Robbie Majzner, MD

Project Summary:

Neuroblastoma is the most common solid tumor outside the brain in children and remains one of the deadliest childhood cancers. While CAR T-cell therapy has transformed treatment for some cancers, it has shown limited success in solid tumors like neuroblastoma.

With support from a $2 million CureSearch Catapult Award, Dr. Majzner and his team are launching a Phase I clinical trial evaluating a novel CAR T-cell therapy for children and young adults with relapsed or refractory neuroblastoma. The therapy targets GD2 - a molecule found on neuroblastoma cells - and is engineered with a novel ZAP70 signaling domain designed to help CAR T cells stay active longer and continue fighting cancer more effectively.

In preclinical studies, the approach demonstrated stronger anti-tumor activity, improved persistence, and signs of a potentially improved safety profile compared with traditional GD2-targeted CAR T-cell therapies

Potential Impact

This research could lead to a new treatment option for children and young adults with relapsed neuroblastoma while helping advance the future of CAR T-cell therapy for other pediatric and adult cancers.

Beyond neuroblastoma, the project may provide important insights into how engineered immune cells can be designed to overcome challenges that have limited the effectiveness of CAR T-cell therapy in solid tumors, potentially influencing future cellular therapies across multiple cancer types.