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

Abstract

Sorafenib produced unexpected and unprecedented acceleration of tumor growth in children with PLGA, irrespective of NF1 or tumor BRAF status. In vitro studies with sorafenib indicate that this effect is likely related to paradoxical ERK activation. Close monitoring for early tumor progression should be included in trials of novel agents that modulate signal transduction.

Molecular pathology of adamantinomatous craniopharyngioma: review and opportunities for practice

“…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 …”

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

“…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).…”

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

“…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).…”