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CureSearch for Children’s Cancer, a leading national nonprofit dedicated to accelerating the development of new pediatric cancer treatments, has announced a significant $2 million investment to advance a groundbreaking immunotherapy for neuroblastoma. This investment, formalized through the 2026 CureSearch Catapult Award, is co-funded by the Jeff Gordon Children’s Foundation and will support a Phase I clinical trial led by Dr. Robbie Majzner at the Dana-Farber Cancer Institute. The project represents a critical effort to address the high mortality rates associated with relapsed or refractory neuroblastoma, a condition that has long lacked effective therapeutic options.
The funding is specifically earmarked for the evaluation of a next-generation Chimeric Antigen Receptor (CAR) T-cell therapy. Unlike traditional treatments, this approach utilizes a patient’s own immune system, re-engineered to recognize and destroy malignant cells. The 2026 Catapult Award focuses on bridging the "valley of death" in drug development—the precarious gap between laboratory discovery and human clinical trials—ensuring that high-potential scientific breakthroughs do not stall due to a lack of early-stage funding.
The Urgent Challenge of Pediatric Neuroblastoma
Neuroblastoma remains one of the most formidable challenges in pediatric oncology. As the most common extracranial solid tumor in children, it accounts for roughly 7% to 10% of all childhood cancers but is responsible for a disproportionate 15% of all pediatric cancer deaths. The disease originates in the primordial crest cells of the sympathetic nervous system, typically manifesting in the adrenal glands, though it can also develop in the neck, chest, or pelvis.
While low-risk and intermediate-risk neuroblastomas often respond well to standard treatments, "high-risk" cases present a much grimmer prognosis. Despite an intensive regimen of high-dose chemotherapy, surgery, radiation, and stem cell transplants, the five-year survival rate for high-risk neuroblastoma remains below 50%. For children who experience a relapse or whose cancer is refractory (resistant to initial treatment), the survival rates drop even further, often leaving families with few, if any, remaining clinical options. Furthermore, the survivors of current aggressive protocols frequently suffer from long-term "late effects," including hearing loss, growth impairment, and secondary malignancies.
The Evolution of CAR T-Cell Therapy and the GD2 Target
The therapeutic innovation at the heart of Dr. Majzner’s trial involves CAR T-cell therapy, a modality that has already revolutionized the treatment of certain blood cancers, such as B-cell acute lymphoblastic leukemia (ALL). CAR T-cells are "living drugs"—T-cells harvested from a patient, genetically modified in a laboratory to express a specific receptor that targets cancer proteins, and then infused back into the patient.
However, translating the success of CAR T-cells from "liquid" blood cancers to "solid" tumors like neuroblastoma has proven difficult. Solid tumors create a hostile microenvironment that can suppress immune responses, and finding a target protein that is present on cancer cells but absent on healthy tissue is a complex task.
Dr. Majzner’s research focuses on GD2, a disialoganglioside expressed on the surface of nearly all neuroblastoma cells. While GD2 has been a target for monoclonal antibodies for years, existing CAR T-cell therapies targeting this molecule have faced a recurring obstacle: T-cell exhaustion. In the dense and immunosuppressive environment of a solid tumor, CAR T-cells often become "tired" or dysfunctional before they can fully eradicate the cancer.
Technical Innovation: The ZAP70 Signaling Protein
The specific breakthrough being funded by the Catapult Award involves a redesigned CAR architecture. To combat the issue of exhaustion, Dr. Majzner’s team has incorporated a novel signaling protein known as ZAP70. In natural immune responses, ZAP70 plays a crucial role in how T-cells communicate and sustain their activity. By integrating this protein into the CAR T-cell structure, the researchers aim to create a more resilient immune cell.
Preclinical data generated in Dr. Majzner’s laboratory suggests that these ZAP70-enhanced CAR T-cells demonstrate significantly stronger anti-tumor activity compared to earlier iterations. More importantly, they show improved persistence, meaning the cells stay active in the body longer, providing a sustained defense against the tumor. This persistence is often the deciding factor between a temporary remission and a long-term cure. The redesigned cells also showed signs of a potentially safer profile, a vital consideration in pediatric medicine where toxicity can have lifelong consequences.
The Catapult Award: Bridging the Funding Gap
The CureSearch Catapult Award is a strategic instrument designed to accelerate the pace of pediatric drug development. In the pharmaceutical industry, pediatric cancers are often classified as "orphan diseases" because the number of patients is relatively small compared to adult cancers like breast or lung cancer. This often results in a lack of commercial incentive for large biotech companies to invest in early-stage pediatric trials.
CureSearch addresses this market failure by providing the necessary capital to move science out of the academic lab and into the clinic. By partnering with the Jeff Gordon Children’s Foundation, CureSearch has pooled resources to ensure that Dr. Majzner’s Phase I trial has the financial runway required to reach its milestones.
"Current GD2 CAR T cells only work in patients with small amounts of disease," explained Dr. Majzner. "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."
Official Responses and Institutional Support
The selection of Dr. Majzner and Dana-Farber Cancer Institute followed a rigorous peer-review process by CureSearch’s Scientific Advisory Council, which comprises leading experts in pediatric oncology and drug development. The council identified this project as one of the most promising avenues for changing the standard of care for neuroblastoma.
Dr. Paisley Myers, Senior Director of Research & Programs at CureSearch, emphasized the organization’s commitment to high-impact science. "One of the greatest challenges in pediatric cancer research is ensuring that promising discoveries reach children," Dr. Myers stated. "Through the Catapult Award, CureSearch helps move innovative therapies from the laboratory into clinical trials. Dr. Majzner’s work exemplifies the bold, high-potential research we were created to support."
The Jeff Gordon Children’s Foundation, founded by the four-time NASCAR Cup Series champion, has a long history of supporting pediatric cancer research. Their co-funding of this award underscores a shared belief that the next generation of cancer treatments will come from immunotherapy and precision medicine.
Chronology and Future Implications
The timeline for the project begins with the formal award in 2026, which will initiate the regulatory and logistical preparations for the Phase I clinical trial. This stage typically involves finalizing the manufacturing process for the CAR T-cells—a complex procedure that must meet stringent FDA standards for safety and purity.
Once the trial commences, it will focus on safety and dose-escalation. Researchers will monitor patients for side effects, such as cytokine release syndrome (CRS) or neurotoxicity, which are known risks of CAR T-cell therapies. Simultaneously, the team will gather preliminary data on the therapy’s efficacy by measuring tumor shrinkage and the persistence of the modified T-cells in the patients’ bloodstreams.
If the Phase I trial proves successful, it will pave the way for larger Phase II trials and potential regulatory approval. The implications, however, extend beyond neuroblastoma. Because GD2 is also expressed in other pediatric and adult malignancies—including osteosarcoma, melanoma, and certain types of brain tumors—the success of this ZAP70-enhanced CAR T-cell architecture could provide a blueprint for treating a wide array of solid tumors that have previously been resistant to immunotherapy.
By funding this trial, CureSearch and the Jeff Gordon Children’s Foundation are not only investing in a single treatment for neuroblastoma but are also contributing to the broader scientific understanding of how to overcome immune exhaustion in the fight against cancer. This investment reflects a shift in the oncology landscape toward highly targeted, "smart" therapies that aim to maximize survival while minimizing the devastating side effects of traditional oncology.
