A British child with relapsed T cell leukaemia is recovering, in remission, after being given base-edited CAR-T cells in a world-first treatment, it has been announced.
UK researchers presented data for the first time at the American Society of Hematology annual meeting in New Orleans, USA. They told how Alyssa, a 13-year-old girl from Leicester, with T-cell acute lymphoblastic leukaemia (T-ALL), received the therapy in a ‘bench-to-bedside’ collaboration between UCL and Great Ormond Street Hospital for Children (GOSH).
After being diagnosed in 2021, Alyssa was treated with all current conventional therapies, including chemotherapy and a bone marrow transplant, but she relapsed and there were no further treatment options.
She was the first patient to be enrolled onto the TvT clinical trial and in May 2022 and was admitted to the Bone Marrow Transplant (BMT) Unit at GOSH, to receive ‘universal’ CAR T-cells that had been manufactured from a healthy volunteer donor.
The cells had been edited using new base-editing technology that had been designed and developed by a research team at UCL, led by Professor Waseem Qasim, who is also an honorary consultant at GOSH.
Alyssa was found to be in remission 28 days after treatment and received a second bone marrow transplant to restore her immune system. She is now recovering well and continues to have post-BMT follow-up at GOSH.
Prof Waseem Qasim, professor of cell and gene therapy at UCL GOS ICH and consultant immunologist at GOSH said: “We designed and developed the treatment from lab to clinic and are now trialling it on children from across the UK – in a unique bench to bedside approach.
“Alyssa’s story is a great demonstration of how, with expert teams and infrastructure, we can link cutting edge technologies in the lab with real results in the hospital for patients. It’s our most sophisticated cell engineering so far and paves the way for other new treatments and ultimately better futures for sick children.
“We have a unique and special environment here at GOSH and UCL that allows us to rapidly scale up new technologies and we’re looking forward to continuing our research and bringing it to the patients who need it most.”
Previous treatments have relied on techniques called TALENS or CRISPR/Cas9 to deliver changes to genes through cuts made by molecular ‘scissors’.
This new base editing approach acts without causing breaks in the DNA, allowing more edits, with fewer risks of unwanted effects on chromosomes, the researchers say.
The technique is also being investigated to try and correct harmful changes in DNA code for some inherited conditions.
Dr Robert Chiesa, consultant in bone marrow transplant and CAR T-cell therapy at GOSH, said: “Since Alyssa got sick with her leukaemia in May last year, she never achieved a complete remission, not with chemotherapy and not after her first bone marrow transplant.
“Only after she received her CD7 CAR-T cell therapy and a second bone marrow transplant in GOSH has she become leukaemia free.
“This is quite remarkable, although it is still a preliminary result, which needs to be monitored and confirmed over the next few months.”
Dr Louise Jones, head of translation at the Medical Research Council, which funded the project through its developmental pathway funding scheme, added: “If replicated, this ‘off the shelf’ universal cell therapy will mark a huge step forward in these types of therapies providing life-changing benefits to more patients.”
The TvT clinical trial for this treatment is currently open and aims to recruit up to 10 patients with T-cell leukaemia who have exhausted all conventional treatment options.
If it is widely successful, the Bone Marrow Transplant and CAR T-cell therapy teams at GOSH hope to offer the treatment earlier, when patients are less sick.
Source: ASH Conference December 2022
https://ash.confex.com/ash/2022/webprogram/Paper169114.html
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