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Results: 269

Robotic screening for novel therapies for diffuse pontine glioma

Diffuse pontine gliomas are the most devastating of all brain tumours.  They mostly affect young children and, as there are no effective treatments, almost all will die of their tumour within 12 months.  In this project we will collect tumours from patients, grow the cells in the laboratory, and ...

Rapid Preclinical Development of a Targeted Therapy Combination for DIPG

    Patients with diffuse intrinsic pontine gliomas (DIPGs) have a uniformly dismal prognosis with a median survival of 9 months and long-term survival of less than 1%.  Radiotherapy provides only temporary improvement of symptoms.  No chemotherapy has ever proven effective. Novel therapies are desperately needed in this vulnerable population. ...

Single-cell profiling and targeting of developmentally regulated signaling programs in ...

Background:  As in other malignant gliomas, there is evidence that DIPG is organized as an aberrant developmental hierarchy originating from rare, treatment-resistant tumor-initiating cells (TICs), also referred to as “cancer stem cells”. Although TICs are potentially important drug targets, they are a challenge to study directly because ...

Establishment and characterization of renewable tissue resources for dipg research

Background:  Due to their vital location, diffuse intrinsic pontine gliomas (DIPGs) are not treated surgically, and the lack of tumor material for research has greatly limited research efforts [1]. Recently, our group and others have used autopsy tissue, and rare biopsy tissues, for genetic analyses of DIPG to identify the ...

Comparative Proteomic and RNA Sequencing of DIPGs

Scientific Merit: Pediatric diffuse intrinsic pontine gliomas (DIPGs) are high-grade cancers of the brainstem that are rarely biopsied due to the morbidity of the procedure.  Recent advances in the understanding of DIPG biology include the discovery of a mutation in the gene coding for histone 3.3 (H3.3K27M).  ...

Evaluating TORC1/2 kinase inhibition in murine and nonhuman primate (NHP) ...

Scientific Merit: New therapies are desperately needed for children with DIPG. Our group participated in recent collaborative efforts that led to identification of panobinostat as an active agent in DIPG. However, additional agents are likely required in combination with panobinostat to improve long-term survival for these children. We identified mutations ...