Research And Grants

Developing New Epigenetic Combination Treatments Against DIPG.

Diffuse intrinsic pontine glioma (DIPG) is the most aggressive of all childhood cancers. Standard treatment with radiotherapy is only palliative and single drug chemotherapy is ineffective. We have found that DIPG cells are sensitive to a novel anticancer treatment. CBL0137 is a novel class of anticancer agents that can inhibit the action of a key protein complex called Facilitates Chromatin Transcription (FACT). The FACT complex controls multiple cancer –associated pathways that can lead to aberrant tumor growth. We have found that CBL0137 can inhibit further the growth of DIPG tumors when combined with other anticancer drugs such as panobinostat and JQ1. Furthermore preliminary testing has shown that CBL0137 can also be combined with other chemotherapeutic agents such as olaparib and ACT001. Olaparib is a clinically approved agent for ovarian and breast cancers where as ACT001 is currently in Phase 1 testing in pediatric patients with DIPG. In this project, we aim to develop these new combination therapy strategies in preclinical models of DIPG and obtain the necessary quantum of data to begin the transition of CBL0137 therapy from the bench to the bedside to directly benefit children with this devastating and currently incurable tumor.