Research is a critical component of better understanding VCP disease. It is our mission at Cure VCP Disease to partner with researchers and provide opportunities for connection and collaboration. We are excited to feature some of these incredible researchers and the work they are doing for VCP disease.
Peter Shen, PhD is an Associate Professor in the Department of Biochemistry at the University of Utah. Having connected with Cure VCP Disease five years ago, he has been profoundly impacted by meeting patients and witnessing first hand how his research translates into real-world impact. We hope you enjoy learning a little more about him, his lab, and his involvement with Cure VCP Disease.
Can you share a little about your lab at the University of Utah?
I started my lab in 2017 and the theme of my lab is to understand the processes underlying protein quality control in cells. The types of questions that my lab is interested in include how cells make and fold proteins, and how they remove proteins that are either no longer needed or might otherwise be toxic.
One of the major tools that my lab uses is known as cryogenic electron microscopy (or cryo-EM). It's a form of microscopy that we can use to look at individual proteins or their assemblies and determine their high-resolution structures, even at the atomic level. Many protein quality control, including those involving VCP, require multiple components to form these larger assemblies. Cryo-EM along with other complementary methods enable us to understand how they work.
How did you first connect with Cure VCP Disease and what do you enjoy most about working with them?
I first connected through Nathan Peck, CEO of Cure VCP Disease. He reached out to me via email and introduced himself. It was the first time that I had heard of the organization and it was in fact the first time that I had met someone who is affected by a VCP variant.
It was through that email introduction that Nathan informed me of the VCP scientific focus group; a community of scientists who have a unified theme of understanding all aspects of VCP from basic to clinical research. It was an eye-opening experience to be connected with this group. I've presented and given scientific presentations to the community. I've learned that it's a highly engaged community. What I enjoy most about working with them is how collegial the environment is.
Earlier this year we had an in-person meeting, the VCP International Scientific Conference where I got to meet many other patients as well as researchers within the same venue. It really brought life to the research questions that I'm interested in.
What has been the most significant thing you’ve learned about VCP?
The most significant thing I've learned about VCP, in work that my lab has been engaged in over the past several years, is how it works in unfolding its protein substrates. VCP's major job in the cell is to recognize and unfold proteins, usually as part of their degradation pathways. Over the years, we've determined several high-resolution structures of VCP that've captured VCP in the act of unfolding various substrates.
We are fascinated by the question of how VCP is regulated in the cell, and one of the active areas of research we have right now is how VCP interacts with dozens of different binding partners.
VCP plays many roles in the cell, and understanding what controls VCP's interactions with different substrates and at different parts of the cell is something that we aim to better understand through structural biology.
How do you stay motivated when researching a protein that involves a rare disease with many unknowns?
What motivates our research is the fact that there are so many unknowns. As a scientist, I think it would not be a very fulfilling career if we could predict the results of all our experiments.
We have interesting questions that we are uniquely positioned to answer. Our expertise and our access to the provisions and the tools to address these unanswered questions is what makes this work really exciting.
I think what also adds to my motivation is having met many people with VCP disease and having the optimism that the work that we're doing might have an impact in benefiting society.
What do you hope to see in the future for VCP disease and/or other rare diseases?
I am very hopeful about the future for developing treatments and possibly cures for VCP disease and other rare diseases. We live in a golden age of scientific discovery and technological advances. The rate at which we're making discoveries, of which we're developing new tools to enable new discoveries, is unprecedented and I think the tools are only going to get better.
You think about the great strides that have been made and the transformations that have been made in tools for genetic engineering, for drug discovery, and even in my own field of structural biology and biochemistry. We're really at the frontier of having the tools available for eradicating many diseases, and I'm certainly hopeful that that will be the future for VCP disease as well.
Disclaimer:
This interview has been edited for clarity and brevity. While the content reflects the views and responses of the interviewee, certain portions may have been condensed or rephrased for readability and flow.