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A breakthrough in finding the first drug with the potential to treat the deadliest type of childhood brain tumour was revealed today.
Scientists at the Institute of Cancer Research, in Chelsea, hope a new type of drug could offer hope for some children diagnosed with the aggressive tumours, which typically cause death in nine months.
Their studies have been part-funded by the Abbie’s Army charity, which was founded by Amanda and Ray Mifsud in memory of their daughter Abbie, who died from a DIPG (diffuse intrinsic pontine glioma) tumour aged six in 2011. They have raised more than £450,000 for research.
There have been no new drugs licensed to treat brain cancer in adults or children for 20 years.
DIPG is an inoperable cancer in the brainstem which has no cure. Surgery is impossible and radiotherapy is of limited effect. Abbie, from Ashford in Kent, died five months after diagnosis.
The hope is that trials of the new drug, which has been shown in tests to shrink tumours and extend survival by 25 per cent, could start within two years.
Mrs Mifsud said: “We’re so pleased to see some of the pre-clinical research work that Abbie’s Army has supported heading towards a biologically relevant treatment.
“Our hope very much is that further translation and combination studies will provide a successful therapy for this sub-set of DIPG patients in the near future.”
The drug targets a weakness in the ACVR1 gene to kill cancerous cells. The science company M4K (Medicines for Kids) Pharma will build on the work of the ICR and its international partners and expects to start trials in 2021.
It could also work for “stone man syndrome”, a rare genetic disease where muscles and ligaments turn to bone. About 40 UK children a year are diagnosed with DIPG. The research is published in Communications Biology.
Professor Chris Jones, of the ICR, 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.”
