First-ever drug to combat ‘untreatable’ brain cancer in kids could offer a ‘cure’

A NEW drug could become the first-ever designed to combat "untreatable" childhood brain cancer, experts hope.

No new medications have been licensed to treat the disease in kids or adults in the last 20 years.

Getty – Contributor1 Scientists have discovered a new drug designed to tackle one of the most dangerous childhood brain cancers

But, a team of sicentists at The Institute of Cancer Research in London believe their new findings could offer hope to patients with a very aggressive tumour.

Kids found to have diffuse intrinsic pontine glioma (DIPG) tumours are typically given nine to 12 months to live after diagnosis.

Scientists found the new drug targets a genetic weakness of the DIPG tumour.

It targets the protein molecule produced by versions of the ACVR1 gene, found in the deadly childhood cancer.

Those mutated genes are only found in DIPG tumours, and people with the rare and devastating condition, stone man syndrome – where a person's muscles and ligaments turn to bone.

The new therapy can kill brain cancer cells and shrink tumours in mice, tests have shown.

Company M4K (Medicines for Kids) Pharma plans to start conducting trials on kids with brain cancer in 2021.

DIPG tumours are notoriously brutal

Kids with DIPG tumours are only expected to live up to a year after diagnosis and there are no cures or life-extending treatments available for it, beyond radiotherapy.

Chemo doesn't work and because of the sensitive area they develop in, surgery isn't an option.

This new study has funded by Abbie's Army, Children with Cancer UK, Cancer Research UK and the DIPG collaborative.

What is Diffuse Intrinsic Pontine Glioma (DIPG)?

Diffuse Intrinsic Pontine Glioma also known as 'DIPG' is the second most common type of primary, high-grade brain tumour in children.

DIPG tends to grow quickly and is more likely to spread to other parts of the brain or spinal cord.

They originate in an area of the brain, and more specifically the brainstem, called the pons.

The pons is an area deep within the lower part of the brain which is responsible for a number of critical bodily functions, such as breathing, sleeping and blood pressure.

The cause of brain tumours, including DIPG, is not yet known.

The signs of a DIPG vary as the pons and surrounding structures (where DIPGs are located) are responsible for a variety of different body functions.

A child with a DIPG may display:

  • Abnormal alignment of the eyes or/and double vision (diplopia)
  • Weakness of facial muscles or facial asymmetry (one side of the face appearing different from the other)
  • Arm and leg weakness
  • Unstable balance and coordination
  • Difficulties walking and speaking

The standard of treatment for DIPGs is radiotherapy, which is usually administered over 3 to 6 weeks depending on the type of radiotherapy that is deemed best for your child (with a daily dose given Monday to Friday).

Your child might also be given steroids during this period to help reduce some of the pressure caused by the tumour and radiation treatment.

Source: The Brain Tumour Charity

Amanda and Ray Mifsud lost their six-year-old daughter Abbie to DIPG brain cancer in 2011.

Together they set up Abbie's Army in November 2012 so that one day parents of children diagnosed with DIPG will not be told that there is no cure and no hope, as they were.

Amanda said: "We're so pleased to see some of the pre-clinical research work that 'Abbie's Army' has supported in the Jones lab with ACVR1 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 drugs extended survival by 25 per cent

Scientists tested 11 prototype drugs with anti-ACVR1 activity on brain cancer cells grown in a lab.

They found that two of the prototypes were particularly effective at blocking signals sent out by ACVR1 ad killing ACVR1-mutant cells – while having very little effect on healthy brain cells.

Researchers then transplanted human DIPG tumour cells into pice and found that the new drugs shrunk the 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, where there is less market incentive and that aren't well served by current business models.

'We owe it to kids to do better'

Professor Chris Jones, professor of paediatric brain tumour biology at The Institute of Cancer Research, London, said that it's "simply not good enough that we can cure some cancers, but in others, we've seen no progress in decades".

"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."

Professor Paul Workman, chief executive of The Institute of Cancer Research, London, said that this latest finding was "an important study".

"I'm proud that the ICR is working in an academic-industry partnership with the biotech company M4K Pharma to take a drugs forward to the clinic.

"Where the patient population is very small, it can be difficult to get companies to take such drugs on.

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"And even where this does happen the result is often a very expensive drug that the NHS struggles to afford.

"One important solution, as shown here, is for academic researchers to take a leading role in working closely with companies to create genuinely innovative treatments at an acceptable price.

"I hope that a new drug from this research can be taken to the clinic as quickly as possible to help children with DIPG tumours, and of course it would also be excellent to see patients with stone man syndrome benefit from the treatment as well."

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