Potent anti-tumor efficacy of palbociclib in treatment-naive H3.3K27M-mutant diffuse intrinsic pontine glioma.

ABSTRACT:

Background

Diffuse intrinsic pontine glioma (DIPG) is a rare and fatal pediatric brain cancer without cure. Seeking therapeutic strategies is still a major challenge in DIPG research. Previous study has shown that dysregulation of G1/S cell cycle checkpoint was common in DIPG and this dysregulation is even more enriched in the H3.3K27 M mutant subgroup. Here we assess potential anti-tumor efficacy of palbociclib, a specific and cytostatic inhibitor of CDK4/6, on high grade H3.3-K27 M-mutant DIPGs in vitro and in vivo.

Methods

We established patient-derived cell lines from treatment-naïve specimens. All the lines have H3.3K27 M mutation. We used a range of biological in vitro assays to assess the effect of palbociclib on growth of DIPGs. Palbociclib activity was also assayed

SUBMITTER: Sun Y 

PROVIDER: S-EPMC6558223 | biostudies-literature | 2019 May

REPOSITORIES: biostudies-literature