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Results: 51

Advanced RNAi therapeutics for DIPG

Diffuse intrinsic pontine glioma (DIPG) is a form of brain tumor that arises in the brainstem of children and is nearly 100% fatal with a median survival of 9-12 months after diagnostic. The location of these highly infiltrating tumors in the ventral pons precludes surgical resection and clinical trials with different ...

Short-pulse ultrasound delivery of panobinostat for the treatment of diffuse ...

Children with diffuse intrinsic pontine gliomas (DIPGs) have a 2-year survival rate of less than 10%. Compared to other brain cancers, DIPG is particularly difficult to treat because it spreads behind an intact blood-brain barrier (BBB) affecting healthy developing paediatric brain tissue. There are currently no effective treatments for this disease. ...

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 ...

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 ...

Determine the efficacy of BMP and MEK inhibitors with RT ...

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 (...