The death of the six-year-old has resulted in a new drug for childhood cancer | Science


Researchers say they are close to a drug test that could tackle unrecognized brain cancer. DIPG is called the intrinsic diffuse dialogue gline – and every year it affects between 30 and 40 boys and girls, between five and 10 years of age. They develop all tumors due to condition and death, often within one month of diagnosis.

But now scientists believe they may soon be able to cope with the condition – and one of the main organizations involved is a wonderful 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 provided vital support for research by Professor Chris Jones, from Cancer Research Institute, London.

His work demonstrated the role of a mutant gene who is involved in leading the progression of DIPG tumors and, as a result, some drugs were developed which could be tested early to see if they were blocking their progress.

“We have reached a very sensitive stage in our upstream work and the first full clinical drug trial for DIPG may be seen in a few years time,” Jones added. Observer. “When that happens, we will have developed the first new treatment specifically developed for childhood cancer. The assistance of Abbie's Army and other children's cancer charity is very important in this endeavor. ”

Abbie with parents Ray and Amanda Mifsud.

Abbie with parents Ray and Amanda Mifsud.

Early features of DIPG include problems with vision, listening and balance. These are worse as the underlying tumor grows and expands until a patient becomes repressed. Because of the location of the tumor, deep in the brain, the surgery cannot be done. In addition, the relative disparity of the DIPG cases meant that it was not a focus of interest for major pharmaceutical companies.

The only standard treatment currently in use is palliative radiotherapy – given to Abbie. “During two months she suffered and treated her,” she remembered her mother. Abbie died on the morning of 13 September 2011. She was six years old.

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

As a result of their experience the Mifsuds established their charities, and since then he has raised funds to research DIPG which has played an important role in helping Jones direct his work on the disease. Two years ago, he discovered that there were mutations in the ACVR1 gene, which had never been detected in any other type of cancer previously, with mutations caused by the disease. The mutation means that the gene is susceptible to being turned on and to guide the manufacture of proteins when it is supposed to turn off.

This information suggests that ACVR1 is involved in biochemical events produced by DIPG tumors and which provided a specific target for Jones and his colleagues to tackle the condition. The first one is for scientists who are working on DIPG. Working with the pharmacy group M4K Pharma, based in Toronto, they have developed a new class of drugs which may be treatments for the condition.

“We identified five main candidate drugs for further clinical studies and we are looking forward to starting clinical trials within two years,” said Owen Roberts, chief executive of M4K Pharma.

The first effective life-saving medicine to treat children affected by DIPG is seen only as quickly as the first trials are conducted.

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

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