title: Vismodegib Exerts Targeted Efficacy Against Recurrent Sonic Hedgehog-Subgroup Medulloblastoma: Results From Phase II Pediatric Brain Tumor Consortium Studies PBTC-025B and PBTC-032.
abstract: OBJECTIVE
  Two phase II studies assessed the efficacy of vismodegib, a sonic hedgehog (SHH) pathway inhibitor that binds smoothened (SMO), in pediatric and adult recurrent medulloblastoma (MB).
  
  
  METHODS
  Adult patients enrolled onto PBTC-025B and pediatric patients enrolled onto PBTC-032 were treated with vismodegib (150 to 300 mg/d). Protocol-defined response, which had to be sustained for 8 weeks, was confirmed by central neuroimaging review. Molecular tests to identify patterns of response and insensitivity were performed when tissue was available.
  
  
  RESULTS
  A total of 31 patients were enrolled onto PBTC-025B, and 12 were enrolled onto PBTC-032. Three patients in PBTC-025B and one in PBTC-032, all with SHH-subgroup MB (SHH-MB), exhibited protocol-defined responses. Progression-free survival (PFS) was longer in those with SHH-MB than in those with non-SHH-MB, and prolonged disease stabilization occurred in 41% of patient cases of SHH-MB. Among those with SHH-MB, loss of heterozygosity of PTCH1 was associated with prolonged PFS, and diffuse staining of P53 was associated with reduced PFS. Whole-exome sequencing identified mutations in SHH genes downstream from SMO in four of four tissue samples from nonresponders and upstream of SMO in two of four patients with favorable responses.
  
  
  CONCLUSIONS
  Vismodegib exhibits activity against adult recurrent SHH-MB but not against recurrent non-SHH-MB. Inadequate accrual of pediatric patients precluded conclusions in this population. Molecular analyses support the hypothesis that SMO inhibitor activity depends on the genomic aberrations within the tumor. Such inhibitors should be advanced in SHH-MB studies; however, molecular and genomic work remains imperative to identify target populations that will truly benefit.
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA. giles.robinson@stjude.org.
authors.forename: Giles W
authors.initials: GW
authors.lastname: Robinson
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Brent A
authors.initials: BA
authors.lastname: Orr
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Gang
authors.initials: G
authors.lastname: Wu
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Sridharan
authors.initials: S
authors.lastname: Gururangan
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Tong
authors.initials: T
authors.lastname: Lin
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Ibrahim
authors.initials: I
authors.lastname: Qaddoumi
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Roger J
authors.initials: RJ
authors.lastname: Packer
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Stewart
authors.initials: S
authors.lastname: Goldman
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Michael D
authors.initials: MD
authors.lastname: Prados
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Annick
authors.initials: A
authors.lastname: Desjardins
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Murali
authors.initials: M
authors.lastname: Chintagumpala
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Naoko
authors.initials: N
authors.lastname: Takebe
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Sue C
authors.initials: SC
authors.lastname: Kaste
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Michael
authors.initials: M
authors.lastname: Rusch
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Sariah J
authors.initials: SJ
authors.lastname: Allen
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Arzu
authors.initials: A
authors.lastname: Onar-Thomas
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Clinton F
authors.initials: CF
authors.lastname: Stewart
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Maryam
authors.initials: M
authors.lastname: Fouladi
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: James M
authors.initials: JM
authors.lastname: Boyett
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Richard J
authors.initials: RJ
authors.lastname: Gilbertson
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Tom
authors.initials: T
authors.lastname: Curran
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: David W
authors.initials: DW
authors.lastname: Ellison
authors.affiliation: Giles W. Robinson, Brent A. Orr, Gang Wu, Tong Lin, Ibrahim Qaddoumi, Sue C. Kaste, Michael Rusch, Sariah J. Allen, Arzu Onar-Thomas, Clinton F. Stewart, James M. Boyett, Richard J. Gilbertson, David W. Ellison, and Amar Gajjar, St Jude Children's Research Hospital, Memphis, TN; Sridharan Gururangan and Annick Desjardins, Duke University Medical Center, Durham, NC; Roger J. Packer, Children's National Medical Center, Washington, DC; Stewart Goldman, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Michael D. Prados, University of California San Francisco, San Francisco, CA; Murali Chintagumpala, Texas Children's Cancer Center, Houston, TX; Naoko Takebe, National Cancer Institute, Bethesda, MD; Maryam Fouladi, Cincinnati Children's Hospital, Cincinnati, OH; and Tom Curran, Children's Hospital of Philadelphia, Philadelphia, PA.
authors.forename: Amar
authors.initials: A
authors.lastname: Gajjar
mesh_terms: D000328:Adult; D000813:Anilides / Q000627:therapeutic use*; D001932:Brain Neoplasms / Q000188:drug therapy* / Q000235:genetics / Q000378:metabolism; D005260:Female; D053823:Hedgehog Proteins / Q000235:genetics / Q000378:metabolism*; D006801:Humans; D008297:Male; D008527:Medulloblastoma / Q000188:drug therapy* / Q000235:genetics / Q000378:metabolism; D008875:Middle Aged; D011725:Pyridines / Q000627:therapeutic use*; D055815:Young Adult
chemical_list: D000813:Anilides; D053823:Hedgehog Proteins; C538724:HhAntag691; D011725:Pyridines; C509239:SHH protein, human