Further hope for base-edited T-cell therapy to treat resistant leukemia uclnews
Three young patients with relapsed T-cell leukemia have now been treated with base-edited T-cells, as part of a"bench-to-bedside" collaboration between University College London and Great Ormond Street Hospital for Children ., shows how donor CAR T cells were engineered using cutting-edge gene-editing technology to change single letters of their DNA code so they could fight leukemia.
This first human application of base-editing technology was designed and developed by a team of researchers at UCL, led by Professor Waseem Qasim , working with Dr. Robert Chiesa and the Bone Marrow Transplant/CART/Haematology teams at GOSH. To generate banks of"universal" anti T-cell CAR T-cells for the study, the researchers used healthy donor T-cells. They then made changes to the cells using"base editing," which works by chemically converting single nucleotide bases which carry instructions for a specific protein, in order to prevent them being produced.Removing existing receptors so that T-cells from a donor could be banked and used without matching—making them"universal.
Adding a Chimeric Antigen Receptor that recognized the CD7 T-cell receptor on leukemic T-cells. The cells became armed against CD7, and recognized and fought T-cell leukemia. If shown to be widely successful, the teams hope that it can be offered to more children and earlier in their treatment journey when they are less sick. With additional funding, they also hope to make it available for adults in the future.
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