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Results: 51

COllaborative Network for NEurooncology Clinical Trials (CONNECT)

CONNECT’s goal is to establish an international clinical trials collaboration for the conduct of scientifically rational, pilot studies to assess feasibility and early efficacy of incorporating promising novel agents to established frontline therapeutic regimens in children with newly-diagnosed, high-risk brain tumors. This collaboration builds on the existing infrastructure ...

EPIGENETIC ALTERATIONS AS THERAPEUTIC TARGETS IN PEDIATRIC BRAIN TUMORS

We and others co-discovered the presence of activating mutations in ACVR1 in 25% of human DIPGs in 2014. Subsequently, my laboratory has developed a murine DIPG model incorporating R206H ACVR1 and observed that R206H ACVR1 significantly accelerates brainstem gliomagenesis. In addition, short-term treatment with a bone morphogenetic protein pathway inhibitor (...

Developing Novel Combination theraputic approaches for DIPG targeting Polo-like Kinase 1

Whole genome sequencing on DIPG biopsy and autopsy samples have identified specific mutations in Histone H3, ACVRI, TP53 and ATRX causing heterochromatin silencing, genetic instability and alterations in DNA damage response pathways. Using a unique panel of patient derived DIPG cultures, we have further examined the molecular profile of DIPG ...

POLYAMINE PATHWAY METABOLISM AS A NOVEL THERAPEUTIC OPTION FOR DIFFUSE ...

Diffuse intrinsic pontine glioma (DIPG) is the most aggressive of all childhood cancers. Standard treatment with radiotherapy is only palliative and chemotherapy has been found ineffective. Polyamines are organic compounds essential for key functions of living cells. They can be made by human cells but also generated from intestinal microorganisms ...

Toward a multimodality cure for DIPG: investigation of intratumoral drug ...

Diffuse intrinsic pontine glioma (DIPG) is a currently incurable childhood brain tumor that, despite many past clinical trials, has never been shown to respond to systemic chemotherapy. Radiation therapy (RT) is effective in extending life but is not curative; median overall survival is 11 months and long-term survival is extremely rare.1 ...