Short Course in the Exosome

Short Course in Extracellular Vesicles: 
The Transition from Tissue to Liquid Biopsies

 Downloadable Handout                                            Summary Article Reprinted from The Journal of Circulating Biomarkers 2014

Session 4- October 2, 2014 Panel on Regulatory and Funding Issues   
Click to View Recording                            

Extracellular vesicles (EVs), including exosomes and microvesicles, have been shown to carry a variety of bio-macromolecules including mRNA, microRNA and other non-coding RNAs. EVs have emerged as a promising minimally invasive novel source of material for molecular diagnostics and may provide a surrogate to tissue-based biomarkers for a variety of diseases. Although EVs can be easily identified and collected from biological fluids using commercial kits, further research and proper validation is needed in order for them to be useful in the clinical setting.

Currently, there are several EV-based research and diagnostic companies that have developed research based kits and are in the process of working with clinical laboratories to develop and validate EV-based assays for a variety of diseases. 

Successful clinical application of EV-based diagnostic assays will require a close collaboration between industry, academia, regulatory agencies and access to patient samples. We expect that international, integrative and interdisciplinary translational research teams, along with the emergence of FDA-approved platforms, will set the framework for EV-based diagnostics. We see the EV field offer new promise for personalized/precision medicine and targeted treatment in a variety of diseases.

This short course, taught by pioneers and experts in the extracellular vesicle domain, will cover a broad range of topics from an overview of the field to its applications, the current state and challenges in the commercialization of extracellular vesicles for research and into the clinic. We will then conclude with a panel discussion on the regulatory aspects and funding opportunities in this field.

This Course is Developed by Winston Kuo, CEO, IES Diagnostics, with participation from Göteborg University, Exosome Diagnostics, and Thermo Fisher.

Jan Lötvall, Professor, Department of Internal Medicine, Göteborg University

Dr. Lötvall has been at Göteborg University since 2002, and chairman of the Krefting Research Centre. He has clinical training in both Clinical Allergy and Clinical Pharmacology, and has a long-term experience in performing translational studies in asthma and allergy.  

Dr Lötvalls current research interest also includes the biological function of exosomes and other extracellular vesicles, and their natural ability of transferring RNA between cells, after having published the original study documenting this process in 2007 in Nature Cell Biology.  

Previously, Dr Lötvall has been a fellow at the Department of Thoracic Medicine, Imperial College, London and the Department of Respiratory Medicine, McMaster University, Canada, and has approximately 240 scientific papers published or in press (PubMed).

He is co-Editor-in-Chief of the open access scientific journal “Respiratory Research”, and has in the last years been instrumental in starting two open access journals, including “Journal of Extracellular Vesicles”. He is also the first elected President of the International Society of Extracellular Vesicles (ISEV), rapidly growing non-profit organisation in the field of exosomes, microvesicles and other extracellular vesicles (www.isev.org; with 700 delegates 2013), and is the Past President of European Academy of Allergy and Clinical Immunology (EAACI).

Johan Skog, PhD, Chief Scientific Officer, Exosome Diagnostics

During his time at Massachusetts General Hospital/Harvard Medical School in Boston, MA, Dr. Skog discovered that tumor derived mutations can be detected in the microvesicle RNA from serum and other biofluids, which was published in Nature Cell Biology in 2008.

Additionally, he demonstrated that microvesicles serve to deliver messages to other cells, inducing changes favorable to the proliferation of cancer. His research background includes virology, gene therapy, brain tumors, RNA and biomarker discovery, and he is an inventor on several patents. He earned his Master of Biomedical Sciences and Doctorate at Umeå University, Sweden.

Sasha Vlassov, Senior Staff Scientist, Group Leader at Thermo Fisher Scientific

Dr. Alexander “Sasha” Vlassov has developed multiple product lines including reagents for exosome research, siRNA, miRNA mimics/inhibitors, and transfection reagents. Currently, he is focusing on development of advanced technologies for exosome analysis and improved methods for extracting nucleic acids from clinically relevant samples. Prior to joining Life Technologies (now part of Thermo Fisher Scientific), he was a Sr. Scientist at Somagenics (California), Postdoctoral fellow at UC Boulder (Colorado), visiting student fellow at the IBMC (France) and research scientist at ICBFM (Russia). During his scientific career, Dr. Vlassov co-authored over 70 publications and 8 patents.

Angel Ayuso Sacido, Ph.D., Director of the Thoracic and Brain Tumor Laboratory, CIOCC-IMMA, Fundacion Hospital de Madrid, Spain

Dr Ayuso-Sacido made his PhD at Merck Sharp and Dhome and worked as a postdoctoral fellow within the Medicine Department at Mount Sinai School of Medicine and the Neurosurgery Department at Cornell Medical Center. Back to Spain, he co-founded the Glioblastoma Spanish Network (REIG). Currently, he is the Director of the Thoracic and Brain Tumour Laboratory at Hospital de Madrid Foundation, and President of the REIG. His research is focused on the use of extracellular vesicles as biomarkers for diagnostic and follow-up treatment as well as for the evaluation of drug sensitivity in patients diagnosed with high-grade glioma.

Eva Rohde, MD, Head of the Transfusion Medicine Department and Director of the GMP-Laboratory, Paracelsus Medical University Salzburg, Austria

Eva Rohde made her medical education at Karl-Franzens-University, Graz, Austria. She finished her training in Transfusion Medicine in 2005 and worked as postdoctoral fellow at the Stem Cell Research Unit of the Medical University Graz. Her research activities focused on cellular therapy and regenerative medicine, especially on human mesenchymal stromal and endothelial progenitor cells from various tissues and the manufacturing of cell-based therapeutics. The postgraduate training “Pharmaceutical Quality Management” enabled  her to act as “Qualified Person for the Pharmaceutical Manufacturing of Blood, Cells and Tissues”. Since 2013 she is a Member of the European COST-Action “ME-HaD BM1202, Microvesicles and Exosomes in Health and Disease”. Currently she is establishing a GMP Laboratory at the Paracelsus Medical University Salzburg for the Production of human advanced therapy medicinal products (ATMPs) and/or cell-based biopharmaceuticals such as extracellular vesicles, EVs.

IES Diagnostics is developing molecular immune diagnostics tests for use in personalized medicine. IES's proprietary technology from the FDA makes use of this natural enrichment to achieve high sensitivity and specificity for gene transcripts and the expression of genes responsible for auto-immune diseases as a primary focus, though they can be used for cancers and other diseases.

Dr. Kuo is well known in the life science ecosystem. He founded the Laboratory for Innovative Translational Technologies at Harvard to provide the university's biomedical research community with early access to enabling leading edge translational technologies - an integral part of the Harvard Catalyst Clinical and Translational Science Center at Harvard Medical School. The Lab's collaborative model interfaces between academics and industry, and its goal is to build communities including public private partnerships around a myriad of technologies in one place and put them to use in the hands of inventors and thought leaders, accelerating the translation of laboratory research in therapeutics and diagnostics into the clinic.

He has initiated and participated with the FDA in a large-scale comprehensive study evaluating the clinical utility of gene expression technologies, which was published in Nature Biotechnology.

Dr. Kuo's clinical and translational initiatives have expanded globally in developing countries such as Brazil, China, Mongolia, Nigeria, Saudi Arabia and South Africa with the hopes to create a framework to accelerate the understanding of mechanisms of human disease and its heterogeneity of expression, to catalyze the identification and development of biomarkers that aid in this process, and to speed the development of therapeutics in patients. Dr. Kuo has also instructed in the Innovation for Economic Development program at the Harvard Kennedy School of Government.

He has been an active member of NIH/SBIR grant review study section for the past 7 years and a peer reviewer for the Qatar National Research Fund for the past 2 years. He sat and advised on the Public-Private-Partnership and Translational CTSA Key Function Committees at the NIH, and sits on numerous Scientific Advisory Boards including the Framingham Heart Study, ShanghaiBio, Exosomics, HansaBioMed and Genomic Expression. He is on the editorial board of several international journals and is Editor-in Chief of the Journal of Circulating Biomarkers and NanoBioMedicine (both he co-established). He is a founder and Trustee of the Otto Heinrich Warburg Cancer Research Foundation, established to foster, fund, and realize biomedical scientific research aimed at advancing knowledge in science and discovering effective and affordable cancer treatments with a focus on cancer metabolism.

Previously, he was Assistant Professor in the Department of Developmental Biology at Harvard School of Dental Medicine and Director and founder of the Harvard Clinical and Translational Science – Laboratory for Innovative Translational Technologies at Harvard Medical School. Prior to leaving Harvard Medical School to industry was the recipient of the prestigious Young Mentor Award.