Presentation Descriptions



Wednesday, June 12, 2019, 1:00 pm – 5:00 pm
 Safety Considerations Using Humanized Mice as Research Tool
Scott Kitchen, PhD, Director, UCLA CFAR/JCCC Humanized Mouse Core Laboratory; Timothy Mandrell, DVM, DACLAM, Consultant What is a humanized mouse and how is it created? Why are they useful, and what are some of the current research projects using them? Dr. Kitchen will address the science behind the creation of these mice and how mice bearing human cells are used in biomedical research. Dr Mandrell will then lead a discussion about occupational health and safety considerations for researchers, animal care staff, and others. The session will conclude with an activity in which audience teams will review information from IBC and IACUC protocols involving humanized mice. They will need to ask the researcher, Dr. Smart, questions about his protocols, and make recommendations on health and safety procedures.
 Activity: Best Practices
Thursday, June 12, 2019, 1:00 pm – 5:00 pm
 IBC Oversight of Transitional Research: Changes in Perspective When rDNA Moves From the Lab to the Clinic
Team from Duke University: Richard Frothingham, MD, IBC Chair, Wayne Thomann, DrPH, Director, Occupational and Environmental Safety, Pat Condreay, PhD, Safety Manager, Antony Schwartz PhD, Director of Biological Safety, Carol Epling, MD, Director, Occupational Health We will describe the development, by a Duke University investigator, of a recombinant chimeric poliovirus that showed promise as an oncolytic virus treatment for glioblastoma multiforme in cell culture and animal models; and eventually was proposed for human clinical trials. Thus, the Duke IBC has had a rare opportunity to oversee a wide spectrum of issues surrounding a single recombinant virus for more than 15 years.
Over the course of the session we will explore the following aspects:
• The evolution of the focus of oversight by the Duke University IBC from issues of laboratory biosafety to those of informed consent and biosafety in a clinic setting
• How Occupational Health expertise on the IBC has influenced approaches to evaluation of recombinant virus research and facilitated institutional preparedness for emerging issues related to novel therapies
• The challenges that novel therapeutic approaches such as PVS-RIPO present in the evolving regulatory environment for human gene transfer clinical trials
In each of these discussions we will provide opportunities for the audience to provide perspectives from their experiences with challenging registrations and contribute to sharing sound oversight practices.
The Future of HGT Research Oversight
Kathryn Harris, Ph.D., RBP This presentation will describe the changes taking place in the Human Gene Transfer Research Oversight.
 IBC Case Studies
A.  IBC Oversight of Human/CRISPR Trials
Ellyn Segal, PhD, Biosafety Manager, Stanford University The previous time span from bench to bedside for human gene transfer using viral vectors was 20 years; the time span from bench to bedside for CRISPR based human clinical trials was 5 years. This accelerated time scale necessitates an in-depth comprehension of a technology that has yet to be thoroughly understood and presents an IBC with numerous unknowns relating to review and oversight.
This session will provide a discussion of the state- of- the -art human gene transfer clinical trials currently being performed and discuss some of the scientific questions associated with these trials. Time will be available for Q/A and audience discussion. .
 B.  IBC Oversight of Prion-like Protiens
Susan Vleck, PhD, Stanford University IBC oversight of infectious agents includes prion protein work as a BMBL-designated BSL2 (or sometimes BSL3) agent. A growing body of data regarding proteins associated with neurodegenerative proteinopathies indicates that these proteins may act in a similar manner to prions. These so-called prion-like proteins misfold and aggregate, are capable of cell-to-cell and cross-species transmission, and may be similarly resistant to common methods of protein degradation. This session will discuss the expansion of IBC oversight beyond the “accepted” list of infectious proteins, touching on issues of risk assessment, decontamination and disposal, and biosafety review.