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CureSearch for Children’s Cancer, a leading national non-profit foundation, has officially named Dr. Robbie Majzner of the Dana-Farber Cancer Institute as the recipient of the 2026 CureSearch Catapult Award. This prestigious $2 million grant, co-funded in partnership with the Jeff Gordon Children’s Foundation, is earmarked for the launch and execution of a Phase I clinical trial that seeks to redefine the treatment landscape for children and young adults suffering from relapsed or refractory neuroblastoma. By targeting the "valley of death"—the precarious gap between laboratory discovery and clinical application—this investment aims to accelerate the delivery of a highly sophisticated Chimeric Antigen Receptor (CAR) T-cell therapy that has shown significant promise in preclinical models.
The Critical Challenge of High-Risk Neuroblastoma
Neuroblastoma is a complex and aggressive cancer that originates in early nerve cells, known as neuroblasts, of the sympathetic nervous system. It is most commonly diagnosed in infants and very young children, frequently appearing in the adrenal glands, but also capable of developing in the neck, chest, abdomen, or spine. As the most common extracranial solid tumor in the pediatric population, neuroblastoma accounts for roughly 7% to 10% of all childhood cancers and is responsible for a disproportionate 15% of all pediatric oncology deaths.
Despite intensive multi-modal treatment regimens—which typically include high-dose chemotherapy, surgical resection, radiation, and autologous stem cell transplantation—the prognosis for patients with high-risk neuroblastoma remains sobering. Long-term survival rates for this cohort hover below 50%. For those who experience a relapse or whose cancer is refractory to standard frontline therapies, the outlook is even more dire, with few effective options remaining. Furthermore, the "cure" often comes at a steep price; survivors frequently face lifelong "late effects," including hearing loss, cardiac dysfunction, growth impairment, and a heightened risk of secondary malignancies. The urgent need for targeted, less toxic, and more effective therapies has led researchers toward the burgeoning field of immunotherapy.
Engineering the Next Generation of CAR T-Cell Therapy
CAR T-cell therapy has already achieved revolutionary success in treating certain "liquid" cancers, such as B-cell acute lymphoblastic leukemia. This process involves extracting a patient’s own T-cells—the "soldiers" of the immune system—and genetically modifying them in a laboratory to express a synthetic receptor (the CAR). These modified cells are then infused back into the patient, where they are programmed to seek out and destroy cells expressing a specific protein.
However, replicating this success in solid tumors like neuroblastoma has proven exceptionally difficult. Solid tumors present a hostile microenvironment that can suppress immune activity. A primary hurdle is "T-cell exhaustion," a state where the engineered cells become fatigued and lose their ability to kill cancer cells or persist long enough to prevent a recurrence.
Dr. Robbie Majzner’s research at Dana-Farber focuses on overcoming these specific barriers. The new therapy targets GD2, a disialoganglioside expressed on the surface of nearly all neuroblastoma cells. While GD2-targeted therapies already exist, they often suffer from limited persistence. Dr. Majzner’s team has re-engineered the internal signaling architecture of the CAR T-cell by incorporating a novel signaling protein called ZAP70.
In traditional CAR designs, the signaling components can sometimes lead to "tonic signaling," which wears the cell out before it can complete its mission. By integrating ZAP70, the team aims to create a more "physiologic" signal that mimics how natural T-cells operate. Preclinical data indicates that these redesigned cells remain active for longer periods, demonstrate superior anti-tumor potency, and may offer an improved safety profile by reducing the risk of off-target toxicity.
The Strategic Mission of the Catapult Award
The CureSearch Catapult Award is a strategic funding mechanism designed to address a systemic failure in the drug development pipeline. In the pharmaceutical industry, pediatric cancers are often overlooked due to the relatively small number of patients compared to adult cancers like lung or breast cancer. This creates a funding void where promising laboratory breakthroughs languish because they lack the capital required to move into human clinical trials.
The $2 million investment provided to Dr. Majzner is specifically intended to propel his GD2-targeted ZAP70 CAR T-cell therapy through this transition. By funding the Phase I trial, CureSearch and the Jeff Gordon Children’s Foundation are providing the necessary resources for regulatory filings, cell manufacturing, and clinical monitoring.
"The 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," stated Dr. Majzner. He emphasized that current GD2 CAR T-cells typically only show efficacy in patients with low disease burdens. The new architecture is designed to break through those limitations, offering hope to those with more advanced or resistant disease.
A Chronology of Innovation and Partnership
The path to the 2026 Catapult Award is the result of years of collaborative effort and rigorous scientific vetting. The timeline of this initiative reflects a structured approach to oncology innovation:
- 2020–2023: Preclinical Validation. Dr. Majzner and his colleagues conducted extensive laboratory research, utilizing mouse models and in-vitro assays to test the ZAP70-enhanced CAR T-cells. The results showed a marked increase in the cells’ ability to survive and multiply within the tumor environment.
- 2024: Selection Process. CureSearch’s Scientific Advisory Board, composed of international experts in pediatric oncology, reviewed numerous high-potential projects. Dr. Majzner’s work was selected for its high degree of innovation and its potential to be "practice-changing."
- 2025: Preparatory Phase. With the award announced, the focus shifts to finalizing the clinical trial protocol, securing Institutional Review Board (IRB) approvals, and preparing the manufacturing facilities at Dana-Farber and partner institutions to produce the patient-specific cells.
- 2026: Clinical Trial Launch. The Phase I trial is scheduled to begin enrolling pediatric and young adult patients. This stage will primarily focus on establishing the safety of the therapy and determining the optimal dosage.
The partnership with the Jeff Gordon Children’s Foundation adds a significant layer of support. Founded by the four-time NASCAR Cup Series champion, the foundation has a long-standing commitment to pediatric cancer research. This co-funding model allows for larger, more impactful grants that can sustain the high costs associated with cell therapy trials.
Supporting Data and Clinical Expectations
The upcoming Phase I trial will be closely watched by the global oncological community. Data from previous iterations of GD2 CAR T-cells showed that while some patients achieved complete remission, many experienced "antigen escape" or T-cell disappearance within weeks.
Supporting data from Dr. Majzner’s preclinical studies suggests that the ZAP70 modification allows for a "slow and steady" activation of the T-cells, which prevents the rapid burnout seen in earlier models. If the clinical trial mirrors these results, it could lead to a paradigm shift in how solid tumors are treated.
Safety is also a paramount concern in Phase I. GD2 is present on some healthy nerve tissues, which can lead to significant pain or neurological side effects during treatment. The new CAR architecture includes features designed to minimize these side effects, potentially making the treatment more tolerable for young children.
Broader Implications for Pediatric Oncology
The implications of Dr. Majzner’s work extend beyond neuroblastoma. GD2 is also expressed in other difficult-to-treat pediatric cancers, including osteosarcoma (a bone cancer) and certain types of brain tumors like Diffuse Midline Glioma (DMG). If the ZAP70 signaling architecture proves successful in this trial, it could serve as a "plug-and-play" platform for other CAR T-cell therapies.
Furthermore, this award highlights the evolving role of non-profit organizations in the medical ecosystem. By acting as venture philanthropists, organizations like CureSearch are not just "donating" to research; they are strategically investing in the most viable paths to a cure.
"One of the greatest challenges in pediatric cancer research is ensuring that promising discoveries reach children," said Dr. Paisley Myers, Senior Director of Research & Programs at CureSearch. "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."
Conclusion: A New Horizon for Families
As the 2026 trial approaches, the medical community remains cautiously optimistic. For the families of children with neuroblastoma, the Catapult Award represents more than just a financial investment; it represents a tangible move toward a future where a diagnosis of relapsed neuroblastoma is no longer a death sentence. By bridging the gap between the lab bench and the bedside, the collaboration between Dana-Farber, CureSearch, and the Jeff Gordon Children’s Foundation underscores a collective resolve to provide every child with the opportunity for a healthy, cancer-free future. The success of this trial could mark the beginning of a new era in precision medicine, where engineered immune cells provide a durable and potent defense against the most stubborn of childhood cancers.
