Scientist whose friend died at 13 of bone cancer has made a huge breakthrough in the fight against the disease

A scientist who devoted his career to treating bone cancer after his best friend died of the disease at the age of 13 has achieved the biggest breakthrough in his treatment in fifty years. Dr. Darrell Green of the University of East Anglia helped develop a new generation of drug that he believes could save thousands of lives around the world.

The current treatment for bone cancer, which mostly affects children, is debilitating and usually involves outdated chemotherapy cocktails as well as limb amputations. Despite this, the five-year survival rate is only 42 percent.

A new drug called “CADD522” has been shown to block a gene associated with the spread of bone cancer.

A new lab study published in the Journal of Bone Oncology shows that the revolutionary treatment increases survival by 50 percent—without the need for surgery or chemotherapy.

And unlike chemotherapy, it does not cause toxic side effects such as organ damage, hair loss, fatigue, and nausea. The researchers are currently trying to find funding for human trials, which could begin within two years if they manage to raise enough money. Human trials could begin within two years.

Dr Green said: “Primary bone cancer is the third most common solid cancer in children after brain and kidney, with over 600 cases in the UK and about 52,000 new cases each year worldwide.

“It can quickly spread to other parts of the body, and this is the most problematic aspect of this type of cancer. Once the cancer has spread, it becomes very difficult to treat.”

Dr Green of Norwich Medical School at UEA decided to study bone cancer in children after seeing his best friend Ben Morley suffer and die from the disease.

Dr Green recalls: “Ben and I grew up together. I met him on my first day at school. We arrived first and became best friends from that day on. One day in late 2000, Ben felt pain in his left knee. He was 12 years old. We thought he might have been kicked while playing football, but the pain only got worse and his knee became swollen.”

Ben was diagnosed with an aggressive childhood bone cancer known as Ewing’s sarcoma. It is one of three types of bone cancer targeted by the new treatment.

Dr. Green said: “Ben is lucky to have been diagnosed so quickly – it takes an average of seven doctor visits before a diagnosis of bone cancer is made. He underwent chemotherapy and had his left leg amputated in early 2001.”

Despite the best efforts of his doctors, on September 11, 2001, Ben’s parents were told that there was nothing more the doctors could do to save him. He died on February 23, 2002 from severe pain and was unable to speak due to his weakness.”

Dr. Green said: “This was my first experience of death. I think about Ben every day. Whenever something gets difficult, or I work late into the night, or something doesn’t work and I want to turn it all on, I just remember that Ben is not here to be given the opportunity to do things himself, what he wants. There are many other families and child patients that I have met who will not have these opportunities either. This gives me extra enthusiasm and encourages me to improve our understanding of the underlying biology of how cancer spreads so that we can intervene with new treatments so that patients don’t have to go through what my friend Ben went through.

“Ultimately, we want to save lives and reduce the amount of disability caused by surgery. And now we’ve developed a new drug that potentially promises to do just that.”

Dr Green and his team collected bone and tumor samples from 19 patients at the Royal Orthopedic Hospital in Birmingham and used next-generation sequencing to identify types of genetic regulators called small RNAs that differed as bone cancer progressed.

They showed that a gene called RUNX2 is activated in primary bone cancer and that this gene is associated with the spread of cancer.

They then developed a new drug called CADD522, a small molecule that blocks the action of the RUNX2 protein, and tested it in mice.

Dr. Green said: “In preclinical trials, metastasis-free survival has been increased by 50 percent when using the new drug CADD522 without chemotherapy or surgery. I am optimistic that when combined with other treatments such as surgery, this survival rate will increase even more.

“Importantly, since the RUNX2 gene is not normally required by normal cells, the drug does not cause side effects such as chemotherapy.

“This breakthrough is really important because bone cancer treatment hasn’t changed in over 45 years.

“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. This means that the treatment of children with bone cancer will be much gentler compared to the debilitating chemotherapy and limb amputations that are life-changing for patients today.”

“We hope this will save many lives,” he added.

The drug is currently undergoing formal toxicological evaluation before the team collects all the data and applies to the MHRA for approval to begin clinical trials in humans.

The study was conducted by UEA in collaboration with the University of Sheffield, Newcastle University, Birmingham Royal Orthopedic Hospital and Norfolk and Norwich University Hospital.