20/01/2020
An exciting, new clinical trial has launched after seven years of game-changing research, none of which would have been possible without funding.

The single greatest limiting factor for those working in the field of kids’ cancer research was once technology, says Dr Raelene Endersby, Co-Head, Brain Tumour Research at the Telethon Kids Institute. But that’s no longer the case. Today, the only factor slowing down greater success in the battle against kids’ cancer is funding.

“If we had more money, we could put many more people onto the various problems we’re facing and we would be able to move forward much faster,” says the highly lauded researcher. “We can do all sorts of things with technology now. But if my team was twice the size, we’d be able to do twice as much work.”

Dr Endersby has proven – with her work over the last seven years resulting in clinical trials of a new drug for use in the treatment of currently incurable forms of medulloblastoma (see timeline below) – that research can be relied upon to come up with the goods.
 
She and her team of clinicians, neurosurgeons and laboratory scientists have developed a new drug that appears to make cancer cells more vulnerable to traditional treatments such as radiotherapy and chemotherapy. The drug works by switching off a specific protein in cancer cells that sense damage and begin the cell repair process. In other words, it removes the cancer cells’ ability to recover.
 
This could make traditional treatment significantly more effective, and might also reduce chances of relapse.
 
Partnered in her work with Dr Nick Gottardo, a Consultant Paediatric Oncologist/Neuro-Oncologist and Head of Department of Paediatric Oncology and Haematology at Princess Margaret Hospital for Children, Dr Endersby says all of her work has a highly practical application.


The work Dr Raelene Endersby and Dr Nick Gottardo do in the lab has a highly practical application.

“I have always been interested in cells and genetics,” she says. “We have a lot of face-to-face interactions with families and kids with cancer. We see the effects every day. That motivates me to do translational research and make a real difference.”
 
“Some of the traditional treatments we give these kids are very toxic, and so they’re damaging. We know so much in this space, but we need to do better when it comes to treatment.”

“I see the deficiencies in the current treatments. I see kids who were treated, and I see what they go through over 12 months or more. Then sometimes, after all of that, the tumours return. It’s heart-breaking. So we’re not there yet, but I can recognise the potential.”

Dr Endersby can see a time in the future when treatments will be refined to a point where survival rates will not only be dramatically improved, but where solutions can be individualised for particular patients and cancer types.
 
“What we need to do is find the new treatments for all of the different molecular subgroups of medulloblastoma and other cancers,” she says.

“From this project, which began in 2013, we’ve established a pipeline that allows us to evaluate compounds in vitro. We can then take them through mouse models and we have all the expertise and technology we need to assess toxicity and efficacy. So this drug could simply be the first of many.”

“What I envisage is many more molecularly targeted agents combining with conventional agents to target specific features in the cancers. In the future oncologists will have a menu they can go through. From this they can marry features of a child’s cancer with the knowledge we’ve generated through research to identify the most effective drugs for that particular patient. Then they have a clinical protocol that has been tested and is established.”
 
It’s a bold and glorious goal, and it’s one that can become a reality, Dr Endersby says. All it needs is funding.
 

Timeline
  • 2013: A drug screen using human medulloblastoma cells, looking for drugs that are effective against the brain cancer, begins.
  • 2014: Initial drug screen is expanded to test the most effective drugs in combination with current clinical treatments. The researchers are filtering drugs that are potentially able to combine with and improve current treatments from those that are not.
  • 2016: Less than 50 drugs pass the stringent, in-vitro tests, and around 30 of these are removed as other research had shown they would not get into the brain. Eventually, just a few from the original 4,000 are shown to inhibit a specific protein that helps cancer cells resist eradication.
  • A project grant from The Kids’ Cancer Project helps to compare these drugs from several different pharmaceutical companies.
  • 2017: The team begins animal modelling work to optimise the treatment regimen (the sequence, dosage and side effects) with the new compound, following the standard-of-care protocol of surgery, radiation therapy and chemotherapy’s potential use of four different drugs.
  • Funding from The Kids’ Cancer Project helps the team use advanced equipment, the first of its kind in the southern hemisphere. This equipment allows effects of radiation therapy with the new drug to be precisely measured.
  • 2018: The researchers develop detailed models around the types of tumours, or genetic mutations, on which this new compound will be effective. On two different molecular sub-groups of medulloblastoma, both currently incurable, different combinations of the new drugs appeared as if they might be effective. This makes it clear which specific patients might benefit from the new treatment regimen.
  • 2019: A clinical trial opens in Australia, Germany and the USA.

 
 
 
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