Approximately 100 attendees gathered over three days to exchange multidisciplinary learnings across three roundtable discussions and 34 scientific presentations, covering core science to clinical research and more.
On February 22-25 at Caltech, Cure VCP Disease and Dr. Tsui-Fen Chou co-organized the first in-person VCP International Scientific Conference. In addition to discussion sessions, participants connected with presenters of 32 scientific posters and nine patient/care partner posters. Many attendees shared that the interaction between patients, clinicians, and researchers was UNPRECEDENTED.
During the scientific sessions, we dove into the basic science of VCP with innovative studies on the structure and function of VCP, and the therapeutic approaches of VCP across diseases, from single-molecule tracking VCP inhibitors to gene therapy and virology. Academia and industry were involved in active and fruitful Q&A and debates. The conference went on to cover the clinical aspects of VCP in neuromuscular diseases and dementia, as well as the discovery of a new population of VCP patients. The convening ended with a forum discussion on the future strategy for VCP disease, where we saw patients, clinicians, scientists, and industry setting the next steps in our field, and collaborative international projects emerge.
We had the honor of two keynote speakers sharing their perspectives on VCP. Dr. Margherita Milone, Professor of Neurology and Director of the Muscle Pathology Lab at Mayo Clinic College of Medicine, opened the conference with an outstanding lecture on all the clinical aspects of VCP disease. Dr. Raymond Deshaies, Senior Vice President of Global Research at Amgen, recapitulated 20 years of chemistry and discoveries of VCP in a remarkable presentation that pointed to a potential research hypothesis for future research. The conversation with Dr. Deshaies continued as he shared his deep knowledge throughout a Q&A session. Both keynote speakers also participated in the diverse roundtable discussions.
Roundtable discussions covered:
fundamental questions in outcome measures
clinical approaches comparing the pros and cons of gene therapies versus traditional small molecules in chronic and heterogeneous diseases such as VCP
roadblocks of genetic testing in adult-onset diseases
Dr. Peter Shen, Assistant Professor from the University of Utah, reflected ... "Meeting patients at the conference brought real world relevance to the basic research that is conducted in my lab. It was humbling to interact with patients, and it gave me a renewed sense of determination to accelerate our research efforts. And, the face-to-face interactions with other researchers provided a motivating environment to create new collaborations. It was great to hear about the latest research in the field and how very smart people can have a range of well-informed opinions on the priorities to pursue.”
Are you interested in joining the VCP Research Network?
We meet monthly and are launching several collaborative initiatives.
Congratulations to the poster winners:
Three young scientists were recognized for having outstanding posters in the respective categories: Structure and Function, Translational Research, and Clinical Research.
Each was recognized and gave the following oral presentations to conclude the conference:
Chad Altobelli of UCSF presented “VCP/p97 structure and activity are altered by multi-domain interactions with UBXD1”
Sukanya Banerjee of the University of Pittsburgh presented “PINK1 and VCP mediated neuroprotection against 2N4RWT-Tau pathology:
Shruthi Balasubramaniyan of the University of Pittsburgh presented “Characterizing a novel mutation in the Valosin Containing Protein D1 domain - VCPT262A”
Thank you to our conference sponsors for helping us make this event a success:
Additionally, we want to recognize two organizations, the National Organization of Rare Disease and Uplifting Athletes, for providing essential travel stipends to several patients and five young investigators.
We are looking forward to the next VCP International Conference!
Yours in Hope,
Armelle Pindon
Chief Scientific Officer
Cure VCP Disease
