Abstract

Clinical Translation of Extracellular Vesicle Research

Jan Lötvall, Professor/Chairman, University of Gothenburg

Over the last ten to fifteen years, we have seen a massive increase in the understanding of extracellular vesicle (EV) biology and function, primarily based on in vitro models and animal experimentation. Currently, there is a strong push to translate EV science to the clinic, primarily as diagnostic and therapeutic tools. For diagnostics, tests are becoming available primarily for the diagnosis of different types of cancer, and initially RNA or DNA in the vesicles have been used to phenotype and stage disease. However, a significant number of EV-membrane proteins are starting to identify subpopulations of EVs, and these are putative biomarker candidates in different diseases. In this lecture, I will describe a work process to identify EV subpopulations of different human cell- or tissue origin, and present how they can function as biomarkers. Perhaps even more important is the current investments in developing EVs as therapeutics in different diseases. Several venture capital financed startup biotech companies have been established to develop EV therapeutics in diseases such as cancer or rare diseases such as Neiman-Pick type C. Most EV startups focus on delivering different types of cargo to a recipient cell’s cytoplasm, for example a large protein or an siRNA molecule that otherwise would not be taken up in a recipient cell. I will discuss the different therapeutic opportunities with EVs, and present some of our own efforts in developing highly efficient EV-mimetics directly from cell membranes at very high yields. I predict that in the next few years, EV therapeutics will become a clearly established modality as the next generation of biological medicines, and we are likely to see medical revolution evolving before our eyes.