Groundbreaking gene therapy helps fight aggressive cancer

Patients with a previously incurable blood cancer are benefiting from pioneering ethical gene immunotherapy.

In a clinical trial by researchers at University College London and Great Ormond Street Hospital (GOSH), seven out of eleven participants with T-cell acute lymphoblastic leukaemia remain disease-free after receiving the ‘base editing’ treatment BE-CAR7.

Scientists modified a type of white blood cell (T-cell) from a donor. When given to the patient, the edited T-cells (BE-CAR7) ‘seek and destroy’ the body’s T-cells, clearing it of leukaemia.

Strength to strength

Alyssa, now 16 years-old, was the first patient in the world to receive the cutting-edge treatment in 2022.

Prior to joining the trial, she and her family were discussing the possibility of palliative care, but three years on Alyssa has been discharged to long-term follow up and is going from strength to strength.

Reflecting on her experience, she said: “I really did think that I was going to die and I wouldn’t be able to grow up and then do all the things that every child deserves to be able to do.”

She told BBC News: “I’m looking into doing an apprenticeship in biomedical science, and hopefully one day I’ll go into blood cancer research as well.”

Hope

Team leader Professor Waseem Qasim explained: “A few years ago this would have been science fiction.

“Now we can take white blood cells from a healthy donor and change a single letter of DNA code in those cells and give them back to patients to try to tackle this hard-to-treat leukaemia.”

Dr Rob Chiesa, study investigator and Bone Marrow Transplant consultant at GOSH said: “Although most children with T-cell leukaemia will respond well to standard treatments, around 20% may not.

“It’s these patients who desperately need better options and this research provides hope for a better prognosis for everyone diagnosed with this rare but aggressive form of blood cancer.”