Bone cancers such as osteosarcoma and Ewing’s sarcoma predominantly affect children. It is the third most common childhood solid cancer, after brain and kidney, with approximately 52,000 new cases each year worldwide. It can spread rapidly to other parts of the body, especially the lungs, which is the most problematic aspect of this type of cancer. Once the cancer has spread, it becomes very difficult to treat it with curative intent: current treatment is grueling, with outdated chemotherapy cocktails and amputation. And yet, the 5-year survival rate is just 42%. But a new study published today shows how a new drug called CADD522 blocks a gene associated with driving the spread of cancer in mice implanted with human bone cancer. Breakthrough drug increases survival rates by 50% without the need for surgery or chemotherapy. And unlike chemotherapy, it doesn’t cause side effects like hair loss, tiredness, and malaise.
Lead researcher Dr Darrell Green, of the University of East Anglia’s Norwich Medical School, was inspired to study childhood bone cancer after his best friend died of the disease as a teenager. Now, the team has made what may be the most important drug discovery in the field for more than 45 years. The team collected bone and tumor samples from 19 patients at the Royal Orthopedic Hospital in Birmingham. However, this small number was more than enough to detect some obvious changes in the tumors. The team used next-generation sequencing to identify the types of genetic regulators called small RNAs, which were different during the course of bone cancer progression. They also showed that a gene called RUNX2 is activated in primary bone cancer and associated with its spread. They went on to develop a new drug called CADD522, which blocks the RUNX2 protein from having an effect, and tested it in mice.
The drug is now undergoing a formal toxicology evaluation before the team gathers all the data and gets closer to approval to start a human clinical trial. Green said: “In preclinical studies, metastasis-free survival increased by 50% using the new drug CADD522 alone, without chemotherapy or surgery. I’m optimistic that combined with other treatments like surgery, this survival figure would increase even further. Importantly, because the RUNX2 gene isn’t usually required by normal cells, the drug doesn’t cause the classic side effects of chemo. The new drug we have developed is effective in all major subtypes of bone cancer and, so far, our experiments show that it is not toxic to the rest of the body. That means it would be a much kinder treatment for children with bone cancers, compared to the grueling chemotherapy and life-changing limb amputation of today’s patients.”
- By Dr. Gianfrancesco Cormaci, PhD, specialist in Clinical Biochemistry.
Scientific publications
Green D et al. J Bone Oncology 2023 Apr; 39:100474.
Kamolphiwong R et al. Gene. 2023 March; 856:147106.
Huang X et al. Genet Mol Biol. 2023; 46(1):e20220136.
– Degree in Medicine and Surgery in 1998 (MD Degree in 1998) – Specialist in Clinical Biochemistry in 2002 (Clinical Biochemistry residency in 2002) – Doctorate in Neurobiology in 2006 (Neurobiology PhD in 2006) – Stayed in the United States, Baltimore (MD ) as a researcher employed by the National Institute on Drug Abuse (NIDA/NIH) and then at Johns Hopkins University, from 2004 to 2008. – Since 2009 he has been involved in Personalized Medicine. – Emergency medical care in private structures since 2010 – Holder of two patents on the preparation of gluten-free products starting from regular enzymatically neutralized wheat flour (owner of patents concerning the production of gluten-free bakery products, starting from regular wheat flour). – Head of the Research and Development department for CoFood srl (Leader of the R&D for the partnership CoFood srl) – Author of articles on medical and health information on the website www.medicomunicare.it (Medical/health information on website) – Author of ECM FAD courses advertised on the website www.salutesicilia.it