PLX3397 attenuated tumor growth and remodeled tumor microenvironment of recurrent glioblastoma.

The tumor microenvironment (TME) is critically implicated in glioblastoma (GBM) recurrence, therapeutic resistance, and immune evasion. By analyzing both primary and recurrent GBM from Chinese Glioma Genome Atlas (CGGA) and clinical samples, we identified significant differences in TME. To overcome the limitations of traditional murine recurrent GBM model, we successfully established a new murine model and characterized the TME differences between primary and recurrent GBM in vivo. Based on immune chemokine profiling within the CGGA dataset, we selected PLX3397, a Colony stimulating factor 1 receptor (CSF1R) inhibitor, to target recurrent GBM in our murine model. PLX3397 treatment significantly attenuated tumor growth and remodeled TME. Collectively, our study firstly analyzed TME combine a large number of database samples and clinical samples, and made the first application of PLX3397 in a murine recurrent GBM model, thereby providing a novel therapeutic strategy and experimental foundation for recurrent GBM research.