The main focus of the group is to find effective combination therapies for children diagnosed with DIPG and DMG. It is recognized worldwide that over 1000 children die every year following being diagnosed with a specific brain cancer called Diffuse Intrinsic Pontine Glioma (DIPG)/Diffuse Midline Glioma (DMG). These tumors arise in the midline structures of the brain such as the thalamus, pons as well as spinal cord.
Over the last 50 years, clinical trials have not resulted in improved outcome for children diagnosed with DMGs. Radiotherapy -the only standard of care- often results in a transient tumor response but does not significantly impact the long-term outcome. The mean overall survival for diagnosed children with DMGs is about 9-12 months. However, in recent years, due to analyses of postmortem DIPG tumor specimens as well more and more upfront tissue sampling, our understanding of the biology of these tumors has significantly improved and it is now clear that DMGs including DIPGs are a heterogeneous group of tumors requiring a personalized therapy approach. Given the heterogeneity on a molecular level of these tumors, a “One size fits all” approach will not improve the outcome for these children.
The DMG-Act group consists of an international and multidisciplinary team of experts across multiple institutions, with the combined aims of:
i) Elucidating drug mechanisms of action.
ii) Creating robust in vitro and in vivo data on drug efficacy as single and combination therapy.
iii) Creating rapid and efficient pipelines for mapping drug targets and identify clinically relevant predictive biomarkers.