Published: 15 December 2022
A teenage girl's incurable cancer has been cleared from her body using genetically modified immune cells. It is the first time this form of the treatment has been used in a patient, thanks to research supported by the NIHR.
Alyssa, 13, from Leicester, became the first reported patient in the world to receive genetically modified immune cells called CAR T-cells. The cells originally came from a healthy donor and were used to treat her T cell leukaemia, which had not responded to existing treatments.
Alyssa was diagnosed with T-cell acute lymphoblastic leukaemia (T-ALL) in 2021 and received all current conventional treatments for her cancer including chemotherapy and a bone marrow transplant. But her disease returned and there were no further treatment options available as part of routine care.
CAR T-cell treatment normally uses a patient’s own T-cells (a type of immune cell) to fight cancer by changing them in the lab so that they can find and destroy cancer cells. But because Alyssa’s cancer was caused by rogue T-cells, the technique could not work in the usual way. T-cells designed to recognise and attack cancerous T-cells also end up killing each other during the manufacturing process before they can be given as a treatment.
New CAR T-cell cancer treatment
Research teams have been working to create a new type of CAR T-cell therapy that can attack cancerous T-cells. They use a method called base editing to change single letters of the DNA code in the T-cells.
The result is that the edited CAR T-cells can be given to the patient so that they find and destroy cancerous T-cells in the body. If the treatment is successful, the patient then receives a bone marrow transplant to restore their immune system.
In May 2022, Alyssa was the first patient to join a new clinical trial at Great Ormond Street Hospital (GOSH). She was treated with T-cells from a healthy donor, which had been edited using the new technology. 28 days later, she was in remission and went on to receive a second bone marrow transplant to restore her immune system. Six months after the bone marrow transplant, she is now doing well at home recovering with her family and continues her follow-up at GOSH.
NIHR senior investigator Professor Waseem Qasim was part of the team that developed the base-editing therapy. He has received NIHR funding for related work and is part of NIHR GOSH Biomedical Research Centre.
He said: “This is a great demonstration of how, with expert teams and infrastructure, we can link cutting edge technologies in the lab with real results in the hospital for patients.
“It’s our most sophisticated cell engineering so far and paves the way for other new treatments and ultimately better futures for sick children."