A study coordinated by the CR UK & UCL Cancer Trials Centre has shown very promising results for children with previously incurable acute lymphoblastic leukaemia (ALL).
The CARPALL study, which tests a new kind of CAR T-cell therapy, is led by Professor Persis Amrolia and Dr Sara Ghorashian at Great Ormond Street Hospital (GOSH) and the UCL Great Ormond Street Institute of Child Health. Taking part are patients from GOSH, University College Hospital London and the Royal Manchester Children's Hospital.
The CAR T-cell therapy uses a patient’s own immune cells (T cells), altering them to recognise a protein on the surface of the leukaemia cells which the T cells then attack. The aim of the study is to see whether the treatment is safe, whether it can cure a patient’s disease, and whether it can prevent the disease from coming back. Results published in Nature Medicine show that 12 out of 14 participants with ALL cleared their disease after three months, while 5 patients remain leukaemia-free after receiving the CAR T-cell treatment.
Lead author Sara Ghorashian said: “The safety profile emerging from this paediatric study is encouraging. It is very promising to see these strong remission rates and excellent Car-T cell expansion and persistence.
Professor Amrolia added: ‘CAR-T therapy is a fantastic example of using the power of the immune system to specifically target cancer cells. We’re just at the beginning of this new treatment and over the next few years I hope we can refine it further.’
The results garnered nationwide press attention (see below). You can read the article in Nature Medicine here.
The study was funded by Great Ormond Street Hospital Children’s Charity, Children with Cancer UK and the J P Moulton Charitable Foundation. The experimental CAR T-cell therapy, known as CAT-19 or AUTO1, was developed collaboratively by both UCL Cancer Institute and UCL Great Ormond Street Institute of Child Health, with support from Autolus Therapeutics, and is being commercialised by Autolus. Autolus is a public biotechnology company that was spun out from UCL in 2014.
Press coverage for CARPALL
Pictured: Professor Peris Amrolia, Dr Sara Ghorashian