Active project
Robbie Majzner, MD
Dana-Farber Cancer Institute / Boston Children’s Hospital
GD2-ZAP70 CAR T Cells in Relapsed and Refractory Neuroblastoma
Dr. Majzner’s team is bringing a new CAR T-cell therapy into a Phase 1 trial for children and young adults whose neuroblastoma has returned or has not responded to treatment. The therapy uses engineered immune cells designed to remain active longer and attack the cancer more effectively.
CureSearch Catapult Award
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.
Project at a glance
- Program
- Catapult Award
- Institution
- Dana-Farber Cancer Institute / Boston Children’s Hospital
- Focus
- Neuroblastoma
Current GD2 CAR T cells only work in patients with small amounts of disease. We’ve designed a brand-new CAR architecture that breaks past limitations to success in lab models, and this trial will allow us to bring this advanced therapy to the children who need it most. The CureSearch Catapult Award is vital to our mission; it gives us the momentum to catapult this science out of the lab and directly to the forefront of patient care.
Robbie Majzner, MD
Dr. Robbie Majzner is Director of the Pediatric and Young Adult Cancer Cell Therapy Program at Dana-Farber Cancer Institute and Boston Children’s Hospital. His research focuses on developing and advancing immunotherapies for children with cancer, with a particular emphasis on improving CAR T-cell therapies for solid tumors and overcoming treatment resistance.
Through both laboratory research and clinical trials, Dr. Majzner works to translate promising scientific discoveries into new treatment options for children and young adults facing difficult-to-treat cancers.
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