England (Commonwealth Union) – Gene editing technology continues to make advancements in plant, veterinary and medical biotechnology. Scientists at the University College London (UCL) and Great Ormond Street Hospital for Children (GOSH) have engineered donor T-cells in an attempt to treat seriously ill children having resistant leukaemia, who had otherwise used up all available therapies.
The Phase I trial is the first application of “universal” CRISPR-edited cells in humans, making a huge leap forward in the application of gene-edited cells for cancer treatment.
The team, led by Professor Waseem Qasim of the UCL Great Ormond Street Institute of Child Health & Consultant Immunologist at GOSH, used CRISPR/Cas9, which makes an incision in the cells’ DNA by inserting a new genetic code. The genetic code permits the T-cells to express a receptor known as chimeric antigen reception (CAR), able to recognize a marker on the surface of cancerous B-cells and eliminate them. The T-cells had to undergo more gene editing so that they can be applied “of the shelf” with no requirement of donor matching.
Professor Qasim, stated that it was a good thing that this kind of unresponsive leukaemia is very rare, but they are pleased to be capable of bringing in new therapies for some of the most difficult to treat childhood leukaemias, especially when all other options have failed. “Whilst there are challenges to overcome, this study is a promising demonstration of how emerging genome-editing technologies can be used to tackle unmet health needs in some of the sickest children we see,” he said.
While several CAR T-cell therapies are currently available, they depend on gathering and engineering a patient’s own cells, which is costly and does not always work in a short period of time. As a result, researchers are currently evaluating genome editing to permit donated cells to be pre-manufactured and utilized in multiple patients, hoping to make it more economical and have treatments more accessible.
