Evaluation of BMP Inhibitors in a Mouse Model of DIPG

Determine whether an ACVR1 inhibitor, VU002 penetrates DIPG tumors and demonstrates target engagement/pathway inhibition. The process of tumor formation and tumor response to therapy is extremely complex and cannot be modeled in vitro. Thus, we propose to use a mammalian model system in order to increase the relevance of our observations to human disease, and mice are the lowest species on the phylogenetic scale suitable for our study. Investigate the ability of ACVR1 inhibition increase survival of tumor-bearing mice and to sensitize DIPG to Radiation Therapy (RT).