Nanoparticles with RNA can fight and prevent cancer and HIV

Researchers at Fred Hutchinson Cancer Research Center have developed a cheaper and faster therapy for diseases such as cancer and HIV, which is considered as a promising new breakthrough as the approach is said to be as simple as “just add water.”

The researchers have developed a new biomedical tool that uses nanoparticles to administer gene changes to targeted cells. Researchers developed this nanoparticle delivery system to extend the therapeutic potential of messenger RNA. This in turn delivers molecular instructions from DNA to cells in the body, directing them to make proteins to prevent or fight disease. The tool was tested in preclinical models and has been described in detail in a paper published in Nature Communications.

About the overall vision, the lead author Dr. Matthias Stephan, a faculty member in the Fred Hutch Clinical Research Division stated, “Our goal is to streamline the manufacture of cell-based therapies." About the tool, he also says, "In this study, we created a product where you just add it to cultured cells and that's it -- no additional manufacturing steps." The researchers focused on T cells of the immune system and blood stem cells and how to deliver mRNA directly to the cells, triggering short-term gene expression.

Previous attempts to engineer mRNA had not yielded positive results a large messenger molecule would be used which would degrade quickly. The body's immune system would recognize it as foreign and destroy it. The nanoparticle system is gentler to cells and many more cells survive in the process. The technology is not yet available as a treatment as researchers are waiting for commercial partners to facilitate the move towards additional applications and into clinical trials.

DOI: 10.1038/s41467-017-00505-8