A British teenager has made medical history by becoming the first person to survive a previously untreatable form of leukaemia thanks to an experimental gene-editing procedure. Alyssa Tapley, then 13, was given weeks to live after chemotherapy and a bone marrow transplant failed to halt her aggressive T-cell acute lymphoblastic leukaemia. Her life was saved by a pioneering CRISPR base editing trial conducted at Great Ormond Street Hospital (GOSH) in London.
Alyssa's ordeal began in 2021 when, after returning to school post-lockdown, she experienced extreme fatigue. Following an emergency admission for double pneumonia, she was diagnosed with T-cell leukaemia, a less common and often harder-to-treat form than B-cell leukaemia. Despite a month of chemotherapy at Leicester Royal Infirmary and a subsequent bone marrow transplant at Sheffield Children’s Hospital, her cancer persisted.
With conventional treatments exhausted, Alyssa's parents were told there were no further options, leaving them with little hope. However, a consultant from Sheffield learned of a trial led by Professor Waseem Qasim and Dr Robert Chiesa at GOSH. This innovative approach involved modifying CAR T-cells – immune cells engineered to fight cancer – using CRISPR base editing. Crucially, the cells were altered to prevent them from attacking each other, a significant challenge in treating T-cell leukaemia where traditional CAR T-cell therapy is ineffective.
Alyssa, despite her parents' initial reservations about her spending her final weeks in hospital, made the courageous decision to participate in the trial. She expressed a desire to contribute to medical advancement, even if it didn't save her own life. After two weeks of conditioning, she received the modified CAR T-cells. Her remarkable recovery has opened new doors for the potential treatment of similar aggressive cancers.
This groundbreaking procedure represents a significant leap forward in gene-editing technology and its application in oncology. The success of this trial could pave the way for wider clinical use of CRISPR base editing, offering a lifeline to patients with cancers that currently have no effective treatment options. The NHS regularly reviews new treatments and technologies, with bodies like the National Institute for Health and Care Excellence (NICE) assessing their clinical and cost-effectiveness for potential integration into standard care.