Tuesday, February 18, 2025

Woman in remission of a rare neuroblastoma cancer for record 19 years after CAR-T immune treatment as a 4 year old child

Good news! Cancer is history (soon)!

"The girl was four years old when she arrived at Texas Children’s Hospital in Houston to receive a highly experimental therapy for nerve-cell cancer. Standard treatments had been unable to hold the cancer back. It had spread to her bones, and the prognosis was poor.

Nineteen years later, she is cancer-free and the mother of two children. The remarkable success story ... is the longest reported cancer remission following treatment with engineered immune cells called CAR T cells. ..."

From the abstract:
"In a phase 1 clinical trial open to accrual from 2004 to 2009, we treated children with neuroblastoma with Epstein–Barr virus (EBV)-specific T lymphocytes and CD3-activated T cells—each expressing chimeric antigen receptors (CARs) targeting GD2 but without an embedded co-stimulatory sequence (first-generation CARs). These CARs incorporated barcoded sequences to track each infused population. We previously reported outcomes up to 5 years and 
now report long-term outcomes up to 18 years.
Of 11 patients with active disease at infusion, three achieved a complete response that was sustained in two patients, one for 8 years until lost to follow-up and one for more than 18 years.
Of eight patients with no evidence of disease at the time of CAR-T administration, five are disease free at their last follow-up between 10 years and 15 years after infusion. Intermittent low levels of transgene were detected during the follow-up period with significantly greater persistence in those who were long-term survivors. Despite using first-generation vectors that are no longer employed because of the lack of co-stimulatory domains, patients with relapsed/refractory neuroblastoma achieved long-term disease control after receiving GD2 CAR-T cell therapy, including one patient now in remission of relapsed disease for more than 18 years."

Woman in cancer remission for record 19 years after CAR-T immune treatment "CAR-T-cell therapy treated a girl with a rare childhood cancer, raising hopes for future recipients of the approach."



A CAR-T cell (orange) attacks a cancer cell (green), which is starting to contract.

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