On Wednesday 25 January, details of two patients successfully treated for their leukaemia at Great Ormond Street Hospital using gene-editing technology were published in the journal, Science Translational Medicine.
One of the patients was Layla Richards who was the first person in the world to receive gene-edited immune cells to treat drug resistant leukaemia. Her story received widespread national and international media coverage.
Commenting on the publication, Professor Waseem Qasim, Professor of Cell and Gene Therapy at UCL Great Ormond Street Institute of Child Health (ICH), Consultant Immunologist at Great Ormond Street Hospital and who carried out the work, said:
“We are delighted to have shown that gene-edited cells can successfully treat leukaemia that has not responded to standard treatments.
“While both patients are now at home and are doing well, we must treat these results with some caution as we don’t yet know if the technique will be successful in treating a larger number of patients. Clinical trials are currently underway to assess this.”
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Notes to Editors
Great Ormond Street Hospital for Children NHS Trust is the country’s leading centre for treating sick children, with the widest range of specialists under one roof.
With the UCL Great Ormond Street Institute of Child Health, we are the largest centre for paediatric research outside the US and play a key role in training children’s health specialists for the future.
Great Ormond Street Hospital Children’s Charity needs to raise money to support the hospital to give children who need help the most the best chance for life. The charity funds patient and family support programmes, provides the latest medical equipment and supports the essential redevelopment of the hospital. It has also launched a five-year strategy to support research in some of the most serious and complex childhood diseases. Great Ormond Street Hospital Children’s Charity is the largest dedicated funder of paediatric research in the UK and our work is entirely funded through the generosity of supporter donations. For more information visit www.gosh.org