Children with aggressive blood cancer received treatment and hope for a cure

Children with aggressive blood cancer could benefit from a new treatment launched in Great Ormond Street which experts hope will cure the disease.

Young people with T-cell acute lymphoblastic leukemia (T-ALL) will be offered CAR T-cell therapy targeting a specific protein, which was recently identified by researchers as occurring only in cancerous white blood cells.

CAR T-cell therapy involves genetically modifying the body’s immune cells to recognize and kill cancer cells and has demonstrated success in other types of cancer, but not in T-ALL.

But experts now believe they have a much clearer target for attacking this deadly cancer because they have identified the CCR9 protein on cancerous T cells (an important type of white blood cell).

As part of a new study into the treatment, Dr Sara Ghorashian, consultant haematologist at Great Ormond Street Hospital (GOSH), and her team at GOSH and University College London’s Great Ormond Street Institute of Child Health, are recruiting 12 children .

All have relapsed T-ALL or are resistant to conventional treatments, such as chemotherapy and bone marrow transplantation.

The study will open around April next year at GOSH and University College London Hospital (UCLH), with the CAR T-cell therapy, which is administered by infusion, created at UCL.

The Great Ormond Street Hospital Charity donated £2.4 million to support the study.

Furthermore, in a radical change, the research will also test a new approach to clinical trials, carrying out a study with children and adults at the same time.

According to GOSH, research projects dedicated to childhood cancer are rare, with pediatric clinical trials traditionally starting much later than those carried out in adults and taking on average 6.5 years longer to complete.

The new method could save time and money and would help ensure children and adults can access targeted therapies on the NHS at the same time, rather than children being forced to wait.

Researchers believe the therapy can work – it has been shown to eliminate leukemia in laboratory and animal studies – and it is known that children tend to tolerate CAR T-cell therapy better than adults.

Although cancer is the biggest cause of death in children aged 14 and under in the UK, only 12 anti-cancer medicines have been authorized specifically for pediatric cancer in the last decade, compared with more than 150 for adult cancers, according to the GOSH.

Dr Ghorashian told the PA news agency there was a “perception that there shouldn’t be early phase clinical trials in paediatrics” but that this created problems for children accessing new medicines and therapies.

She added: “Often things like the specifics of how a medicine is administered are just borrowed from adults and not necessarily adapted for children.

“We don’t have as many new medicines for childhood cancer as we do for adult cancer, and medicines reach children later.

“They come in doses that are borrowed from adults, and what we should be doing is testing them in parallel – making sure they are safe in the first place – but not preventing access to effective treatments for children.”

Ghorashian said no CAR T cell drug has been effective for T-ALL to date. But it has shown success in another type of leukemia called B-cell acute lymphoblastic leukemia.

She said: “Our target, the CCR9 target, is only present in a very, very small proportion – about one to 2% of T cells in total – which means that when we treat with CAR T cell therapy, we hope that we will gain We will not be eliminating healthy T cells in significant numbers.

“This means that the patient’s leukemia will be eliminated if we are lucky, but the patient’s healthy T cells will remain and be able to continue to fight the infection.”

After treatment, study patients would not need a bone marrow transplant to replace healthy T cells that were killed by CAR T.

“If we can offer a CAR T cell therapy that does not need to be consolidated with a salvage transplant, that would be beneficial,” said Dr. Ghorashian.

“That’s why we’re particularly excited to test this and see if it will be effective.”

Although CAR T cell therapy can cause side effects, these are generally manageable compared to the long-term effects of chemotherapy and bone marrow transplant.

Dr. Ghorashian hopes the trial will help set a precedent for future studies in children and adults, adding: “Often, children wait unacceptable periods of time to access a new cancer treatment, which can make all the difference.

“This study will pave the way for research to always include children and will make it natural for both pediatric and adult patients to be included in clinical trials at the same time.

“Our goal is to never have a situation where children are left behind and we hope to be able to prove the benefit of this new approach to influencing a change in the law in the future.”

Maris Hanson, 47, lost her son Frank to T-ALL in 2019, just after his second birthday.

Frank Hanson died of T-cell acute lymphoblastic leukemia (T-ALL) in 2019, just after his second birthday. Brochure

Ms Hanson, who is legal and people director at Giant Group, is now raising funds and awareness for GOSH Charity.

She told PA: “Frank was an incredibly happy child, very curious and always looking for new experiences. He was very active as a boy, he always wanted to be outside.”

Frank fell ill before his second birthday in January 2019 when his parents noticed a persistent type of infection with a low temperature.

He recovered but became very ill on a flight to Estonia and was flown back to the UK on a medical plane for treatment at GOSH.

Mrs Hanson said: “When Frank was diagnosed I felt like my legs had been ripped off.

“Even when the leukemia spread to the brain, we still had hope. We hoped there would be a miracle; something would click and Frank would improve.

“But there comes a point where you can no longer treat the child if it means they won’t have quality of life…

“After three and a half months, it was Mother’s Day weekend, we were basically told ‘there’s nothing more we can do because… his form of leukemia is unresponsive’.

“So in four months, this journey has basically ended for us in a way that I would never want any other parent to go through, let alone the child themselves, because it is cruel.”

Mrs Hanson, who lives in west London, said she was determined to raise awareness about clinical trials for children.

“I was in a situation where I lost my son,” she said. “And I thought: what can I do to, in some way, benefit him too? This is one way I can continue to live my life.”

GOSH Charity is holding its biggest fundraising appeal to build a new Children’s Cancer Center on the site.

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