Saheli Sadanand Highlights a Study on CAR T Cells Showing Promise in High-Risk Neuroblastoma
Saheli Sadanand, Deputy Editor of Nature Medicine, shared a post on LinkedIn about a paper by Franco Locatelli et al. published in Nature Medicine:
“New in Nature Medicine – final results from a phase 1/2 trial and a case series showcase the clinical potential for GD2-targeting CAR-T cells in 54 children with high-risk metastatic, relapsed, or treatment-refractory neuroblastoma.
Some background: While CAR-T cells have been highly effective for various adult and pediatric hematological malignancies, there have been few demonstrations of strong clinical efficacy for solid tumors to date. Neuroblastoma is a type of solid tumor. It is one of the most common pediatric cancers and expresses high levels of GD2. In 2023, Franco Locatelli and colleagues published interim results from this trial in the New England Journal of Medicine.
KEY findings of this new study:
- no new safety signals
- overall response rate (ORR) of 66% in the clinical trial with a 5 year overall survival (OS) of 42.7% (excluding 3 patients treated with no evidence of disease)
- ORR of 77% and 5-year OS of 67.6% in children with low disease burden treated with the recommended CART dose (including 30 patients across the trial and case series)
- CARTs exhibited robust expansion and persistence, detectable up to 5 years post-infusion in some patients with sufficient follow-up
These data support the efficacy of this therapy for children with neuroblastoma, particularly when given early, and showcase the potential for CARTs in solid tumors. A larger phase 2 trial to validate this approach is now in the works.
Congratulations to the author team and especially the children and families who participated in this study!”
Title: GD2-targeting CAR T cells in high-risk neuroblastoma: a phase 1/phase 2 trial
Authors: Franco Locatelli, Daria Pagliara, Maria A. De Ioris, Marco Becilli, Giada Del Baldo, Annalisa Serra, Angela Mastronuzzi, Maria G. Cefalo, Giuseppina Li Pira, Giovanna Leone, Valentina Bertaina, Francesco Fabozzi, Matteo Di Nardo, Chiara Rosignoli, Maria Luisa D’Andrea, Alessandro Crocoli, Sabina Vennarini, Matilde Sinibaldi, Stefano Di Cecca, Marika Guercio, Laura Iaffaldano, Barbarella Lucarelli, Mattia Algeri, Pietro Merli, Giovanna S. Colafati, Biagio De Angelis, Concetta Quintarelli, Francesca del Bufalo
You can read the Full Article on Nature Medicine.
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