Researchers across multiple institutions have identified a novel therapeutic target for a rare and aggressive childhood cancer, neuroblastoma and are collaborating to accelerate drug development. The work, involving scientists from the University of Torino, Dana-Farber Cancer Institute, Texas Tech University Health Sciences Center, and the Cancer Research UK Manchester Institute, focuses on a specific vulnerability in high-risk neuroblastoma tumors.
The collaborative effort, detailed in recent research publications, centers on the role of the MYCN oncogene, which is amplified in a significant proportion of neuroblastoma cases. Researchers discovered that MYCN drives the expression of a specific protein that is essential for tumor survival. Targeting this protein, rather than MYCN directly, offers a potentially more effective and less toxic therapeutic strategy.
Matteo Menotti, currently at the Cancer Research UK Manchester Institute, and Ramesh Choudhari, now at Texas Tech University Health Sciences Center, were key contributors to the research. Their work, alongside colleagues Chiara Ambrogio and Taek-Chin Cheong, revealed a critical dependency within neuroblastoma cells. The team identified a specific pathway that, when disrupted, leads to tumor cell death.
The research builds upon Manchester’s established cancer research heritage, as highlighted by the Cancer Research UK Manchester Institute, a leading institute within the University of Manchester. The institute, core-funded by Cancer Research UK, integrates basic and clinical translational cancer research, fostering collaboration between scientists and clinicians at The Christie NHS Foundation Trust. The institute is currently recruiting PhD students to start in October 2026.
The Manchester Cancer Research Centre (MCRC), a partnership between The University of Manchester, Cancer Research UK, and The Christie NHS Foundation Trust, aims to be a world-leading center for translational cancer research. Established in 2006, the MCRC focuses on transforming clinical care through personalized medicine strategies. Professor Rob Bristow currently directs the MCRC.
Further investigations at Dana-Farber Cancer Institute and Harvard Medical School, led by researchers including Seth H. Cassel and Cigall Kadoch, validated the findings and identified potential drug candidates that could selectively target the identified protein. The team is now working to optimize these compounds and evaluate their efficacy in preclinical models.
Researchers at the University of Torino, led by Roberto Chiarle, contributed to the understanding of the molecular mechanisms underlying the vulnerability. Their work, alongside contributions from the National Institutes of Health and the Centro Nacional de Biotecnología in Spain, provided crucial insights into the protein’s function and its role in neuroblastoma progression.
The collaborative nature of this research is underscored by the involvement of multiple institutions and the sharing of resources and expertise. The team is currently focused on advancing these findings towards clinical trials, with the goal of providing new treatment options for children with high-risk neuroblastoma. Further research is planned to explore the potential of this therapeutic strategy in other cancers driven by MYCN amplification.
Leave a Reply