Featured Publications

Expression profiles of 151 pediatric low-grade gliomas reveal molecular differences associated with location and histological subtype

Expression profiles of 151 pediatric low-grade gliomas reveal molecular differences associated with location and histological subtype

In our April featured publication, investigators from our Program and their collaborators analyzed the gene expression profiles of 151 pediatric low-grade glioma samples.  They discovered significant differences associated with the location of the tumor, the appearance of the tumor and BRAF mutation status.  In particular, supratentorial juvenile pilocytic astrocytomas were enriched with genes involved in […]

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BRAF Mutation and CDKN2A Deletion Define a Clinically Distinct Subgroup of Childhood Secondary High-Grade Glioma

BRAF Mutation and CDKN2A Deletion Define a Clinically Distinct Subgroup of Childhood Secondary High-Grade Glioma

In this month’s featured publication, Dr. Ligon and colleagues investigate the genetic changes that lead to the transformation of pediatric low-grade glioma to secondary high-grade glioma. Using advanced sequencing techniques, they analyzed 886 patient samples and showed there were recurrent alterations in BRAF (to BRAFV600E) and deletion of CDKN2A, amongst other changes in tumors that […]

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Development of covalent inhibitors that can overcome resistance to first-generation FGFR kinase inhibitors

Development of covalent inhibitors that can overcome resistance to first-generation FGFR kinase inhibitors

In this month’s featured publication, researchers in Dr. Gray’s group expand upon previous studies of the FGFR receptor, a protein found mutated in a subset of pilocytic astrocytomas.  However, a common problem in cancer is development of tumor resistance to chemotherapy over time.  Using structure-based drug design, they developed two next-generation covalent inhibitors that can […]

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Time to Rethink the Unthinkable: Upfront Biopsy of Children With Newly Diasnosed Diffuse Intrinsic Pontine Glioma (DIPG)

Time to Rethink the Unthinkable: Upfront Biopsy of Children With Newly Diasnosed Diffuse Intrinsic Pontine Glioma (DIPG)

In November’s featured publication, Dr. Kieran discusses diffuses intrinsic pontine glioma (DIPG) biopsy.  During the past 50 years, biopsy for these tumors have been discouraged due to excessive toxicity of the procedure.  However, Dana-Farber Cancer Institute and Boston Children’s Hospital have recently opened a multi-site national clinical trial of DIPG biopsy and chemotherapy to change the […]

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Successful Treatment of a Progressive BRAF V600E-Mutated Anaplastic Pleomorphic Xanthoastrocytoma with Vemurafenib Monotherapy

Successful Treatment of a Progressive BRAF V600E-Mutated Anaplastic Pleomorphic Xanthoastrocytoma with Vemurafenib Monotherapy

In October’s Featured Publication, Dr. Sandro Santagata treated a patient with BRAF V600E mutated pleomorphic xanthroastrocytoma (PXA), a rare brain tumor in children and young adults. His team used Vemurafenib monotherapy  for the first time in the treatment of V600E-mutated glioma, and saw an encouraging response by the tumor to this approach, leading to the […]

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Final results of a prospective multi-institutional phase II study of everolimus (rad001), an mtor inhibitor, in pediatric patients with recurrent or progressive low-grade glioma. A poetic consortium trial.

Final results of a prospective multi-institutional phase II study of everolimus (rad001), an mtor inhibitor, in pediatric patients with recurrent or progressive low-grade glioma. A poetic consortium trial.

In this article in Neuro Oncology, Dr Kieran and others from Dana-Farber Cancer Institute and Harvard Medical School report the results of the RAD001 clinical trial in pediatric patients.  They show that treatment with RAD001 was well tolerated in patients with recurrent or progressive low-grade gliomas, and that incorporation of this drug into front line […]

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Recurrence after gross-total resection of low-grade pediatric brain tumors: the frequency and timing of postoperative imaging.

Recurrence after gross-total resection of low-grade pediatric brain tumors: the frequency and timing of postoperative imaging.

In this month’s article in Journal of Neurosurgery: Pediatrics, Mark Kieran and colleagues studied the frequency of brain imaging (through MRI) a patient receives to evaluate how often tumors recurred.  They found that at either 2 or 5 years of recurrence-free survival there were no clinical symptoms or mortality observed in the patient population studied, […]

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Targeted treatment for sonic hedgehog-dependent medulloblastoma

Targeted treatment for sonic hedgehog-dependent medulloblastoma

This month’s featured publication by Dr. Kieran discusses the medulloblastoma subgroup with activations of the sonic hedgehog pathway, the preclinical data currently known and the development of novel therapies to overcome chemoresistance.

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Targeting BRAF in Pediatric Brain Tumors

Targeting BRAF in Pediatric Brain Tumors

In this e-book chapter, Dr Kieran discusses the signalling molecule BRAF, and how mutations in BRAF can cause pediatric brain cancer.   Also explained is how our scientists are beginning to understand the biology of BRAF signalling, and how these discoveries can be exploited for new therapy targets.

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Pediatric low-grade gliomas: How modern biology reshapes the clinical field

Pediatric low-grade gliomas: How modern biology reshapes the clinical field

In this review article, Drs. Stiles, Segal, Beroukhim, Ligon and Kieran discuss low grade glioma clinical features, current therapies and new genomic discoveries, and how these most recent findings can be applied to diagnose and treat low grade tumors in the future.

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