Experimental drug giving hope to kids with cancer


Australian scientists have published new findings into an experimental drug that could help eradicate one of the most common childhood cancers.

Neuroblastoma is diagnosed in up to 70 Australian children under the age of five every year, and less than half of those with aggressive forms of the disease will survive.
It is a cancer of the nervous system that's usually diagnosed when it's well advanced.
But research by the Children's Cancer Institute of Australia (CCIA) has revealed how the cancer progresses and how an experimental drug could hold the key to interrupting its progress.
"We've known for some time that one gene has been a driver in that cancer," one of the study's authors Professor Glenn Marshall said.
"What we've discovered is another gene that acts like an accelerant on a fire. It gives the cancer cell this infinite capacity for mutation.'
The CCIA's report identifies a so-called "feedback loop" where a gene known as MYCN works together with a molecule called FACT to drive the cancer.
When chemotherapy is used to fight the cancer, damaged DNA cells become vulnerable to being repaired by cancer cells. 
But a drug known as CBL-0137 was found to have stopped this, while also targeting the FACT molecule.
"The tumor is almost completely gone or is showing signs of death after 24 hours," another author of the study, Dr Dan Carter said.
"It's extremely rapid, extremely potent, it has a very good side effect profile in our model system. So we're hoping it has all the requisites of a good cancer therapy."
Hugo Prosser was just 10 months old when doctors found five solid tumours throughout his body. 
Now aged seven, he has undergone multiple surgeries and rounds of chemotherapy over the years, taking a huge toll on him and his family.
"We look at things much differently," his father Jason told SBS News.
"Our focus is on positive things and we realise we've had some hard knocks, but we get to take him home. There are a lot of parents who don't take their kids home and we're a lot more fortunate than some people."
"To have that condition taken out of the dictionary - put a line through it, doesn't exist anymore - that would be great."
The drug is currently being trialled on adults in Russia and the United States. 
The next step would include testing it on children here in Australia, which doctors hope will begin in the next 18 months.
If successful, it could lead to less hospital time for patients and fewer rounds of chemotherapy.

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