[HTML][HTML] RBPJ maintains brain tumor–initiating cells through CDK9-mediated transcriptional elongation

Q Xie, Q Wu, L Kim, TE Miller, BB Liau… - The Journal of …, 2016 - Am Soc Clin Investig
The Journal of clinical investigation, 2016Am Soc Clin Investig
Glioblastomas co-opt stem cell regulatory pathways to maintain brain tumor–initiating cells
(BTICs), also known as cancer stem cells. NOTCH signaling has been a molecular target in
BTICs, but NOTCH antagonists have demonstrated limited efficacy in clinical trials.
Recombining binding protein suppressor of hairless (RBPJ) is considered a central
transcriptional mediator of NOTCH activity. Here, we report that pharmacologic NOTCH
inhibitors were less effective than targeting RBPJ in suppressing tumor growth. While …
Glioblastomas co-opt stem cell regulatory pathways to maintain brain tumor–initiating cells (BTICs), also known as cancer stem cells. NOTCH signaling has been a molecular target in BTICs, but NOTCH antagonists have demonstrated limited efficacy in clinical trials. Recombining binding protein suppressor of hairless (RBPJ) is considered a central transcriptional mediator of NOTCH activity. Here, we report that pharmacologic NOTCH inhibitors were less effective than targeting RBPJ in suppressing tumor growth. While NOTCH inhibitors decreased canonical NOTCH gene expression, RBPJ regulated a distinct profile of genes critical to BTIC stemness and cell cycle progression. RBPJ was preferentially expressed by BTICs and required for BTIC self-renewal and tumor growth. MYC, a key BTIC regulator, bound the RBPJ promoter and treatment with a bromodomain and extraterminal domain (BET) family bromodomain inhibitor decreased MYC and RBPJ expression. Proteomic studies demonstrated that RBPJ binds CDK9, a component of positive transcription elongation factor b (P-TEFb), to target gene promoters, enhancing transcriptional elongation. Collectively, RBPJ links MYC and transcriptional control through CDK9, providing potential nodes of fragility for therapeutic intervention, potentially distinct from NOTCH.
The Journal of Clinical Investigation