Scientists use AI to discover new treatment for deadly childhood cancer


Scientists have used artificial intelligence (AI) to create a drug regimen for children with a type of deadly brain cancer, where survival rates have not improved for 50 years.

Diffuse intrinsic pontine glioma (DIPG) is a rare, rapidly growing type of brain tumor in children.

These types of tumors are difficult to remove surgically because they are diffuse, which means that they do not have well-defined boundaries suitable for operations.

A quarter of children with DIPG have a mutation in a gene known as ACVR1, but there is currently no approved treatment to target this mutation.

In a new study, scientists at the Institute of Cancer Research in London (ICR) and the Royal Marsden NHS Foundation Trust were able to use AI to discover that combining the drug everolimus with another called vandetanib could improve the ability of the vandetanib to pass into the blood. -brain barrier to treat cancer.

The combination has been shown to be effective in mice and was initially tested on four children.

Experts now hope to test the drug combination on a larger group of children in clinical trials.

The study showed that the combination of the two drugs increased the amount of vandetanib in the brains of mice with DIPG by 56%.

The treatment also prolonged the survival of the mice by 14% compared to those receiving standard control treatment.

The team behind the research said the study shows how AI could open up new avenues for cancer treatment by spotting new ways to combine existing drugs.

The two study drugs are already approved to treat other types of cancer.

Chris Jones, professor of pediatric brain tumor biology at ICR, said: “DIPG is a rare and aggressive childhood brain cancer, and survival rates have not changed over the past 50 years we have therefore desperately need to find new treatments for this disease.

“Our study demonstrates how AI can contribute to drug discovery for cancers like DIPG, by providing new combinations of treatments that may 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 cross the blood brain barrier first.

“The treatment extended survival when we tested it in a mouse model, and we have already started testing it in a small number of children.

“We still need a large-scale clinical trial to assess whether the treatment can benefit children, but we have moved to this stage much faster than it would ever have been possible without the help of the IA. “

The initial plan for the study came from BenevolentAI, a company that built an AI drug discovery platform.

ICR researchers worked with those at BenevolentAI to use the platform to identify drugs that could be used to target ACVR1 mutations in DIPG.

The platform hosts a knowledge graph that contains all publicly available biomedical data, as well as other information that BenevolentAI’s machine learning system has ingested and “read” from scientific papers.

Scientists were able to explore the information in the graph and uncover information they could not have found using only human thought.

Dr Fernando Carceller, consultant in pediatric and adolescent neuro-oncology at the Royal Marsden NHS Foundation Trust and leader of the pediatric and adolescent neuro-oncology and drug development team at ICR, said: “This encouraging research highlights highlight the possibilities of harnessing intelligence to find better treatments for childhood cancer.

“Close collaboration between scientists at ICR and clinicians at Royal Marsden has made this bedside laboratory approach possible.

“The clinical results are too preliminary to draw definitive conclusions, but we are working to advance this combination in a clinical trial for children with ACVR1 mutant DIPG.

Prof Peter Richardson, Vice President of Pharmacology at BenevolentAI, said: “AI-enhanced approaches are already proving their value in expanding the ability of researchers to find new, innovative therapeutic approaches – whether by discovering new ones. therapies or reusing existing ones – not only in DIPG, but also other diseases in the future.

The research is published in the journal Cancer Discovery, with funding from (among others) Brain Research UK, DIPG Collaborative, Children with Cancer UK and the Royal Marsden Cancer Charity.

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