Research And Grants

Phase I/II Study of Oral DFMO and AMXT1501 for DIPG/DMG

Brain cancers are the deadliest of all childhood cancer and the leading cause of disease associated death in children. Diffuse intrinsic pontine glioma (DIPG), and diffuse midline glioma (DMG) remain the most aggressive childhood cancers and the only available treatments for this group of brain cancers are palliative.

Our group has made the ground-breaking discovery that the polyamine pathway is a critical driver of DIPG/DMG and represents a novel therapeutic target. We have shown that dual targeting of the polyamine pathway is the most effective drug treatment strategy published to date in orthotopic animal models of childhood brainstem gliomas.

This project translates this preclinical work into a clinical trial to make this promising treatment available to children with DIPG. The combination of AMXT 1501 and DFMO potently inhibits the polyamine pathway and represents an exciting new treatment strategy for DIPG/DMG. Since DFMO is clinically available and the combination of AMXT 1501 and DFMO is currently being tested in adult patients in an open clinical trial, this combination can be rapidly translated to children with these aggressive, incurable brain tumours. Furthermore, this clinical trial will open through the international collaborative network, CONNECT, which will allow it to be open in at least 18 children’s hospitals around the world with access for children internationally.