This is the first time that a group of international basic and translational scientists and physicians 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.
Background- Little was known about the biology of these tumors until recently. The availability of autopsy and some biopsy materials from children with DIPGs has led to a new understanding of the biology of these tumors. Important biological pathways in DIPGs are being identified. These pathways may be readily targetable with currently available molecularly-targeted agents. In addition, human DIPG tumors have been grown from human postmortem DIPG tumors from autopsy materials in petri dish (cell lines and neurospheres). Finally, mouse DIPG models have been developed– a key resource to functionally testing potential therapies.
Aim- The researchers hypothesize that they can identify a promising combination of molecularly-targeted agents using a functional drug screening approach by first testing the potentially effective molecularly-targeted drugs in the laboratory from DIPG cell lines (16 available cell lines),
They will test the two or three most effective drugs in the mouse models in combination. 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.
Website- (regular progress updates)
Funding- The DIPG Symposium Collaboration through the Cure Starts Now including Reflections of Grace Foundation, The Jeffrey Thomas Hayden Foundation, Cancerfree Kids, Carly’s Crusaders, The Max Lacewell Foundation, Smiles for Sophie Forever Foundation and Benny’s World Foundation
The Lyla Nsouli Foundation for Children’s Brain Cancer Research
Curesearch for Children’s Cancer
ABC2- Accelerate Brain Cancer Cure