Phase II study of sorafenib in children with recurrent or progressive low-grade astrocytomas

Karajannis, Matthias A.; Legault, Geneviève; Fisher, Michael J.; Milla, Sarah Sarvis; Cohen, Kenneth J.; Wisoff, Jeffrey H.; Harter, David H.; Goldberg, Judith D.; Hochman, Tsivia; Merkelson, Amanda; Bloom, Michael; Sievert, Angela J.; Resnick, Adam; Dhall, Girish; Jones, David; Korshunov, Andrey; Pfister, Stefan M.; Eberhart, Charles G.; Zagzag, David; Allen, Jeffrey C.

doi:10.1093/neuonc/nou059

Cited by 192 publications

(173 citation statements)

References 32 publications

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“…The experience of use of SHH pathway inhibitors in pancreatic cancer, or BRAF inhibitors in BRAF-fusion gene-positive low-grade gliomas highlights this best, as treatment was found to promote rather than inhibit tumor growth. 27,32,43 …”

Section: Molecular Biology Of Tumor Recurrencementioning

confidence: 99%

Molecular pathology of adamantinomatous craniopharyngioma: review and opportunities for practice

Apps¹,

Martinez-Barbera²

2016

FOC

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Since the first identification of CTNNB1 mutations in adamantinomatous craniopharyngioma (ACP), much has been learned about the molecular pathways and processes that are disrupted in ACP pathogenesis. To date this understanding has not translated into tangible patient benefit. The recent development of novel techniques and a range of preclinical models now provides an opportunity to begin to support treatment decisions and develop new therapeutics based on molecular pathology. In this review the authors summarize many of the key findings and pathways implicated in ACP pathogenesis and discuss the challenges that need to be tackled to translate these basic science findings for the benefit of patients.

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Section: Molecular Biology Of Tumor Recurrencementioning

confidence: 99%

Molecular pathology of adamantinomatous craniopharyngioma: review and opportunities for practice

Apps¹,

Martinez-Barbera²

2016

FOC

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“…Initial experiences with certain chemotherapy regimens [i.e., cisplatin/carboplatin and etoposid; (Tsugu et al, 2009)] applied to PMAs provided promising results and underline the importance of further research of aggressive therapy strategies often required by PMA. In addition, there are ongoing discussions regarding the question if PMA and PA patients may qualify for a novel therapeutic approach with inhibitors targeting the RAF/MAPK/ERK pathway that was found to be activated in almost all tumors (Aktas et al, 2014;Karajannis et al, 2014).…”

Section: Discussionmentioning

confidence: 99%

Differential imaging characteristics and dissemination potential of pilomyxoid astrocytomas versus pilocytic astrocytomas

et al. 2015

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“…It has been shown to inhibit tumor growth in a mouse plexiform model [112]. Unfortunately, it was not tolerated by pediatric patients in a Phase I study [113] and either no tumor response or acceleration of tumor progression was observed in studies on OPG (NCT01338857) [114], PNF (NCT00727233) [113] and MPNST (NCT00245102).…”

Section: • Sunitinibmentioning

confidence: 99%

The role of the immune system in neurofibromatosis type 1-associated nervous system tumors

Karmakar

Reilly

2016

CNS Oncol.

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With the recent development of new anticancer therapies targeting the immune system, it is important to understand which immune cell types and cytokines play critical roles in suppressing or promoting tumorigenesis. The role of mast cells in promoting neurofibroma growth in neurofibromatosis type 1 (NF1) patients was hypothesized decades ago. More recent experiments in mouse models have demonstrated the causal role of mast cells in neurofibroma development and of microglia in optic pathway glioma development. We review here what is known about the role of NF1 mutation in immune cell function and the role of immune cells in promoting tumorigenesis in NF1. We also review the therapies targeting immune cell pathways and their promise in NF1 tumors.

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Phase II study of sorafenib in children with recurrent or progressive low-grade astrocytomas

Cited by 192 publications

References 32 publications

Molecular pathology of adamantinomatous craniopharyngioma: review and opportunities for practice

Molecular pathology of adamantinomatous craniopharyngioma: review and opportunities for practice

Differential imaging characteristics and dissemination potential of pilomyxoid astrocytomas versus pilocytic astrocytomas

The role of the immune system in neurofibromatosis type 1-associated nervous system tumors

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