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Clinical Development of RNA Nanoparticle Vaccines Targeting Recurrent Pediatric Brain Cancers

Duke University
Duane Mitchell, MD, PhD
Grant Type: 
Innovation Grants
Year Awarded: 
Type of Childhood Cancer: 
Brain Tumors, Medulloblastoma, Primitive Neuroectodermal Tumor (PNET)
Project Description: 


Cancer vaccines are designed to stimulate the immune system into specifically targeting and killing invasive tumor cells.  If tumor-specific antigens can be identified, these targets may meet the clear and urgent need for the development of safe and effective vaccines for children with recurrent brain tumors. We hypothesize that nanoparticles loaded with tumor RNA can serve as an effective immunization platform for eliciting potent tumor-specific immune responses in children with recurrent brain cancers. The development of an effective therapeutic vaccine platform using a nanoparticle formulation that (1) can be made in large quantities and (2) is suitable for distribution to multiple treatment centers for clinical evaluation would greatly benefit the advancement of cancer vaccines for children with malignant brain tumors.

Project Goals

The specific aims of this research are to explore in a mouse model of medulloblastoma the effects of nanoparticle vaccines on the immune system and tumor growth to determine if nanoparticle vaccines targeting medulloblastoma are safe and effective in treated these tumors. Further studies will explore the ability to manufacture nanoparticle tumor RNA vaccines from surgical tumor specimens collected from children with malignant brain tumors.  The translation of this research into clinical trials for children with recurrent brain cancers may offer a new therapeutic treatment could be both more efficacious and less toxic than current treatment options.