Francesca Del Bufalo, MD, PhD, on Evaluating GD2-Directed CAR-T in Pediatric Neuroblastoma


The medical doctor and scientist at Bambino Gesù Chidren’s Hospital discussed phase 1/2 clinical trial data she presented at ASGCT’s 2024 Meeting.

“CAR T-cells can be effective against solid tumors. We need to identify [first of all] the ideal construct. The preclinical evaluation was very crucial for us. Then you need to identify the ideal population...”

The success seen with chimeric antigen receptor T-cell (CAR-T) therapy in hematological malignancies, which now have 6 FDA-approved CAR-T products available in the United States, has led to interest in replicating this success for the treatment of solid tumors. Academic institutions across the globe are currently pursuing a myriad of diverse methods for bringing the cell therapy modality to a variety of solid tumor types. One such institution is Bambino Gesù Children’s Hospital, in Rome, Italy, which is conducting a phase 1/2 clinical trial evaluating a GD2-directed CAR-T construct in pediatric patients with neuroblastoma. Francesca Del Bufalo, MD, PhD, a medical doctor and scientist at Bambino Gesù Children’s Hospital, recently presented promising results from this study at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD.

Following her presentation, CGTLive® sat down with del Bufalo on the conference floor to learn more. Del Bufalo went over the key results from the trial, pointing out that overall response rates and 3-year event-free survival was improved in patients who started treatment with the CAR-T with a lower burden of disease compared to patients with a high disease burden. She noted that a future avenue of study will seek to evaluate the CAR-T as a second and eventually first-line treatment. Del Bufalo also spoke about a significant challenge encountered in the study with a group of patients that were refractory to the CAR-T therapy. She concluded by discussing the importance of academic institutions pursuing the development of novel treatments for narrow indications that would not be financially feasible for private companies to pursue and emphasized the need for new models of funding later phase clinical trials in academic settings.

Click here to view more coverage of the 2024 ASGCT Annual Meeting.

1. Del Bufalo F. GD2 CAR Clinical Trial. Presented at: ASGCT Annual Meeting 2024, May 7-10; Baltimore, Maryland.

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