[HTML][HTML] Combined VEGF and CXCR4 antagonism targets the GBM stem cell population and synergistically improves survival in an intracranial mouse model of …

A Barone, R Sengupta, NM Warrington, E Smith… - Oncotarget, 2014 - ncbi.nlm.nih.gov
A Barone, R Sengupta, NM Warrington, E Smith, PY Wen, RA Brekken, B Romagnoli…
Oncotarget, 2014ncbi.nlm.nih.gov
Glioblastoma recurrence involves the persistence of a subpopulation of cells with enhanced
tumor-initiating capacity (TIC) that reside within the perivascular space, or niche (PVN). Anti-
angiogenic therapies may prevent the formation of new PVN but have not prevented
recurrence in clinical trials, suggesting they cannot abrogate TIC activity. We hypothesized
that combining anti-angiogenic therapy with blockade of PVN function would have superior
anti-tumor activity. We tested this hypothesis in an established intracranial xenograft model …
Abstract
Glioblastoma recurrence involves the persistence of a subpopulation of cells with enhanced tumor-initiating capacity (TIC) that reside within the perivascular space, or niche (PVN). Anti-angiogenic therapies may prevent the formation of new PVN but have not prevented recurrence in clinical trials, suggesting they cannot abrogate TIC activity. We hypothesized that combining anti-angiogenic therapy with blockade of PVN function would have superior anti-tumor activity. We tested this hypothesis in an established intracranial xenograft model of GBM using a monoclonal antibody specific for murine and human VEGF (mcr84) and a Protein Epitope Mimetic (PEM) CXCR4 antagonist, POL5551. When doses of POL5551 were increased to overcome an mcr84-induced improvement in vascular barrier function, combinatorial therapy significantly inhibited intracranial tumor growth and improved survival. Anti-tumor activity was associated with significant changes in tumor cell proliferation and apoptosis, and a reduction in the numbers of perivascular cells expressing the TIC marker nestin. A direct effect on TICs was demonstrated for POL5551, but not mcr84, in three primary patient-derived GBM isolates. These findings indicate that targeting the structure and function of the PVN has superior anti-tumor effect and provide a strong rationale for clinical evaluation of POL5551 and Avastin in patients with GBM.
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