Thanks to the Good Friday Appeal’s Run for the Kids initiative, newly diagnosed patients with acute lymphoblastic leukaemia (ALL) and lymphoblastic lymphoma at The Royal Children’s Hospital (RCH) have been able to access the latest in personalised medicine.

The trial, known as the TOTAL Therapy Study XVII or TOTAL 17, is an international collaboration led by St Jude Children’s Research Hospital in America.  

Over the past two years, 62 patients at the RCH have been enrolled in the innovative program, far exceeding the initial aim of enrolling 20 patients per year. Recruitment for the trial ceased in mid-2023 due to reaching the trials capacity limit, with patients enrolled continuing with follow up therapy and treatments including neurocognitive assessments.

Once enrolled, the child’s blood and bone marrow samples were sent to St Jude. There, clinicians used state-of-the-art tools and gene mapping to identify the specific cancer-causing gene combinations, providing patients with the opportunity to receive treatment that is specifically designed to fight their type of cancer.  

“The Good Friday Appeal fundraising is critical for every piece of the cancer journey. We need the latest research, diagnostic tools, best clinical trials, and patient and family support networks, and the Good Friday Appeal has been a cornerstone for each part of that.  

“Importantly, thanks to the Good Friday Appeal, we are also able to provide children with leukaemia and lymphoma access to one of the best clinical trials in the world,” said Dr Hanna.  

One of the patients enrolled in the trial is five year old Bobby, who was diagnosed with B-cell ALL when he was three.  

For patients like Bobby, the long term impacts of the TOTAL Therapy Study XVII are life changing.  

“One of the best things the trial has offered us is the additional supports, including the neurocognitive and physio assessments. It has been invaluable for us when considering things like getting Bobby ready for school, including any adjustments we need to make,” said Trudy, Bobby’s mum.  

“Additionally, the idea that we could be part of that larger pool of research is significant to us. Being able to contribute to future developments to benefit other families made our decision an easy one,” she added. 

Impact Milestones

2023 – 2024

• By the end of April 2023, 48 out of the 62 enrolled patients completed baseline tests to determine how the brain works in terms of thinking, learning, remembering and problem solving.
• The tests provided valuable data and insights for early interventions and referrals to relevant departments.
• Samples from patients were sent to St Jude Children’s Hospital for comprehensive genomic profiling of leukemia cells. Out of the 52 patients profiled, 16 received targeted therapy.
• Thanks to the new and targeted therapies, some patients who entered the trial avoided having a bone marrow transplant. 
• Based on sampling, St Jude’s has also helped identify two patients with a variant that may influence cancer predisposition.
• Patients and their families have been linked with RCH Genetic Counselling services.
• Beyond immediate treatment, patients were also offered assessments that look at how treatments affect the brain and thinking abilities.
• As of September 2024, the study remained close to recruitment, with 45 patients still participating in the study. Meanwhile, three have completed the study and are in follow-up therapy.