GIRL TREATED WITH CAR-T FOR NEUROBLASTOMA, IS STILL ALIVE AND HEALTHY 19 YEARS AFTER TREATMENT

In a recent study published in Nature Medicine, researchers report the successful treatment of a neuroblastoma patient with chimeric antigen receptor (CAR) T-cell therapy.

A woman, now twenty-three years old and with two children,
treated in 2006, with a pioneering type of immunotherapy for a solid tumour has been in remission for more than 19 years with no further treatments.

In CAR-T-cell therapy, T-cells are isolated from the patient’s blood and genetically engineered to express CARs on their surface. When administered to the patient, CAR-T-cells recognize and facilitate the clearance of cancer cells by binding to specific proteins on their surface.

Although CAR-T-cell therapy has been widely successful in treating hematological cancers, this treatment approach is limited in its ability to combat solid tumors.

Neuroblastoma is a rare solid tumor that develops in immature nerve cells. This type of cancer primarily affects children five years of age and younger and is associated with high relapse rates.

Between 2004 and 2009, the researchers of the current study conducted a phase I clinical trial. During this trial, activated T-cells were engineered to target disialoganglioside 2 (GD2), a protein often overexpressed in neuroblastoma cells. This therapy involved both activated T-cells (ATCs) and Epstein-Barr virus (EBV)- specific T-cells (VSTs), which were trained to recognize and attack tumor cells.

The trial enrolled a total of 19 children, 11 of whom had active relapsed neuroblastoma. Among eight patients with no evidence of active disease, five had a history of relapsed disease, and three were infused with the GD2 CAR-T cell therapy after completing therapy for high-risk disease.
Among eleven patients with active disease at the time of infusion, three had complete responses, and one had a partial response. One of the three patients with complete responses subsequently experienced disease relapse. However, the remaining two patients had sustained responses. One experienced beneficial effects for eight years until being lost to follow-up, while the other continued to report positive outcomes for more than 18 years.

Source:

https://www.nature.com/articles/s41591-025-03513-0

https://www.news-medical.net/news/20250217/Cancer-free-for-18-years-CAR-T-therapy-sets-new-milestone-in-neuroblastoma-treatment.aspx