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Results: 51

Establishment of an International Diffuse Intrinsic Pontine Glioma Registry (DIPG ...

Brainstem gliomas account for up to 20% of all CNS tumors in children with a median age at presentation of 6-7 years.1  Diffuse intrinsic pontine gliomas (DIPG) comprise 80% of all brainstem gliomas. In North America and Europe, approximately 300 children develop brainstem gliomas per year. 2 Prognosis for patients with DIPGs remains ...

Validation of TAK228 as an effective drug for treating children ...

Validation of TAK228 as an Effective Drug for Treating Children Diagnosed with DIPG Diffuse intrinsic pontine glioma (DIPG) is an untreatable childhood cancer.  DIPGs develop in pons making them surgically unresectable.  We and others have identified mutations in histone and their obligate partner mutations that seem to drive ...

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 ...