28/11/2019
As brain cancer survival rates have traditionally refused to improve, this is one researcher who believes he may have found a potential path to success.

While survival rates for blood cancers have been gradually improving over the last few decades, the same cannot be said for brain cancers. As a result, the biggest disease-related killer of children in Australia is brain tumours. 

Medulloblastoma is a brain cancer connected to the cerebellum, a part of the brain that helps coordinate the body’s movement and balance. This type of cancer is associated with poor survival rates, and those who do survive often suffer reduced quality of life as a result of treatments. 

“Right now, standard therapy involves surgery to remove as much of the tumour as possible, then radiation therapy which involves radiating the brain and sometimes the spinal cord, then chemotherapy,” said Professor Brandon Wainwright from the University of Queensland.

“Radiotherapy and chemotherapy work on the basis that they damage the DNA inside the tumour cell, so the cell kills itself. The problem is that they work by acting on cells that are growing."

"That makes sense, as tumours are caused by cells that don’t understand what they are meant to be doing, so their response is simply to continue growing. But these treatments also act on normal cells in the bodies of babies and kids, who are growing,” he said.

If a child survives the cancer, the treatment can result in permanent disabilities such as loss of sight, touch and hearing, intellectual disabilities, lowered prospects of independent living later in life, and more.
 
Wainwright is driven by the hope that he could do something to improve survival rates and also to boost quality of life for survivors, and he just might be on the right track.

 
The difference between a normal cell and a brain cancer cell is that the genetic program inside the cancer cell, the program that usually tells a cell what its job is within the body, is broken. It’s like a computer with a faulty operating system – it simply won’t do the job it is supposed to do. Instead, as mentioned, the cell simply grows.
 
Unfortunately for researchers, brain cancer is not one disease, it is many. There are also varying types within each specific brain cancer. Medulloblastoma, for instance, may have up to 11 different versions, each of which requires a unique solution.
 
The standard therapies – surgery, radiotherapy and chemotherapy – are general therapies and are not helping to shift the needle on survival rates. A new approach is required, and Wainwright is closing in on a potential solution. 

“Right now, the gold standard therapy for kids with brain cancer is not acceptable,” he said. “So our process is to try to understand the genetic networks in types of tumours, to come to understand where the software became broken, and develop tailor-made therapies for each type of cancer.”

The “broken software” is what causes the tumour, so Wainwright has been hunting for the cause of the broken software. For one type of medulloblastoma, one cause is a genetic syndrome called naevoid basal cell carcinoma syndrome, which is passed on genetically along a pathway known as ‘Hedgehog’.
 
“It turns out that this one genetic pathway, when it is switched on in the skin, causes non-melanoma skin cancer,” he explains. “When it was switched on in the brain, it causes medulloblastoma.”
 
The gene, known as ‘Patched’, which controls the Hedgehog pathway and therefore drives a predisposition for developing medulloblastoma, was the first gene ever discovered to cause brain tumours.
 
“Using our original findings as the basis for further research, our lab has identified many of the core genetic components that lead to the development of medulloblastoma,” Wainwright said. “This work will enable the therapeutic targeting of every medulloblastoma, not just a subset, leading to more powerful clinical trials and ultimately more effective treatment options.” 

“This seems very promising. But before we can get to a clinical trial, we have to prove that our new therapy combines well with standard therapy."

"The Kids’ Cancer Project has been supporting our testing process. It’s important that people realise that we couldn’t do this, we couldn’t do the work that could make a real difference for families and kids, without the support of The Kids’ Cancer Project,” he said.

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