Six-year-old’s death leads to hope of new drug for childhood cancer – The Guardian

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Six-year-old’s death leads to hope of new drug for childhood cancer – The Guardian


Parents of Abbie Mifsud created the charity that has funded vital research on brain stem tumours

Professor Chris Jones, who is heading research into DIPD.






Professor Chris Jones, who is heading research into DIPD.
Photograph: Institute of Cancer Research

Researchers say they are close to testing a drug that could tackle a previously untreatable childhood brain cancer. The condition is known as DIPG – diffuse intrinsic pontine glioma – and every year it affects between 30 to 40 boys and girls, aged between five and 10. All develop tumours caused by the condition and die, often within months of their diagnoses.

But now scientists believe they may soon be able to tackle the condition – and one of the key organisations involved is a remarkable charity set up by Amanda and Ray Mifsud, whose daughter Abbie died of DIPG in 2011. Their charity, Abbie’s Army, has raised money that has provided vital backing for research by Professor Chris Jones, of the Institute of Cancer Research, London.

His work has revealed the role of a mutant gene that is involved in driving the progression of DIPG tumours and this work has, in turn, led to the development of a number of drugs that might soon be tested to see if they inhibit their progress.

“We have reached a very promising stage in our preclinical work and that could see the first full clinical trials of a drug for DIPG in a couple of years,” Jones told the Observer. “When that happens, we will have produced the first new treatment developed specifically for a childhood cancer. Crucially, the help of Abbie’s Army and other children’s cancer charities has been a major boost in that effort.”

Early symptoms of DIPG include problems with vision, hearing and balance. These worsen as the underlying tumour grows and spreads until a patient becomes bedridden. The tumour’s location, deep in the brain stem, makes surgery impossible. In addition, the relative rarity of DIPG cases has meant it has not been a focus of interest for major pharmaceutical companies.

The only standard treatment currently in use is palliative radiotherapy – which was given to Abbie. “Over the course of two months she endured and battled through her treatment,” recalled her mother. Abbie died in the morning on 13 September 2011. She was six years old.

“Abbie was our whole world,” said Amanda. “And no parent ever wants to hear that there is no cure and no hope and they should never have to.”

As a result of their experience the Mifsuds set up their charity, and this has since raised funds for research on DIPG that have played an important role in helping Jones direct his work on the disease. Two years ago, he discovered that tumours caused by the disease carried mutations of the gene ACVR1, which had never been found in any other kind of cancer before then. The mutation means the gene is susceptible to being switched on and to direct the manufacture of proteins when it is supposed to be switched off.

This knowledge suggests ACVR1 is involved in a cascade of biochemical events that eventually produce DIPG tumours and has provided Jones and his colleagues with a specific target for tackling the condition. It is a first for scientists working on DIPG. Working with the pharmaceutical group M4K Pharma, based in Toronto, they have developed a novel class of drugs which could act as treatments for the condition.

“We have identified five lead candidate drugs for further preclinical studies and expect to begin clinical trials within two years,” said Owen Roberts, chief executive of M4K Pharma.

Just how quickly these trials will lead to the launching of the first effective, life-prolonging medicine for treating children afflicted by DIPG remains to be seen.

“You have to go through careful protocols when carrying out drug clinical trials and these cannot be rushed,” said Jones. “On the other hand, this disease is terribly rapid in its clinical course. The average survival time is nine months. That means that when we get a drug that is in any way effective we will see very quickly that it is making improvements to a child’s life.”

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