New clinical trial at GOSH gives hope to children with aggressive blood cancer

Researchers at GOSH and UCL Great Ormond Street Institute of Child Health (UCL GOS ICH) are collaborating on a novel approach to clinical trials to give hope to children with an aggressive type of blood cancer, T-cell acute lymphoblastic leukaemia (T-ALL).

Thanks to £2.4million of funding from GOSH Charity, Dr Sara Ghorashian, a Consultant Haematologist at GOSH, and her team will be running a trial for young people whose T-ALL relapses or is resistant to conventional treatments, including chemotherapy and bone marrow transplant. There is currently no standard of care for these children, and long-term survival can be as low as 30%.

As well as potentially providing a new treatment option for children and young people with relapsed T-ALL, the trial is also adopting a novel approach which will see it running alongside an adult’s clinical trial - taking place at University College London Hospital (UCLH) - for the first time in the hope of creating a more efficient trial design.

Dedicated research projects for children’s cancers are rare, with paediatric clinical trials traditionally starting later than adults’ and taking an average of six and a half years longer to complete. Only 12 anti-cancer medicines have been authorised for paediatric cancer specifically in the last ten years, compared to over 150 for adult cancers.

The phase one clinical trial will treat 12 children with relapsed T-ALL. It will be testing the effectiveness of a CAR T-cell therapy targeted at a specific protein called CCR9, that has been newly identified by researchers at UCL to occur exclusively on cancerous T-cells. Through the trial, Dr Ghorashian and her team are hoping to develop a new treatment that is more effective and targeted than current options.

CAR T-cell therapy, which involves genetically modifying the body’s immune cells to recognise and kill cancer cells, has already shown remarkable success in children with B-cell acute lymphoblastic leukaemia. However, this has proven difficult to replicate in T-ALL due to the CAR T-cells targeting either each other or the healthy T-cells, weakening the immune system even further. However, with the recent discovery of the CCR9 protein, there is now a promising, clearer target.

With the required funding in place, the trial is now going through all the necessary reviews and approvals with the expectation that it will be open in early 2025.

Any patients eligible to receive treatment under the NHS and interested in this trial should approach their specialist healthcare provider. In the interim, clinical teams will be happy to discuss alternative trials that are currently open for children with T-ALL.

One family who understands the urgent need for child-focused research is Maris Hanson and her husband Aivar, whose son Frank passed away from T-ALL in 2019, only months after receiving his initial diagnosis.

Born in 2017, Frank was a child who rarely got ill. But just before he turned two, he developed a cold with his health further deteriorating on a flight to celebrate his upcoming birthday with relatives and close friends in Estonia. Blood tests at a local hospital revealed he had T-ALL.

Within two days, instead of enjoying his birthday party, Frank was rushed to GOSH by air ambulance and started intensive chemotherapy treatment to tackle the aggressive cancer that was spreading throughout his body. Frank unfortunately didn’t respond to treatment and passed away after only four months in his family’s arms.

Since then, Maris has dedicated her life to raising vital funds and awareness for GOSH Charity, to help fund more pioneering research for children with rare and difficult-to-treat cancers.

Maris said: “When Frank was diagnosed, I felt like my legs were taken out from under me. Even when the leukaemia spread to his brain, we still had hope. We hoped there would be a miracle; something would click and Frank would get better.

“As a grieving parent, it’s incredibly important to me that I can see something is happening and progress is being made with children’s cancer research. People often ask me how I feel when I see some new research or treatment that could have saved Frank and they expect to me to say ‘why me’. While of course I wish there had been a treatment available for Frank at the time, I am so happy to see these treatments being developed. When I see another child pass away, I live through that same devastation as when we lost Frank. Whereas, when children get help and other parents don’t have to experience what we will go through for the rest of our lives, it’s the best feeling for me.

“I feel like I have an obligation to get people to understand the need for more research into children’s cancers, so that children don’t get left out in any way and can benefit from better treatments. Also, it makes me feel that I am still able to do something for Frank as he would want me to carry on his voice and seek help for others. That’s my mission.”

Not only will this new trial design potentially result in significant time-savings and cost, it will help ensure that children and adults have access to targeted therapies at the same time on the NHS. This will be a change from traditional clinical trial methods where adults take part first, which can mean that children don’t even get the opportunity to try these potential treatment options if the trial fails in adults. This is particularly pertinent as children and young people often tolerate CAR T-cell therapy more robustly than adults.

Dr Sara Ghorashian, Consultant Haematologist at GOSH and Honorary Associate Professor at UCL, said:

“Too often, children wait unacceptable amounts of time to access new cancer treatment which could make all the difference. This study will pave the way for research to always include children, and make it a matter of course that both children and adults are incorporated within clinical trials at the same time. Our goal is to never have a situation where children are left behind and we hope that we can prove the benefit of this novel approach to influence a change in the law in the future.”

Replacing outdated buildings on Great Ormond Street, our new Children’s Cancer Centre will mean that children with rare and complex cancers will receive care in the best possible environment, making it easier for them to be able to play, continue with school and participate in normal activities. The CCC will also enhance our ability to research and innovate to develop new and kinder treatments for cancer.

With significant outside space, a new hospital school, child centre inpatient wards and day care spaces, imaging, theatres and critical care services linked to the existing hospital, children and young people coming to GOSH will have access to the very latest technologies and receive care and treatment in environments that reflect their needs.

For more information visit Children’s Cancer Centre.

GOSH Charity is in the midst of its biggest ever fundraising appeal to help build the Children’s Cancer Centre, find out more about how you can be part of Build it Beat it.