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A personalized immunotherapy approach developed by Washington University School of Medicine for the treatment of pediatric patients with recurrent or refractory brain tumors. The platform utilizes tumor/normal exome sequencing and cDNA-capture sequencing to identify up to 20 tumor-specific neoantigens (mutations unique to the patient's tumor). These neoantigens are then incorporated into a custom-designed vaccine, which has been evaluated in two primary forms: a DNA-based plasmid platform and a synthetic long peptide platform. The DNA vaccine is typically delivered via the TDS-IM v2.0 electroporation device to enhance cellular uptake, while the peptide vaccine is administered in combination with the adjuvant poly-ICLC (a TLR3 agonist) to stimulate a robust T-cell mediated anti-tumor immune response. The primary objective of these early-phase trials is to assess the safety, feasibility, and immunogenicity of this personalized approach in pediatric neuro-oncology.
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