World-first clinical trial in ‘man’s best friend’ could unlock treatment breakthrough for kids with cancer.

Telethon Kids Institute is leading a unique clinical trial in pet dogs that could pave the way for a new immunotherapy treatment for one of the most common childhood cancers, Sarcoma. Sarcoma is a cancer of the bones and soft tissues and is the third most common cancer in children, with one in three dying from the disease.

The treatment is a polymer-filled gel which is loaded with immunotherapy drugs and can be applied inside the surgical wound when a patient has a sarcoma tumour removed.

The head of Telethon Kids Institute’s Cancer Centre, Associate Professor Joost Leisterhuis, said the first step for almost all sarcoma patients after diagnosis is surgery to remove the tumour. “The surgeon will try to remove as much tissue as possible, but often some cancer cells are left behind and the cancer just comes back,” he said.

“With the immunotherapy gel, the surgeon would remove the tumour and apply the gel before closing the wound as they normally would. Then over time, the immunotherapy drugs get to work – drawing immune cells from all over the body and activating them at the site of the tumour to mop up any remaining cancer cells.”

The gel has shown positive results in lab models and is now being used at Perth Vet Specialists to treat pet dogs who have been diagnosed with sarcomas.

“Surprisingly, sarcoma is one of the most common cancers in pet dogs and it presents exactly the same way as it does in children with sarcoma, which is a lump in the tissue of the muscle or bone,” Associate Professor Leisterhuis said. “The treatment is also the same – to surgically remove the tumour. And often, as with children, some cells will remain, and the cancer will come back.

“This trial is giving beloved family dogs access to cutting-edge treatments for their cancer, and their owners can know that they are playing a role in getting this treatment one step closer to helping kids with cancer.”

Veterinary oncologist, Dr Wyatt from Perth Vet Specialists said so far seven dogs had been treated with the gel in surgery and the results have been overwhelmingly positive. “Sarcoma is actually more common in dogs than it is in people – we would see multiple patients per week with sarcoma and they can be fatal if they are left unchecked or spread into nearby organs,” he said.

“There doesn’t seem to be any problem in the healing of the dogs who’ve taken part in the trial so far, we seem to be getting a really good response from the gel. Everybody’s winning – the dogs are getting treatments they would otherwise not have access to and they’re also doing something that could ultimately help children with cancer.”

The gel was developed in collaboration with molecular scientists at The University of Western Australia, led by Associate Professor Killugudi Swaminatha Iyer. Associate Professor Iyer, from UWA’s School of Molecular Science, said the gel is made from natural materials.

“The gel is made of long polymers which are natural and that are broken down by the body itself,” he said. “So for this treatment, the team has tagged on some immunotherapy to those long polymers which are then slowly released in the body and the polymers themselves are broken down.”

The Telethon Kids Cancer Centre is dedicated to finding new, more gentle treatments for kids with cancer. Associate Professor Lesterhuis said there have been very few developments in sarcoma treatment in the last 30 years and children are still exposed to high doses of toxic radiotherapy and chemotherapy.

“Immunotherapy is one of the most exciting developments in cancer treatments but so far it has not had much success in sarcoma treatment.” he said.

“This is a cruel disease and the life-long side effects that kids suffer from more traditional treatments include learning difficulties, infertility, speech and vision problems and even secondary cancers – where the treatments themselves cause new cancers to develop.

19-year-old Angus Hollington (pictured in the banner image with Bull Terrier Maggie) has survived cancer twice. He was diagnosed with Ewing’s Sarcoma at age 11 and underwent intensive treatment. As a result of his treatment, he developed leukaemia at age 17. Around the same time, Angus ended up having his leg amputated as a result of infection from bone grafts from his sarcoma treatment.

Angus is the perfect example of why we need less toxic treatments for kids with cancer.

“Our dream in the end is that with these sorts of immunotherapies we can replace chemotherapy and radiotherapy all together,” said Associate Professor Lesterhuis. “But at this stage I would see it as an addition, where we might be able to reduce some of the chemo or radiotherapy. We have to do better for these kids and their families.”

Our Foundation is proud to help make this exciting research possible, alongside the support of other generous donors. This is the kind of ground-breaking research you are helping to fund when you make a donation toward the Child Cancer Research Foundation.

 

Fostering the next generation of researchers at Telethon Kids Institute

One of the great things about the research projects that CLCRF funds is the opportunity to foster the next generation of researchers. The Telethon Kids Institute has around 100 students enrolled from several universities around Australia, conducting research projects from laboratory-based projects to population-wide studies.

We recently met with Taylor Ferguson, a Curtin University student who has commenced the first year of her PhD in Associate Professor Rishi Kotecha and Dr Laurence Cheung’s Leukaemia Translational Research laboratory. Taylor is continuing her studies from last year, where she completed her Honours degree alongside the respected researchers.

As part of this team, Taylor has been looking at a specific subtype of leukaemia, which has a rearrangement of the KMT2A gene and is associated with a poor prognosis. “Previously, my lab did a large drug screen to identify some novel agents that could be used,” said Taylor. “So, my project is to further investigate these novel agents in detail and move them forward in the preclinical setting.” The ultimate goal is for these novel agents to be translated into clinical trials.

Confessing that this was the only Honours project she applied for, Taylor had her heart set on the opportunity to help make a difference for future generations of children. “I did a mini research project at university which was to do with infants,” Taylor explained. “This drew my attention as to how vulnerable they are, especially to the side effects of chemotherapies. So, when I saw this project, I decided that it would be something I’d be interested in, to work towards improving outcomes for these infants.”

The prognosis of infants diagnosed under the age of one with KMT2A-rearranged acute lymphoblastic leukaemia is poor, with a 5-year event-free survival rate of less than 40%. “Because they are babies, they don’t have the ability to tolerate the chemotherapies the same way that older people might be able to,” explained Taylor. “So, if you’re trying to reduce the leukaemia disease burden, you have to increase the intensity of the chemotherapy. Unfortunately, babies can’t tolerate these high doses.”

There is a significant need to find new therapies to improve outcomes for these babies, which can only come with more funding. “Just to give these babies a fighting chance, especially those with this aggressive gene rearrangement, is the reason why extra funding is needed.”

When asked what her favourite thing about her job has been so far, Taylor stated that she has enjoyed becoming part of a team. “At university, it’s very much everyone for themselves and you do your own work,” she explained. “But in this team environment, you realise everyone is working towards a common goal. And so, it’s like a little community. Everyone wants to improve the outcomes for children with cancer, and specifically in my team, it’s for infants with leukaemia.”

Although now finding her feet in research, Taylor did not always know that this was the work she wanted do. “I kind of went through every career that I possibly could. When I first went to university, I wasn’t quite sure what I wanted to do. The more I got into science, the more I decided how much I loved it. And research is like focused science in a specific area. You also have outcomes that you can build off, and which other people can use to improve treatments or therapies.”

Grateful to be placed in this role, Taylor expressed how lucky she feels to be at the Telethon Kids Institute. “It’s a very good environment and the team that I have is just amazing,” she said. “Everyone’s been very supportive, and they all help me whenever I need it, which, at the beginning was a lot.”

Taylor explained that she would love to stay in the realm of leukaemia research. “I really enjoy the work,” she said. “It’s such a horrible disease, if there’s anything I can do to contribute to the research for it, that’s the research I want to do.”

We are delighted to be able to support you­­ng researchers like Taylor as they aim to improve the lives of children with cancer. You can help support Taylor’s research, and give infants with leukaemia a fighting chance by making a donation towards childhood cancer research today. Simply head to www.clcrfgiving.org

 

Behind the lab coat: 

What do you like to do in your spare time? 

I’m a bit of a homebody. I really enjoy spending time with my family. We moved from Scotland and it’s only the five of us here, so we’re very close.

I like to just relax and just have a good time reading books, going out with friends, things like that. I’m a very simple person.

What’s your favourite genre to read?

I usually read sci-fi or fantasy.

What’s your favourite quote?

My parents always tell me take every opportunity and you only fail if you never tried. They actually say that to me quite a bit.

It’s especially relevant in research, because not everything goes according to plan but you have to keep trying and keep persisting. Resilience is what gets outcomes at the end.

Local Immunotherapy for Sarcoma

Our Foundation has helped fund so many important research projects at the Telethon Kids Institute, working together with the Institute for decades to help improve the lives of children battling cancer. One of the exciting research projects that we are currently helping to fund is the Local Immunotherapy for Sarcoma project, headed by Associate Professor Joost Lesterhuis.

Joost is the Head of the Sarcoma Translational Research team at the Telethon Kids Cancer Centre, a team that aims to discover and develop safer and more effective treatments for sarcoma through inventive and rigorous research. One of the team’s current focuses is on developing immunotherapy approaches for childhood sarcoma that can be applied during surgery to prevent relapse.

Sarcomas are a group of cancers of the bone, muscle, or connective tissue. About 15% of cancers in children and adolescents are sarcomas, whereas it makes up only 1% of cancers in adults. “The biggest problem that has been facing sarcoma is that there hasn’t been a lot of progress in the last three decades,” said Joost. “Basically, we treat children with sarcoma the same as we did three decades ago.”

The relatively high incidence of the disease in children and the regularity of relapse makes the development of more effective treatments a high priority. “We really need to do better,” said Joost. “Because, at the moment, the primary treatment that we have available is surgery. With surgery, you really want to get rid of the tumour with a wide margin around it. Which, in some cases actually means amputations of limbs.”

Unfortunately, in many cases, some cancer cells will remain after surgery, which can then grow out in time and cause the cancer to come back. This means children must also go through harsh chemotherapy or radiotherapy before and after surgery. Even after all this, some children will still relapse.

“Once sarcoma returns, the prognosis becomes rather grim,” explained Joost. “One in three children will actually die from their sarcoma. So, we really have to do better.”

Currently, Joost and his team are developing a gel that can be left behind in the wound bed by the surgeon after sarcoma surgery. Children can then go home while the gel locally releases immunotherapy in the period that follows the surgery.

The aim is for the gel to attract and activate immune cells to mop up any remaining cancer cells, hopefully preventing the relapse of the sarcoma. The nature of the gel itself means that it safely degrades on its own.

This is such an exciting project and one that our Foundation is extremely proud to help fund. By continuing to fund this project, with the help of our generous community of supporters, we can see outcomes improve for so many children with sarcoma.

When you donate towards CCRF, you are helping to support world-class researchers like Joost and his team at the Telethon Kids Cancer Centre. This helps us get closer to seeing a future where children can live the cancer-free and side-effect-free lives that they deserve. If you would like to donate, simply head to our online donation page.

Images provided by Telethon Kids Institute

 

 

Lightening the leukaemia load for kids with Down syndrome

Source: First published Monday 14 September 2020 by the Telethon Kids Institute

Kids born with Down syndrome are at high risk of an array of health problems – including issues with sight, hearing, heart defects, bone complications, immune disorders and learning difficulties. One of the lesser-known complications is their increased risk of childhood leukaemia.

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