Researchers at Great Ormond Street Children’s Hospital (GOSH) and Great Ormond Street Institute of Child Health (UCL GOSH ICH) have used a new technology to engineer T cells to treat seriously ill children.

The first phase of the trial, published in Science Translational Medicine, is said to be “the first use of ‘universal’ CRISPR-edited cells in humans and represents a significant step forward in the use of gene-edited cells to treat cancer.”

The CRISPR technique involves making a cut in a cell’s DNA, allowing researchers to insert brand new genetic code. Researchers can then allow the T cells to express a receptor called a chimeric antigen receptor (CAR), which can recognize the makers of the cancer cells and destroy them.

GOSH have noted that although a number of CAR T-cell therapies are now provided by the NHS, they rely on harvesting and engineering the patient’s own cells, which is expensive and not always possible in a short time frame. In this trial, GOSH describes how their team “produced their banks of donor CAR T cells using a single inactivated virus to transfer both the CAR and a CRISPR targeting system, and then applied cutting-edge mRNA technology to activate the steps to edit gen. “

The CRISPR technique was then further used to edit T cells so that they could be used without the need for a donor match. Researchers are investigating how donated cells can be pre-produced and used in multiple patients to lower costs and make treatment more affordable.

Speaking about the impact of this new research, Dr Kanchan Rao, Consultant Bone Marrow Transplant at GOSH said: ‚ÄúThis study adds to the growing body of evidence that genome-edited T cells may be a viable alternative to currently available treatments. “

The study included six children aged 14 months to 11 years who had relapsed; all children had previously received standard treatment for B-ALL in the UK, but their disease had relapsed multiple times. They were given the edited cells by intravenous infusion, and the treatment remained active for about four weeks. If successful, patients are eligible for a bone marrow stem cell transplant. Four of the first six children went into remission within 28 days, and two of these children remained in ongoing remission nine and 18 months after treatment, respectively.

The study’s lead author, Professor Waseem Qasim, spoke about the future of the trials as follows: “Although there are challenges to overcome, this study is a promising demonstration of how emerging genome editing technologies can be used to address unmet health needs in some of the sickest children we see.’

GOSH researchers use technology to engineer donor T-cells for children with resistant leukaemia

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