New bone cancer drug unveiled
Thursday March 9th 2023
A new drug could treat the main types of primary bone cancer without toxic side effects, British researchers say.
Scientists at the University of East Anglia and University of Sheffield, UK, say the drug, CADD522, increases survival rates by 50% without the need for surgery or chemotherapy.
Study co-author 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."
Writing in the Journal of Bone Oncology, the researchers say when they implanted mice with human bone cancer the drug blocked a gene associated with driving the cancer's spread.
They collected bone and tumour samples from 19 patients at the Royal Orthopaedic Hospital in Birmingham, which was more than enough to detect some obvious changes in the cancers.
The team used next generation sequencing to identify small RNAs that were different during the course of bone cancer progression and showed the RUNX2 gene is activated in primary bone cancer and also that it is this gene that is associated with driving the cancer's spread.
They went on to develop CADD522, a small molecule that blocks the RUNX2 protein from having an effect, and tested it in mice.
Lead researcher Dr Darrell Green, from UEA's Norwich Medical School, said: "In preclinical trials, metastasis-free survival was increased by 50% using the new CADD522 drug on its own, without chemotherapy or surgery. I'm optimistic that combined with other treatments such as surgery, this survival figure would be increased further.
"Importantly, because the RUNX2 gene is not usually required by normal cells, the drug doesn't cause side effects like chemotherapy. This breakthrough is really important because bone cancer treatment hasn't changed for more than 45 years."
The new drug is undergoing formal toxicology assessment, in the hope of being used in a human clinical trial.
Green D, Singh A, Tippett VL et al. YBX1-interacting small RNAs and RUNX2 can be blocked in primary bone cancer using CADD522. Journal of Bone Oncology. March 2023.
Tags: Cancer | Child Health | Pharmaceuticals | UK News
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