Exploiting Mutant IDH1/2-induced DNA Repair Defects in Pediatric Glioma to improve outcomes

Pediatric Brain Cancer
Sabine Mueller, MD, PhD
University of California, San Francisco


Pediatric gliomas are the most commonly encountered tumor types in children and also contribute to the most cancer related deaths in children. There is an urgent need for high-grade gliomas which lack any meaningful standard of care. IDH1/2 gene mutations are common in low grade gliomas (>70%). Pre-clinical data has suggested that this mutation has vulnerability that can be targeted by drugs known as PARP inhibitors. Dr. Mueller and team hope to test the safety and tolerability of BGB-290, a PARP inhibitor, in combination with temozolomide – a chemotherapy utilized in neurological cancer – in children, adolescents, and young adults with newly diagnosed and recurrent gliomas that express IDH1/2-mutations.

Trial Registration: ClinicalTrials.gov Identifier: NTC03749187