A groundbreaking gene-editing therapy is offering new hope to patients with aggressive T-cell acute lymphoblastic leukaemia, a cancer once considered almost impossible to treat. Doctors in the UK report that a cutting-edge treatment, which rewrites DNA inside white blood cells, has successfully reversed the disease in several patients.
The therapy transforms healthy donor T-cells into a “living drug” capable of targeting and destroying cancerous T-cells. What once seemed like science fiction is now saving lives.
Sixteen-year-old Alyssa Tapley from Leicester became the first person in the world to receive this innovative treatment in 2022. Before the trial, she had exhausted all other treatment options, including chemotherapy and bone marrow transplants. Today, she remains cancer-free and is planning a future in biomedical science.
Doctors at University College London and Great Ormond Street Hospital used a revolutionary technique called base editing. This method allows scientists to zoom into a specific part of DNA and alter a single genetic “letter,” enabling precise and powerful changes without damaging the rest of the genome.
To build this highly specialized therapy, researchers began with healthy donor T-cells and applied four critical DNA edits.
First, they switched off the cells’ natural targeting system to prevent them from harming the patient.
Second, they removed the CD7 marker found on all T-cells, ensuring the treatment would not destroy itself.
A third edit created resistance against a chemotherapy drug, allowing the modified cells to survive the treatment process.
Once infused, the edited cells aggressively seek out and destroy harmful T-cells. Out of ten additional patients, eight children and two adults, nearly two-thirds reached remission.
With Alyssa now enjoying school, preparing for her driving lessons, and dreaming of becoming a cancer researcher, this breakthrough marks a transformative moment for patients fighting one of the toughest blood cancers.
