Children with refractory leukemia can be treated using new gene editing techniques

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  • Researchers at UCL and Great Ormond Street Hospital for Children (GOSH) have developed donor T cells to treat critically ill children with drug-resistant leukemia who would otherwise have exhausted all available treatments.

The phase I study, published in Science Translational Medicine, is the first human application of ‘universal’ CRISPR-edited cells and represents an important advance in the use of gene-edited cells to treat cancer.

Researchers, led by Professor Waseem Qasim, UCL Great Ormond Street Institute of Child Health & Consultant Immunologist at GOSH, used a technique known as CRISPR/Cas9 to cut a cell’s DNA and insert new genetic code. In this case, the genetic code allows T cells to express a receptor called a chimeric antigen receptor (CAR) that can recognize and destroy markers on the surface of cancerous B cells.

The T cells were then further gene-edited and made available “off-the-shelf” without the need for donor matching. “Fortunately, this type of unresponsive leukemia is extremely rare, but we are excited to introduce new treatments for some of the most difficult childhood leukemias to treat,” said lead author Professor Qasim. I’m exhausted.”

The study builds on previous work by Professor Qasim’s team using older techniques.

“While there are challenges to overcome, this study demonstrates how novel genome-editing techniques can be used to address the unmet health needs of the sickest children we see. It’s a promising demonstration.”

A 1-year-old patient who had leukemia cleared from his body using CAR-T cell therapy in 2015. Here, white blood cells were harvested from a healthy donor and used “molecular scissors” called TALENS (precursor of CRISPR). and processed in a special lab to create cells that are suitable for transplantation into every patient so that leukemia can be prevented and eliminated.

Following the success of this experimental treatment, a larger study reported on her 21 patients, both treated adults and children. Several CAR T-cell therapies are currently offered by the NHS, but they rely on harvesting and generating the patient’s own cells. This can be expensive and is not always quick to do.

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