Grants - Designated Grants - California - 2014

Sabine Mueller, M.D., Ph.D.

Vintner Grant in Memory of Shunsuke Yamamoto

Children with diffuse intrinsic pontine glioma– a specific brain tumor type- continue to have a dismal prognosis and most children die from this disease within months from diagnosis.  Despite multiple national clinical trials, no change in outcome has been achieved over the last several decades. Two potential reasons why we have not made any progress in this disease are a) treatment is not matched to each child’s individual tumor characteristics and b) due to the presence of a tight blood-brain barrier medications given either by mouth or vein are not getting in sufficient enough concentrations to the tumor. To address these issues we are currently conducting a clinical trial through the Pacific Neuro-Oncology Consortium (www.pnoc.us, PNOC003). In this trial we will profile each child’s tumor with state of the art next generation sequencing and determine a treatment plan based on the specific characteristics of the tumor. A specialized tumor board that consists of several neuro-oncologist, pharmacologists and researches with an expertise in next generation sequencing meet and discuss the results and determine a specialized treatment plan, which consists of up to four FDA approved drugs. Specific attention is being paid to the drug brain penetration of recommended drugs. Correlative aims of this feasibility study is to develop patient derived mouse models as well as to test if tumor specific DNA can be detected in blood and be used as a marker for clinical response.

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