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‘Untreatable’ childhood brain cancer gets its first drug therapy

A new type of drug that targets a genetic weakness in an untreatable childhood brain cancer could become the first ever treatment designed to target the disease.

The new type of drug targets the protein molecule produced by mutated versions of the ACVR1 gene found in the deadly childhood brain cancer ‘diffuse intrinsic pontine glioma’ (DIPG).

In the new study, the ICR-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.

They 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.

The researchers transplanted human DIPG tumours into mice and found the potential new drugs stopped ACVR1 activity, shrunk tumours and extended survival by 25 per cent (from 67 to 82 days).

The drugs have now been taken on as the first project of a new open science company called M4K Pharma, which aims to discover and develop affordable drugs for childhood diseases that are rare.

Read full, original post: New treatment could become first ever targeted therapy designed for ‘untreatable’ childhood brain cancer

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