THE MARVEL MOLECULE

Thanks to your gifts, UEA researchers edge closer to a new treatment for childhood bone cancer.

It has been the most significant breakthrough in the treatment of childhood bone cancer for more than 40 years. But Bensofib, UEA’s new marvel molecule that could double the survival rate of children with cancer with significantly less toxicity than chemotherapy, is reliant on philanthropy to become a medical reality.

Today, being diagnosed with bone cancer is a deeply frightening prospect for children. Just two in five will survive for five years. Many who do survive are left disabled due to amputations or the metal rods that replace affected bones. With the support of donors, we want to double the survival rate and avoid unnecessary treatments.

The Science Behind The Discovery

Dr Darrell Green (BIO06) is the scientist we introduced you to in the previous issue of Pioneer. He leads UEA’s childhood bone cancer research. His breakthrough discovery was made by isolating and analysing circulating tumour cells in patients’ blood. After a year spent searching for these one-in-a-billion cells, his team identified a potential gene responsible for the spread of cancer from one part of the body to another, a process known as metastasis.

“Babies develop in the womb thanks to specific embryonic genes that instruct cells to move around and make the baby, which become less active after birth as they are no longer needed,” explained Dr Green. “But we discovered that bone cancer cells reactivate these embryonic genes and pathways to achieve their goal of spreading to distant parts of the body.

“With support from donors, we pioneered an experiment that deactivated those genes in cancer cells. With our new molecule, tumours were less aggressive and stopped spreading to the lungs, representing extraordinary levels of potential as a treatment. We called the new molecule Bensofib.

“Our drug is named for Ben Morley, my childhood friend who died from bone cancer aged 13 and inspired my work; Sophie Taylor, a research participant who lost her life to bone cancer at five years old; Professor Bill Fraser, my academic mentor; and the gene inhibitor.

“It’s one thing being able to understand how or why cancer spreads at the molecular level, but it’s another to produce a new molecule or drug that can modify genetic processes in real time in a living being. That’s exactly what philanthropic support has allowed us to do.” u

u THE NEXT STAGES

There are two kinds of bone cancer that Bensofib is targeting. In 2020-21 pre-clinical trials, the new treatment led to the complete remission of Ewing sarcoma. In the second kind of cancer, osteosarcoma, the team saw a 50% increase in metastasis-free survival. And excitingly, there were no side effects.

The next stage for our researchers will be to subject Bensofib to extensive testing to make sure that it is safe to proceed with clinical trials. We will decide on a safe and effective dose for the new drug through toxicology studies. Following MHRA approval, clinical trials will begin, and Bensofib will start its first test phase in children with bone cancer to prove its effectiveness against treatments that are currently being used.

Enormous Potential

Alongside the main research focus on childhood bone cancer, Dr Green’s team has also seen Bensofib’s potential to treat other conditions.

“One of the genes we are studying is also involved in the spread of skin and lung cancer,” said Dr Green. “Our new drug also shows positive signs for preventing other cancers from spreading to the bones. There is just so much possibility here.”

There has been a historical lack of funding for childhood bone cancer. Just £147,462 was spent last year on research into the disease by the National Cancer Research Institute

THERE IS JUST SO MUCH POSSIBILITY .

Dr Darrell Green

and partners, including Cancer Research UK and the Medical Research Council. This is less than 0.02% of the total national UK cancer research budget of £669m. This is a year-on-year reduction of 25% and goes some way to explain why bone cancer standard care, chemotherapy and surgery haven’t changed since the 1970s.

Developing new drugs and treatments has become significantly more expensive since the mid-20th century. In recent years, drug breakthroughs have become more and more challenging to come by. A phenomenon known as ‘Eroom’s Law’, coined by industry consultant Jack Scannell, observes that the cost of developing new drugs doubles, in real terms, every nine years.

That is why Dr Green’s research is such an exciting opportunity. This is a chance to complete significant research into treatments for a disease that has received little attention up to this point. Meaning that donors could make a breath-taking impact in return for sums far lower than those contributed to better-known conditions.

Uea Expertise

We are privileged to have some of the foremost minds in research here at the University. With more than 300 researchers and £60 million worth of ongoing research, we are expanding the world’s understanding of ageing, cancers and cardiometabolic and gut health.

In the most recent Research Excellence Framework, 85% of our research output was rated as world-leading or internationally excellent.

The accomplishments of our researchers maintain the University’s standing as one of the top destinations for great minds – in the Times Higher Education World University Rankings 2022, UEA was rated in the World Top 200 universities and the World Top 50 for research citations.

Philanthropy Is Essential

A debt of gratitude is owed to the generous individuals who have allowed us to get to this position and those who will help us reach the finish line.

Without the extraordinary philanthropic support we have received, none of the significant progress made at UEA would have been possible. Children diagnosed with bone cancer would be no closer to new treatments that save lives and avoid debilitating amputations.

A donation today could result in fewer children losing their lives in years to come. Fewer families left with an irreparable hole in place of their son or daughter or struggling to support little ones left with a permanent disability. Could there be a more precious gift?

“Bone cancer remains one of the deadliest cancers and one of the most brutal to treat,” said Dr Green.

“The support from our donors places us on the verge of the first new treatment in more than 40 years. We are very grateful to everyone who has backed this transformative research. There is still lots of work needed to get over the finish line, so I also reach out to new donors. They will, quite literally, watch in real time the progress their generous donations will accomplish. Thank you all very much.”