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mRNA Vaccine Targeting GPC2 Reduces Neuroblastoma Tumor Size in Preclinical Models

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A new mRNA vaccine targeting the aggressive childhood cancer neuroblastoma has shown promising results, reducing tumor size by 70% in preclinical models.

Dublin, Ireland – A collaborative study led by researchers at RCSI University of Medicine and Health Sciences and the School of Pharmacy at Queen's University of Belfast has tested a novel mRNA vaccine against neuroblastoma. The findings, published in the journal Molecular Therapy Oncology, represent a significant step forward in the fight against this deadly disease.

Vaccine Mechanism and Findings

The vaccine targets the protein Glypican 2 (GPC2), which is found on the surface of neuroblastoma cells. To deliver the therapy, the team used self-assembling peptide nanoparticles carrying mRNA that instructs the body to produce the GPC2 protein. This trains the immune system to recognize and attack neuroblastoma cells.

In preclinical models, the results were striking:

  • Tumor size was reduced by 70%.
  • Tumor development was delayed by 10 to 11 days.

"The mRNA vaccine technology is modular, allowing for precise tailoring. This study is a first milestone in vaccine development, showing promising potential."
— Dr. Olga Piskareva, Senior Lecturer at RCSI and lead author of the study.

Background on Neuroblastoma

Neuroblastoma accounts for an estimated 15% of childhood cancer deaths. In Ireland, between five and ten cases are diagnosed annually. Current treatments are insufficient, with 80% of patients showing no significant response, and relapsed cases are often resistant to existing therapies.

Broader Implications

The GPC2 protein is not exclusive to neuroblastoma; it is also present on other types of cancer cells. This suggests the vaccine technology could potentially be adapted for use against a variety of other cancers, broadening its impact.

Funding and Publication

The study was funded by:

  • The Irish Research Council
  • The Higher Education Authority
  • The Health Research Board
  • The Conor Foley Neuroblastoma Cancer Research Foundation

The full research was published in the journal Molecular Therapy Oncology.