Diffuse Intrinsic Pontine Glioma (DIPG). Also known as Diffuse Midline Glioma (DMG) is responsible for more Australian paediatric cancer deaths each year than any other cancer type. There are no treatments offered to patients (other than palliative radiation therapy) so families are told their child is expected to survive 9-11 months post diagnosis. ONC201 is an experimental drug that Prof Dun and his team have been studying since 2018. Initial investigations in the lab suggested there was some benefit in DMG, so they put their 'pharmaco-proteogenomics' skills to work to understand exactly how the drug was working and how they could further enhance its beneficial effects.
The treatment ONC201 appears to improve outcomes, extending median survival from 11 months to 20 months, although the benefit is not experienced equally among all DMG patients. With the help of Fight For A Cure funding Prof Dun and his team have discovered the mechanisms controlling resistance to ONC201, and a strategy to combat this through a combination with a second agent, ‘paxalisib’. The findings so far are that the combination is beneficial and safe, prompting them to progress the strategy to an international clinical trial. More than 100 children are receiving the therapy at last count.
As well as uncovering the drug combination providing benefit to kids with DIPG, Matt's efforts has made sure the therapies can be accessed in Australia. And Prof Dun has been able to achieve this in less than 4 years when the standard is 13 years. It really is a substantial advancement for kids and families facing DIPG. And we are proud at Fight For a Cure to have been part of this.