These children experienced similar outcomes to the children treated with cells that were not frozen. Ten of the children in the study were treated at UCLA using a frozen preparation of corrected stem cells. Over 200 patients with various genetic conditions across the world have now been treated with this type of gene therapy – this is another very significant breakthrough.” Prof Adrian Thrasher, Professor of Paediatric Immunology at GOSH and Wellcome Principal Research Fellow, said “We started gene therapy at GOSH around 20 years ago and now have refined the process to offer a potential cure for children born with this debilitating condition. We need and want guidelines to change so we can start offering this potential cure to children and provide it as a first-choice treatment – this research could set those wheels in motion.” These corrected stem cells are then returned to the patient where, once engrafted, they go on to produce a seemingly continual supply of healthy immune cells capable of fighting infection.ĭr Claire Booth, co-lead author, Mahboubian Associate Professor in Gene Therapy and GOSH consultant in Paediatric Immunology and Gene Therapy: “If approved in the future, this treatment could be standard for ADA-SCID, and potentially many other genetic conditions, removing the need to find a matched-donor for bone marrow transplant and the toxic side effects often associated with that treatment. Next, a viral vector is used to deliver a new copy of the ADA gene into the DNA of the patient's cells. The gene therapy approach described in the paper involves first removing some of the patient's blood-forming stem cells, which create all types of blood and immune cells. Now, lentiviral gene therapy technology has advanced significantly and offers the potential to improve the safety and efficacy profile of this treatment option for patients. In earlier trials of gamma-retroviral gene therapy for other similar diseases, serious side effects including leukaemia have been seen in some patients. Everything had to be highly sterilised to keep her safe Sarah's mum, Maria. I wasn’t allowed to kiss my daughter or sleep next to her. “I now know how it feels to have your child as a patient and to be so sick, and I want to use my experience to help others in a similar position.” Sarah’s treatment has had a huge impact on the whole family, but it also inspired Maria to go back to studying and train to become a nurse. She’s doing so well now and can do everything a normal child her age would do.” Six months after she was given the gene therapy her skin was clean and healthy and the other symptoms got a lot better. “Before, her nappy rash was always bleeding and sore, she was being sick and losing weight. Sarah was referred to GOSH for gene therapy and several years after her treatment is doing very well: It was horrible to not be able to do normal things with my own daughter.” Everything had to be highly sterilised to keep her safe. I saw it was SCID and I was incredibly upset… I wasn’t allowed to kiss my daughter or sleep next to her. “I remember the exact time I saw the paper that had the blood test results on and the diagnosis. She was less than ten days old when her mum Maria became concerned about her daughter’s weight loss and sore, bleeding nappy rash that wouldn’t heal. One of the patients treated was four-year-old Sarah from South Yorkshire. In the two cases in which treatment wasn’t successful, both children were able to return to current standard treatments, with one eventually receiving a bone marrow transplant.No serious side effects have so far been reported, with generally mild or moderate complications experienced from the necessary preparation for the gene therapy. Of these, 48 are no longer showing symptoms of ADA-SCID, although they will have lifelong monitoring. Two to three years after the treatment, all of the 50 children treated with the new gene therapy at Great Ormond Street Hospital, UCLA Mattel Children’s Hospital and the National Institutes of Health (NIH) are alive and well. If approved, gene therapy would be a welcome new treatment option for ADA-SCID as it is a one-time procedure that has the potential to provide life-long results. These treatments are expensive and therefore out of reach for patients in many countries. If a matched bone marrow donor is not available, patients require lifelong ADA injections along with preventative medicines. The standard treatment for ADA-SCID involves once or twice weekly injections of the ADA enzyme until a matched bone marrow donor – usually a close family member – can be found. In a new study published in the New England Journal of Medicine, co-lead authors Professor Donald Kohn (UCLA) and Dr Claire Booth (GOSH) reported the two- to three-year outcomes of 50 children who were treated in clinical trials with an experimental stem cell gene therapy for ADA-SCID between 20.
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