First leukaemia patient to undergo ‘innovative’ treatment living healthy life, pioneer doctor says

The trial involved editing healthy immune cells to fight the disease and was trialled by adults and children with a rare and aggressive form of the disease.

Alyssa Tapley, now 16, became the first patient with an aggressive form of leukaemia to have the experimental treatment in 2022 and is now leading a healthy life.

Nine children and two adults were treated as part of the study at Great Ormond Street Hospital and King’s College Hospital, with some 64 per cent remaining disease-free.

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Almost two thirds of patients with T-cell acute lymphoblastic leukaemia (T-ALL) involved in a clinical trial of the treatment – known as BE-CAR7 – now remain disease-free.

BE-CAR7, which was developed by scientists at Great Ormond Street Hospital (GOSH) and University College London (UCL), is a highly advanced version of an immunotherapy known as CAR T-cell treatments.

Speaking to Charlotte Lynch on LBC News, Professor Waseem Qasim said: “It is a breakthrough in demonstrating the power and the potential of some of the new technologies that are coming through.

“So what we’ve done here is, instead of trying to. Encourage a patient’s own immune system to do the job, we’ve realised some of these patients have been through lots of chemotherapy.

“The immune system’s quite weak. Can we take cells from a healthy donor and engineer them in such a way that they’re going to hunt down the remaining leukaemia cells but not cause damage to any of the normal tissues?

“And can they do that in a way that’s going to allow us then to seek out and stir up any leukaemia that’s left behind in the system, whether it’s in the blood or the bone marrow, sometimes in the brain fluid compartment.

“So it’s quite a big ask for the treatments and we’ve only recently been able to do this because the engineering steps required have only really been developed in the last few years.”

Speaking about Alyssa, Mr Qasim, professor of Cell and Gene Therapy at University College London and honorary consultant immunologist at Great Ormond Street Hospital, said “[She] was 13 when she came to us from a hospital in Leicester where she’d already had beaten, including a transplant, and her leukaemia had come back.

“Normally in that situation, the kind of statistics we’re looking at is probably a very low chance of the disease then going away and children really, or young adults being offered symptom care and palliation.

“In that situation we had approval from an ethics committee and the trial was ready to start and she was in fact the first patient that was treated back in 2022.

“She’s now three years post the treatment and I have to say the treatment’s already quite intense. What we’re doing is we’re using the engineered cells to clear out the leukaemia, but in that process we are also dismantling the entire immune compartment, which then has to be rebuilt from a bone marrow transplant.

“So it’s a very intensive treatment. There’s risks and side effects associated with the various stages. But she’s done really well and she’s now three years after the intervention and is basically living an unrestricted lifestyle.”

CAR T-cell therapy typically involves a doctor collecting T cells from a patient which are then modified in a lab with proteins called chimeric antigen receptors (CARs) placed on the surface to recognise and kill cancer.

The immune cells are then placed back into the patient’s bloodstream using a drip.

However, developing CAR T-cell therapy for leukaemia stemmed from abnormal T cells has been challenging.

A clinical trial explored whether BE-CAR7 could clear leukaemia ahead of a planned bone marrow transplant in the hopes it could stop the cancer returning.

Nine children and two adults with T-ALL were treated as part of the study at Gosh and King’s College Hospital.

Now, some 64 per cent remain disease-free, with the first patients off treatment three years on.

More than eight in ten achieved deep remission and were able to get a stem cell transplant without disease. Side effects, including low blood count and rashes, were tolerable, according to researchers.

Waseem Qasim, a professor of cell and gene therapy at UCL and honorary consultant immunologist at Gosh, said: “We previously showed promising results using precision genome editing for children with aggressive blood cancer and this larger number of patients confirms the impact of this type of treatment.

“We’ve shown that universal or ‘off-the-shelf’ base-edited CAR T-cells can seek and destroy very resistant cases of CD7+ leukaemia.”

BE-CAR7 was first administered to Alyssa Tapley, then 13, from Leicester in 2022. She was diagnosed with T-cell leukaemia in May 2021 and did not respond to standard therapies.

Alyssa, who is now 16, said: “I chose to take part in the research as I felt that, even if it didn’t work for me, it could help others.

“Years later, we know it worked and I’m doing really well.”

Alyssa now has hopes of becoming a research scientist.

“I’ve done all those things that you’re supposed to do when you’re a teenager,” she said.

“I’ve gone sailing, spent time away from home doing my Duke of Edinburgh Award, but even just going to school is something I dreamed of when I was ill.

“I’m not taking anything for granted. Next on my list is learning to drive, but my ultimate goal is to become a research scientist and be part of the next big discovery that can help people like me.”

Dr Deborah Yallop, consultant haematologist at King’s College Hospital, said “We’ve seen impressive responses in clearing leukaemia that seemed incurable – it’s a very powerful approach.”

The results of the clinical trial have been published in the New England Journal of Medicine and presented at the 67th American Society of Hematology Annual Meeting in Florida.

Reacting to the findings, Dr Tania Dexter, senior medical officer at stem cell charity Anthony Nolan, said: “The results of the study are promising, with most patients achieving levels of remission allowing them to receive a stem cell transplant.

“Considering these patients had a low chance of survival before the trial, these results bring hope that treatments like this will continue to advance and become available to more patients.”