Researchers at the University of East Anglia and The University of Sheffield have developed a new bone cancer treatment for children.
Currently, bone cancer treatment depends on the type of bone cancer you have, how far it has spread and your general health; however, the main options are surgery, chemotherapy and radiotherapy, which are brutal and can lead to life-long disabilities.
To improve treatment options, teams from the University of Sheffield and the University of East Anglia have developed a new drug that works against the main types of primary bone cancer. They posted their findings in the Journal of Bone Oncology.
Urgent interventions are needed to tackle the current survival rates are poor
Current bone cancer treatments are painful and uncomfortable, yet the five-year survival rate remains poor at just 42% – largely because of how rapidly bone cancer spread to the lungs. These rates haven’t improved in nearly half a century.
The researchers have developed a new bone cancer treatment called ‘CADD522’ that blocks a gene associated with driving the cancers spread in mice implanted with human bone cancer. This drug increases survival rates by 50% without surgery or chemotherapy and does not cause toxic side effects like hair loss and tiredness seen in chemotherapy.
Co-author of the study, Professor Alison Gartland from the University of Sheffield’s Department of Oncology and Metabolism, said: “Primary bone cancer, although rare, occurs most often in children and young adults between the ages of 10 and 20 usually during a growth spurt. It is a difficult cancer to treat as it can spread very rapidly to other parts of the body – especially the lungs.
“Currently, children have to undergo very toxic treatment which has very unpleasant and sometimes life-long side effects and sometimes life-changing amputation. This, coupled with the low survival rate, is why this drug is so incredibly important and could make a huge difference to patients and their families.
“This breakthrough was only possible due to the extensive collaboration between teams at the Universities of Sheffield and East Anglia, and I sincerely hope that with further research and backing this drug can be used in clinical trials in the near future.”
Testing the drug in 19 patients
The researchers collected bone and tumour samples from 19 patients at the Royal Orthopaedic Hospital in Birmingham. However, this small trial was enough to detect some obvious changes in the cancers.
The team used next-generation sequencing to identify types of genetic regulators called small RNAs that were different during bone cancer progression. They also showed that a gene called RUNX2 is activated in primary bone cancer and that this gene is associated with driving the cancer’s spread.
They went on to develop CADD522, a small molecule which blocks the RUNX2 protein from having an effect and tested it in mice.
Lead researcher Dr Darrell Green, from UEA’s Norwich Medical School: “In high school, my best friend Ben Morley became ill with primary bone cancer. His illness inspired me to do something about it myself because during my studies I realised that this cancer has been all but left behind others in terms of research and treatment progress.
“I wanted to understand the underlying biology of cancer spread so that we can intervene at the clinical level and develop new treatments so that patients won’t have to go through the things my friend Ben went through.
“Ultimately, we want to save lives and reduce the amount of disability caused by surgery. Now we have developed a new drug that potentially promises to do just that.”
The new drug is now undergoing formal toxicology assessment before the team assemble all of the data and approaches the MHRA for approval to start a human clinical trial.
