Grantee Name: Dr. Javad Nazarian
Organization: University Children's Hospital Zurich
Project Name: Correlative studies of an open label Phase 1 study in diffuse intrinsic pontine glioma (DIPG)
Funding Year: 2020
Project period: 4 years
Correlative studies of an open-label Phase 1 study in diffuse intrinsic pontine glioma (DIPG)
Based on previous studies and preliminary data, a phase 1 clinical trial of ONC206 is scheduled to start at the University Children’s Hospital Zurich. This trial is designed as a multi-arm trial based on different disease stages to enable the largest possible population to access ONC206. The proposal seeks funding for the correlative study of this clinical trial.
There is an urgent need to develop and evaluate new therapies for children with brain tumours given their high mortality rates. This is particularly true for Diffuse Midline Gliomas (DMGs), which include Diffuse Intrinsic Pontine Gliomas (DIPGs), which affect over 2,000 children and young adults in the US and Europe every year. Sadly, despite decades of research, there are no effective treatments for this disease and over 90% of children die less than 2 years from being diagnosed.
Promising drugs and treatment strategies are emerging, but they require extensive research at the preclinical stage. This is both to understand the mechanisms of action of these drugs, but also to identify clinically relevant predictive biomarkers of drug efficacy. Clinically relevant predictive biomarkers allow us to gauge, in real time, how well they will respond to treatment.
Our project's aim is to assess tumor response to therapy. Such studies will allow for designing more effective therapies for patients diagnosed with DMG. Our study is designed as a multi-arm trial based on different disease stages to provide access for the largest possible population of patients. This trial offers a new and promising agent to children in Europe where little to no innovative therapies are available, and limited clinical phase 1 trials are open. The results of our study will have a global impact on treatment strategies for children suffering from DMGs.