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

Researchers at Great Ormond Street Hospital (GOSH) and University College London (UCL) 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 Great Ormond Street Hospital Charity (GOSH Charity), Dr Sara Ghorashian, a Consultant Haematologist at GOSH, and her team from GOSH and the UCL Great Ormond Street Institute of Child Health, 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%. This paediatric study will open at both GOSH and University College London Hospital (UCLH), meaning that children aged up to 18 will have the opportunity to be involved.

As well as potentially providing a new treatment option for children and young people with relapsed T-ALL, this paediatric clinical trial is also adopting a novel approach which will see it running alongside an adult’s clinical trial (taking place at 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. Although cancer is the biggest killer of children aged 1-14 in the UK, 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 1 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.

Not only will this new trial design potentially result in significant cost and time-savings, it will help ensure that children and adults have access to targeted therapies at the same time on the NHS. This is a step 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.

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. But there comes a point when you can no longer treat the child if it means they’ll have no quality of life. Every parent wants to have their child no matter what, and it is the most difficult thing in the world to let go, but you also don’t want them to suffer and have to let nature take its course.

“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.”

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 paediatric and adult patients 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.”

GOSH Charity is the UK’s largest dedicated charitable funder of child health and has just launched its first ever cancer research strategy, pledging a minimum of £15million in this area to help develop new treatments and cures, improve the experiences and quality of life for patients and families through research, and support infrastructure and staff careers to create a thriving research culture.

Improving the lives of children with cancer is at the centre of GOSH Charity’s biggest ever fundraising appeal, to help build a world-leading new Children’s Cancer Centre at GOSH and transform children’s cancer care. Designed with the needs of children and families at its heart, the Children’s Cancer Centre will support every aspect of care for children and their families, from diagnosis to remission, and help to offer opportunities for children to participate in research.

Dr Aoife Regan, GOSH Charity’s Director of Impact and Charitable Funding, said: “In line with our new cancer research strategy, we are really proud to be funding this important study which will hopefully, not only provide a potential new treatment option for children with a particularly aggressive form of cancer, but also lay the foundations for a new, more efficient clinical trial design that would benefit even more seriously ill children in the future.

“At GOSH Charity, we strive to give seriously ill children the best chance and the best childhood possible and are currently in the midst of the most ambitious appeal in our history to help build a new Children’s Cancer Centre at GOSH. This game-changing new facility will help enable pioneering research to take place to deliver new breakthrough therapies for children who desperately need them.”

Find out more about the Children’s Cancer Centre at GOSH and donate to the Build it. Beat it. appeal.

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With the required funding in place, this FRACTALL clinical 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.

For any media enquiries or more information, please contact:

Grace Waters, GOSH Charity press office – grace.waters@gosh.org / 020 3841 3237

About Great Ormond Street Hospital Charity (GOSH Charity)

Great Ormond Street Hospital Charity (GOSH Charity) stops at nothing to help give seriously ill children childhoods that are fuller, funner and longer. For the hundreds of children from all over the UK who are treated by Great Ormond Street Hospital (GOSH) every day, for children with rare or complex illnesses everywhere, for this generation and all those to come.

GOSH has been transforming the lives of seriously ill children since opening its doors in 1852 and has always depended on charitable support. GOSH Charity funds groundbreaking research into children’s health, cutting-edge medical equipment, child-centred medical facilities and the support services children and families going through the toughest journey of their lives urgently need. But there is so much more we need to do.

Together with our supporters, we can give seriously ill children the best chance, and the best childhoods, possible. Because we believe no childhood should be lost to serious illness.

Join us, visit gosh.org today.

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