Media release

From: Springer Nature

Long-term remission in patient following CAR-T therapy

A patient has achieved over 18 years of remission from neuroblastoma, a type of nerve cell cancer, following treatment with chimeric antigen receptor (CAR) T cell therapy and without requiring any additional treatments. The findings are published in Nature Medicine. This may represent the longest reported remission in a patient with cancer treated with CAR-T cell therapy to date, the authors suggest. The findings are based on follow-up from a phase 1 clinical trial that tested an engineered CAR-T cell therapy in children with neuroblastoma.

CAR-T cell therapy is a type of treatment that modifies a patient’s T cells — a type of white blood cell that is a part of the immune system — to specifically recognize and kill cancer cells. This therapy has been approved for the treatment of patients with some blood cancer types, such as leukaemia and lymphoma, but has been less effective in patients with solid tumours. Neuroblastoma is a type of rare solid tumour cancer that typically occurs in children and is a challenging disease to treat, with high relapse rates despite therapy.

Helen Heslop and colleagues conducted a phase 1 clinical trial involving 19 children with neuroblastoma, between 2004 and 2009, during which they tested T cells that were engineered to recognize GD2, a protein with high levels of expression in neuroblastoma. 11 of these patients had active disease, which could be detected using imaging studies. Although the phase 1 trial determined the treatment was safe, 12 patients died between 2 months and 7 years after treatment, owing to relapsed neuroblastoma. Of the remaining 7 patients, 5 continued in follow-up for at least 13 years after treatment. Heslop and colleagues observed that one of the patient’s cancer has gone into remission for more than 18 years without receiving other cancer treatments. She has also given birth to two healthy infants. The authors also found evidence of CAR-T cells persisting for at least 5 years in five of the treated, including the patient with 18 years of remission.

The authors note that the CAR-T cells used in this trial lack design elements of modern CAR-T cells, which now include co-stimulatory molecules, and patients with active disease at the time of treatment may not have benefited as much as those with no evidence of disease or with a lower burden of disease when treated. These data suggest that CAR-T cells have the potential to provide long-term benefits to patients with solid tumours, and also provides biological insights into CAR-T cell behaviour that could be informative for other studies.