A recent correction to published clinical trial data offers renewed optimism for children and young adults battling high-risk neuroblastoma,an aggressive cancer that develops from immature nerve cells. The findings, published online September 8, 2025, in Nature Medicine, detail the results of a phase 1/2 trial investigating GD2-targeting chimeric antigen receptor (CAR) T-cell therapy.
Neuroblastoma is most commonly diagnosed in young children, with high-risk cases - those that have spread to other parts of the body - carrying a particularly poor prognosis. Standard treatments include surgery, chemotherapy, radiation therapy, and stem cell transplant, but relapse rates remain high.