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A groundbreaking therapy once regarded as science fiction has now succeeded in reversing aggressive, previously incurable blood cancers in some patients, according to doctors involved in the treatment. This innovative approach uses precise DNA editing in white blood cells to create a “living drug” capable of fighting cancer within the body.
The treatment was first administered to a young girl whose story was covered in 2022; she remains free of the disease and aspires to pursue a career in cancer research. Since then, an additional eight children and two adults diagnosed with T-cell acute lymphoblastic leukaemia (T-ALL) have undergone the therapy. Results show that nearly two-thirds of these patients have achieved remission.
Ordinarily, T-cells function as defenders of the body, identifying and destroying harmful invaders. However, in T-ALL, these cells multiply uncontrollably, causing the disease. For many individuals enrolled in the trial, conventional treatments like chemotherapy and bone marrow transplants had failed, leaving comfort care as the only previous option. Sixteen-year-old Alyssa Tapley from Leicester, who was the very first recipient of this treatment at Great Ormond Street Hospital, shared her experience: “I really did think that I was going to die and I wouldn’t be able to grow up and do all the things that every child deserves to be able to do.” Now, Alyssa is embracing life fully.
Three years ago, Alyssa underwent a transformative procedure involving the eradication of her old immune system followed by the growth of a new one. This process required a four-month hospital stay, during which she was not allowed contact with her brother to minimize infection risk. Following treatment, her cancer is now undetectable, and she only needs to attend annual check-ups. Beyond her health recovery, Alyssa is engaged in her A-level studies, the Duke of Edinburgh Award program, and is considering driving lessons. She expressed her ambitions clearly: “I’m looking into doing an apprenticeship in biomedical science, and hopefully one day I’ll go into blood cancer research as well.”
The research, led by teams at University College London (UCL) and Great Ormond Street Hospital, utilizes a technique known as base editing. This process involves altering the fundamental units of genetic code—adenine (A), cytosine (C), guanine (G), and thymine (T)—which together compose the DNA instructions for the body. By precisely targeting and converting one base into another, scientists can rewrite genetic instructions to modify cell behavior. In this instance, they aimed to empower healthy T-cells to identify and eliminate the cancerous T-cells causing leukaemia.
Achieving this complex goal required meticulous genetic modification to ensure the engineered T-cells could attack malignant cells without annihilating themselves. Initially, healthy donor T-cells were altered through multiple steps: disabling their ability to attack the patient’s own body, removing a key chemical marker called CD7 to prevent the therapy from destroying itself, applying an “invisibility cloak” to shield the cells from chemotherapy drugs, and finally programming the cells to seek out any T-cells bearing the CD7 marker. This design allows the modified T-cells to eliminate both healthy and cancerous T-cells carrying CD7 while sparing each other. Patients then receive an infusion of these engineered cells. If no cancer is detected after four weeks, they undergo a bone marrow transplant to rebuild a healthy immune system.
Professor Waseem Qasim from UCL and Great Ormond Street Hospital highlighted the significance of this advancement: “A few years ago, this would have been science fiction,” he remarked. “We have to basically dismantle the entire immune system. It’s a deep, intensive treatment, it’s very demanding on the patients, but when it works, it’s worked very well.”
A study published in the New England Journal of Medicine reports on the first 11 patients treated at Great Ormond Street and King’s College Hospital. Nine of these individuals achieved deep remission, making them eligible for bone marrow transplantation. Seven patients remain disease-free, with follow-up periods ranging from three months up to three years. While risks such as infections arise due to immune system depletion, the therapy shows great promise. In two cases, the cancer cells lost their CD7 markers, enabling them to evade treatment and causing the disease to relapse. Dr. Robert Chiesa from Great Ormond Street’s bone marrow transplant department commented on the outcomes: “Given how aggressive this particular form of leukaemia is, these are quite striking clinical results, and obviously, I’m very happy that we managed to offer hope to patients that otherwise have lost it.” Dr. Deborah Yallop, haematologist at King’s, added, “We’ve seen impressive responses in clearing leukaemia that seemed incurable – it’s a very powerful approach.” Similarly, Dr. Tania Dexter from the stem cell charity Anthony Nolan noted the encouraging nature of the findings for patients with previously low survival odds, seeing the treatment as a hopeful step toward broader future availability
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