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Researchers in Ireland have reported the first preclinical study of an mRNA vaccine designed specifically to treat neuroblastoma, one of the deadliest childhood cancers.
This vaccine is designed to target Glypican-2 (GPC2), a protein found at high levels on many neuroblastoma cells and also expressed in medulloblastoma and certain other high-grade gliomas. Instead of using traditional lipid nanoparticles for the vaccine, they employed tiny self-assembling peptide nanoparticles called RALA to deliver the mRNA instructions to the immune system.
The vaccine successfully stimulated a strong anti-tumor immune response in lab models, increasing production of several immune signaling molecules, including IFN-γ, IL-2, and TNF-α. In mice with aggressive MYCN-amplified neuroblastoma, the vaccine delayed tumor growth by roughly 10-11 days and reduced tumor volume by ~70% compared with untreated animals.
These results are preclinical, and significant additional work will be needed before human trials can test the safety and efficacy of this vaccine. Nevertheless, we will keep an eye on this promising research.