title: Sorafenib plus valproic acid for infant spinal glioblastoma.
abstract: Spinal glioblastoma multiforme (GBM) is rare in children. New therapeutic options should be explored given the poor outcomes reported. We describe the case of an infant with spinal GBM whose condition worsened despite radiotherapy and chemotherapy. Immunohistochemical analysis of the tumor sample showed activation of the Raf-MEK-ERK pathway. Targeted pharmacologic therapy with sorafenib plus valproic acid led to decrease in the size of the tumor and improvement of symptoms. We conclude that regulation of the mitogen-activated protein kinase pathway using sorafenib plus valproic acid warrants further investigation for the management of childhood GBM.
authors.affiliation: Children's Cancer Hospital, The University of Texas MD Anderson Cancer Center, Houstan, TX 77030, USA.
authors.forename: Christopher Alan
authors.initials: CA
authors.lastname: Rokes
authors.forename: Mark
authors.initials: M
authors.lastname: Remke
authors.forename: Nandita
authors.initials: N
authors.lastname: Guha-Thakurta
authors.forename: Olaf
authors.initials: O
authors.lastname: Witt
authors.forename: Andrey
authors.initials: A
authors.lastname: Korshunov
authors.forename: Stephan
authors.initials: S
authors.lastname: Pfister
authors.forename: Johannes E
authors.initials: JE
authors.lastname: Wolff
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001557:Benzenesulfonates / Q000008:administration & dosage; D003131:Combined Modality Therapy; D048049:Extracellular Signal-Regulated MAP Kinases / Q000378:metabolism; D005260:Female; D005909:Glioblastoma / Q000188:drug therapy* / Q000378:metabolism; D006801:Humans; D007150:Immunohistochemistry; D007223:Infant; D020930:MAP Kinase Kinase Kinases / Q000378:metabolism; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy* / Q000378:metabolism; D009536:Niacinamide / Q000031:analogs & derivatives; D010671:Phenylurea Compounds; D011725:Pyridines / Q000008:administration & dosage; D011878:Radiotherapy; D000077157:Sorafenib; D013120:Spinal Cord Neoplasms / Q000188:drug therapy* / Q000378:metabolism; D014635:Valproic Acid / Q000008:administration & dosage; D048490:raf Kinases / Q000378:metabolism
chemical_list: D001557:Benzenesulfonates; D010671:Phenylurea Compounds; D011725:Pyridines; D009536:Niacinamide; D014635:Valproic Acid; D000077157:Sorafenib; D048490:raf Kinases; D048049:Extracellular Signal-Regulated MAP Kinases; D020930:MAP Kinase Kinase Kinases