Priming the blood-brain barrier to improve drug delivery and treatment outcomes in diffuse midline glioma

There is currently a lack of effective treatments for children with the brain cancer diffuse midline glioma (DMG). This is because the blood-brain barrier, which protects the brain, prevents drugs from being delivered to the tumour. We have developed a new way to overcome this barrier by targeting the blood vessels surrounding the tumour. This will enable the safe and effective delivery of potent cancer drugs that we know can target and kill DMG tumour cells.

The proposed study will generate essential preclinical data demonstrating the efficacy of a target molecule in increasing CNS drug delivery and enable effective treatment of DMG while limiting side-effects. We anticipate that the outcomes of the proposed studies will form the basis for a clinical trial to assess a drug combination therapy in the effective treatment of DMG tumours. This has the capacity to revolutionise DMG treatment, thereby improving outcomes for affected children who would otherwise do very poorly.