Potential first ever targeted therapy designed for ‘untreatable’ childhood brain cancer

Scientists at The Institute of Cancer Research, London have discovered a new type of drug that targets a genetic weakness in ‘diffuse intrinsic pontine glioma’ (DIPG), an untreatable childhood brain cancer, which could become the first ever treatment designed to target the disease.

The international team of researchers have found that the new drug class can kill brain cancer cells with mutations in the ACVR1 gene and shrink tumours in mice.

The new type of drug targets the protein molecule produced by mutated versions of the ACVR1 gene found in the deadly cancer, which is brilliant news for patients as there are currently no life-extending treatments other than radiotherapy for DIPG tumours and this is never curative, with children expecting to live only nine to 12 months after diagnosis.

An Institute of Cancer Research-led team tested 11 prototype drugs with anti-ACVR1 activity – including some from the Structural Genomics Consortium, as well as other drugs previously investigated for stone man syndrome – on brain cancer cells grown in the lab, and found that two of the prototypes from the new series were particularly good at blocking signals sent out by ACVR1 and killing ACVR1-mutant cells, while having very little effect on healthy brain cells.

Results show that the prototype treatment could also offer hope for patients with the rare and devastating ‘stone man syndrome’, in which muscles and ligaments turn to bone.

Professor Chris Jones, Professor of Paediatric Brain Tumour Biology at The Institute of Cancer Research, London, said: "It’s simply not good enough that we can cure some cancers, but in others we have seen no progress in decades. We owe it to children and their families to do better.

"DIPG is a relatively rare childhood brain cancer, but it is always deadly. Learning more about the biology of DIPG, and trying to find ways to translate that knowledge into new treatments, has been a passion of mine for years. My lab discovered that mutations in the ACVR1 gene occur in a quarter of DIPG cancers and it’s incredibly exciting to see this now lead to potential new drugs for the disease. I can’t wait to see how they perform in patients.

Open science company M4K (Medicines for Kids) Pharma has taken on development of the ACVR1 inhibitor drugs, with clinical trials in children with brain cancer expected to begin in 2021.

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