DIPG research breakthroughs

“We’ve seen what we think is the most active drug that anyone’s ever tested in the lab for DIPG. The tumour’s stopped growing. We believe this is one of the first really important breakthroughs for DIPG.”

Associate Professor David Ziegler and Dr Maria Tsoli, from the team behind Levi’s Project at Children’s Cancer Institute, have discovered a potentially game-changing treatment for DIPG called polyamine depletion therapy. This treatment is a drug combination comprising DFMO, which targets the polyamine pathway critical to DIPG tumour growth, and AMXT1501, which can block the transport of polyamines into the cancer cell. Dr Ziegler and Dr Tsoli have tested this treatment on live models of DIPG in the labs and world-first 3D models of the tumour, and say it’s “spectacularly effective in eradicating cancer cells”. This research has been published this week in Nature, the leading science journal in the world.
Polyamine depletion therapy will go to clinical trial in 2021, thanks to your generous support.

More information on this ground-breaking researchch can be found here, and you can read the Nature article here https://www.nature.com/articles/s41467-021-20896-z

In another potential DIPG breakthrough, research published this week in the international journal, Cell Reports, offers an exciting new therapeutic approach for the treatment of DIPG by using a new anti-cancer compound, CBL0137, developed from the antimalarial drug quinacrine. The researchers found that CBL0137 directly reverses the effects of the key genetic drivers in DIPG, and has a profound effect against DIPG tumour models, even more so when combined with a second drug, panobinostat, a new type of drug known as a histone deacetylase (HDAC) inhibitor.

A/Prof Ziegler will lead an international clinical trial of CBL0137 for children with DIPG that will open in the top children’s hospitals in the US and Australia. Plans to launch the trial are boosted by the fact that CBL0137 has recently successfully completed testing in phase I clinical trials in adults with solid tumours.

Find out more about this exciting research here.

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