Experimental Drug Gives Hope To Children With Neuroblastoma Cancer

By Alyssa Navarro | Tech Times | November 7, 2015 | See Original Here

[COMMENT: Although not Rhabdo specific, this should provide the Rhabdo community with some hope that what's being discovered in the lab is moving towards clinical application (Alan)]

Researchers from the Children's Cancer Institute (CCI) in Australia recently developed an experimental drug which could give hope to children who are diagnosed with neuroblastoma cancer.

Neuroblastoma, the cancer of the nervous system, is one of the most common solid tumors that could grow during early childhood. Experts say that up to 70 Australian children are diagnosed with the disease at the age of five years old every year, and that only less than half of these patients who experience the disease's aggressive form survive.

A seven-year-old boy named Hugo Prosser was just 10 months old when he was diagnosed with the cancer. He has gone through a lot of chemotherapy rounds and multiple surgeries over the years.

"To have that condition taken out of the dictionary — put a line through it, doesn't exist anymore — that would be great," said his father Jason.

Researchers hope that the CCI study published in the journal Science Translational Medicine would pave way for more effective cancer treatment.

In the study, scientists identified a molecular feedback loop that helps initiate and accelerate the development of neuroblastoma. They discovered that an experimental drug which is currently used in clinical trials for adult cancer has the potential to stop the loop and the progression of the tumor.

The combination of the drug known as CBL0137 and conventional chemotherapy agents is more effective than just the drug alone, researchers said. Conventional chemotherapy often damages the DNA and cancer cells repair it. Researchers said that CBL0137 prevented the cancer cells from restoring DNA, ensuring cell death.

"We've known for some time that one gene has been a driver in that cancer," said Professor Glenn Marshall, one of the researchers of the study.

Marshall and his colleague Dr. Daniel Carter focused on the molecular and genetic mechanisms that make the feedback loop work, and how it is interrupted by the experimental drug. They found that behind the loop is a gene called MYCN and a molecule called FACT which modifies DNA and is the target of CBL0137 in order to stop the process.

Carter said that the neuroblastoma tumor showed signs of cell death after 24 hours of being attacked by the experimental drug. He said the drug is extremely potent and rapid, as well as a good side effect profile in their system.

Meanwhile, the Children's Oncology Group of the United States will take the experimental drug into clinical trials in 2016.

"It's the first type of experiment of its kind and hopefully will lead to more experiments and discoveries that could eventually lead to prevention, because in cancer, prevention is much better than cure," added Marshall.

Researchers believe that the experimental drug has all the requisites of a good cancer therapy, and that there will be greater and more rapid shrinkage of the tumor because of the treatment.