(ANSA) – ROME, AUG 28 – Complete remissions in 40% of cases, a 77% overall response rate in low-burden patients, and a 5-year survival rate approaching 90% when administered early: GD2 CAR-T cells, developed and tested at the Bambino Gesù Children’s Hospital in Rome, are proven safe and effective in the treatment of refractory or relapsed neuroblastoma.
The definitive confirmation comes from the scientific journal Nature Medicine, which published the results of the Phase I/II clinical trial, launched in 2018 and now in its final analysis.
The study involved 54 children, who were infused with GD2 CAR-T cells produced from their own lymphocytes and modified in the laboratory to recognize and destroy tumor cells.
Overall, two out of three patients responded positively to the therapy, and 40% achieved complete remission six months after infusion.
In children with a low disease burden, the overall response rate reached 77%, with a five-year survival rate of 68% and an event-free survival rate of 53%.
Even better results were observed in patients treated early, after one or two lines of therapy, with a five-year survival rate approaching 90%, compared to 43% in children who had already received three or more lines of therapy.
Treatment during the consolidation phase also yielded promising outcomes: seven of the eight children treated in this condition (at least four of whom were expected to relapse) remain disease-free, with a median follow-up of 15 months.
Another notable finding concerns the 13 patients whose T lymphocytes had been collected at diagnosis, before exposure to chemotherapy.
In this cohort, 5-year overall survival reached 100% and event-free survival was 66.5%, compared to 33.2% and 22.6%, respectively, in children treated with cells harvested later, at the time of relapse.
“The data published today tell us that this is the right path and that we are ever closer to making this therapy an integral part of standard care,” commented Franco Locatelli, head of the Center for Clinical Studies in Hematology, Oncology and Cellular Therapies at Bambino Gesù Hospital. (ANSA).
Read article…
The definitive confirmation comes from the scientific journal Nature Medicine, which published the results of the Phase I/II clinical trial, launched in 2018 and now in its final analysis.
The study involved 54 children, who were infused with GD2 CAR-T cells produced from their own lymphocytes and modified in the laboratory to recognize and destroy tumor cells.
Overall, two out of three patients responded positively to the therapy, and 40% achieved complete remission six months after infusion.
In children with a low disease burden, the overall response rate reached 77%, with a five-year survival rate of 68% and an event-free survival rate of 53%.
Even better results were observed in patients treated early, after one or two lines of therapy, with a five-year survival rate approaching 90%, compared to 43% in children who had already received three or more lines of therapy.
Treatment during the consolidation phase also yielded promising outcomes: seven of the eight children treated in this condition (at least four of whom were expected to relapse) remain disease-free, with a median follow-up of 15 months.
Another notable finding concerns the 13 patients whose T lymphocytes had been collected at diagnosis, before exposure to chemotherapy.
In this cohort, 5-year overall survival reached 100% and event-free survival was 66.5%, compared to 33.2% and 22.6%, respectively, in children treated with cells harvested later, at the time of relapse.
“The data published today tell us that this is the right path and that we are ever closer to making this therapy an integral part of standard care,” commented Franco Locatelli, head of the Center for Clinical Studies in Hematology, Oncology and Cellular Therapies at Bambino Gesù Hospital. (ANSA).
Read article…
