Collaborating To Bring New Treatments for Rare Pediatric Brain Tumor

This is the first time that a group of basic and translational scientists and physicians from eight institutions throughout North America and Europe have come together as a consortium to focus on DIPGs and to focus on a bench-to-bedside approach to rationally target therapy for children with DIPGs.

Patients with the high grade glioma called diffuse intrinsic pontine glioma (DIPG) have a uniformly dismal prognosis with a median survival of 9 months and long-term survival of less than 1%.  Novel therapies are desperately needed in this vulnerable population.  

Little was known about the biology of these tumors until recently.  The availability of autopsy and some biopsy materials from children with DIPGs has finally led to a new understanding of the biology of these tumors.  The researchers, led by Dr. Charles Keller of the Oregon Health & Science University (OHSU), are identifying potentially important biological pathways in DIPGs that are readily targetable with currently available molecularly-targeted agents.  In addition, consortium researchers have successfully grown human DIPG tumors from autopsy materials in the petri dish and have developed mouse models of DIPGs—a key resource to functionally testing potential therapies.
Since the number of children with this unfortunate disease is limited, and the number of available targeted agents is quite large, researchers hypothesize that they can identify a promising combination of molecularly-targeted agents using a functional drug screening approach.  
The ultimate goal is to move the most effective single agent or combination therapy forward to early phase clinical trials in the next 18-24 months.

The study was originally made possible by a grant from TheCureStartsNow.  An additional supplement from The Lyla Nsouli Foundation for Children’s Brain Cancer Research further allowed the project to expand with two European research collaborators.  ABC2 and CureSearch for Children's Cancer jointly provided additional funding and strategic advice that helps the research consortium perform DNA (exon) sequencing of all DIPG cell lines, their primary tumor and paired normal DNA via the laboratory of Dr. Paul Spellman at OHSU.  From this data, the consortium will be able to prioritize combination drug testing on the most clinically ‘representative’ samples.

Click HERE for updates on the project.

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