Precision Medicine Approaches for Children with DIPG

2nd Annual CBTTC Investigator Meeting, The Westin New Orleans Canal Place, New Orleans

May 25, 2016
Sabine Mueller, MD, UCSF Benioff Children’s Hospital


Recent advances in gene expression and sequencing technologies allow oncologists for the first time to use these technologies in real time for clinical decision making and to determine an individualized treatment plan based on each child’s tumor characteristics. Our first feasibility trial used a precision based medicine approach in collaboration with the Translation Genomic Research Institute (TGEN) for children with newly diagnosed diffuse intrinsic pontine glioma (DIPG). Based on the molecular characteristics of each child’s tumor a specialized tumor board was used to recommend a treatment plan based on the results of the molecular profiling. All current FDA approved drugs were considered for the treatment plan. This was the first trial in the US to test such an approach for children with DIPG.