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A research project at Children’s Cancer Institute that could lead to the first-ever effective drug to treat the deadly brain cancer

Photo from Children's Cancer Institute/Linkedin

Jul 21, 2024, 16:25

A research project at Children’s Cancer Institute that could lead to the first-ever effective drug to treat the deadly brain cancer

Children’s Cancer Institute shared a post on LinkedIn:

“A bold research project at Children’s Cancer Institute that could lead to the first-ever effective drug to treat the deadly brain cancer, DIPG (diffuse intrinsic pontine glioma) received a major boost this month, with a $US400,000 grant awarded to Dr Jean Borges Bertoldo by the US-based charity, ChadTough Defeat DIPG Foundation.

One of eight new DIPG grants awarded by the Foundation, and the only one to be awarded to an Australian researcher, the grant will fund Dr Bertoldo over the next three years to develop and test an exciting new drug candidate for DIPG which uses a completely new approach to any other conventional cancer drug so far developed.

In the past, certain genetic mutations in DIPG tumours have been found to drive the aggressive growth of this cancer. These have been hailed as potential ‘therapeutic targets’ — genes that can be targeted by anticancer drugs. However, so far these have proven ‘undruggable’, meaning that no one has been able to develop a drug that successfully binds to the targets and negates their effects.

Now, Dr Bertoldo hopes to change that. Using the cutting-edge tools of chemoproteomics and artificial intelligence, he has developed a small molecule agent called JNSY1 that specifically targets a genetic mutation regarded as the main driver of DIPG, the histone H3K27M mutation.

‘I’ve already shown that this agent can bind to this mutation, restore normal gene function, and induce selective toxicity in DIPG cells,’ he explains. ‘Now, thanks to this grant, I intend to develop this agent into a novel drug candidate that can be taken into clinical trials in children with DIPG.’

Dr Bertoldo believes that if he is successful, the drug he develops will be unique, known as ‘first in class’, and could prove to be a breakthrough in the treatment of cancer.

‘We aim to be the first in the world to develop a drug that specifically targets the main cause of DIPG.’

Excitingly, the method Dr Bertoldo is applying in the lab can be applied to other genetic targets in other paediatric cancers, making the implications of this research very broad.”

Source: Children’s Cancer Institute/Linkedin