abstract
  32. How neuronal activity regulates glioma cell proliferation. Neuro Oncol. 2015 Dec; 17(12):1543-4. View abstract
  33. Metallothionein-I/II Promotes Axonal Regeneration in the Central Nervous System. J Biol Chem. 2015 Jun 26; 290(26):16343-56. View abstract
  34. 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
  35. 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
  36. Coordinate activation of Shh and PI3K signaling in PTEN-deficient glioblastoma: new therapeutic opportunities. Nat Med. 2013 Nov; 19(11):1518-23. View abstract
  37. 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
  38. Rolipram promotes functional recovery after contusive thoracic spinal cord injury in rats. Behav Brain Res. 2013 Apr 15; 243:66-73. View abstract
  39. 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
  40. Feasibility and tolerability of bevacizumab in children with primary CNS tumors. Pediatr Blood Cancer. 2010 May; 54(5):681-6. View abstract
  41. 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
  42. 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
  43. Live or let die: CCM2 provides the link. Neuron. 2009 Sep 10; 63(5):559-60. View abstract
  44. 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
  45. Tumor stabilization under treatment with imatinib in progressive hypothalamic-chiasmatic glioma. Pediatr Blood Cancer. 2009 Apr; 52(4):476-80. View abstract
  46. Neurotrophin 3/TrkC-regulated proteins in the human medulloblastoma cell line DAOY. Electrophoresis. 2009 Feb; 30(3):540-9. View abstract
  47. Pharmacokinetics and safety of intrathecal liposomal cytarabine in children aged <3 years. Clin Pharmacokinet. 2009; 48(4):265-71. View abstract
  48. 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
  49. Mitosis-dependent protein expression in neuroblastoma cell line N1E-115. J Proteome Res. 2008 Aug; 7(8):3412-22. View abstract
  50. 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
  51. 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
  52. The role of cyclic AMP signaling in promoting axonal regeneration after spinal cord injury. Exp Neurol. 2008 Feb; 209(2):321-32. View abstract
  53. 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
  54. Therapeutic approaches to promoting axonal regeneration in the adult mammalian spinal cord. Int Rev Neurobiol. 2007; 77:57-105. View abstract
  55. Myelin-associated inhibitory signaling and strategies to overcome inhibition. J Cereb Blood Flow Metab. 2007 Jun; 27(6):1096-107. View abstract
  56. 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
  57. How inflammation promotes regeneration. Nat Neurosci. 2006 Jun; 9(6):715-7. View abstract
  58. Nectin-like molecule 1 is a high abundance protein in cerebellar neurons. Amino Acids. 2006 Jun; 30(4):409-15. View abstract
  59. 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
  60. 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
  61. MAG induces regulated intramembrane proteolysis of the p75 neurotrophin receptor to inhibit neurite outgrowth. Neuron. 2005 Jun 16; 46(6):849-55. View abstract
  62. Overcoming inhibitors in myelin to promote axonal regeneration. J Neurol Sci. 2005 Jun 15; 233(1-2):43-7. View abstract
  63. 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
  64. Expression of proteasomal proteins in ten different tumor cell lines. Amino Acids. 2004 Oct; 27(2):129-40. View abstract
  65. 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
  66. 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
  67. cAMP and Schwann cells promote axonal growth and functional recovery after spinal cord injury. Nat Med. 2004 Jun; 10(6):610-6. View abstract
  68. A role for cAMP in regeneration of the adult mammalian CNS. J Anat. 2004 Jan; 204(1):49-55. View abstract
  69. 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
  70. Myelin-associated inhibitors of axonal regeneration in the adult mammalian CNS. Nat Rev Neurosci. 2003 Sep; 4(9):703-13. View abstract
  71. New roles for old proteins in adult CNS axonal regeneration. Curr Opin Neurobiol. 2003 Feb; 13(1):133-9. View abstract
  72. 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
  73. Myelin-associated glycoprotein interacts with the Nogo66 receptor to inhibit neurite outgrowth. Neuron. 2002 Jul 18; 35(2):283-90. View abstract
  74. Spinal axon regeneration induced by elevation of cyclic AMP. Neuron. 2002 Jun 13; 34(6):895-903. View abstract
  75. A role for cAMP in regeneration during development and after injury. Prog Brain Res. 2002; 137:381-7. View abstract