Fig. 7: PDGFR inhibition targets pericytes and decreases tumor cell population in FBO and in vivo.

A Left: mETMR-FBOs were treated with the PDGFR-inhibitor CP-673451 (10 μM) or vehicle at every medium exchange from day 15 (D15) to 30 (D30), followed by scRNA-seq at D30 (1 experiment). Right: UMAP of integrated dataset, colored by treatment - CP-673451 (n = 3), vehicle (n = 4) and untreated FBO controls (n = 2); n = biological replicates. B Cell type annotation of the mETMR-FBO dataset. ETMR subpopulations (RG-like, NProg-like and Nb-like) form distinct clusters from non-tumor FBO cells. C, D Boxplots showing the relative proportion (%) of PC cells (C) or ETMR cells (D) by condition in the dataset described in (A). Wilcoxon rank sum test, one-sided. Boxes represent the median (middle) and interquartile ranges (upper/lower hinges); whiskers show 1.5× IQR. C The PC population decreased from 0.44% (IQR 0.16–2.04) in vehicle-treated to 0.04% (IQR 0.03–0.52) in CP-673451-treated samples (p = 0.43). D The tumor population decreased from 3.87% (IQR 2.23–12.68) in vehicle-treated to 1.29% (IQR 1.19–2.02) in CP-673451-treated samples (p = 0.2). E Representative H&E and Ki67-stained histological images of ETMR-harboring murine brains at embryonic day E18.5, after 4 days of CP-673451 or vehicle treatment. Scale bar = 1 mm. F, G Quantification of tumor area (F) and ki67 positivity (G) in ETMR-bearing mice: vehicle-treated (n = 4) vs. CP-673451-treated (n = 3 animals); Wilcoxon rank-sum test, one sided. Boxes represent the median (middle) and interquartile ranges (upper/lower hinges); whiskers show 1.5× IQR. F Tumor fraction decreased in CP-673451-treated animals (mean = 18,78%; sd = 5,36) compared to vehicle-treated ones (mean = 48,09%; sd = 4,02). G Ki67+ areas decreased in CP-673451-treated animals (mean = 10,17%; sd = 1,72) compared to vehicle-treated ones (mean =2 28,61%; sd = 2,23). Source data of C, D, F and G are provided as a Source Data file. Sample type symbols in A–C, E, F created with BioRender.com.