Diffuse intrinsic pontine glioma (DIPG) is a rare and fast-growing type of tumour that forms where the spine is connected to the brain.
Children with DIPG are typically expected to live only nine to 12 months after their diagnosis, and survival rates have not improved for 50 years.
DIPG tumours are difficult to remove surgically because they do not have well-defined borders suitable for operating on.
A quarter of children with DIPG have a mutation in a gene known as ACVR1, but there are no treatments approved to target this mutation.
In a new study, scientists at the Institute of Cancer Research, London (ICR) and the Royal Marsden NHS Foundation Trust were able to use AI to find that it could be beneficial to combine the immunosuppressant drug everolimus with an anti-cancer medication called vandetanib.
The combination has proved effective in mice and has been initially tested on four children.
The study showed that combining the two drugs increased the amount of vandetanib in the brains of mice with DIPG by 56 per cent.
The treatment was also able to extend survival in mice by 14 per cent compared with those receiving a standard control treatment.
The scientists say that everolimus could enhance vandetanib’s capacity to pass through the blood-brain barrier in order to treat the cancer.
Experts now hope to test the drug combination on a wider group of children in clinical trials.
Chris Jones, professor of paediatric brain tumour biology at the ICR, said: “DIPG is a rare and aggressive childhood brain cancer, and survival rates have not changed over the past 50 years so we desperately need to find new treatments for this disease.
“Our study demonstrates just how much AI can bring to drug discovery for cancers like DIPG, in proposing new treatment combinations that would not have been obvious to people.
“The AI system suggested using a combination of two existing drugs to treat some children with DIPG – one to target the ACVR1 mutation, and the other to sneak the first past the blood-brain barrier.
“The treatment extended survival when we tested it in a mouse model, and we have already started testing it out in a small number of children.
“We still need a full-scale clinical trial to assess whether the treatment can benefit children, but we've moved to this stage much more quickly than would ever have been possible without the help of AI.”
The initial plan for the study came from BenevolentAI – a company that has built an AI drug discovery platform.
Dr Fernando Carceller, consultant in paediatric and adolescent neuro-oncology at the Royal Marsden NHS Foundation Trust, and leader of the paediatric and adolescent neuro-oncology and drug development team at the ICR, said: “The clinical results are too preliminary as to draw firm conclusions, but we are working to take this combination forward in a clinical trial for children with ACVR1-mutant DIPG.”
The research is published in the journal Cancer Discovery, with funding from groups including Brain Research UK, the DIPG Collaborative, Children with Cancer UK and the Royal Marsden Cancer Charity.
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